Response to NINDS Request for Information on ALS Strategic Plan

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February 11, 2022

Dear NINDS,

As we set our sights on shaping the strategic future of ALS research, we thank you for the opportunity to identify key research and priorities at NIH and beyond.  We also recognize that many individuals with ALS need better treatments for their disease today, and we are encouraged by your efforts to advance new therapies as quickly as possible through development of this ALS Strategic Plan.

The Les Turner ALS Foundation has tirelessly advocated for the ALS community for better research, care, and support for 45 years, designing and implementing new programs by working alongside people with ALS, their families, clinicians, and researchers. In response to your request for information, below are a set of research priorities that will transform research and care.

A. Opportunities for understanding the genetic, molecular, cellular, or neural pathway changes that cause ALS or influence disease progression

  • Draw collaborative innovators together to establish the tools and infrastructure necessary to accelerate our understanding of the genetic and environmental causes of ALS
  • Establish a large natural history study to collect real-world clinical and genetic data about ALS, to enhance our understanding of the disease and enable improved care practices for people living with ALS

B. Strategies for translating discoveries into therapies

  • Identify where directed investment can help drive fundamental discoveries through the various stages of treatment development, from laboratory to clinic.
  • Develop and optimize research models to systematically evaluate which therapies are most likely to have clinical impact for people with ALS
  • Ensure better coordination of ALS research efforts by creating an infrastructure that facilitates collaboration and helps coordinate research efforts across all NIH institutes as well as across private and public funders involved in ALS research

C. The most important factors to consider when conducting clinical research

  • Explore and mandate innovative and more patient-centric clinical trial design
  • Ensure equitable access to the latest therapies and treatment through a broad clinical trials network, with specific outreach to people living with ALS who are part of underrepresented populations-people of color and people living outside of major medical centers.
  • Identify new outcome measures for clinical trials that include biomarkers and novel measures of function

D. Biggest challenges for people currently living with ALS and their caregivers

  • Ensure that clinical management of ALS is informed by the priorities of people living with ALS and their caregivers and supported by evidence
  • Ensure equitable access to the latest therapies and treatment through a broad clinical trials network with specific outreach to people living with ALS who are part of underrepresented populations- people of color and people living outside of major medical centers.

E. Interventions, such as technologies or symptomatic therapies, that will improve the quality of life of people living with ALS and their caregivers

  • Evaluate innovative ways and potential public and private collaborations to sustain care model and deliver care more efficiently and effectively (multidisciplinary care, home health and caregiving options)
  • Invest in support and outreach to help optimize mental health and emotional wellness services for people living with ALS and caregivers

F. Opportunities for collaborations and partnerships that will accelerate the discovery of effective interventions for the diagnosis, treatment, management, prevention, or cure of ALS

  • Identify new funding resources available to invest in quality improvement in each of the above areas
  • Understand barriers to managing everyday care
  • Create resources to help people with ALS and families guide decisions about their treatment in collaboration with their ALS care team
  • Invest in programs to create a sustainable pipeline of ALS researchers through research training grants

G. Inequities, if any, that limit the ability of people living with ALS, their families, and caregivers to participating in the research process

  • Understand the impact of social determinants of health and identify and fund opportunities to minimize their effect on optimal ALS care