Sunday, April 26, 2015

Doctor: Biggest Kidneys He has Ever Seen; Discuss both sides of organ donation; Gift of Life for 2 year old Taylum

Living With PKD

From New Delhi TV

2.75 Kilogram (6 lbs) Kidney, World's Largest, Removed From a Patient in Delhi

Doctors have removed the world's largest kidney

A kidney weighing 2.75 kg, claimed to be the world's largest, has been removed from a 45-year-old patient, the authorities at a hospital in Delhi where the operation was performed said on Tuesday.

Subash Yadav was suffering from autosomal dominant polycystic kidney disease (ADPKD) for a couple of years and was admitted to the Sir Ganga Ram hospital (SRGH) last month following chronic renal failure with severe pain in abdomen and blood in urine.

According to hospital authorities, the case was analysed closely and a decision was taken to remove both of his kidneys and transplant new ones. Also the other reason was the deteriorating condition despite being admitted to the hospital.

The doctors remove the 2.75 kg kidney after a three-hour-long surgery.

"We found the procedure quite challenging. Normal kidneys weigh 130 grams approximately. This kidney weighed 2.75 kg which is 20 times more and was stuck to the surrounding intestines," said Manu Gupta, consultant urological surgeon at SGRH and who performed the surgery.

"Even the second kidney of the patient also weighed 2.5 kg and had to be removed in another surgery a week later," Mr Gupta said and added that altogether, the patient's body was bearing an extra weight of about 5 kg.

Mr Gupta said that the dimensions of this huge kidney were 33 X 20 X 20 cm. This happens to be the largest kidney removed to date in the world.

The Guinness Book of World Records reports a kidney weighing 2.15 kg as world's largest kidney till now which was removed in Dhule, Maharashtra in 2011. The dimensions of that kidney were 33.72 X 14.14 X 15.05 cm.

Vinant Bhargav, consultant nephrologist at the hospital said that pre-transplant nephrectomies (removal of kidneys) is occasionally necessary in such situations.

"The patient is recovering well now and awaiting kidney transplant," he told IANS.




From Shelbyville Times-Gazette, By JOHN I. CARNEY, Tennessee

McTigue remains positive despite waiting, setbacks

(Photo)

When Brad McTigue's phone rings, and the Caller ID is either "Vanderbilt" or "Indiana," he has to be ready to drop everything. What that call means is that there's a possible match for a life-saving kidney transplant.

That call has come nine times -- and, nine times, Brad has been disappointed. Six of those nine times, the donor turned out to have health problems. One motorcycle accident victim turned out to have undiagnosed tuberculosis. A potential live donor had breast cancer. McTigue's own brother-in-law flew in from Hawaii, and he and McTigue drove up to Indiana for the transplant, only to discover that the brother-in-law also had a kidney disease.

Lost chance

Last year, though, was the cruelest disappointment of all. A friend of a friend who was an organ donor had passed away, and appeared to be a good match. Doctors harvested and transplanted six different organs, the last of which was the kidney meant for Brad McTigue. It took all day on March 31, and when he looked at the clock he realized it was after midnight -- April Fool's Day.

The donated kidney failed, within a day of the transplant. They originally assumed it was a problem with the organ, but it now appears to have been an artery that was kinked during the transplant surgery.

McTigue tries to stay positive, but describes that experience as "a cruel April Fool's joke."

Fewer matches

Now, he's gone back to waiting. To make matters worse, the anitbodies his body generated, in misguided self-defense, during the failed transplant make it harder to find a compatible match.

It all started when Brad was 20 and his father was diagnosed with polycystic kidney disease, or PKD, which refers to the formation of multiple cysts in the kidney, interfering with its function. The disease is hereditary, so Brad was immediately tested.

"I started seeing a nephrologist when I was 20," he said.

Brad's kidneys weigh 15 pounds each.

"I've never met you," one doctor told him, "but you have the biggest kidneys I've ever seen."

Seeking transplant

In March 2013, after years of care, he made the decision to begin seeking a transplant. He went onto Vanderbilt University Medical Center's transplant list in 2013 and onto Indiana University's in 2014.

While the road to this point has been frustrating, McTigue takes solace in the incredible support he's received from his wife Andrea and from his in-laws. There are two online sites seeking potential donors and informing friends and family of Brad's progress. Andrea has created a Facebook page, Team Brad. Brad's older sister, Susan Clark, is active in the National Kidney Foundation and has created the webpage "Batting For Brad," a reference to McTigue's work as a softball umpire. He said she has gotten so agitated at the false alarms that the family has stopped letting her know about prospective transplants before they've been checked out.

Sudden difference

McTigue drove to Susan's house in Alexandria, Va., last May to attend the National Kidney Walk, but he was unable to walk -- he was still recuperating from the failed surgery in April and unable to walk because blood vessels in his legs had been damaged during the surgery. A few weeks later, in June, he had stints put in his legs, which had an overnight positive impact.

"The next day, I walked a mile," he said. "That's how much of a difference it made."

Brad also gets encouragement from the number of live donors who are willing to consider giving up one of their two kidneys. Vanderbilt has told him that they've never had another patient generate as many phone calls from potential donors.

"My support is incredible," he said. [Read more]




From Cyprus Mail, By Bejay Browne

Working overtime to upgrade kidney treatment

Working overtime to upgrade kidney treatment



THE NUMBER of patients in need of life saving dialysis treatment at Paphos renal unit saw a huge increase in the first two months of the year.

According to the chairman of the Cyprus Kidney Association-Paphos, there is usually around a twenty per cent increase in patients year on year, but during the first two months of 2015 alone numbers increased by ten per cent.

According to Graham Brown 2,500 people in Paphos need dialysis imminently and 5,000 with kidney problems attend the kidney outpatient unit.

The prevalence of thalassemia, an inherited genetic disease, passed on from parents to children is one reason why the demand for dialysis is high in Cyprus. This blood disorder is three times more prevalent in Cypriots than elsewhere. It is also due to modern living, he noted.

“Our way of life means that our bodies are not able to cope with all of the toxins we are putting in them – from processed foods, shampoos, sun cream and so on. All of these are chemicals,” he said.

He went on to explain that there are only three ways in which a body can get rid of toxins, through the kidneys, liver and sweat glands.

“There are 82,000 Cypriots affected by kidney disorders and 32, 000 are predicted to need dialysis treatment before they are 70,” he said.

Increasing numbers of patients and his own need for regular lifesaving dialysis, is keeping Brown focused on improving facilities available at Paphos Renal Unit,. Since June 2014, the association and its supporters have raised in excess of 60,000 euros. Some of this has been used to replace old and ageing machines, purchase a number of chair beds at a cost of 3,500 each and also to allow for a small extension to the unit.

“We have completed a small extension, by knocking through walls, so that three more machines have been put in. This will enable 15 more patients to be treated. The bed chairs are due to arrive from Portugal in two weeks’ time,” he added.

Brown, 65, only took over as chairman of the association in April 2014, determined to raise the profile of the charity. He was diagnosed with a hereditary condition of polycystic kidney disease and following the removal of one of his kidneys at a Paphos hospital, he started dialysis treatment at the general in November 2013.

He is now one of many patients who need regular dialysis treatment for five hours, three times a week, to keep him alive.

Brown pointed out that the association has recently changed its name and logo from Cyprus Kidney Association Expats to The Cyprus Kidney Association Paphos, to reflect the work they undertake.

“We changed our name and logo on World Kidney Day – March 12 – as people were asking why is it only for expats. But it’s not, it’s for everyone.” [Read more]




Gift of Life

From Tallahassee Democrat, Florida, by Karl Etters Democrat staff writer


-Helen and Robert.jpg_20150417.jpg


Robert Mayo never really considered being an organ donor, but from the moment he felt compelled to become one, there were no second thoughts.

Helen Schwarz felt she would never find a kidney to replace hers after being diagnosed with polycystic kidney disease in 2013.

The two will come together Thursday to talk about their experience on both sides of organ donation and urge others to help save a life during an Organ Donor Month screening of the documentary film "Perfect Strangers" at Tallahassee Memorial HealthCare.

Mayo, 60, was only a few sentences into a story about Helen written by former Tallahassee Democrat Executive Editor Bob Gabordi, when he said there was a sense of absolute direction from God to reach out to Helen and doctors at the University of Maryland Medical Center in Baltimore.

"From that moment, I had no fear of the consequences," said Mayo of Tallahassee. "I've never had a moment in my life when I felt such a strong sense of, wow; you've got to do this."

The former administrative manager of the Tallahassee Democrat newsroom, 62-year-old Schwarz didn't know Mayo until he contacted her daughter soon after the transplant operation.

At one point, they were just down the hall from each other in the hospital but had no idea.

Schwarz, who later received a liver transplant from a deceased donor in February 2014 after being diagnosed with polycystic liver disease, was comforted in their similar faith after Mayo reached out using the name "Padre."

"I was over the top excited. Over the top scared. Over the top everything and I didn't know who this person was," Schwarz said.

The option of dialysis was out there, but Schwarz had three other reasons to push for a transplant – her grandchildren who are now 2, 3 and 5-years-old.

"My whole reason why I was so desperate and so anxious to live was I wanted to be the fun Grammy and watch my grandchildren live and grow," she said.

The two have remained close and often have dinner or just chat by phone.

Mayo first met with Schwarz, her daughter and son-in-law and their children around New Year's Day 2014 and spent time with her entire family again at Easter that year.

Mayo said he feels like family. His mild discomfort from the surgery means little compared to what he was able to give.

"It is far surpassed by the joy I have every single day when I think about Helen being alive," he said. "That is irreplaceable. I will take it to my grave." [Read more]




From CBC Sudbury, Canada

Taylum Lamoureux to undergo kidney transplant

Sudbury's Taylum Lamoureaux has found a kidney donor and is scheduled for a transplant next month.


It's news Darren and Desiree Lamoureux have been waiting for — their son Taylum, will be getting a new kidney next month.

Taylum was born with Polycystic kidney disease and for the last two years, he's been waiting to get a transplant.

This means the family will be allowed to stay at Ronald McDonald House in Toronto. Earlier this year, there was uncertainty about the family's housing situation because the house is only intended for short term stays.

Desiree said a woman who the family didn't know reached out to them through social media, and said she was willing to donate a kidney to Taylum.

"It was an immediate feeling … just a gut instinct, like, I think this is the person we have to go with," she said.

"I kind of wanted to exhaust our options of people we knew beforehand, but something told me to go with this person."

The donor went through testing and was a match.

Taylum's transplant surgery is scheduled to take place May 13.




PKD Fundraising

From Enid News, Oklahoma, by Melissa Thomas

Fundraiser is set up for kidney transplant

Melissa Thomas



Four years ago, Melissa Thomas was trying to get healthier when she received some terrible news.

“I was trying to lose and weight and I went to the doctor and was talking to her about why I couldn’t lose it on my mid-section,” Thomas explained. “She said, ‘Let’s run some tests.’ She ran a bunch of tests showing my kidney function was really low.”

Following an ultrasound, Thomas was told she suffered from polycystic kidney disease and would need a kidney transplant.

“It’s genetic. Both of my parents are deceased, so I don’t know which one of them would have had it,” she said. “The disease itself causes the kidneys to fill up with cysts. At this point, I have hundreds of cysts in both kidneys.”

Thomas said the disease causes her 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.

“The pain isn’t so much as the pressure. It causes the kidneys to grow,” she said. “Instead of being the size of your fist, they’re about five times that size and they kind of push on the other organs.”

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.

“My kidneys will eventually fail, which is what they are doing,” she said. “The new one will not get the disease, but it won’t stop the liver cyst from forming and it won’t stop the other issues it causes. You still have the disease forever.”

Thomas participated in a three-year study of PKD, undergoing bloodwork and MRIs every six months to help study the disease. When her kidneys are removed, they will be donated to the PKD Foundation for further study.

Sunday, April 19, 2015

I may look pregnant but I'm living with PKD; Save a life; Kidney Transplant Chain Saves 34 Lives; Managing PKD

Save A Life

From The Trentonian, Trenton, New Jersey, by Jeff Edelstein

Sheri Gould needs a kidney, and is hoping to find someone to help


Sheri Gould of Lawrence has PKD, a kidney disease that will result, for her, in kidney failure. She’s hoping to find someone who is willing to donate one of their kidneys.


Sheri Gould is terrified.

It’s not necessarily because she’s going to have to begin kidney dialysis soon. It’s not necessarily because she knows a lifetime of dialysis is going to be difficult on her body and mind. It’s not necessarily because for many people, dialysis is just a weigh station to the end of the line.

Nope. She’s terrified because she’s seen what happens to people with Polycystic Kidney Disease (PKD) who go on dialysis. Specifically, her own mother.

“I saw my mom struggle,” Gould, 50, of Lawrence told me. “I don’t want to go through that.”

She spoke of infections that would make your skin crawl. Extreme fatigue. Quality of life going down the tubes.

Her mother died at age 70 after 14 years on dialysis.

So Gould — like many sufferers of PKD, a hereditary disease that affects roughly 1 in 10,000 people worldwide — is seeking to do something about it.

Sheri Gould is in the market for a kidney.

Got one to spare?

If so, reach out to Sheri at her website KidneyForSheri.com.

And don’t worry — you don’t need to be a perfect match, as through the Living Kidney Donor Network, your kidney might go to someone you match with say, in California, while Gould would get someone’s else’s kidney. Think of it like four first graders swapping snacks at lunchtime. You get Greg’s Snickers, Jane gets your Twix, Paul gets Jane’s potato chips, etc. You get the picture.

Only one snag to this whole scenario though, and Gould knows it: “Yes, of course,” she said. “If I had two good kidneys, I’d be afraid to give one away.” [Read more]


From WJLA, Channel 7 ABC Affiliate, Washington D.C. , By Alison Starling


With 34 donors and 34 recipients, Chain 357, nicknamed a “chain of love,” is the country's largest-ever multi-hospital kidney transplant chain. The National Kidney Registry worked with 26 hospitals across the country to make sure every link of the chain connected.

Since Jan. 6, the chain has bounced across the country, including stops at MedStar Georgetown Transplant Institute in Washington, D.C.; Walter Reed National Military Medical Center in Bethesda, Md.; University of Virginia Hospital in Charlottesville, Va.; and two bouts at the University of Maryland Medical Center in Baltimore, Md.

LaTwanya Goslee, 43, tried to donate a kidney to her brother, 41-year-old Charles Muse, for several years. Their mother, Edith Summers, had donated a kidney to Muse in 2003, after he suffered from kidney failure due to hypertension. But that kidney eventually failed; he went on dialysis, and needed another transplant.

Goslee and Muse hoped the second transplant would be as simple. And initially, they were a match. But after Muse was diagnosed with cancer in one of his native kidneys, they were no longer compatible. And so, they entered the Paired Kidney Exchange Program, through the National Kidney Registry.

So, even though Goslee couldn't donate directly to Muse, by donating a kidney to a stranger, her brother would be eligible to receive a kidney from one of his matches in the system. It turns out his new kidney came from Harley Florez, a man from Colombia. He donated in South Carolina, so that his aunt living there with Polycystic Kidney Disease could receive a kidney, too. And the kidney Goslee donated at the University of Maryland Medical Center was driven by courier immediately to Allegheny General Hospital in Pittsburgh, Pa. to be transplanted into a 65-year-old retired heavy equipment operator named Gary Watson. [Read more]



Living With PKD
From Huffington Post Canada, by Cheri Barton




I'm 47 years old and people are still asking me if I am pregnant. I try to take it as a compliment about how young I must look, but when people first started asking me, I would turn very red and uncomfortable. Then they would also turn red and it was embarrassing for both of us. I have the dominant form of polycystic kidney disease (ADPKD) and polycystic liver disease (PLD), causing my kidneys and liver to develop multiple fluid-filled cysts. As a result of cyst growth due to ADPKD, kidneys become significantly enlarged and, over time, kidney function deteriorates. ADPKD is one of the most common life-threatening genetic diseases, affecting approximately one in 500 people.

Only once, while buying a decaf coffee, did I say "yes" when asked by another customer if I was pregnant, and what a big mistake that was! I tried answering her first question, "when are you due?" and was in the middle of trying to calculate my imaginary due date when she was already on to the next: "Is it a boy or a girl?" After escaping, I looked at my husband and said that I would never pretend to be pregnant again. About 10 minutes later, someone else asked me, and we both burst into laughter.

ADPKD is a genetic disease -- and a parent carrying the gene has a 50 per cent chance of passing it on to their children -- but I mutated the disease, which means my parents don't have it, and I hadn't even heard of it until I was diagnosed about 13 years ago. At that time, our third child was about a year old when I noticed protruding lumps in my stomach and my waistline getting bigger. My doctor sent me for an ultrasound where I learned both of my kidneys and my liver were filled with cysts. I was then referred to a nephrologist who diagnosed me with both ADPKD and PLD.

I have three hernias in my abdomen from the weight and pressure of my liver. I have to wrap bandages around my abdomen every day to hold in hernias. The surgeons told me that they can't operate and I will need a liver transplant eventually, probably before a kidney transplant.

Recently, my worst fears about ADPKD came true: both of our daughters, now 20 and 17 years old, have ADPKD. Our 17-year-old daughter already has over 30 cysts in each kidney and is taking blood pressure medication. Our 14-year-old son has not been tested yet. It has been a roller-coaster journey full of disappointing news and challenges, but also opportunities and evaluating the most important things in life. Here are some of the things I've learned along the way:

Get involved with the patient community (and your local community!)

After being diagnosed, I started researching, and found out that the PKD Foundation of Canada has a chapter in Toronto and it hosts annual Walk for PKD events in September. That year, I held a bake sale in my small town of Vankleek Hill, got Scotiabank to match my fundraising, and ended up bringing over $8,000 to Toronto. The next year I started my own Walk for PKD in Vankleek Hill and 11 years later, we have raised over $170,000 for the PKD Foundation of Canada from my little community alone. It wasn't easy at first being so forthcoming about my disease, but the support from my community has been more positive than I could have ever imagined.

If you're living with a chronic illness or know someone who is, be proactive and get involved with a foundation or patient community. Knowing you're not going through it alone and have a whole network of support relieves a lot of stress and questions.

Learn to love your body

[Read more]




From Air Force Global Strike Command, by Senior Airman Stephanie Morris, Minot Air Force Base Public Affairs, North Dakota

In sickness and in health

in sickness and in health

One old adage of marriage is the vow to be there for each other, for better or for worse in sickness and in health. For many couples, this is a line they recite at their ceremony and store in the back of their minds for many years. However, for one couple, that vow was tested only a couple years after they made it.

Essence Minter was diagnosed at age 18 with polycystic kidney disease while serving in the United States Navy. Both her mother and grandmother also had the disease, but they carried mutation one -- Essence was host to mutation two.

This meant she was at a higher risk of needing a kidney transplant in the future and may even need to be placed on dialysis one day.

Essence's husband, Airman 1st Class Karlton Minter, 5th Medical Operations Squadron emergency medical technician, didn't let this news keep him from meeting her at the end of the aisle.

"I knew about the disease when I met her," Karlton said. "I wasn't afraid of it."

So Karlton and Essence exchanged vows one year after he left for basic training.

Upon graduating from Lackland Air Force Base in San Antonio, Texas, Karlton and Essence found themselves at his first duty station -- Minot Air Force Base, North Dakota.

Unfortunately, it wasn't long after they arrived before they received devastating news. Essence's kidney function was barely above 20 percent, and she had no alternative but to get a transplant.

"The kidney center at Sanford will only do a transplant if the kidney is below 20 percent function," Essence explained while exchanging a pained look with her husband.
Because of this, the two would have to wait, and Karlton was forced to stand by as his wife's health waned.

"As a husband and a man, I felt it was my duty to take care of her," Karlton said. "I was just focused on my wife and on finding a way to help her."

He began speaking to his leadership and the staff at Sanford, trying to find a way to replace his wife's ailing kidneys with one of his own.

Karlton was able to garner advice on donating his kidney to his wife from a co-worker who had recently gone through a similar situation with their mother.

Gaining permission from his leadership and the Air Force was only the first of many hurdles Karlton would face.

The staff at Sanford conducted blood work, echocardiograms and a battery of other tests on Karlton, all of which finally culminated in an extensive interview.

"They must have asked me 500 questions on that survey," Karlton remarked, sharing a laugh with his wife. "They had to make sure I wasn't being pressured to donate my kidney and I was there of my own free will."

With a blood type of O positive, and test results that showed compatibility, Karlton was finally given the green light to go under the knife to save the love of his life.

"I knew the surgery was going to be successful," Karlton said. "It was something I felt in my body, in my bones. I felt spiritually that we met for a reason and this was it."

Many hours later, Essence and Karlton awoke from the first surgery either had ever undergone surrounded by hospital staff and family members. The procedure was a success. [Read more]




From News Medical, Interview conducted by April Cashin-Garbutt, BA Hons (Cantab)

Managing autosomal dominant polycystic kidney disease: an interview with Dr Richard Sandford, University of Cambridge


Please can you give a brief introduction to autosomal dominant polycystic kidney disease (ADPKD)?



Autosomal dominant polycystic kidney disease (ADPKD) is one of the most common inherited kidney diseases. It's also one of the most common reasons that people require dialysis or transplantation.

European-wide figures suggest that about 10% of people receiving dialysis or transplantation have ADPKD. Therefore, there are many more people being followed-up with this condition or who remain undiagnosed because they haven't yet developed symptoms or kidney failure.

ADPKD is a condition that is often diagnosed in adulthood and the typical features are very easy to see on a kidney ultrasound scan. The scan usually reveals enlarged kidneys that contain multiple cysts. Liver cysts are also common.

Sometimes, the kidneys can become so enlarged that they each weigh several kilograms and occupy most of the abdomen or tummy.

In the majority of cases, there is also a progressive decline in kidney function.

As a genetic condition, it typically affects subsequent generations of the same family, so many families that we see will have several family members who are affected.

Please can you explain what “autosomal dominant” means in the context of ADPKD?

Autosomal dominant refers to the inheritance pattern of ADPKD. “Autosomal” means that the ADPKD genes are carried of chromosomes apart from the sex chromosomes: therefore it affects men and women equally.

“Dominant” means that, of the two copies of each polycystic kidney disease genes we have, only one copy contains a genetic alteration or mutation. This mutation is sufficient to cause the disease.

Anybody who has ADPKD and therefore a PKD gene mutation has a 50/50 chance of passing the mutation onto their children. If a child inherits the gene they will then develop the disease later in life.

How is ADPKD currently managed and how much variation is there of clinical standards throughout Europe for ADPKD?

As there are no widely adopted, disease-specific guidelines about managing this condition, ADPKD is generally managed in the same way as any other potentially progressive forms of chronic kidney disease. This involves paying attention to the control of symptoms and blood pressure, as well as the management of cardiovascular risk.

The ‘translating science into policy’ report, the first publication of the European ADPKD Forum (EAF) has been developed to help improve the management of autosomal dominant polycystic kidney disease (ADPKD) throughout Europe. One of the objectives of the EAF initiative is to recommend strategies to improve ADPKD care within the context of health policy development at the European and national levels. [Read more]




From Maney Online

Weight loss in a patient with polycystic kidney disease: when liver cysts are no longer innocent bystanders

Abstract

Objective and importance:

Autosomal dominant polycystic kidney disease (ADPKD) is the most frequent inherited kidney disorder, and liver involvement represents one of its major extra-renal manifestations. Although asymptomatic in most patients, polycystic liver disease (PLD) can lead to organ compression, severe disability and even become life-threatening, thereby warranting early recognition and appropriate management.

Clinical presentation:

We report the case of a 56-year-old woman with ADPKD and severe weight loss secondary to a giant hepatic cyst compressing the pylorus. Partial hepatectomy was required after failure of cyst aspiration and sclerotherapy, and patient's condition improved rapidly.

Discussion and conclusions:

We discuss the presentation and classification of compressing liver cysts, and the available therapeutic alternatives for this potentially severe complication of ADPKD.

Sunday, April 12, 2015

Pot for PKD?, Talk With Yours Kids About PKD; Organ Donation Awareness

PKD Treatment

From KHQA, channel 7, Connect TriStates, by David Amelotti

More diseases and conditions could qualify for medical marijuana use in Illinois

ILLINOIS -- Some Illinois residents are petitioning 14 medical conditions or diseases to be added to the Compassionate Use of Medical Cannabis Pilot Program Act.

The 14 conditions or diseases include anorexia nervosa, anxiety, chronic post-operative pain, diabetes, Ehlers-Danlos Syndrome, essential thrombcythemia with a JAK 2 mutation, irritable bowel syndrome, migraine, Neuro-Behcet's Autoimmune Disease, neuropathy, osteoarthritis, polycystic kidney disease, post-traumatic stress disorder, and superior canal dehiscence syndrome.

Illinois Department of Public Health Communications Manager Melaney Arnold said Governor Bruce Rauner will appoint a board of specialists to review the petitions.

The 16-member board is comprised of physicians, nurses, and patient advocates with background in specialized areas such as psychiatry, mental health, oncology.

"They are going to look at the medical conditions. They're going to look to see if there is any scientific or medical evidence that supports it," Arnold explained. "The benefits, if there are conventional therapies that can handle this type of condition. Those are all things the board will consider."

After review, the board will hold a hearing May 4 in Chicago to discuss and listen to technical evidence regarding the proposed conditions and diseases.

"They are not to have any kind of association with a dispensary or cultivation center so that way it is not someone advocating on their own behalf as far as beneficial, adding a condition for their own benefit," Arnold said.

The public is allowed to petition to add conditions or diseases to the medical cannabis pilot program act twice a year, in January and again in July




Living With PKD

From PKD Foundation of Canada

Sun. May 17 2015 – Hamilton Chapter Informational Meeting: “Talking to your Children about PKD” Webinar

MAY
17


Time: 2:00pm - 4:00pm
Location: Classroom B (T2208), Juravinski Innovation Tower, St. Joseph’s Healthcare Hamilton, 50 Charlton Ave. East, Hamilton ON.

We hope you can join us for this informational support meeting in May!

If there has been a diagnosis of polycystic kidney disease (PKD) in your family, it may not be easy to talk about it, let alone discuss it with youngsters. This webinar features Howard Winokuer, PhD. offering information about grief and loss, as well as his advice about facilitating the healing process of children. The webinar includes Katie and Fran Towey sharing their personal experience of talking about PKD with their own kids.



From CTV, Toronto, Canada

Family of sick toddler searching for apartment near Sick Kids hospital

Taylum Lamoureux in Toronto's Sick Kids Hospital


A Sudbury, Ont., family with a sick toddler is thankful for the support they received following an online plea for help to find an apartment near the downtown Toronto hospital.

Taylum Lamoureaux, 2, needs daily treatment at the Hospital for Sick Children (Sick Kids). Taylum has a rare form of polycystic kidney disease and is awaiting a transplant. He was born with kidney failure and transported by air ambulance to Sick Kids just hours after his birth.

"We have been very moved by the love and support we have been shown by the city of Toronto and beyond," the toddler's mother told CTVNews.ca on Monday.

Only one in four babies with his condition survive, and Taylum wasn't expected to live more than a week. His kidneys were successfully removed when he was an infant, and he receives dialysis to clean his blood. He's undergone more than 1,000 hours of hemodialysis and nine surgeries.

For two years, his mother, Desiree, has stayed at Ronald McDonald House so she can be by Taylum's side during treatments. Her husband, Darren, works 12-hour shifts as an electrician in a mine in Sudbury so they can pay their bills and keep up his health insurance.

To help ease the burden of travelling back and forth, the couple was given a new 2015 Toyota Corolla and a cheque for $25,000 on CTV's Canada AM in December.

The couple was brought to tears, and said the donations meant they'd be able to spend more time together as a family.

"This will mean that Darren can spend more time here with his son," she told Canada AM. "Less FaceTime and more face-to-face time with Taylum."

Months later, the family turned to the Internet to find a new place for that face-to-face time.

The family had been paying $465 per month to rent a small apartment at Ronald McDonald House in downtown Toronto, but were told earlier this year they could no longer stay at the residence, in order to make room for new patients. [Read more]




From Goldendale Sentinel, Goldendale, Washington, By Akasha Spino-Bybee

Kidney disease fighter still needs help

In an earlier story, The Sentinel reported on Joanne Davenport, life-long resident of Goldendale, who was diagnosed with Polycystic Kidney Disease (an inherited disorder in which clusters of cysts to develop in the kidney) 27 years ago. This disease has caused her numerous medical problems and her condition will only get worse as time goes on. Recently we got an update on her situation.

Davenport’s nephrologist has recently informed her that, since there is no cure for this disease, her only chance of survival is with a kidney transplant. She is currently undergoing extensive medical testing and is expected to be put on the active transplant list at Virginia Mason Medical Center in the very near future. Although the transplant will be a heaven sent for Davenport, the leftover costs of co-pays, deductibles, doctor’s visits, travel fees, lodging expenses and temporary relocation fees will add up to thousands of dollars.

Along with this financial burden, the transplant is only possible once Davenport is able to meet the $5,000 deductible. Not to mention, she’ll have to spend around $8,000 a month on her post-surgery, anti-rejection medication, for the rest of her life. To help with the financial burden Davenport faces, a fundraising campaign has been established with HelpHOPELive, a non-profit organization that provides community-based fundraising for patients and their families and has been doing so for over 30 years.

Davenport is calling out to our community for help. There are a few ways that you can donate today:

Go online to http://www.helphopelive.org and in “Find a Campaign”, enter “Joanne Davenport”. Once you’ve been redirected to her campaign, you will be prompted to donate.

Send a check to HelpHOPELive in honor or Davenport at Two Radnor Corporate Center 100 Matsonford Road Suite 100, Radnor, PA 19087. Please make the check payable to HelpHOPELive and in the memo section, write “In honor of Joanne Davenport”.

All donations are tax-deductible, are held by HelpHOPELive in the Northwest Kidney Transplant Fund, and are administered by HelpHOPELive for transplant-related purposes only. To make a donation over the phone or to call for more information, dial (800) 642-8399. There will also be a raffle held at the Klickitat County Fairgrounds during the 2015 Home and Garden Show to benefit the Northwest Kidney Transplant Fund on Davenport’s behalf. Drawings for the raffle will take place from noon to 7 p.m. on May 1; from 10 a.m. to 6 p.m. on May 2; and from 10 a.m. to 3 p.m. on May 3. Prizes include gift certificates, horseback riding lessons, great prizes and more. To donate raffle items or for more information, call Janice Woolley-Coder, (360) 991-4318.




Organ Donation Awareness

From The Edinburgh Reporter, United Kingdom, By John Hislop

Hibs back organ donation campaign


photo


Hibs are backing a national campaign which urges Scots to discuss their organ donation wishes with loved ones.

Seven players donned number 7 shirts in a bid to raise awareness of how the seven words, ‘I’d like to be an organ donor’ can save up to seven lives, and Organ Donation Scotland street teams will be at Sunday’s Edinburgh derby chatting to fans as they head to Easter Road.

Research has shown almost half of people in Scotland (45 per cent) haven’t discussed their organ donation wishes with their family and other football clubs including St Johnstone, Hamilton Accies and Rangers have got behind the number 7 drive in a bid to get more people talking.

Although many think that joining the NHS Organ Donor Register is enough, sharing organ donation wishes remains fundamental to relatives honouring a loved one’s choice in the event of their death.

It’s a subject close to the heart of Hibs fan Claudia Bell, 51, from Edinburgh, whose life has recently been transformed by organ donation. Claudia received a kidney transplant this January after living with polycystic kidney disease (PKD) for almost thirty years.

Due to her deteriorating kidney function, Claudia began dialysis in 2010 and underwent surgery to have a kidney removed in 2011 – meaning she was able to go on the transplant list later that year.

Claudia’s kidney transplant was made possible thanks to an altruistic donor coming forward, and the Hibs fanatic is now making a steady recovery.

Claudia said: “I can’t believe that a total stranger would show such generosity and offer the most amazing gift to me. The difference in how I feel after my transplant to how I felt before is remarkable. I have so much more energy and I don’t have to dialyse anymore which gives me so much more freedom.

“It’s the best gift I’ve ever received. People need to be aware of the huge difference organ donation can make to someone’s life. If your family know what your organ donation wishes are, then should they ever have to make that decision, they can maybe make a positive impact on someone else’s life. This is why it’s so important to discuss your wishes.”

Speaking about her love of Hibernian FC, Claudia said:“I’m a massive Hibs fan. When I was on dialysis, I actually changed the days I was in hospital so I’d still be able to watch their matches.

“I think getting football clubs on board with this campaign is a brilliant idea. It’s going to reach a whole new audience of people and a real cross-section of society who might not really have thought about organ donation before or have discussed it with their family. [Read more]




PKD Research

From Science 2.0, Scientific Blogging

ZS-9 Normalizes Blood Potassium In 98 Percent Of Kidney Patients

Patients with chronic kidney disease may be treated with a class of medications called Renin Angiotensin Aldosterone System inhibitors (RAASI's) but though they protect the heart and kidney, a significant percentage of patients develop a dangerous side effect; high potassium levels in the blood, a condition known as hyperkalemia.

Elevated potassium puts patients at risk of death from cardiac arrhythmias. Lacking a drug to treat the problem, doctors either stop these beneficial drugs or may use kidney dialysis to quickly lower the potassium.
It is estimated that more than 20 million people in the U.S. have chronic kidney disease in varying levels of severity. Risk increases with age, and the disease is most common in adults over 70, particularly those with diabetes. Between 5 percent and 30 percent of patients, depending on the type and stage of chronic kidney disease and whether they are treated with one or more RAASI's, may develop a high potassium condition.

Two national studies of ZS-9, a new oral drug that has been tested in more than 1,000 patients with high blood potassium, had a subgroup of patients with chronic kidney disease treated with RAASI's. Results of the ZS-9 studies were reported in recent months in the New England Journal of Medicine and the Journal of the American Medical Association.

"Dialysis is logistically difficult for patients, requires a catheter and is expensive," said Wajeh Y. Qunibi, M.D., a professor of medicine in the School of Medicine at The University of Texas Health Science Center at San Antonio, at the National Kidney Foundation Spring Clinical Meetings in Dallas. "In our studies, patients took a dose by mouth three times daily with meals, and potassium normalized in 98 percent of them within 48 hours. This is a major change in the way to treat hyperkalemia."

Sunday, April 5, 2015

Treating PKD in Kids; Kidney Health; Building PKD Awareness; Too Much Ice Tea

Living With PKD

From The Toldeo Blade, Ohio, BY ALAN BAVLEY, KANSAS CITY STAR


Early detection is key so it’s easier for doctors to intervene

KANSAS CITY — Two-year-old Marley Martinac has a serious chronic kidney disease, but she’s going to have a better shot at a healthy life than kids born just a decade earlier.

Thanks largely to an ongoing national study with leadership at Children’s Mercy Hospital in Kansas City, doctors now have a clearer picture of the best ways to stop or slow the progression of kidney disease in children like Marley.

The goal is to keep the children growing and thriving for as long as possible, preventing, or at least postponing, the need for kidney dialysis or a transplant.

“Thank God, we’re not at that point at all,” said Marley’s mother, Katie Martinac of Bates City, Mo. “She’s pretty spunky. She hasn’t lost that.”

About 16 percent of the U.S. population has chronic kidney disease, a gradual loss of kidney function caused by high blood pressure, diabetes and a variety of other conditions. How many children have these conditions isn’t known, but 2,500 children nationwide are on dialysis and more than 5,000 have received kidney transplants.

“We’re aiming to head things off in childhood,” said Children’s Mercy kidney specialist Bradley Warady, “to prevent children from requiring dialysis or a kidney transplant, or maybe delay it to give children more chance to grow and for their minds to develop.”

Warady, along with researchers at Children’s Hospital of Philadelphia, is coordinating the Chronic Kidney Disease in Children (CKiD) Cohort, a study that since 2003 has been following close to 900 children with mild to moderate kidney disease recruited from medical centers across the United States and Canada. Their research, funded by the National Institutes of Health, is now the largest long-term study in North America of any chronic childhood disease.

Research by the CKiD group, published recently in the American Journal of Kidney Diseases, shows that the severity of three common conditions in children with kidney disease — high blood pressure, anemia and protein loss through urine — predict how quickly their disease will worsen. For example, when urine has high protein levels, kidneys deteriorate twice as fast.

Because these conditions are all treatable, Warady said, the study offers doctors guidance for improving their patients’ care.

“We want to detect it when it’s mild and easier to intervene,” he said. “We have the tools to treat these things. But the treatment hasn’t been done yet in a consistent way. This emphasizes the importance of these factors. It raises awareness of how significant they are.”

Doctors have not had much data on childhood kidney disease, said Joseph Vassalotti, chief medical officer of the National Kidney Foundation.

This research “really advances our understanding of the natural history of chronic kidney disease in children.”

These diseases include birth defects, such as abnormally developed kidneys that don’t work well or blockages in the kidney’s plumbing; genetic conditions such as polycystic kidney disease, in which fluid-filled cysts destroy kidney tissue; and a variety of diseases that attack the hundreds of thousands of tiny clusters of blood vessels that alter the blood.

“There’s a lot to childhood kidney disease,” Warady said. “It’s a lot more complicated than ‘I just don’t pee.’”

Marley has a condition called nephrotic syndrome that causes her kidney damage. Doctors at Children’s Mercy aren’t sure what is responsible, but they’ve been able to keep the condition under control. [Read more]



From Charleston Daily Mail, West Virginia, by Charlotte Ferrell Smith

After transplant, woman vows to take advantage of second chance at life

Anna Faye Ray, 77, was 66 when she was given another chance at life thanks to the unselfish gift of two kidneys from a family who lost a 2-year-old child.

“I’ll do everything I can to take care of my body so a part of their child will continue to live,” Ray said.

Ray, a resident of Edgewood Summit, works part-time as a ministerial assistant and is a major advocate for organ donation as she frequents health fairs and serves as a speaker at numerous venues.

However, there was a time that poor health took such a toll that she barely had the energy to walk across a room.

In 1971 she was diagnosed with polycystic kidney disease, a potentially fatal condition she inherited from her father. She tried medication to slow down the disease, but it continued to progress.

By 2003 she was placed on dialysis. At age 65, she was put on a transplant list and told it would likely take up to five years to find a match.

After 12 months, a match was found and she received two kidneys from a 2-year-old who had died.

Her surgery was performed 9 p.m. Feb. 15, 2004 at Charleston Area Medical Center’s General Division. When her pastor came to pray with her before the operation, she told him she was not afraid because she would be fine whether she continued life on earth or in heaven.

“I told him ‘if anything happens, I am going to the best home I ever had.’” she said.


She asked hospital staff if someone else could have those healthy kidneys if she did not survive. She was told it was necessary to be registered as an organ donor before that could happen. She was surprised to learn that, despite health issues, many other parts of her body could be used as well. She immediately became an organ donor.

While she continues to take necessary medications to care for her new kidneys, they have remained healthy and continued to grow.

Her life was saved thanks to a family she may never know because the entire process is confidential. However, she thinks of that family often and strives to do what she can to help others by working hard as an advocate for organ donation. Prior to the surgery, she was very shy but has forced herself to become an extrovert in order to be a good speaker. [Read more]




From ABC News, Good Morning America

Drinking Gallon of Iced Tea Daily May Have Caused Arkansas Man's Kidney Failure, Doctors Say

Doctors believe a man's excessive iced tea drinking -- about a gallon daily -- is what caused his previously unexplained kidney failure last year that's been keeping him on dialysis ever since.

The unidentified 56-year-year-old man was admitted to John L. McClellan Memorial Veterans Hospital in Little Rock last May with weakness, fatigue, body aches and an elevated serum creatinine level, which meant his kidneys weren't functioning properly, according to the doctors' letter published in the New England Journal of Medicine Thursday.



The patient's urine sample was "remarkable" for its abundance of calcium oxalate crystals, Dr. Fyed Syed and Dr. Alejandra Mena-Gutierrez told ABC News today. Both doctors treated the patient and co-authored the letter along with Dr. Umbar Ghaffar, who was not immediately available for comment.

Calcium oxalate crystals are molecules that can put someone at high risk to develop kidney stones, Syed and Mena-Gutierrez said.

High levels of oxalate in the body is usually due to a genetic disorder, complications from gastric surgery or consumption of antifreeze, the doctors said, but the patient's history outlined in the letter didn't match any of those explanations.

An explanation was finally found after the patient admitted to drinking 16 eight-ounce glasses of iced tea daily, Syed and Mena-Gutierrez said, adding that black tea, "a rich source of oxalate," accounts for 84 percent of tea consumed in the U.S. [Read more]




PKD Awareness

From Clayton News Daily, Morrow, Georgia, By Johnny Jackson

Morrow students to walk, raise awareness of kidney disease

Assistant principal in search of kidney donor

MORROW— More than 800 students, parents and faculty of Morrow Middle are expected to walk Friday in support of Assistant Principal Derrica Davis, who has a story to tell about her search for a kidney donor.

Davis, 36, suffers from a rare kidney disease called polycystic kidney disease.

She will be the featured guest at the Morrow Middle School Kidney Walk is a daylong event that will take place on the school’s athletic fields.

She said she was diagnosed at 23. She has been on a waiting list for the past 14 months as the disease has progressed.

“I’m an only child, and I’m the child of an only child, so getting a donor is a challenge,” said Davis, the mother of a 6-year-old boy. “Only 1 percent of the population would be a match for me.”

She said she manages these days with 8 percent of her kidney functions.

“I don’t believe in holding on to hardships in life,” said Davis, who receives notifications with scriptures on her phone several times a day.

She said she tries to stay positive, even when she feels pain and fatigue from her condition.

“There is little help for my disease,” she said. “There is not a lot of research for people with my disease, so giving to organizations such as the National Kidney Foundation is important.”

Davis said residents with the O-blood type, who are interested in donating their kidney, may call the Piedmont Transplant Clinic at 404-605-4605.

“Even if they can’t help me, it’s quite possible they can help somebody else,” she said.

Learn more about Davis’s cause on Facebook by searching “akidneyfor.Derrica.” Also, find out what the school plans to do for its National Kidney Walk event May 30.




From Kokomo Perspective, Indiana, by Alyx Arnett
Knocking out polycystic kidney disease

Community to hold fund raiser to benefit Williams family April 11

Jeremy Williams


Nine years ago Jeremy Williams’ mother was in need of a kidney after she was diagnosed with polycystic kidney disease (PKD). She didn’t ask her son to donate a kidney, but Williams couldn’t sit back and watch her suffer, he said.

He went in for testing to see if he would be a good match to donate a kidney to his mother when, at age 30, he learned something he expected to hear—but not nearly this soon. He too had PKD.

“They did the scans and just by the questions that I asked the girl in the room that she wouldn’t answer, I could tell there was a problem,” Williams said, now 39. “I wasn’t really that upset at the time. I guess I more or less expected it.”

PKD is an inherited disorder that causes clusters of cysts to develop primarily within a person’s kidneys, which can lead to kidney failure. The chance of inheriting PKD is 50 percent, a statistic that rings true in Williams’ family.

His mother had three brothers, and out of the four of them, two had PKD. His uncle passed away from it in his early 40s. Williams’ grandfather also had it and died from it in his 40s.

“I guess I expected all the time growing up that if I was going to get it, I would have serious problems by the time I was in my 40s. I didn’t really expect that I would hear about it when I was 30,” he said.

After Williams’ initial diagnosis, he had his condition monitored twice a year. His kidney function was holding steady around 50 percent. But in December 2013, Williams found out his kidneys only were functioning at 17 percent, and within another year, his kidney function dropped to 9 percent. Williams said, best case scenario, it’s at 9 percent today, though he said it’s likely worse now. It can’t be tested anymore since Williams now undergoes daily peritoneal kidney dialysis.

“It’s so unexpected for me to be as bad as I was,” he said. “I had no reason to believe that I was in any way going to go this far down the hill. The doctor said she was surprised, that she hadn’t ever seen someone’s [kidney function] drop that fast.”

The unexpectedness came as a shock to the Williams family, as they were planning their family. Williams and his wife, Jenni, had their first child, Ashton, in 2003. That was several years before Williams knew he had PKD. He said he and his wife tried for years to have a child before resorting to in vitro fertilization.

“Through the in vitro process, we got her pregnant, and she had him,” he said.

Soon, Williams’ wife wanted more children, but Williams had found out he had PKD. Williams resisted for a while, but he said he caved at the point when he realized his disease wasn’t really affecting him other than high blood pressure at that time.

Through in vitro, Williams’ wife got pregnant again, this time with twins, who are now 2. It was soon after the children were born that Williams’ kidney function plummeted. [Read more]



From The Chattanoogan, Tennessee 

Flag Raising Honors 35 Erlanger Organ Donors In 2014

On Wednesday, officials with Erlanger will raise a “Donate Life” flag in the hospital courtyard to recognize and honor 35 Erlanger patients who gave the ultimate gift of life as organ donors in 2014.

Officials said, "Thanks to the organ donations of these 35 individuals, 123 lives were saved last year."

At 11 a.m., Erlanger will join thousands of hospitals and organizations across the nation by flying the flag in recognition of Donor Awareness Month, and in honor of the lives touched by organ, eye and tissue donation. Representatives from the Erlanger Health System, Tennessee Donor Services, Donate Life, Erlanger’s transplant program and other medical professionals will join Paula Palmer, a kidney transplant recipient, and Paula Boring, mother of an organ donor. [Read more]




Kidney Health

From Santa Monica Mirror, CA, by ALIA TUQAN, M.D

Filtering The Facts On Kidney Health

March is “National Kidney Month,” highlighting the role of our kidneys in maintaining overall health and the importance of keeping them healthy. This month’s column covers kidney health and disease and what can be done to protect these vital organs.

Our kidneys filter our body’s waste to produce urine. They help balance electrolytes, the charged particles needed for cell functioning. They also are involved in maintaining blood pressure and producing red-blood cells.

As we grow older, our kidneys slow down. Their ability to filter waste diminishes. That’s why older people are more susceptible to the effects of alcohol and medications.

Various diseases can decrease kidney function as well. Uncontrolled high blood pressure, also known as hypertension, and diabetes can damage kidneys. Less common diseases, such as lupus, an autoimmune disease, and polycystic kidney disease, a genetic disorder, can affect kidneys too.

In addition, certain medications can affect kidneys. Non-steroidal anti-inflammatory drugs (NSAIDs) – a class of pain medications, including over-the-counter ibuprofen and naproxen – can damage kidneys if taken for prolonged periods of time.

People who take diuretics – certain types of medications commonly used to treat high blood pressure and leg swelling – need periodic blood work to make sure electrolytes and kidney function remain stable on these drugs.

People with kidney disease can be more sensitive to drugs and susceptible to drug side effects because their kidneys no longer filter the body’s waste as efficiently as they once did. In addition, they are at risk of high blood pressure, low red-blood-cell counts, also known as anemia, and bone diseases.

What can you do to maintain kidney health and prevent kidney problems? These recommendations can help reduce your risk:

• Get regular checkups with your doctor, who may order blood tests and review medications.

• Get medical treatment for high blood pressure or diabetes.

• Take over-the-counter and prescriptions drugs as prescribed.

• Review herbal supplements with your doctor to ensure they are safe.

• Eat a healthy diet.

• Exercise regularly, but discuss any new activities with your doctor beforehand.

Follow these simple steps for better kidney health – and a healthier you!

Dr. Alia Tuqan is a board-certified geriatrician with the highly regarded UCLA Geriatrics Program in Santa Monica and Westwood. For more information, call 310.319.4371 or visitwww.uclahealth.org.




PKD Research

From MiBiz, Written by Mark Sanchez



Metabolic Solutions Development Co. plans to seek $25 million to $35 million in additional capital later this year to support the continued development of its new drug targets.

Founded in 2006 by former Upjohn Co. researchers Jerry Colca and Ralph Kletzien, the Kalamazoo-based Metabolic Solutions has already raised about $70 million from investors in the last seven to eight years to develop compounds to treat diseases connected to metabolic dysfunctions. In particular, the company has targeted compounds that could treat kidney, liver, Parkinson’s and Alzheimer’s diseases, as well as Type 2 diabetes.

The additional capital Metabolic Solutions will seek to raise toward the end of 2015 will “accelerate pipeline development and position (the company) for access to public markets in 2016,” CEO Stephen Benoit said during a presentation this month at the BioCentury Future Leaders event during the Biotech Industry Conference in New York City.

“We’re in a pretty good position to achieve a pretty significant inflection in value here over the next 24 months,” Benoit said. “The real benefit here in being where we are relative to what we need to accomplish over the next 24 months is quite a step up in enterprise value for investors.”

Current investors will “continue to participate going forward,” Benoit said.

MOVING BEYOND DIABETES

The company has redirected its lead compound, known as MSDC-0602, to be used on nonalcoholic steatohepatitis (NASH), a liver inflammation caused by the buildup of fat, and polycystic kidney disease (PKD), a genetic disorder where cysts develop in the kidneys and impair function. PKD presently has no treatment in the U.S.

An insulin sensitizer, the MSDC-0602 compound was proven to be safe and effective in treating Type 2 diabetic patients in a Phase 2 human clinical trial.

Given the regulatory requirements and an “almost complete inability to raise capital around an asset in Type 2 diabetes,” the company decided to refocus the compound around liver and kidney disease “where the timeline and the cost to market are a lot more reasonable,” Benoit said in the presentation.

Metabolic Solutions plans to move later this year into Phase 2b clinical trials for MSDC-0602 and another compound, called MSDC-0160, which has the potential to treat neurodegenerative diseases such as Parkinson’s and Alzheimer’s, Benoit said.

The Phase 2b study for MSDC-0602 will involve 320 NASH patients over 12 months in at least 60 sites globally and the same number of PKD patients over a year that will include sites in Europe and Japan.
PARTNERSHIPS AHEAD?

Metabolic Solutions should have “some interesting news in the next few months on the partnering front” involving the upcoming clinical studies on NASH and PKD and the capital raise as the company converts from a middle-stage to a later-stage drug development pipeline, Benoit said. [Read more]



From 7th Space Interactive

Urine Fetuin-A is a biomarker of autosomal dominant polycystic kidney disease progression

Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder characterized by numerous fluid-filled cysts that frequently result in end-stage renal disease. While promising treatment options are in advanced clinical development, early diagnosis and follow-up remain a major challenge.

We therefore evaluated the diagnostic value of Fetuin-A as a new biomarker of ADPKD in human urine.

Results: We found that renal Fetuin-A levels are upregulated in both Pkd1 and Bicc1 mouse models of ADPKD. Measurement by ELISA revealed that urinary Fetuin-A levels were significantly higher in 66 ADPKD patients compared to 17 healthy volunteers or 50 control patients with renal diseases of other causes .

Receiver operating characteristics (ROC) analysis of urinary Fetuin-A levels for ADPKD rendered an optimum cut-off value of 12.2 μg/mmol creatinine, corresponding to 94% of sensitivity and 60% of specificity (area under the curve 0.74. Furthermore, urinary Fetuin-A levels in ADPKD patients correlated with the degree of renal insufficiency and showed a significant increase in patients with preserved renal function followed for two years.

Conclusions: Our findings establish urinary Fetuin-A as a sensitive biomarker of the progression of ADPKD.

Further studies are required to examine the pathogenic mechanisms of elevated renal and urinary Fetuin-A in ADPKD.

Author: Nathalie PiazzonFlorian BernetLinda GuihardWouter N LeonhardSéverine UrferDmitri FirsovHassib ChehadeBruno VogtSophia PiergiovanniDorien PetersOlivier BonnyDaniel B Constam
Credits/Source: Journal of Translational Medicine 2015, 13:103