Two children with an aggressive form of childhood leukemia showed no evidence of the disease after being treated with an experimental cell therapy, a research team from The Children’s Hospital of Philadelphia and the University of Pennsylvania confirmed on Monday, March 25, 2013.
The case study of 7-year-old Emily Whitehead comes as encouraging news to those who suffer from a stubborn form of the most common childhood cancer, acute lymphoblastic leukemia (ALL), even suggesting the novel treatment may someday replace the need for chemotherapy and bone marrow transplants.
Emily was featured in news stories in December, 2012. Experimental therapy had led to her dramatic recovery after she relapsed following conventional treatment.
Emily remains healthy and cancer-free nearly a year after receiving bioengineered T cells.
The specialized T cells — white blood cells that mature in the thymus — reprogrammed Emily’s immune cells to rapidly multiply and destroy leukemia cells.
The case report was published online ahead of print in The New England Journal of Medicine. It will appear in the April 18, 2013 print issue.
However the researchers said there’s a need for more research to improve on the therapies being tested.
Sadly, another patient, a 10-year-old girl who also had a complete response to the same treatment, suffered a fatal relapse two months later when other leukemia cells appeared. Those cells didn’t harbor the specific cell receptor targeted by the experimental therapy, according to the case report.
"This study describes how these cells have a potent anticancer effect in children," said co-first author Stephan A. Grupp, M.D., Ph.D., of The Children's Hospital of Philadelphia, where both patients were treated in this clinical trial.
"However, we also learned that in some patients with ALL, we will need to further modify the treatment to target other molecules on the surface of leukemia cells."
After decades of research, oncologists can currently cure 85 percent of children with ALL. Both children in the current study had a high-risk type of ALL that stubbornly resists conventional treatments.