Video by: Charles River
In 2018, a seven-year-old girl named Mila with a fatal neurogenetic disease became the first person in the world to receive a drug tailored to a single patient. The drug was named milasen. In a remarkable collaboration between scientists, physicians, drug developers, foundations and regulators, this novel medicine was created, tested and delivered in just one year from her diagnosis. While Mila would eventually succumb to her condition, milasen suppressed her seizures and improved her quality of life. The 2019 publication of this effort in the New England Journal of Medicine drew international attention as the first example of individualized genomic medicine, setting a precedent and giving new hope to patients with conditions considered too rare to support treatments.
Further customized drugs for other rare genetic diseases have followed. In 2021, with growing interest in pursuing this new treatment approach, the US Food and Drug Administration issued its first ever set of guidances to govern the development of individualized drugs like milasen.
In response to the unique opportunity and challenges, the N=1 Collaborative was launched in 2021 as the first international hub for individualized medicines for rare disease. The organizing committee is comprised of scientific leaders from around the world, including Dr. Tim Yu, the designer of milasen, and Julia Vitarello, Mila’s mother. With founding support from the Oligonucleotide Therapeutics Society N-of-1+ Taskforce and the Chan Zuckerberg Initiative, the N=1 Collaborative and its hundreds of participants are dedicated to guiding this rapidly developing new branch of medicine.