Houston Chronicle by Todd Ackerman 9/17/2018
Sharon Bradley got the jitters on the drive from Pearland to the hospital where her husband was scheduled to receive an experimental treatment never before tried in an ALS patient.
The couple hoped the immunotherapy would buy Larry time in his battle with amyotrophic lateral sclerosis (ALS), the terminal neurodegenerative disorder that traps patients in an increasingly paralyzed body. But they also knew something could go wrong in a new therapy only tested in mice, that it could hasten the disease's development rather than slow it.
One patient initially interested in the treatment got cold feet.
"Are you sure you're game?" Sharon asked.
"No matter what, we don't have a lot of time, " Larry, unperturbed, told his wife. "I want to try this for myself. But at the end of the day, if it doesn't make a difference for me, maybe it'll make a difference for someone else."
Researchers haven't had much luck devising treatment to make a difference in the lives of ALS patients, who on average die three to five years after diagnosis. Now Dr. Stanley Appel, a Houston Methodist Hospital doctor who's considered a world leader in the field, is hoping he's unlocked an answer.
Appel's approach, funded by an Ice Bucket Challenge grant, involves injecting patients with their own immune cells, cells that had become dysfunctional in the body but were normalized and expanded in the laboratory after their removal. The idea is that again functioning properly in the body, the cells will help prevent the deterioration that characterizes ALS.
If clinical trials confirm the approach works, Appel thinks it might provide chronic treatment, much like insulin for diabetics.
Lou Gehrig’s diseaseALS, also known as Lou Gehrig's disease, is one of medicine's most devastating disorders, an attack on nerve cells in the brain and spinal cord that control voluntary movement. The resulting muscle degeneration leaves patients unable to walk, talk, swallow and, ultimately, breathe.
More:
https://www.houstonchronicle.com/local/prognosis/article/New-immunotherapy-treatment-funded-by-the-Ice-13231335.php
