A team of researchers in Philadelphia, including former Damon Runyon Scholar Mark A. Lemmon, PhD, announced that the recently-approved lung cancer drug crizotinib (Xalkori) could potentially be used to treat neuroblastoma patients. The most common cancer among infants, neuroblastoma is a disease of the developing nerve cells.
In 2008, Mark’s colleague Dr. Yaël Mossé found that mutations in the ALK gene can cause a form of neuroblastoma. They began experimenting with crizotinib, which blocks ALK activity, and found that tumor response varied according to the type of mutation. “We predicted that…we’d see differences in how the drug binds to the ALK protein,” Yaël said. “That was completely wrong.” They instead found that certain tumors simply required a much higher dose of the drug.
Treatment options are currently limited to surgery and chemotherapy. Unlike chemo, crizotinib is a targeted therapy that would inflict less damage on healthy, non-cancerous cells. According to Mark, “If we can better individualize treatment to a child’s genetic profile by understanding the effects of mutations on protein structure and function, we may achieve better results for patients.”
> Read more about the discovery at CHOP.edu
Quotes adapted from Children’s Hospital of Philadelphia article
