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Clinical trial results published on September 3, 2020 indicate that the new drug, AMX0035, slowed ALS functional decline in a meaningful way.
The ALS Association, which supported the development of AMX0035, says that it “could mean the difference between a person with ALS being able to feed themselves versus being fed, or the difference between needing a wheelchair versus not needing one.” |
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These promising results, however, come from a phase 2 clinical trial. Typically, a more lengthy phase 3 trial is needed to confirm the effectiveness and safety of a new drug before it can be made available to the public. Advocates are already pushing the FDA to make the medication available sooner so that current ALS patients have the chance to benefit.
AMX0035 is a powder that is dissolved in water and taken twice a day either orally or through a feeding tube. It combines two existing drugs (sodium phenylbutyrate and taurursodiol), both of which have been found safe in prior studies. Researchers believe that AMX0035 is safe. Some trial participants experienced mild side effects.
This six-month study involved 137 participants who were diagnosed with ALS within 18 months prior to the start of the study and showed fast-progressing symptoms. Clinical trials were conducted by leading ALS researchers at medical centers across the United States. Eighty-nine people received the drug and 48 got a placebo. Those who received AMX0035 performed two points better on the ALS Functional Rating Scale. Most participants were already taking riluzole (Rilutek) and/or Radicava (edaravone).
AMX0035 is not a cure, does not restore function, and does not stop decline. It appears to slow the progression of ALS. The lead researcher said that the trial results were modest but meaningful. She believes that AMX0035 will become one of a handful of medications patients will take together to slow the progression of ALS until there is a cure.
There is no cost estimate yet for AMX0035 and it is unclear whether insurers would cover this drug based on phase 2 trial results.
Learn about other medications for ALS on our Research and Treatment page.
This six-month study involved 137 participants who were diagnosed with ALS within 18 months prior to the start of the study and showed fast-progressing symptoms. Clinical trials were conducted by leading ALS researchers at medical centers across the United States. Eighty-nine people received the drug and 48 got a placebo. Those who received AMX0035 performed two points better on the ALS Functional Rating Scale. Most participants were already taking riluzole (Rilutek) and/or Radicava (edaravone).
AMX0035 is not a cure, does not restore function, and does not stop decline. It appears to slow the progression of ALS. The lead researcher said that the trial results were modest but meaningful. She believes that AMX0035 will become one of a handful of medications patients will take together to slow the progression of ALS until there is a cure.
There is no cost estimate yet for AMX0035 and it is unclear whether insurers would cover this drug based on phase 2 trial results.
Learn about other medications for ALS on our Research and Treatment page.
