Stanford’s Laboratory for Cell and Gene Medicine (LCGM), the university’s first manufacturing facility specifically designed to adhere to the Food and Drug Administration’s (FDA’s) current good manufacturing practices (CGMPs), opened this month in Palo Alto.
This recent partnership between the Stanford School of Medicine, Stanford Health Care and Stanford Children’s Health is dedicated towards developing biological products for phase-1 and phase-2 clinical trials.
LCGM’s main focus is “to develop cell and gene therapy products that exist as research projects within Stanford but are intended to use clinically to provide a curative therapy for a wide variety of diseases,” said laboratory director David DiGiusto, Ph.D.
These biological products are currently manufactured for a wide range of projects at LCGM. Judith Shizuro, Ph.D. M.D., an expert in blood and marrow transplantation, is collaborating with LCGM on stem cell transplants. She has a trial open to treat patients with severe combined-immunodeficiency. Other biological products worked on in the lab include corrective skin cells to develop skin grafts for children with epidermolysis bullosa and developing chimeric antigen receptors (CAR) T-cells for cancer immunotherapy.
There are also ongoing projects to explore possible treatments for graft versus host disease, sickle cell anemia and autoimmune diseases.
“There are about 35 projects the lab is currently working on,” DiGiusto said. All of LCGM’s customers are Stanford affiliated. While there room in the future for services to others outside of Stanford, “the priority would be given to those who have collaborative efforts with Stanford.”
According to DiGiusto, the hope is that projects will flourish under the stringent procedures needed to comply with the FDA’s CGMPs. In its official website, the FDA describes how CGMPs assure safe design for pharmaceutical systems that help prevent occurrences with contamination and other mishaps. These regulations, dealing with both facility and operation, were designed to be flexible to fit the needs of each individual manufacturer.
“The flexibility in these regulations allows companies to use modern technologies and innovative approaches to achieve higher quality through continual improvement.” (FDA)
To achieve this high quality, the lab uses cleanrooms, controlled environments designated for conducting research and/or manufacturing products. Cleanrooms employ technology that are used to keep rooms sanitized, to reduce particulate contamination and to control other factors such as temperature, humidity and pressure.
There is an emphasis and a requirement for highly-trained professionals to work at LCGM. Many assume that LCGM is fit for researchers, but the lab needs those who are knowledgeable in both scientific acumen and the regulatory affairs of drug development.
“Because we work with biological drugs (i.e. cells), we need those who understand how to develop cells and viruses as drugs,” DiGiusto explained.
Other challenges stem from the range of the 35 projects the lab is currently working on.
“We can’t be subject experts in every field, so it’s challenging to have stem cells on one day and T cells on another day,” DiGiusto said.“The challenge is accommodating the diversity of the pipeline that we have at Stanford.”
With this in mind, LCGM is intended as part of the Stanford Medicine initiative to make “precision health” a reality — preventing diseases rather than trying to cure them after-the-fact. With precision medicine, the goal is to identify effective treatments for different patients based on genetics, environments and lifestyles.