Remember the 2014 “Ice Bucket Challenge”? Around the world, people threw buckets full of ice water at their heads and promised to donate to a good cause.
The challenge was initiated by two people with amyotrophic lateral sclerosis, more commonly referred to as “ALS” or Lou Gehrig’s disease, to help raise awareness of the neurological condition.
And what you may not know is that a social media trend has raised $115 million in donations to the ALS Association.
According to a press release, the association invested part of the donation in the development and testing of a treatment for ALS called Relyvrio.
This year, Relyvrio was approved by the Food and Drug Administration for the treatment of ALS.
“Thank you to the millions of people who have made it possible to donate, participate and invest in promising therapies like AMX0035. [Relyvrio] It will immediately help people living with ALS,” said Calaneet Balas, president and CEO of the ALS Association, in a statement.
ALS is a rare nervous system disease that affects the nerve cells that control voluntary muscle movements, according to the National Institute of Neurological Disorders and Stroke.
Most patients experience worsening symptoms over time and respiratory failure within three to five years of disease onset, said Eufrosina Young, a board-certified neurologist and ALS expert in the Department of Neurology at Upstate University Hospital. and lead to death.
Relyvrio, combined with other ALS treatments, can extend patients’ lives by nearly seven months, based on data collected from that trial, Young said. The patient has already called her clinic and asked if she could add the drug to her treatment, she said.
“First risk Hospitalizations were measured to be 44% lower in patients receiving this drug,” she notes, adding, “These are very important markers of disease progression.”
There is hesitation over Relyvrio’s approval timeline
But while ALS patients are excited and hopeful about the drug’s approval, some experts are nervous that it has been approved too soon and without enough scrutiny.
“There is a general concern that the trials are wrong,” David Rind, chief medical officer of the Institute for Clinical and Economic Review, told NPR.
For experts like Lind, part of the hesitation may stem from the fact that the FDA approved Relyvrio after just one study involving just 137 patients.
Typically, two small, independent studies with fewer than 1,000 patients showed similar results or had overall positive results for a drug to be approved by the FDA. Two large-scale studies are needed.
“Given the serious, life-threatening nature of ALS and the substantial unmet need, this level of uncertainty is acceptable in this case,” the agency wrote in its Benefit-Risk Assessment.
Young said he would have liked more participants to participate in the trial, but he agrees with the FDA’s decision.
“Overall, everyone wants bigger trials, everyone wants more evidence,” she says.
Another trial with more participants is underway to further test the drug’s efficacy, and Young hopes it will provide more assurance.
At this time, Relyvrio monotherapy is not recommended and is currently given as add-on therapy in combination with existing drugs for ALS, she said.
But another potential issue to consider is access to the drug itself. After Relyvrio’s approval, the drug’s maker, Amylyx, said it would cost $158,000 a year.
According to Futurism, an Amylyx spokesperson said the company hopes to negotiate with insurance companies to lower prices, and plans to use the money from the sale to develop new treatments in the future. said it was.
“After the FDA grants approval, the main battle is with medical insurance to cover costs. Even with medical insurance, patients may or may not have copays. Yes,” says Young.
“These patients are terminally ill and deserve this treatment.”
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