Faces of Gene Therapy: Meet Lesa Brackbill
- msevcik1
- May 29
- 2 min read
Lesa Brackbill is a passionate mother, advocate, author, and member of IGT’s Patient Advocacy Advisory Council. After her daughter Victoria was diagnosed with Krabbe Disease in 2015, Lesa turned heartbreak into purpose. Her leadership has helped transform newborn screening policies across Pennsylvania and the nation, and she believes that knowing the root cause of diseases at birth can lead to better outcomes for families that stand to benefit from innovative treatments like gene therapy.
Lesa recently reflected on her advocacy journey and shared her perspective on why gene therapy is vital for families like hers:
Q: How did you come into the advocacy space?
Lesa: Ten years ago, my daughter, Tori, was diagnosed symptomatically with Krabbe disease. Being told on diagnosis day that there was nothing we could do, but that had we caught this at birth we could have treated it, was the most traumatic news we could have received. My advocacy launched that day. I began with changing the Newborn Screening system in PA along with advocating for the addition of Krabbe disease to state panels elsewhere.

Q: Why is gene therapy so important for your community specifically?
Lesa: Right now, our only available treatment is a bone marrow transplant, and it isn't a cure. It doesn't stop the peripheral nervous system damage. We now have zero clinical trials, but the one that recently ended because of a lack of funding was showing SUCH promise! The data was so good.
Q: What are some of the major barriers to access you see and how can we address them?
Lesa: Payers and lack of newborn screenings. That's why I am passionate about protecting and expanding the newborn screening program. And payment barriers are hurting patients - what good is an approved treatment if no one can access it? I recently wrote an op-ed about payers making decisions that rob parents of life-saving opportunities. You can read the op-ed here.
Q: Why do you believe IGT’s mission of modernizing the policy space for gene therapies is so important?
Lesa: There are so many gene therapies in development, so many lives to potentially save. The quicker we can solve these problems and reduce barriers, the better.

Lesa’s advocacy journey embodies the strength and determination of our patient community. Her insights serve as a reminder that policy is personal - and that by modernizing our policies and regulations to keep pace with innovation, we can ensure a future where all patients have the opportunity for better, life-changing health outcomes.
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