Researchers Encouraged By Results Of Recent ALS Phase 2 Drug Trial
PITTSBURGH (KDKA) -- For someone diagnosed with ALS in 2020, the prognosis is still grim.
"The outlook is from three to five years survival. But we do have outliers who beat all the odds and will survive longer, up to seven years, nine years," says Allegheny Health Network ALS specialist Dr. Sandeep Rana.
ALS is diagnosed by physical exam and nerve and muscle tests. Why it happens still isn't known, but some risk groups have emerged, such as soldiers.
"Like professional athletes, involved in contact sports, they have a higher incidence of developing ALS. Trauma may be playing a role," says Dr. Rana.
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But treatment has changed.
"I wouldn't say we have the cure, but there are options that we can slow down the progression of the illness," Dr. Rana says.
The treatment includes Riluzole, a pill, and an intravenous drug called Radicava, which requires a special IV port to remain in the chest.
"Patients hesitate to consider that as an option, and there are some who elect not to proceed that route," said Dr. Rana.
Radicava was approved in 2017, and the first patient in the U.S. to get it was in Pittsburgh. One-third of the patients who get it hold steady, one-third of the patients show no response and another third decline rapidly.
Dr. Rana says the timing may matter.
"Start early and this gives the patient the best opportunity to retain their level of functioning. It's worth trying. It's worth offering to all patients," the doctor said.
And a new medicine may be coming soon. In phase 2 trials of about 100 patients, those getting the drug — along with Riluzole and Radicava — had a slower progression of the disease over several months. The drug company is encouraged by these results.
"So they approached the FDA to allow them to get the approval even though traditionally it would require a phase 3 study," Dr. Rana explains.
Meanwhile, other drugs are in the works.
"There are up to 30 products worldwide that are being studied for ALS, very exciting time for ALS," said Dr. Rana. "We have a couple of new trials that we are initiating and offering in our clinic."
This degenerative disease of certain nerve cells leads to weakness, trouble walking and speaking, cramps and twitching, and changes in behavior. Eventually, it affects the breathing muscles.
