National Cancer Institute Announces Genetic Sequencing Trial

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NCI launches trial to assess the utility of genetic sequencing to improve patient outcomes.

The National Institutes of Health’s (NIH) National Cancer Institute (NCI) announced the launch of the Molecular Profiling based Assignment of Cancer Therapeutics (M-PACT). M-PACT is a pilot trial designed to assess if assigning treatment based on genetic screening can improve the rate and duration of response in patients with advanced solid tumors. Researchers hope the trial will identify patient sub-groups that are likely to benefit from certain treatments and result in new treatments being developed quickly for some cancers. The results of the trial could lead to smaller, more definitive clinical trials, which would be helpful to clinicians and patients in terms of cost and time.

“Patients will have their tumors genetically screened and if a pre-defined mutation is found, they will receive treatment with targeted agents,” said Shivaani Kummar, MD, head of NCI’s Developmental Therapeutics Clinic and the principal investigator of the trial, in a press release. “What we don’t know, however, is whether using this approach to assign targeted treatments is really effective at providing clinical benefit to patients, as most tumors have multiple mutations and it’s not always clear which mutation to target and which agent is most likely to provide maximal benefit. This study hopes to address some of these questions in the context of a prospective, randomized trial.”

According to NIH, a mutated gene can lead to the activation of multiple pathways, resulting in disease progression and potentially requiring multiple interventions. NCI’s M-PACT trial will determine if “people with specific mutations that have been demonstrated in laboratory systems to affect drug effectiveness will benefit from a specifically chosen targeted intervention and if these interventions lead to better outcomes.”

The study will include 180 patients with solid tumors that are resistant to standard therapy based on their genetic profile. Tumors will be genetically sequenced to look for a total of 391 different mutations in 20 genes that are known to affect the utility of targeted therapies. Patients with mutations of interest will be enrolled in the trial and randomly assigned to one of two treatment arms to receive one of the four treatment regimens that are part of this study. It is expected that patients with specific tumor types will have received certain therapies prior to being enrolled in NCI’s M-PACT. Patients with all types of solid tumors will be considered for trial eligibility.


“We believe that this study will aid patients in the trial that will be conducted initially at the NCI, and subsequently expanded to clinical trials sites participating in the NCI-supported Early Therapeutics Clinical Trials Network,” said James Doroshow, MD, NCI deputy director for clinical and translational research, in a press release. “We also believe that M-PACT can be a model for trials nationwide, particularly those that employ genetically-driven treatment selection approaches in their design.”