Ultra-Rare Affliction: Three Possible Patients for First CIRM Gene Therapy Identified at UCLA

UCLAis aware of three patients with a deadly, ultra-rare condition who could possibly take advantage of the first gene therapy that the state of California has helped to commercialize. In response to a question late last week,Donald Kohn, the UCLA scientist who led the clinical trial there for the treatment, said, 'We know of 3 LAD-1 patients who might be eligible for the therapy.' UCLA is the only site in the country for the treatment and is ready to continue when it is put on the market later this year. All nine of the children in the trial at UCLA were treated successfully, including three in one family. The condition is so rare that it is estimated to generate only 25 new cases a year in the United States. LAD-1 leaves children vulnerable to dangerous, recurrent bacterial and fungal infections. 'Survival beyond childhood is rare without treatment,' according to UCLA. The therapy was produced byRocket Pharmaceuticalsof New Jersey. TheCalifornia Institute for Regenerative Medicine (CIRM), which was created by state voters through a ballot initiative, awarded $5.9 million to the firm for the trial specifically at UCLA. Other trial sites were located in London and Spain. The provisions of the initiativerequire that recipients of clinical awards develop access and affordability plans for Californians. Rocket's plan deals with state residents who have 'no other means' to pay for the treatment. Estimated treatment costs indicate it could run upwards of $4 million. That does not include other, often quite large expenses for the families. It is not known whether any of the three potential patients fit the Rocket criteria for financial assistance. Tomorrow, CIRM has scheduledan online, public hearingon Rocket's plan that begins at 9 a.m. PDT. Written comments are also being accepted at legal@cirm.ca.gov until 5 p.m. tomorrow (Sept. 8). It is authoritative and independent and the only regular source of news about the $12 billion California research program. Subscribe paid today to theCalifornia Stem Cell Reportto help keep the news flowing. It costs less than a San Francisco latte and is better for you. CIRM expects to take action on the Rocket plan by Sept. 15, according to a CIRM response Friday to questions posed five days earlier. Rocket's access/affordability plan applies only to patients who are 'legal' residents of California, but it does not define what it means by 'legal.' California has different definitions of legal residency depending on their purpose. The six-page plan was heavily redacted, raising other questions about its meaning. In response, CIRM simply reiterated that the redactions complied with state law and CIRM regulations. The concealed information includes sections dealing with the proposal's scope, eligibility, insurance, documentation, patient and provider access, financial assistance, and governance and monitoring. TheCalifornia Stem Cell Reportasked CIRM whether it has queried 'patient groups or other interested parties to respond to the plan or the stateOffice of Health Care Affordability.' 'No,' said the CIRM statement. 'The IP (intellectual property) regulations provide that CIRM is responsible for reviewing the access plan and determining whether it satisfies the applicable IP requirements. We have, however, consulted with subject matter experts to inform our review and determination.' CIRM also said the plan is not subject to review by the CIRM board or its affordability committee, which includes outside parties. Both the board and the committee 'have been kept informed throughout the review process,' CIRM said. CIRM has investeda total of $12.7 millionin Rocket for clinical-stage work. In addition to the award for LAD-1 in 2019, the awards include $5.8 million for Danon disease in 2024 and $1 million for infantile malignant osteopetrosis in 2020. Thanks for readingThe California Stem Cell Report!This post is public so feel free to share it. Share Leave a comment