Whose life is it anyway? Building patient needs and goals into stem cell clinical trials

by Lisa Willemse

In a traditional view of medical research, advances tend to be measured against the overarching goal of cure. Noble as this might be, research is rarely such a black and white affair -- if we have learned anything, it’s that there are innumerable shades of grey.

Even the goal itself can be questioned, especially when achieving it could be 50 years into the future.

Back in 2004, Dr. Kim Anderson published a rather shocking study that revealed a discrepancy between the goals of the spinal cord injury (SCI) population and those of medical researchers. While research had been largely aimed at finding an all-encompassing “cure” for SCI, the patients themselves were more pragmatic, identifying incremental goals focused on partial functional recovery and improved quality of life. 

According to the study, recovering the ability to walk is not the first priority of the majority of the SCI population, which lists the top three priorities as hand/arm functionality (for people with tetraplegia), regaining sexual function and recovery of bladder/bowel function.

While many branches of research have been working towards quality of life and functional recovery improvements for people with more chronic SCI, the stem cell field has been largely focused on recent spinal cord injuries with hopes of achieving that overall goal of spinal cord repair or regeneration. Media coverage of the recently-halted Geron trial illustrates just how intense the focus is on stem cells as a potential cure for SCI.

Now there is more to consider. Just last month, another study led by Dr. Brian Kwon was published, which questions whether the SCI population is being left behind in the rush to initiate stem cell clinical trials on patients with recent spinal cord injuries.  In the study, the team found that the chronic SCI population (more than 18 months post-injury) demonstrated a much greater awareness of the risks and was averse to undertaking any experimental therapy that might reduce their quality of life. In contrast, the sub-acute (between 1-7 months post-injury) had a greater willingness to consider a clinical trial, despite being less knowledgeable about their condition or about stem cells in general. 

As noted by both the researchers and the patients themselves:

“...this is the time when a person with SCI is most vulnerable, poorly informed about the evolution of the condition and options for intervention and is unlikely, therefore, to be able to make an informed decision about participation.”

This is a critical disconnect in research process to the detriment not just of the patient but also of the research community:   

“While researchers produce results, and physicians and health care professionals create the bridges between the science and practice, people with SCI must not only seek the fruits of those gains, they must have the opportunity to contribute their experiential, contextualized knowledge to the unfolding story.”

As one participant noted: “Put my voice in there, so it’s actually not just the doctors and physiotherapists doing all the research stuff without having input from the people with the actual spinal cord injuries.”

While the research community takes a collective pause in the aftermath of the Geron trial closure, perhaps this is a suitable time to review shared goals so that, even if we know it’s not all black and white, at least we’re looking at the same shade of grey.