The ISSCR’s Global Policy Work Protects and Advances Stem Cell Science

Defining Embryo Models

Amid an exceptional 12 months of advances for stem cell science and regenerative medicine, the ISSCR remains focused not only on facilitating scientific exchange through our meetings and events, but also on working to ensure pivotal areas of research can continue to move forward. Following the publishing of significant advances in embryo model research last year and the release of the ISSCR’s statement on embryo models, we recognize the need for our Guidelines to keep pace. To address the growing need for clarity, we have formed a working group to summarize the state of science. The international group comprises researchers, embryologists, ethicists, and clinicians who are reviewing the current recommendations for research on embryo models in order to provide funders, governments, scientific journals, and oversight committees guidance on how to proceed.

Advising Regulatory Policy

Over the last few years, ISSCR has met with the U.S. Food and Drug Administration (FDA) in order to raise issues that researchers face when pursuing the commercialization of stem cell-based therapies. The goal of these meetings is for ISSCR members to share the latest scientific developments and to provide recommendations on how the agency can incorporate this information into its regulatory process.

In late 2023, the ISSCR responded to an FDA draft guidance on Manufacturing and Comparability for Human Cellular and Gene Therapy Products. The ISSCR’s comments were aimed towards improving organization of the document, requests for clarification and examples, and recommendations for adding new sections. The society plans to respond to several more guidance documents in the coming months including one issued last week on potency assurance for cell and gene therapies, a topic we previously discussed with the FDA. We plan to meet again with the agency in the first half of this year.

While our work with the FDA is ongoing, the ISSCR is also pursuing meetings with regulatory agencies around the globe. In 2023, the ISSCR held its first regulatory meeting with the U.K.’s Medicines and Healthcare products Regulatory Agency to discuss manufacturing issues and regulatory convergence. And ISSCR plans to host its inaugural meetings with the European Medicines Agency (EMA) and with Japan’s Pharmaceutical and Medical Devices Agency later this year to discuss similar topics. We have engaged our European-based and Asia-based members to help facilitate and plan these meetings.

To learn more about what regulators have to say about manufacturing cell and gene therapies, consider attending the Industry Innovation Showcase at ISSCR 2024 in Hamburg, Germany where members who participated in these meetings will share insights and information learned from our regulatory advocacy.

Informing Legislative Policy

To provide opportunities for our members to have a strong voice with policy makers, the ISSCR is hosting its annual U.S. Congressional Advocacy Day in March 2024. This event brings in members from key states and institutions to advocate for continued federal funding for research with human fetal tissue, to encourage Congress to crack down on the marketing of unproven cellular therapies, and to request robust, sustained, and predictable funding for stem cell and biomedical research. Learn about last year’s advocacy event.

For members interested in public policy, in the coming months we will be opening applications for the Goldstein Fellows program. Goldstein Fellows participate in policy discussions as members of the Public Policy Committee and plan a policy session at the annual meeting.  In 2024, the Goldstein Fellows are developing a session on the importance of considering cell line diversity in order to improve inclusivity in stem cell research. I would also like to highlight their recent authoritative review published in Stem Cell Reports on the merits of fetal tissue research. Stay tuned for a new podcast featuring the fellows discussing this paper.

As envisioned several years ago, the dual prioritization of legislative and regulatory policy initiatives that are integral for stem cell research to thrive, and clarifying the path for the commercialization of stem cell therapies, position ISSCR as the leading global advocate for our field.

We cannot do this work without you. Your commitment to membership is essential to ISSCR’s advocacy work and contributes to our ongoing success on behalf of the entire field.

 —Amander