Aug. 22, 2017: Overcoming Challenges to Move Stem Cell Therapies to the Clinic

The promise and perplexity of stem cells have captivated me for decades. During my medical training, I took care of patients with neurological diseases caused by death of brain cells — such as Alzheimer’s, Parkinson’s and stroke — diseases we imagined could someday be treated by new neurons made from stem cells. During my research training, I sought to understand the basic biology of stem cells in the brain, what signals instruct them to become one type of cell or another during normal development, and what happens when that process goes wrong. The idea that stem cells, which are capable of becoming any cell in the human body, could be produced at will in the lab from adults — including the patients in my clinic — seemed to be a far-fetched pipe dream. Like so many other remarkable advances in science over the last three decades, that pipedream is now a reality. Induced pluripotent stem cells (iPSCs) are now being produced routinely, usually from adult skin cells. Particularly when combined with new gene-editing technologies, the promise has never been greater for stem cell-based therapies to help treat millions of patients with spinal cord injuries, diabetes, Alzheimer’s disease and scores of other disorders.

Despite this amazing progress, we have yet to achieve the hoped-for promise of stem cell therapies in treating disease. The barriers are typical of those encountered in all areas of translational science. We need to understand how to create, characterize and differentiate patient-specific iPSCs in well-defined, reproducible and efficient ways that meet regulatory standards, and systematically understand the beneficial and potential adverse effects of these cells in people.

To address these challenges, the NIH Common Fund Regenerative Medicine Program helped NCATS create the Stem Cell Translation Laboratory (SCTL), which officially opened last month. The planning, design and construction of this purpose-built, state-of-the-art laboratory took more than two years and marks a new and exciting phase for stem cell translational science. NCATS will use the its signature, systems-driven collaborative approach to characterizing stem cells and their differentiated progeny, creating efficient and scalable differentiation protocols, and disseminating all its protocols and data for use by the scientific community. Among other technologies, the SCTL will integrate multiple “‑omics” technologies (e.g., genomics and proteomics) with single-cell analysis and functional cell characterization. The SCTL is now seeking collaborators from academic, biopharmaceutical and nonprofit organizations to partner in technology development, demonstration and dissemination projects.   

SCTL is just one NCATS initiative to advance stem cell translation to the clinic. Investigators at Boston University and three other CTSA hubs funded through NCATS’ Clinical and Translational Science Awards (CTSA) Program established a national network of iPSC repositories and core facilities. The iPSC network was funded through NCATS’ first round of CTSA Program Collaborative Innovation Awards and already includes more than 1,000 patient-derived iPSCs with plans to add thousands more. Leading the effort to openly share advances in the stem cell field, the repository also contains gene-editing tools and protocols for CTSA Program investigators to enhance basic and clinical research, including better disease modeling and development of regenerative medicine therapies.

On Sept. 26, 2017, NCATS will host a workshop on translational challenges related to iPSCs. This meeting will convene diverse scientists on the NIH campus to discuss barriers to progress and work toward concrete solutions. Register now to attend!

Christopher P. Austin, M.D.
National Center for Advancing Translational Sciences