The UCLA Human Gene and Cell Therapy Program (HGCTP) is responsible for:
- Clinical cell and gene processing laboratories infra-structure
- Clinical/regulatory monitoring:
Support documentation and monitoring oversight of gene and cell product manufacture
Review and verify eligibility of enrolling gene therapy subjects
- Training and certification of GMP lab personnel and users
- Academic program in gene medicine. Annual symposium & Gene Therapy Interest Group
Regulatory Approvals before Implementation for HGCTP protocols at UCLA
- UCLA Institutional Review Board (IRB)
- Jonsson Comprehensive Cancer Center Internal Scientific Peer Review Committee (ISPRC)
- NIH/RAC (if a gene therapy trial)
- Medical Radiation Safety Committee (MRSC)
Good Manufacturing Practices Facility
The Good Manufacturing Practices (GMP) facility at UCLA was specifically developed to support investigator-initiated clinical trials in which a cell or gene therapy product is manufactured by the investigative team under an FDA IND. This facility is a joint effort by the David Geffen School of Medicine, Human Gene and Cell Therapy Program (HGCTP), the Jonsson Comprehensive Cancer Center, and the Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Research.
The GMP facility is a 2,395 square foot BioSafety level 2 contained area (class 10,000) with 7 HEPA-filtered rooms. The 4 positive pressure laboratories are designed for ex vivo cell manipulation of cells, since it minimizes the possibility of outside pathogen contamination. The 3 negative pressure laboratories are designed for gene transfer procedures, and ensure that the recombinant vectors do not contaminate the surrounding environment.
Current Clinical Trials Utilizing the GMP
- Immunotherapy of melanoma, sarcoma and myeloma with TCR-modified T cells (Ribas)
- Immunotherapy of advanced malignancies with TCR Engineered PBMC (Singh)
- Immunotherapy of glioblastoma with tumor antigen-pulsed Dendritic Cells (DC) (Liau/Prins)
- Immunotherapy of glioblastoma with allogeneic Cytotoxic T Lymphocytes (CTL) (Kruse)
- Immunotherapy of renal cell carcinoma with Ad-CA6/DC (Kabbinavar)
- Gene therapy for ADA-deficient SCID, XSCID, XALD (Kohn)
- Retinal pigment epithelial cells for Stargardt and Age Macular Degeneration (Schwartz)
Operations and Staffing GMP Facility
The HGCTP provides a dedicated Facility Manager and Quality Assurance Officer to maintain the GMP facility and equipment, and provide training, oversight and support of GMP manufacturing and cGMP compliance. Individual investigative teams provide the personnel for product manufacturing and characterization and are responsible for documentation and regulatory reporting, generally under investigator-initiated IND. The Jonsson Cancer Center Office of Regulatory Compliance provides clinical monitoring and auditing oversight for institutional trials using the GMP facility, if this is not provided by a different entity.
Access and Equipment for GMP Facility
Access to this facility is restricted to investigators who have had specific training for entering and working in this environment. Access is controlled by the UCLA Human Gene and Cell Therapy Program. Please contact the Facility Manager for more information. Priority for room use is given to NIH/CIRM-funded investigator-initiated clinical trials. The overall operational plan for the facility for compliance with cGMP standards incudes: controlled access, facility cleaning, continuous monitoring of environment, air and equipment; scheduled routine preventive maintenance and calibration of equipment; document control, etc.
Costs Associated with Using the GMP Facility
To ensure the successful operation of the GMP facility, the facility will be transitioning to a sales and services model. This model will result in a direct fee-for-service facility. Investigators planning to utilize the GMP facility for future research studies should include anticipated GMP charges in new grant application budgets. For further information, including costs for inclusion in grant budgets, please email the HGCTP team at firstname.lastname@example.org.
The Human Gene and Cell Therapy Program Investigator’s Course is mandatory for all investigators, lab personnel, data managers and clinical personnel involved with an active (or soon to be active) clinical trial that involves gene or cell therapy. If the attendee is not involved with making the test article or manipulating stem cells in the JCCC GMP or BSCRC GTP facility, then he/she does not need to take the cGMP class listed below.
The current Good Manufacturing Practices [FDA cGMP] class is required for principal investigators and lab personnel who have responsibility and oversight and/or perform manufacturing of test article(s) or manipulate biological entities, such as stem cells or viral vectors, in the JCCC GMP or BSCRC GTP suites. Personnel wanting to perform work in the GMP/GTP suites must take the cGMP class in order to meet the requirements for GMP/GTP suite access established by the JCCC and BSCRC leadership.
Both courses are given by the GMP Quality Assurance Manager/ HGCTP.
These classes will provide the attendee with a better understanding of regulatory requirements for clinical research involving gene transfer and good manufacturing practices. There is a written take home exam for each class that must be satisfactorily completed by the participant with a passing grade of >70%. Passing the written test will result in the issuance of a certificate of completion valid for 3 years.