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.