ALS Association’s Advice to NINDS: Move Quickly on Research

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by Mary Chapman |

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In response to the National Institute of Neurological Disorders and Stroke‘s (NINDS) request for community input regarding what it should prioritize to accelerate amyotrophic lateral sclerosis (ALS) research, the ALS Association’s overarching message is this: move swiftly to help patients.

NINDS, which is part of the National Institutes of Health (NIH), sought contributions last month from patients, families, caregivers, researchers, advocates, and clinicians. The agency’s chief objective is to determine the community’s top priorities for studies into more effectively diagnosing, treating, managing, and preventing ALS.

More specifically, NINDS sought feedback regarding perceived knowledge gaps, investigation opportunities, and which clinical trials it should prioritize, so that it can complete its strategic planning process to set its research priorities. The deadline for responses closed Feb. 11.

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The ALS Association’s research plan is based on funding for three key areas: finding new treatments and cures; optimizing current care; and preventing or delaying harms caused by ALS.

“The ALS Association is focused on ensuring people with ALS live longer, with improved quality of life, and their loved ones are prevented from getting ALS,” the organization said in a press release.

Regarding the organization’s top priority, finding new therapies, the ALS Association is focused on increasing research funding and the number of clinical trials, and improving regulatory science for ALS, particularly biomarkers and trial endpoints.

“We are placing these objectives above the fundamental science needed to better understand ALS, because our priority is to improve the health of people with ALS as quickly as possible,” the organization’s response states.

“There are numerous risk factors, pathways and genes that have been identified as playing a role in ALS, and we can find more effective treatments if we place greater emphasis on testing their treatment potential quickly,” it continued. “As new treatments come to clinic, the ALS community needs the capacity to rapidly test their effectiveness in the real world, and in combination with other treatments taken by people with ALS.”

In terms of optimizing current care, the organization wants the NIH to focus on interventions that improve the health of patients and their caregivers, while continuing the search for disease-modifying treatments. To that end, the Association itself is focusing on optimizing the use of assistive technologies, reducing ALS complications, and increasing access to high quality care.

As for preventing or putting off harms caused by the progressive neurodegenerative disorder, the organization mainly wants to see interventions that can prevent new ALS cases, whether those interventions are achieved through medicine, behavior, or legislation.

“This has never been done before and requires the ALS community to overcome both scientific and organizational challenges,” the association states.

All responses, including those from the ALS Association, will be compiled by working groups of community stakeholders that will present their findings at free public hearings on Oct. 26–27, at which time the public can provide input.

Based on that input, the groups may modify their research priorities. Those priorities will be posted online for public comment before being submitted for approval to the National Advisory Neurological Disorders and Stroke Council.

“We value the work of NINDS and other research funders in the ecosystem of ALS research, and our research priorities are, to some extent, set by our unique position within that ecosystem. Still, we would encourage NINDS and the rest of NIH to focus on their mission and find ways to use research to advance the health of people living with ALS as soon as possible,” the ALS Association wrote.

“The urgency of the ALS patient community necessitates a very aggressive timeline — speed matters!,” it concluded.