Delivering Drugs to Target PKD; VEGFC and PKD; Geography Affects Dialysis Life and Death

Targeting PKD Treatments

From The Current, University of California, Santa Barbara, by Julie Cohen

Delivering Drugs to the Right Place

Renal cyst fluid contains growth factors that cause the excessive proliferation and cyst growth associated with polycystic kidney disease.

A human polycystic kidney can grow to the size of a regulation size football. A normal kidney is the size of a fist.

Thomas Weimbs has developed a targeted drug delivery method that could potentially slow the progression of polycystic kidney disease

For the 12 million people worldwide who suffer from polycystic kidney disease (PKD), an inherited disorder with no known cure, a new treatment option may be on the horizon.

PKD is a condition in which clusters of benign cysts develop within the kidneys. They vary in size, and as they accumulate more and more fluid, they can become very large. Among the common complications of PKD are high blood pressure and kidney failure.

Now, Thomas Weimbs, a professor in UC Santa Barbara’s Department of Molecular, Cellular, and Developmental Biology, has developed a novel strategy for targeting therapeutic antibodies to polycystic kidneys. He notes that the strategy opens up the possibility of repurposing a large number of existing drugs for PKD therapy.

Therapeutic antibodies — a class of biologics — are already being used extensively to treat a variety of diseases from cancer to autoimmune disorders. These biologic therapies use an antibody class called immunoglobulin-G (IgG) to bind to and prevent the activity of specific proteins or growth factors. However, in PKD, the growth factors shown to promote cyst growth reside in fluid trapped in the interior space of a cyst, called a lumen, to which IgG antibodies have no access.

Weimbs and his team have found a method that enables another class of antibodies, immunoglobulin-A (IgA), to penetrate the cyst wall. The researchers’ results appear in the Journal of Biological Chemistry. [Read more]

From Nature

VEGFC and microvascular changes in PKD

New research shows that a disorganized pericystic network of vessels expressing vascular endothelial growth factor receptor 3 (VEGFR3) is present from the early stages of polycystic kidney disease (PKD). Huang and colleagues also found that the major VEGFR3 ligand, VEGFC, was downregulated during the early stages of cystogenesis in mice…[Read more]

From Zawya, Kuwait

Researchers uncover new treatment for world's most common inherited kidney disease

A new technique for treating polycystic kidney disease has been identified by researchers based at the UCL Institute of Child Health (ICH), the research partner of Great Ormond Street Hospital. Published in the Journal of the American Society of Nephrology, the treatment, which involves targeting tiny blood and lymphatic vessels inside the kidneys, is shown to improve renal function and slow progression of disease in mice.

Polycystic kidney disease (PKD) is a genetic disorder where fluid filled cysts grow in kidneys and destroy normal renal tissue. It is the world's most common inherited kidney disease affecting between 1 in 400 and 1 in 1000 people worldwide - around 12.5 million individuals. A rarer form of the disease, which occurs in about one in every 20,000 live births in the UK, leads to a third of these babies dying before or just after birth.

Treatment for the condition has traditionally targeted proteins which are thought to play a role in causing the condition and are located in hair-like structures and tissue that line the inside of cysts. These treatments can help alleviate some of the symptoms of PKD but they can't currently cure the condition.

Researchers have now discovered that the blood and lymphatic system surrounding cysts may also be important in the development of the condition and could be a new target for treating the disease.

By looking at mouse models of both the common and rarer form of the disease, the team noticed that tiny blood vessels surrounding the cysts were altered very early in cyst development. They therefore treated the mice with a potent 'growth factor' protein called VEGFC, and found that patterns of blood vessels normalised and the function of the kidneys improved. In the mice with the rare form of the condition, it also led to a modest but significant increase in lifespan.

David Long, lead researcher and Principal Research Associate at the ICH, explains: "With further testing, treatments that target blood vessels surrounding the kidney cysts, perhaps in combination with currently used drugs, may prove to be beneficial for patients with polycystic kidney disease."

Adrian Woolf, Professor of Paediatric Science at the University of Manchester and co-author of the study added: "If we could target these blood vessels early in the development of the condition it could potentially lead to much better outcomes for patients."

Elaine Davies, Research Director at Kidney Research UK and Dr Richard Trompeter, Chairman of Kids Kidney Research, said: "This is an exciting piece of work we are extremely proud to be supporting. By identifying a treatment plan that can prevent further deterioration of kidney function in patients with this particular disease, our researchers have given fresh hope to thousands of people in the UK with this kidney condition. However, research like this can only continue with the support of the public."

Living With PKD: Dialysis

From MedicalXpress

Geography is destiny in deaths from kidney failure, study shows

The notion that geography often shapes economic and political destiny has long informed the work of economists and political scholars. Now a study led by medical scientists at Johns Hopkins reveals how geography also appears to affect the very survival of people with end-stage kidney disease in need of dialysis.

"If you are a person with kidney failure in Texas you're in trouble, but if you're in New England you're golden, and that's profoundly troubling because the quality of care shouldn't be predicated on your ZIP code," says senior investigator Mahmoud Malas, M.D., M.H.S., an associate professor of surgery at the Johns Hopkins University School of Medicine.

A report on the findings, published online June 24 in JAMA Surgery, maps out the geographic gaps in end-stage kidney care and—researchers believe for the first time—quantifies the difference in death risk stemming from such regional disparities.

Tracking nearly 465,000 people with end-stage kidney disease who started dialysis in the United States between 2006 and 2011, Malas and team found that the likelihood of getting the right kind of dialysis access ranged from 11 to 22 percent, depending on where a person lived, a disparity that added up to as much as 30 percent difference in risk of dying.

In addition, the analysis showed that no region met the target rate of treating at least half of its patients with the recommended form of dialysis, one done by creating a surgical access in the patient's arm by stitching an artery to a vein. This method, known as AV fistula, makes it far less likely that a patient will develop life-threatening bloodstream infections and blood clots.

Other forms of dialysis—those that involve accessing a patient's bloodstream through a catheter in the chest or via the abdomen—carry a notably higher risk of deadly complications, Malas and team say.

Dialysis is a life-sustaining therapy that cleans a person's blood once a patient's kidneys can no longer do so. A decade-old initiative, Fistula First, developed by professional societies and federal agencies calls for at least half of all patients with kidney failure to get their first dialysis using this approach.

Overall, New England and the Pacific Northwest had the highest scores, with one in five patients getting fistula-based dialysis. Florida, Texas and Southern California had the lowest scores, with slightly more than one in nine patients receiving fistula-based treatment. Not surprisingly, the researchers note, mortality and survival followed the same geographic pattern, with patients in the high-scoring areas having a nearly 30 percent lower risk of dying, on average, compared with those in the bottom-scoring regions.

"Dialysis with an AV fistula is superior to other methods and offers a dramatic survival advantage," says lead author Devin Zarkowsky, M.D., who conducted the research as a surgery resident at Johns Hopkins and is now a chief resident at the Dartmouth-Hitchcock Medical Center in New Hampshire. "The fact that fewer than one in five people start dialysis with a fistula is a real public health concern." [Read more]

Finding Kidneys; Dialysis Decision; Home Town Help; September 4 is PKD Awareness Day in Canada

Finding Kidneys, You Never Know

From Herald Journal, Wellsville, Utah, By Amy Macavinta

Cross-country move brings woman to her kidney donor

During the summer of 2013, Noreen Pollard and her two kids moved across the country from Mississippi to Utah. She was recently divorced, and her name had been added to a nationwide organ transplant list — it was a good time to return to Utah.

Pollard was born in Ogden and has family in the area, but she said she felt drawn to Wellsville, which worked out nicely because her daughter was able to transfer her studies to Utah State University.

However, when she moved into her cozy little home on Center Street, she never dreamed her future kidney donor lived just two doors down.

“I think we were led here,” she said.

Now 45 years old, Pollard was diagnosed at 32 with polycystic kidney disease, an inherited disorder that causes non-cancerous cysts to develop inside the kidneys, interfering with the body’s ability to dispose of toxins and eventually leading to kidney failure.

By the time she moved to Utah, Pollard was experiencing a near-constant pain, especially in her right kidney. She described it as a dull, throbbing ache with periodic sharp stabs. Her blood pressure was elevated, she was tired all the time, and she said she often lived in a fog she couldn’t quite describe — all common responses to the toxins in the body.

For two and a half years, Pollard hooked herself up to a machine every night at bedtime for peritoneal dialysis, a process that helped her body get rid of those toxins while she slept.

In November, the congregation in Pollard’s LDS ward held a special fast for her, but two days later, Pollard said she learned her liver was not looking good, and doctors wanted to do a biopsy — putting her kidney opportunities on hold temporarily.

Pollard’s kidney donor, Heidi Gordon of Wellsville, says she is fortunate — she has the ability to stay home and care for her family.  [Read more]

Dallas Morning News, Dallas, Texas, By MARC RAMIREZ

Shared kidney ailment brings Arlington dad, son closer

A quarter-century apart, Moses Cabrera and son Moses Jr. had kidney transplants, performed by the same surgeon. This Father’s Day, the two will toast each other’s health — and recall the health scares that brought them closer together.

They share the name Moses, and from there the similarities just keep going: They dress alike, have similar parenting styles and when prompted by a memory, they’ll both break into song.

They’re men of few words and great faith. And a quarter-century apart, Moses Cabrera and son Moses Cabrera Jr. both had kidney transplants, performed by the same surgeon.

This Father’s Day, the two will toast each other’s health — and recall the health scares that brought them closer together.

“It’s just amazing that we both had to go through this,” says the elder Cabrera, a retired postal carrier. “But God has been good to us.”

Both have been separately diagnosed with polycystic kidney disease, a mostly hereditary condition that causes cysts to form around the kidney, limiting and eventually destroying its function. It affects about 500,000 people nationwide.

The two Cabreras, like gym partners, encourage each other to pursue healthy habits, take their medications and stay the course.

“Not a day goes by that it’s not in my head,” Cabrera Jr. said. “We ask each other, ‘How you feeling?’ Or ‘Are you drinking plenty of water?’”

The experience has given them even more in common, a relationship that amazes Cabrera’s wife, Mary Ann.

“I always tell Moses — that’s not his son, that’s his clone.”
Family trait

In the mid-1950s, Cabrera’s own father died of kidney failure at age 43, but little was known about the disease, known as PKD, and its genetic nature.

As a result, Cabrera, now 70, said he lived carelessly, even as his brother and sister succumbed to the disease.

He rarely checked his blood pressure. At work, he started blacking out for a split second — and then he’d gather himself.

When the dizzy spells started, wife Mary Ann made him go to the doctor. Blood tests told the story: He had PKD. [Read more]

Kidney Dialysis

From KLS, Salt Lake City, Utah, By Suzanne Carlile

Making the dialysis decision: What you need to know

Hundreds of thousands of Americans with kidney failure undergo dialysis treatments to keep them alive. While their reasons for needing the treatment can vary widely, many patients with kidney failure ultimately face the same dialysis decision.

Here's what you need to know:


What is dialysis?

When your kidneys fail, your body cannot filter toxins, regulate body fluids, or sustain electrolyte balance vital to survival. Dialysis takes the place of kidney function by cleaning a person's blood.

The first successful dialysis machine, or “artificial kidney,” was invented in 1945 by Willem Johan Kolff, then a doctoral student at the University of Groningen in Holland. Today, about 450,000 Americans are on dialysis,according to the National Kidney Foundation.

Dialysis is used as a bridge to kidney transplant, in recovery from acute kidney failure, or for an end-of-life, life-sustaining purpose. In general, life expectancy after starting dialysis is five years.


Who gets dialysis treatment?
The No. 1 reason a person needs dialysis treatment is kidney failure due to diabetes. The disease is responsible for 44 percent of all dialysis patients’ kidney failure, according to the U.S. Renal Data System's 2013 Annual Data Report.

The report names the second-largest group of dialysis patients, at 28 percent, as people with kidney failure due to high blood pressure. Other conditions often leading to dialysis treatment are glomerulonephritis, polycystic disease, kidney inflammation, genetics, auto-immune disease, drug use, etc.

Signs and symptoms of kidney failure are often hard to pinpoint because they mimic multiple other medical conditions. For early detection, lab testing should be part of your yearly physical. By analyzing a person's blood urea nitrogen and creatinine levels, a doctor will be able to determine the level of kidney function.

Patients who have lost more than 75 percent of their kidney function are often referred for dialysis treatment. The worse your kidney function is, the more complications you will have.

The decision to undergo dialysis treatments is the first and most important discussion you will have to make with your health care provider, and you should understand doing so will complicate any other medical problems you have.


Realities of dialysis treatment

It’s important to note dialysis is not a cure for kidney failure. Dialysis is a life-support treatment — without functioning kidneys you cannot live. [Read more]

Home Town Help

Enid News, Oklahoma, by Cass Rains

Golf tournament to aid woman to raise funds for kidney transplant

Registration still is open for the Spare Parts Golf Tournament next month to help an Enid woman raise funds for a kidney transplant.

Four years ago, Melissa Thomas was diagnosed with polycystic kidney disease and told she would need a kidney transplant.

The disease causes the kidneys to swell, and because the kidneys control so much in the rest of the body, it causes multiple complications, such as liver cysts, heart problems and brain aneurysms.

Last July, Thomas was referred for a transplant. Because polycystic kidney disease, or PKD, has no cure, it is the only way to save her life.

Before she can receive a transplant, Thomas must raise $20,000.

The costs mostly are associated with two months of medication, totaling about $12,000, and for a four-week stay in a post-surgery recovery facility.

The tournament is set for July 11 at Meadowlake Golf Course. Registration is $75 for individuals, with $40 tax deductible, and $300 for a foursome, with $160 tax-deductible. Hole sponsorships are available for $150 and are entirely tax-deductible.

Registration can be done at bit.ly/ sparepartsgolftournament.

Donations also can be made by visiting Thomas’ HelpHopeLive campaign at m.helph opelive.org/campaign/6162.



From WSBT Channel 22, CBS Affiliate Elkhart, Indiana, by Zach Crenshaw

Local family uses moving billboard to search for kidney

A local family with a history of kidney disease is going to great lengths to try and find one.

The dialysis machine casts a perpetual shadow over Kim Lara’s life.  “Basically, when I started dialysis, my life stopped,” she said.  Every four hours she has to be home to plug in for treatment.

“I can’t do anything, I can’t go anywhere.”

The reason for the pain is Polycystic Kidney Disease, which Kim has had since she was 11 years old.

“I was one of the youngest ever diagnosed,” she said.  In December, she had to have one of her enlarged kidneys removed.  “It was the largest one they had ever seen,” Kim said. “A normal kidney weights about five ounces, and mine weighed right about 10 pounds when it was in me and I still have the other kidney and it’s just as large.”

The removed kidney was so large the doctor actually submitted it to the Guiness World Book of Records.

With stage 5 renal failure, Kim needs a transplant to survive. Her family has been disqualified, including her husband who was a perfect match.  “He went through all the testing and everything was going great. Then at the last test they found out he had a heart valve problem,” Kim said.

After being ruled out, Kim’s family came up with another plan.  “We put a billboard on our cars. It says ‘Need a kidney for my daughter, my sister, my mom, my wife’ – depending on whose car it is,” said Brenda Richards, Kim’s mom.

Their moving billboards are an outward expression of their love for Kim.

“Wherever our cars have been, [the hospital has] gotten phone calls wanting to know more information,” said Richards.  Kim’s mom and daughter have the same disease.  “My brother donated to me nine years ago and he said it was the greatest experience of his entire life,” said Brenda.

But now, Kim’s mom can only watch as her daughter waits in pain.  "It's just heartbreaking because you feel helpless, because I don't have any of my kidneys."  Strong in her faith, Kim continues to hook up to her machine every four hours, but she's hoping her signs will be a sign for someone else to come forward and change her life again.

"I'll get my life back. I'll be able to take a walk with my husband."

Kim's blood type is A positive, so all 'A' and 'O' blood types are matches for her.  If you're interested in donating on behalf of Kim, call Lutheran Hospital at 260-435-7209. If you would like to donate privately, contact zcrenshaw@wsbt.com to get in contact with the family.

You can learn more about Kim and her journey through her Facebook page, Kidney Search for Kim.

From Journal Star,Lincoln, Nebraska

Spaghetti feed to benefit Hickman man with kidney disease

A spaghetti dinner and raffle Saturday from 4 to 8 p.m. at the American Legion Club in Hickman will benefit Hickman resident Curtis Rust, who suffers from polycystic kidney disease and needs a kidney transplant.

Rust, who is hooked up to dialysis at night and works during the day, also has heart disease.

An offering at the door will cover spaghetti, garlic bread, dessert and a beverage. Donations may also be made at Union Bank and Trust or at www.gofundme.com/nnfg4c.

PKD Awareness

From York Region, York, Ontario, Canada, by Simone Joseph

Battling rare kidney disease family affair

Growing up, Jeff Robertson knew his mother was being held captive by a powerful force.

From an early age, he witnessed her struggles.  He was born on Oct. 28, 1981 to Jan Robertson.

Not long after, her liver enlarged to 40 pounds because of cysts.  “She looked nine months pregnant for the first 15 years of my life,” Robertson said.  ‘It is a great step forward. Prior to that, there were no options available.’  In part, because of the weight she was already carrying, she often couldn’t pick up her two young children.

The Richmond Hill resident was diagnosed with polycystic kidney disease after having her first child — Robertson’s sister — in 1979.  “Little was known in the medical community about the disease and we didn’t know anyone who had it,” said Robertson, now 33.

Polycystic kidney disease affects an estimated one in 500 people. Kidneys are usually the most severely affected organs, but the disease can cause cysts to develop in your liver and elsewhere in the body.

Growing up, Robertson did his best to help fight his mother’s disease.  His school projects usually focused on the genetics of polycystic kidney disease.

He sold bags of peanuts and candy to benefit first the Kidney Foundation of Canada and later the foundation his mother created — the Polycystic Kidney Disease Foundation of Canada in 1993.

Robertson has been executive director of the foundation since June 2008.  The foundation promotes research, advocacy and education programs in the hopes of discovering treatments and a cure for polycystic kidney disease and to improve the lives of all it affects.  This past year has been a big boost for the cause.

In 2014, Health Canada recognized Sept. 4 as National Polycystic Kidney Disease Awareness Day.

At the end of February of this year, Health Canada approved an oral medication, called Jinarc, to help adult patients combat the disease. It slows progression of kidney enlargement and protects kidneys from damage and failure.

“It is a great step forward. Prior to that, there were no options available,” Robertson said.

Three and a half years ago, Jeff Robertson made his own important discovery.  He and his sister were genetically tested for polycystic kidney disease. Neither carries the gene.  Meanwhile, the foundation is looking to set up its first York Region chapter, possibly this year, in Richmond Hill, where Ms Robertson still lives.

Robertson feels his work at the foundation is making a difference.  “It (awareness) gets better every year,” he said.  But he wants to emphasize that at the moment, there is no cure for this disease.

He is still hoping for more awareness of the disease and the foundation.  “We want people to know we are here and ready to help in any way possible”.

To that end, he is walk co-ordinator for the 2015 Toronto PKD fundraising walk, with registration opening June 1.

Go to endpkd.ca for more info.

PKD Research

From University of Groningen, Netherlands, Research Database

Urine and Plasma Osmolality in Patients with Autosomal Dominant Polycystic Kidney Disease: Reliable Indicators of Vasopressin Activity and Disease Prognosis?

Vasopressin plays an essential role in osmoregulation, but has deleterious effects in patients with ADPKD. Increased water intake to suppress vasopressin activity has been suggested as a potential renoprotective strategy. This study investigated whether urine and plasma osmolality can be used as reflection of vasopressin activity in ADPKD patients. Methods: We measured urine and plasma osmolality, plasma copeptin concentration, total kidney volume (TKV, by MRI) and GFR (I-125-iothalamate). In addition, change in estimated GFR (eGFR) during follow-up was assessed. Results: Ninety-four patients with ADPKD were included (56 males, age 40 +/- 10, mGFR 77 +/- 32 ml/min/1.73 m(2), TKV 1.55 (0.99-2.40) l. Urine osmolality, plasma osmolality and copeptin concentration were 420 +/- 195, 289 +/- 7 mOsmol/l and 7.3 (3.2-14.6) pmol/l, respectively. Plasma osmolality was associated with copeptin concentration (R = 0.54, p <0.001), whereas urine osmolality was not (p = 0.4). In addition, urine osmolality was not associated with TKV (p = 0.3), in contrast to plasma osmolality (R = 0.52, p <0.001) and copeptin concentration (R = 0.61, p <0.001). Fifty-five patients were followed for 2.8 +/- 0.8 years. Baseline plasma and urine osmolality were not associated with change in eGFR (p = 0.6 and p = 0.3, respectively), whereas baseline copeptin concentration did show an association with change in eGFR, in a crude analysis (St. beta = -0.41, p = 0.003) and also after adjustment for age, sex and TKV (St. beta = -0.23, p = 0.05). Conclusions: These data suggest that neither urine nor plasma osmolality are valid measures to identify ADPKD patients that may benefit from increasing water intake. Copeptin appears a better alternative for this purpose.

Slowing Progression of PKD; Protecting Your Heart; Salvation Army Donation: Exercise & Dialysis

PKD Research

From News Medical

New technique slows progression of polycystic kidney disease in mice

A new technique for treating polycystic kidney disease has been identified by researchers based at the UCL Institute of Child Health. Published in the Journal of the American Society of Nephrology, the treatment, which involves targeting tiny blood and lymphatic vessels inside the kidneys, is shown to improve renal function and slow progression of disease in mice.

Polycystic kidney disease (PKD) is a genetic disorder where fluid filled cysts grow in kidneys and destroy normal renal tissue. It is the world’s most common inherited kidney disease affecting between 1 in 400 and 1 in 1000 people worldwide – around 12.5 million individuals. A rarer form of the disease, which occurs in about one in every 20,000 live births in the UK, leads to a third of these babies dying before or just after birth.

Treatment for the condition has traditionally targeted proteins which are thought to play a role in causing the condition and are located in hair-like structures and tissue that line the inside of cysts. These treatments can help alleviate some of the symptoms of PKD but they can’t currently cure the condition.

Researchers have now discovered that the blood and lymphatic system surrounding cysts may also be important in the development of the condition and could be a new target for treating the disease.

By looking at mouse models of both the common and rarer form of the disease, the team noticed that tiny blood vessels surrounding the cysts were altered very early in cyst development. They therefore treated the mice with a potent ‘growth factor’ protein called VEGFC, and found that patterns of blood vessels normalised and the function of the kidneys improved. In the mice with the rare form of the condition, it also led to a modest but significant increase in lifespan.

David Long, lead researcher and Principal Research Associate at the ICH, explains:


"With further testing, treatments that target blood vessels surrounding the kidney cysts, perhaps in combination with currently used drugs, may prove to be beneficial for patients with polycystic kidney disease." [Read more]

From WFMZ, Channel 69 News HealthBeat, by Nancy Werteen

Health Beat: Kidney patients: Protecting your heart

ATLANTA - David MacKenzie gets a workout most days, even if it's just a quick, brisk walk. He's tried to maintain a healthy lifestyle for most of his adult life. "Early on, probably in my mid to late-30s, I began to have elevated blood pressure," said MacKenzie, now 66. MacKenzie didn’t know it growing up, but he and one of his sisters would also develop polycystic kidney disease, an inherited condition that affects kidney function.

"Even though it's adaptive to have this fight or flight response in the situation when you need it, if it's revved up all the time, then it's not good for your body," explained Dr. Jeanie Park, assistant professor at Emory University School of Medicine in Atlanta. Park found that a drug already FDA-approved for a metabolic disorder dials down the adrenaline levels in kidney patients. It's called tetrahydrobiopterin. Park studied 32 men with moderate kidney disease and found a decrease in sympathetic nerve activity in those who took the drug for 12 weeks. "It could be a novel way of reducing cardiovascular risk and potentially reducing blood pressure in patients with hypertension or chronic kidney disease," Park said. For patients like MacKenzie, that would mean managing just one chronic condition, kidney disease, instead of two. Right now, doctors use beta blockers and another drug, clonidine, to treat high blood pressure and the over-activation in the sympathetic nervous system. Park said those drugs are often hard for patients to tolerate. [Read more]

Living With PKD

From MedScape, by Pam Harrison

Exercise During Dialysis Delivers Major Gains

LONDON, United Kingdom — A personalized exercise program integrated into routine dialysis can improve strength, endurance, and quality of life, new research indicates.

Fitness "improved significantly over a 1-year period, after which patients stabilized," said Kirsten Anding-Rost, MD, from the KfH Dialysis Center in Bischofswerda, Germany.

The program "was a really big success," she explained. "Normally in dialysis patients, health status steadily declines, so if you do something that maintains health status, it's actually very good."

Dr Anding-Rost presented results from the study here at the European Renal Association–European Dialysis and Transplant Association 52nd Congress.

The 46 study participants were typical dialysis patients. Mean age was 63 years, there were a lot of comorbidities, and three patients had undergone leg amputation.

The exercise program consisted of 30 minutes of combined resistance training that worked eight major muscle groups and 30 minutes of endurance training in the supine position on a bicycle ergometer.

Patients trained two times a week for 5 years, and were assessed with maximum strength testing and mean cycling power per training session.

"To adapt the training intensity to the personal fitness of each patient, we had to first measure the personal fitness of each patient," Dr Anding-Rost explained. And throughout the study period, exercise intensity was continuously adjusted to match improvements in fitness.

Of the initial 46 participants, 36 completed 1 year of the study and 20 completed 5 years.

Improvements in strength depended on how adherent patients were to their training sessions. For those with high adherence, improvements were significant in all eight muscle groups targeted by the resistance exercises.

In the moderate-adherence group, "we still saw an improvement in some muscle groups, but it was not as good as in the high-adherence group," Dr Anding-Rost reported.

There was a "nice improvement" in the average cycling power gained during the first 3 months of the exercise program, she said. After that, patient endurance remained more or less stable.

During the first 3 months, there was a 55% improvement in maximum exercise capacity in the high-adherence group and a 45% improvement in the moderate-adherence group.

There were also significant improvements on all three tests of physical function and in quality of life, ranging from 11% to 31%.

Physically weak patients actually had greater improvements in physical function than stronger patients.

Gift of Life

From KDKA, CBS Affiliate Pittsburgh, by Brenda Waters

Salvation Army Employee Donates Kidney To Co-Worker

Thousands of people die every year waiting for a kidney transplant, but a minister at the Salvation Army is making sure her co-worker doesn’t end up on that list.

Michael Riemer, Director of Emergency Disaster Services at The Salvation Army’s Western Pennsylvania Division, is to receive a kidney Tuesday from co-worker Kate Esker.

“It’s the biggest day of my life, because I get a second chance at life,” Riemer tells KDKA’s Brenda Waters.

Riemer, 55, found out in his mid-20’s that he was diagnosed with polycystic kidney disease (PKD), a genetic disorder that took the lives of his mother, grandmother, and great-grandmother.

Riemer says PKD’s progression is long and slow, and remained private about his diagnosis for 30 years.

He has devoted his life to helping others. Riemer served Mt. Lebanon as a police officer for 25 years, and currently oversees disaster relief for The Salvation Army’s Western PA Division’s 28 counties.

When Allegheny General Hospital proposed to Riemer several months ago to go public in order to find potential donors, Esker, Core Officer and minister at McKeesport’s Salvation Army Worship and Service Center, immediately wished to help.

“I told my husband on the way home I wanted to see if I was a match,” says Esker. “I know Mike pretty well, and I like the life that he lives… there is so much life ahead of him.”

Riemer says he is forever indebted to Esker and her family.

“How do you begin to say thank you to someone who’s given you life a second time over? There’s not enough words; there’s not enough money in the world for this gift, a second time.”

Esker is “more excited than nervous.” She and Riemer would encourage anyone who knows someone who needs a kidney to check if they are a fit.

From WTSP, CBS Affiliate Tampa Bay, by Bobby Lewis

Friends' kidney swap will save them and strangers

It's one thing to loan a friend some money or a book but you have to have a special bond to offer up a body part.

"She's outstanding," said Subash Vajja. "She started saying, 'I'm going to give you a kidney,' and she never changed her mind. She stick with what she say."

Vajja and Beth Dillon, one of his wife's former co-workers, have become so close that they consider each other family. Vajja has been waiting a year and a half for a new kidney and Dillon didn't hesitate to give up hers for her friend.

The only problem was, the two weren't a match. They did a lot of research and found a Paired Kidney Exchange option – an opportunity to give and receive kidneys for those who are facing illness. Vajja has been fighting Polycystic Kidney Disease.

They went through all of the tests. Even after finding out her kidney wouldn't match Vajja's needs, Dillon still wanted to give up her organ as a way to honor her uncle.

"He was never healthy enough to receive a kidney," she said. "He passed away almost two years ago. June 13, 2013."

Now, two years later, on June 12, 2015, Vajja and Dillon are both having surgery to save and be saved by strangers.

Both will travel to Birmingham to have surgery. They are part of an 11-person kidney swap group that will all have surgery on Friday.

"I'm going to tease the doctor and say, 'Since you're in there, can you do a little lypo or something?'," joked Dillon. "I think it will just enlarge our family because we're all going to be touched by each other and I think it's going to be a life-long connection with people."

From Brookings Register, Brookings, South Dakota, by Eric Sandbulte

A 10-way gift of life

Brookings resident Greg Enz participated in a kidney exchange program at the Mayo Clinic in Rochester, Minn., in March so that his best friend’s mother could receive a lifesaving kidney donation herself. About 10 people were involved in the exchange program at the time, donors and recipients included. Pictured are Greg Enz at right, Kathy Allen at center and her son and Enz’s friend Ethan Allen at left.

“Greg is an absolutely wonderful person. He is just so generous and selfless, and myself and my family are just so grateful to him. He’s just a gem.”

Those are the words of Kathy Allen, who received a kidney transplant thanks to the generosity of Greg Enz and the Mayo Clinic’s kidney exchange program.

Enz, who works as a correctional officer at the Brookings County Detention Center, is a lifelong friend to Allen’s son, Ethan, ever since they met in the seventh grade. But Allen was diagnosed roughly 30 years ago with polycystic kidney disease (PKD), a common but life-threatening genetic kidney disease.

PKD Research: New Treatment shows promise; Jinarc (tolvaptan) now available in Canada; Schedule that Walk!

PKD Research

From Nephrology News

New polycystic kidney disease treatment method shows promise in mouse study

A new treatment that involves targeting blood and lymphatic vessels inside the kidneys has improved renal function and slowed the progression of polycystic kidney disease in mice, according to a study published in the Journal of the American Society of Nephrology.

Treatment for polycystic kidney disease has traditionally targeted proteins that are thought to play a role in causing the condition and are located in hair-like structures and tissue that line the inside of cysts. These treatments can help alleviate some of the symptoms of PKD but they can't cure the condition.

Researchers have now discovered that the blood and lymphatic system surrounding cysts may also be important in the development of the condition and could be a new target for treating the disease

"If we could target these blood vessels early in the development of the condition it could potentially lead to much better outcomes for patients," said Adrian Woolf, Professor of Paediatric Science at the University of Manchester and co-author of the study.

By looking at mouse models of both the common and rarer form of the disease, the team noticed that tiny blood vessels surrounding the cysts were altered very early in cyst development. They treated the mice with a potent 'growth factor' protein called VEGFC, and found that patterns of blood vessels normalized and the function of the kidneys improved. In the mice with the rare form of the condition, it also led to a modest but significant increase in lifespan.

"With further testing, treatments that target blood vessels surrounding the kidney cysts, perhaps in combination with currently used drugs, may prove to be beneficial for patients with polycystic kidney disease," said David Long, lead researcher and Principal Research Associate at the ICH.

From Business Wire, Press Release

Critical Path Institute Secures Regulatory Support for Autosomal Dominant Polycystic Kidney Disease (ADPKD) Biomarker

TUCSON, Ariz.--(BUSINESS WIRE)--The Critical Path Institute (C-Path) announced today that the U.S. Food and Drug Administration (FDA) has issued a Letter of Support to C-Path’s Polycystic Kidney Disease Outcomes Consortium (PKDOC) for the use of 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 is a debilitating genetic disease affecting more than 600,000 Americans and 12 million people worldwide. It is characterized by progressive enlargement of the kidneys due to the formation and growth of cysts. TKV is a measurement of the impact of ADPKD on the size of the kidneys and is believed to be predictive of a future decline in kidney function.

This Letter of Support is intended to encourage the use of TKV as an exploratory prognostic biomarker in clinical studies to identify patients likely to experience a progressive decline in renal function. “This represents a very significant milestone in the PKDOC’s continuing effort to qualify TKV as a prognostic biomarker,” said Martha Brumfield, PhD, President and Chief Executive Officer of C-Path. “It also constitutes a significant endorsement of the innovative consensus science model that C-Path has helped foster for the past 10 years.”

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

David Baron, PhD, Chief Scientific Officer of the PKD Foundation, said, “As a patient advocate, I believe this is important because it shows that the FDA acknowledges variances in the disease progression of ADPKD patients. This also encourages drug companies to investigate potential treatments for ADPKD that can be used earlier in the progression of the disease.” [Read more]

From National Institutes of Health, U.S. National Library of Medicine

Molecular genetic diagnosis of autosomal dominant polycystic kidney disease in a newborn with bilateral cystic kidneys detected prenatally and multiple skeletal malformations.

Abstract

We report a case of an unusual prenatal presentation of polycystic kidneys associated with multiple skeletal limb defects, including polydactyly, syndactyly, bilateral agenesis of the tibia, and club foot. The ultrasonographic picture was consistent with a diagnosis of polycystic kidney disease, either the adult onset autosomal dominant type (ADPKD) or the early onset autosomal recessive form (ARPKD). However, there was a positive family history for ADPKD. Linkage analysis was performed in 10 family members, of whom four were affected, using six flanking DNA markers tightly linked to the PKD1 locus on chromosome 16p, and one marker linked to the putative PKD2 locus on chromosome 2p. Lod score determinations indicated that the affected gene in the family is most likely PKD1. The patient inherited the disease linked haplotype from his affected mother.

PKD Treatment

From CNW, Canadian News Wire, Press Release

Now Available in Canada: First-ever treatment for adults living with a life-threatening kidney disease (ADPKD)

On February 25, 2015, Health Canada approved JINARC™ (tolvaptan) as the first pharmaceutical treatment available in Canada for patients with autosomal dominant polycystic kidney disease (ADPKD). JINARC™ was discovered in Japan by Otsuka Pharmaceutical and was first approved there for the treatment of ADPKD in 2014.

Effective today, JINARC™ is now commercially available for use in Canada.

JINARC™ slows the progression of kidney enlargement in patients with ADPKD, which should help protect the kidneys from damage and failure.

MONTREAL, QC, June 3, 2015 /CNW/ - Today, Otsuka Canada Pharmaceutical Inc. announced that JINARC™ (tolvaptan), is now commercially available for use in Canada. JINARC™ is indicated to slow the progression of kidney enlargement in adults with autosomal dominant polycystic kidney disease (ADPKD).

"ADPKD can have a significant impact on a person's health and quality of life, and often afflicts many individuals in a family across the generations. Ultimately it leads to kidney failure and the need for kidney transplant or dialysis in most people affected by the disease," says Dr. Phil McFarlane, nephrologist from Toronto. "With the availability of JINARC™, Canadians living with ADPKD and the clinicians who care for them now have an option that can potentially slow cyst growth, delay disease progression, and improve symptom control."

Approximately, half of polycystic kidney disease (PKD) patients reach end stage renal disease (ESRD) and require renal replacement therapy in the form of dialysis or a kidney transplant by age 54.1 According to a recent Canadian survey, the majority of ADPKD patients say the disease has impacted their ability to: complete everyday activities, such as working or spending time with family (66 per cent); travel and go on vacation (58 per cent); and lead a healthy and active lifestyle (56 per cent).2 Furthermore, on average, two-thirds (67 per cent) of ADPKD patients have other family members who have been diagnosed with the disease.*

"When I was first diagnosed with ADPKD 14 years ago, it was hard for me to accept that there weren't any treatment options that could slow the progression of my disease," says Cheri Barton, Ottawa, ON. "With the availability of JINARC™, there's finally hope for our family and for the PKD community." [Read more]

Gift of Life

From Canada AM

'My heart made the choice': Woman donates kidney to save life of toddler

A two-year-old boy from Sudbury, Ont. is recovering after receiving a kidney from an organ donor. Now that donor is sharing her own story of losing her own son to kidney disease, and what ultimately compelled her to give the gift of life.

Late last year, Desiree and Darren Lamoureux made a plea for their son Taylum, who was born with a rare form of polycystic kidney disease. Only one in four babies with Taylum's condition survive.

Because of the disease, Taylum required daily dialysis treatment in hospital. This forced Desiree Lamoureux and Taylum to move to Toronto so he could receive treatment, while his father remained in Sudbury to continue working as an electrician.

Last December, the Ontario Toyota Dealers Wishmaker campaign gave the family a cheque for $25,000 and a new Toyota Corolla. But it was ultimately another mother who had gone through a similar experience that would end up giving the biggest gift of all.

Donor Michelle MacKinnon's son David also suffered from kidney disease. When he became a teenager, MacKinnon and her husband decided to get tested to see if they would be a match for donation.

It was determined through testing that she was a perfect match for her son. However, days before the scheduled surgery, David's condition worsened.

"Unfortunately, David had a stroke three days prior to our transplant date," she told CTV’s Canada AM. "He died on the day I was to give him my kidney."

David died in June 2011. On that very day, MacKinnon said she pulled the doctors aside and told them that she would still like to proceed with organ donation to a family in need.

Years later, MacKinnon came across a Facebook post detailing the Lamoureuxs' story. When she read about their ordeal, MacKinnon said she knew she wanted to help.

"I found the post on Facebook completely by accident. (It was) Desiree's plea for a kidney. I thought, 'Maybe that's what I am supposed to do,'" she said.

So MacKinnon messaged Desiree Lamoureux about the possibility of donating her kidney to Taylum, and on May 13, she and the toddler underwent transplant surgery. Both are now recovering. [Read more]

From KDNO/KDNU NBC Affiliate, Pasco, Washington

Random Act of Kindness: Stranger Donating Kidney to Help Save Local Man's Life

PASCO, WA. -- All week we've been keeping track of your random acts of kindness for Random Acts of Kindness Week in the Tri-Cities. And we've found perhaps, the ultimate random act of kindness.

Honestly we don't know if you can find a better random act of kindness than this.

On Thursday, we met with Darla and Tanaka Modoc in their Pasco home. They called us after hearing about Random Acts of Kindness Week in the Tri-Cities and the story they told us, is simply amazing.

Tanaka for the last 3-years has been forced to do dialysis several times every week because of a hereditary disease called Polycystic Kidney Disease. And get this, later this month, Tanaka will be receiving a kidney from a complete stranger.

Tanaka met Vaughn Jensen through a friend and he offered to go through years of tests and even lost a ton of weight just to be eligible for the surgery. Tanaka and Darla can hardly believe that this stranger is willing to help save Tanaka's life.

"It was like a miracle, he said, "I'm going to donate a kidney to you," just like that," Tanaka Modoc said. "And he kept saying it and saying it and saying it. I got goose bumps."

"It's amazing what he's doing," Darla Jensen said. "I'm very grateful to him. I will never be able to repay him for what he's given my family."

Tanaka says to celebrate the new kidney, he and Vaughn plan to chug a huge bottle of water after the hopefully successful surgery. Darla and Tanaka reached out to us to publicly tell Vaughn a big "thank you" for everything he's done for them.

They want to help bring awareness for the shortage of live kidney donors needed around the country.

And they also could use your help. They're fundraising through Go Fund Me to help pay for Vaughn's medical expenses. If you want to help out, click here.

 

PKD Chapter Events

From PKD Foundation of Canada

Sun. June 14 2015 – Toronto Chapter Meeting: Genetic and Clinical Aspects of Autosomal Dominant Polycystic Kidney Disease

Dr. David Chitayat, MD, FABMG, FACMG, FCCMG, FRCPC is a Professor and Medical Director for both Mount Sinai Hospital and SickKids Hospital. During this informational presentation, Dr. Chitayat will discuss the genetic aspects of autosomal dominant polycystic kidney disease (ADPKD), prenatal and pre-implantation genetic diagnosis, as well as the clinical manifestations and treatment of ADPKD. [Read more]

From PKD Foundation of Canada

2015 Montreal Marche pour PKR / Walk for PKD

September 12, Montreal, Quebec. We are volunteers, friends, family and co-workers walking to END PKD!

We are proud to announce that Québec will have its first WALK FOR PKD on Saturday, September 12, 2015!!! A walk to support people affected by this hereditary disease, for which there is no cure.

Accompanied by your family, friends, or your colleagues, come join us for a morning full of fun, music, and complimentary food. In a beautiful environment located in the heart of Dollard des Ormeaux, Centennial Park on the island of Montreal is the ideal location for our event.

On-site registration will commence from 9:00am-10:00am, with announcements and the Walk starting at 10:00am sharp!

We hope to see you there!

From PKD Foundation of Canada

2015 Vankleek Hill Walk for PKD

September 13, Vankleek Hill, Ontario. Join us as we Walk for PKD in the village of Vankleek Hill, ON only 1 hour from Ottawa or Montreal. Come early to enjoy lunch at the annual ‘Festival of Flavours’ taking place on Main Street, and visit Beau’s All Natural Brewery for a sample of some award winning beer! Our 4km walk will take us straight through the Festival and through our picturesque town on the hill. Stay after the walk for some homemade snacks and drinks. We can’t wait to welcome you to our small town.