Donald Kohn, a longtime UCLA professor and researcher, is leading the development of the one-time gene therapy treatment.
His team, along with other researchers, published results last year after treating 50 patients with a type of severe combined immunodeficiency, or SCID, called ADA-SCID. All 50 children were alive for at least two years after treatment and more than 95% required no additional treatment.
Despite the success, a small biotech company gave up on Kohn’s therapy and re-licensed him earlier this year after failing to seek regulatory approval. As a result, at least 28 children with SCID are now on a waiting list hoping to access treatment.
Without gene therapy, doctors treat children with SCID using enzyme replacement therapies if they are not eligible for bone marrow transplants. Despite the availability of enzyme therapy, about one in five children with ADA-SCID die before they turn 20.
“buy time”
Earlier this month, the U.S. Food and Drug Administration gave Kohn permission to resume his studies, Kohn told Insider. David Jensen, an independent journalist for The California Stem Cell Report, first reported on the news.
In an email to Insider, Kohn said he expects to treat between three and six patients with his current funding. Kohn hopes that UCLA’s institutional review board will sign off on his research plans in January so he can begin enrolling patients. He hopes to treat the first patient in March and an additional patient every two to three months.
“Right now we are buying time,” Paola Andrea Fernndez de Soto Abdul-Rahim, the mother of a child with ADA-SCID, told Insider in May. “We can’t wait forever.”
Insider reached out to her to discuss the news of Kohn returning to college. She said she doesn’t know when doctors will be able to treat her son, but hopes he will get the chance sometime in 2023.
An uncertain future for rare disease treatments
Despite the positive update, much remains unclear about the future of Kohn’s treatment and gene therapies for rare diseases in general. Pharmaceutical companies have shown little interest in the treatments because the complexity of the treatments and the small number of eligible patients limit potential profits.
Kohn said UCLA and University College London, which control the intellectual property for this therapy, are in talks with several commercial-stage gene therapy companies about licensing the therapy.
Pharmaceutical companies have deprioritized or suspended gene therapy programs to treat a range of rare diseases, including Wiskott-Aldrich syndrome, MPS, Batten disease, chronic granulomatous disease, Rett syndrome, and Fabry disease. An expert previously called this deprioritization and suspension “the great abandonment” in a conversation with Insider.
Some researchers have begun discussing nonprofit models that could make these therapies accessible, but much of that work is still in the early stages. Kohn added that his team plans to hold discussions to see if they can also make this therapy available in a non-commercial, academic setting.
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