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Celia Grace Hamlett was 4 when she was diagnosed with metachromatic leukodystrophy (MLD), a rare, genetic neurological disease that leads to severe disability and eventual death. The devastating news thrust her family into a quest for treatment, a journey that led them from their Alabama home to our team at M Health Fairview Masonic Children’s Hospital.
While the Hamletts grappled with this diagnosis, we lobbied the U.S. Food and Drug Administration (FDA) for special permission to perform the experimental procedure. After multiple hurdles with insurance coverage, Celia Grace became the first MLD patient in the U.S. to be treated with gene therapy. As the process unfolded, a local documentary film crew picked up cameras to capture this turning point in modern medicine.
After three years of filming and about a month after the FDA approved the same therapy that Celia Grace received in 2021, “Sequencing Hope” premiered at the Minneapolis-St. Paul International Film Festival. It documented Celia Grace’s path to treatment and her life in the years since. For Celia Grace and many others facing similar challenges, gene therapy represents more than just treatment — it offers the possibility of a future once deemed impossible.
As physicians and researchers with M Health Fairview and the University of Minnesota Medical School, we have devoted our careers to exploring treatments and cures for conditions such as inherited neurological disorders, enzyme deficiencies and sickle cell disease. Through rigorous clinical trials, we have witnessed firsthand the life-changing potential for gene therapies to cure people with conditions who previously lacked safe and effective treatment options. We are deeply concerned, however, that without comprehensive payment coverage from Medicaid and private insurers, these groundbreaking discoveries will remain out of reach for many.
The steep toll of gene therapies, ranging from $2 million to more than $4 million, poses significant challenges for health care delivery systems, which are not designed to account for the multimillion-dollar upfront costs of these therapies. Without comprehensive coverage from Medicaid and private insurers, equitable access to transformative gene therapies is becoming a greater obstacle than the development of the therapies themselves.
At the core of this issue lies the imperative of equitable access to these revolutionary treatments. Communities of color, disproportionately affected by diseases like sickle cell disease, may bear the brunt of inadequate access to care. As the medical community works to explore the promise of gene therapy, private and public payers and drug manufacturers must work with providers who are familiar with these conditions to develop a payment model that will ensure equitable access to this lifesaving care.
