In the fight against cancer, researchers constantly strive to discover new treatments.
A recent study led by the Translational Genomics Research Institute (TGen), part of City of Hope, sparks hope for people battling glioblastoma, the deadliest form of brain cancer.
This study could pave the way for the approval of the first new glioblastoma drug in over a decade.
Glioblastoma: A Deadly Foe
Glioblastoma is a fast-growing type of brain cancer. Despite advances in medical science, the average survival for glioblastoma patients is around 15 to 18 months.
It is expected to cause 18,000 deaths this year, making it the seventh leading cause of cancer death in America. The last glioblastoma drug, Avastin, was approved back in 2009.
A New Approach
Researchers believe a new treatment could potentially help up to a third of all glioblastoma patients.
They aim to use precision medicine, which involves choosing participants with a specific genomic “signature of vulnerability”.
The Drug Duo
The researchers’ new approach involves a combination of two drugs – MLN4924 (also known as Pevonedistat) and Etoposide.
Preclinical study results, published in Neuro-Oncology, suggest this combination could benefit glioblastoma patients whose cancer cells lack PTEN, a tumor-suppressing gene.
Genomic sequencing (a method for analyzing a sample of DNA) revealed that when PTEN is missing, there is an increase in the expression of another gene, TOP2A.
This gene is thought to resist the effectiveness of MLN4924. The second drug, Etoposide, is believed to block TOP2A, thus weakening the cancer cells enough for MLN4924 to kill them.
A Long Journey to Discovery
Finding this drug combo wasn’t quick. Almost ten years ago, researchers screened over 100,000 different drugs.
They aimed to find an effective molecule small enough to cross the blood-brain barrier, a network of small blood vessels protecting the brain.
After extensive screening, four drugs were identified: Arsenic Trioxide, Methoxyamine, Selinexor, and MLN4924/Pevonedistat.
The first three drugs underwent clinical trials for glioblastoma but didn’t extend survival for enough patients.
However, Pevonedistat, originally developed for another disease, has not been tested in glioblastoma patients yet.
The Next Steps
The TGen team, led by Dr. Michael Berens, is planning a series of nationwide clinical trials. They aim to test the safety and effectiveness of the drug combo MLN4924/Pevonedistat with Etoposide.
These trials will form part of the Glioblastoma Umbrella Signature Trial (GUST), which will test this and other drug combinations.
Associate Professor Patrick Pirrotte, Ph.D., a study author, said, “Our use of multiple vulnerability signatures in the GUST trial will demonstrate how a precision-medicine model can support an efficient clinical trial for diseases, such as glioblastoma, characterized by multiple mutations that result in multiple tumor types.”
The discovery of the potential MLN4924/Pevonedistat and Etoposide combination for treating glioblastoma represents a significant leap in cancer research.
If successful, this new treatment could be a game-changer, offering a new lifeline for thousands of glioblastoma patients and their families.
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The study was published in Neuro-Oncology.
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