There is an urgent need for new and improved therapies for ALS, as there is still no cure. The aim of the Lawrence and Isabel Barnett Drug Development Program is to develop new treatments for ALS. This program builds on the Association’s funding of basic and translational research. The Association seeks applications for the preclinical assessment of pre-investigational new drug (pre-IND) therapeutics for ALS that have a high probability of reaching the clinic within three years. The proposed studies are expected to be product-driven and focused on therapeutics.
Academic institutions, biotechnology and pharmaceutical companies are encouraged to apply.
- RFP open: August 19, 2019
- Application due: October 7, 2019
- Review decision issued with award agreement: December 2019
- Earliest potential start date: January 2020
- Proof-of-concept testing of lead molecules from pharmacological treatments, biological therapies, or gene therapy in animal models of ALS. Both novel programs and repurposing of approved or clinically safe therapies from other disease indications are appropriate for this RFA.
- Determination of target engagement, pharmacokinetics, pharmacodynamics, and preclinical toxicology/safety.
- Development of pharmacologic agents through Adsorption, Distribution, Metabolism, Excretion, and Toxicity (ADMET) phase; This can be performed by a contract research organization in collaboration with the PI.
Please Note: Clinical trials will not be supported with this funding opportunity. Studies proposing high-throughput screening, hit-to-lead, and lead optimization will not be supported through this funding opportunity.
- The maximum period of performance is two years.
- The maximum budget for the entire period of performance is $250,000 per year (500,000 total).
- Up to 10% in indirect costs, and additional costs such as travel and open science fees (e.g. publication fees, data sharing fees) may be included in this $250,000 per year total.
- Principal Investigator(s) salary will not be allowed.
- Payment structure will be negotiated and based on milestone achievements. Time lines must be clearly described.
- Regardless of the period of performance proposed, the applicant may not exceed the maximum allowable costs.
- Equipment costs should not exceed 20% of annual funding.
- Pre-IND therapeutics for ALS that have a high probability of reaching the clinic within three years.
- Successful applications will provide strong and compelling data supporting the biological rationale for the proposed treatment and a clear plan, including essential “Go/No-Go” decision milestones, for moving the approach through the essential stages of development.
- The Association expects a royalty from discoveries in proportion to its contributions to the direct activity leading to the drug discovery. Grantee shall have the first right, but not the obligation, to prepare, file, prosecute, and maintain all registerable rights in Intellectual Property arising from implementation of the Research Plan at its sole expense. It shall notify the Association if it does not intend to pursue Patent protection for any invention, discovery or idea arising from the implementation of the Research Plan and assign all rights to the Association.
- Applications must address at least one of the areas of programmatic interest listed in the eligible topics section above. Applications that do not focus on at least one of the following areas will be administratively withdrawn.
- For an overview of Association policies and terms of award, please click here.
The preclinical drug development process will likely require resources beyond those available at a single organization. Therefore, applications are open to investigators participating in synergistic collaborations, focused testing and developing lead agents for the treatment of ALS.
If a collaboration is proposed, letters confirming/supporting the collaboration are required. Specific roles and responsibilities for each collaborator need to be clearly articulated. If the collaboration is multi-organizational, participating organizations will ensure the success of the collaboration by resolving potential intellectual and material property issues and by removing organizational barriers that might interfere with achieving high levels of cooperation.
Instructions for submission
- Access application and instructions here.
- Please also fill out the drug scoresheet form and attach with your application.
- Submit questions and other related correspondence to [email protected].
Review Criteria for Proposals:
- Impact: Potential for the proposed studies to make an important contribution to ALS therapeutic development and how does the approach fit the current landscape of ALS treatments in development.
- Target/Pathway: Rationale for developing compounds against the given target/pathway.
- Research Strategy: Feasibility, timeline and design of experiments, and whether the development plan will result in go-no go decisions in moving the therapeutic forward.
- Research Team: Qualifications of the PI, key personnel, and collaborators/consultants are appropriate to perform the proposed research project.
- IP/Patent landscape: Impact on future development/commercialization of the therapeutic.