New Drug Slows Fatal Brain Cancer

Summary of New Drug Delays Progression of Deadly Brain Cancer:
A new targeted therapy drug, vorasidenib, has been shown to more than double the progression-free survival in patients with a glioma subtype, a slow-growing but deadly brain cancer. The drug inhibits mutant IDH1/2, delaying chemotherapy and radiation by almost 17 months, providing a breakthrough treatment for this deadly brain tumor. The study is the first clinical trial analyzing a targeted therapy specifically developed to treat brain tumors. The drug vorasidenib more than doubled progression-free survival in people with recurrent grade 2 glioma with IDH1 and IDH2 mutations, suggesting a possible new treatment option for people with the slow-growing but deadly brain tumor. The disease did not progress for an average of 27.7 months among those who received the drug, significantly longer than the 11.1 months for those who received a placebo. The FDA has not yet approved the drug for clinical use.

*****
A new targeted therapy drug called vorasidenib has proven successful in delaying the progression of a specific glioma, a slow-growing but deadly brain cancer. In an international study co-led by the University of California, Los Angeles (UCLA), 331 people were examined. The drug was shown to extend the time people with a glioma subtype are on treatment without their cancer worsening. The study was published in the New England Journal of Medicine and confirms that a new treatment option is now available for this slow-growing but deadly brain tumor.

Understanding the challenge

New treatment approaches for glioma are required because current treatments, including chemotherapy and radiation, can cause neurological deficits. The team found that the drug vorasidenib more than doubled the progression-free survival in people with recurrent grade 2 glioma with IDH1 and IDH2 mutations. The current standard treatment – a combination of radiation and chemotherapy – causes neurological deficits that make it hard for patients to learn, remember new things, concentrate, or make everyday decisions. With a patient cohort typically younger than most, the impact of this disruption on their lives can be significant.

A new hope

The availability of a treatment that enables patients to go for more extended periods between chemotherapy and radiation treatments could have a significant impact. Doctor Timothy Cloughesy, a professor of neuro-oncology at the David Geffen School of Medicine at UCLA and co-senior author of the study, acknowledged just how significant this breakthrough is, “having the ability to hold off on getting radiation therapy to the brain with an effective therapy is really critical and very meaningful to this population of patients.”

The effectiveness of vorasidenib

Vorasidenib is classified as a dual inhibitor of mutant IDH1/2. This means it prevents the formation and accumulation of the onco-metabolite 2-Hydroxyglutarate or 2-HG. 2-HG is formed when genetically altered versions of two enzymes, IDH1 and IDH2, are present in a tumor and areresponsible for forming and maintaining IDH-mutant gliomas. Vorasidenib is a brain-penetrant inhibitor that can cross the blood-brain barrier.

In the study, 331 people aged 12 and older who had been diagnosed with recurrent grade 2 glioma with the IDH1 and IDH2 mutations underwent brain tumor surgery. One hundred sixty-eight were randomly assigned to receive vorasidenib, and 163 received placebos. Among those who received vorasidenib, the disease did not progress for an average of 27.7 months, significantly longer than the 11.1 months for those who received the placebo. The disease progressed in just 28% of people receiving vorasidenib, compared to 54% receiving placebos.

The future of the drug

Doctors permitted a switch to vorasidenib for patients initially in the placebo group whose cancer began progressing during the study. The researchers observed limited adverse side effects from vorasidenib. “This is the first targeted treatment that shows unequivocal efficacy in this population and is precedent-setting for this disease,” Cloughesy said. Servier Pharmaceuticals manufacture Vorasidenib, which the FDA has not yet approved for clinical use. Discussions regarding approval of the drug will now begin.

Read the Source Here