Saving Mila: How a tailor-made therapy, developed in a flash, may have halted a young girl’s rare disease
As 6-year-old Mila’s favorite movie, “Frozen,” played, her father held a newspaper up in front of her face. She was rapt — her favorite song was on — but she didn’t even flinch.
That’s the moment Mila’s parents realized that she had gone completely blind. And they finally knew, now, after months of concern and sinking suspicion, that something was dangerously wrong with their bright, engaging daughter.
Mila has Batten disease, an ultra-rare, neurodegenerative genetic disorder. For every child diagnosed, the prognosis has been tragic: Batten is always fatal.
But Mila’s parents, Julia Vitarello and Alek Makovec, now have a glimmer of real hope. Thanks to a remarkable effort on the part of scientists, regulators, and Mila’s family, an experimental therapy — developed in record time — could very well be halting her disease in its tracks.
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Drug development typically takes several years before a new therapy can even make it into clinical trials. Mila, however, made it from diagnosis to bespoke therapy in just over a year. Her case serves as a proof-of-concept in efforts to rapidly develop and deliver precision medicine — as tailored to a single patient. The drug was
