Regis Kleiss, 28, of Royal Oak, Mich., is taking part in a clinical trial to combat amyotrophic lateral sclerosis, led by the University of Michigan.
By Kimberly P. Mitchell, Detroit Free Press
USA TODAY
DETROIT - 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 2½ years ago with Lou Gehrig's disease, formally known as amyotrophic lateral sclerosis, or ALS. It is, he writes, "as if God is torturing me."
The disease kills with swift and 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 the disease at any given time; 5,000 are diagnosed yearly. Most die within a few years. There is no cure.
The disease has reduced Regis Kleiss, 28, a formerly thick-bodied shot and discus thrower and captain of the high school track team, to a bony echo of himself.
Paralyzed except for some minor movement he can make with his head, he will spend his final days on a feeding tube, unable to hold his bassett hound, Riley, or hug his parents or his niece or even scratch his head. And the cruelest thing of all? 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's 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 Michigan has requested approval from the U.S. Food and Drug Administration to expand it and move it to Ann Arbor. 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, though Michigan's Dr. Eva Feldman, who heads the research, cautions not to read too much into that.
The trial involves injecting 500,000 to 1 million stem cells into the spine. The ancestry of the line of cells dates to the spinal cord of an aborted fetus in 2000.
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 the expansion of the human trial, 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, Alzheimer's or Huntington's.
Last month, Feldman flew down with a team of Michigan staff for the 17th surgery in the trial, in anticipation that the trial might soon move to Ann Arbor. The trial has been at Emory University in Atlanta since it began.
Feldman is clear and she repeats this often: This part of the trial tests safety only. By design, it doesn't assess the efficacy of the treatment yet.
So the clinical trial patients so far - all from the Atlanta area - know the experimental stem cell therapy probably will not cure them. Still, they're empowered, knowing their participation might one day cure others, said Ed Tessaro, a retired Macy's executive.
The following morning, Tessaro, 66, lay on an operating room table at Emory's hospital, as doctors removed part of the bone in his spine and laid bare a pulsating, bright-white spinal cord for a second infusion of stem cells.
"It may kill us," Tessaro said of the disease, "but it's not going to defeat us before we die."