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Progress slow but steady for cell, gene therapies for AMD
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WAIKOLOA, Hawaii — Progress in cell and gene therapies for the treatment of wet and dry age-related macular degeneration is evidenced by advancement, albeit slow, of clinical trials, according to a speaker here.
Janssen and Ocata are both initiating phase 2 trials of cell therapy for atrophic AMD, and a phase 2 study of NeuroTech’s implant platform that combines cell and gene therapy for treatment of wet AMD is ongoing, Allen C. Ho, MD, told colleagues at Retina 2016.
Allen C. Ho
“Part of why we may be currently have a hiccup in gene therapy for treatment of advanced AMD is that, for all these biologic therapies, there are many more variables in terms of what can go wrong or what may lead to lack of response,” Ho said, citing vectors, cell lines, delivery systems and surgical procedures as some of the factors. “These are some of the reasons why things take longer.”
The Ocata AMD phase 2 study is expected to enroll 60 patients with geographic atrophy secondary to AMD, he said. The treatment arm will undergo transplantation of 200,000 retinal pigment epithelium cells in the subject’s study eye. The study’s objective is to evaluate the safety of three regimens of short-term, low-dose systemic immunosuppression therapy as rejection prophylaxis prior to or following transplantation, according to Ho’s presentation.
In the Janssen phase 2b randomized controlled masked study of CNTO 2476 human umbilical tissue derived cells, 210 patients with geographic atrophy are expected to be enrolled. Subjects will be randomized 1:1:1 to receive 60,000 cells, 300,000 cells or sham procedure. Efficacy end points will be both functional and anatomic, Ho said.
In the NeuroTech trial, 90 subjects are expected to be enrolled. A single NT-503 Encapsulated Cell Technology implant will be compared for noninferiority to Eylea (aflibercept, Regeneron) dosed every 8 weeks in patients with demonstrated response to anti-VEGF, according to the presentation.
“This is intended to address burden of treatment or persistent injections after initial series of anti-VEGF injections,” Ho said. – by Patricia Nale, ELS
Reference:
Ho AC. Cell and gene therapy trials for age-related macular degeneration. Presented at: Retina 2016; Jan. 19, 2016; Waikoloa, Hawaii.
Disclosure: Ho reports he receives research grant funding and is a consultant for Avalanche, Genentech, Janssen/Johnson & Johnson, NeuroTech and Regeneron.