Grant Guidelines for Researchers

Consistent with the urgency felt by all families impacted by this disease, the A-TCP’s current focus is on funding innovative research strategies that look at A-T in new ways and that include a clear “yes” or “no” answer to the hypotheses rather than merely defining a mechanism.

Our greatest interest is in funding translational and clinical research projects focused on the neurological problems faced by all patients with A-T. We are desperate to deliver new ideas from A-T research with practical relevance for patients into the hands of experts involved in drug discovery and clinical development. We have interest in disease-modifying strategies that would prevent, slow, or stop the progression of the disease as well as symptom-improving strategies that address neural circuit function, immune system health and lung function – but especially the neurological symptoms.

We may occasionally support earlier-stage discovery research, but only if the results of those early-stage projects will clearly (a) reveal and accelerate a path to new treatments (such as validating a drug target) or (b) produce a tool or resource that will help accelerate the development of treatments (such as a viral vector that can carry the ATM gene, or a validated disease biomarker that can more quickly measure or predict the efficacy of treatments for the neurological problems of A-T).

Because many governments, non-profits and companies worldwide already spend billions of dollars annually on cancer research – some of which is likely to benefit A-T patients – we are unlikely to award grants to applications proposing research on the role of the ATM protein in cancer or focused on strategies for treating cancer in A-T patients.

Unfortunately, although we are wildly enthusiastic fans of good science, our small organization cannot afford to support researchers who simply want to use a popular methodology (such as making iPS cells from patients and then differentiating them into neuronal cells, or doing single-cell sequencing) in their lab, or who want us to fund their favorite area of focus around which they have built their career, if its relevance to A-T requires a real stretch of the imagination. It’s natural for passionate scientists to sincerely believe that their field or approach is most relevant to A-T, but we need to be more objective. We hope that you can appreciate our position on this.

With advice from our Board of Directors and Scientific Advisors, we have established some rules that we want you to understand before you put any effort into applying to us for funding.

• 100% of the funding we provide to you must be used for the direct costs of your A-T-related project. Because we are a small organization and have made promises to our donors about how we will use their donations, our grants cannot be used for administrative, overhead, or indirect costs that your institution may want applied to this project. Please let your institution know that this point is not negotiable. We appreciate that many fine institutions must somehow find support for their overhead, but as an organization desperately striving to leverage every dollar we raise from donor and grass-roots events for an ultra-rare disease, we simply cannot afford to cover overhead.
• Upon awarding a grant to you, we will set a grant start date together. Regardless of your reason, if your research does not begin as planned within six months of that grant start date, we will terminate the grant. We will not be willing to allow “no-cost extensions” beyond that date, as you will be preventing us from supporting other researchers who are ready to start their work. Therefore, we urge you to think hard about when your project will be able to start.
• We require you to submit progress reports every six months. Both the shorter mid-year progress report and the longer year-end progress report are not burdensome, especially compared to other grant-giving organizations.
• We do not believe that we should need to remind you that a progress report is due, and we believe that an investigator capable of running a rigorous research project should be able to keep track of something as simple as a due date. If we do not receive your progress report on time, we may terminate your grant.
• We may support the purchase of equipment that is critical for an A-T research project, but usually, we prefer to fund an investigator who works in a lab that already has the necessary equipment.
• As we are determined not to waste any of our donors’ hard-earned and generously given dollars, we do not usually pay for travel unless it will support collaborations necessary for the achievement of a proposal’s goals.
• Funding for proposals requiring Institutional Review Board (IRB) approval will be dependent upon and commence after IRB approval of the study protocol has been received.
• We will fund neuroscience-related research proposals using Atm-/- mice as a model system only if the selected model has a clear, obvious neurological phenotype.
• We will not fund any additional researchers eager to make iPS cell lines derived from A-T patient cells or by disrupting the ATM gene in normal or carrier cells. This work has been performed many times.

• We are not impressed by your educational pedigree or the name of your current institution. But we are very impressed by a truly original, clearly written, well-thought proposal that is as concise as possible while anticipating and addressing the questions that we and our advisors may have while reading it.
• We do not care if your research is performed in an academic or industry setting. We care only about innovative, novel research approaches to A-T performed by rigorous scientists in a cost-effective way.
• We know what A-T is. Please do not spend any time describing the clinical presentation of or the general biology underlying the disease in your proposal.
• In recognition of the growing need for increased reproducibility in all areas of research, certain points regarding experimental design, minimizing bias and the discussion of results should be considered when preparing a grant application. These points are delineated in detail as part of the NIH/NINDS guidance document Improving the Quality of NINDS-Supported Preclinical and Clinical Research through Rigorous Study Design and Transparent Reporting.
• Scientifically amazing proposals from highly respected, world-class investigators may still not be funded if:
  • The proposed research is too far from being relevant to a therapeutic intervention.
  • Our scientific advisors find the research redundant and similar prior work does not need validation.
  • The proposed research is likely to happen anyway, without our support.
  • The proposed research cannot realistically be achieved with the proposed budget.
  • Our recent grant commitments have unfortunately surpassed our fundraising.