Judy Illes, CM, PhD, FRSC, FCAHS, University of British Columbia

Creating a space for informed hope

Like drugs and other biologics, the development of stem cell therapies typically follows a standard path through clinical trials before they are approved for medical use. But this is not always the case. There are occasions when an already approved therapy could be used for “off-label” purposes – for a condition other than what Health Canada approved it for. One example of this is the use of hematopoietic (blood) stem cell transplantation, normally used for patients with severe blood disorders, being offered by hospitals as an off-label treatment for patients with aggressive multiple sclerosis (MS). These treatments are associated with higher risk and are not part of a clinical trial.

Within this context, Dr. Judy Illes at the University of British Columbia wanted to know if and how the decision-making processes of the MS patients differed from those of the clinicians responsible for their care. With support from a SCN Impact award, Illes and her team interviewed 11 patients and 15 specialists.

What they found is that patients who had not responded to standard MS therapies had an urgent desire to access a hematopoietic stem cell transplant. By contrast, clinicians emphasized the risks of the transplantation procedure and were reluctant to choose it as a first line of therapy. These risks were more acceptable to patients as an alternative to rapid disease degeneration and they also cited a desire to help advance knowledge by participating in an investigational stem cell procedure. Such an altruistic motive was not echoed by clinicians, who were ambivalent about the societal benefits and cited concerns that choosing an experimental therapy outside a clinical trial might result in reduced knowledge generation and/or delays getting clinical approval.

The team’s findings point to a need for greater dialogue and shared decision making between the two groups. Providing evidence-based information that supports informed choices and informed hopes will benefit research and patient care for those choosing a hematopoietic stem cell transplant for MS as well as other stem cell therapies currently in the translational space.

Project Outcomes

• New knowledge about patient decision-making process, priorities and concerns regarding off-label stem cell therapies
• Development of evidence-based resources and recommendations for patients and providers