ISSCR Voices Concern as Italian Government Authorizes Unproven Stem Cell Therapy

  • 9 April, 2013

Stem cells hold great promise for the treatment of a wide range of diseases and injuries; however, years of laboratory research followed by rigorous clinical trials are required to deliver safe and effective therapies to patients. Currently, the range of diseases for which stem cell treatments have been demonstrated to be safe and beneficial remains limited. ISSCR believes that ongoing research will eventually lead to new therapies for additional diseases but until the research has been completed we cannot be sure which diseases can be treated effectively with stem cells, or how the stem cells should be used to ensure safety and effectiveness.

The excitement about the promise of stem cells has resulted in confusion for patients struggling to cope with incurable diseases. The ISSCR is concerned that inadequately tested stem cell treatments are being marketed around the world to patients and their families without the necessary safeguards in place to ensure safety and efficacy.

The recent decision announced by Italy’s health minister, authorizing the administration of cells that have been described as mesenchymal stem cells to patients with neurological disorders, has raised concern in the international research community. It is not clear based on the scientific literature that mesenchymal stem cells have any ability to ameliorate neurological conditions nor is there compelling evidence from clinical trials that such cells provide benefit to patients with neurological conditions. The Italian Medicines Agency (AIFA) had previously denied this treatment.

In 2008, the ISSCR published “Guidelines for the Clinical Translation of Stem Cells,”that call for rigorous standards in the development and use of stem cell therapies. The ISSCR Guidelines recommend that stem cell therapies should be tested in structured clinical trials, which under internationally accepted standards are subject to independent and ongoing assessment of safety, efficacy, and ethical soundness.

Shinya Yamanaka, MD, PhD, ISSCR President said “We sympathize with patients with incurable diseases. However, there is little objective reason to believe that these patients have the possibility of benefitting from a mesenchymal stem cell therapy and treatment decisions should not be made outside of a controlled clinical trial without data on safety and efficacy.”

The ISSCR believes that innovative and compassionate care is important, but untested therapies should only be offered outside of clinical trials in limited circumstances where there is sound theoretical reason to believe the patient could benefit. This exception does not justify commercializing unproven therapies. The principles of medical ethics review and regulatory oversight were developed over several decades, largely in response to instances where people were harmed during human experimentation.

The ISSCR emphasizes the critical role that expert independent review and oversight plays in the development and practice of stem cell treatments, in particular that provided by local bodies and national regulatory authorities who aim to protect human health and to assure the quality, effectiveness and safety of medical treatments.

Click here to learn more about how science becomes medicine.

See also:

# # #

The International Society for Stem Cell Research is an independent, nonprofit membership organization established to promote and foster the exchange and dissemination of information and ideas relating to stem cells, to encourage the general field of research involving stem cells and to promote professional and public education in all areas of stem cell research and application.