International Society Cell and Gene Therapy, the global professional society of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell and gene therapy sector, announces its reaction to the US Food and Drug Administration (FDA) seeking court orders preventing two US stem cell clinics administering unproven cellular therapy treatments to patients.

The FDA action takes place one week after the ISCT Presidential Task Force Session on the Use of Unproven Cellular Therapies on May 4, 2018 a headline session at the ISCT Annual Meeting, Montreal, Canada. The session involved regulators including Peter Marks, US FDA and Nadine Kolas, Health Canada, as well as representatives from academia, industry, bio-ethics and patients.

This session enabled these representatives to discuss in person strategies to combat unproven cellular therapy from different perspectives. The session provided the opportunity to discuss the effectiveness of existing responses, and coordinate and agree joint international strategies amongst regulators, health providers, physicians and patients.

The panel discussions at the meeting involved:initial results of a global collaborative survey on how unproven cell therapies are impacting biomedical research and clinical practice the size of the unproven cell therapy market - currently worth up to USD 2.4 billion and involving approximately 60,000 patients annually, paying up to USD 40,000 per treatment the need to distinguish between medical intervention with appropriate informed consent, including clinical studies and compassionate use therapies, versus therapies marketed to patients without the regulatory framework, with misleading or fraudulent information the rights of patients to be the final decision makers of their own care, and greater sensitivity from the medical community for experimental procedures, especially for high unmet medical needs the need for international regulatory-harmonisation the extensive approval processes that regulators, including the.FDA, use for cell therapies, and how these processes are changing and evolving, including the introduction of new programs, such as the

FDA’s Regenerative Medicine Advanced Therapy Designation (RMAT) program to expedite the development and review of regenerative medicine advanced therapies the challenges of communicating with multiple stakeholders about cell therapies the need for patients and families to distinguish between realism and hope, and the devastating effects that false hope can have the sharing of information for physicians and patients, and the precise definition of products and procedures.

“The most recent FDA actions highlight to the entire cell therapy community that the UCT business stretches across the globe, not just in specific regions where regulations are less tight,” said Dr. Massimo Dominici, chair of the ISCT Presidential Task Force (PTF) on the Use of Unproven Cellular Therapies (UCT), and professor of oncology at the University of Modena and Reggio Emilia, Italy. “It is vital that regulators, as recently demonstrated by the FDA, continue to identify and shut down clinics conducting unproven and potentially harmful cellular interventions. However, all representatives of the ISCT Presidential Task Force agree that the job cannot be left to regulators only. There is no magic bullet for UCT with complex challenges. Patients affected by diseases with a high-unmet medical need have few options. It is up to the cell therapy community to collectively drive more therapies through approval, reduce costs through innovative development and manufacturing strategies and educate the public and patients about the dangers of unproven cellular therapy. As a result, ISCT will continue to coordinate global interdisciplinary efforts to implement these longer-term solutions.”

ISCT has for a number of years been bringing groups together spanning the international cell and gene therapy community, including collaborations with more than 30 scientific societies and institutions, to lead and coordinate direct action to combat and raise awareness about unethical and unproven cellular therapies. It first discussed unproven cell therapies with the FDA in 2012. Direct action has ranged from establishing a multilateral task force, implementing a long term program of regulatory harmonisation, to publishing a reference guide, translated in six languages, authored by 23 leading figures in the field, for the patient community.  

“Cell and gene therapies are addressing a variety of currently unmet medical needs. Several products have been approved after demonstration of safety and clinical efficacy. Many more are currently progressing through clinical development. However, clinics offering potentially dangerous unproven cell therapies present a significant risk of eroding public confidence in our new, emerging field,” said John Rasko, President, ISCT and Head, Cell & Molecular Therapies, RPA Hospital, Sydney, Australia. “The FDA action, and wider collaborations at the ISCT Annual Meeting this month, have reinforced the need for ISCT and its members numbering over 1,500 across 50 countries to continue on its mission of promoting the development of, and access to, properly manufactured and documented cell and gene therapies to patients. As these approved therapies are delivered, this will ultimately reduce the demand for unproven treatments.”