Barb Bailus, PhD
Today we start a series of posts to introduce our newest FAST Scientific Advisory Board members; it is an honor to welcome such an amazing group of scientists! Several of these scientists have been members of the Angelman community for a long time, and others are fairly new but offer exciting insights and expertise that will help FAST in racing toward a cure! They have also been kind enough to answer some questions about themselves and their research. Read more about first new member below!
Dr. Timothy Yu quickly learned what it takes to move a novel drug from the lab-bench to bedside as swiftly as possible. At the 2018 FAST Gala he discussed his groundbreaking work on developing Milasen, a custom drug developed for a single patient with a unique form of a devastating neurodegenerative disorder called Batten disease. Batten is a devastating and rapidly progressive disease. Within a year of being diagnosed, young Mila had lost her vision, most of her ambulatory ability, and was having consistent and severe seizures. The family contacted Dr. Yu shortly after Mila’s diagnosis and in the following year his lab found the mutation in her CLN7 gene, developed a “drug” to treat it, called an antisense oligonucleotide (ASO), had it manufactured to a clinical grade material, got FDA approval to start a single patient clinical trial, and administered it. Dr. Yu is a bicoastal scientist, having done his PhD and MD work at University of California San Francisco and then establishing his laboratory at Harvard, where he focuses on neurodevelopmental disorders. It is no surprise that he cites the 2018 FAST Gala as one of his favorite AS memories, his enthusiasm at the event was contagious, where he met with both families and researchers to discuss the promising path forward for AS treatments. In the future, Dr. Yu envisions the opening of interventional genomic clinics that can diagnose and synthesize tailor made therapies for patients with neurological disorders. When not working in the lab on developing life changing therapies, he enjoys photography, music, biking and playing with his three young daughters.