STAT+: The quest to save Grace — and clear the way for rare disease patients everywhere
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- Matt Wilsey has spent a decade and $70 million trying to cure his 15-year-old daughter Grace's NGLY1 deficiency, recruiting Nobel Prize-winning advisers and assembling an A-team of investors and donors.
- Wilsey's company has treated 10 patients, including Grace, in a clinical trial but is out of money and still lacks what the FDA says it needs for approval.
- Grace received the gene therapy three weeks before the story was written, but the drug landed her back in the hospital "feebler than she had ever been," according to STAT.
- Wilsey is pressing the FDA anyway, framing his push as "a pivotal test case" for regulators and for anyone developing treatments for patients with deadly rare diseases.
- Wilsey told his staff his trial could be "an ice breaker" for a "decimated" rare-disease field and said advocates hope his game plan serves as a guide to curing hundreds of other rare diseases.
Why it matters: With 10 patients treated, no cash left, and data short of FDA requirements, Wilsey's confrontation with the agency becomes the focal point for an entire field — how the FDA handles ultra-rare-disease approvals will shape whether other small developers and desperate families can get therapies across the line, with Grace's life and a precedent for hundreds of similar programs hanging in the balance.
