In New Jersey, families like Tim and Kelsey Drury are taking on the daunting challenge of finding a cure for infantile neuroaxonal dystrophy (INAD), a rare and fatal neurological disorder affecting their son Jack and other children. With no existing treatments and limited interest from pharmaceutical companies, these families, through the INADcure Foundation, are driving groundbreaking gene therapy research. This blog explores their fight, the science behind INAD, and the hope for a clinical trial.
Human Toll
The diagnosis of INAD is devastating. Jack Drury, diagnosed at age 2, faces progressive loss of motor and cognitive skills, unable to crawl or roll over. His parents, Tim and Kelsey, endured six months of uncertainty before learning their son’s condition has no cure, with most children not surviving past age 10. Similarly, Leena Panwala’s daughter Ariya, now 11, requires round-the-clock care due to INAD’s debilitating effects. The emotional and physical toll on families is immense, compounded by the lack of treatment options.
Community Response
Through Jack’s Miracle Mission, the Drurys have raised over $545,000 to fund a gene therapy clinical trial, offering hope to families worldwide. The INADcure Foundation, based in Fairfield, NJ, supports 36 families globally, fostering a community of advocacy and resilience in the face of this rare disease.
Key Facts About INAD and Research Efforts
- What is INAD?: Infantile neuroaxonal dystrophy is a rare, autosomal recessive disorder caused by a PLA2G6 gene mutation, affecting approximately 150–250 children worldwide. Symptoms include wobbly eye movements, muscle weakness, and loss of motor skills, often leading to death by age 10.
- Research Progress: The INADcure Foundation, founded by Leena Panwala, is pioneering gene therapy research, partnering with institutions like Baylor College of Medicine and Oregon Health and Science University. A clinical trial is planned, pending FDA approval expected by September 2025, with Atlantic Health as the host.
- Funding Challenges: The foundation has raised $553,439 in 2023 but needs an additional $2 million for the trial. Big pharma’s lack of interest due to INAD’s rarity has forced families to fund research independently.
Scientific and Social Context
INAD’s rarity—estimated at 1 in 1,000,000—makes it a low priority for pharmaceutical companies, leaving families and nonprofits to fill the gap. The INADcure Foundation functions as both a fundraiser and a biotech entity, a rare model driven by necessity. Research includes a natural history study, a biobank for patient samples, and mouse model studies showing promise for gene therapy. These efforts aim to produce enough data for an Investigational New Drug application to the FDA, a critical step toward clinical trials.
Why This Matters
The fight for an INAD cure highlights the power of community-driven science. Success could not only save lives like Jack’s but also set a precedent for addressing other ultra-rare diseases, proving that even small groups can drive medical breakthroughs when traditional systems fall short.
What Lies Ahead
The INADcure Foundation is racing against time, with plans to submit an FDA application by September 2025 and begin manufacturing a clinical trial drug, a process that takes six months. If approved, the trial, led by Dr. Darius Adams at Atlantic Health, could start in 2026. However, raising the remaining $2 million remains critical. Community support, amplified by campaigns like Jack’s Miracle Mission, is vital to sustaining momentum.
Conclusion
New Jersey families are leading the charge for an INAD cure, transforming grief into action through Jack’s Miracle Mission and the INADcure Foundation. Their efforts to fund gene therapy offer hope for children facing this devastating disease. By supporting these initiatives, we can help turn the tide against INAD and inspire solutions for other rare disorders. Join the fight for a cure today.
