Expansion of the Alpha Stem Cell and Gene Therapy Clinic at UCLA

The UCLA Alpha Clinic (AC) seeks to support CIRM’s strategic plan and integrated into the infrastructure of the long-standing Clinical and Translational Science Institute (CTSI) to take advantage of specialty clinical research program areas that facilitate the execution of clinical trials. Over the years, the UCLA AC has been known for its innovation and forward development progress within a diverse portfolio of therapeutic areas. Many of our investigators have opened previously unattainable avenues of treatment for rare diseases and those with significant unmet needs. We assist in facilitating the study activation for these trials ensuring a robust review of the science, availability of potential patients, and the clinical research staffing expertise to execute these complex protocols within the highest standards of ethics, safety, and good clinical practice. The AC responds to requests for pre-clinical and clinical trial assistance within UCLA, CIRM and AC network referrals, and those from external sponsors to conduct trials. CIRM-funded research receives priority access to facilitation and navigation through the process. Our focus areas are in rare diseases and sickle cell disease (SCD). This facilitation includes consultations with standing expert teams in FDA and Regulatory Affairs, Community Engagement, Finance and Coverage Analysis and manufacturing. All of our partners and teams work together to ensure that investigators are able to access the resources they need with project management support. We also alert our investigators to opportunities for FDA expedited programs that lead to faster therapeutic product development. From the patient perspective, we review trials in therapeutic areas relevant to the prevalence of disease conditions within our local community. We seek out consultation with our community engagement and trial diversity experts on clinical trial education and participation. We respond to patient inquiries about our clinical trials and refer when applicable to the CIRM website and ct.gov for additional resources. We are leveraging academic-community collaboratives to provide underrepresented communities with access to AC network clinical trials, education and community care. More specifically, we are working with our SCD clinical and research teams and our Community Engagement group to capitalize on opportunities in this specific area. The outreach to advocacy organizations within the Los Angeles area fosters relationships directly with affected communities. Within the network, we have participated in the Steering Committee and Program Manager meetings throughout the first year. We have representation on many of the collaborative working groups within the network that extends the reach of CIRM’s mission and takes advantage of unique clinical trial resources/expertise at each site. These include leading the Coverage Analysis Group, FACT accreditation, and membership in the Social Determinants of Health and Scientific Monitoring Committee groups. We were well-represented at the UCI Director’s meeting this fall and engaged in the dialogue and discussion relating to elevating the presence and capacity of the AC Network. We also were a hear from patient advocates and meet other attendees at the UCI annual symposium. Our first AC Executive Advisory Board meeting was held in December with 13 members present to hear about the background of the AC, strategy, operational milestones, and overall operations. There were a number of action items detailed during the discussion which included ensuring access to resources for cellular therapy laboratory cores, patient recruitment and cohort finding UC-wide, retention of trained research staffing, and future communications about the AC network and its resources and events. We have convened an AC publicity and communications team internally to collaboratively work with CIRM on disseminating information on strategies, opportunities, and clinical trials of importance to the community. Our website is now live with the information required for both study teams and the public to access information on the UCLA AC. Our CTSI newsletter serves as a resource for study teams to become aware of new developments within the UCLA AC and the entire AC Network. From the regulatory standpoint, we are working on evaluating the best platforms for submitting regulatory materials electronically that are efficient, user-friendly, and satisfy both federal regulatory agency and institutional requirements. We are providing network services for data and safety monitoring to ensure that investigator-initiated trials have the resources available to satisfy CIRM-funded trial requirements as well as those of the regulatory agencies for oversight and safety. Professional development continues in CIRM working group meetings, FDA and CTTI training sessions and future plans for the Association of Clinical Research Professionals and ISSCR.

The UCLA Alpha Clinic (AC) continues to strategically plan for meeting the needs of patients through clinical trials, community engagement, workforce development training and healthcare partnerships. We maintain an active list of clinical trials assisting at the development phase, start-up, and execution. We are working with the investigators who are positioning themselves for new CIRM grant submissions this year, continuing to target the unmet needs of patients with serious disease in a multitude of therapeutic areas. This year we focused on the clinical trials, coverage analysis network deliverable, community engagement (CE), and workforce training and development programs. We continue to leverage community partnerships that align with our strategy and patient-oriented perspectives. Integrating our capabilities within the infrastructure of the UCLA Clinical and Translational Science Institute (CTSI) in the David Geffen School of Medicine and the UCLA Health System has led to exponential advances in our reach for community engagement and diversity programs. We have several community partners and are actively working on the CIRM Community Care Centers of Excellence (CCCE) developments in their foundational phase. As part of creating a systematic approach to program development, AC leaders are members of the Adult Hemoglobinopathy Program Steering Committee and responsible for the Clinical Trials Oversight workgroup. One of the Committee’s initiatives is to expand days/hours for routine and urgent sickle cell disease (SCD) care, as well as clinical trial access. We continue to examine the ethical issues of clinical trials especially with newly approved therapeutics in SCD. Dr. Amie Patel is a SCD consultant and healthcare advocate for patients including clinical trials at UCLA. She created a transition skills lab to help pediatric/young adults advocate for themselves in the adult emergency room working in conjunction with the Sickle Cell Foundation of California. She meets with Cayenne Wellness on a bi-weekly basis and is also working with Pfizer on developing patient education materials. Our AC Directors continue to engage new investigators in the targeted fields of regenerative medicine, gene, and cellular therapy, assisting with the resources they need to move forward with their translational and/or clinical research. Specifically, Dr. Noah Federman is the Medical Director of the CTRC, where many complex clinical trials in diverse therapeutic areas are conducted and overseen by the respective DSMB, chaired by him. He is a key leader in the FACT accreditation process at UCLA and is a member of the AC Network Working Group on this topic. He represented the UCLA AC at the Los Angeles Regenerative Medicine Summit. Dr. John Adams serves as the Chair of the CTSI Scientific Review Committee, moderator for the AC Executive Advisory Board, and as a Program Director for the UCLA Regenerative Musculoskeletal Medicine Training Program. Our Program Manager has been spearheading many of the educational and training opportunities within the AC ensuring that these remain a rewarding experience for those involved. The UCLA pathway training programs continue to excel from high school through clinical research professionals. MATCH (high school), FIAT LUX (college freshmen) CTSI-RAP (UCLA undergraduates), LAUNCH (post-graduate), and Navigating Careers in Clinical Research (job seekers and faculty/staff) remain in high demand. The AC focuses on Regenerative Medicine, Gene, and Cell Therapy curricula, including Clinical Trials Day during Undergraduate Research Week and a Community Engagement and SCD-focused Navigating Careers in Clinical Research seminar (156 registrants). The AC took the initiative to assimilate a UCLA CIRM Trainee Educational Collaborative for co-developing curriculum and events from the BRIDGES, COMPASS, CIRM Scholars, and GMP internship programs. We have presented to our Grants Submission Unit (for future relevant CIRM and NIH grant trainee applications), Teen Cancer of America, and at senior community housing. Our Program Coordinator continues to build out our website and curate the content along with the CIRM Communications team and their resources. We have partnered with our CTSI Clinical Trials Navigator team to ensure that our clinical trials are highlighted on our UCLA Clinical Trials and Medical Study Opportunities page and are pushed electronically to referring physicians for their qualifying patients and to patients directly. We look forward to partnering with the CIRM-selected clinical trial portal for ongoing collaborative recruitment efforts. Professional development of the AC team includes participation in an internal retreat with leadership to discuss and strategize for further refinements of AC services and targeted priority areas, attendance at the Association of Clinical Research Professionals Meeting, and recently accepted educational program abstracts at Translational Science 2025.