Stem cell-based treatment for retinitis pigmentosa is given the go ahead by the US FDA for clinical trial

A clinical trial testing a first-of-its-kind stem cell-based treatment for retinitis pigmentosa has been given the go ahead by the US FDA.

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May 15, 2015
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There is currently no treatment for retinitis pigmentosa, which causes blindness and affected more than 1.5 million people worldwide. A clinical trial testing a first-of-its-kind stem cell-based treatment for retinitis pigmentosa has been given the go ahead by the US FDA, and is a promising approach. The treatment aims to intervene when the photoreceptors (rods and cones) that are degenerating can be protected, and possibly reactivated.

Drs Henry Klassen, Jing Yang and their colleagues developed the treatment, and a startup company, iCyte Inc., cofounded by Klassen and Yang, will commercialize the therapy and administer the trial at University of California, Irvine (UCI; CA, USA). Their research project has been running for more than 10 years, and was accelerated when they received support from the state's stem cell agency, the California Institute for Regenerative Medicine (CIRM). They have received US$21 million.

By the middle of the year, the trial will enrol up to 16 patients at UCI with the possibility of enrolling more at a second site. They will be looking at the safety of a single injection of retinal progenitor cells. The injector will be administered into the eyes of patients with retinitis pigmentosa, and any changes ocular function will also be looked at.

"This milestone is a very important one for our project," said Klassen, "It signals a turning point, marking the beginning of the clinical phase of development, and we are all very excited about this progress."

"Without the backing of CIRM and the people of California, we would have never made it this far this quickly," Klassen commented. "To the patients and their families who have been waiting all these years, I am delighted to finally be taking our research out of the lab and into the clinic."

Klassen further explained that the agency also tutors grantees in many aspects of translational development, especially during the later preclinical phase, where much is at stake.

"One of CIRM's goals is to provide the support that promising therapies need to progress and ultimately get into clinical trials with patients," said Jonathan Thomas, chair of the agency's governing board.

– Written by Emily Hargrave

Source: UCI receives FDA consent to study stem cell-based treatment for retinitis pigmentosa in clinical trial: www.news-medical.net/news/20150508/UCI-receives-FDA-consent-to-study-stem-cell-based-treatment-for-retinitis-pigmentosa-in-clinical-trial.aspx

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