UC San Diego Health CIRM Alpha Stem Cell Clinic

The UC San Diego CIRM Alpha Clinic funded through a $8 million CIRM grant with $8 million in matching funds from the UC San Diego Sanford Stem Cell Clinical Center (SSCCC) serves as the fulcrum for Cell and Regenerative Medicine clinical studies at UC San Diego. The SSCCC was established in 2013 with a $100 million gift from T. Denny Sanford that was predicated on obtaining the first Alpha Clinic grant in 2015. Based on a 3-fold return on investment with regard to contracts, grants, and philanthropy as well as successful clinical trials and 2 FDA approvals that were supported by the UCSD CIRM Alpha Clinic and obtaining the second CIRM Alpha clinic grant, a $150 million philanthropic donation was provided by T. Denny Sanford to establish the Sanford Stem Cell Institute (SSCI) in September 2022 combined with substantial stem cell research faculty and infrastructure support from the offices of the UC San Diego Chancellor, Vice Chancellor of Health Sciences, Vice Chancellor of Advancement, and Vice Chancellor of Research. Directed by Professor Catriona Jamieson, the Sanford Stem Cell Institute includes: 1) the Sanford Stem Cell Clinical Center; 2) the Sanford Stem Cell Discovery Center; 3) the Sanford Advanced Therapy Center; 4) the Sanford Integrated Space Stem Cell Orbital Research (ISSCOR) Center; 5) the Sanford Stem Cell Fitness and Space Medicine Center; 6) the Sanford Stem Cell Innovation Center. The UC San Diego CIRM Alpha Clinic, directed by Professor Catriona Jamieson, is the “jewel in the crown” of the Sanford Stem Cell Clinical Center. For the benefit of patients in California and beyond, our UCSD CIRM ASCC objectives are to accelerate 1) development, 2) implementation, and 3) completion of innovative and accessible regenerative medicine clinical trials related to three major disease areas, including therapeutically recalcitrant malignancies, metabolic diseases, and neurological disorders. During this reporting period, the UCSD CIRM ASCC has continued to accelerate accrual to ongoing studies in neurology, oncology, cardiovascular disease, gastroenterology, orthopedics, and infectious diseases. Specifically, we would like to focus on three pivotal trials. First, enrollment in the CIRM-funded (CLIN2-13355) Neurona Therapeutics Phase l/ll trial involving intracerebral administration of NRTX-1001, a GABAergic stem cell product, for patients with drug-resistant mesial temporal lobe epilepsy (NCT05135091), has been led by UCSD site PI Professor Shih. This trial was highlighted on National NBC News because of early results showing reduced seizure activity and improved memory in study participants. This national NBC News story (https://health.ucsd.edu/news/press-releases/2023-07-26-new-uc-san-diego-health-among-first-in-nation-to-perform-regenerative-brain-cell-procedure-for-epilepsy/) shone a national spotlight on the importance of Regenerative Medicine clinical trials and the CIRM Alpha Clinic program. Second, the SSCCC and Alpha Clinic team supported NIH UO1-funded (NIH/NIAAAU01AA029019) phase 1 study of guselkumab (NCT04736966), an IL-23 inhibitory monoclonal antibody for the prevention of alcohol-related liver disease, including liver fibrosis and precancer stem cell generation, completed enrollment of all 12 participants. Third, the Genascence Phase 1b IL-1Ra-expressing mesenchymal stromal cell (MSC) gene therapy trial (GNSC-001) for patients with knee osteoarthritis, directed by Dr. Susan Bukata, has screened multiple patients and enrolled two patients who met eligibility criteria with the first treatment scheduled for February. This randomized, placebo-controlled, double-blind dose-finding Phase 1b clinical trial (NCT05835895) is evaluating the safety, tolerability, and pharmacodynamics of intra-articular administration of GNSC-001 in up to 50 patients with knee osteoarthritis in the US, including at the UCSD CIRM Alpha Clinic. In summary, the UC San Diego Alpha Clinic and the SSCCC continue to accelerate the discovery, development, and implementation of innovative research to enhance stem cell molecular diagnostic strategies and enhance precision regenerative medicine opportunities. The Alpha Clinic has seen high demand from both CIRM-funded and industry-sponsored trials that are expanding the diversity of disciplines that employ regenerative medicine approaches. The goal going into 2024 is to continue to work with researchers on CIRM and other funding agency grants to develop new products and investigator-initiated trials (IITs) and deliver a complete array of CRO services for both IIT and industry-sponsored clinical trials. We will expand Alpha Clinic operations to meet the demand of upcoming studies by increasing FTEs for project management, clinical trial coordination, regulatory support, data management, and monitoring. This will be supported in part by the SSCCC and SSCI. Since the inception of the SSCCC and UCSD CIRM ASCC, our team has enrolled 594 participants in 62 trials.