Notice of Special Interest (NOSI): Common Fund ALS-related Transformative Research Award (R01 Clinical Trial Optional)
Notice Number:

Key Dates

Release Date:

June 17, 2020

Related Announcements

NOT-RM-21-004 - Notice of Clarification to the High-Risk, High-Reward Research Program: Pioneer: RFA-RM-20-011, New Innovator: RFA-RM-20-012, Transformative Research (RFA-RM-20-013, RFA-RM-20-020 & NOT-RM-20-019), and Early Independence (RFA-RM-20-014 & RFA-RM-20-021).

RFA-RM-20-013 - NIH Director’s Transformative Research Awards (R01 Clinical Trial Optional)

Issued by

Office of Strategic Coordination (Common Fund)

National Institute on Aging (NIA)

National Institute of Environmental Health Sciences (NIEHS)

National Institute of General Medical Sciences (NIGMS)

National Institute of Neurological Disorders and Stroke (NINDS)


The purpose of this Notice is to inform potential applicants of the special interest of the Office of Strategic Coordination (Common Fund), the National Institute of Neurological Disorders and Stroke (NINDS), the National Institute on Aging (NIA), the National Institute of Environmental Health Sciences (NIEHS), and National Institute of General Medical Sciences (NIGMS) in supporting exceptionally innovative research into the basic biology of Amyotrophic Lateral Sclerosis through the NIH Director’s Transformative Research Award initiative.


Amyotrophic lateral sclerosis (ALS) is a devastating disease with no known cure. It is a neurodegenerative disease that causes the death of motor neurons that control voluntary muscles. This results in weakness and ultimately loss of voluntary muscle function. ALS is rapidly progressive and always fatal. Many people die within 3-5 years of developing symptoms. The cause of ALS is unknown but likely involves a combination of genetic and environmental risk factors. About 5 – 10% of cases are familial and the remaining cases are considered to be sporadic. Hampered by the unknown etiology of ALS despite extensive efforts, only two drugs have been developed that are FDA approved and these drugs extend life by just a few months and do not improve symptoms. Thus, the development of effective ALS therapeutics can benefit tremendously by investing in basic ALS research that tests highly novel concepts, brings together researchers from different scientific perspectives, and applies powerful emerging technologies from a variety of disciplines. Though such highly innovative research may be inherently risky, the potential payoff in our understanding of ALS may warrant the risk.

To solicit and support such high-risk, high-reward ALS-related research, the Accelerating Leading-edge Science in ALS (ALS2) is being created. ALS2 will use the existing NIH Director’s Transformative Research Award initiative to receive and review applications. This initiative is part of the NIH Common Fund’s High-Risk, High-Reward Research (HRHR) Program. The HRHR program offers a time-tested, powerful approach to sparking innovation and impact. The Transformative Research Award initiative is a particularly well-suited initiative within the HRHR program for supporting interdisciplinary teams of scientists proposing to combine their expertise and pursue highly innovative ideas. Transformative Research Award applications do not require preliminary data or a detailed experimental plan. Rather, the emphases are unusually high magnitude of potential impact, an exceptional degree of innovation, and a highly compelling logic of the approach. Of note, an anonymized review process of the Transformative Research Award applications is being piloted this year to help maintain focus on these emphases. Large budgets (exceeding $500,000 in direct costs in any given year) are acceptable without pre-approval but must be commensurate with the scope of the project.


The objective of this Notice is to advance dramatically our understanding of the complex biology of ALS. Thus, applications that use one or more of the following elements are encouraged:

  • Adapt emerging tools and technologies from neuroscience, cell biology and other disciplines to identify what causes ALS and how the disease progresses, forming the basis for new potential therapeutic strategies.
  • Attract new talent from a range of scientific disciplines, including cell biology, bioengineering, chemistry, biophysics, environmental health sciences, and computational science, and initiate new interdisciplinary collaborations.
  • Explore potential similarities between ALS and other neurodegenerative disorders and beyond, including, but not limited to, frontotemporal dementia, chronic traumatic encephalopathy, Kennedy’s disease, spinal muscular atrophy, primary lateral sclerosis, and aging-induced neuromuscular degeneration.

Application, Review, and Funding Information

Applications must be submitted in response to FOA RFA-RM-20-13. All instructions in the FOA must be followed. In addition, applications must indicate “NOT-RM-20-019” (without quotation marks) in the Agency Routing Identifier field (Box 4b) of the SF424 (R&R) Form. Applications without this information in Box 4b will not be considered for support by the ALS2 program. The receipt date is September 30, 2020.

Applications will be reviewed using the same review process as for all other Transformative Research Award applications as described in RFA-RM-20-013.

Funding for applications submitted through this Notice will be considered separately from other TRA applications and will be based on the results of peer review and programmatic priorities.


Please direct all inquiries to:

Amelie K. Gubitz, Ph.D.
National Institute of Neurological Disorders and Stroke (NINDS)
Telephone: 301-496-5680

Ravi Basavappa
Office of the Director (OD)
Telephone: 301-435-7204

Lisa Opanashuk, Ph.D.
National Institute of Aging (NIA)
Telephone: 301-82705422

Jonathan A. Hollander, Ph.D.
National Institute of Environmental Health Sciences (NIEHS)
Telephone: 984-287-3269

Oleg Barski, Ph.D.
National Institute of General Medical Sciences (NIGMS)
Telephone: 301-496-1511

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