2024 has been a hopeful year for FTD Research, and it’s also been an exciting year for the AFTD research team. It isn’t easy to summarize all that’s been accomplished in a year, but we’ve provided some interesting highlights!

This year, the AFTD team welcomed two new staff members, Dr. Nicole Bjorklund as Director of Research and Grants and Dr. Kate Still as the first AFTD Research Outreach Manager. After nearly a decade and a tremendous amount of work advancing innovation in FTD science, Dr. Debra Niehoff retired as AFTD’s Director of Research and Grants.

AFTD’s research team and the rest of AFTD are guided by our current Strategic Plan. The funding that we provide is central, yet only one component of our strategy. We want to attract and retain talent and funding to the FTD field, empower an informed and active community of research participants and partners, make it easier for innovators and companies to apply their resources to the needs of FTD, create a community of FTD researchers, and develop tools to enable FTD research.

The first research strategic objective is to foster a diverse, global community of FTD-focused scientific investigators.   AFTD has several initiatives focused on galvanizing the core community of researchers, advocates, and others committed to scientific progress for FTD. In addition to creating opportunities for FTD research mentorship and connection between FTD researchers, AFTD sponsored, presented at, and hosted meetings as part of the 2024 International Conference for FTD. One such meeting was convening seasoned FTD researchers with the AFTD Holloway Scholars, a fellowship made possible by the support of the Holloway Family Fund. In 2024, AFTD also sponsored and attended conferences for organizations that work in FTD and related disorders, including the End the Legacy’s Genetic ALS and FTD Community Summit and the Cure PSP International Research Symposium.

The second objective is to expand, inform, and empower a research-ready community. The AFTD research team works to ensure people can make informed decisions regarding the science of FTD. In 2024, these initiatives included webinars presented in collaboration with the FTD Disorders Registry. Scientific resources also included scientific sessions at the May 2024 AFTD Annual Education Conference. In partnership with the University of Pennsylvania FTD Center, AFTD collaborated on the first-ever FTD Clinical Research Learning Institute, a one-day virtual workshop designed to empower people impacted by FTD with knowledge about clinical research. The research team also developed several resources related to FTD genetics, including a new resource on Gene Therapy and FTD.

The AFTD research team works to ensure the values, priorities, and concerns of people impacted by FTD are understood by research stakeholders. In 2024, AFTD hosted the first Industry Advisory Panel, where community members shared their experiences with companies working on FTD clinical trials. In 2024, AFTD also brought personal experiences with FTD to professional audiences through participation in working groups, presentations at scientific conferences, and a publication on the need for more diversity in FTD research.

The third strategic objective is to develop or strengthen tools and resources needed for FTD research to succeed. New projects launched in January with support from the new AFTD Digital Assessment Tools for FTD and ALS Grant awards. This unique program advances the development of digital tools with broad applicability across the FTD-ALS spectrum. In 2024, the FTD Disorders Registry, co-founded by AFTD and the Bluefield Project to Cure Frontotemporal Dementia, announced the official launch of an enhanced platform. This comprehensive database will facilitate the global recruitment required for rare disease clinical trials, as well as serve as a portal for a global community of researchers.

A major challenge in the development of treatments is the expertise, consensus, and validation of tools to run the clinical trials to test if a treatment is safe and effective.  In 2024, AFTD hosted the second annual FTD Research Roundtable to help the field overcome shared challenges in drug development and clinical trials. Guided by the shared priorities of 15 biopharmaceutical company members, regulatory experts at the FDA and EMA, academic research leaders, non-profits, advocates, and people directly impacted by FTD, the 2024 Roundtable focused on the availability of validated biomarkers for clinical trials, as well as strategies to identify participants and to quantify whether a treatment has had a meaningful change. This conference built upon the insights on FTD Biomarkers gained through the November 2023 Holloway Summit.

AFTD also hosted a workshop on how clinical trial might be designed to test a preventative treatment for FTD and/or ALS caused by the C9orf72 genetic variant, with collaborative leadership by FTD and ALS experts, support from the ALS Association, and participation by both clinical research fields, regulatory experts from the FDA, and people with lived experience of both C9-FTD and C9-ALS.

The final research strategic objective is to accelerate and broaden research approaches to address unmet needs in FTD research. The AFTD research team spent time in 2024 working on projects to align FTD experts on key challenges to diagnosis and treatment. Across the AFTD grant portfolio, 31 research projects are ongoing and are being actively stewarded. These studies span from basic research to clinical trials to improving the quality of life for those living with FTD. Numerous research grant cycles are underway, and we expect to award several promising new studies in the coming year. As FTD lies at the intersection of ALS, Alzheimer’s Disease, and Parkinson’s Disease, AFTD works to influence priorities in neurodegenerative research, including collaborating with other advocacy groups, attending and presenting at conferences for related disorders, and speaking to government and funding bodies about FTD needs. For example, Dr. Penny Dacks was invited on behalf of AFTD as one of four experts to provide input on non-Alzheimer’s forms of dementia to a congressionally mandated committee review of dementia research priorities. AFTD has also partnered with NIH, biopharmaceutical companies, and other non-profit organizations to support The Accelerating Medicines Partnership® in Amyotrophic Lateral Sclerosis (AMP® ALS) Program to make sure that the scientific solutions for ALS are developed with understanding of the needs of people jointly at-risk and/or experiencing FTD.

Looking ahead to 2025, as AFTD works on a new Strategic Plan that will guide organizational priorities for the future, we expect to deepen our focus on the factors slowing the development of the solutions needed by families. As AFTD looks to the future, we will continue to work attracting brilliant minds, companies, and funders to focus on this space to ensure people impacted by FTD are receiving timely and accurate diagnoses and that the infrastructure exists to support FTD clinical trials, the key to unlocking effective treatments.