Five Upsides of PKD; CRISPR Model of PKD; Tiny Cell Bubbles; HALT-PKD Trial; PKD & Medical Marijuana

Living with PKD

From Huffington Post, by Bill Brazell

5 Upsides of a Chronic Disease

At my 25th college reunion, a classmate whom I hadn't met kindly asked me to talk about some of the positive things that had come out of inheriting a disease. This is what I said:

TRANSCRIPT

That's my grandmother Ann on her wedding day. Her husband would die in front of her when he was 44 years old. My poor Nana would later attend the funerals of both of her children, and her oldest grandchild. Three of those four sudden deaths had a single cause: polycystic kidney disease, or PKD -- a disease that was passed down to five or her six grandchildren, including me.

This is a normal kidney. It's the size of a fist.

That on the right is a polycystic kidney. "Polycystic" means "many pockets of fluid."

A healthy kidney weighs about five ounces. [Polycystic kidneys] can weigh 40 pounds each. Carriers are prone to high blood pressure and aneurysms. Along with some height and intelligence, I inherited those kidneys from my dad.

But I'm here to talk about the upside -- the good that has come of our family's history with these vile organs.

The first upside is that because he knew he might die young, my dad bought a lot of life insurance. I'm afraid that some of you may regret your laugh when I tell you what happened next. In 1984, my father drowned at the age of 42. He drowned. He certainly hadn't expected to die like that. But the insurance money put all four of his kids through college. And by the way, I don't hold it against anybody for laughing, believe me.

First Upside: Dad believed in life insurance. Which came in handy.

Two years after my father died, I needed a physical to maintain my spot as the worst rower on the freshman heavyweight crew. I asked the doctor at Holyoke Center to check for PKD. He found it.

Shaken, I walked back to my dorm. Alone, I looked out the window at the students, professors and tourists ambling through the Yard and I thought, "They can expect a full life. Mine may be half over."

Fourteen years passed, and I did very little about the disease.

Then my cousin Mike died. That's Mike and his dad, my Uncle Dick. Both would die of PKD, but Mike died first. He was just 35 when an aneurysm killed him, leaving two young kids behind.

Mike's death shocked me. We had grown up together, sneaking into movies, teasing each other about sports and politics. His death also brought my life into focus. I vowed to do all I could to help cure this disease before it took more of us.

Upside #2: A greater sense of purpose.

By telling Mike's story in email, I raised more than $10,000 for the following year's Walk for PKD. That brought me to the attention of a PKD Foundation volunteer who told me about a promising experimental drug.

As part of an ongoing trial, I've been taking that drug, tolvaptan, for 10 years. Its existence convinced me that it would be ethical for me to get married and have kids -- even though each of my kids would have a 50 percent chance of inheriting these kidneys.

Which leads me to upside #3: My wife, Victoria, who's up there with our little 5-month-old. She's sort of back in the shadows trying to keep the baby from being too loud. By our fourth date, I knew that I liked Victoria a lot, and so I knew I had to tell her about the disease. She took it extremely well. That was a lot for us to learn about each other on date #4. Usually fourth dates focus on... other things.

Upside #3: Learned something great, early, about future spouse.

Upside #4 is the deep generosity that I have received from just about everyone else.

When I email people about this, I'm asking somebody who has nothing to do with my disease -- and no chance of ever getting it -- to donate money to help cure it. What's in it for them? Here are four people, among many, who help anyway.

This is Steve Buchman. Each year he donates a large sum to support my Walk. The last time, I thanked him, and he said, "Look. For some reason, Bill, I get selfish pleasure from doing this. So you don't need to thank me. [Pause.] But you're welcome."

Angela Olivo is part-owner of Select Office Suites, which rents office space to small businesses in New York. It's been years since I was her tenant. Yet Angela and SOS generously support my Walk every year. She is a lovely woman. And I would never have known how lovely if I hadn't asked her for help.

That's my friend David Simons and me. When that photo was taken, David knew that he was dying of colon cancer. He died a few weeks after that photo was taken -- six years ago today, in fact. In his will he left $10,000 to the PKD Foundation in my name -- to cure a disease that he did not have. There are no words for how it felt when I learned that, that he had done that. And this past January, David's mom, Pat Bonardi, without my asking her, donated to my upcoming PKD Walk. Now this is a woman who has had the worst loss any parent can have -- but it was to cancer, not PKD. Yet she is supporting us, without ever being asked.

If I didn't have PKD, I would never have asked these people for support. And I probably would never have imagined that anyone could give as generously as these four people, and so many others, including some in this room, have done.

Upside #4: People have been so generous. How can I ever be cynical again?

Then the PKD helped me experience something very unexpected: I found out what it must feel like to be Brad Pitt.

Some friends made me a kidney costume. I first wore it to an evening benefit at a Manhattan museum where nobody knew me.

En route to the benefit, alone in a cab at night, I felt very anxious and insecure. I wasn't sure it was a good idea at all, and I expected some attendees to ridicule me. And then I thought about my young nieces, who may develop this disease as well. I knew that the costume would make them smile, and I told myself I was wearing it for them.

When I walked in, feeling odd, three lovely young women ran toward me, and said, "Oh, please, please -- will you please take a picture with us?"

A gorgeous woman said to me, "What are you?" I said, "I'm a kidney, darling, what are you?" She said, "I'm just a girrrrl."

And as I was leaving, another beautiful woman took off her heels, ran barefoot on cobblestones, crashed into my foam and said, "You have to come to our afterparty. I don't know you, but you're the only real thing here."

Upside #5: Got to feel like a sex symbol.

I skipped the afterparty. I went home and, like Cinderella, became normal again. I'm a happily married father of three lovely girls, and I never need to feel like Brad Pitt again.

I swear.

Thank you, Kim Harris Gardner, for inviting me to focus on the good things that have come out of this disease. And thank you all for listening. When I had just been diagnosed, and was looking out the dorm window, I may have seen some of you walking by. I didn't think then that I would be able to see you again, when I was 47 years old. But I'm seeing you now. Thank you for being here.

I welcome your help at support.pkdcure.org/goto/billbrazell. To learn more about PKD and join the fight, please visit The PKD Foundation. Thank you.

From Medical Marijuana Inc

Polycystic Kidney Disease – Medical Marijuana Research Overview

Polycystic kidney disease is an inherited kidney disorder that causes abnormal cysts to develop and grow. Studies have shown cannabis reduces kidney damage and helps manage side effects like high blood pressure and pain.
Overview of Polycystic Kidney Disease

Polycystic kidney disease (PKD) is an inherited disorder that causes clusters of cysts to grow in the kidneys. The cysts are filled with fluid and can enlarge the kidneys, replacing much of their normal structure. The abnormal shape leads to chronic kidney disease, which can then over time lead to reduced kidney function and even kidney failure. While most cysts develop in the kidneys, PKD cysts can also develop in the liver and elsewhere throughout the body.

Polycystic kidney disease is caused by genetic defects that are inherited. In rare cases, a mutated gene can be the cause of the disease. There are two types of polycystic kidney disease, each of which is caused by different genetic flaws. The most common type is autosomal dominant polycystic kidney disease (ADPKD), which typically develops between the ages of 30 and 40 and requires only one parent to have the abnormal gene. Autosomal recessive polycystic kidney disease (ARPKD) is a less common type of PKD that typically develops shortly after birth or during childhood. Both parents must have abnormal genes for this type of disease.

PKD commonly causes high blood pressure. Other symptoms caused by the disease include back or side pain, headaches, blood in urine, an increase in abdomen size, frequent urination, kidney stones, urinary tract or kidney infections and kidney failure. Brain aneurysms, heart valve abnormalities, colon problems and chronic pain can also develop from the disease.

There is no cure for polycystic kidney disease, but lifestyle changes and medical treatments can help reduce kidney damage and manage blood pressure. By controlling high blood pressure, the progression of the disease and subsequent kidney damage can be delayed. Medication is often used to manage chronic pain. Additional treatments are necessary if bladder or kidney infections, blood in the urine, kidney failure or aneurysms, develop.
Findings: Effects of Cannabis on Polycystic Kidney Disease

While clinical studies on cannabis’ effect on polycystic kidney disease are lacking, findings in preclinical studies suggest cannabinoids found in cannabis could offer therapeutic benefits for treating kidney disease. One animal study found that a major cannabinoid found in cannabis, cannabidiol (CBD) effectively reduced oxidative stress, inflammation and cell death in the kidney and improved renal function, causing the researchers to conclude CBD may be effective at preventing kidney disease (Pan, et al., 2009). These protective effects of CBD are likely due to its activation of the endocannabinoid system’s CB2 receptors, which another study found was effective at protecting the kidney from damage by minimizing inflammation and oxidative stress (Mukhopadhyay, et al., 2010).

Cannabis may also limit kidney damage caused by polycystic kidney disease by potentially lowering blood pressure. Both animal and human studies have found that cannabinoids cause blood vessels to vasodilate, which in turn improves blood flow and reduces blood pressure (Stanley & O’Sullivan, 2014a) (Stanley & O’Sullivan, 2014b) (Herradon, Martin & Lopez-Miranda, 2007) (Batkai, et al., 2004). CBD specifically has shown provide vasodilation, which researchers found allowed for greater blood flow and reduced damage (Stanley, Hind & O’Sullivan, 2013).

In addition, cannabis can help polycystic kidney disease patients manage their chronic pain levels. Cannabis has been shown to significantly lower both neuropathic and nociceptive pain; even managing chronic pain that had previously proven refractory to other treatments (Boychuck, Goddard, Mauro & Orellana, 2015) (Wallace, et al., 2015) (Lynch & Campbell, 2011). Because of cannabis’ effectiveness, surveys have found that use is prevalent among those experiencing chronic pain (Ware, et al., 2003). In one study, 12 of 15 chronic pain patients who smoke herbal cannabis reported an improvement in pain (Ware, Gamsa, Persson & Fitzcharles, 2002). Cannabis has also been shown to offer a therapeutic role for headaches, which are common in those with polycystic kidney disease (Baron, 2015).
States That Have Approved Medical Marijuana for Polycystic Kidney Disease

Currently, no states have approved medical marijuana specifically for the treatment of polycystic kidney disease. However, in Washington DC, any condition can be approved for medical marijuana as long as a DC-licensed physician recommends the treatment. In addition, a number of other states will consider allowing medical marijuana to be used for the treatment of polycystic kidney disease with the recommendation from a physician. These states include: California (any debilitating illness where the medical use of marijuana has been recommended by a physician), Connecticut (other medical conditions may be approved by the Department of Consumer Protection), Massachusetts (other conditions as determined in writing by a qualifying patient’s physician), Nevada (other conditions subject to approval), Oregon (other conditions subject to approval), Rhode Island (other conditions subject to approval), and Washington (any “terminal or debilitating condition”).

Also, 14 states have approved medical marijuana specifically to treat “chronic pain,” a symptom commonly associated with polycystic kidney disease. These states include: Alaska, Arizona, California, Colorado, Delaware, Hawaii, Illinois (Chronic Post-Operative Pain), Maine, Maryland, Michigan, Montana, New Mexico, Oregon, and Rhode Island. The states of Nevada, New Hampshire and Vermont allow medical marijuana to treat “severe pain.” The state of Washington has approved cannabis for the treatment of “intractable pain.”.

PKD Research

From The American Society for Cell Biology

A new gene editing technique turns human pluripotent stem cells into a model system for polycystic kidney disease

CRISPR/Cas9 is hot. News of the revolutionary gene editing technique that is already shaking up bioscience has finally reached the news media and the public. Now comes a first rate example of how CRISPR is changing the pace of biomedical research by linking up with another cutting edge technology—human pluripotent stem cells (hPSCs). Benjamin S. Freedman, now at the University of Washington, and his colleagues in Joseph Bonventre’s lab at Harvard Medical School, have used CRISPR/Cas9 to guide hPSCs into becoming a human cell-based lab model system for polycystic kidney disease (PKD). The most common inherited kidney disorder, affecting one in 500 Americans, PKD currently is not curable and, without long-term dialysis or kidney transplant, can be fatal. Freedman will speak about the PKD model at ASCB 2015 in San Diego on Monday, December 14, following its earlier publication in Nature Communications.

PKD’s hallmark is the formation of damaging, balloon-like cysts in kidney tubules. In the early 2000s, cell biologists linked cyst formation to gene mutations that affect the primary cilia, hair-like projections from cells that seem to act as sensory antennae. These fundamental discoveries were made in non-human organisms such as the algae,Chlamydomonas reinhardtii, and the zebrafish, Danio rerio. But the exact disease mechanism in humans is still not well understood in part because there hasn’t been a good human model of PKD in kidney cells.

CRISPR gave Freedman et al. a more precise tool to remodel the hPSC genome to include PKD mutations in the disease-linked genes, PKD1 and PKD2. The researchers then used a 3-D cell culture system to coax their mutant and healthy hPSCs down the differentiation pathway into becoming kidney progenitor cells and finally the proximal tubule cells found in kidney nephrons. In the mutant tubule cells, they observed the formation of large, translucent cyst-like structures but not in their healthy controls. These observations and others have convinced the researchers that their CRISPR/Cas9 and hPSC system produces a stable, biologically accurate human model for a common genetic disease where new understanding and new therapies are desperately needed.

From Rutgers University, By Robin Lally

Healthy or Sick? Tiny Cell Bubbles May Hold the Answer

Rutgers researchers provide insight into genes that can spread cancer or heal wounds


Rutgers scientists have uncovered biological pathways in the roundworm that provide insight into how tiny bubbles released by cells can have beneficial health effects, like promoting tissue repair, or play a diabolical role and carry disease signals for cancer or neurodegenerative diseases like Alzheimer’s.

In a new study, published in Current Biology, Rutgers scientists isolated and profiled cells releasing these sub-micron sized bubbles, known as extracellular vesicles (EVs), in adult C. elegans and identified 335 genes that provide significant information about the biology of EVs and their relationship to human diseases.

They and colleagues from Princeton University, the University of Oxford and Albert Einstein College of Medicine, determined that 10 percent of the 335 identified genes in the roundworm regulate the formation, release, and possible function of the EVs. Understanding how EVs are made, dispersed and communicate with other cells can shed light on the difference between EVs carrying sickness or health.

“These EV’s are exciting but scary because we don’t know what the mechanisms are that decide what is packaged inside them,” said Maureen Barr, lead author and a professor in the Department of Genetics in Rutgers’School of Arts and Sciences. "It’s like getting a letter in the mail and you don’t know whether it’s a letter saying that you won the lottery or a letter containing anthrax.”

For decades scientists believed that the EV material released by some human cells – which can only be seen through high-tech electron microscopes – was nothing more than biological debris.

While this theory has changed – with researchers now having a better understanding of the role these EVs play in cancer, infectious diseases and neurodegenerative disorders – scientists still aren’t certain how they are made or why the same parcels can result in different outcomes.

But Barr said by using C. elegans, which have many genes similar to humans, Rutgers scientists have identified new pathways that could control the production of EVs and the cargo they carry, including the proteins responsible for polycystic kidney disease, the most commonly inherited disease in humans. The polycystic kidney disease gene products are secreted in tiny EVs from both humans and worms and no one knows why these proteins are in the EVs, she said.

“The knowledge gained from this tiny worm is essential for determining the biological significance of EVs, for understanding their relationship to human diseases like polycystic kidney disease, and for harnessing their potential therapeutic uses, “Barr said.

Because EVs are found in bodily fluids like urine, blood and cerebral spinal fluid, Barr said it is nearly impossible to determine the cellular source from which they are derived. This is why very little is known about how EVs are made and how the molecular cargo is released.

By understanding how a cell makes and packages proteins, lipids and nucleic acids into EVs, Barr said, pharmaceutical treatments and therapies could be developed, for instance, that would prevent cancer cells from producing EVs carrying cargo necessary for tumor growth.

“When we know exactly how they work, scientists will be able to use EVs for our advantage,” said Barr. “This means that pathological EVs that cause disease could be blocked and therapeutic EVs that can help heal can be designed to carry beneficial cargo.”

From Renal and Urology News, by Jody A. Charnow, Editor

Severe Hyperkalemia Rare Despite Dual RAAS Blockade

Severe hyperkalemia rarely occurs as a result of dual blockade of the renin-angiotensin-aldosterone system (RAAS) or intensive blood pressure (BP) control, according to a study.

Ronald D. Perrone, MD, of Tufts Medical Center in Boston, and colleagues analyzed the frequency and severity of hyperkalemia in the HALT-PKD trial, a prospective, randomized study designed to determine the effects of dual RAAS blockade and BP reduction on progression of autosomal dominant polycystic kidney disease.

The trial had 2 study groups. Study A participants had an estimated glomerular filtration rate (eGFR) greater than 60 mL/min/1.73 m2. These patients were randomly assigned to receive lisinopril plus placebo or lisinopril plus telmisartan, with 2 levels of BP control: standard BP (SBP, 120–130/70–80 mm Hg or low BP (LBP, 95–110/65–75 mm Hg). Study B included patients with an eGFR of 25–60 mL/min/1.73 m2 randomized to lisinopril plus placebo or lisinopril plus telmisartan, with SBP control only.

Hyperkalemia rarely developed in study A participants. Mild hyperkalemia (potassium levels higher than 5.5 but not more than 6 mEq/L) occurred in 3%, 1%, 3%, and 2% of subjects in the lisinopril/telmisartan, lisinopril/placebo, SBP, and LBP arms, respectively, Dr. Perrone's group reported at Kidney Week 2015 in San Diego. Moderate hyperkalemia (potassium levels higher than 6 but not more than 6.5 mEq/L) developed in 2%, 0.4%, 1%, and 1%, respectively. Severe hyperkalemia (potassium levels above 6.5 mEq/L) developed in 0.4%, 0%, 0%, and 0.4%.

Mild hyperkalemia was more common among study B participants, but it was easily managed, according to the investigators. It occurred in 17% of the lisinopril/telmisartan arm and 16% of the lisinopril/placebo arm. Moderate hyperkalemia developed in 4% and 12%, respectively, and severe hyperkalemia developed in 0% and 0.4%, respectively.

“With careful management, dual RAAS blockade and intensive BP control were safe in the HALT PKD trial,” the researchers concluded in their study abstract.

Gift of Life

From Tap Into Morristown, Summit, NJ, By GREG ELLIOTT

Summit Man's Selfless Act Gives New Hope, Gift of Life to Girlfriend's Mother

Hilltop City resident Dana Henderson and Lauren Karsen have been a couple for just over a year now.

Henderson — a Summit High School Class of 2000 alum — is giving a unique anniversary present to Karsen, who is a records clerk with the Summit Police Department.

Henderson's gesture of love to his significant other?

Flowers? No. A heart-shaped box of chocolates? Uh-uh.

Henderson is donating a kidney to Lauren's mom, Karen, who has Polycystic Kidney Disease (PKD), a hereditary disease that, according to atlantichealth.org, is passed down through families and in which many cysts form in the kidneys, causing them to become enlarged.

Symptoms often do not appear until middle age, with statistics showing 1 in 1,000 Americans are affected with PKD, however the actual number may be more, because some people do not have symptoms.

Persons with PKD have many clusters of cysts in the kidneys. What exactly triggers the cysts to form is unknown. The disease is associated with the aortic aneurysms, brain aneurysms, cysts in the liver, pancreas, and testes, and diverticulaof the colon.  Currently, no preventative treatment can prevent the cysts from forming or enlarging, and the disease gets worse slowly. Eventually it leads to end-stage kidney failure, the treatments for which may include dialysis or a kidney transplant.

Henderson, a Muhlenberg College nursing student with two semesters remaining, works full-time as a patient care technician at Overlook Medical Center in Summit. He also volunteers one day each week at the Summit Volunteer First Aid Squad.  His passion for, and proximity to, the medical profession is allowing him to face what is undeniably an emotional and personal journey with a high degree of logic. "I am pretty confident, I have a lot of faith in modern medicine, and transplants in particular."

One of the body's miraculous capabilities is that the lone remaining kidney essentially takes on twice the functioning capacity, making up for the organ that was removed.  When Karen was diagnosed this past February she, like many who find out they have the disease, had no symptoms. At 61, she is in otherwise good health, which makes transplant an more viable option.

"It's all been crazy," said Henderson. "Once we found out, it was like 'who's gonna step up?" Tested for compatibility, Henderson was not the lone option, but assumed that role once another compatible donor was ruled out for health reasons.

Further, If one parent carries the gene, their children have a 50% chance of developing PKD. Once her mother was diagnosed, Lauren and her sister had to get tested. Lauren does not have PKD however, unfortunately, her sister tested positive. Lauren will likely eventually be her sister's donor.

The procedure involving Henderson and Karen will be performed at St. Barnabas Medical Center in Livingston on December 1, appropriately a day that has been designated at #GivingTuesday and calls for folks to help causes and those less fortunate post-Black Friday and after Cyber Monday.

Assuming all goes smoothly, Henderson will be in the hospital for two to three days, and then face another 4-6 weeks of monitored recovery before being able to assume his normal routine.

Henderson's impending, amazing act of selfless giving does not, however, overshadow the fact that his rehab time will place him in some real financial hardship, as his full-time work schedule allows him to pay for his schooling.  Prodded by others, the low-key Henderson finally relented and established a gofundme page, which is collecting donations to make up for his lost wages. In true form, Henderson placed a goal figure on the page of $1,500, which will not be enough to cover the wages he is foregoing. [Read more]

Year End Donations; 2016 National PKD Convention; Running Out of Time

PKD Foundation

From PKDCure.org

Thank you for joining our family of year-end donors!

We are humbled and grateful for your decision to invest in research, education, advocacy and patient support funded by the PKD Foundation!

Because of you, exceptional funding initiatives like the recently announced Jared J. Grantham Research Fellowship in partnership with the American Society of Nephrology are paving the way for brand new discoveries that will change and save lives for patients with polycystic kidney disease.

Don't miss the matching gift and tribute giving options below!

From PKDCure.org

We can't wait to see you in Orlando!

June 24 to 26, 2016, Orlando, Florida, Walt Disney World Resort

If you or a loved one is affected by PKD, join us at the PKD National Convention 2016 – a special opportunity to:
Learn the latest research about the disease
Connect with others in the PKD community
Become an advocate for your own health
Meet the doctors and researchers dedicated to fighting PKD

Join us to discover ways to manage your or a loved one's health from an all-star assembly of PKD experts.

Registration opens this January!

Living With PKD

From Mission record, Mission City, British Columbia, Canada, by Kevin Mills

Mission mom running out of time

Mission’s Jacquelyn Sumpton prepares to start her peritoneal dialysis treatment, which she needs every night. The 29-year-old mother of three has been diagnosed with polycystic kidney disease and needs to find a suitable kidney donor
— Image Credit: Kevin Mills Photos

Walking inside Jacquelyn Sumpton’s house, one’s attention is drawn to the dozens of stacked cardboard boxes marked Baxter. They contain medical supplies that keep Sumpton alive.

The 29-year-old Mission mother of three has been diagnosed with polycystic kidney disease and has to have peritoneal dialysis every night. Her prognosis is for eventual kidney failure, which is fatal. And time is slowly running out. Her only hope is to find a suitable person willing to donate a kidney so she can have a transplant.

As of today, the search is ongoing.

Her ordeal began about 18 months ago while pregnant with her third daughter. A routine blood test came back showing that she was anemic. Further tests revealed that Sumpton had kidney disease. Initially, she was told it was nothing serious to worry about. Doctors advised her to keep having blood tests to monitor the condition.

After Evangeline was born, three weeks early, life went back to normal.

Two months later, the doctor’s office called and told Sumpton she needed to go back for more bloodwork That test revealed that Sumpton’s GFR – which measures how much her kidneys actually filter – was down to nine per cent.

“They said, ‘You need to come in here today. We are putting a catheter in next week; we have set you up an appointment and you are going on dialysis the week after that.’

“Like it is happening now.”

She was horrified by the suddenness of it all.

“I think, a few times, I listened to a song and I just drove around and cried.”

But she is determined to stay upbeat and keep fighting. She doesn’t really have a choice.

“I come at it positive. Because if I don’t, I’ll fall into the deepest depths of depression and my family couldn’t take it and I couldn’t take it. And crawling out would be much harder than staying above it.

“Every day I have to consciously prepare myself and strengthen myself and ready myself and then address the day.”

Sumpton and her husband Sean Magnusson, also 29, have been together since they were in high school. They have three daughters – 10-year-old Genevieve, Aliya, 8, and Evengeline, who is one. Sumpton and Magnusson remain hopeful that a suitable kidney donor can be found.

“We knew from the start that polycystic kidney disease is not curable, so the only way to fix it is to put a new kidney in,” said Magnusson.

The search began immediately as they asked family and friends to get tested for compatibility. But slowly, those potential candidates were ruled out for a variety of reasons.

“The screening process is very thorough. The donor has to be a good match in order to have success.”

Magnusson is currently going through the testing process, hoping he might be a match.

“Our blood type matches and I’m healthy enough,” he said.

But now he has to go through more tests to determine if the tissue is able to co-mingle and not reject.

The waiting game continues.




From The Ellsworth American, Ellsworth, Maine, by Steve Fuller

Sorrento man takes search for kidney donor for stepson to the streets

Gerry Mehl of Sorrento stood at the intersection of State, Main and Water streets Friday morning carrying a sign looking for potential kidney donors. Mehl’s stepson, 51-year-old Mark Harrell of Milbridge, has polycystic kidney disease. ELLSWORTH AMERICAN PHOTO BY STEVE FULLER

While many spent Black Friday standing in line at a big-box store looking for an incredible deal on a flat-screen TV, Gerry Mehl spent his day standing on a street corner in downtown Ellsworth looking for a kidney donor for his stepson.

Mark Harrell, 51, lives in Milbridge and has polycystic kidney disease (PKD). It is an inherited genetic disorder that causes fluid-filled cysts to form in kidneys and sometimes other organs, and in many cases leads to kidney failure.

t also causes kidneys to grow in size, and Harrell said his are the size of footballs. The pain leaves him unable to sleep at times.

Harrell does in-home dialysis at his parents’ house in Sorrento four days a week, with each session lasting five to five and a half hours. While dialysis helps, it is neither easy nor the ideal solution.

“The life expectancy of someone on dialysis is half that of someone with a transplanted kidney,” Harrell wrote in a letter to the editor earlier this year.

For months, he and his parents have driven around with decals on their vehicles that read, “KIDNEY DONOR NEEDED 207-422-1119.” The number is Mehl’s, as Harrell does not have reliable phone service where he lives.

As Black Friday drew closer, Mehl decided it would be a good opportunity to share that same message with the large number of people out shopping that day.

He began at around 8:30 a.m. by standing at the corner of Main and High streets, then moved to Walmart for a bit before heading down to the busy corner of State, Main and Water streets.

The sign made of bright yellow posterboard that Mehl held up as cars passed by carried the same message as the decals on his family’s vehicles. After being out on the streets for a few hours, he said he had received a few friendly honks and “quite a few waves.”

Mehl said he does not want to watch his stepson suffer anymore — seeing Harrell get hooked up to the dialysis machine and watching the two needles go in, day after day.

“Until you see it — until you see him on the machine…” Mehl said, emotion evident in his voice. “It’s not pleasant.”

Harrell is on a transplant list, but he is one of more than 1,000 people in Maine waiting for a kidney transplant and one of more than 60,000 around the country. The average wait time for a kidney from someone who has died is three to five years, according to the KidneyLink program.

That is why Harrell and his family are looking for a living donor, and that is why they are being proactive in their search.

“Sitting back and just waiting is not an option for me,” said Harrell, who called his parents his heroes for their assistance. While Mehl was out with the sign on Friday morning, his wife, Sonny, was at home helping her son with his dialysis.

Information about the living donation process can be found by visiting www.mmc.org/living-donation.

Anyone who is interested in becoming a living donor, or knows someone who might be, can contact Mehl by calling 422-1119 or calling the transplant center at Maine Medical Center in Portland directly at (800) 870-5230 and selecting option number four.

Artifical Kidney; Run for PKD; TKV: Prognostic Biomarker; Mark Your Calendar

Artificial Kidney

From Tech Times, by Katherine Derla

Implantable Artificial Kidney Offers Economical Alternative To Dialysis And Organ Transplant: How It Works

In the United States, approximately 26 million people are affected by kidney disease with an estimated 5 percent annual increase in end-stage renal disease (ESRD) or kidney failure. Patients with ESRD rely on routine dialysis treatment which can extend their lives but leaves patients with very poor quality of life.

The Kidney Project, a collaboration between the Vanderbilt University and the University of California aims to revolutionize ESRD treatment. Led by Dr. William H. Fissell and Dr. Shuvo Roy, a team of researchers developed a new implantable bioartificial kidney that improves the health and financial implications of ESRD, particularly among patients who rely on dialysis treatments that can accumulate an annual cost of up to $89,000.

The implantable artificial kidney, which is about the size of an average coffee cup, imitates the capabilities of a normal, healthy kidney. To address the increasing deaths among waitlisted transplant patients, The Kidney Project's bioartificial kidney offers patients with less expensive and non-invasive continuous ESRD treatments that enable them to be more mobile.

"We aim to conduct clinical trials on an implantable, engineered organ in this decade, and we are coordinating our efforts with both the National Institutes of Health (NIH) and the U.S. Food and Drug Administration," said Roy.

The device will be implanted beside the patient's own kidneys, which will not be removed, and will be connected to the patient's bladder and blood supply. The team employed methods involved in the production of microelectromechanical systems and semiconductor electronics to create the bioartificial kidney, which works using blood pressure and without the aid of electronic power of pump. The bioartificial kidney has a silicon nanofilter that mimics a normal kidney by removing water, small molecules, toxins and salt from the blood.

The Kidney Project is currently in its second stage of development. Phase Two involves extensive refinement of the bioartificial kidney, device test, development of pre-clinical testing prototypes and human clinical tests which involves ESRD patients. Set to conclude in 2017, the project's timeline depends on funding. While the team works with funds from federal grants, The Kidney Project is still in need of $8 million to complete Phase Two.

The Kidney Project has already reached out to donors and investors. It has also created online crowdsourcing campaigns to help raise the needed budget, which has been supported by the ESRD patient groups.



From Mass Device, by FINK DENSFORD

FDA grants expedited access for UW’s wearable artificial kidney

The FDA granted expedited access pathway status for a Wearable Artificial Kidney being developed at the University of Washington’s Medical Center in Seattle, the school reported earlier this month.

The Wearable Artificial Kidney is a battery-powered hemodialysis machine composed of miniaturized components worn as a tool belt and connected to the patient via catheter, UW Medical said.

The approval came based off the successful results of a 7-patient clinical trial of the device carried out at the UW Medical Center between October 2014 and April 2015, despite technical issues that halted the trial early on, the group said.

“Treatment with the WAK was safe, well-tolerated and effectively cleared patients’ uremic toxins while maintaining electrolyte and fluid balance. These results serve as proof-of-concept that, after the device is redesigned to overcome technical problems, a wearable artificial kidney can be developed as a viable dialysis technology,” Kidney Research Institute director Dr. Jonathan Himmelfarb said in prepared remarks.

Patients wore the device for a 24-hour period, Himmelfarb said. Investigators in the trial said patients equipped with the device “expressed excitement to walk around hospital hallways” while the device was functioning, and that the device functioned “admirably.”

“When patients perform hemodialysis at home, only a few options exist and they are large, heavy and stationary. The WAK represents the first truly portable hemodialysis machine, which could enhance patients’ quality of life and enable them to work and travel as they would otherwise,” study investigator Dr. Matthew Rivara said in a press release.

Because the WAK continuously filters blood, subjects were also temporarily freed from food restrictions associated with salt, potassium and phosphorous, which accumulate in patients undergoing chronic dialysis.

“I was amazed at how well it worked for me. It was heavy and cumbersome, but I’d be wearing it today if I could. It just gives you so much more freedom,” test subject Chuck Lee, who has had diabetes for 40 years and received thrice-weekly dialysis for over 2 years, said in a prepared statement.

Despite eating foods that would normally be restricted, the device reported metabolic and other fluid counts in a healthy range, according to Lee’s wife.

Expedited access pathways grants the group more immediate review from the FDA as they submit material to the agency.

“If we can complete the design expeditiously, they’ll look at it very fast, and even during redesign, they’ll give guidance so we’re not making decisions that compromise our chances to get to the next stage. The WAK showed it could dialyze patients for the full 24 hours, with pretty good results, and that’s a big proof of concept. We have a lot of work to do, but this technology has potential to change quality of life in a big way,” trial co-lead Dr. Larry Kessler said in a press release.



PKD Fundraising

From Express & Star, United Kingdom

WATCH: Burntwood marathon runner hopes donations will 'tumble' in

A marathon runner from Burntwood has found a unique way to up the ante - by strapping a four stone tumble dryer to his back.

That is the length Phil 'the tumble dryer' Box goes to to raise money for his nephew and step-brother who suffer from life debilitating condition Polycystic Kidney Disease (PKD).

The 29-year-old from Oakdene Road said he started running a few years ago simply to 'clear his head' but having realised he had a natural talent for it he soon ran out of ways to make it more challenging.

He said: "I started running if I had an argument at home or something and it just progressed from there.

"I joined a running club, then ran a marathon, then started doing the ultra marathons which are 100 miles, then I thought what 'am I going to do now?'

"Eventually it just came to me 'I'll try it with a tumble dryer on my back', I was in the kitchen and it was just staring me in the face."

Remarkably, the carpenter and joiner was told to 'calm down' by doctors in December after he was admitted to hospital with chest pains and a suspected heart attack.

But undeterred, Mr Box was out a week later scampering around Chasewater.

And when he came up with the idea to stick a tumble dryer to his back - partly inspired by another runner called Tony the Fridge - he did not exactly ease himself in.

Instead he took on three marathons in three days managing to carry it for around half of the distance.

"It's quite awkward. From the start you are in quite a bit of pain which you carry with you all the way until the end," he said.

But it is worth it to raise money for the PKD charity which he has already generated more than £1,000 for through various running events.

His step brother Craig |Turner and nephew four-year-old Charlie Turner both have the disease which causes a range of health problems and can eventually lead to kidney failure.

Mr Box said: "PKD is treatable and they can pre-long it but at the moment there isn't a cure.

"That's why I am doing this. If people like me don't raise awareness for PKD then there will never be a cure."

Mr Box's next running challenge is on Sunday, when he will be running on a treadmill - without the tumble dryer this time - at Burntwood Leisure Centre for the venue's entire opening time, some 14 hours 30 minutes.

To sponsor Mr Box text 'Tumb85' followed by the donation amount to 70070.

From Michigan City - La Porte Indiana... Mark Your Calendar

Cruis'n For a Cure for PKD

Sunday, May 15, 2016
12:00PM - 2:30PM

Cruis'n For a Cure for PKD Car show and Car cruise.

Car Show and 40-mile car cruise, $10 per vehicle, all wheels welcome. Rain or Shine. Concessions, trophies, prizes, LaPorte County Fairgrounds

Tracking PKD Progression

From Business Wire

C-Path’s Polycystic Kidney Disease Outcomes Consortium Secures EMA Qualification Opinion for Enrichment Biomarker in ADPKD


The Critical Path Institute (C-Path) announced today that the European Medicines Agency (EMA) rendered a positive qualification opinion to C-Path’s Polycystic Kidney Disease Outcomes Consortium (PKDOC) for total kidney volume (TKV) as a prognostic biomarker to select patients for clinical trials of new therapies for Autosomal Dominant Polycystic Kidney Disease (ADPKD).

ADPKD affects approximately 12 million people worldwide. There is only one medication developed to treat ADPKD, called tolvaptan, which has not yet been approved in the U.S.

TKV is a measurement of the impact of ADPKD on kidney size and is considered to be predictive of decline in kidney function. The EMA opinion states, “CHMP supports baseline total kidney volume, in combination with patient age and eGFR as a prognostic biomarker to identify patients likely to experience a progressive decline in renal function, as characterized by a decline in eGFR or progression to end-stage renal disease.

“From the data provided it is reasonable to expect that baseline TKV can predict disease progression and is a biomarker valuable for risk stratification."

”This qualification, along with a similar one from FDA, confirms the relationship between TKV and ADPKD disease progression, and will help in the design of clinical trials for new therapies for ADPKD” says C-Path COO and PKDOC Co-Director Steve Broadbent.

The PKDOC created a Clinical Data Interchange Standards Consortium (CDISC) data standard for ADPKD and used it to remap data from patient registries and observational studies. The database was used to develop a joint model linking baseline TKV with clinical outcomes.

About the organizations:

C-Path, a nonprofit, was established in 2005 to deliver on FDA’s Critical Path Initiative. C-Path has established 12 global, public-private partnerships that include regulatory agencies, academia, patient advocacy organizations, and pharmaceutical companies. C-Path has headquarters in Tucson, AZ, and has been funded by public and private support. For information, visit www.c-path.org.

The PKD Foundation is the only US organization solely dedicated to finding treatments and a cure for PKD, through promoting programs of research, education, advocacy, support, and awareness on a national level, along with services in local communities. Visit pkdcure.org to learn more.

Wanted: A Hero; Tinder to the Rescue; Wearable Artificial Kidney Passes 3rd Proof of Concept; Nano-Tech

Living with PKD

From County Record, Blountstown, Florida, by Kelli Peacock Dunn

WANTED: A Hero

Tonya Stewart Lawrence and her family are praying for a kidney donor. Could you be the match she so desperately needs?

A Blountstown native who works as a nurse in our community now finds herself on the receiving end of medical care. Just recently, a chronic illness she has been managing for a decade took a turn for the worse and she is now on a waiting list to receive a kidney.

A 1996 graduate of Blountstown High School, Tonya Stewart Lawrence, 38, and her husband, Brett, reside in Two Egg with their four children. By day, Tonya works as a nurse at TMH Blountstown Family Practice.

“Back in 2005, I was diagnosed with polycystic kidney disease,” says Tonya. “Basically, cysts overtake your kidneys. I went on several different medications and every once in awhile I’d have a kidney stone.”

The condition was completely manageable until May when Tonya was preparing for a sinus procedure and was told she couldn’t have the surgery. “My creatinine levels were too high which is related to kidney function,” she explains. “They were hesistant to put me to sleep.”

A short time later, Tonya was referred to the Mayo Clinic where she received some dreaded news: She is facing dialysis and a transplant. Just two weeks ago, the plan of action went a step further when she was placed on a transplant list.

Finding a match isn’t always a quick process. “Average wait times are two to four years,” says Tonya. To receive a kidney from a donor at time of death, the wait is up to five years.

Tonya’s brother was the first to step up and see if he could be the match she so desperatedly needs. At first, it seemed he would be the ideal donor. “He was the perfect match,” she remarks. “But then they did a urinalysis on him and he had too much sodium. He is always going to produce kidney stones so they didn’t want to take that kidney.”

So now the search goes on for anyone who could possibly be a match and be willing to give one of their kidneys so this wife and mother can live a normal life.

How can you be the hero Tonya needs?

-Donors must have Type O blood as Tonya is O+ (Blood type O is the universal donor and can give to all blood types)

-Be between the ages of 18 and 70

-No diabetes

-No hypertension

-No active infections

-No active cancer

-Have a healthy BMI (body mass index) less than 31% for donors under age 30, 35% for ages 31-45 and 40 percent for ages 46 and older

If you’d like to be tested to see if you could be a match for Tonya, call Lisa Leath, Living Donor Coordinator, at 904-956-3249. You can also message Tonya on Facebook for more information.

Please watch for details on fundraisers for Tonya as the surgery and recovery process will be costly for her. Even with insurance, her monthly medication costs alone will be $1500 out of pocket.

“This is such a humbling experience,” says Tonya. “I’m just an independent person. It’s very difficult to ask for help and reach out to your community.

There are a lot of emotions are involved.”

From The Tribune, Greeley, Colorado

Greeley sports bar Double Play holds event to raise awareness, funds for hereditary kidney disease

The smoky smell of barbecue, bright orange jerseys and tinny sound of sports broadcasters made for a proper tailgate, but Broncos fans didn’t pack the Double Play parking lot for just a football game.

The locally owned sports bar, which sits in the Greeley Mall parking lot on 23rd Avenue, hosted a fundraising event Sunday afternoon.

Their goal: raise awareness about polycystic kidney disease and maybe find a kidney donor.

PKD is a hereditary disease that can cause painful swelling and eventually lead to kidney failure, according to the PKD Foundation. Funds from Sunday’s event went to the nonprofit.

Parents have a 50 percent chance of passing it along to their children.

Double Play co-owner Jeff Woltemath is invested in the cause. His wife, Autumn, has PKD. He said not many people are aware the disease exists.

“You hear about cancer, you hear about heart disease,” he said.

PKD doesn’t get the same attention.

He’s been working with his wife and a few other people to get the word out about the disease, using Facebook and events like Sunday’s.

They’re also looking for a donor. Autumn’s father, Bob Ellis, has PKD, too. His has progressed far enough to land on the transplant list.

A big blue poster with a photo of Ellis in a cowboy hat hung in the massive tailgating tent, encouraging fans to consider donating.

Ellis was wearing the same cowboy hat Sunday, sitting near the TV inside the tent. He said he didn’t love the attention, but he was grateful his daughter was so passionate about his health and helping him.

Ellis’ wife, Lesa, also was at the tailgate. She said Autumn was Bob’s kidney donor champion.

Most people don’t realize they only need one kidney and can donate the other, Lesa said.

“If you’re on the kidney transplant list, you’re waiting for someone to die,” she said. “It really doesn’t have to be that way.”

From Custom Ink

PKD Walk Nashville, TN

"PKD Walk Nashville, TN Oct 31st, 2015 @ Shelby Park.We raised money for research and treatment of PKD, a deadly genetic disease that affects millions. There is currently no meds or treatment available. CustomInk provided great service and a great product!!! We had many complements on our T-shirts! Thank you!! " - http://nashvillewalk@pkdcure.org - Rebecca Bush (Becky) (Nov 11, 2015)

Gift of Life

From Tampa Bay Times, Tampa Bay, Florida, by Chelsea, Tatham

Kidney donor enters couple's lives thanks to swiping right on Tinder

Almost four months ago, Rich O'Dea took Jennifer Thomas out on a blind date.

Both were trying to get over recent breakups. O'Dea told Thomas about his passion for running marathons. He told her about his close friend, Scott Bragan, who leads a team of marathoners who raise awareness and funds for the Polycystic Kidney Foundation.

His wife, Erika Bragan, suffers from polycystic kidney disease, a genetic disorder that can cause kidney failure. She was facing a lifetime of dialysis or a kidney transplant.

Thomas wanted to help. She has O-positive blood.

Erika Bragan also has O-positive blood.

Thomas got tested. After weeks of tests, she got the news: She was a match.

Now, Erika Bragan is set to get a new kidney from someone she had never met before.

And all because two people swiped right on Tinder.

Erika Bragan was diagnosed with the disease, known as PKD, 14 years ago while she was pregnant with her daughter.

Parents have a 50 percent chance of passing the disease along to their children. The Bragans have two: Madison, 14, and Spencer, 11. The parents said their children have not yet been tested for the disease because of insurance reasons.

About 600,000 Americans suffer from PKD, the fourth leading cause of kidney failure. More than 50 percent will have failing kidneys by age 50, according to the PKD Foundation.

Erika Bragan, 44, has spent two years on the transplant list. Scott Bragan, 47, was once a match for his wife. But then his blood pressure medication eliminated him as a donor.

So, he turned to marathons. Since 2009, he said his Team Tampa PKD has run eight marathons and raised about $115,000 for the PKD Foundation.

"As Erika got more sick," he said, "my way of dealing with it was to run and raise money and awareness."

Through the team's efforts, four potential donors came forward to try to help Erika Bragan. They went through the testing process, but none were matches. [Read more]

PKD Research

From Medscape

Wearable Artificial Kidney Potential Alternative to Dialysis

A wearable artificial kidney has passed a third proof-of-concept trial: patients receiving hemodialysis who wore the device remained hemodynamically stable for 24 hours, and fluid removal was consistent with prescribed ultrafiltration.

"We do not do a good job at answering the unmet needs of our dialysis patients," said Victor Gura, MD, a nephrologist at the Cedars Sinai Hospital, Los Angeles, who is also from the David Geffen School of Medicine at the University of California, Los Angeles.

"The draconian impositions we make on these patients in terms of not only being tethered to a machine ('doing time' as patients say) but also the limitations of diet — two glasses of orange juice and a bag of potato chips is enough to kill these patients" — don't make for a very good life, he said.

Dr Gura, who invented the wearable device, spoke at a press briefing here at Kidney Week 2015: American Society of Nephrology Annual Meeting.

Dialysis on a Belt

The wearable artificial kidney is a battery-operated belt-like device (think construction workers' belts from Home Depot) that miniaturizes the functions of a 300-pound dialysis machine in just 11 pounds, which is light enough to allow patients the freedom to move.

The device also uses just a half a liter of water compared with the 40 gallons of water required during a normal session of hemodialysis.

In an earlier proof-of-concept study involving eight patients requiring hemodialysis published in theLancet, Dr Gura and colleagues report that their wearable hemodialysis device showed promising safety and efficacy results, although further studies would be necessary to confirm their findings (Lancet. 2007;370:2005-2010).

In the current trial, Dr Gura and colleagues tested the wearable artificial kidney on seven more patients receiving hemodialysis at the University of Washington in Seattle. The researchers aimed to determine the safety and efficacy of the device in achieving solute electrolyte and volume homeostasis during a 24- hour period.

"The difference between this trial and the one we did previously was that the regulators allowed patients to wear the [device] for 24 hours for the first time," Dr Gura explained.

Of the seven patients who tested the device, five patients completed the planned 24 hours of study treatment, whereas the remaining two patients completed 4 and 10 hours of treatment, respectively.

Six of the seven patients were mobile while undergoing dialysis with the wearable kidney.

Laboratory and hemodynamic parameters remained stable during the 24-hour test run.

From Nano Technology Now

Nanotechnology advances could pave way for implantable artificial kidney

Research that could lead to the development of a surgically implantable, artificial kidney, was presented at ASN Kidney Week 2015 November 3-8 at the San Diego Convention Center in San Diego, CA. Advances in nanofilter technology were produced in collaboration between investigators from UCSF and Vanderbilt University. The research recently received a new $6 million grant through the National Institute of Biomedical Imaging and Bioengineering's Quantum Program.

A surgically implantable, artificial kidney could be a promising alternative to kidney transplantation or dialysis for people with end stage renal disease (ESRD). Currently, more than 20 million Americans have kidney diseases, and more than 600,000 patients are receiving treatment for ESRD. U.S. government statistics indicate kidney care costs the U.S. health care system $40 billion annually, accounting for more than 6 percent of Medicare spending.

"We aim to conduct clinical trials on an implantable, engineered organ in this decade, and we are coordinating our efforts with both the NIH and the U.S. Food and Drug Administration," said Shuvo Roy, PhD, a UC San Francisco bioengineer who led the research together with Vanderbilt University nephrologist William Fissell, MD.

Roy is the technical director of The Kidney Project at UCSF, a multi-institutional collaboration that has prototyped and begun testing key components of the coffee-cup-sized device, which mimics functions of the human kidney.

One component of the new artificial kidney is a silicon nanofilter to remove toxins, salts, some small molecules, and water from the blood. Roy's research team designed it based on manufacturing methods used in the production of semiconductor electronics and microelectromechanical systems. The new silicon nanofilters offer several advantages -- including more uniform pore size -- over filters now used in dialysis machines, according to Roy. The silicon nanofilter is designed to function on blood pressure alone and without a pump or electrical power.

Fissell, associate professor in the Department of Medicine at Vanderbilt and medical director for The Kidney Project, said the project's goal is to create a permanent solution to the scarcity problem in organ transplantation. "We are increasing the options for people with chronic kidney disease who would otherwise be forced onto dialysis," Fissell added.

The artificial kidney being developed by Roy and Fissell is designed to be connected internally to the patient's blood supply and bladder and implanted near the patient's own kidneys, which are not removed. Along with Roy at UCSF and Fissell at Vanderbilt, a national team of scientists and engineers at universities and small businesses are working toward making the implantable artificial kidney available to patients.

In September the project was designated for inclusion in the FDA's new Expedited Access Pathway program to speed development, evaluation, and review of medical devices that meet major unmet needs in fighting life-threatening or irreversibly debilitating diseases.

Artifical Kidney Development, Living with PKD, ASN Kidney Week 2015, Wife Donor to Husband

PKD Research

From Medical Xpress

Development of a surgically implantable, artificial kidney

Development of a surgically implantable, artificial kidney—a promising alternative to kidney transplantation or dialysis for people with end-stage kidney disease—has received a $6 million boost, thanks to a new grant from the National Institute of Biomedical Imaging and Bioengineering (NIBIB), one of the National Institutes of Health, to researchers led by UC San Francisco bioengineer Shuvo Roy, PhD, and Vanderbilt University nephrologist William Fissell, MD.

"We aim to conduct clinical trials on an implantable, engineered organ in this decade, and we are coordinating our efforts with both the NIH and the U.S. Food and Drug Administration," Roy said.

Roy is a professor in the Department of Bioengineering and Therapeutic Sciences in the Schools of Pharmacy and Medicine, and technical director of The Kidney Project at UCSF, a multi-institutional collaboration. The Kidney Project team has prototyped and begun testing key components of the coffee-cup-sized device, which mimics functions of the human kidney.

Roy and Fissell will present updates on development of the device November 3-8 at Kidney Week 2015 in San Diego, part of a major meeting of the American Society of Nephrology.

NIBIB is overseeing and funding the continuation of their work for four years under a cooperative agreement through its Quantum Program, created to support the development of "biomedical technologies that will result in a profound paradigm shift in prevention, detection, diagnosis, and/or treatment of a major disease or national public health problem." This is the second major grant the researchers have received through the program.

In part because the U.S. population has grown older and heavier and is more likely to develop high blood pressure and diabetes, conditions often associated with kidney failure, the number of individuals diagnosed with kidney failure is growing year-over-year and has risen 57 percent since 2000, according to the National Kidney Foundation. More than 615,000 people now are being treated for kidney failure. U.S. government statistics indicate that kidney failure costs the U.S. healthcare system $40 billion annually and accounts for more than six percent of Medicare spending.

The waiting list for kidney transplants in the United States has grown to more than 100,000 people. The number of available kidneys has remained stagnant for the past decade, and only about one in five now on the list is expected to receive a transplant.

More than 430,000 of those with kidney failure now undergo dialysis, which is more costly and less effective than transplantation and typically requires hours-long stays at a clinic, three times weekly. Only about one in three patients who begins dialysis survives longer than five years, in comparison to more than four in five transplant recipients.

Fissell, associate professor in the Department of Medicine at Vanderbilt and medical director for The Kidney Project, said, "This project is about creating a permanent solution to the scarcity problem in organ transplantation. We are increasing the options for people with chronic kidney disease who would otherwise be forced onto dialysis."

Along with Roy at UCSF and Fissell at Vanderbilt, a national team of scientists and engineers at universities and small businesses are working toward making the implantable artificial kidney available to patients.

According to B. Joseph Guglielmo, PharmD, dean of the UCSF School of Pharmacy, "The grant from NIBIB is a striking affirmation of the promise associated with this device, as well as NIH confidence in the ultimate success of The Kidney Project. Patients with chronic kidney failure are in real need of alternatives to transplant and dialysis; this School of Pharmacy and campus priority clearly demonstrates the research rewards of working collaboratively."

In September the project was designated for inclusion in the FDA's new Expedited Access Pathway program to speed development, evaluation, and review of medical devices that meet major unmet needs in fighting life-threatening or irreversibly debilitating diseases. The program evolved from an earlier FDA program called Innovation Pathway 2.0, in which The Kidney Project team was one of three device-development groups selected for a pilot initiative focused on kidney failure. Members of the FDA regulatory staff have continually been in communication with Roy and other project leaders to help guide device testing and criteria for data collection.

The aim of the new program is to speed the FDA's premarket approval (PMA) process for scientific and regulatory review of safety and effectiveness of Class III medical devices—those with the potential to provide major benefits, but that also might potentially pose major risks.

"The new program brings FDA reviewers, scientists, and leadership together with our team to define a roadmap to regulatory approval and product launch," Roy said.

Early Studies of Artificial Kidney Prototype Are Encouraging

One component of the new artificial kidney is a silicon nanofilter to remove toxins, salts, some small molecules, and water from the blood. Roy's research team designed it based on manufacturing methods used in the production of semiconductor electronics and microelectromechanical systems (MEMS). The new silicon nanofilters offer several advantages—including more uniform pore size—over filters now used in dialysis machines, according to Roy. The silicon nanofilter is designed to function on blood pressure alone and without a pump or electrical power.

The second major component is a "bioreactor" that contains human kidney tubule cells embedded within microscopic scaffolding. These cells perform metabolic functions and reabsorb water from the filtrate to control blood volume. A project collaborator, H. David Humes, MD, professor in the Department of Internal Medicine at the University of Michigan, earlier showed that such a bioreactor, used in combination with ultrafiltration in an external device, greatly increased survival in comparison to dialysis alone in the treatment of patients with acute kidney failure in a hospital intensive care unit.

The artificial kidney being developed by Roy and Fissell is designed to be connected internally to the patient's blood supply and bladder and implanted near the patient's own kidneys, which are not removed.

Unlike human kidney transplant recipients, patients with the implantable artificial kidney will not require immunosuppressive therapy, according to Roy. Preliminary preclinical studies indicate that the non-reactive coatings developed for device components are unlikely to lead to filter clogging or immune reactions, he said, and that bioreactor cells can survive for at least 60 days under simulated physiological conditions.

About one-half of the new funding from NIBIB will support lab studies on methods for optimizing performance of the bioreactor's kidney cells. The remainder will enable refinements for the mechanical design of the nanofilter unit and biocompatibility of the artificial kidney. The filter will be evaluated in preclinical studies aimed at achieving clot-free operation and stable filtration for 30 days.

Private philanthropy and UCSF support already have been vital in sustaining The Kidney Project, and even with the FDA's new and more flexible pathway, additional funding will be required to meet project timelines, Roy said.



From Business Wire

Angion Biomedica to Present Clinical and Preclinical Data at the Upcoming American Society of Nephrology Conference in San Diego

UNIONDALE, N.Y.--(BUSINESS WIRE)--Angion Biomedica Corp. announced today that it will present Phase 2 data from one clinical program and three preclinical programs at the American Society of Nephrology Kidney Week, November 3-8, 2015 in San Diego, CA

... In addition, Angion will give presentations on two other compounds in its pipeline. Data on Angion’s novel anti-fibrotic compound ANG3070 in polycystic kidney disease will be presented in “Therapeutic Effects of the Fibrokinase Inhibitor ANG3070 in Polycystic Kidney Disease.” Angion’s novel compound ANG3586 for chronic kidney disease is the topic of “Identification of a New Aldosterone Synthase Inhibitor with Anti-Fibrotic Activity in Animal Models.” These compounds are Angion’s proprietary small molecules on track for IND submissions in 2016. [Read more]

From Market Wired

Kadmon Corporation to Present Clinical Data on Tesevatinib for the Treatment of Polycystic Kidney Disease at ASN's Kidney Week 2015

Kadmon Corporation, LLC today announced data from an ongoing Phase 1b/2a clinical trial demonstrating the tolerability of tesevatinib, the Company's investigational oral tyrosine kinase inhibitor, in patients with autosomal dominant polycystic kidney disease (ADPKD). The data will be presented in a poster session (SA-PO865) at the American Society of Nephrology (ASN) Kidney Week 2015, being held November 3-8, 2015 in San Diego, CA.

ADPKD is an inherited disorder characterized by the formation of fluid-filled renal cysts, leading to loss of kidney function and end-stage renal disease. Tesevatinib inhibits the molecular pathways central to the progression of ADPKD, namely EGFR and Src family kinases. In addition, tesevatinib accumulates in the kidneys, 15-fold greater than in blood, making it an excellent potential therapeutic product candidate for PKD.

The Phase 1b portion of the study demonstrated that tesevatinib was generally well tolerated at 50, 100 and 150 mg QD, with rash occurring in the 150 mg QD dose cohort. The Phase 2a portion of the study evaluated tesevatinib 150 mg administered twice or three times weekly. The tolerability of these intermittent dosing schedules was improved over 150 mg QD, but rash still occurred. In order to better characterize the safety profile of tesevatinib, an additional 20 patients dosed at 50 mg QD are being enrolled into the study.

"We believe that tesevatinib's accumulation in the kidneys and specific inhibition of EGFR and Src allow for lower dosage in ADPKD patients, making it potentially suitable for long-term use with reduced adverse events," said Mark S. Berger, M.D., Senior Vice President, Clinical Research at Kadmon. "Results from this ongoing study have indicated that tesevatinib 50 mg QD appears to be an optimal dose to treat ADPKD."

"Tesevatinib is ideally suited to treat ADPKD, a disease for which there are no FDA-approved therapies," said Harlan W. Waksal, M.D., President and CEO at Kadmon. "Since ADPKD is a chronic disease requiring lifelong treatment, we will confirm the safety profile of tesevatinib at the 50 mg QD dose before pursuing a potential registration study in this indication."

In addition to ADPKD, Kadmon is developing tesevatinib for the treatment of autosomal recessive PKD (ARPKD), a rare but more severe form of the disease affecting newborns. The Company plans to initiate a Phase 1b/2a clinical trial of tesevatinib in ARPKD in 2016.

Living with PKD

From Bel Marra Health, by Mohan Garikiparithi

Polycystic kidney disease causes renal failure, hematuria and kidney stones

Polycystic kidney disease can cause renal failure, hematuria and kidney stones. Polycystic kidney disease is an inherited disorder where clusters of cysts develop within the kidneys. These cysts are noncancerous and are small sacs of fluid. Their size can vary as they accumulate more fluid, which allows them to grow quite large.

Polycystic kidney disease doesn’t solely affect the kidneys, it can also affect the liver and other parts of the body as well. Although noncancerous, the disease can still lead to other complications, including hematuria and kidney stones.

Hematuria – or blood in the urine – is a common complication of polycystic kidney disease – it occurs in 30 to 35 percent of patients. Hematuria is associated with an increased kidney size and a faster kidney growth rate. Hematuria is commonly caused by bleeding into a cyst that communicates with the urinary tract and bladder. This can occur during strenuous activity and can result in pain. Hematuria can be temporary, lasting up to seven days, and can be resolved with bed rest and staying hydrated. If bleeding does not stop, a procedure may be required to stop it.

Kidney stones, on the other hand, occur within 25 percent of polycystic kidney disease patients. Kidney stones can be quite painful and block urine flow. If blockage is not relieved, kidney function can become lost.
Polycystic kidney disease causes

Polycystic kidney disease is inherited (genetic) so the primary cause is a genetic defect that often runs in families. There are two different types of polycystic kidney disease based on different defects: autosomal dominant polycystic kidney disease (ADPKD) and autosomal recessive polycystic kidney disease (ARPKD).

ADPKD develops commonly in those in their 30s or 40s but children can develop it as well. For a child to develop ADPKD only one parent needs to have the genetic defect. ADKPD accounts for 90 percent of polycystic kidney disease causes.

ARPKD is a far less common type of polycystic kidney disease and the symptoms are common right after birth. In this type, both parents must have the genetic defect in order to pass it on to their child.
Polycystic kidney disease symptoms

Symptoms of polycystic kidney disease include:
High blood pressure
Back or side pain
Headache
Increased size of abdomen
Hematuria – blood in urine
Frequent urination
Kidney stones
Kidney failure
Urinary tract or kidney infections
Polycystic kidney disease treatment

Treating polycystic kidney disease involves managing and treating the symptoms associated with it. By controlling the symptoms you can slow down progression and avoid complications associated with the disease. Here are some common methods of polycystic kidney disease treatment.
Controlling high blood pressure
Medications to control pain
Draining cysts
Using antibiotics to treat bladder or kidney infections
Drink plenty of fluids to drain blood in urine
Dialysis or a kidney transplant to improve kidney function
Treating liver cysts
Getting regularly screened for intracranial aneurysms if patient has history of aneurysms in the family
Polycystic kidney disease diet

Although there is no specific diet for polycystic kidney disease, there are still some important aspects of diet to keep in mind to help maintain kidney health as a whole. A polycystic kidney disease diet should include:
Low salt, sugar and fat
Minimal alcohol
Plenty of fruits and vegetables
Moderate amounts of protein
Lots of water and liquids (hydration is very important)

Aside from these diet tips, it’s also important to not smoke, use illegal drug and always remember to exercisefor overall good health.

From RTV, ABC Affiliate, Indianapolis, IN, by Chance Walser

At 39, Jeremy Williams doesn't have much time left for life-saving kidney transplant

A Kokomo man is fighting for his life while he waits desperately on the kidney transplant list.

Jeremy Williams spends eight hours a day on home dialysis. He's one of approximately 600,000 Americans who live with polycystic kidney disease.

Williams was first diagnosed in 2006 with the disease, which causes cysts to grow on his kidneys. The cysts take the organs over and shut them down.

The disease runs in Williams' family. His uncle and grandfather both had it. They both died in their early 40s.

At 39 years old, Williams' kidney function is at just 7 percent of normal.

"I figured in my 40s I'd be done for, so, you know, it wasn't a good feeling to have," Williams said. "I'm past that now and I believe that I can make it and I can beat this."

The Williamses are worried they don't have 3-4 years to wait on the cadaver transplant list. Williams' best chance to beat the disease is to get a donation from a living donor. That's the only thing Ashton Williams wished for on his 12th birthday.

"He always feels really horrible," Ashton said. "He says he doesn't feel good every day, I guess, and I hope … I hope every day he gets better."

For more information about organ transplants and how to become a donor, click here . The Williams family also operates a Facebook page to spread the word about his situation. Find that page here .

Gift of Life

From ABC Affiliate WHAM, Rochester, NY

Wife Organ Donor to Husband

Julie Boneberg always knew her husband would eventually need an organ donation. The 39-year-old just didn't realize, she would be the donor. 

Brian Boneberg, 40, has polycystic kidney disease. It's a genetic disease that has even taken the lives of his family members. "My mother had a transplant, my uncle had a transplant, my grandmother never made it to a transplant. One of my cousins passed away from it," said Brian. 

Doctors say Brian's kidneys are the size of a football, when they should be the size of a fist. "You don't have to be a spouse. Anybody can donate a kidney and there are so many people who have been waiting years and years and he's very lucky," says Julie of her husband Brian. 

"He doesn't have to do that, but unfortunately there are a lot of people who are out there waiting for a kidney." 

According to the National Kidney Foundation, there are 123,000 Americans currently on a waiting list for a lifesaving organ transplant. Of those, more than 101,000 need a kidney, but only 17,000 people receive one each year. It's rare, but the couple ended up being a good match for an organ transplant. 

They have two children, Kaylee, 20, and Zachary, 15, who say their parents are a perfect match in more than one way. "They still act like teenagers in love," says Kaylee. "They cuddle on the couch and hold hands. It's nice living in a household with parents who really love each other." 

Kaylee and Zachary have created a GoFundMe page for their parents, as they will not be able work during surgery and for some time after. "To know how hard he works now with the kidney that he has, and the kidney he is going to get, and what he is going to be able to do when he has that kidney," says Zachary, "is going to be amazing." 

The couple recently celebrated 19 years of marriage. Their surgery is scheduled for December 1.

CRISPR Gene-Editting Studies PKD; NHS OK's Tolvaptan in UK; PKD Foundation News

PKD Research

From MedicalResearch.com

Gene-edited Kidney Organoids Re-Create Human Disease

MedicalResearch.com Interview with:
Benjamin Freedman, Ph.D.
Assistant Professor | University of Washington
Department of Medicine | Division of Nephrology
Member, Kidney Research Institute
Member, Institute for Stem Cell and Regenerative Medicine
Seattle WA 98109

Medical Research: What is the background for this study? What are the main findings?

Dr. Freedman: We are born with a limited number of kidney tubular subunits called nephrons. There are many different types of kidney disease that affect different parts of the nephron. The common denominator between all of these diseases is the irreversible loss of nephrons, which causes chronic kidney disease in 730 million patients worldwide, and end stage renal disease in 2.5 million. Few treatments have been discovered that specifically treat kidney disease, and the therapeutic gold standards, dialysis and transplant, are of limited availability and efficacy.

Pluripotent stem cells are a renewable source of patient-specific human tissues for regeneration and disease analysis. In our study, we investigated the potential of pluripotent cells to re-create functional kidney tissue and disease in the lab. Pluripotent cells treated with a simple chemical cocktail matured into mini-kidney ‘organoids’ that closely resembled nephrons. Using an advanced gene editing technique called CRISPR, we created stem cells with genetic mutations linked to two common kidney diseases, polycystic kidney disease (PKD) and glomerulonephritis. Mini-kidneys derived from these genetically engineered cells showed specific ‘symptoms’ of these two different diseases in the petri dish.



Medical Research: What should clinicians and patients take away from your report?

Dr. Freedman: Pluripotent stem cells are a new area for kidney study and care. We have shown that the cells can turn into primitive mini-kidneys that can perform certain basic functions of the kidney in a petri dish. What is perhaps more impressive is that these very simple tissues can actually re-create ‘symptoms’ of kidney disease, such as PKD cysts from tubules and junctional defects in glomerulonephritis. This is especially important because these types of cells are typically hard to grow and study in the lab.

Gene editing techniques like CRISPR allow us to modify the human genome, which is breathing new life into the field of gene therapy. In our study, the only difference between mini-kidneys that showed disease symptoms and those that did not was a small mutation in a single stretch of DNA. By inference, correcting disease in patients may be as simple as correcting a single gene. Technologies like CRISPR and pluripotent cells are in their infancy, and are not yet ready for the clinic, but might someday revolutionize the way we practice medicine.

Medical Research: What recommendations do you have for future research as a result of this study?

Dr. Freedman: In the short term, our lab and others like it will use these genetically engineered mini-kidneys to investigate how diseases like PKD, glomerulonephritis, and nephrotoxicity actually work. The organoids can be produced in multi-well format, which raises the possibility of performing ‘clinical trials in a dish’ for thousands of candidate drugs to rescue disease symptoms. This high-throughput discovery approach will lead to better-informed and more successful clinical trials. The approach can be extended to many different types of kidney disease.

In the long term, pluripotent cells are a way to produce tissue on-demand that would be 100 % immunocompatible with the original patient. There is still a long way to go. The mini-kidney cells are trying to form a kidney nephron, but key structural components are missing or need to be integrated. We show in this study that mini-kidney tubules can survive after transplantation into mice. The holy grail is to further develop these grafts to be safe, efficacious, and disease-free for clinical use in human patients.

PKD Treatment

From Coventry Observer, United Kingdom

Lifechanging drug trialled in Coventry now available across the UK

A LIFECHANGING drug which was trialled and tested in University Hospital Walsgrave, Coventry, is now available to members of the public as of today (October 28).

People living with Autosomal dominant polycystic kidney disease (ADPKD) will now be able to access Jinarc (tolvaptan) – the first treatment shown to slow progression of the life-threatening condition which affects the kidneys.

ADPKD is an inherited condition that causes small, fluid-filled sacs called cysts to develop in the kidneys.

The condition can cause a wide range of problems including abdominal pain, high blood pressure and kidney stones.

Though children are born with the condition, it is not often noticeable until someone reaches 30 years old.

For many people, kidney failure at a young age can be daunting prospect.

However the introduction of tolvaptan will ensure those living with ADPKD can slow their progression towards kidney failure and reduce the number of symptoms they experience.

Tess Harris, CEO of the PKD charity who raise awareness and funds to help combat the condition, explained how the new drug will – in the long run – save thousands of pounds and provide a better quality of life for those diagnosed.

Ms Harris told The Observer: “This is the first ever drug in the world to treat this condition.

“As chief executive of the charity, our aim has been to fundraise to help support patient education, raise awareness of the condition and give verbal evidence to the public about the problems ADPKD cause.”

Tess continued and stated that to fund someone on a dialysis machine each year costs the NHS £40,000.

Not only will the new drug save money, but it will also make more transplants available to those who suffer from poor kidney function – who will not be eligible to take the drug.

Tess said: “The majority of my family is affected by the condition – my father passed away when he was 57 whilst myself, my brother, sister, niece and nephew are all affected also.

“A new quality of life will be discovered for those eligible to take the drug.”

Kieran McGovern – currently in his 30s – is just one of the 64,000 people affected by the condition in the UK.

After trialling the drug for the past eight years, Kieran revealed that taking tolvaptan has provided him with huge psychological boosts and that he feels as though he can lead a normal life.

Kieran added: “I knew that my Dad had the ADPKD so when I was 21 I decided to go and get checked up to see if I also had the condition.

“The result displayed that I did and it massively affected me – there were various complications that I had to adapt to such as my diet where I was restricted on various protein and dairy items.

“At the time I was living over in Ireland, where my blood pressure was continuously high.

“However since being on the drug I have discovered that it gives you a perk.”

Judith Dignum, chair of trustees at the PKD charity, said: “Up until now if you have been diagnosed with ADPKD you would’ve had no treatment – you’d just have to wait and worry about yourself and your children.

“This drug will help make a massive difference by delaying the progression of the disease, it will allow for extra healthy years.

“There are five stages of a kidney disease – the fifth stage would be when someone requires dialysis, this drug is required when someone would be at the second of third stage of treatment.”

PKD Foundation

Latest News

Congressional Update October 2015

On October 1, Congress gave final approval to HR 719, the FY16 Continuing Appropriations Act (or “CR”). In general, the law funds federal programs at their FY15 level until December 11, 2015. Most people hope that Congress will fund the government through the rest of FY16 by the end of this calendar year.

The next eight weeks will be very busy in Congress, starting with the election of a new Speaker of the House and Republican leadership team. In addition to annual appropriations, Congress needs to work on a host of other important legislative proposals: a budget deal to prevent automatic cuts in both domestic and defense spending (sequestration); an increase in the national debt ceiling; a transit and highway bill; a bill extending various tax breaks; the Export-Import Bank; and maybe other matters. Given the nature of the legislative process (especially at the end of a calendar year), it is too early to even speculate on when Congress may complete action on any of these proposals. Quite possibly, several of these key bills may be combined into an “omnibus” bill. How changes in the Republican House leadership may affect any of these issues and their timetables remains to be seen.

New FDA Commissioner

President Obama has nominated Dr. Robert Califf to be the new FDA Commissioner. As you know, the FDA is responsible for approving new drugs and medical devices. One of those drugs would benefit PKD patients.

The Senate Health, Education, Labor and Pensions (HELP) Committee will have to confirm Dr. Califf for his new position. A confirmation hearing provides an opportunity for Senators to question the nominee on FDA policies and procedures.


21st Century Cures Act

A significant portion of the funding increase for NIH in the 21st Century Cures bill (HR 6) would come from selling oil from the national Strategic Petroleum Reserve (SPR). Several Senators, including Sen. Lisa Murkowski (R-AK), object to the use of SPR for non-energy purposes. On October 6, the Senate Energy and Natural Resources Committee, which Sen. Murkowski chairs, held a hearing on the SPR and Energy Security.

In July, the Committee approved S. 2012, a bipartisan bill introduced by both Murkowski and ranking Democrat Maria Cantwell (D-WA). The key provision is Section 2103(b), which restricts the uses of funds raised from any drawdown to purposes directly related to either the operation of the Reserve or projects that enhance U.S. energy security. The Senate has not yet debated S. 2012.

Murkowski: In her opening statement on October 6, Sen. Murkowski stated that the SPR is “not an ATM for new spending” that could cause the country to lose the substantial benefits of the SPR. She added that some in Congress are treating our own SPR as a “cash machine.” Sen. Debbie Stabenow (D-MI) agreed that using SPR for other purposes makes “no sense”.

Sen. Elizabeth Warren (D-MA) focused on the transportation and 21st Century Cures bills. The proposed sales in these bills would fund the government with no flexibility. If oil drops to $1 a barrel, that oil would have to be sold to fund those programs. Mandating an inflexible sell-off is another bad idea for funding the federal government. She’d prefer, by inference, closing tax loopholes and getting serious about tax reform. Note that Warren also serves on the HELP Committee, which will draft its own 21st Century Cures bill.

As anticipated, most of the Senators on the Committee support the Murkowski position. Finding adequate and guaranteed funding for 21st Century Cures is imperative, but it will not be easy. [Read more]