In a study from the Translational Genomics Research Institute, scientists found that, if successful, a drug combo could lead to FDA approval of the first new drug treatment in more than a decade for glioblastoma, the most common and aggressive type of brain cancer.
The researchers expect the new treatment could help up to a third of all glioblastoma patients.
An estimated 18,000 Americans are expected to die this year from brain cancer, the nation’s seventh leading cause of cancer death.
In the study, the team suggest that a drug combination of MLN4924, also known as Pevonedistat, when given in combination with a second drug called Etoposide, could help glioblastoma patients whose cancer cells have lost PTEN, a tumor-suppressor gene.
The team says there hasn’t been a new drug approved for glioblastoma in 13 years.
Knowing the right patients to enroll in the clinical trial, through the study of molecular features of sensitive and resistant tumors, and only treating patients whose tumors have the signature of vulnerability, should set researchers on a path to a newly approved drug treatment.
The last drug to receive FDA approval for glioblastoma was Avastin in 2009. The average patient survival for glioblastoma remains stuck at about 15–18 months.
The identification of MLN4924/Pevonedistat grew out of a high-throughput screen nearly a decade ago of more than 100,000 different drugs.
Scientists tried to find an effective molecule that also was small enough to pass through the protective web of extra-small capillaries that surrounds the brain, forming what is known as the blood-brain barrier.
From those initial screenings, researchers identified four drugs—Arsenic Trioxide, Methoxyamine, Selinexor, and MLN4924/Pevonedistat.
The first three drugs have already undergone clinical trials for glioblastoma patients but were considered failures because they didn’t extend the survival of a sufficient number of patients.
However, they did work for a small percentage of patients with particular genomic profiles. Pevonedistat, which was originally developed for another disease, has never been tested in patients with glioblastoma.
The team is planning a series of clinical studies at sites across the nation that will test the safety and effectiveness of MLN4924/Pevonedistat with Etoposide.
If you care about brain health, please read studies about how the Mediterranean diet could protect your brain health, and the MIND diet could improve cognitive health in older people.
The study was conducted by Michael Berens et al and published in Neuro-Oncology.
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