NCI Pilot Trial to Assess Value of Genetic Sequencing for Improving Patient Outcomes


The National Cancer Institute (NCI) has launched a clinical trial to determine whether selecting therapies based on genetic mutations can improve outcomes in patients with metastatic solid tumors. Molecular Profiling based Assignment of Cancer Therapeutics (M-PACT) is one of the first randomized trials to assess if using genetic mutation as the basis for treatment selection can improve the rate and duration of response in patients with advanced-stage solid tumors.

A second goal is to see if the use of genetic sequencing can identify a subpopulation of patients who could benefit from specific targeted treatment, which would help to expedite drug development for these patients based on their genetic profile, and could lead to smaller clinical trials and reduce cost and time of drug development.

“Patients will have their tumors genetically screened, and if a predefined mutation is found, they will receive treatment with targeted agents,” said Shivaani Kummar, MD, head of NCI’s Developmental Therapeutics Clinic and the trial’s principal investigator. “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.”

The M-PACT trial is designed to determine whether patients with mutations that have been shown in the laboratory to affect drug effectiveness will benefit from a specific targeted therapy, and if this approach results in better outcomes.

The NCI is now screening hundreds of people to select 180 patients with advanced refractory solid tumors based on their genetic profile. u

National Cancer Institute Press Release; January 30, 2014.

FDA Approvals, News & Updates, Web Exclusives - February 5, 2020

Regenerative Medicine Advanced Therapy Designation Granted to ADP-A2M4 for Synovial Sarcoma

On December 3, 2019, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to the biopharmaceutical company Adaptimmune Therapeutics for ADP-A2M4 for the treatment of synovial sarcoma. Earlier this year, the FDA granted Orphan Drug designation to the agent for the treatment of soft tissue sarcomas.

FDA Approvals, News & Updates, Web Exclusives - February 5, 2020

Tazverik Receives FDA Approval as First Treatment Specifically for Metastatic or Locally Advanced Epithelioid Sarcoma

On January 23, 2020, the FDA granted accelerated approval to tazemetostat (Tazverik; Epizyme), a methyltransferase inhibitor, for the treatment of metastatic or locally advanced epithelioid sarcoma in adults and pediatric patients aged ≥16 years who are not eligible for complete resection.