The USC+CHLA Alpha Clinic (AC) aims to be a leader in advancing and supporting the development of cell and gene therapies for people from all backgrounds.
During the current reporting period, we:
• launched the operational infrastructure,
• enhanced translational and clinical support capabilities,
• expanded our collaborative relationships with our investigators as well as other ACs specializing in cell and gene therapies,
• developed and offered new training and educational programs, and
• generated a comprehensive patient outreach and community engagement concept.
Below is a description of our progress and impact in three main areas.
Clinical Trial Operations:
Through collaborative efforts with the Southern California Clinical and Translational Science Institute (SC CTSI), Norris Comprehensive Cancer Center and the AC network, we created clinical trial intake protocols to support clinical trial activation. Our AC also assisted several investigators and companies in the preparation of clinical study proposals and product manufacturing. Our Good Manufacturing Practice (cGMP) facility has made significant strides by securing the CIRM INFR5 funding to enhance manufacturing capabilities and by joining the CIRM manufacturing consortium. Our group continues to meet regularly to discuss AC operational plans, team progress, and community engagement planning. We are currently supporting three active trials at USC and nine at CHLA.
USC clinical trial progress and outcomes:
At USC, the three trials are in the initial stages of activation. The Regenerative Patch Technologies trial should commence recruitment by September 2024. The Neurona Trial is pending IRB approval for recruitment and the team is working with our cGMP facility for processing the therapeutic product. The AC team has linked Abata Therapeutics with an eligible and interested principal investigator, and they are currently assessing site feasibility.
CHLA clinical trial progress and outcomes:
Trials at CHLA are focused on CAR T-cell therapy for childhood leukemia. To date the CIRM AC team has assisted with project oversight and logistical support, including biospecimen coordination. Support will be expanded when the AC Clinical Research Care Coordinator is fully hired.
Education:
1. Cell and Gene Therapy Development
Content: This annual 4-unit course provides training in cGMP manufacturing, quality management, and regulatory science related to cell therapy, complemented by hands-on training in our laboratories. Participants also engage in practical case studies during 15 weekly sessions over a four-month period.
Impact: The first offering was Spring 2023. Nine participants reported significant increases in understanding of translational requirements from the in-depth case studies and lectures/discussions with industry representatives and local expert instructors. There are currently 13 participants registered for Spring 2024.
2. Innovations in Regenerative Medicine
Content: This all-day symposium was co-sponsored by the CIRM AC and the Regulatory Support core of the SC CTSI. Experts from academia, government, and industry provided updates in regulatory aspects of regenerative products, including case studies in orthopedics and ophthalmology, as well as target identification for gene therapy and cell therapy manufacturing.
Impact: 82-96% of the 122 attendees had positive survey responses. Participants found the information useful, plan to implement it and are likely to recommend it to others.
3. International Conference on Relapse After Transplant and Cellular Therapy
Content: This 6th annual meeting of the ASTCT/EBMT was supported by a CIRM EDUC1 grant to AC site PI, Dr. Alan Wayne. The Conference brought together internationally renowned experts to share their knowledge and current research relative to the biology, natural history, prevention, and treatment of relapse with over 250 participants from around the world.
Impact: The majority (96%) of survey respondents agreed that the conference sufficiently met learning objectives, offering new information and skills to overcome treatment barriers, provide patient-centered care, and improve patient outcomes (https://www.astct.org/Attend/ASTCT-EBMT-Relapse-Conference).
Patient Outreach & Community Engagement:
Progress: We have developed a dual strategy for community outreach. One component will be focused on engaging and educating the lay community on cell and gene therapy in partnership with the SC CTSI. The second component will focus on engaging community health care providers in underserved communities in South Los Angeles. We are providing community outreach support for the Regenerative Patch Technology trial in macular degeneration mentioned above. We are also in discussion with other ACs on development of aligned and complementary approaches to community outreach and engagement.
Impact: Our community outreach efforts are in the planning phase, so we do not have specific impact to report yet.
Grant Application Details
Application Title:
Alpha Clinic Network Expansion for Cell and Gene Therapies
Public Abstract:
The CIRM Alpha Clinic will expand capacities and workforce expertise to accelerate development, clinical testing, and dissemination of novel cell and gene therapies for a broad range of conditions that impact Californians across the lifespan and from diverse backgrounds and underserved communities.
Statement of Benefit to California:
In 2020, Californians voted to increase their investment in regenerative medicine to address unmet medical needs. Expansion of CIRM’s Alpha Clinics Network is critical to achieving this goal. The Alpha Clinic will speed the development of exciting new cell- and gene-based treatments to improve health across the lifespan, from infants to seniors, and for a broad range of diseases that affect Californians from all backgrounds.
