From Nephrology News
New polycystic kidney disease treatment method shows promise in mouse study
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
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
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.
Now Available in Canada: First-ever treatment for adults living with a life-threatening kidney disease (ADPKD)
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
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
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
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.
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
From PKD Foundation of Canada
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!
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.
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
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