From Business Wire
The Critical Path Institute’s Polycystic Kidney Disease Outcomes Consortium Secures FDA Qualification for Enrichment Biomarker in Autosomal Dominant Polycystic Kidney Disease (ADPKD)
ADPKD is a debilitating genetic disease affecting approximately 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. There is only one medication developed specifically to treat ADPKD, called tolvaptan, which has not been approved in the United States.
“The current gold standard for evaluating kidney damage in PKD is Glomerular Filtration Rate which does not change appreciably until very late in the disease stage. This presents a challenge for drug developers because it requires a clinical trial with patients in late stages of the disease or a clinical trial that runs for decades,” says C-Path Chief Operating Officer and PKDOC Co-Director Steve Broadbent. “The qualification of TKV imaging as a prognostic biomarker is a fundamental step towards enabling pharmaceutical companies to develop new therapies for a disease that, as of now, has no available treatments approved for use in the United States.”
TKV is a measurement of the impact of ADPKD on the size of the kidneys and is considered to be predictive of a future decline in kidney function. This draft guidance provides qualification recommendations for the use of TKV, measured at baseline, as a prognostic enrichment biomarker to select patients with ADPKD at high risk for a progressive decline in renal function (defined as a confirmed 30% decline in the patient’s estimated glomerular filtration rate (eGFR)). The use of TKV as a biomarker – along with the patient’s age and baseline eGFR – can help those conducting clinical trials in finding appropriate candidates, potentially improving the accuracy and efficiency of those trials.
To arrive at this determination, the PKDOC first 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. [Read more]
PKD Awareness
From Channel 28, Fox Affiliate, Columbus, OH
Teen Cheerleaders Unite to Raise Awareness about Kidney Disease
Two Delaware County teenagers are leading a battle against life-changing disease they both face: kidney disease.
Sara-Ann Dietrich and Katherine Coomes met as teammates on the cheerleading squad. Each quickly learned they share a similar condition.
Sara-Ann Dietrich and Katherine Coomes met as teammates on the cheerleading squad. Each quickly learned they share a similar condition.
Sara-Ann, a freshman at Delaware Hayes High School, has Polycystic Kidney Disease, or PKD. The large number of cysts in her kidney will progressively decrease kidney function.
Katherine Coomes, a sophomore, has Focal Segmental Glomerulosclerosis, which causes the kidney's filters to scar, also decreasing kidney function.
"If I get a bad report from the doctor I can talk to Sarah and she understands," said Coomes. "She is on the same road as I am."
The 14-year-old Dietrich responded the same. "We go through some of the same stuff, and it's cool to share that some of that stuff with her," said Dietrich.
The girls are now on a mission to educate fellow students about the disease.
"Other diseases are more popular and I feel like kidney disease isn't paid as much attention to because people don't know about them," said Coomes.
"Other diseases are more popular and I feel like kidney disease isn't paid as much attention to because people don't know about them," said Coomes.
A walk to raise awareness about PKD and to raise funds to fund research will be held in Columbus, October 3.
For more information and to register for the walk, you can go to the website here.
A friend is also selling tees and sweatshirts, and will donate the proceeds to the National Kidney Foundation in the girls' names. Find more information about that fundraiser here.
From KLAS-TV, CBS Affiliate, Las Vegas, NV, By Patranya Bhoolsuwan
The phrase "Vegas Vacation" means a lot of things, but for most visitors, it does not mean raising awareness of a disease.
Valen Keefer is using her stop through Las Vegas to raise awareness about Polycystic Kidney Disease (PKD).
PKD is one of the most common, life-threatening, genetic diseases, yet most have never heard of it.
There is no cure and no treatments available in the United States.
Keefer had both of her kidneys removed in college and received a transplant 13 years ago.
"This has become my passion of helping other people, so they don't feel alone and go through what I went through," she said.
Thursday is National PKD Awareness Day, so she and her family emblazoned their message on their van to help tell everyone who sees it that PKD exists.
Their message is one of hope with their chant, "PKD will not beat me."
From KENS-TV, San Antonio, TX
Did you know that a sick kidney can grow to be the size of a football? The "Polycystic Kidney Disease (PKD) Foundation" says that you can help people with this disease get a donor. Join them on September 12 and "Walk For PKD".
Living With PKD
From McGill University Health Centre, Montreal, Canada
The Journey of acceptance and determination of a woman living with PKD
From the time she was a young girl, Diane* knew she had a 50/50 chance of having polycystic kidney disease (PKD), an incurable genetic condition in which fluid-filled cysts develop in the kidneys and affect their function. Over time, PKD can lead to renal failure and require dialysis or a kidney transplant. Diane was in her mid-forties when she was diagnosed.
“I inherited the defective gene from my father and saw how badly he coped with his condition, and that’s why I made important decisions early on. I told my doctor I wanted to avoid dialysis and I asked him to put me on the waiting list for a kidney transplant.”
Unfortunately, things didn’t work out quite that way for Diane. She didn’t get on the list fast enough and had to go on dialysis while waiting for a kidney. Throughout that time, Diane hardly missed a day of work as owner of an importing company.
“I would get up very early and go to the Royal Victoria Hospital for the dialysis. I was functioning well and was able to take care of my two kids,” she says. “My condition wasn’t something I wore on my sleeve.”
A diligent approach
Diane thinks patients with PKD should be diligent about their disease.
“You have to find the right doctor and ask the important questions. You have to push yourself, because otherwise you’ll reach a point where you’re too sick and have no other choice but to go on dialysis.”
Dr. Dana Baran, a nephrologist at the Royal Victoria Hospital of the McGill University Health Centre (RVH-MUHC) and Diane’s physician, agrees that patients need to be proactive and seek the best and most up-to-date information about their disease.
“Patients with PKD, or with a family history of the disease, should be referred to a tertiary care centre for the first discussions about the condition,” Dr. Baran says. “We also look at medical issues that could have an impact on the progression of the disease.”
According to Dr. Baran, patients should have yearly check-ups, even if they have no symptoms. As more and more cysts develop, the kidney enlarges and symptoms can arise: pain, bleeding in the urine, kidney stones, urinary tract infections and high-blood pressure. After a while, patients can show signs of progressive kidney failure and will need dialysis or transplantation.
A year after she started dialysis, Diane underwent a successful kidney transplant. She must maintain a healthy lifestyle and take anti-rejection medication for the rest of her life. In spite her difficult journey, Diane believes patients living with PKD should never despair.
PKD Research
Dialysis device developed by Imperial team a step closer to patients
Living With PKD
From McGill University Health Centre, Montreal, Canada
The Journey of acceptance and determination of a woman living with PKD
From the time she was a young girl, Diane* knew she had a 50/50 chance of having polycystic kidney disease (PKD), an incurable genetic condition in which fluid-filled cysts develop in the kidneys and affect their function. Over time, PKD can lead to renal failure and require dialysis or a kidney transplant. Diane was in her mid-forties when she was diagnosed.
“I inherited the defective gene from my father and saw how badly he coped with his condition, and that’s why I made important decisions early on. I told my doctor I wanted to avoid dialysis and I asked him to put me on the waiting list for a kidney transplant.”
Unfortunately, things didn’t work out quite that way for Diane. She didn’t get on the list fast enough and had to go on dialysis while waiting for a kidney. Throughout that time, Diane hardly missed a day of work as owner of an importing company.
“I would get up very early and go to the Royal Victoria Hospital for the dialysis. I was functioning well and was able to take care of my two kids,” she says. “My condition wasn’t something I wore on my sleeve.”
A diligent approach
Diane thinks patients with PKD should be diligent about their disease.
“You have to find the right doctor and ask the important questions. You have to push yourself, because otherwise you’ll reach a point where you’re too sick and have no other choice but to go on dialysis.”
Dr. Dana Baran, a nephrologist at the Royal Victoria Hospital of the McGill University Health Centre (RVH-MUHC) and Diane’s physician, agrees that patients need to be proactive and seek the best and most up-to-date information about their disease.
“Patients with PKD, or with a family history of the disease, should be referred to a tertiary care centre for the first discussions about the condition,” Dr. Baran says. “We also look at medical issues that could have an impact on the progression of the disease.”
According to Dr. Baran, patients should have yearly check-ups, even if they have no symptoms. As more and more cysts develop, the kidney enlarges and symptoms can arise: pain, bleeding in the urine, kidney stones, urinary tract infections and high-blood pressure. After a while, patients can show signs of progressive kidney failure and will need dialysis or transplantation.
A year after she started dialysis, Diane underwent a successful kidney transplant. She must maintain a healthy lifestyle and take anti-rejection medication for the rest of her life. In spite her difficult journey, Diane believes patients living with PKD should never despair.
PKD Research
From Imperial College London, by Colin Smith
A project to develop technology that could improve outcomes for people with kidney disease received a boost, following a £1 million grant.
More than 2.5 million people worldwide have kidney conditions that require their blood to be routinely filtered by a dialysis machine. Most patients undergo a surgical procedure to prepare their veins for coping with the filtration process. This involves a surgeon creating a connection in the arm, called a fistula, between an artery and a vein, which is then connected to a dialysis machine. Although they are the gold standard in medical care, fistulas are unreliable, often blocking up and requiring repeated costly repair operations. In the US alone, $US 4.6 billion is spent annually to treat failing fistulas.
Now researchers from Imperial College London are developing the first minimally invasive procedure that uses a synthetic tube, called a stent graft, to form the fistula for dialysis. The team have also developed the prototype technology for inserting the stent graft between a patient’s artery and vein with precision and accuracy.
The device is called an Electronic Percutaneous Anastomosis Technology for Haemodialysis or ePATH. The researchers have received the £1 million funding from the NHS National Institute for Health Research to further develop the system and take it through patient trials.
The ePATH system will help some patients with kidney failure to avoid open surgery before beginning dialysis treatment. This is because the ePATH catheter system creates a vascular access site in a minimally invasive manner. This would mean less trauma for patients, leading to faster recovery times, and them being able to use dialysis machines sooner.
A St. Louis company is moving closer to releasing a first of its kind product to help people avoid devastating kidney problems. Dr. Kevin Martin specializes in kidneys and also works for MediBeacon in Creve Coeur. Dr. Martin said, “I think it’s very exciting it’s a huge development in the field.”
Employees at MediBeacon are very excited about a yellow liquid. It glows in the presence of light and could dramatically prevent kidney damage in tens of millions of people.
Dr. Martin said, “One would know within a short period of time within in minutes if the kidney is having trouble.”
Currently a patient has to undergo a series of blood tests, which can take hours or even days, before doctors know if the organs that rid the body of toxins are failing. And even that can be a guessing game. Dr. Martin said, the quicker you can correct the injury to the kidney the better off the patient would be.” Failing kidneys can lead to dialysis or even death. Steven Hanley is the CEO of MediBeacon, “With ours you’ll notice with an hour how your kidneys are doing.” It’s as simple as holding a light against the patient’s body. Here’s how the new system works. It has already passed some human trails.
The patented agent, the yellow liquid, is injected into the patient. A light is held against the person’s skin, a doctor can determine in real time if the kidneys are working and properly ridding the body of waste, they don’t have to wait and guess.
The liquid is nontoxic and is not radioactive. This start-up bio tech company could be good for St. Louis’ future. Hanley said, “We have less than ten employees today we expect that double we’re going to be using chemists and very highly educated people.”
He said he hopes to have the state of the art kidney monitoring system in hospitals by the end of 2017.
From Nature Reviews Nephrology, by Friedhelm Hildebrandt
Employees at MediBeacon are very excited about a yellow liquid. It glows in the presence of light and could dramatically prevent kidney damage in tens of millions of people.
Dr. Martin said, “One would know within a short period of time within in minutes if the kidney is having trouble.”
Currently a patient has to undergo a series of blood tests, which can take hours or even days, before doctors know if the organs that rid the body of toxins are failing. And even that can be a guessing game. Dr. Martin said, the quicker you can correct the injury to the kidney the better off the patient would be.” Failing kidneys can lead to dialysis or even death. Steven Hanley is the CEO of MediBeacon, “With ours you’ll notice with an hour how your kidneys are doing.” It’s as simple as holding a light against the patient’s body. Here’s how the new system works. It has already passed some human trails.
The patented agent, the yellow liquid, is injected into the patient. A light is held against the person’s skin, a doctor can determine in real time if the kidneys are working and properly ridding the body of waste, they don’t have to wait and guess.
The liquid is nontoxic and is not radioactive. This start-up bio tech company could be good for St. Louis’ future. Hanley said, “We have less than ten employees today we expect that double we’re going to be using chemists and very highly educated people.”
He said he hopes to have the state of the art kidney monitoring system in hospitals by the end of 2017.
Advances in genome sequencing and genetic manipulation techniques over the last decade have helped identify numerous single-gene causes of early-onset kidney diseases and risk alleles for complex, polygenic traits. Subsequent studies regarding the underlying disease mechanisms will help lead to personal genetic diagnoses and unique therapeutic interventions in the future.
Gift of Life
From Burlington Times, Burlington, NC. By Bill Cresenzo
John Blythe of Burlington, right, donated a kidney to one of his employees, Rick Ingoglia, recently in Orlando, Fla. Blythe's job as a district manager for a music instrument company takes him up and down the east coast. Ingoglia works as a shop manager in Florida.
Rick Ingoglia got a very big bonus from his boss, John Blythe of Burlington, this year: a kidney.
Blythe is the district manager of repair for instrument company Music & Arts, covering a region that spans from Virginia to Florida. Ingoglia is a shop manager and repairs brass instruments in Orlando.
They have been friends and colleagues for about seven years.
Ingoglia, 58, has polycystic kidney disease, meaning that he has cysts on his kidneys that with time, multiply and get larger.
Earlier this year, Ingoglia called Blythe to let him know that his kidney function was worsening and that he would have to take time off, advising that his kidneys were functioning at 20 percent and he would have to start dialysis unless he had a transplant.
He had just put his name on a kidney transplant list, along with thousands of others.
Dialysis is tedious and arduous. Most patients spend several hours three days a week as a machine filters their blood.
“The average wait for my blood type is two to three years for a cadaver kidney,” Ingoglia said. “I surely would have been on dialysis by now and would have remained on dialysis until I got a new kidney. Dialysis is no picnic, especially for working people like myself.”
Through previous conversations, Blythe knew that Ingoglia had a Type “B” blood.
“I said, ‘Rick, I’m going to check to see if I am a possible match, but I can’t make any promises.”
Ingoglia was optimistic.
“Going forward both of us had a very very good feeling about it: We’re both Christians, we’re both believers and we believed it was going to work out.”
In June, Blythe traveled to Florida and went through a battery of tests at the Orlando Transplant Center. Everything was clear and surgery was a go.
Naturally, Blythe and his family were worried about the possible health implications of giving away a kidney. What if his other kidney failed? What if, God forbid, his daughter ever needed a kidney?
“According to the transplant center and the kidney foundation, if I were ever to have problems or need a transplant of my own, I would have priority,” he said. My wife and I talked through process and decided that if God put me in the position to make a donation for somebody else, God would put someone in my daughter’s life to cover that need. You step out in faith.”
In July, Bythe traveled back to Orlando — his collegues helped with travel expenses.
The transplant occurred on July 22. The surgery lasted about four hours, with Blythe and Ingoglia side-by-side. Surgeons performed it laparoscopically, meaning that Blythe only has a small incision near his abdomen.
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