Cell therapy breakthroughs, such as chimeric antigen receptor T-cell (CAR-T) therapy, have made a significant impact on the oncology landscape, but many potential breakthrough cell-based therapies are emerging in other therapeutic areas, such as neurodegenerative diseases and macular degeneration.
“What we see in the cell-based therapeutic research area today represents just the tip of the ‘iceberg’ – and underneath it is a reservoir of innovative science that has the potential to bring new therapies to the market and impact patients’ lives,” said Brian McIntosh, PhD, Study Director and Scientific Lead of Cell and Gene Therapy.
“Many biotech’s and academic labs are exploring pluripotent and multipotent stem cells to address unmet needs for patient populations,” he added. “They need to understand the challenges that they’ll face in developing their investigational products, from the preclinical phase to testing in humans in clinical trials.”
Starting a dialogue around stem cell products
To provide a more in-depth conversation on this topic, we hosted “Practical Considerations and Solutions in the Preclinical Development of Investigational Stem Cell Products,” a live focus session on June 24th at the 2020 virtual meeting for the International Society for Stem Cell Research (ISSCR). If you were a registered attendee of the meeting at ISSCR.org, you can listen on-demand to insights from leading pharma and biotech subject matter experts on this topic and others from the virtual meeting.
“This educational session was designed to inform and create a dialogue around scientific and regulatory considerations for developing pluripotent and multipotent stem cell products,” explained Dr. McIntosh, who served as the session chair.
“ISSCR attendees can listen to the five speakers who discussed their experiences and lessons learned with developing cellular therapeutics,” he said. “We designed this session to provide practical advice surrounding key scientific and regulatory aspects of preclinical development. We know that investigational stem-cell products are much different from traditional therapeutics with respect to justifying safety and efficacy. Sponsors today are required to understand more about the critical elements of a regulatory submission to initiate human clinical studies.”
Uniting a wide range of expertise
Kicking off the virtual on-demand focus session, attendees heard about preclinical pharmacology considerations of a cell therapy product, presented by Kapil Bharti, PhD, Senior Investigator, National Eye Institute, Ocular and Stem Cell Translational Research Section at the National Institutes of Health. Next, preclinical safety assessment of induced pluripotent stem cells (iPSCs) and embryonic stem cells (ESCs) were discussed by Jeanne Loring, PhD, Founder and Chief Scientific Officer of Aspen Neuroscience, and Professor and Founding Director of the Center for Regenerative Medicine in the Department of Molecular Medicine at Scripps Research Institute.
Drawing on professional experience, Paul Baldrick, PhD, Executive Director, Nonclinical Regulatory Strategy within the Global Regulatory Affairs Department spoke about nonclinical regulatory guidance in the development of cell therapies. His presentation was followed by Jane Lebkowski, PhD, President of Regenerative Patch Technologies, a biotechnology firm developing composite stem cell-based implants targeting restoration of retinal architecture and function in patients with macular degeneration. She shared critical quality attributes of cell therapy products and best practices for establishing and tracking manufacturing processes for cell therapy products.
Biomarker selection was the final topic, presented by Akanksha Gupta, PhD, Head of Immunology, Biomarker Solution Center. She discussed her perspectives on developing biomarkers in a preclinical study to monitor pharmacokinetics and how these efforts can help set up sponsors for success when they reach the clinic.
Continuing the stem cell conversation
At the end of each topic, attendees participated in question and answer sessions that occurred during the live virtual format.
“Given the sheer newness of the pluripotent stem cell derived transplantation field, we anticipated many questions,” said Dr. McIntosh. “Attendees should get a lot of value from these presentations, which we hope will help them accelerate the development of their cell therapy candidate and catapult it into the clinic.”
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