The Committee for Advanced Therapies (CAT) of the European Medicines Agency (EMA) advises patients and citizens not to use unregulated cell therapies, which may be unsafe or ineffective.
The CAT opinion was formulated in response to individuals, companies and hospitals promoting cell therapies that are not scientifically validated as remedies for a wide range of diseases, including cancer, cardiovascular disease, autism, cerebral palsy, muscular dystrophy and loss of vision.
Risks of unproven or unregulated treatments
These treatments can expose patients to serious risks, with little or no benefit. Patients who have used cell therapy products that are not scientifically based or are unregulated have reported serious, sometimes fatal, side effects including infections, adverse immune reactions, tumour formation, loss of vision and cerebral bleeding.
What are cell therapies?
Cell therapies are treatments that use the patient's own cells or those from a donor. The use of blood and cells for transplantation is an established medical practice. However, if the cells are not used for the same essential function in the recipient patient as in the donor or if they are manipulated in a substantial way, this does not constitute transplantation. Therefore, their safety and benefits cannot be guaranteed. For this reason, these therapies are regulated in the European Union (EU) as medicines.
The rapid development of technology in the field of cell therapies offers new opportunities for the treatment of various diseases, many of which are currently considered incurable.
Clinical trials ensure safety and protect patients
The CAT stresses that well-designed clinical trials are essential for the safety and effectiveness of these therapies so that patients can benefit from the potential benefits of these cell therapies. These trials are necessary not only to understand the safety and efficacy of innovative therapies, but also to protect the safety, dignity and rights of patients. Well-designed clinical trials also educate patients about the potential benefits and risks of treatments and can be used to support their authorisation in the EU, thereby generating benefit for more patients.
By evaluating data from clinical trials conducted with cell therapies, the CAT also verifies that the quality of these products is properly controlled. Once these products are authorised in the EU, the EMA and national medicines agencies, such as the FAMHP, constantly monitor their safety and share information to allow rapid decisions to be made at EU level to protect the health of patients.
Avoiding marketing authorisation procedures and clinical trials makes it difficult to understand and document the effects of these cell therapies, thereby denying future patients access to potentially curative treatments.
The CAT will continue to work to contribute to the development of new and other advanced cell therapies, with the goal of increasing timely access to life-altering treatments.
Patients or their families who plan to use cell therapies should contact their healthcare professionals to obtain information on the benefits and risks of treatment and to find out which authority has approved them. They can also contact the FAMHP or the EMA directly.