Studying patient’s genome helped to suggest personalised treatment options for children with cancer. 

Precision medicine could be the best treatment option for children with rare or aggressive forms of cancer, according to a new study. Using genetic information researchers from the University of Michigan Comprehensive Cancer Center and C.S. Mott Children’s Hospital were able to suggest personalised options for half of patients. 

“We found that for some children with rare, difficult-to-treat and aggressive cancers, this technology can dramatically change the course of their treatment,” says lead author Rajen Mody, M.D., M.S., pediatric oncologist at U-M’s C.S. Mott Children’s Hospital.

“We have made significant strides in cancer treatment but for some kids, especially those with metastatic or relapsed disease, even the most advanced, proven therapies have not been able to improve their outcome. Our approach in precision oncology showed its greatest promise in these difficult to treat patients – 80 percent of our study patients had relapsed or refractory disease, and those are the ones who benefited most from our study.”

Of the 46% of patients had actionable findings, such as a specific genetic anomaly that can be targeted with an approved or experimental drug, or in some cases a change in diagnosis. As a result, 25% of patients went on to recieve a novel therapy, which in 10% of cases lead to a partial or complete cancer remission that lasted six months or longer. 

“We were excited to see an actionable finding in such a substantial percentage of patients, and we think it could potentially be higher over time. These are patients who had exhausted all proven therapeutic options or who had an extremely rare diagnosis. If we can find a clinically actionable event and have a chance to act upon it, we show in this study that it can have a big impact on that patient,” says senior study author Arul Chinnaiyan, M.D., Ph.D., director of the Michigan Center for Translational Pathology.

In time the researchers want to offer sequencing to as many pediatric cancer patients at Mott as they can, but there are still a few barriers to overcome. In particular, the cost of sequencing (around $6000 per patient) and the turnaround time for a sequence. During this study it took seven to eight weeks to report the results to the patients and their families. 

“These are early days and the full promise of precision medicine is yet to be fully realized,” cautions Mody. “We need better targeted therapies designed for children, and turnaround time for sequencing needs to be less than two weeks for it to be a regular part of a patient’s treatment plan.”