A Matter of Health

Remembering the promise; honoring today

By Ruth SoRelle, M.P.H.

New Year's Day represents beginnings and even renewed beginnings. Yet, at a recent party, I was confronted with old realities.

A gentleman who had just been diagnosed with a serious neuromuscular disorder asked me what Baylor College of Medicine was doing with stem cell research as it related to his disorder.

"I do not want to take pills for the rest of my life," he said.

Vigorous stem cell program

BCM has a strong stem cell program. The Stem Cell and Regenerative Medicine Center specializes in stem cell work – both so-called adult and embryonic stem cells. The work runs the gamut – from stem cells of the blood-forming system to tumor stem cells to those in the nervous system. Researchers in the Center for Cell and Gene Therapy at BCM use stem cells to treat cancers and related problems.

Unfortunately, that work at BCM does not extend to his disease, although there are researchers pursuing such work in other parts of the country and world. All are still in the experimental stages. The most recently published study involved a single teenager who received blood stem cells from a sibling whose tests showed that the two were a good match. The young patient had received all possible treatments before undergoing the blood stem cell treatment and none of them worked to halt its devastating progress. Forty months after the transplant, his most serious symptoms had gone away. A few remained. The treatment – a procedure that has its own high risk of injury and death – appeared to provide an option for people who have his disease, who are seriously debilitated by it and for whom accepted treatment is not working. They must have a matched donor as well. However, the scientists warn that long-term follow-up will determine how effective the treatment is and how long it lasts.

A new field and an old one

The use of bone marrow and/or peripheral blood stem cell treatments from a donor is decades old. Its success depends on how close a match a donor is to the person receiving the treatment as well as a host of other factors. It represents wiping out a person's immune system – often with toxic drugs – and then restoring it with stem cells. When it works, it is great.

Embryonic stem cells are a new phenomenon. Because they can become so many tissues, they have much promise that is yet unrealized. We have studied them in detail for no longer than a decade. We have to understand them better before we can use them to treat people.

The same is true of many uses of adult stem cells. At present, their use is reserved for the most serious cases – where benefit outweighs risk.

While we wait

Certainly, no one wants to have a chronic or deadly disease. At the end of my father's life, he read reports about stem cells avidly. Perhaps, he thought, they could replace his failing heart, kidneys and liver. Those advances have yet to be realized. They gave him hope.

In the meantime, he took his medications, watched his diet, exercised and extended his life by about five years in doing so. Daily, I watch friends with chronic diseases take their medicine, lose weight and go on with life. A few decades ago, those medicines would not have been available, and their lives would have taken a more sharp downward course.

Taking medicine is not ideal. Nor is getting older, but as my father used to say, "It beats the alternative."

Old friends

As the gentleman at the party and I talked about his condition, the medications he took and the effects they had, I remembered the first time I met a patient with his condition. She was a co-worker. I was 18; she was an old woman of 25. Already, she was weakening.

She could not work a full day. She described to me the future she faced, and it was bleak.

"They say there's nothing they can do," she told me. "I just wish there was a pill I could take."