Objective:

People Against Leigh Syndrome (PALS) is seeking proposals for independent research that needs funding for the final push to advance a project from the bench top to the bedside. Ideally, funded projects will positively impact patient care in the near future.

Timing:

PALS will begin accepting written proposals on 15 August 2021 and will close the submission period at 11:59 p.m. EST on 31 October 2021.  Awards will be announced by December 2021 with funding to begin no later than January 2022 pending execution of a grant award agreement.

About PALS:

PALS was formed in 2013 after William Martin, son of Lori and Neil Martin, was diagnosed with Leigh syndrome. After discovering the disease was progressive and potentially fatal, supporters of the Martin Family decided to take action and through PALS have built awareness of this rare disease, so far raising more than $500,000 to support the medical and research communities in their quest to develop viable treatments and ultimately a cure.  

PALS currently supports TRiaLS: The International Registry for Leigh syndrome through the UTHealth Mitochondrial Center of Excellence as well as the Leigh Syndrome International Consortium and will continue to do so. Through this RFP, PALS seeks to fund therapeutic-focused and mechanism-based research that will quickly help patients battling Leigh syndrome.

Scope of Projects:

All submissions must have a Leigh syndrome focus. The following research categories and priorities have been established:

1. Translational Research - Projects that contribute new tools for advancing basic research from the bench top to the clinic, especially novel cellular and animal models or their application to deepen insights, biomarkers, diagnostics, or therapeutics for Leigh syndrome.

2. Clinical Research - Patient-centered projects that focus on development of therapeutic approaches for treating Leigh syndrome patients.

3. Interventional Pilot Clinical Trials – Small, patient driven proof of concept clinical trials that could lead to large-scale or industry sponsored funding.

Funding Mechanisms:

Applicants must be established independent investigators. Postdoctoral fellows are NOT eligible for awards under this RFP.

PALS intends to fund up to 25,000 USD in project(s) under this RFP.  Budgets should not exceed 25,000 USD over 12 months (to include both direct and indirect costs with indirect costs not to exceed 10 percent of total budget).  Small budget, shorter duration submissions are highly encouraged.  Applicants may be based anywhere in the world.

Application Process:

The application process will proceed as follows:

• Click on "Download File" below to download application.

• Completed application and supporting materials should be emailed to peopleagainstleighsyndrome@gmail.com no later than 11:59 p.m. EST on 31 October 2021.  Applicants are reminded to leave sufficient time for any necessary institutional approvals so as to meet the above timeline. Late submissions will not be considered.

Review Process:

Applicants may be assured that all submitted information is handled as confidential with any potential reviewer conflicts of interest taken very seriously.

• All submitted proposals must include a 750 word (maximum) scientific abstract that provides sufficient detail to allow for further consideration.

• Submissions will be reviewed by PALS Leadership, including director Lindsey Klingensmith and the Board of Directors Lori Martin, Tahra Peterson, Peyton Jones and Melissa Knight, as well as a medical committee including Mary Kay Koenig, MD; Sumit Parikh, MD; Russell P. Saneto, DO, PhD; and Nivedita Thakur, MD.

• Scoring/Review criteria include (but are not limited to): Responsiveness to the intent of the RFP, relevance to Leigh syndrome, scientific novelty, scientific premise and rigor, and potential to impact the lives of those affected with Leigh syndrome quickly.

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