New Experimental Drug Shows Promise for Brain Cancer

VIDEO | 02:08

“This represents the first new treatment option in low-grade glioma in more than 20 years,” says Ingo Mellinghoff, MD, Chair of MSK’s Department of Neurology, who led a clinical trial showing the drug’s effectiveness.


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We finally have a new tool that might really make a difference for our brain tumor patients.

Patients with low-grade glioma have a very difficult choice to make. They know that the tumor is continuously growing in the absence of treatment, but they also know that the treatment, which is standard of care with radiation and chemotherapy, comes with considerable toxicity.

Having a drug available would be a game-changer for these patients.

I was diagnosed with a grade 2 astrocytoma.

I'm a nurse anesthetist and I need to continue to do my job and do it well. So, I can't have radiation to the brain yet. I'm young and I just wanted to keep working.

The drug Vorasidenib was specifically designed to get to the tumor cell.

The INDIGO trial with Vorasidenib was a double-blinded placebo-controlled trial. Neither the patient nor the physician knew who was getting the drug.

The data analysis occurred quicker than we expected because the result was so clear.

I was on the placebo and they switched me over to the drug. I’ve had no growth, nothing. Like, it was slowly, slowly growing and all of a sudden plateau since I started the drug.

It's very exciting to tell patients that their brain tumor is not growing.

We've been very fortunate at Memorial Sloan Kettering to play a leading part in this effort. I think we’ve kicked the door open. It is a very successful, clinically meaningful result that I believe will have a large impact on many patients worldwide.