Wouter Van’t Hof, PhD
Contributing Editor
Cleveland Cord Blood Center, USA
The first letter in the ISCT acronym stands for International. (If you didn’t know this, you are hereby admonished and instructed to read this editorial in full). At ISCT, five Regional Executive Committees represent the global membership in North America, Europe and the Middle East, Asia, South and Central America and Australia and New Zealand regions. For each region, the Vice-President serves as the regional envoy on the Board of Directors. In pre-and post-pandemic times, regional in-person meetings are organized periodically, and the annual ISCT meeting alternates between the regions.
The Telegraft Hub is another platform for expressing and sharing your experiences in cell and gene therapy development across the globe and for highlighting unique benefits or challenges in your area for bringing new well-controlled, safe, and efficacious cell-based medicines to patients. The pursuit of new cell therapies is rapidly expanding in China and Japan. In March 2023, the Telegraft Hub posted a Committee Spotlight for the ISCT Asia Regional Early-Stage Professionals (ESP) Subcommittee, which aims to grow its collaboration network to promote clinical translation and successful services of cell and gene therapies.
In February 2023, the South & Central America Legal and Regulatory Affairs Watchdog provided a Latin American perspective on the Challenge of Unregulated Regenerative Medicine. This interesting piece highlights the challenges in Central and South America in developing guidelines and regulations by which cell therapies may move from research to clinical application under high-quality standards and ways to prevent uncontrolled stem cell tourism. In contrast to well-established agencies such as the FDA and the EMA in the northern hemisphere, the pursuit of regulated CGT efforts remains scattered across South and Central America. The bigger countries in the area, Brazil, Colombia, or Argentina, are leading the region in defining CGT development paths and, as such provide a source for standardization in other countries that may have patchwork or even missing legislation for CGTs.
The main objective of ISCT is to ensure patient access to new therapies in any region of the world. In the US and Europe, CGT approval rates have been increasing in recent years, but downstream hurdles to patient access, such as sky-high pricing of approved products, remain. Other complications in transferring novel therapies across regions are based on limitations in the procurement of specimens (e.g., adult allografts or cord blood) used as starting material for the manufacture of specialty products, such as NK, regulatory T cells and others. FDA requires that CGT products tested in clinical trials in the US are manufactured from starting materials sourced in the US. This is largely based on the agency’s experience and comfort with its own donor screening, testing and eligibility assessment criteria for the prevention of the transfer of communicable diseases. Likewise, the EMA has its own specific stipulations for infectious marker disease testing panels that are not in place in other areas. Due to globalization and migration, every region in the world is becoming more diverse and forced to be less reliant on its own internal gene pool, especially for CGTs requiring HLA matching. Thus, a need for “universal” guidance on sharing of starting materials is becoming more and more relevant.
Telegraft Hub provides a prime opportunity for all CGT researchers and developers in all regions of the world to share their local, regional experiences and offer examples of successful and unsuccessful approaches and regulations. It is also a place to offer recommendations on optimization and broadening access to CGTs to patients of different backgrounds across the global regions.
We invite you to consider your contributions and follow our updates on new international advances in cell and gene therapy. Check us out at https://www.isctglobal.org/telegrafthub, or follow us on Twitter (@telegraft) or on LinkedIn https://www.linkedin.com/company/isctglobal/.
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