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]

Creating Mini-Kidney Organoids; Sister's Gift; COTA Fundraising for Life; Getting Donor Time-off; Living Dreams

PKD Research

From Science Codex

Mini-kidney organoids re-create disease in lab dishes

Mini-kidney organoids have now been grown in a laboratory by using genome editing to re-create human kidney disease in petri dishes.

The achievement, believed to be the first of its kind, resulted from combining stem cell biology with leading-edge gene-editing techniques.

The journal Nature Communications reports the findings today, Oct. 23. The work paves the way for personalized drug discovery for kidney disease.

The mini-kidney organoids were grown from pluripotent stem cells. These are human cells that have turned back the clock to a time when they could develop into any type of organ in the body. When treated with a chemical cocktail, these stem cells matured into structures that resemble miniature kidneys.

These organoids contain tubules, filtering cells and blood vessel cells. They transport chemicals and respond to toxic injury in ways that are similar to kidney tubules in people.

"A major unanswered question was whether we could re-create human kidney disease in a lab petri dish using this technology," said Benjamin Freedman, who led the studies at Brigham and Women's Hospital in Boston. He is now an assistant professor of medicine in the Division of Nephrology at the University of Washington and a UW Medicine researcher.

"Answering this question," he said, "was important for understanding the potential of mini-kidneys for clinical kidney regeneration and drug discovery."

To re-create human disease, Freedman and his colleagues used the gene-editing technique called CRISPR. They engineered mini-kidneys with genetic changes linked to two common kidney diseases, polycystic kidney disease and glomerulonephritis.

The organoids developed characteristics of these diseases. Those with mutations in polycystic kidney disease genes formed balloon like, fluid filled sacks, called cysts, from kidney tubules. The organoids with mutations in podocalyxin, a gene linked to glomerulonephritis, lost connections between filtering cells.

"Mutation of a single gene results in changes kidney structures associated with human disease, thereby allowing better understand of the disease and serving as models to develop therapeutic agents to treat these diseases," explained Joseph Bonventre, senior author of the study. He is chief of the Renal Division at Brigham and Women's Hospital and a principal faculty member at Harvard Stem Cell Institute.

"These genetically engineered mini-kidneys," Freedman added, "have taught us that human disease boils down to simple components that can be re-created in a petri dish. This provides us with faster, better ways to perform 'clinical trials in a dish' to test drugs and therapies that might work in humans."

The researchers found that genetically matched kidney organoids without disease-linked mutations showed no signs of either disease.

"CRISPR can be used to correct gene mutations," explained Freedman. "Our findings suggest that gene correction using CRISPR may be a promising therapeutic strategy."

In the United States, costs for kidney disease are about 40 billion dollars per year. Kidney disease affects approximately 700 million people worldwide. Twelve million patients have polycystic kidney disease and two million gave complete kidney failure. Dialysis and kidney transplantation, the only options for patients in kidney failure, can cause harmful side effects and poor quality-of-life.

"As a result of this new technology," Freedman said, "we can now grow, on demand, new kidney tissue that is 100 percent immunocompatible with an individual's own body."

He added, "We have shown that these tissues can mimic both healthy and diseased kidneys, and that the organoids can survive in mice after being transplanted. The next question is whether the organoids can perform the functions of kidneys after transplantation."

source: University of Washington Health Sciences/UW Medicine

Gift of Life

From Grand Island Independent, Nebraska, By Lauren Sedam

Woman is living because of her sister's gift of a kidney

BURWELL — Korlyn Goff is the queen of casseroles.

Just ask her sister, Lisa Obermier.

It’s something Goff and Obermier said they learned from their mother, Dee Conner, when they were growing up in Burwell.  Whether it was helping harvest crops or showing up with food in someone’s time of need, the sisters saw how important it was to be there.

“I live by the philosophy that you have to be a neighbor to have a neighbor,” Goff said.

But last year, Goff took that giving spirit above and beyond.  Obermier had long been living with polycystic kidney disease, and she was in need of a donor. Goff stepped up, and almost a year later, the two are thriving. But to Goff, the gift wasn’t some heroic effort.

It was what she had to do for her sister.

“It was the right thing to do,” Goff said. “It was the only thing to do.”

Finding the match

Polycystic kidney disease is a disorder in which cysts form in the kidneys, causing complications, including kidney failure.  It is hereditary, and several members of Goff’s and Obermier’s family have the disease, which can also present itself in the liver. Some have had transplants as well, and other family members have donated to some of them.

About 25 years ago, Conner was diagnosed with the disease in her liver. At the time, Obermier was tested, but her liver was fine.  It wasn’t until about 10 years ago that Obermier, who now lives in York, discovered she had the disease in her kidneys.

“That’s when they said it leads to transplant or dialysis or death,” she said.

For a long time, Obermier was on the list for an “angel donor,” but a living donor was also an option.

It became more of a possibility for Goff, who still lives in Burwell and does not have the disease, when her sister was forced to go on dialysis.  She saw her struggling, and she knew then that she had to get tested.

“I thought the sooner I make this decision, the sooner we can get on with life,” she said.

Other family members, including their father, Delmer, had wanted to donate, but Goff said she knew she was the match.  Around Thanksgiving, she started the process. After ensuring she was current on a variety of exams and after having preliminary blood work done, Goff underwent more extensive testing at Mayo Clinic in Minnesota.

“They tested you from one end to the other in that two days,” she said.

At the end of it, Goff heard the news she’d always expected: She was a match.  Those at Mayo Clinic told her to go home and talk about it with her family, but Goff knew what she needed to do.

Even so, others weren’t so sure.

Goff’s husband, Dave, is an only child, she said, and it was hard for him to think about his wife and the mother of Dustin, 26, and Kaden and Kortney, 21, going through something so big.  For the Conners, it was hard to imagine both of their daughters in this situation. It was a scary time, Dee Conner said, but she knew Goff had her mind made up.

“She had told me that she thought it was something she needed to do to save her sister’s life,” Conner said.

Obermier, too, worried about putting her sister in danger.

"It was mixed feelings because it’s very humbling to have to accept that kind of gift from anyone,” she said. “And I’d rather be on the other end of it any day.”

Their biggest fears

The operation was set for Dec. 30.  Goff, who works at a salon, wanted to recover during her slower months, and the transplant teams were able to make it work.

Obermier went to Minnesota on Christmas Eve for testing, and Goff came the day after Christmas.

On the day, they were supposed to be the second operation, but they were bumped to third. Goff was finally taken in at about noon, and Obermier was left to wait until they were ready for her a few hours later.

The sisters both came through the procedure, but they soon had a brush with some of their biggest fears. [Read more]

From WHO-TV, West Des Moines, Iowa, BY MICHAEL DASILVA

Kidney Donor Runs into Obstacle for Time Off from Work

"It`s been a big gift," said Heather Watson-Vance, who received great news last week when she found out that her step-daughter Cori Vance was a match to be an organ donor. "I was born with polycystic kidney disease, which is a terminal genetic illness. There is no cure. Pretty much all we can do is a kidney transplant or dialysis," said Heather Watson-Vance.

So when she found out Cori was approved to be a living donor and give her a kidney, she was ecstatic; until she found out Cori's employer - Windsor Windows & Doors in West Des Moines - wasn't so thrilled.

Cori says she kept Windsor in the loop throughout the whole process, and told them that she was being tested to see if she was a match, but that when the company found out she was a match, she was met with opposition.

"The HR lady had said that they`ve been talking and we need to talk about the days off and if I would be coming back, and so she recommended that she would give me a week to come back," said Cori.

Cori says she was later told by Windsor that they would give her two weeks off tops, and that that was them being generous, and that there would be no restrictions. "I had informed them that, you know, I wasn`t gonna be able to come back without any restrictions, and what I do is the paperwork, that I would have a ten pound restriction, and then she asked me would you be able to stand? For 8-10 hours? and I said it`s all up to my doctor, like I don`t know right now and she said no, so she just told me that my last day would be October 30," said Cori. At her job, in addition to paperwork, Cori also moves metal. "I unload it and load it into a cradle," said Cori.

Channel 13 News went by Windsor Windows & Doors in West Des Moines to hear what they had to say about all of this, but we were told by the front desk that the Divisional Human Resources Manager - Pete Crivaro - had no comment. "And then I spoke with you guys and when you guys had showed up there, I don`t think he liked that very much. So, he told me that if I do speak with you guys to let him know so that he could prepare and that to let you guys know that he`ll grant me 4-6 weeks," said Cori.

But Cori and Heather say this story is bigger than just their situation. "And that`s not my point is for me to get the 4-6 weeks, because they weren't gonna give it to me anyways, until all of this happened. I just like, it`s not okay, and I don`t` want other people who like have thought about donating, I don`t want them to change their mind, not to do it because they're scared or companies aren`t giving them a chance neither," said Cori.

"We just really feel like with an organ donation that`s gonna impede other people from maybe wanting to donate an organ if they were treated this way at their job or they're not going to receive their job back after the donation, I mean you can`t put your own family in jeopardy, you know even to save a life," said Heather Watson-Vance. [Read more]

Fundraising

From Plainfield Patch, Plainfield IL, by Scott Viau

Fundraiser for Plainfield Boy in Need of Kidney Transplant

When five-year-old Drew Duszynski was born, he was diagnosed with a rare kidney disease and liver disorder: Autosomal Recessive Polycystic Kidney Disease and Congenital Hepatic Fibrosis.

On June 8, 2015 Drew had both of his oversized, diseased kidneys removed and after a month in the hospital he returned home. He receives nightly dialysis (10 hours) and, if all continues to go well, he will soon complete the transplant process and receive a new kidney.

Despite his condition and all the challenges, Drew is just a normal little boy – bright eyed, smart, sweet, playful, funny, inquisitive and full of life

With the total cost of a transplant often exceeding $500,000, transplant patient’s families are unable to shoulder the financial burden of such a procedure. While insurance will cover a great deal of the costs for Drew’s kidney transplant, the expected out-of-pocket costs are estimated to be in excess of $50,000.

In connection with the Children’s Organ Transplant Association, a fundraiser for Drew will be held from noon to 3 p.m. Saturday, Nov. 7 at Katie O’Connor’s Pint House and Eatery at 13717 US Route 30 in Plainfield.

The event will include raffle drawings and 50/50, kids activities, food and a cash bar. Tickets are available at the door. Families will pay $12 or $6 per person. RSVP by Nov. 3 and receive a discounted ticket price of $8 for families or $4 per person. To RSVP, contact Dave Duszynski at 773.251.8464 ordaveduz@ameritech.net or Sara Swenson at773.677.7360 or sararenae_1@yahoo.com.

COTA (Children’s Organ Transplant Association) is a national charity dedicated to organizing and guiding communities in raising funds for transplant-needy patients. The organization helps children and young adults who need a life-saving transplant by providing fundraising assistance and family support. COTA does not charge for its services and does not take a percentage of funds raised.

Find our more about Drew and COTA at COTAforAndrewD.com.

Living with PKD

From Northampton Chronicle, United Kingdom

Northampton firm backs 11-year-old girl’s dream to play football while fighting serious kidney disease

A talented young footballer is playing out her dream thanks to a generous sponsorship from a Northampton company.

Bethan Edgell, 11, from Buckingham, continues to fight a serious kidney disease, Autosomal Recessive Polycystic Kidney Disease (ARPKD), however she has been recognised by the MK Dons. And to help her realise her footballing ambitions, pump specialist firm Process Systems International (PSI) has agreed to pay for her elite training.

The football crazy youngster plays for Moretonville Harriers as a Striker and is now training with the MK Dons Girls Skills Elite Centre U13 squad.

Her proud dad Chris, who is an engineering co-ordinator for PSI, said: “Bethan has to overcome so many health hurdles to play her various sports, that this opportunity is really important and rewarding for her. There is no cure for her condition and her kidneys will gradually deteriorate, so she is determined to make the most of every sporting opportunity given to her.

“To have the support of PSI to pay for her additional training is very touching indeed.”

As for Chelsea loving Bethan, it’s early days but so far she is enjoying her elite status. She said: “I am truly grateful to PSI for giving me this opportunity with the MK Dons. The various sports I play, in particular football, is a large part of my life and keeping fit is helping with my condition.”

Waiting; Urgent Message; Finds Donor on Facebook; New Medicare Dialysis Model; Home based Dialysis

Living with PKD

From The Press and Journal, Aberdeen, United Kingdom, by Davis Kerr

Man forced to wait four years for kidney transplant

An Orkney man has criticised the Scottish Government after being forced to wait for years for a new kidney.

Steve Sankey spent four years waiting for and organ transplant and had to endure three years of dialysis and regular flights to Aberdeen for check-ups.

But he believes he and others in his situation could be spared the wait if the government adopted an “opt out” system for organ donations.

At present people have to sign up to the organ donor register for their organs to be used in transplants.

The government said it was committed to increasing the number of donors but evidence was split on whether an opt-out system would work.

Mr Sankey had been living with adult polycystic kidney disease, which left him with only 5% of organ function and meant he had to undergo dialysis.

The 59-year-old wildlife guide finally received a new kidney on Tuesday, September 15 at Edinburgh Royal Infirmary.

He said “Naturally I’m delighted with my new kidney, which after a month is working well and thus far all appears to be fine.

“The medics are really pleased with progress, as are all my family and friends. I had great support from everyone, especially down in Edinburgh, where I used to work, and I’d like to pay tribute to the professionalism of the transplant team in Edinburgh and the renal team in Aberdeen.”

Mr Sankey recently travelled to the Scottish Parliament for the launch of a private member’s bill being led by Labour MSP Anne McTaggart to try to change the law on organ donation.

He said: “It’s hugely disappointing that the Scottish Government has chosen not to include this piece of legislation in their programme – it could help so many others in my predicament.

“Instead we’re having to rely on a private member’s bill to try and get the organ donation system into a ‘soft opt-out’ one like many other European countries, including Wales.

“If I had been a resident of Spain, for example, I would have probably had a transplant several years ago, as the supply of organs through the different system is much greater than in the UK.”

From People, BY TIARE DUNLAP

Washington Woman Finds Unique Way to Search for a Kidney Donor for Ailing Husband

Amy Malancioiu is driving around Duvall, Washington, in an SUV emblazoned with her personal cellphone number and a desperate plea.

"Urgent!" reads a messagein large letters on her family's car. "Please help!! Husband needs a kidney. 206-930-2517."

Amy's husband, 57-year-old Sorin Malancioiu, needs a kidney, and it could take as long as six years for him to receive one through the organ transplant waiting list as his type O blood is the most difficult to match, KOMO newsreports.

Sorin suffers from an inherited disorder called Polycystic Kidney Disease, his wife explained on GoFundMe. The disease caused cysts to grow on the father of two's kidneys until they became too compromised to function and were removed in 2014.

"Just seven years ago, Sorin led a very active lifestyle," Amy wrote. "Since the surgery this past June, he has been on dialysis 3 days a week for 4 hours a day. He is exhausted, listless and in pain after each session."

Faced with six more years of these painful treatments and uncertainty about the future, Amy decided to take matters into her own hands.

"I don't want to wait for [a kidney] to come to us," Amy told the news station. "I want to go out and get it."

Amy, one of the couple's daughters and a close family friend are driving around with the plea written on their car windows.

"It's amazing the response I'm getting," Amy said. "It's time to spread the word as fast as we can hoping that somebody would be willing to donate – to get him a kidney faster so he doesn't have to live like he does now."

The couple said they've received 20 serious offers from people who say they're willing to donate. Finding a match will still be extremely difficult as donors must have type O blood, be non-smokers, over 21 years old and in good health.

Still, they remain hopeful their unusual tactic will yield results.

"I believe this could lead to a donor, to the right person," Sorin said.

Until it does, Amy will continue the search.

"It's just incredible to have somebody like her," Sorin said. "I'm the luckiest man. I am so lucky to have such a wonderful marriage."

From Global News Canada, By Erika Tucker

Kidney ‘want ad’ on Facebook leads to donor match for Calgary woman

CALGARY – A Calgary woman who has suffered from polycystic kidney disease for 15 years posted her plea for a donor on Facebook in June, writing, “the only way I will get a kidney before I die is to find one on my own and social media will be the best way.”

She was right.

“I wrote it and I shared it to a friend, and that friend, Linda, shared it to everybody,” said Lynne Prodaniuk, who has been on the donor list since 2007 with three attempted matches, but no success.

She said she nearly died in 2000 as doctors weren’t initially sure what was wrong. At one point in her treatment, her kidney function fell to 12 per cent. She said she had a catheter put in her abdomen to start dialysis, but by 2014, the dialysis was no longer cleaning her blood well enough, and her toxicity levels increased. She said she had surgery and started hemodialysis in hospital, and has been going for eight-hour treatments since then.

Prodaniuk said when she joined the donor list, the wait was estimated between four and six years. When she got to six years, she was told the wait time had increased to eight years. She passed her seven-year anniversary July 26.

Then, another friend’s Facebook post turned things around for Prodaniuk.

“I posted on Facebook that I needed a handyman, a window company recommendation, and a kidney,” Erin Kelly told Global News.

“Mostly I was kidding about the kidney but I put it out there anyway because my Facebook friends are awesome and I was secretly hoping someone would at least inquire.”

Kelly’s friend, Shamus Neeson, replied right away. Neeson works for Canadian Blood Services and has donated blood many times, so he was curious about the living donor process.

“It was something that I felt that I should look into, if I could help save someone’s life…it just made sense,” said Neeson. “I put a call in to the living donor program, they sent me a package of very basic screening questions on my family history and what not and then we just started going from there.”

Neeson has now gone through all required kidney and cardiovascular testing and been given the go-ahead. The two new friends are waiting for the green light from Alberta’s living donor program, and from there will schedule surgery.

“I can’t believe that somebody would want to do that for a stranger,” Prodaniuk said of Neeson’s generosity, but he said it was “easy.”

“I was surprised at how easy this whole process has been,” he said. “I know that I’ve talked about how much medical testing you have to go through [but] it’s been so easy. I’m surprised more people don’t do it.”

“I always believe that we’re here to help each other.”

READ MORE: How Albertans can become living organ donors

Prodaniuk said she’s tired of “living in a prison” and can’t wait to get her life back.

“We’re going to Australia because I have a friend in Sydney and we are going to have Christmas on the beach with a barbecue.”

If you would like more information on how to become a living donor, you can contact one of Alberta’s transplant programs.

For living kidney donor services, call the Southern Alberta Transplant Program at 403-944-4635.

For information on living kidney, liver or lung donation, call the Northern Alberta Transplant Services Living Donor Program at 780-407-8698.

Dialysis News

From Senior Journal

Medicare launches new dialysis model to improve kidney care

More than 600,000 Americans have end-stage renal disease (ESRD), also known as kidney failure, and require life sustaining dialysis treatments several times per week. Medicare has a new plan. In 2012, ESRD beneficiaries comprised 1.1% of the Medicare population but accounted for an estimated 5.6% of total Medicare spending.

These individuals typically have many health problems, are at higher risk of hospital readmissions, and suffer from fragmented care.

The Centers for Medicare & Medicaid Services (CMS) has announced the participants for the Comprehensive ESRD Care (CEC) Model, a new accountable care organization (ACO) model made possible by the Affordable Care Act and conducted by the CMS Innovation Center.

CMS says it is part of the Department of Health and Human Services’ approach to building a health care delivery system that results in better care while using taxpayer dollars more wisely,

ACOs are groups of physicians and other health care providers who collectively take on responsibility for the quality and cost of care for a population of patients. The CEC Model is designed specifically for beneficiaries with ESRD and builds on experiences from other models and programs with ACOs, including the Pioneer ACO Model and the Medicare Shared Savings Program.

In the CEC Model, dialysis facilities, nephrologists, and other providers have joined together to form ESRD Seamless Care Organizations (ESCOs) to coordinate care for ESRD beneficiaries. ESCOs will be financially accountable for quality outcomes and Medicare Part A and B spending, including all spending for dialysis services, for their ESRD beneficiaries.

This model will encourage dialysis providers to think beyond their traditional roles in care delivery and support beneficiaries as they provide patient-centered care that will address beneficiaries’ health needs in and out of the dialysis facility.

“This new ACO model represents a paradigm shift in care for beneficiaries with end-stage renal disease; it promotes a patient-centered approach to their dialysis and non-dialysis care needs that will help accomplish our delivery system reform goals of better care, smarter spending, and healthier people,” said Patrick Conway, M.D., MSc, acting deputy administrator and chief medical officer, CMS.

The CEC Model includes separate financial arrangements for ESCOs with large and small dialysis organizations. ESCOs with participation by a dialysis facility or facilities owned by a large dialysis organization, which is an organization that owns 200 or more dialysis facilities, will be eligible to receive shared savings payments. They will also be liable for shared losses, and will have higher overall levels of risk compared with their smaller counterparts.

ESCOs with participation by a dialysis facility or facilities owned by a small dialysis organization, which is an organization that owns fewer than 200 dialysis facilities, will be eligible to receive shared savings payments, but will not be liable for shared losses.

The CEC Model is part of the Department’s efforts to create opportunities for providers to enter into alternative payment models and meet the Secretary’s goal, announced on January 26th, to have 30% of traditional Medicare payments paid through alternative payment models by the end of 2016 and 50% by the end of 2018.

From Med Device Online, by Jof Enriquez

FDA Clears Baxter's Web-Connected Peritoneal Dialysis System

Renal solutions company Baxter International, Inc. announced recently that the U.S. Food and Drug Administration (FDA) has granted 510(k) clearance for its AMIA automated peritoneal dialysis (APD) system with SHARESOURCE web-based remote connectivity. The AMIA device is used by patients to perform peritoneal dialysis exchanges at home, by themselves or with the help of a caregiver.

"PD is an important treatment option for many patients with kidney disease seeking home-based therapy," said Jaime Uribarri, M.D., Department of Nephrology, Mt. Sinai Hospital, NY, in the announcement. "We welcome new technology that allows patients to start and administer their physician-prescribed PD therapy at home."

Baxter collaborated with DEKA Research and Development Corporation (DEKA) in developing the AMIA APD system, which the company claims is the only device cleared in the U.S. that incorporates patient-centric features like voice guidance, a touchscreen control panel with animated graphics, and the SHARESOURCE two-way connectivity platform, which allows remote access by healthcare providers.

"Enabling patients to actively work with their healthcare providers to initiate and confidently manage their dialysis therapy is the ultimate goal in the development of AMIA with SHARESOURCE," said Bruce Culleton, M.D., VP, renal therapeutic area lead, Baxter, in the announcement. "Baxter's focus is to understand and support access to the best renal replacement options for all patients, and then bring technology like AMIA and SHARESOURCE to healthcare providers so they can help their patients achieve their best possible outcomes."

According to Baxter, it will launch the AMIA APD system with SHARESOURCE this year in the U.S., where some 600,000 individuals are living with end-stage renal disease (ESRD), which is treated with either hemodialysis, PD, or a kidney transplant.

Unlike hemodialysis, which draws out blood from the body to be cleansed by a dialyzer, peritoneal dialysis makes use of the abdominal lining — called the peritoneum — as a dialyzing membrane to cleanse the blood and clear excess fluid. Automated PD devices called cyclers utilize clamps to control the flow of fluids in and out of the abdomen. The machines are pre-programmed and the fluid exchanges are performed automatically while the patient sleeps.

Wing-walking for PKD; Donation Laws Need Changing; Adult Stem Cell Kidneys?

PKD Awareness

From GetBucks, United Kingdom

Wingwalking ex-head teacher raises nearly £2000 for charity

Barbara Smith recently raised nearly £2000 by wing walking, a present bought for her by the staff and pupils at her last school


Recently retired Barbara Smith, from Chalfont St Giles, took part in the challenge for a leading kidney disease research charity

A retired head teacher raised nearly £2,000 for a leading kidney disease research charity by taking part in a wing walking experience recently.

Babara Smith, Sylecroft Road, Chalfont St Giles, suffers from polycystic kidney disease (PKD), a genetic and hereditary disorder whereby abnormal cysts grow in the kidneys.

Staff and pupils at Selborne School, Ealing, London, got together and bought her the wing walking experience as a retirement gift, which was presented to her at her retirement party in July this year.

Mrs Smith, who was a head teacher at various schools for the past 20 years, and was teaching for the past 40, said of the flight, which she did in September: “It was scary and exhilirating.”

But added: “It was a really exciting thing to do.”

Mrs Smith highlighted the fact that there are only three and a half thousand people on the transplant list, and said that the majority of those were waiting for a new kidney, which is one of the reasons why she wanted to do this.

It was inspired, she said, after she gave a motivational assembly to some of the school children when she came up with the idea of doing another challenge, having done a number of fundraisers for the charity in the past.

“It was a motivational assembly and that was when I came up with the idea of doing another challenge,” she said.

And added: “They [the children] all think I’m totally mad, but why not? You have got to do something, haven’t you? Something you have got to enjoy. Material things, they come and go, but experiences like this are really special.”
The challenge, as mentioned in the Chalfont St Giles community email newsletter, was inspired by trips to air shows when she was younger. [Read more]

From Longview News-Journal, Longview, Texas, by Ellen Goodacre

90 turn out for kidney disease walk in Longview

About 90 participants walked the exterior loop around LeTourneau University on Saturday to help in the fight against a leading cause of kidney failure.

The walk was to raise funds and awareness for polycystic kidney disease, at the 9th Annual East Texas Walk for PKD hosted by the East Texas PKD Foundation.

"The things we really focus on are education, information from top research scientists to help the patients know the best things they can do for themselves," said Teresa Andrus, Volunteer Chapter Coordinator for the East Texas PKD Foundation. "We also really encourage people to come for support. All of us need encouragement; this is a tough disease — chronic pain and fatigue are involved for most of us."

Andrus was diagnosed with PKD at age 22 in 1968 and was part of a clinical, surgical research study, which she credits with saving her life.

Polycystic kidney disease is a genetically inherited disorder that causes clusters of cysts to develop on the kidneys, according to the Mayo Clinic website.

"My husband suffers from PKD, and he just received a transplant in June and his donor set up a booth here," 39-year-old Melissa Navarro of Tyler said. "There is not a cure, and there's a lot of people who suffer from PKD and they need donations and support from the community."

While there are treatments for PKD and lifestyle changes patients can make to manage the disease, there is no cure. PKD is the fourth leading cause of kidney failure, and about half of PKD patients develop kidney failure by age 50, according to the PKD Foundation website.

One Longview man with PKD, Roger Kulig, has had both kidneys removed and is on dialysis awaiting a transplant because of the severity of his cysts on his kidneys caused by the disease.

"People need to be aware that (PKD) could already be in their body, and there's no way to know unless you go to the doctor and designate to do a sonogram," Kulig said. "PKD is something you just don't happen to know (you have). Normally, people walking around don't know they have it until they have a pain that's unknown."

Kulig began showing symptoms of PKD at age 55 and had to have both kidneys removed after cysts on the organs had grown so large that he had trouble walking and eating.

East Texas PKD Foundation member Madalene Miller received a kidney transplant in 2008 at Memorial Hermann hospital in Houston.

"I want people to know that there is no cure and there is no real treatment, other than a transplant," Miller said. "If you have the disease in your family, there is hope and they can extend that hope by participating in some of the research studies that the foundation is sponsoring. That's why we do this walk, so we can raise more funds."

There are more than 40 clinical studies recruiting patients for PKD in the U.S., according to the PKD Foundation website, www.pkdcure.org.

From Ottumwa Post, Ottumwa, Iowa, by Pam Credille, Managing Editor

Local Family Hopes Kidney Walk will be a Step Towards Transplant

A National Kidney Foundation walk hits close to home for one local family. Approximately ten years ago, James Elementary School Principal Jay Green was diagnosed with Polycystic Kidney Disease.

“There is no cure for it, so they basically treat him for it to prolong the inevitable. The end stage is renal failure and he will need a transplant,” said Cindy Green, Jay’s wife.

On Saturday, October 10, the Southeast Iowa Kidney Walk will take place on the Indian Hills Community College campus. Registration will be open at the walk and there is no fee to enter. A free-will donation is accepted, but you do not have to donate to take part in the event.

The walk will begin at 10 a.m. outside of IHCC’s Advanced Technology Building. The walk is approximately one mile in length.

All proceeds, from the Southeast Iowa Kidney Walk, will go to the National Kidney Foundation to aid in research. None of the monies collected will go to the Greens.

“He is going to have to have a transplant soon, he is on the waiting list for a new kidney,” said Cindy Green.

Unfortunately, Cindy is not a match for Jay. However, there family is holding out hope and trusting in God that Jay will receive a compatible kidney donation.

If you would like to find out more information about becoming a kidney donor, click here.

Gift of Life

From Wales Online, United Kingdom

Mum backs changes to donation laws in Wales after her life is saved in rare, three-way organ exchange

Health worker Helen Williams is backing changes to the organ donor law in Wales after her husband gave up one of his kidneys so she could have a transplant.

A health worker whose husband gave up one of his kidneys so she could have a lifesaving transplant is backing changes to the organ donor law in Wales.

Mum Helen Williams was born with polycystic kidney disease, an inherited condition which would have resulted in kidney failure.

Her husband Craig, who works as a consultant anaesthetist at Princess of Wales Hospital in Bridgend, made the selfless decision to give her one of his kidneys to make sure that never happened.

But while they shared the same blood group, Helen’s very high antibody count meant there was a serious risk her body would reject the kidney.

So the couple took part in a rare, three-way exchange which meant Craig’s kidney was donated to a stranger and resuscitation officer Helen received another stranger’s healthy kidney in return.

Helen, who has an 11-year-old daughter called Eleri, has backed December’s introduction of a new law in Wales which means people will be regarded as having consented to organ donation after their death unless they have opted out.'Live life, love life and donate life'

Today, Assembly Members will consider exceptions where consent should still be expressed, such as body parts including the brain.

Living donors who lack the mental capacity to express a view could also be deemed to consent to donation by experts acting in their best interests.

Speaking of the changes, which come into effect on December 1, Helen said: “It’s a personal choice, but I strongly believe if you are prepared to receive a donated organ to save your life you should be prepared to give when you are dead and no longer need it.

“My motto is: live life, love life and when you go donate life." [Read more]

PKD Research

From The Australian, by John Ross

Stem cell kidneys offer new hope for organ replacement

Australian scientists have ­created tiny kidneys in a dish in a key advance towards lab-grown ­replacement organs and crash test dummy tissues for gauging drug toxicity.

The achievement, reported in the journal Nature, represents a major step forward in stem cell technology.

The researchers made headlines two years ago when they grew disc-like kidney “organoids” in the lab. But the new version is bigger and more developed, comprising all dozen or so cell types found in the kidneys of a three-month-old human foetus. The earlier ­attempt comprised just two cell types.

The new organ was produced almost entirely from “pluripotent” stem cells, which are re-engineered from adult tissue such as skin. The 2013 version was grown from embryonic stem cells.

Team leader Melissa Little said the improved attempt still lacked the structure of a real kidney, with major blood vessels and exit plumbing for urine. “It’s not a kidney, it’s a kidney model,” said Professor Little, of the Murdoch Children’s ­Research Institute in Melbourne. “But it’s a hell of a lot better than we’ve had before.”

She said the approach could be used for drug screening in as little as two years, potentially saving drug developers hundreds of millions of dollars. The kidney is one of the ­organs typically poisoned by drugs, along with the liver and heart.

The kidney is one of the organs typically poisoned by drugs, along with the liver and heart, and problems often only show up during expensive phase two and three trials. Using lab-derived organs could circumvent this, exposing toxicity issues early on.

Other possible applications include using lab-grown kidneys to identify inherited genetic mutations, or to generate reconditioned cells for injection back into patients. A longer term goal would be to grow replacement kidneys with no danger of rejection by the immune system, because they were derived from patients’ own cells — a godsend for the tens of thousands of Australian kidney disease patients who need dialysis to survive.

Professor Little said this would be an “enormous undertaking” requiring buy-in from biotechnology companies. “Even if we knew how to do it — and there’s a huge amount of additional research we’d have to do — just to build a facility of sufficient quality control and safety (would cost) tens of millions of dollars. You don’t do that on research grants.

“(But) now we now have an opportunity to make the right cell types for the patient, and in large numbers.

From MedGadget

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