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.

Jinarc (tolvaptan) Approved in Europe; Cooking for a Cure; Social Media Help; Be a Hero

PKD Treatment

From PharmiWeb, Press Release

OTSUKA’S JINARC® THE FIRST-EVER TREATMENT APPROVED IN EUROPE FOR ADULTS LIVING WITH ADPKD A CHRONIC GENETIC KIDNEY DISEASE

Otsuka Pharmaceutical Co. Ltd. announced today that the European Commission has granted marketing authorisation for JINARC® (tolvaptan) for the treatment of ADPKD in adults who have chronic kidney disease (CKD) stage one to three at initiation of treatment with evidence of rapidly progressing disease. In receiving this marketing authorisation tolvaptan becomes the first pharmaceutical therapy to be licensed in Europe for the treatment of the underlying pathophysiology of ADPKD.

“Until now healthcare professionals have focused on treating the signs and symptoms of ADPKD with no specific treatment available to treat the disease” said Professor Ron T. Gansevoort University Medical Centre Groningen the Netherlands an expert in the field of polycystic kidney disease. “Tolvaptan represents a significant medical breakthrough in the management of ADPKD. For the first time healthcare professionals can modify the progression of the disease and preserve kidney function with the potential to improve patients’ quality of life and long-term outcomes.”

The marketing authorisation for tolvaptan is based on the findings of the pivotal Phase III randomised double-blind and placebo-controlled TEMPO 3:4 trial – the largest clinical study conducted in ADPKD to date.1 In the three-year study the rate of TKV increase over 3 years was significantly less for tolvaptan-treated subjects than for subjects receiving placebo: 2.80% per year vs 5.51% per year respectively (ratio of geometric mean 0.974; 95% CI 0.969 to 0.980; p <0.0001); these data demonstrate an approximate 50% significant reduction in the annual increase in TKV versus placebo.3 Furthermore tolvaptan showed a statistically significant reduction in the risk of multiple events of worsening kidney function kidney pain hypertension or albuminuria (hazard ratio=0.87 95% CI: 0.78-0.97 p=0.0095).3 The result of the key secondary composite endpoint is primarily attributed to effects on worsening kidney function (61.4% less likely with tolvaptan than with placebo) and medically significant kidney pain (35.8% less likely in tolvaptan-treated patients).3

Other than side effects associated with the mechanism of action of tolvaptan (eg thirst polyuria polliakuria) most side effects observed in ADPKD patients administered tolvaptan were comparable with those administered placebo.1 However a risk of liver injury was identified in patients with ADPKD taking tolvaptan. Elevation of alanine transaminase (ALT) was observed in 4.4% of patients on tolvaptan and 1.0% of patients on placebo.3 Two (2/957 0.2%) tolvaptan treated-patients as well as a third patient from an extension open label trial exhibited clinically significant increases in ALT with concomitant elevations in total bilirubin (BT).3 While these concomitant elevations were reversible with prompt discontinuation of tolvaptan they represent a potential for significant liver injury and patients taking tolvaptan will have to undergo monthly blood tests for the first 18 months of treatment with tolvaptan and three-monthly thereafter to mitigate this risk.3 Tolvaptan treatment must be initiated and monitored under the supervision of physicians with expertise in managing ADPKD and a full understanding of the risks of tolvaptan therapy including hepatic toxicity and monitoring requirements.3

“The progressive and hereditary nature of ADPKD is a physical and emotional burden on those living with the condition as well as their families and loved ones” said Tess Harris President of PKD International. “This approval is welcomed by the ADPKD community as it represents a step forward for the thousands of patients and carers throughout Europe who are affected by the disease.”

ADPKD is the most common inherited kidney disease primarily characterised by the proliferation and growth of multiple fluid-filled cysts in the kidney.16 Cyst growth and expansion in both kidneys leads to slow deterioration of kidney function and approximately half of patients reach end-stage renal disease (ESRD) and require renal replacement therapy (RRT) in the form of dialysis or a kidney transplant by the age of 54.27 ADPKD is the fourth leading cause of ESRD in adults8 and accounts for around 10% of patients requiring RRT.9

“It is a great honour to deliver the first treatment for ADPKD in Europe” said Tatsuo Higuchi President and Representative Director of Otsuka Pharmaceutical Co. Ltd.. “This approval is testament to the invaluable endeavours of the researchers and patients involved in the discovery and development of tolvaptan.”

Tolvaptan was first approved for patients with ADPKD in Japan in March 2014 and was approved for ADPKD in Canada in February 2015. Following this European marketing authorisation Otsuka will continue to work with local authorities in countries throughout Europe to help ensure that eligible ADPKD patients are able to access tolvaptan.

About JINARC® (tolvaptan) in ADPKD

Tolvaptan is a potent vasopressin V2 receptor antagonist that has been proven to slow cyst growth and renal function decline in patients with ADPKD in CKD stage one to three with evidence of rapidly progressive disease.1

The formation of cysts associated with ADPKD can lead to significant kidney damage even when kidney function is not affected10 and cause complications that include chronic and acute pain hypertension and kidney failure.1 In addition to the physical symptoms ADPKD causes a profound psychological and emotional burden for those living with the disease their families and loved ones. 11-13

By selectively blocking vasopressin from its V2 receptor tolvaptan decreases cyst cell proliferation and fluid secretion ultimately reducing cyst development and renal clinical events associated with disease progression.14 [Read more]

Funding the Cure

From KSHB TV, Channel 41, Kansas City, by Terra Hall

Cooking for a cure: Reality TV star brings awareness to Kansas City nonprofit organization

KANSAS CITY, Mo. - While he didn't win the grand prize in the Food Network's cooking competition, Chopped All-Stars, New York City Chef Michael Psilakis still brought a ton of awareness to a Kansas City charity. He rallied for the Polycystic Kidney Disease Foundation throught the fourth season of the show.

Psilakis' dad died from complications related to polycystic kidney disease. The disease causes cysts to grow on a person's kidneys. A normal kidney is the size of an adult fist, but the kidney's of people living with the disease can blow up to the size of a football and weigh up to 30 pounds each.

If Psilakis wins, he'll donate $75,000 cash prize to the Polycystic Kidney Disease Foundation, a local nonprofit organization.

To learn more about the disease, including the warning sighs you should look out for, head over to Polycystic Kidney Disease Foundation website.

Seeking Life

From KMBC TV, Channel 9, Kansas City, By Kisha Henry

Family turns to social media to find kidney donor

Kevin Heuerman's kidney function is down to 10 percent

SEDALIA, Mo. —A Missouri family is using an unusual technique to try to find a kidney donor.

Kevin Heuerman, a Sedalia husband and father, has polycystic kidney disease. He said his biggest wish is to get a call to come to Research Medical Center in Kansas City for a kidney transplant.

“A healthy kidney is about the size of a fist,” Heuerman said. “Mine, right now, they say are the size of footballs.”

The disease has covered Heuerman’s kidneys with growing cysts, causing the organs to lose function. He spends 12 hours each week at a clinic for dialysis, time that keeps him away from his family. He said it’s been tough on his son.

“He’ll be 10 in July,” Heuerman said. “It’s hard to tell him sometimes that I have to rest or I can’t go out and do this or that.”

With his kidneys functioning at less than 10 percent, Heuerman has been placed on the donor registry. The average wait is three to five years, so his wife has gone in search of that kidney herself.

“We’re best friends and there’s nothing I wouldn’t do for him,” said Melissa Heuerman.

Her first stop was Facebook, where a page calling attention to the family’s need gained traction overnight.

“We’ve had people share it in California, Hawaii and Arkansas,” she said.

She also has a sign she takes with her everywhere she goes.

“Impact Signs and Lighting called us and said that they would like to put it on my car for free,” Melissa Heuerman said.

“I knew when it was going to happen and we started doing all the testing – I knew that she’s not the kind of person that can be quiet about anything,” Kevin Heuerman said. [Read more]

Giving Life

From The Seattle Times, By Joyce F. Jackson

Be a hero: Donate a kidney

STRANGE as it may sound, the nonprofit dialysis organization I lead works very hard to help patients leave dialysis, or avoid it entirely. Instead, we help them get a transplant.

For many people with chronic kidney failure, a kidney transplant offers the best chance at the lifestyle they enjoyed before diagnosis. But until quite recently, nature often thwarted good intentions because of tissue mismatches. Seattle Times reporter JoNel Aleccia recently reported a very hopeful story about technology to arrange kidney swaps, matching willing donors with compatible recipients, greatly extending the possibilities for transplants.

Without a transplant, people with kidney failure must go on dialysis three days a week or daily for life, relying on a machine to do the waste-removal work of a kidney.

Transplant recipients often live longer, feel better and have more independence than people on dialysis. So we actively encourage our patients to seek a transplant. More than a quarter of Northwest Kidney Centers’ dialysis patients now qualify for the waitlist to get a kidney.

We work with each individual to clear barriers to transplant. Some receive free treatment from volunteer dentists who make sure oral infection doesn’t bump them off the list. We coach patients to speak out about their need for a kidney wherever they go, in hopes of appealing to a prospective living donor.

Last year, 87 of our patients received the gift of a new kidney. A kidney patient here is almost twice as likely to get a transplant as in other parts of the country. That’s a testament to hard work by our doctors and staff to make transplants a priority and to support patient enthusiasm to get a new kidney. Seattle is fortunate to have four excellent transplant centers — Swedish Medical Center, the University of Washington Medical Center, Virginia Mason Hospital and Medical Center, and Seattle Children’s Hospital.

While many of our waiting patients have a friend or relative ready and willing to give a kidney, others aren’t as lucky. That’s where you come in.

Be sure your driver’s license has a red heart indicating you’ll be an organ donor after you die. Take care of that task when you renew your license, or better still, go now to www.donatelifenw.org and sign up.

Open your mind to the possibility of becoming an organ donor while you live by giving one of your two kidneys. If you don’t already know someone who needs it, chances are you will hear of a friend (or a friend of a friend) in need. Your recipient’s insurance would cover the cost of your surgery and you would need a few weeks off to recover. [Read more]

Gift of Life from Stranger; Miracle in Iowa; Police Service; Dialysis on Demand: At Home; Pakistan Singer coming to Atlanta for Surgery

Gift of Life

The Herald News, New Lenox, IL, By DENISE M. BARAN–UNLAND

New Lenox woman received donor kidney from a stranger

NEW LENOX – In early 2014, a Facebook friend of Kellie Lonchar, 51, of Ohio shared a post from a New Lenox woman seeking a kidney donor.

Lonchar, widowed at a young age when she had three small children, scrolled through the comments assuring the woman – Michelle Doyle – of prayers and well-wishes.

But Lonchar, who also had the rare Type O-negative blood Doyle had, thought she needed to do more.

“Her story resonated with me, and I could not stop thinking about this woman,” Lonchar said. “I don’t know why.”

On Sept. 30, Lonchar met Doyle. On Oct. 2, Lonchar donated one of her kidneys to Doyle and saved her life.

Virtual call for help

By January 2014, Doyle’s surgeon warned her she was a ticking time bomb. She no longer had any kidneys, and her doctor told her she needed to turn to social media to help find a live donor. She needed to stop worrying people might pity her.

So – reluctantly – Doyle did. She is humbled at Lonchar’s generosity and called her amazing.

“She’s like the sister I never knew,” Doyle said.

Looking back, Doyle said she knew her kidneys were in trouble, but she didn’t want to admit it. Her grandmother, Lorraine Steinquist, (deceased) had begun hemodialysis when she was 45 for polycystic kidney disease.

Doyle’s mother, Cheryl Doyle, also was 45 when she began dialysis. Fifteen years ago, Pam Vanderbilt of New Lenox, Cheryl’s neighbor, donated one of her kidneys to Cheryl. One of Doyle’s uncles also had a kidney transplant, also for polycystic kidney disease.

According to the National Institutes of Health, www.niddk.nih.gov, polycystic kidney disease is a genetic disease in which cysts form in the kidney and fill with fluid, causing the kidneys to enlarge and leading to possible kidney failure.

But when back pain began when Doyle was 24, she attributed it to working out. When her abdomen enlarged, Doyle blamed it on middle-age spread. Her nocturnal shortness of breath was from anxiety, she decided. But it was so bad that Doyle made a doctor’s appointment for blood work.

Emergency situation

On Oct. 6, 2011, Doyle was at the gym with her fiance, Mike Tomaska, when she received a phone call from her doctor. Get to the hospital now, the message said. You are in end-stage renal failure.

The blood work for Doyle’s kidneys was so bad, the doctor couldn’t believe the number, Doyle said. So Doyle – who is terrified of needles – and Tomaska went to the hospital. A permanent catheter was put into her chest, and dialysis was begun. [Read more]

From The Des Moines Register, by Deb McMahon

Kidney donation provides miracle for Iowa family

My family received a miracle at the Mayo Clinic! My dear sister Pam will not have to receive dialysis treatments due to polycystic kidney disease.

Pam received that life saving kidney just in the nick of time. Her kidney function had deteriorated to 12 percent, she had a constant headache, her back felt like it was on fire and she threw up every morning. Yet, she remained the eternal optimist, never complaining, asking "why me?", moaning or groaning. Just seeing her, one would not guess she was suffering from end stage renal disease.

The miracle happened at the Mayo Clinic under the able hands of Dr. Mikel Prieto and his assistants. Of course the miracle could only happen with a donor. Our angel donor changed my sister's life and there is no way we could ever repay her.

What do you say to someone who so selflessly made the decision and never wavered? She willingly "shared her spare" so my sister could have a chance at life unencumbered by dialysis.

She gave her the gift of life and feels as blessed by her decision as we feel by the miracle outcome. That is true grace.

I am still in awe of the gift given by our angel and by the speedy recovery of my sister. She is at The Gift of Life Transplant House in Rochester where she will stay for two weeks. With her positive attitude, her grateful heart and her treasured family and friends, she will continue to be a shining example of what a miracle looks like!

Being able to see a miracle as it played out at The Mayo Clinic was a gift. The bond formed with the angel donor and her angel husband, the sharing together when we heard good news, the applause when they were out of surgery and the group hugs with happy tears are memories I will not forget.

We are family now. We have shared an experience that was life changing. It is true that there are angels among us and our angel just saved my sister's life!

From Daily Telegraph, St. Marys, Australia, ALISON BALDING

St Marys police officer donates kidney to colleague

St Marys Senior Constable Tony Mascherin recently received a new kidney from a colleague. Pictures: Matthew Sullivan

Senior Constable Tony Mascherin will live to dance at his children’s weddings because a kind-hearted colleague gave him a kidney.

The St Marys police officer hopes sharing his story will encourage more people to register as organ donors after Detective Senior Constable Tony Blair donated a kidney.

“It saved my life,” Sen-Constable Mascherin said. “I’ll see my children grow up and get married, and have a better quality of life.”

YOU’RE NEVER TOO OLD TO DONATE

HUNDREDS OF PATIENTS MISSING OUT ON ORGAN DONATIONS


The father of four was diagnosed with polycystic kidney disease in 2007.

By 2011 his kidney function was below 8 per cent and he was put on dialysis for up to six hours a day.

When word started to spread through St Marys police station, Detective Sen-Constable Blair felt compelled to step forward.

He did not want recognition for the selfless act but spoke to The Standard to encourage others to become organ donors. “He needed a kidney and I had one spare,” Sen-Constable Blair said. “I know it’s an innocuous statement but it’s the truth. Anyone would do the same, I just happened to get in first.”

His advice to others considering becoming donors was “just do it”. The pair have the same first name, have been police officers for eight years and each has four children but, until this generous gesture, they were not particularly close.

Less than three months after the operation, Sen-Constable Mascherin said he was still trying to work out a way to say thank you.

“I still can’t believe someone could be that nice, he saved my life,” he said. “That is just who Tony is, a very kind-hearted person.”

St Marys crime manager Detective Inspector Barry Vincent praised the “selfless act”. “I hold him in the highest regard,” he said.



Living with PKD

From Dawn, Pakistan, by MEHREEN HASAN

Veteran singer Alamgir set to leave for Atlanta for kidney removal

Veteran singer Alamgir has been battling polycystic kidney disease for years and is ready to take the next step towards relief from the pain he has long suffered.

Alamgir is about to leave for Atlanta, where his kidneys will be removed as a preliminary procedure for his kidney transplant next year.

Also read: Alamgir: Ailing pop star sings at media event

The surgery is expected to take place in mid-June at Atlanta's Emory Hospital, which is "one of the best in America," said Alamgir to Dawn.com.

"Once I arrive, I'll undergo lots of tests, so my doctors are sure that I'll be able to tolerate the long surgery," he added.

Typically, a third kidney can be fitted in without the removal of the existing kidneys, but Alamgir's are so enlarged that there is no room for the third, he said. He added to Dawn.com that he finds it hard to breathe due to this reason.

It will take Alamgir five to six months to heal after the surgery, after which he will return to Pakistan to wait out the interval before his transplant.

Responding to a question about his treatment funding, Alamgir said that the government hasn't fully made good on its promise to donate for his kidney transplant. "They had promised me 50 lakhs, but I only got 20. It's been three years since 2012 (when I received the first donation) and there has been no follow-up. I managed to raise the remaining money through my own hard work."




Dialysis Developments

From The Wall Street Journal, By TIMOTHY HAY

Outset Medical Raises $60 Million for Miniaturized Dialysis Machines

The Tablo dialysis system Outset Medical

The hundreds of thousands of patients in the U.S. who get kidney dialysis face multiple appointments at the clinic each week, where they hook up to big, bulky machines for hours at a time.

The technology involved, which essentially does the work for kidneys that are malfunctioning, hasn’t been significantly upgraded in decades.

One startup medical-technology company, San Jose, Calif.-based Outset Medical Inc., aims to make the experience easier on patients, and eventually turn dialysis into something that can be done in the home.

The company is making a small, lightweight system that can do the same work that today is done by machines that take up most of a room, Chief Executive Leslie Trigg said.

Outset Medical is in the midst of raising a large Series B round to get its system into dialysis clinics, and begin the process of moving the technology into patients’ homes, Ms. Trigg said.

The company has thus far closed on $45 million in new equity investment plus $15 million from converted warrants in a Series B round that is expected to grow significantly larger in the near future, she said.

The funding was provided by return investors Warburg Pincus and Vertical Group, as well as new public-equity investors whose names the company isn’t disclosing. The second phase of funding, which is expected to close soon, will also involve new and returning investors, Ms. Trigg said.Among Outset Medical’s innovation is the miniaturization of the water-filtration component, a breakthrough that could lead to the first consumer version of dialysis with a device small enough to be kept in the home. [Read more]

PKD Fundraising

From News-Dispatch, Michigan City, By MATT CHRISTY

Car show helps raise money for PKD research

La PORTE — The annual Cruisin' for a Cure for PKD, which was held at the La Porte County Fairgrounds on Sunday, brought classic car enthusiasts and owners together to help raise money for the genetic kidney disorder.

Organized by Laura Moyer, who suffers from polycystic kidney disease and comes from a family where 15 members share the disease, the event consisted of a car show followed by a 40-mile cruise. All money raised went toward PKD research.

All makes and models of vehicles were welcomed as the event kept focus on the cause. More than 50 cars participated in the event, from 1931 Ford Model A's to brand new Ford Mustangs. Prizes also were given away, as well as trophies to three judge's choice winners.

La Porte County Sheriff John Boyd served as guest judge for the car show and led the cruise afterward.

"I'm honored because it's such a great event," Boyd said. "It's nice to see the community come out for something positive where they rally behind a cause that's so beneficial for people."

PKD is one of the most common life-threatening genetic diseases and affects 1 in 500 Americans. The disease causes cysts to form on both kidneys, eventually resulting in the organs shutting down and requiring those suffering from PKD to need organ transplants and anti-rejection medicine.

Moyer, who serves as chair of the Northern Indiana Chapter of the PKD Foundation, said in attendance were organ donor recipients who totaled more than 27 years of life with their new organs. Moyer is one of those, as she received a kidney thanks to her husband's donation three years ago.

"Everybody is awesome," Moyer said of the turnout for the event. "Even the people that come through and maybe just want to go look at the old cars and like to do that are willing to give a donation because they know it's to a great cause."

The money raised will continue to go to research and clinical trials for cures to PKD, as well as research to help organ donor recipients keep their new organs inside of them.

"It's important and not only just about kidney disease, but also about organ donation," Moyer said. "We're trying to teach both here about how important organ donation is. Unfortunately there are lots of people dying everyday because there isn't donations. We're trying to teach both."

For more information about PKD and the Northern Indiana Chapter of the PKD Foundation, as well as upcoming events, visit www.pkdcure.org.

Pain & Burden of PKD; Unique Treatment for PKD; Tattoos for PKD

Living with PKD

From MPR

Pain in Autosomal Dominant Polycystic Kidney Dz Often Complex

(HealthDay News) — The etiology of pain in autosomal dominant polycystic kidney disease is complex, and management of pain should be approached in a stepwise manner, according to a review published in the May issue ofThe Journal of Urology.

Matthew W. Tellman, from the Indiana University School of Medicine in Indianapolis, and colleagues conducted a systematic literature review of the etiology and management of pain in autosomal dominant polycystic kidney disease and anatomy of renal innervation.

The researchers found that for most patients with autosomal dominant polycystic kidney disease, pain occurs due to renal, hepatic, and mechanical origins. Patients may experience different types of pain complicating confirmation of etiology. Anatomical and histological assessment of renal innervation can help elucidate the mechanisms that can lead to renal pain. A stepwise approach is recommended for management of pain in autosomal dominant polycystic kidney disease. Due to the high incidence of acute causes of renal pain in autosomal dominant polycystic kidney disease, these must be ruled out first. Non-opioid analgesics and conservative interventions can be used first for chronic pain, before considering opioid analgesics. Surgical interventions such as renal cyst decortication, renal denervation, and nephrectomy can target pain from persistent renal or hepatic cysts.

"Chronic pain in patients with autosomal dominant polycystic kidney disease is often refractory to conservative, medical, and other noninvasive treatments," the authors write.  [Read more]

From AJMC

Burden of Autosomal Dominant Polycystic Kidney Disease: Systematic Literature Review

ABSTRACT 

Objectives: Autosomal dominant polycystic kidney disease (ADPKD), the most common hereditary kidney disorder, is a leading cause of end-stage renal disease. While there is no pharmacologic ADPKD therapy indicated, earlier supportive treatment may reduce disease burden, which may lead to the reduction or prevention of healthcare utilization and costs. To help US healthcare payers to understand this genetic disorder and the related healthcare utilization and costs, we performed a literature review on ADPKD. 

 Study Design: This literature review includes information on ADPKD incidence and prevalence, diagnostic criteria and risk factors, and the humanistic and economic burden. The information was summarized to characterize the impact of ADPKD on patients and healthcare systems. 

 Methods: PubMed and EMBASE databases from January 2003 to March 2013 were searched for articles containing relevant key terms, which were then screened for exclusion criteria to focus on data for ADPKD. For the selected publications, data were extracted and summarized. 

 Results: The results indicate that prevalence studies are outdated and have generally been on small populations. Additionally, diagnostic criteria are established, and a few possible disease progression prognostic factors have been identified. Pain is a commonly recognized element of humanistic burden, and a correlation between reduced kidney function and increased healthcare costs has been demonstrated [Read more]

From Business Wire

Unique Treatment for Polycystic Kidney Disease: Two Surgeries, One Anesthesia

FARMINGTON, CT--(BUSINESS WIRE)--Only a handful of transplant centers in the country treat polycystic kidney disease (PKD) with removal of both dysfunctional kidneys with a replacement kidney from a living donor in one operation.

BroadcastMed, Inc. and University of Maryland Medical Center present Terry J. Watnick, MD who works with the transplant team to help patients avoid multiple surgical operations, increased costs and potential prolonged dialysis by performing one procedure.

Dr. Watnick is an Associate Professor of Medicine at the University of Maryland School of Medicine and the Director of the Baltimore Polycystic Kidney Disease Research and Clinical Core Center.

Physicians can learn more at the UMMC Physician Learning Channel.

About University of Maryland Medical Center

The University of Maryland Medical Center (UMMC) performs at least 300 kidney transplants per year and has one of the nations largest kidney transplant programs. UMMC surgeons are also at the forefront of laparoscopic living donor nephrectomy, which has made kidney donation much easier, and allows for patients to get transplant faster. For more information visit umm.edu/transplant.

PKD Fundraising

From Northern Life, Canada, By: Sudbury Northern Life Staff

Sudburians urged to buy a temporary tattoo for Taylum

Two-year-old Taylum Lamoureux is expected to receive a kidney donation May 13, after spending his entire life living in Toronto's Ronald McDonald House due to his kidneys failing shortly after birth. Supplied photo.

Fundraising event aims to help toddler, Ronald McDonald House

A fundraising event intended to help a Chelmsford toddler who is receiving a kidney transplant begins Monday and runs until Wednesday, when he's scheduled for surgery.

Taylum Lamoureux has Polycystic Kidney disease and has spent his entire life in Toronto living at Ronald McDonald House waiting to grow big enough to receive a kidney transplant. Now two years old, he will receive a transplanted kidney from a selfless stranger who will donate one of hers to him.

Starting this week, friends and supporters of the family will be selling temporary tattoos for $1 each and organizers are “encouraging all of our students, teachers, friends and family to wear them on May 13, the day of his transplant.

“The proceeds raised will be donated to Ronald McDonald House Toronto, and a portion of the proceeds will be used to purchase a play structure for Taylum when he is finally able to return home,” wrote Marymount Academy teacher Tammy Jutila, in an email. “It is the hope that each secondary school will be participating, and the campaign will only run for three days, May 11-13.”

Many local businesses will also be selling the tattoos to help the family. And anyone interested in buying a tattoo can contact one of the following organizers: Jenni Preen, 705-566-960; Jen Geddes, 705-969-2212; MacKenzie Crowther, 705-523-9235; and Tammy Jutila, 705-674-4231.

Since he was brought to Toronto, Taylum has lived at Ronald McDonald House, near the Hospital for Sick Children, with his mother Désirée Lamoureux. If all goes well, after the operation he'll be visiting the hospital every day for three months, for blood work and checkups, to make sure his new kidney is functioning properly.

After three months, if there are no complications, Taylum and his mother will be able to return home to Sudbury. But he will need to return to Toronto every two weeks for more checkups, over a three- to six-month period.

From CJME, Radio 980, Regina, Canada, Reported by Lasia Kretzel

'You have your life back,' transplant recipient, donor support annual walk

Deb Leisle and Bonnie Cockrum support annual Transplant Trot

Deb Leisle and Bonnie Cockrum share a unique bond: their kidneys.

Born with polycystic kidney disease, Deb Leisle started dialysis eight years ago. Four years later, while visiting a friend at St. Paul's hospital, she met nurse Connie Cockrum. Cockrum learned of Leisle's disease and need of a new kidney, and decided to donate one her own.

"It was just something from within that said, 'hey you should do this,'" Cockrum said.

The two were among the more than 100 people who came out Saturday to support and take part in the Transplant Trot. The annual run raises awareness for the need for organ donations and celebrates recipients and donors.

Provincial director of Canadian Transplant Association Phil Gleim said there are 100 people people on the Saskatchewan waiting list to receive a kidney. Many more are waiting on lungs, hearts, livers, bones and corneas.

As many as 50 per cent of all kidney transplants are through living donors while liver, bone and amniotic membranes can also come from live patients.

Deceased donors can save up to eight lives if their donate their organs, Gleim said.

"If people were to come out to events like these or meet someone who has had an organ transplant, I think they realize you're not just saving someone's life, you're giving somebody back a quality of life," he said, adding it is an individual decision.

Cockrum said on the day of the transplant, she wasn't nervous.

"That kind of thing really excites me, to be able to change someone's life like that," she said.

Leisle said the transplant gave back her life.

"You're not tied to a machine three days a week, you have your life back, you're able to eat (what you want)," she said. "Now I can go on a holiday."

Today, Leisle and Cockrum both live healthy lives and share a unique friendship.

"We're kind of hitched at the kidney," Leisle said with a laugh.

"I have visiting rights now," Cockrum added.

PKD Research

From JCI, Journal of Clinical Investigation

Macrophage migration inhibitory factor promotes cyst growth in polycystic kidney disease

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is characterized by renal cyst formation, inflammation, and fibrosis. Macrophages infiltrate cystic kidneys, but the role of these and other inflammatory factors in disease progression are poorly understood. Here, we identified macrophage migration inhibitory factor (MIF) as an important regulator of cyst growth in ADPKD. MIF was upregulated in cyst-lining epithelial cells in polycysitn-1–deficient murine kidneys and accumulated in cyst fluid of human ADPKD kidneys. MIF promoted cystic epithelial cell proliferation by activating ERK, mTOR, and Rb/E2F pathways and by increasing glucose uptake and ATP production, which inhibited AMP-activated protein kinase signaling. MIF also regulated cystic renal epithelial cell apoptosis through p53-dependent signaling. In polycystin-1–deficient mice, MIF was required for recruitment and retention of renal macrophages, which promoted cyst expansion, andMif deletion or pharmacologic inhibition delayed cyst growth in multiple murine ADPKD models. MIF-dependent macrophage recruitment was associated with upregulation of monocyte chemotactic protein 1 (MCP-1) and inflammatory cytokine TNF-α. TNF-α induced MIF expression, and MIF subsequently exacerbated TNF-α expression in renal epithelial cells, suggesting a positive feedback loop between TNF-α and MIF during cyst development. Our study indicates MIF is a central and upstream regulator of ADPKD pathogenesis and provides a rationale for further exploration of MIF as a therapeutic target for ADPKD. [Read more]