Session 7: Advocacy as the Current
Overview
This year’s summit theme, “A River Runs Through Advocacy and Science,” found its clearest expression in this closing session. As Kendall Davis, MPH, framed it in her introduction, individual stories gather momentum when they are joined by science and by community, and together they create currents strong enough to change how research gets done for people living with Telomere Biology Disorders (TBDs) and other rare diseases.
Session 7 opened with Jenni and Cate Hargett, a mother and daughter whose years-long search for a diagnosis, and now for a bone marrow donor match, showed what it means to live inside the uncertainty that so many TBD families know well. From there, Dr. Matthew Hall of the National Institutes of Health described how existing, already-approved medicines can sometimes be redirected toward rare diseases like TBDs, which offers a faster path toward treatment than starting from scratch. Abbey Hauser then widened the lens further still by emphasizing how the health and medical care access of people in rural communities is shaped by policy decisions far outside the clinic, and how anyone, in any setting, can use their voice to help close that gap. Katie Stevens closed the session, and the summit, by looking at how far Team Telomere has come and what is now possible when patient advocates are recognized as full partners in research and policy, not just participants in it.
Together, these talks made the case that advocacy, community, and science are not separate efforts. They are currents that only grow stronger when they run together.
Highlights from Each Talk
Translational Science and Drug Repurposing, by Dr. Matthew Hall, National Institutes of Health (NIH)
Dr. Matthew Hall leads a translational science program at NIH that works to move promising science toward actual treatments. His team focuses on drug repurposing, which tests medicines already approved for one disease to see if they might help a different one, because new drugs take years to develop and most rare diseases lack a commercial path to one. His group screens a library of nearly 3,000 approved drugs against disease models. He shared a success story, a repurposed cancer drug that received fast-track approval for a specific brain tumor, alongside a more mixed case in Sanfilippo syndrome, and was clear that repurposing is useful but not a guaranteed path to treatment.
Y’all Means All: Rural Advocacy and Rare Disease, by Abbey Hauser
Abbey Hauser, who previously worked at Team Telomere and now works in health equity policy at the EveryLife Foundation for Rare Diseases, spoke about rural advocacy and rare disease and encouraged the audience to see poor health outcomes as rooted in systems and policy, rather than individual choice. She shared how Hennepin County Medical Center in Minneapolis avoided closure by showing that training physicians at an urban hospital also supports rural care, illustrating how urban and rural systems depend on each other. Her central message: people not directly affected by an inequity shouldn’t leave the work of fixing that inequity entirely to those who are.
Next Steps, by Katie Stevens, CEO, Team Telomere
Katie Stevens, CEO of Team Telomere, closed the session and the summit by reflecting on how far the organization has come. She announced that Team Telomere is part of a $161 million ARPA-H grant initiative, collaborating with St. Jude on a $26.5 million portion, a sign that patient advocacy groups are increasingly recognized and resourced as genuine research partners rather than expected to contribute for free. She also described securing IRB approval in just six business days for a blood draw study offered at the summit itself, which drew 33 participants against a budget for just 10 to 20.
What This Means for the Field
Taken together, these talks point to a field that is maturing in real time. A decade ago, families like the Hargetts often carried the full weight of both searching for a diagnosis and searching for a treatment largely on their own. Today, that search runs is supported by structured programs like Dr. Hall’s translational science pipeline at NIH, which is actively building tools to test whether existing medicines can help rare disease patients faster than starting from scratch, and by the work of patient advocacy groups like Team Telomere.
Abbey Hauser’s talk was a reminder that improving outcomes for TBD families is not only a laboratory question. It is also a policy question, in which clinicians, researchers, and advocates who are not themselves fighting against a given inequity have a responsibility to raise their voices in the rooms they already occupy.
Katie Stevens’s closing remarks show that Team Telomere itself is stepping into a new phase: Team Telomere is being invited into regulatory conversations, named as a formal partner on major federal research funding, and trusted enough by its own community to gather 33 blood samples in a single morning. As Katie put it, there is still a long road ahead. However, this summit made clear that the road forward runs through advocacy, community, and science moving together, not any one of them alone. The remarkable and successful framework that Team Telomere has developed has the potential to change the infrastructure of research and treatment for underserved patients with other rare diseases.
