Ampligen Showing Promise for Recurrent Ovarian Cancer in Phase 1/2 Trial, Phase 2 Study Now Recruiting

Hemispherx Biopharma‘s experimental therapy Ampligen (rintatolimod) can change the tumor microenvironment to increase the amount of infiltrating T-cells, an approach that is showing promise in a Phase 1/2 clinical trial for recurrent ovarian cancer, according to the company.

Funded in part by an Ovarian Cancer Specialized Program of Research Excellence from the National Institutes of Health, the study is being conducted at the University of Pittsburgh Medical Center.

Ampligen — approved in Argentina for severe cases of chronic fatigue syndrome — is an immune modulator that activates part of the immune system and is thought to assist in immune infiltration within tumors.

In cellular models, Ampligen promoted an accumulation of killer T-cells, which are immune cells that attack cancerous ones, without attracting regulatory T-cells (T-regs) – potent immunosuppressive cells that promote the progression of cancer through their ability to limit the anti-tumor immune response.

Interim results from the Phase 1/2 trial (NCT02432378) showed a similar behavior in ovarian cancer patients, indicating the investigational therapy has the potential to selectively reprogram the tumor microenvironment and induce immune infiltration without intensifying immunosuppressive responses.

“Ampligen has the potential to be clinically significant because a robust killer T-cell population in the tumor microenvironment without attracting Treg cells is important to help optimize checkpoint blockade induced tumor shrinkage,” Robert Edwards, the trial’s principal investigator and chair of gynecologic services at Magee-Women’s Hospital at the University of Pittsburgh School of Medicine, said in a press release.

These findings suggest that adding Ampligen to chemo-immunotherapy could give the immune system an additional advantage in the fight against ovarian cancer.

“Ovarian cancer is projected to kill 152,000 this year worldwide. Recurrent ovarian cancer is a dire unmet medical need. Working with the University of Pittsburgh, we are able to team up and focus world-class science on developing a meaningful combination therapy with the potential to save the lives of women who may be out of therapeutic options,” said Thomas K. Equels, CEO of Hemispherx.