Novel Drug Improves Survival in Advanced Brain Cancer

A new novel drug has been found to improve survival and reduce cognitive decline in patients with advanced brain cancer, according to research from Duke University School of Medicine.

In specific, the novel drug BMX-001 was created by Ines Batinic-Haberle, a professor at the university, after more than 30 years of research and creating hundreds of compounds to perfect what would become BMX-001.

According to a news release from Duke University School of Medicine, BMX-001 is a drug that mimics the action of superoxide dismutase, a type of enzyme the body naturally makes.

The treatment of BMX-001 was evaluated in a multi-institutional trial in patients who were newly diagnosed with high-grade glioma, a type of brain cancer, which was led by Dr. Katherine B. Peters, a professor of neurosurgery and neurology at the Preston Robert Tisch Brain Tumor Center at Duke University.

The phase 2 trial included 160 patients, according to Clinical Trials.gov, who were randomly assigned into two treatment arms. Eighty patients in one treatment arm received radiation therapy, Temodar (temozolomide) and BMX-001 and 80 patients received radiation therapy plus temozolomide in the second treatment arm. Temodar is a type of chemotherapy drug.

Patients in the trial had a baseline MRI (imaging scan), completed a cognitive test and answered a questionnaire about health-related quality of life, Clinical Trials.gov noted. The trial began in late 2018 and is expected to be completed by July 2024.

Peters and other researchers established that patients in the BMX-001 regimen treatment arm had reduced cognitive decline that often occurs after receiving radiation. In particular, they determined that patients who received the BMX-001 regimen performed better after taking cognitive tests, compared with patients who received radiation and chemotherapy.

Peters noted in the release that many patients fear the cognitive effects they have because of brain tumors. Some of these effects could include difficulties with language or having difficulties while focusing or multitasking, she said.

“Our patients have to contend with both a cancer diagnosis and a neurologic diagnosis all in one," Peters said in the news release.

Based on past findings, researchers also established that radiation therapy may worsen challenges regarding cognitive abilities. They found that 50% to 90% of patients who receive radiation to the brain will experience the side effect of cognitive dysfunction, the release reported.

"This really is an unmet need for our patients,” Peters said. “We are getting better at treating our patients, and now we need to maintain their quality of life as best we can.”

In the 1990s, Batinic-Haberle began designing a synthetic version of the enzyme superoxide dismutase. However, her goal was to design a small molecule that was “very stable, water-soluble and positively charged, therefore able to cross cellular membranes and reach the brain,” she said in the news release, unlike superoxide dismutase.

While BMX-001 has already shown promise for patients with newly diagnosed high-grade glioma, the novel drug will soon be tested in clinical trials for other cancer types, including head and neck cancer, anal and rectal cancers and multiple brain metastases. The drug is also being tested in ovarian cancer, of which Batinic-Haberle said early data show that BMX-001 reduces peripheral neuropathy (weakness or numbness from nerve damage in the hands and feet) and suppresses tumor growth.

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