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.