Sunday, December 27, 2015

NIH Budget; Share the Season; Island & City Gifts; UNOS Rules Working

Budgeting for PKD Research

From Fox News, Opinion By Newt Gingrich, Bill Brazell



Imagine spending four hours a day, three days a week tethered to a dialysis machine just to survive. For many of the more than 20 million Americans—one out of every 10 adults—who suffer from chronic kidney disease (CKD), that life isn’t theoretical. It’s their daily reality.

But all taxpayers, not just those with kidney problems, have a reason to support a drastic increase in government funding for research: We, the taxpayers, are spending tens of billions each year to treat patients with kidney disease—the nation’s ninth-leading cause of death—and yet we are investing very little in trying to cure it.

Every year, the federal government spends $80 billion through Medicare alone to treat CKD (and the real cost to taxpayers is much higher when we count spending through dozens of other programs), but the National Institutes of Health are able to devote less than 0.8% of that amount to research to prevent or cure it.

To make matters worse, the costs of treating CKD are increasing rapidly. Between 2008 and 2012, Medicare spending on CKD patients increased at a rate almost five times as fast as the rest of the program and now makes up one-fifth of all Medicare Parts A and B expenditures. Costs increase as patients progress through the disease, with a dialysis patient requiring nearly $90,000 per year. This makes end stage renal disease one of the most expensive chronic diseases.

Put another way, every patient kept off of dialysis saves taxpayers $250,000.

If we fail to solve this problem, then by 2030, one out of every six American adults will have CKD.

Take for example the particular form of CKD that one of us (Bill) inherited from his father. The NIH spends $41 million on polycystic kidney disease (PKD) research per year. Having sent 5,000 patients to the transplant list, PKD costs taxpayers 50 times more than that— $2 billion—annually via Medicaid and Medicare alone.

Of course, this total does not include the lost productivity of workers forced to retire early. Only 20 percent of the people on dialysis of working age have jobs. Nor does it account for the toll of the disease on caretakers, nor the devastation that too many early deaths wreak on families.

Bill’s cousin Michael, a successful sales executive suffering from PKD, was torn from his family at the age of 35, leaving two young children to grow up without a father.

A treatment to slow the progression of PKD may now be close at hand. Thanks to research funded in part by the NIH and in part by the PKD Foundation, Bill is taking an experimental drug already approved for use in the European Union, the U.K., Japan and Canada. With just a little more spent on PKD research, even better treatments could soon follow, saving billions for years to come.

PKD is just one of more than 200 costly kidney diseases. For each, research is severely underfunded. An additional $1.5 billion over ten years could significantly reduce the $80 billion taxpayers are paying each year through Medicare to manage them. Simply delaying the onset of such illnesses by a few years would save American taxpayers billions of dollars annually—forever.

It is hard to imagine a smarter, and more compassionate, fiscal policy.

Kidney disease, of course, represents just one of many debilitating conditions for which the NIH funds crucial basic research. Heart disease, cancer, stroke, arthritis, Alzheimer’s—swifter progress toward cures would benefit all of those who suffer from these terrible diseases, to say nothing of taxpayers at large.

We have proposed doubling the NIH budget to $60 billion and reforming it to reduce bureaucracy, focus resources on basic research for the most expensive and prevalent health problems, and give the director more flexibility to redirect funds where they are urgently needed.

Leaders in both the House and Senate have taken important steps in this direction. The new budget deal passed by the House would boost NIH’s budget by $2 billion, the largest increase the NIH has received in 12 years. That’s a great first step.

Moreover, the 21st Century Cures Act, which recently passed the House 344-77, is a bipartisan effort to promote medical discoveries using the newest technology. [Read more]




Helping Those with PKD

From The Wichita Eagle, Wichita, Kansas, BY RICK PLUMLEE

Share the Season: Single mom with kidney disease can only work part time

Share the Season is an annual campaign that offers one-time aid to people affected by unforeseen hardships. The people are not identified to protect their privacy.

A painful kidney ailment diagnosed in July has turned this 28-year-old single mom’s life upside down.

But Share the Season recently was able to help her get back on top of her bills, plus provide enough that she can buy a “present or two” for her two children.

“A lot of stress release,” she said of the assistance.

Not much had gone well for the certified medical assistant since she was diagnosed with polycystic kidney disease, which causes cysts to develop on kidneys.

“It’s not curable, just manageable,” she said.

The pain has forced her to work only on “OK days,” she said. “There aren’t any good days.”

Working part time at best has been difficult, emotionally as well as financially.

“I have always been a hard worker,” she said. “I’ve worked full time since I was 16. This is kind of depressing.”

With her income greatly reduced, she and her children, ages 10 and 8, moved in with her parents in August.

But days before she was to move, her apartment was broken into and everything was stolen.

“I mean everything,” she said. “They took furniture, clothes, the children’s stuff. They even took our food. The apartment was trashed.”

Her kidney disease, which right now affects only her left kidney, is being treated with pain medication. But after the first of the year, she hopes to see another doctor and begin managing the ailment better.

“Then I think I can go back to working full time,” she said. “This whole working a little isn’t me.”

Share the Season is a joint project of The Eagle, the Wichita Community Foundation and the Salvation Army.

So far this season, it has raised $173,079.

Send contributions to Share the Season at the Wichita Community Foundation, 301 N. Main, Suite 100, Wichita, KS 67202. To donate online, go to www.sharetheseason.org. If you have questions about donating, call 316-264-4880.



Kidney Transplant Rules

From The Washington Times, By LAURAN NEERGAARD, AP


In this photo provided by the University of Virginia Health System, taken Dec. 9, 2015, Marshall Jones, right, laughs with Dr. John Barcia in the Battle Building at the University of Virginia Children's Hospital in Charlottesville, Va. A shake-up of the nation's kidney transplant system is getting more organs to patients once thought nearly impossible to match, according to early tracking of the new rules. (Coe Sweet/UVA Health System via AP) MANDATORY CREDIT

In this photo provided by the University of Virginia Health System, taken Dec. 9, 2015, Marshall Jones, right, laughs with Dr. John Barcia in the Battle Building at the University of Virginia Children’s Hospital in Charlottesville, Va.

A shake-up of the nation’s kidney transplant system means more organs are getting to patients once thought nearly impossible to match, according to early tracking of the new rules.

It’s been a year since the United Network for Organ Sharing changed rules for the transplant waiting list, aiming to decrease disparities and squeeze the most benefit from a scarce resource: kidneys from deceased donors. Now data from UNOS shows that the changes are helping certain patients, including giving those expected to live the longest a better shot at the fittest kidneys.


The hope is to “really level the playing field,” said Dr. Mark Aeder, a transplant surgeon at University Hospitals Case Medical Center in Cleveland who is chairman of the UNOS’ kidney committee.

In Abingdon, Virginia, 8-year-old Marshall Jones was one of the lucky first recipients. A birth defect severely damaged his kidneys and a failed transplant when he was younger left his immune system abnormally primed to reject kidneys from 99 percent of donors.

Then last January, after four years of searching, organ officials found a possible match, hours away by plane but available under the new policy - and it worked.

“We don’t use the word lightly, but this was really a miracle kidney for him,” said Dr. Victoria Norwood, Marshall’s doctor and the pediatric nephrology chief at the University of Virginia.

There’s a huge gap between who needs a new kidney and who gets one. More than 101,000 people are on the national waiting list, while only about 17,000 kidney transplants are performed each year. Roughly 11,000 of them are with kidneys donated from someone who just died; the rest occur when a patient is able to find a living donor.

The wait for a deceased-donor kidney varies widely around the country, and in 2014, more than 4,500 people died before their turn.

The new kidney allocation system can’t alleviate the overall organ shortage. “The only thing to shorten total wait time for everybody is more organ donors,” Aeder said.

Instead, the policy altered how deceased-donor kidneys are distributed, shifting priorities so that how long you’ve been on the waiting list isn’t the main factor. Among the changes:

-fewer transplants are occurring in which the kidney is predicted to outlive the recipient. Now, the kidneys expected to last the longest - as calculated by donor age and medical history - are offered first to the patients expected to survive the longest. That’s called longevity matching. Before the change, 14 percent of the longest-lasting kidneys went to recipients age 65 or older. That dropped to 5 percent as the new policy kicked in, according to UNOS monitoring. [Read more]




Gift of Life

From South Philly Review, By Bill Chenevert

Giving the biggest gift of all


Mike Farulo is one of nearly 5,000 area residents waiting for a kidney.  Photo by Bill Chenevert


Mike Farulo often goes to bed thinking “Is tonight going to be the night? Any day now my life could change for the better.” Farulo suffers from Polycystic Kidney Disease (PKD), a genetic disease that fills one’s kidneys with cysts and zaps their functionality. Dialysis and donations-turned-transplants are the only solution.

It’s a tiring and frustrating daily existence and Farulo, a resident of Broad and Ellsworth streets, has an impressively balanced perspective on it all.

“I knew from a kid that I might have to deal with it when I got older” – his mother had PKD, too, and went through 22 years of dialysis and two donations. “My mom had it, my grandmother had it, my uncle, my mom’s brother had it,” Farulo said; it’s genetic and hereditary.

Farulo works with Gift of Life, the regional donor organization that covers eastern Pennsylvania, southern New Jersey and Delaware. He volunteers by driving patients, donors and family members to appointments in conjunction with the Gift of Life Family House in Northern Liberties.

“Gift of Life oversees 40,000 organ transplants a year: kidneys, hearts, lungs, livers, pancreas, intestines, all the internal organs,” Howard Nathan, president and CEO of Gift of Life, explained. “In the U.S. there are 122,000 people waiting for an organ – 101,000 are waiting for kidneys. There are 444,000 people on dialysis in the U.S. Regionally, 5,900 people are waiting [for an organ], of which about 4,900 are patients waiting for kidneys.”

Farulo’s childhood was rooted in Elizabeth and Somerset, N.J. He came to Philadelphia at 18 to go to LaSalle University but ended up getting associate degrees from the Art Institute of Philadelphia and the Community College of Philadelphia. He’s sustained himself in the service industry and had a great run of working on Linblad Expeditions cruise boats, which travel the world. He bartended and managed a 65-passenger cruise boat for about 10 years and had to give it up for dialysis. Now he works a few hours a week at Pumpkin, 1713 South St., simply to engage with life outside dialysis and Gift of Life patients.

“The littlest bit of exertion and I start getting very fatigued,” he explained. “I can do some light bicycle riding. I can’t work full-time anymore. I can’t have soup or ice cream. I can’t have beer or alcohol.”

He can ingest up to only 32 ounces of liquid per day. And on days like last Monday, his dialysis wasn’t enough and he had to throw in a fourth session to remove his blood of all liquids.

Nathan explained that the Center for Disease Control establishes criteria for kidneys that may or may not be at-risk due to the deceased’s medical history. They “evaluate nearly 40,000 deaths in [a network] of 129 area hospitals, almost 110 per day. Only about 750 are suitable donors,” he said. “Mike’s getting called because he’s been on the waiting list for a while,” since 2012 in his case. Even before the dialysis began.

The first four calls that Farulo’s received haven’t materialized into a donor. The initial three were highly at-risk and, being even a little sluggish on the chance to jump on the fourth combined with being fifth on the list, both kidneys from the fourth call just missed him.

Every once in a while, the Point Breeze resident tells his story to an interested party and they swear they’ll consider being a live donor. Although he doesn’t hold his breath, he seems peacefully hopeful that his luck could change at a moment’s notice.

“I tell my story to people who want to listen,” he said humbly.

When his mother was dying, his father went to the local newspaper and swears her story resulted in 10 or 12 people that went into the nearby hospital to test their kidney compatibility.

“4.7 million people have it on their license out of 9 million drivers [in PA],” Nathan reported. “In the U.S., 125 million have it on their state ID or driver’s license and that’s half of the driving public. Over 90 percent say that organ donation is a good thing” but, he notes, trauma scenes often don’t include a state ID and that’s why it’s important to register as an organ donor with PennDOT. “One to two percent of all potential people who die are potential donors,” he says. “The need is great [for donors].”




From The Beaufort Gazette, Hilton Head, SC, BY RYAN COPELAND



This will be Linda Keene's second Christmas with one of Sara Oakley's kidneys.

Three years ago, when Keene's organs began to fail, Oakley was there to offer a shoulder to cry on, an ear to listen, and, ultimately, an kidney to donate.

But that is not where the story -- or their bond -- really began.

Over thirty years ago, Oakley serendipitously stepped into Keene's music room at Laurel Bay Elementary. Both teachers, both relatively new to Beaufort, and both about to have their first babies. They also lived one street over from each other and began to carpool to work.

Keene soon took time off to stay at home with her daughter, Meredith, as well as Oakley's son, Hayes. Their lives were running smoothly as the 1970's came to a close.

But as the 1980's began, so did health problems for Keene's family.

Her mother had long suffered from Polycystic Kidney Disease. Two of her three sisters were diagnosed with the illness. Keene was tested and found to be carrying the hereditary gene as well. At that time, all she could really do was monitor her blood pressure and kidney functions. But the time bomb was quietly ticking.

It wasn't until 2006, when Keene's sister Claudia passed away, that Oakley became aware of the serious nature of the disease. She told her husband that if it ever came to Keene needing a transplant, she would provide the kidney.

Her chance to make good on that vow came sooner than either she or Keene imagined.

By the summer of 2012, Keene noticed herself slowing down physically. The smallest effort left her exhausted. Her nephrologist in Savannah set up an appointment at the Medical University of South Carolina in Charleston to discuss treatment options after her kidney function dropped well below the 15% standard for healthy organs. She was in full renal failure.

"It's funny, I had seen my sister Susanne receive a transplant and suffer from some complications and told myself I never wanted to go through with that," said Keene.

But faced with few other options, she accepted the news that she was a good candidate for a transplant.

Several friends immediately got tested as possible donors, a gesture Keene found "meaningful" and well-intended. Most of them, however, never made it past the point of having good enough blood pressure readings.

One who did make it through to the next round was Oakley.

"I procrastinated in filling out the paperwork, but once I passed the 24-hour blood pressure monitoring, I realized I had made it further as a potential match than others," said Oakley.

Calling it the "best physical anyone could have," Oakley passed the stress test, blood matching and every other measuring tool the doctors threw at her. She had not only a clean bill of health, but the health of a woman half her age. That matters only because she was soon to become the oldest transplant donor in the history of MUSC.

"They called me on my birthday to tell me I was a match and everything was a go," said Oakley.

The present, though, was for Keene.

For two people as close as sisters, the match was no surprise.

The transplant was performed in May of 2014.

As Oakley lay on the operating table having the kidney removed, Keene waited in a nearby room to receive it.

The surgeon emerged from the operation room to say the "new" kidney had begun working immediately in Keene's body.

Now, Keene gets compliments about how healthy she looks -- a fact Oakley attributes to having the gift of her kidney.

While Keene knows not to push things too much, the transplant has transformed her.

"It added years to my life and opened up new opportunities I didn't have before," said Keene, including a continuing friendship with Oakley.

Oakley agrees.

"She knows more secrets about me than anyone, but now she can't tell them because she has my kidney," said Oakley.

Sara Oakley gave Linda Kenne the gift of life.

Together they share the gift of one another.




From Panama City News Herald, Panama City FL, By JENNIFER RICH

Read more here: http://www.islandpacket.com/news/local/community/beaufort-news/article50721035.html#storylink=cpy


Bonifay resident celebrates 'sister-like match' this Christmas

Candi Hinson and Becky Raulerson went on a cruise to the Bahamas two years ago.


Even with Christmas just days away, 39-year-old Candi Newsom Hinson says she already has received the biggest gift possible.

“It’s right here,” said Hinson, patting the right side of her abdomen.

Hinson has spent the year recovering since receiving a kidney transplant earlier this year. In the ninth-grade, Hinson first learned about her kidney disorder during a sports physical for the Blue Devils basketball team. As the physician palpated her stomach, he felt Hinson’s spleen was enlarged. The observation led to further testing and a diagnosis of polycystic kidney disease (PKD).
PKD is a genetic disorder in which the kidneys develop cysts over time, causing kidney function to suffer tremendously.

“They take over the kidneys where they can’t filter,” Hinson said.

There’s no known cure for PKD, and more than half of people diagnosed will experience kidney failure by age 50. These patients eventually require dialysis or a kidney transplant.
Hinson said after her diagnosis, she was put on blood pressure medicine to avoid putting unnecessary strain on her kidneys. She lived a normal life with PKD, but had occasional lower back pain and fatigue. Hinson continued to get yearly checkups — until two years ago, when her kidney function had deteriorated enough for doctors to advise it was time to consider a transplant.

“Some people stay on the transplant list for years, and you have to meet all this criteria,” she said.
Hinson’s husband, Travis Hinson, was first to be tested as a possible donor. He was a match to his wife, but he was ruled out due to kidney stones.

Doctors told Hinson her best option would be a live organ donor.
Doctors “see how well people do with transplants before dialysis, so that was the goal, and we met it because of Becky.”

‘It’s going to be you’

Hinson’s best friend since elementary school, 39-year-old Becky Raulerson insisted she would be next to be tested as a potential donor.

“A feeling came over me,” Raulerson said, "and God said, ‘Becky, it’s going to be you.’ ”

In November 2014, Raulerson flew to Walter Reed National Military Medical Center for three days of compatibility testing. After the gamut of blood work and scans, the lifelong friends received the news for which they had hoped — Raulerson was a perfect match.

“They told us it was a sister-like match,” Hinson said.

Raulerson said she wasn’t at all surprised, and a surgery date was set for April 16. The friends saw one another the day following the surgery.


Read more here: http://www.kansas.com/entertainment/holidays/article50694485.html#storylink=cpy


Read more here: http://www.kansas.com/entertainment/holidays/article50694485.html#storylink=cpy

Sunday, December 20, 2015

PKD & EVs: ExtraCellular Vehicles; Gift of Life from 70 year old woman

Gift of Life

From KHOU TV, Houston, Texas, by Kevin Reece, KHOU 11 News


635859781713066541-Reece-pkg-elderly-kidney-donor2.JPG


Two women in Simonton, Texas, with the help of doctors at Houston Methodist, are proving that age is no barrier to a successful living donor kidney transplant – even if that donor and her kidney are 70 years old.

Ann Hammermiller, 73, suffers from a genetic condition called polycystic kidney disease.

She was nearing the point when she would need to begin dialysis or search for a donor.

But as a member of Simonton Community Church, she learned her pastor suffered from the same condition.

He announced his need for a donor and remarkably, a member of the congregation offered to be his living donor and turned out to be a perfect match.

That's where Judy Peters comes in. She was willing to offer one of her kidneys to the pastor too. But learned her friend Ann Hammermiller was also in need. She didn't hesitate. Judy made the offer at a church dessert social.

"And she said what do you think about me being your donor? And of course I thought 'Oh gee she's had too much sugar,'" Hammermiller said with a laugh, politely questioning her friend's state of mind.

Peters turned out to be a perfect match. The surgery happened this past September. Doctors at Houston Methodist now say both women, at 70 and 73, are doing very well.

"We tend to be very conservative with donors. We are very protective of donors," said Dr. A. Osama Gaber, the director of Houston Methodist's J.C. Walter Jr. Transplant Center.

Gaber says the age of the kidney isn't the determining factor but that healthy kidney function is.

"Really our concern isn't the kidney we're going to take. It's what we're going to leave and how healthy the donor is going to be for the rest of their lives. That's really our major concern, said Gaber.

"I can't thank her enough for giving me a new life," Hammermiller said.

"I could say 'I'm too old, I can't possibly do that,'" Peters said when asked about any reservations she might have had about the kidney donation. "But when God calls you to do something, God makes it happen."

When the successful transplant took place at Houston Methodist, with the women recovering with just a four-day hospital stay, Judy Peters became the second oldest kidney donor ever at the hospital. The oldest was 71.

"Living donors are true heroes," said Gaber. "They are just these people that come in and they're willing to give of themselves, truly of themselves, to others. And it's a fantastic thing to watch this and to be part of it."

"There's no words," Hammermiller said seated next to her friend and kidney donor. "What can you say to someone like that? All you can do is say thank you, thank you and you're just so blessed in getting an offer like that. It's just a blessing that someone would step up and say I want to do this for you."

"We were good friends," Peters said.

And now?

"Better friends," she said as both broke into laughter. "We're connected. I'm just privileged to be a part of that. It was just very, very sweet, that is my sweet friend."

A verse in the bible says it is better to give than receive. The two women in Simonton would tell you that both at the same time, are pretty rewarding too.

If you would like to learn more about transplants and living donations click here.



PKD Research

From Medical News Today, by Catharine Paddock PhD
Cells send tiny parcels to each other - in sickness and health

Within the past decade, it has emerged that cells package various molecules into tiny bubble-like parcels called extracellular vesicles to send important messages - in sickness and health. The potential for using these vesicles in diagnosis and treatment is now under intense investigation.

Extracellular vesicles (EVs) are so small they can only be seen through high-tech electron microscopes. The membrane-covered structures are roughly in the same size range as viruses, bacteria and platelets.

For decades, scientists thought the cargo of EVs - which can consist of various molecules such as proteins and genetic material - was just debris.

More recently, they have begun to understand the importance of EVs in health and disease and how they might be used to ferry drugs to their targets.

However, there are still many large gaps in our understanding of EVs.

For example, because EVs are found in bodily fluids like blood, urine and cerebrospinal fluid, researchers are finding it nearly impossible to determine where they come from, how they are made or how they release their cargo of molecules.

Another mystery is why the same EV cargo can result in different outcomes.

Now, a new study - led by Rutgers University in Piscataway, NJ, and published in the journal Current Biology - offers some new clues about EVs.

Significant insight into biology of EVs and their role in human diseases

Lead author Maureen Barr, a professor of genetics at Rutger's, says EVs are both exciting and scary because we do not know the mechanisms that control what goes into them. She explains:

"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."

Prof. Barr says the more we find out about how a cell makes and packages proteins, lipids and bits of genetic material into EVs, the more we can use that knowledge to develop drugs and therapies to treat diseases. For example, one use could be to prevent cancer cells from using EVs to send messages that promote tumor growth.

For their study, the team investigated EVs in Caenorhabditis Elegans. The roundworm is a very useful model for understanding human biology at the level of cells - for example, many of its genes are similar to ours.

The researchers identified 355 genes that offer significant information about the biology of EVs and the role they might play in human diseases.

One in ten of the genes appear to control the formation, release and possible function of the vesicles.

The study also identifies new pathways that might control how cells produce EVs and choose their cargo, including proteins responsible for the most commonly inherited disease in humans - polycystic kidney disease.

Prof. Barr says cells secrete polycystic kidney disease gene proteins in EVs in both humans and worms, and no one knows why. She concludes:

"When we know exactly how they work, scientists will be able to use EVs for our advantage. 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."

Meanwhile, from another recently published study of roundworms, Medical News Today learned that a Velcro-like molecule essential for sperm to be able to attach to eggs during fertilization is the same as one discovered in humans 10 years ago. The researchers suggest the finding could lead to better fertility treatments and contraceptives.


From 7th Space

Effects of pyrrolidine dithiocarbamate on proliferation and nuclear factor-κB activity in autosomal dominant polycystic kidney disease cells


Pyrrolidine dithiocarbamate (PDTC) reduces renal cyst growth in a rodent model of polycystic kidney disease (PKD) but the mechanism of action is not clear. Here, we investigated the hypothesis that PDTC reduces the proliferation of cystic epithelial cells in vitro in a nuclear factor (NF)-?B-dependent manner.

Methods: Immortalized autosomal dominant PKD (ADPKD) cells that are heterozygous (WT9-7) and homozygous (WT-9-12) for a truncating Pkd1 mutation, and immortalized normal human tubular cells (HK-2), were exposed to NF-?B-inducing agents with or without PDTC.

Cell proliferation and apoptosis were assessed by bromodeoxyuridine assay and Annexin V flow cytometry, respectively. NF-?B activity was assessed by luciferase reporter assay and western blotting for nuclear p65, p50, and RelB subunits and cytoplasmic phosphorylated-I?B?.

Results: Serum-induced proliferation was similar in all cell lines over 72 h.

PDTC demonstrated anti-proliferative effects that were delayed in ADPKD cells compared to HK-2. Basal NF-?B-dependent luciferase reporter activity was lower in ADPKD cells compared to normal cells.

Classical NF-?B stimulants, lipopolysaccharide (LPS) and tumor necrosis factor (TNF)-?, increased NF-?B luciferase activity in HK-2, whereas in PKD cell lines, NF-?B activity was only induced by TNF-?. However, neither stimulant altered proliferation in any cell line.

PDTC reduced TNF-?-stimulated NF-?B activity in HK-2 only.

Conclusions: PDTC reduced proliferation in ADPKD cells but did not consistently alter NF-?B activation, suggesting that other signalling pathways are likely to be involved in its ability to attenuate renal cyst growth in vivo.

Sunday, December 13, 2015

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.
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


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 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

Lauren, Dana, and Karen.

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]

Sunday, December 6, 2015

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 - Kevin Mills Photos


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.
PHOTO BY STEVE FULLER

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.

Sunday, November 22, 2015

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

Artificial Kidney

From Tech Times, by Katherine Derla


The Kidney Project


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


University of Washington 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

Phil 'The Tumble Dryer' Box, gets ready for a massive 14.5 hour treadmill run, raising money for PKD (Polycystic Kidney Disease) charity which his nephew suffers from


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.