First patient receives potentially cancer-stopping pill in new clinical trial

LOS ANGELES — Researchers at City of Hope, one of the largest integrated cancer research and care centers, say the first patient has received a new cancer-stopping pill and is healthy and well.

The pill, AOH1996, was named after young cancer victim Anna Olivia Healey, who was born in 1996. For 20 years now, Linda Malkas, Ph.D., professor in City of Hope’s Department of Molecular Diagnostics & Experimental Therapeutics, has dedicated her research and development efforts to AOH1996.

In trials, the pill has been effective at targeting the proliferating cell nuclear antigen (PCNA), which has a crucial role in cell repair and replications. Using mutated cells as a target in this way has not only been shown to halt growth of cancerous human cells and inhibit spread, but its mechanism of action doesn’t confer any toxic effects to normal, healthy cells.

“Imagine cancer as the water filling up a bathtub. Left unchecked, the tumors or water will eventually overflow and damage other parts of your home. The treatment my team at City of Hope created is akin to a watchful homeowner who shuts the water off — stopping the spread of tumors to other parts of the metaphorical house — and then drains the tub, eliminating the cancer,” says Malkas, co-investigator in the trial and the M.T. & B.A. Ahmadinia Professor in Molecular Oncology, in a media release.

What types of cancer can the pill treat?

The first patient to receive the drug is part of City of Hope’s Phase 1 Clinical trial. The goal of this trial is to accurately determine the highest tolerable dose of the pill and to study the efficacy in preliminary stages. Patients eligible for participation are adults with solid tumors who haven’t responded well to standard cancer treatments. The research team instructs patients to take the pill twice per day.

“Since many patients’ cancers become resistant to our standard therapies, we need new therapeutics with new mechanisms of action — for example, non-cross resistant. AOH1996 is just that kind of new therapy,” explains Daniel Von Hoff, M.D., of the Molecular Medicine Division at the Translational Genomics Research Institute, part of City of Hope, and an advisor on the study.

So far, the drug has been promising against breast, prostate, ovarian, skin, and lung cancers. Malkas hopes to see the day when AOH1996 becomes an FDA-approved inhibitor drug that can be combined with other therapies like chemotherapy to boost their benefits while not increasing risk of side-effects.

Additionally, she mentions that there are other targeted therapies out there that have helped several cancer patients through hindering cell growth and metastasis. This only reinforces her confidence that AOH1996 can do the same. For now, Malkas hopes to have the Phase 1 trial last over the next two years and carefully evaluate new findings over this time.

The current findings of the clinical trial are available here.

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