From Mila to Millions: Scaling N of 1 Therapies

When Julia Vitarello learned that her daughter Mila had the CLN7 form of the deadly, neurodegenerative condition Batten disease, it set her off on a search for a treatment that resulted in the development of a customized antisense oligonucleotide. In the wake of Mila’s case, a movement has emerged to develop so-called N-of-1 therapies for people with ultra-rare conditions. Vitarello, along with Boston Children's Hospital researcher Timothy Yu, who developed the ASO to treat Mila, has co-founded the N=1 Collaborative, an international group seeking to enable the development of N-of-1 therapies to treat the thousands of patients in need. We spoke to Vitarello, CEO of Mila’s Miracle Foundation and co-founder of the N=1 Collaborative, about the new organization, the issues it is trying to address, and what it would take to take enable the development of individualized therapies broadly for patients with ultra-rare disease.