Winter 2022 ALS Research Round Up

We’ve talked before here about how research is incremental. How one small discovery leads to another discovery, which informs the direction of another study. But while research progress can at times seem slow, that should not be mistaken for a lack of urgency among those doing the research. In fact, the ALS Association has recently made the case to an important arm of the U.S. government that it wants their help in moving ALS research forward even faster.

The National Institute of Neurological Disorders and Stroke (NINDS) recently issued a request for input to help guide their work in support of ALS research. NINDS has convened a working group to study the current landscape of ALS research. In response to request, the ALS Association submitted input telling the agency that speed matters. In fact, they urged the NINDS to help the Association use research to advance the health of people living with ALS as quickly as possible. In any way they can, the ALS Association asked the NINDS to focus its efforts in investments within three key areas: 1) Finding new treatments and cures, 2) Optimizing current care and 3) Preventing or delaying the harms of ALS. The NINDS is expected to present their findings in October 2022.

In other important ALS research news:

  • The U.S. Food and Drug Administration (FDA) has accepted Amylyx Pharmaceuticals’ New Drug Application (NDA) for AMX0035

And the FDA has granted priority review with target date of June 29, 2022 for completion of review and action. Additionally, Amylyx is preparing to submit an Expanded Access Program (EAP) to the FDA for launch in the U.S. in the coming months for patients who are ineligible for participation in the global Phase 3 PHOENIX clinical trial. “Our team is committed to bringing a potential new treatment, if approved, to people living with ALS as efficiently as possible, as each moment counts,” said Co-CEO, Chairman and Co-Founder of Amylyx Joshua Cohen.

  • The FDA approves phase 2 clinical trial of psychedelic ketamine for treatment of ALS

PharmaTher Holdings Ltd. has announced that the FDA has accepted an investigational NDA to proceed with a Phase 2 clinical trial evaluating the drug ketamine in the treatment of ALS. The Study will be conducted at the University of Missouri led by the Study’s Primary Investigator, Dr. Richard Barohn, M.D.

  • Mitsubishi Tanabe Pharma America has announced the FDA has accepted their NDA for an investigational oral formulation of edaravone

The FDA has accepted Mitsubishi Tanabe Pharma America application for priority review of oral edaravone. The investigational treatment has been developed to have a similar clinical profile compared to the intravenous (IV) formulation of RADICAVA, which was approved by the FDA for the treatment of ALS in May 2017. The FDA grants Priority Review for medicines that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.

  • ALS Focus telehealth survey results

The ALS focus team recently released the results from their telehealth survey to understand the needs and experiences of the ALS community as they transitioned from in-person appointments to more frequent virtual appointment during the pandemic. The survey found that more than 75 percent of respondents had a telehealth appointment during the pandemic, including nearly a quarter of people who had an appointment with an out-of-state provider. Overall, these appointments proved to be a time-saver, with more than 60 percent of people with ALS and caregivers saying the appointments saved them “a lot” of time. Nearly half of respondents also said that the appointments saved them “some” or “a lot” of money.

As to the effectiveness of these telehealth appointments, more than 60 percent of respondents said they felt telehealth appointments were just as good as in-person visits for receiving guidance from their healthcare providers. However, they were less confident that in receiving care through telehealth appointments, with more than half saying they felt less confidence about receiving high quality care through telehealth in place of in-person appointments.

You can see all the results from the ALS Focus telehealth survey here. And if you would like to add your voice and insights to future surveys, you can register here.

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