Dr. Donald Kohn (right) with Seersha (ADA SCID) and her parents Stephen and Shayla
When Seersha was born an investigational gene therapy, a one-time procedure that the researchers say may provide lifelong results was only available in the United States through a waitlist at The University of California Los Angeles (UCLA). This treatment method is a welcome new option for children with ADA-SCID, who otherwise must undergo once- or twice-weekly injections of enzyme replacement therapy (ERT) or locate a matched bone marrow donor. In cases where no donor is found, ERT injections have been a lifelong necessity, but they don’t always work. Now, thanks to the hope provided by this clinical trial there is a better solution.
The gene therapy for ADA SCID has already proven to be highly effective as is described in the New England Journal of Medicine article “Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency” published May 27, 2021. The article describes how with 100% survivability, 48 of 50 ADA SCID children from the US and the UK were successfully treated in clinical trials prior to Orchard walking away from the commercialization of the product. The goal following a successful patient clinical trial is to present your data to the FDA for approval for commercial production of the product. Orchard claimed that the cost to bring the product to market was too high for a treatment that can only treat such a small, ultra-rare disease patient population. Eventually, Orchard agreed to return the license to UCLA and University College London (UCL), however, it took additional time for doctors at UCLA to obtain all the necessary approvals internally from UCLA and also from the FDA to once again begin treating patients. Currently, UCLA is the only location in the United States where this life-saving therapy is available to treat patients with ADA SCID.
Dr. Donald Kohn continues to treat as many patients as he can while looking for new financial avenues to bring ADA SCID gene therapy to more patients. Since restarting the trial, a small handful of patients have been treated and a few more from the lengthy waitlist are being scheduled for 2024. However, the restart of the clinical trial at UCLA is not a long-term solution, as the money to provide this therapy will run out. Without a commercial partner, the therapy cannot proceed through the FDA approval process. The California Institute for Regenerative Medicine (CIRM) has been key to the funding of this investigational gene therapy, but CIRM has placed a hold on applications requesting financing. Currently, all stakeholders of the SCID community are waiting and watching to see how this financial quandary will end. Can a successful clinical trial for a handful of ultra-rare disease patients be converted to a standard of care that will be available to all patients in that rare disease group or will the families who want this therapy for their children eventually be told, “We’re sorry, we can’t provide this therapy because it’s too expensive to produce?”
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