Advanced Cell Technology, Inc.(ACT), a biotechnology company applying cellular technology in the field of regenerative medicine, has announced that its clinical partner, the University of California, Los Angeles (UCLA), has received the US Food and Drug Administration (FDA) approval for its Investigational New Drug (IND) Application to initiate a phase I/II study using ACT’s retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs) to treat myopic macular degeneration (MMD, or myopia), commonly known as nearsightedness.
The study is led by Steven Schwartz, MD, Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at UCLA’s Jules Stein Eye Institute.
The primary focus of the study will be to evaluate the safety in patients with severe myopia of the type that causes fissures in the RPE layer of the eye. Dr Schwartz is the principal investigator in each of ACT’s two phase I/II clinical trials for Stargardt’s macular dystrophy and dry age-related macular degeneration (dry AMD) using RPE cells derived from hESCs. The approval was announced by Dr Schwartz at Bascom Palmer Eye Institute’s tenth annual angiogenesis meeting, “Angiogenesis, Exudation, and Degeneration 2013”.
“We are encouraged by Dr Schwartz’s keen interest in the evaluation of ACT’s RPE cells in potentially treating myopia,” commented Gary Rabin, chairman and CEO of ACT. “We are pleased to be on track to broaden the scope of our RPE program with the initiation of the new Investigator IND. Nearsightedness is a very common condition, worldwide, and it is estimated that roughly 30 per cent of cases are high, or severe, myopia.”
The myopia clinical trial will follow a similar protocol as the company’s three other human clinical trials in the US and Europe using hESC-derived RPE cells to treat forms of macular degeneration. The trial will enroll a total of 12 patients, with cohorts of three patients in an ascending dosage format. The trial is a prospective, open-label study designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with myopia at 12 months, the study’s primary endpoint.
Preliminary results from the US Stargardt’s and Dry Age Related Macular Degeneration trials were reported in The Lancet earlier this year.
“We look forward to initiating the clinical trial,” commented Dr Schwartz. “Myopic degeneration is an increasingly important global cause of permanent central vision loss for which there is no accepted treatment. Applying our key learnings from the ongoing ACT-sponsored stem cell trials allows the research promise of regenerative medicine to include myopic vision loss.”
Robert Lanza, MD, ACT’s chief scientific officer, said, “Myopia is one of the most common medical ailments in the world and myopic patients have a higher risk of permanent vision loss due to complications such as fissures in the RPE layer of the eye. We are anticipating Dr Schwartz’s evaluations, potentially leading to a treatment for those patients at high risk of vision loss from this condition.”
Jules Stein Eye Institute at UCLA created a world-renowned centre dedicated to the preservation of vision and the prevention of blindness. The Institute’s comprehensive programmes for the care of patients with eye disorders, research in the vision sciences, education in the field of ophthalmology and outreach to the community, coupled with its state-of-the art facilities, have brought national and international recognition to UCLA and the Institute, as it continues its mission to advance ophthalmology worldwide.