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27 May 2011

Alport Foundation Awards $100K to Study Rare Kidney Disease

The Alport Syndrome Foundation has awarded $100,000 to Dr. Laura Perin of the Saban Research Institute at Children’s Hospital Los Angeles to examine the use of Amniotic Fluid Stem Cells (AFSCs) as a novel approach to reversing fibrosis in Alport Syndrome.
The study will see if this approach provides a better understanding of how AFSCs modulate the molecular mechanisms that reverse interstitial fibrosis. Perin recently discovered evidence suggesting that a single injection of AFSCs has the capacity to reduce fibrosis, accompanied with an overall improvement of renal function and lifespan in mice.
Understanding how AFSCs behave in an in vivo environment will strengthen the foundation for translating this stem cell research into viable clinical therapies for the future and will possibly significantly delay or prevent End-Stage Renal Failure in Alport Syndrome patients.
AFSCs appear to be a valuable alternative to both embryonic and adult stem cells, offering a new approach to tissue repair and regenerative medicine. Stem cells were discovered in amniotic fluid donated by pregnant women in 2007, representing a novel and ground-breaking finding in the field. AFSCs are readily available via routine antenatal testing, or amniocenteses. AFSCs appear to be much more pluripotent (or able to generate a broad range of cell types) than adult stem cells, yet do not involve harming or destroying life at any stage, thus avoiding the ethical dilemma associated with the use of embryonic stem cells.
“We are very excited about Dr. Perin’s research” said Sharon Lagas, Alport Syndrome Foundation President. “Fibrosis leads to kidney failure and there are not any therapies available aimed directly at this process that can reduce its effects and prolong kidney function. We are hoping Dr. Perin’s research leads to advances in treating, delaying, or reducing fibrosis and kidney failure.”
Alport Syndrome is a genetic kidney disease affecting an estimated 1 in 5,000 people that causes progressive kidney deterioration and profound hearing loss. The majority of those affected by Alport Syndrome are young men who require dialysis or a kidney transplant by their early 20s.
The Alport Syndrome Foundation was launched in 2007 as a grassroots effort by families affected by the disease to provide a support community linking patients, medical providers and the scientific research community.

**published in "RENAL BUSINNES TODAY"

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