Children's Hospital Los Angeles Researcher Wins Grant to Study Stem Cells' Ability to Reverse Renal Fibrosis

Published on 
June 24, 2011

The Alport Syndrome Foundation Awards $100,000 to Laura Perin, PhD, for Her Novel Laboratory Investigations

MEDIA CONTACT:  Ellin Kavanagh at 323-361-8505
Laura Perin, PhD
Laura Perin, PhD, co-director of Research in the division of Pediatric Urology at Children's Hospital Los Angeles

LOS ANGELES (June 24, 2011) – Laura Perin, PhD, co-director of Research in the division of Pediatric Urology at Children's Hospital Los Angeles, has been awarded a $100,000 grant from the Alport Syndrome Foundation for her novel study of amniotic fluid stem cells (AFSCs) in reversing the fibrosis common to inherited kidney disorder.

Dr. Perin will use the grant to examine the underlying biological mechanisms by which stem cells influence the formation of fibrous tissue in Alport Syndrome, which damages tiny blood vessels in the kidneys that process waste. The inherited disorder, which has its origins in a genetic mutation of a protein in connective tissue, most often affects males and can progress to end-stage renal disease at an early age.

Dr. Perin, associate director of Research in the GOFARR Laboratory for Organ Regenerative Research and Cell Therapeutics at The Saban Research Institute has already made important discoveries about stem cells and Alport Syndrome.

Working in the lab, she recently found evidence suggesting that a single injection of AFSCs can reduce kidney fibrosis, accompanied with an overall improvement of renal function and lifespan. She also found that the injected stem cells create this beneficial effect by regulating the genes involved in the progression of fibrosis.

"Amniotic fluid stem cells may represent a new cell source for tissue and organ regeneration with important advantages," said Dr. Perin.

"Dr Perin's work with amniotic fluid stem cells will be pivotal in better understanding the natural progression of this disease which may enable viable therapies for the future,” said Roger De Filippo, MD, associate professor of Urology at Children's Hospital Los Angeles and director of the GOFARR Laboratory for Organ Regenerative Research and Cell Therapeutics at The Saban Research Institute.

For one, AFSCs appear to be more pluripotent (able to generate a broad range of cell types) than adult stem cells. Also, AFSCs sidestep any controversy involving embryonic stem cells and the use of embryos in research since they come from the amnionic fluid that surrounds a fetus during pregnancy and is discarded after delivery.

Going forward, Dr. Perin wants to find out whether multiple injections of AFSCs will have a greater impact on renal function and lifespan. She is also asking other, fundamental questions.

"Very little is known about the main mechanisms by which stem cells affect the progression of fibrosis in Alport Syndrome," said Dr. Perin. "In order to claim that stem cells can be used as an anti-fibrotic therapy, it's crucial that we understand these cellular mechanisms."

Her next-stage research will seek to identify which of the molecular signaling pathways that promote renal fibrosis are modulated by the injection of AFSCs.

The ultimate goal is to develop clinical applications for patients with this rare, life-threatening kidney disorder. Understanding how stem cells behave in an in vivo environment will set the stage for translating such laboratory-based research into viable clinical therapies, including finding a much-needed treatment that could delay or even prevent end-stage renal failure in Alport Syndrome patients and those with other forms of chronic kidney disease.

About Children’s Hospital Los Angeles
Children's Hospital Los Angeles has been named the best children’s hospital in California and among the best in the nation for clinical excellence with its selection to the prestigious US News & World Report Honor Roll.

Children’s Hospital is home to The Saban Research Institute, one of the largest and most productive pediatric research facilities in the United States and is one of America's premier teaching hospitals through its affiliation since 1932 with the Keck School of Medicine of the University of Southern California.

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