New drug slowed progression of ALS for people in middle age, researchers say
The disease that struck down baseball giant Lou Gehrig in his prime still has no cure, but researchers at the University of California, San Francisco, announced Monday that a new treatment appears to slow it down in some patients ages 40 to 65.
The new drug, which is most effective on people who suffer with higher levels of chronic inflammation, stalls the progression of the degenerative neurological disease formally known as amyotrophic lateral sclerosis, or ALS, according to research published Monday in the journal Muscle & Nerve.
People who have the disease lose voluntary control of their muscles as the motor neurons that control this process deteriorate and die in their brains, in their spinal cords and in their muscles.
Discovered in 1869, ALS didn’t become a household name around the world until it forced Gehrig to take himself out of the New York Yankees’ line-up in 1939 after playing a record-setting 2,130 games. He died within two years. Most patients don’t live more than five years after diagnosis.
Those who have the disease lose the ability to eat, speak, move or breathe, but UCSF researchers found that, at higher doses, a drug known as NP001 temporarily suspended the decline of patients who had high levels of a molecules that promotes inflammation.
“My hope is that we can continue the study of patients whose disease progression slowed or halted with NP001 as compared to placebo,” said Dr. Michael S. McGrath, an emeritus professor of medicine at UCSF. “We’ve still got a lot to learn about ALS pathogenesis and how best to treat, but I believe that we’ve found a path forward for use of an immunoregulatory approach to a large subset of patients with ALS.”
Inflammation typically can be a good thing because it trips the immune system and jump-starts healing, but in the case of some diseases, the immune system mistakes perfectly normal molecules as a threat and set off an attack.
McGrath and his team had a hunch that this is what was happening with ALS and set out to develop a drug that would inhibit production of inflammation-promoting factors called cytokines. That was the beginning of NP001.
They tested the drug in 154 people with ALS, ages 32 to 76, and to their dismay, the drug did not significantly slow the progression of the disease when compared with a placebo group, said McGrath, the cofounder of Neuvivo Inc. a pharmaceutical company that sponsored the study.
Then, he said, they took a closer look at the data and discovered that 117 study participants ages 40 to 65 had shown marked improvement. That subset represents 76 percent of the people in the study.
Compared with the placebo group, this subset of participants showed a 36% slowing of the decline of their physical changes and a 51% slowing of the decline in their respiratory function.
“It is plausible that the disease mechanism and trajectory for those patients under 40 differs from older patients and is more likely to have a genetic or heritable component as is often seen in other chronic diseases, although this has not yet been proven,” McGrath said.
This story was originally published February 1, 2022 at 6:00 AM with the headline "New drug slowed progression of ALS for people in middle age, researchers say."
