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RP is a blinding eye disease that affects approximately 150,000 people in the United States. It is caused when the light-sensing cells located in the back of the eye called photoreceptors are lost due to a localized form of degeneration. There is currently no treatment for the condition, and it leads to gradual vision loss and eventually blindness.
In previous clinical trials, also funded by CIRM, Klassen and his team of researchers used human retinal progenitor cells (hRPCs) to treat the disease in study subjects. Injected into the gel-filled space in the center of the eye, the progenitor cells secrete neurotrophic factors that have been shown to rescue weak or dormant photoreceptors, slowing and potentially reversing the degenerative process.
“CIRM funds and accelerates promising early stage research, through development and to clinical trials,” says Maria T. Millan, MD, president and CEO of CIRM. “Programs, such as this, that are novel stem cell or gene therapy approaches addressing a small number of patients, often have difficulty attracting early investment and funding. CIRM’s role is to de-risk these novel regenerative medicine approaches that are based on rigorous science and have the potential to address unmet medical needs. By de-risking programs, CIRM has enabled our portfolio programs to gain significant downstream industry funding and partnership.”
In the recently completed Phase 2b trial, more than 80 subjects were randomized into three treatment arms: a control/sham arm, a medium-dose arm and a high-dose arm. The subjects were monitored over a 12 month period to assess changes in vision function and functional vision. Following completion of the 12 month masked study, subjects randomized into the control group were also given the opportunity to receive cells. This Phase 2b trial showed promising results, as well as a continued strong safety profile.
“I feel so blessed to be born at this time,” said Rosie Barrero, one of the patients in the first trial. She reports being able to see the time on her cell phone, something that hadn’t been possible in many years.
“While not a cure for retinitis pigmentosa, these progenitor cells appear to be a potentially effective and relatively sustained treatment,” said Klassen. “However, patients will still need to continue receiving injections.”
The new grant from CIRM will support repeat dosing in a subset of the previously treated patients, as well as ongoing cell manufacturing activities.
About the UCI School of Medicine
Each year, the UCI School of Medicine educates more than 400 medical students, and nearly 150 doctoral and master’s students. More than 700 residents and fellows are trained at UCI Medical Center and affiliated institutions. The School of Medicine offers an MD; a dual MD/PhD medical scientist training program; and PhDs and master’s degrees in anatomy and neurobiology, biomedical sciences, genetic counseling, epidemiology, environmental health sciences, pathology, pharmacology, physiology and biophysics, and translational sciences. Medical students also may pursue an MD/MBA, an MD/master’s in public health, or an MD/master’s degree through one of three mission-based programs: the Health Education to Advance Leaders in Integrative Medicine (HEAL-IM), the Leadership Education to Advance Diversity-African, Black and Caribbean (LEAD-ABC), and the Program in Medical Education for the Latino Community (PRIME-LC). The UCI School of Medicine is accredited by the Liaison Committee on Medical Accreditation and ranks among the top 50 nationwide for research. For more information, visit som.uci.edu.
Original post https://alertarticles.info
