A mother, shaped by tragedy, embarks on a mission to advance custom medicines

Jared Whitlock (STAT News)

Feb. 9, 2022

For weeks, Julia Vitarello avoided the room in her home. The fairy curtains she sewed. The sheets. The quiet. But one day, she placed her desk by the window. There, she began spending long hours on a single-minded mission: advancing custom medicines — and sparing others the pain her family has endured.

After receiving milasen, the frequency of Mila’s seizures slowed markedly — and significantly decreased in severity. She laughed again during bedtime stories. COURTESY JULIA VITARELLO

Four years ago, Vitarello’s daughter, Mila, was given a drug created just for her, the first time a medicine was specifically tailored to one patient’s genetic disease. The drug, called milasen, halted her rapidly progressing condition and later improved her quality of life. But the disease, already in an advanced stage, eventually resumed its assault. Mila died Feb. 11, 2021, at 10 years old.

Now, Vitarello is immersing herself in the business of personalized drugs — both to speed and scale their development — in the hopes of making more rare diseases treatable. It has meant mobilizing an academic consortium that creates custom medicines, and working to tackle policy issues such as drug reimbursement that are critical to the development of so-called “N of 1” therapies.

The need for these medicines, Vitarello and other advocates say, is huge — 3 in 10 childrenwith a rare disease won’t live to their fifth birthday. But so, too, are the barriers. Currently, the development of each individualized drug requires families and researchers to raise upward of $2 million. It also takes a small army of scientists and mountains of paperwork.

Vitarello imagines, one day, drugmakers routinely whipping up custom medicines for newborns with ultra-rare conditions. She strives toward this future from Mila’s old room.

“Creating Mila’s drug was like climbing Everest without oxygen or guides,” said Vitarello, 45. “But we need a clear path and to lower Everest.”

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Whitlock, J. (2023b, July 31). A mother, shaped by tragedy, embarks on a mission to advance custom medicines. STAT. https://www.statnews.com/2022/02/09/julia-vitarello-mila-rare-diseases-custom-drug-development/