Mass General team develops a gene therapy for recurrent ovarian cancer

MGH's David Pepin

A research team at Massachusetts General developed a gene therapy to deliver a protein that demonstrated signs of successfully reining in recurrent ovarian cancer in a mouse model for the disease.

The team explains that in males, Mullerian Inhibiting Substance--which is secreted in embryonic tissue--prevents the development of female reproductive organs. Building on earlier work that spotlighted the potential role of MIS in curtailing the growth of ovarian cancer, the investigators say they gained new insights into how MIS can target cancer stem cells that linger following chemotherapy.

The investigators already had studied the potential of this approach, but were challenged by their inability to get sufficient quantities of the protein where it was needed. So they came up with a purified form of MIS and used an AAV vector--common in gene therapies--to deliver it straight to the peritoneal cavity where ovarian cancer recurrence often begins.

Not only did they get the desired levels of MIS where it was needed, the team also concluded that delivering the gene therapy weeks in advance of tumor implantation in mice spurred a measurable response.

"Our findings are important because there are currently no therapeutic options for recurrent, chemoresistant ovarian cancer," says David Pepin of the MGH Pediatric Surgical Research Laboratories and lead author of the report. "This is also a proof of concept that gene therapies with the AAV9 vector can be used to deliver biologics for the treatment of ovarian cancer and represents the first time this approach has been tested in this type of ovarian cancer model."

One of the next steps will be to develop biomarkers to identify which patients are most likely to respond to this therapeutic approach. 

- here's the release