A new drug developed by French pharmaceutical company Servier has been shown to slow the progression of brain cancer considerably. The drug, vorasidenib, is a small molecule inhibitor that targets the IDH1 gene, which is mutated in a subset of patients with glioblastoma multiforme (GBM), the most common and aggressive form of brain cancer.
In a phase II clinical trial, vorasidenib was shown to significantly prolong the time it took for tumors to grow or spread (progression-free survival) in patients with recurrent GBM. The median progression-free survival for patients treated with vorasidenib was 16.6 months, compared to 10.6 months for patients who received standard of care treatment.
Vorasidenib was also well-tolerated in the trial, with the most common side effects being fatigue, nausea, and vomiting. The drug is currently being evaluated in a phase III clinical trial, which is expected to be completed in 2024.
If vorasidenib is approved by the FDA, it would be the first new drug for GBM in more than a decade. The current standard of care for GBM is surgery followed by radiation therapy and chemotherapy. However, these treatments are only effective for a short period of time, and most patients with GBM die within two years of diagnosis.
The development of vorasidenib is a significant advance in the fight against brain cancer. The drug's ability to slow the progression of GBM could offer patients with the disease more time to live and to participate in clinical trials of other experimental therapies.
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