ALS stem cell trial is chronicled in Detroit Free Press two-part series
By Robin Erb, Free Press Medical Writer
Sometimes she glares at the painting of Jesus in her dining room.
“I just let it loose,” said Mary Kleiss at her Royal Oak home. “I look at that picture and I say, ‘You get down here and put on your boxing gloves and let’s get this over with.’ I am so damned angry.”
Her son, Regis, was diagnosed two and a half years ago with Lou Gehrig’s disease — amyotrophic lateral sclerosis, or ALS. It is, he writes, “as if God is torturing me.”
The disease kills with stunning efficiency — deadening its victims’ peripheral nerves, withering muscles and, in a final assault, shutting down their ability to breathe. An estimated 30,000 people have it at any given time; 5,000 are diagnosed yearly. Most die within years. There is no cure.
The disease has reduced Regis Kleiss, 28, a formerly thick-bodied shot and discus thrower and captain of the track team at Dondero High School, to a bony echo of himself. Paralyzed except for some minor movement of his head, he will spend his final days on a feeding tube.
ALS leaves its victims’ minds intact.
“It’s a miserable, damned disease,” his mother said.
Now, a clinical trial overseen by the University of Michigan may provide hope. It is cutting-edge and audacious work — the only ALS trial so far in which neural stem cells are injected directly into a patient’s spinal cord. So far, 15 patients have undergone the procedure — two of them twice — as the FDA monitors its safety.
One patient showed a remarkable improvement for a while, though U-M’s Dr. Eva Feldman, who heads the research, cautions not to read too much into that. The other 14 showed no improvement.
The trail is tentative and early. But when the rest of a person’s life has been compressed to an expectancy of two to five years, it is hope nonetheless.
The trial has been based in Atlanta since 2010, but U-M has requested approval from the U.S. Food and Drug Administration to expand it and move it to the University of Michigan in Ann Arbor.
The trial involves injecting 500,000-1 million stem cells into the spine. The ancestry of the cells dates to the spinal cord of an aborted fetus in 2000. The cells are different from the embryonic stem cells that were the subject of a controversial ballot proposal in Michigan in 2008, when voters approved lifting the ban on embryonic stem cell research.
Feldman and others theorize that these new cells act as nursemaids to damaged nerve cells, sending out repair signals, and somehow halting the progression of the disease.
The procedure worked in rats. It has been shown to be safe in pigs.
If the FDA approves moving the trial to Ann Arbor, Michigan patients will have access to an experimental treatment that not only might offer insight into a disease that kills an estimated 15 Americans a day, but also push back the battle lines against other neurodegenerative diseases, such as Parkinson’s and Alzheimer’s or Huntington’s.
For patients: We appreciate your interest in our stem cell trial and as this article mentions we are working to bring it to Michigan. However, we are not at this time able to accept patient applications. Please check this site monthly for any updates.