DoD Amyotrophic Lateral Sclerosis Therapeutic Development Award | W81XWH 20 ALSRP TDA

FAQs: DoD Amyotrophic Lateral Sclerosis (ALS) Therapeutic Development Award (FY20 ALSRP TDA)

1) What is the purpose of the FY20 ALSRP Therapeutic Development Award (TDA)?

The award is designed to advance promising ALS therapeutic candidates along the drug development pipeline once a credible therapeutic lead already exists. It supports work from therapeutic lead validation through FDA Investigational New Drug (IND)-enabling studies, with the goal of producing a data package that can responsibly support first-in-human testing later (even though clinical trials are not allowed under this award).

2) What stage of research is this award meant to support?

This program targets development-oriented research rather than early discovery. Projects are expected to start with identified lead compounds or a lead class of compounds and move forward through preclinical development activities that typically precede IND submission.

3) Are early discovery or exploratory projects a fit for this opportunity?

No. The expectation is that proposals are not early discovery efforts. The Department of Defense is looking for empirical, product-driven work aimed at de-risking a specific therapeutic candidate and generating development-quality evidence.

4) What is meant by having a "credible therapeutic lead" at the time of application?

Applicants are expected to have already identified lead compounds (or a lead class) and to provide solid preliminary data appropriate to the development stage being proposed. At a minimum, there must be evidence of efficacy in at least one ALS-relevant model system.

5) What minimum efficacy evidence is required to be considered responsive?

The opportunity requires evidence of efficacy in at least one ALS-relevant model system, which may include cellular models and whole-animal models.

6) What kinds of supporting drug development data are expected beyond efficacy?

Applicants are expected to demonstrate practical development fundamentals, including proof of compound identity and purity, evidence of selectivity for the intended target (especially against closely related targets), and workable in vitro assays (primary and secondary) that can support optimization and structure-activity relationship (SAR) studies.

7) What types of preclinical development activities does the award support?

The award supports activities commonly performed between initial discovery and IND submission, including: confirming and strengthening candidate therapeutics derived from screening or discovery approaches; optimizing potency and drug-like properties; evaluating derivatives or related compounds to improve performance; and expanding pilot findings with stronger, more definitive study designs.

8) Does the program support optimization work such as SAR and improving drug-like properties?

Yes. The solicitation explicitly references optimization of potency and drug-like properties and the use of in vitro assays to support optimization and SAR studies.

9) Can applicants propose studies that replicate and strengthen earlier findings?

Yes. The opportunity supports moving beyond small pilot findings by replicating results with stronger study designs, for example by incorporating multiple ALS model systems, adding time points, or testing additional doses to better define dose-response relationships.

10) Are formulation and stability studies allowed?

Yes. The program allows formulation and stability studies that can lead into Good Manufacturing Practice (GMP) production methods.

11) What IND-enabling studies are contemplated under this award?

The award supports classic IND-enabling activities such as compound characterization, ADME (absorption, distribution, metabolism, and excretion) studies, and appropriately designed dose-response and toxicology studies in relevant model systems.

12) Are clinical trials permitted under this award?

No. Clinical trials are explicitly not permitted under this mechanism.

13) If clinical trials are not allowed, is any human-related research permitted?

Yes, a specific type of human-data validation is encouraged: testing biological correlates of disease activity or progression using pre-existing, de-identified human specimens from well-characterized patient cohorts, as long as those materials already exist at the time the application is submitted.

14) Can the project collect new human biospecimens during the award period?

No. Collecting new human specimens is not supported under this mechanism. Any human specimen work must rely on existing materials available at the time of application.

15) What kinds of existing human data or specimens are considered acceptable sources?

Acceptable sources include specimens or datasets from controlled clinical trials, observational studies, publicly available biorepositories, and registries. The solicitation includes examples such as the CDC National ALS Registry and Biorepository.

16) What if an applicant wants to use a human specimen repository not specifically named in the solicitation?

If a different repository is proposed, applicants are expected to provide a letter of support describing key details about the resource and explaining how data and samples are broadly accessible.

17) Are military and Veteran ALS populations relevant to this opportunity?

Yes. The opportunity signals that active duty military and Veteran patient populations and related resources should be considered where relevant.

18) What is the biomarker expectation for this award?

Biomarkers are a defining requirement. The Department of Defense makes clear that having validated biomarkers available or conducting biomarker development and characterization in parallel with therapeutic work is a critical component of the program.

19) What types of biomarkers does the program prioritize?

The program prioritizes biomarkers that directly support therapeutic development and eventual clinical trials, such as target engagement biomarkers (does the drug hit its target), pharmacodynamic biomarkers (does it produce a measurable biological effect consistent with the mechanism), and predictive or cohort-selective biomarkers (which patients or subgroups are more likely to respond).

20) Are diagnostic, prognostic, or disease progression biomarkers considered responsive?

Not by themselves. Biomarker work aimed purely at diagnosis, prognosis, or tracking progression without a clear tie to accelerating or strengthening the therapeutic development pathway is not considered responsive.

21) What details should applicants provide about their proposed biomarker strategy?

Applicants are expected to clearly define the biomarker, explain how it will be used, and justify how it improves the efficiency and interpretability of the overall therapeutic development plan.

22) Does the opportunity encourage collaboration for teams with limited ALS experience?

Yes. Because ALS models and endpoints can be technically demanding, teams with limited ALS experience are strongly encouraged to partner with collaborators who have deep expertise in ALS pathophysiology, relevant model systems, and appropriate outcome measures.

23) What agency administers this funding opportunity?

This is a discretionary Department of Defense funding opportunity administered by the U.S. Army Medical Research Acquisition Activity (USAMRAA).

24) What funding mechanisms are used?

The opportunity uses grant and cooperative agreement mechanisms.

25) What is the CFDA number associated with this program?

The listing references CFDA 12.420.

26) Who is eligible to apply?

Eligibility is described as unrestricted (open to any entity type), subject to any additional eligibility clarifications that may appear in the full announcement.

27) What is the Funding Opportunity Number for this award?

The Funding Opportunity Number is W81XWH 20 ALSRP TDA.

28) When was the opportunity released and what was the original closing date?

The opportunity was originally released on January 17, 2020, with an original closing date of July 23, 2020.

29) How many awards were anticipated?

The listing anticipated a small number of awards, with two expected.

30) Is there an award ceiling or budget cap stated in the summary information provided?

The listing shows an award ceiling of 0, which commonly suggests that applicants need to consult the full announcement for budget parameters or that a specific cap was not captured in the summarized field.

31) What is the overall "best fit" profile for a competitive project under this award?

A strong fit is a preclinical, development-focused project that starts with a credible lead and ALS-relevant efficacy evidence, proposes a clear optimization and IND-enabling plan, includes a biomarker strategy tied directly to development decision-making and eventual clinical translation, and (where applicable) reinforces translational relevance using existing de-identified human specimens or datasets.