Mila’s Story and the Future of ‘N-of-One’ Therapies: A Fireside Chat About Lessons Learned from the Development of a Therapy for a Single Patient

Timothy Yu, M.D., Ph.D., co-authored a report in The New England Journal of Medicine for October 24, 2019, that extended the scientific and regulatory boundaries of personalized medicine. The article reviewed how Dr. Yu, a physician at Boston Children’s Hospital, had developed and delivered an “n-of-one” therapy intended to treat only one person. The patient, the late Mila Makovec, was an eight-year-old girl with a genetic form of a neurological disease so rare that it had never been documented before.

Officials at the U.S. Food and Drug Administration gave Dr. Yu permission to use milasen, a custom therapy designed to block the effects of an extraneous portion of DNA whose presence was inhibiting the function of a key gene, to treat Mila’s unusual form of Batten disease, a fatal condition with no cure. To finance the work, Mila’s mother, Julia Vitarello, raised $3 million by creating a nonprofit research foundation and appealing to the users of the GoFundMe crowdfunding platform.

Dr. Yu and Ms. Vitarello will join moderator Walter Kowtoniuk, Ph.D., a venture capitalist focused on translating scientific breakthroughs into improved patient care, to envision a world in which physicians routinely develop n-of-one therapies. With attention to regulatory questions and strategies for ensuring the financial sustainability of the n-of-one treatment model, the conversation will explore how a race to save one girl’s life has turned into a movement to push the boundaries of ultra-personalized medicine for the benefit of tens of thousands of children suffering from ultra-rare genetic diseases in the United States and around the world.

  • Moderator

    Walter Kowtoniuk, Ph.D.
    Venture Partner, Third Rock Ventures

  • Panelist

    Julia Vitarello
    Founder, CEO, Mila’s Miracle Foundation; Co-Founder, N=1 Collaborative , Mila's mom

  • Panelist

    Timothy Yu, M.D., Ph.D.
    Co-Founder, N=1 Collaborative; Staff Physician, Investigator; Associate Professor, Harvard Medical School, Boston Children’s Hospital