Scholar Fights for the Most Vulnerable Patients

People who are struggling with incurable diseases or end-of-life decisions are often targeted by those pushing miracle cures or other forms of false hope. A Robert Wood Johnson Foundation Clinical Scholar seeks ways to protect their interests.

    • March 15, 2011

Stem cell research holds the promise of a cure—or at the very least, dramatic improvements in prognosis—for many people with life-threatening or chronic illnesses. The problem, explains Robert Wood Johnson Foundation (RWJF) Clinical Scholar (2009-2011) Amy Tsou, M.D., M.A.R., is that “people are making unproven claims about stem cells and other treatments. Even the language describing many early phase clinical trials is misleading and it often encourages patients to pursue expensive, but unproven courses of treatment.”

Tsou’s interest in patients struggling with incurable diseases is the outgrowth of her work as a neurologist at the University of Pennsylvania, where she primarily treats people with Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig's disease). “Our patients are particularly vulnerable to scams offering false hope, so some of my research focuses on how to protect them,” Tsou said. “Stem cells have been very helpful in some diseases—such as leukemia. But there are also researchers who present early [phase one or two] stem cell clinical trials for other diseases as being further along than they really are and suggesting benefits that are not yet proven.”

In the case of ALS patients, Tsou explains that the disease progresses because motor neuron cells die. Some proponents of stem cell therapy claim that injecting stem cells into the spinal cord will re-grow the cell and its neuron cable. “But at this point, we have no proof,” Tsou said. “Yet there are United States researchers recruiting patients for stem cell trials with misleading language, and physicians in other countries are promising results and charging large fees.”

To more fully explore this issue, Tsou and bioethicist Arthur Caplan, Ph.D., wrote, “Touting Stem Cells—We Have Seen the Enemy and He Is Us,” which was published in the May 2010 issue of the American Journal of Bioethics: Primary Research.

While Tsou has no objection to the thousands of stem cell trials now being conducted around the world and is hoping for research outcomes that will someday benefit her patients, she said, “There’s still very complex science and a learning curve where stem cells are involved. We are a long way from using them as a cure for ALS, for example, but in America we have a penchant to hope for miracle cures—like gene therapy, antioxidants or even margarine for heart disease. The truth is, much of the research looks very promising, but it’s not there yet. It’s very complicated.”

A Broader Focus: End-of-Life Care

Tsou’s work on stem cells is just part of her wider investigation into ways to help patients and physicians manage the tough issues that come with incurable diseases. Her latest research looks closely at helping medical professionals, patients and families talk about difficult care decisions.

“Overall, I’m committed to improving communication between doctors and patients in end-of-life care and one of the subjects I focus on is breathing tube placement for ALS patients,” Tsou said. ALS patients grow weak and eventually have difficulty swallowing and breathing, so facing the decision of whether or not to place a breathing tube in the throat is inevitable.

“This disease progresses so quickly there’s no reason for us not to have this conversation with patients, yet it can be awkward and many physicians avoid it. The study—which I hope to publish this spring or summer—shows that the number of patients getting the procedure done under emergency circumstances is very high, possibly because it has not been discussed in advance. Performing the procedure in the emergency room is much more expensive and we are investigating whether clinical outcomes are worse under these circumstances as well,” Tsou said.

In this case and in others involving needed end-of-life care, Tsou thinks “physicians need to be willing to have straight talk with patients and their families. Patients appreciate it when we’re honest with them.”

Yet Tsou acknowledges that physicians are often less than prepared to have such challenging conversations. “Physicians need more training in this area—it really is an art—something we need more practice at. Most medical schools offer some training, but it’s not enough. It also needs to take place during residency, when we’re facing real patients and families, rather than in the classroom. You just don’t realize how complicated it really is until it’s a face-to-face conversation with a dying patient or their family,” Tsou adds.

“I came into the Clinical Scholar program frustrated about how doctors often communicate so poorly with patients on these issues. My mentors—Jason Karlawish, M.D.[ a 2008 Investigator in Health Policy Research awardee] and Judith Long, M.D.[associate director of the Penn Clinical Scholars program]— helped me to focus these questions in the context of ALS care and consider different ways to study communication with patients. Physicians need to realize that talking with the public is just as important as the biomedical work. It’s underappreciated in the academy,” Tsou said.

Whether the issue is unproven, supposedly miraculous cures or helping people end life with dignity, Tsou sees a need for medical professionals to do a better job. “The bottom line for me is that dying patients are a very, very vulnerable group and we need to not only provide the best medical care that we can; we need to protect them from false hope and disingenuous people.”

The Robert Wood Johnson Foundation Clinical Scholars program advances the development of physicians who are leaders in transforming health care through positions in academic medicine, public health and other roles.