Meeting of Interest

Toxicity and Efficacy of Stem Cells as Therapeutic Products for Humans and Animals

Tuesday, April 24, 2018, 11:00 AM–12:30 PM ET USA

Stem-cell based therapies are becoming very popular in humans and animals for many conditions. However, much still needs to be understood in terms of their toxicity and efficacy. We will have two speakers from the US Food and Drug Administration (US FDA). The first is from the Center for Biologics Evaluation and Research (CBER) discussing their use in humans and the second speaker is from the Center for Veterinary Medicine (CVM) who will discuss the challenges in their veterinary use.


Stem Cell-based Therapies: FDA/CBER Preclinical Regulatory Considerations presented by Wei Liang, PhD, Pharm/Tox Branch 1, Division of Clinical Evaluation and Pharmacology/Toxicology (DCEPT), Office of Tissues and Advanced Therapies (OTAT), CBER, US FDA

The conduct of a clinical trial for an investigational stem cell-based product is governed by the Code of Federal Regulations (CFR) Title 21, Part 312 to ensure the safety and rights of subjects in all phases of a clinical investigation. According to 21 CFR 312.23(a)(8), the sponsor needs to provide adequate information derived from pharmacology and toxicology studies to support a conclusion that the proposed clinical trial is reasonably safe and scientifically feasible to conduct. Preclinical evaluation of stem cell-based products in vitro and in animals is a key element in the overall benefit:risk assessment. The primary goals of preclinical studies for stem cell-based therapies are to establish the scientific rationale to support the planned clinical investigation and to assess the potential toxicities associated with administration of the stem cell-based product. Considerations for preclinical assessment of stem cell-based products are discussed in the "Guidance for Industry: Preclinical Assessment oof Investigational Cellular and Gene Therapy Products," which was released by CBER in November 2013.

This presentation will discuss (1) potential safety concerns associated with stem cell therapies, (2) preclinical testing programs for stem cell therapies to support an investigational new drug (IND) submission for a first-in-human clinical trial, and (3) early communications with FDA/CBER/OTAT via pre-pre-IND interactions and pre-IND meetings.

Safety of Cell-Based Products for Animal Use: presented by Lynne Boxer, DVM, CVM, Office of Foods and Veterinary Medicine, US FDA

Cell-based products for animal use, including stem cell products, that meet the definition of a new animal drug require US FDA approval to be legally marketed. Thus, approval of these products requires a demonstration of safety, effectiveness, and manufacturing quality. While there is increasing interest in the potential therapeutic applications for cell-based products, there are also challenges associated with assessment of the safety and quality of these products. This talk will discuss the specific safety considerations for cell-based products, as well as an overview of what is known about adverse events in veterinary species.

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