Today, the Food and Drug Administration approved a gene therapy known as CAR T-cell therapy that genetically modifies a patient’s own cells to help them combat pediatric acute lymphoblastic leukemia (ALL), the most common childhood cancer. It is the first gene therapy to be approved by the FDA.
“This represents the progression of the field of gene therapy, which has been developing over the last 30 years,” says gene therapy pioneer David A. Williams, MD, who is chief scientific officer of Boston Children’s Hospital and president of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. “It’s a realization of what we envisioned to be molecular medicine when this research started. The vision — that we could alter cells in a way to cure disease — is now coming true.” …
One of the hot trends in drug discovery could be called drug re-discovery: finding new uses for drugs that have already received FDA approval for a different indication.
It’s an approach that allows researchers and clinicians to rapidly test potential treatments for rare or difficult-to-treat conditions. Because the drug’s safety profile is already known, much of the preclinical and early clinical work that goes into developing a drug can be bypassed.