Novel oral drug therapy combination shows promise for advanced melanoma patients

Medical Xpress / Huntsman Cancer Institute / Cell Reports Medicine ^ | March 21, 2025 | Jared Almazan et al

[ConservativeMind]

A research team is testing a new combination drug therapy that could both treat and prevent melanoma metastasis to the brain.

Holmen first examined what causes melanoma cells to spread to the brain and identified focal adhesion kinase (FAK) as a potential target for new therapies. FAK is an enzyme that regulates cell growth, and, they found, is a major contributor to melanoma metastasis.

"The window of time to treat a patient with brain metastasis is shortened because the average survival from time of diagnosis of brain metastasis is only about a year—even while using these other therapies."

Holmen and her research team found that inhibiting the enzyme FAK in combination with an inhibitor of RAF and MEK—which targets another cellular pathway that regulates cancer cell growth—was effective in prolonging survival rates in preclinical mouse models. They specifically studied a subtype of melanoma triggered by a mutation of BRAF, a gene that helps regulate cell division.

A mutation of this gene has been identified with several types of cancer, including an estimated 50% of patients with metastatic melanoma.

"This combination drug therapy also stopped the development of brain metastasis, and that's where this research is very exciting," says Holmen. "Not only did it treat the tumor once it had spread and was growing in the brain, it also prevented the cells from getting there in the first place."

The oral treatment combines two drugs: defactinib, which blocks a protein called FAK, and avutometinib, which blocks proteins called RAF and MEK.

The study, led by Howard Colman, MD, Ph.D., professor in the Department of Neurosurgery at the U, has now moved into clinical trials for patients at Huntsman Cancer Institute and Holden Comprehensive Cancer Center at the University of Iowa.

The study is open to patients with melanoma with brain metastases.

[ConservativeMind]

The trial is open and is even taking people with brain metastasis.

The two drugs are oral and not available outside of the trial. The trial is out of the University of Iowa.

The drugs appear to work together to prevent and shrink melanoma metastasis to the brain.

[ConservativeMind]

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I was hoping to hear it was Ivermectin being used.