WORCESTER – Founded 150 years ago on the simple idea of taking care of kids, Boston Children’s Hospital still revolves around that core principle, but President and CEO Sandra Fenwick said there’s much, much more to the story.
Before her keynote address to the 558th meeting of the Worcester Economic Club at the College of the Holy Cross Wednesday night, Ms. Fenwick detailed the depth and breadth of research started in labs and clinical trials at Boston Children’s Hospital.
She described the process that teams at Boston Children’s went through over two decades of basic science research and genome study to combat sickle cell anemia; a breakthrough that paved the way for the discovery of a “fetal hemoglobin switch,” and subsequent trials using infusions of gene-modified cells that resulted in the first patient becoming disease-free. The hospital recently infused the second and third patients, and plans to infuse the fourth patient later this month, Ms. Fenwick said.
Another patient Ms. Fenwick described couldn’t talk or walk without assistance and suffered up to 30 seizures a day; she had recently gone blind. In just 10 months, a Boston Children’s doctor and his team identified the girl’s unique genetic mutation, designed a drug to fix it, and launched an FDA-approved clinical trial. After her treatment, her condition stabilized; the research, Ms. Fenwick said, could be scaled to address genetic mutations across many diseases.