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.

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 5-year $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 support, including 6 UC San Diego SSCI Endowed Chairs, and substantial clinical and research infrastructure support from UC San Diego Health and the offices of the UC San Diego Chancellor, Vice Chancellor of Health Sciences, and Vice Chancellor of Research. Directed by Professor Catriona Jamieson, the SSCI 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 Center; 5) the Sanford Stem Cell Fitness and Space Medicine Center; 6) the Sanford Stem Cell Innovation Center and 7) Brain Tumor and Neuro-restoration Program. 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 Alpha Clinic 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. Our Alpha Clinic has 39 active clinical trials with steady increases in accrual and a total of 155 patients enrolled, with two-thirds being industry-sponsored and one-third investigator-initiated trials (IITs), spanning neurodegenerative disorders, cancer, infectious diseases, and metabolic diseases. During the reporting period the UC San Diego CIRM Alpha Clinic registry came online. Aims of the registry are to recruit patients for current and future clinical trials, inform long term safety of products, identify areas of need for patient concerns, public outreach, and educational development. Our first patient was recently treated in the Phase 1 study in collaboration with City of Hope using CAR T HIV cure strategy. This is an incredible operational success for the Alpha Clinic network as well as pilot for the greater adoption of regenerative medicine multisite research collaborations across California. We launched an IIT with Sumitomo using human induced pluripotent stem cell-derived dopaminergic progenitors and will treat our first patient in the coming weeks. In close collaboration with the Advanced Therapy Center in the Alpha Clinic Manufacturing Network, we successfully launched a Tumor-Infiltrating Lymphocyte (TILs) as a new standard of care. Our Stem Cell Genomics and Bioinformatics Core published 30 peer-reviewed articles, completed CIRM-grant funded pre-INDs and INDs, and spun out 1 new start-up company. Our CIRM Clinical Fellowship has enrolled 4 fellows (Neurosurgery, Infectious Diseases, Neurology, Oncology); Sanford Surgical Center has now enrolled 2 fellows and performed 12 large animal model surgical training sessions; Molecular Imaging Core is developing methods to track cell therapy product in the body with 1 tracer in development. To enhance understanding of stem cell research and the potential to deliver a paradigm shift in clinical practice with our scientific, clinical, and community stakeholders, we have held many recurring events, which have drawn considerable attention. Of note, is the 10th annual Sanford Stem Cell Institute Symposium featured a focused panel discussion from the Alpha Clinic leaders from across the Alpha Clinic Network, keynote talks from 3 Nobel laureates, a Gairdner Prize winner and former President of ISSCR, which is now available for viewing on UCTV and YouTube. We have been exploring and expanding partnerships by adding trial experts with the requisite domain expertise to enhance clinical trials development and accrual in our health system, as well as the alpha clinic network, our region, and beyond. In summary, our Alpha Clinic continues to accelerate development of infrastructure to translate both therapeutic and molecular diagnostic stem cell discoveries. With rigorous attention to quality, we continue to enhance clinical research and trials for patients in our health system, California’s Alpha Clinic Network, and beyond.