Regulators need to protect stem cell promise

The report in this week’s issue of the New England Journal of Medicine (NEJM) of three people in South Florida who lost their sight after participating in what they thought was a stem cell clinical trial highlights the need for greater scrutiny by regulators to protect patients.

This is urgently needed, not just in US, but also here in Australia. While great progress has been made over the past decade, the reality is we are only just beginning to evaluate if laboratory results equate to safe and effective treatments.

Clinical trials are expected to be overseen by research ethics committees and other experts to ensure that the approach is justified. It is expected that the interests and welfare of the participants are protected, and that they are fully informed of all possible risks. It is not usually expected that patients pay to participate.

However, due to regulatory ambiguity a growing number of clinics around the world are marketing so-called stem cell treatments without first showing that they work or are safe. They are effectively bypassing the clinical trials framework or, as in this Florida case, imply that they are performing clinical research while in fact operating a business selling hope.

A quick search online finds dozens of clinics in Australia that, for a hefty fee, will treat patients suffering osteoarthritis, infertility, multiple sclerosis and hair loss with stem cells. All of these treatments are offered outside clinical trials and regulatory oversight. Evidence of benefit is weak. As they use the patient’s own cells, usually obtained from liposuction, the therapies are presented as “safe”.

For many years we and others have been concerned that patients may suffer infections or other complications, and have called on regulators to curb these exploitative practices. Even after the Coroner’s report into the death of an Australian woman who had an unproven stem cell ‘treatment’ for dementia and died following complications associated with the procedure, little has changed.

It remains business as usual for many of these clinics.

In the absence of effective regulation, all that can be done is to continue to encourage anyone exploring stem cell therapies to do their research.

Ask lots of questions and take information back to your treating GP or specialist to discuss the implications for your condition. Raise red flags if you are asked to pay for treatment, if the same treatment is being offered for many different conditions, or where the clinic uses anecdotal stories or patient testimonies as evidence of success.

Only participate in clinical trials that have had ethics committee approval and are listed in a respected repository such as the Australian New Zealand Clinical Trial Registry. Finally, don’t given up hope and maintain an active interest in the progress of stem cell research for your condition.

Despite the tragic story of the three women who are now blind, stem cell treatments to restore vision loss do hold promise. After 20 years of research, Italian scientists recently received European regulatory approval for a stem cell-based treatment for a type of blindness that results from damage to the cornea, the surface of the eye.

Other groups in the US and Japan are advancing clinical trials for macular degeneration. They first cultivate stem cells to form the retinal pigmented epithelial cells that are needed to restore a damaged retina.

Animal studies show that injecting such cells below the surface of the retina can slow the disease. We now need to responsibly assess whether this can be replicated in humans. Doing so is technically challenging, will require expert surgeons, long-term support and follow-up for the patients.

There are no guarantees that such an approach will work, although another NEJM report is promising.

Until then, regulators must step in to safeguard patient welfare and the work of those trying to support legitimate clinical translation.