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Recent clinical trial suggests the drug vorasidenib could offer a new treatment option for low-grade glioma.

An international phase 3 clinical trial has shown that a new targeted therapy to treat low-grade gliomas can slow the progression of the disease. These findings have the potential to offer a new treatment option for those diagnosed with a grade 2 glioma.

These findings have the potential to offer a new treatment option for those diagnosed with a grade 2 glioma.

In this post, we’ll discuss: 

What is vorasidenib?

The drug vorasidenib inhibits the enzymes IDH1 and IDH2 and is able to cross the blood-brain barrier.

What are IDH1 and IDH2 enzymes? And how does vorasidenib work against them?

IDH or isocitrate dehydrogenase are a group of enzymes that are important in cell metabolism (how cells get their energy). They play an important role in various biological processes in the body.

Mutations in the DNA that code for IDH1 and IDH2 occur in the majority of low-grade gliomas (1). Therefore the discovery of a drug that can target IDH1 and IDH2 and reduce disease progression could offer a new treatment option for those diagnosed with this type of tumour.

Who was part of the trial?

The trial involved 331 patients aged 12 and over. They had residual or a recurrent grade 2 glioma with an IDH mutation.

Of the 331 patients, 168 were randomly assigned vorasidenib and 163 were randomly assigned the placebo.

After the trial ended, those who had received the placebo were then given access to vorasidenib due to its ability to slow disease progression.

What did the trial show?

The clinical trial showed that vorasidenib more than doubled the length of time until tumour progression compared to placebo (27.1 months vs 11.1 months).

Results also showed that the time to next treatment intervention was significantly reduced in those treated with vorasidenib compared to control (85.6% likelihood of not receiving intervention in the next 18 months vs 47.4% likelihood).

Additionally, the trial showed that treatment with vorasidenib had low levels of toxic effects for patients.

The researchers published these results in the New England Journal of Medicine.

Why is this so important?

Gliomas with an IDH mutation are the most common primary, malignant brain tumour affecting people under 50 years old (2). They are not curable and will continue to grow if they are not treated.

Nearly all grade 2 gliomas have an IDH mutation and include astrocytomas and oligodendrogliomas.

Current standard-of-care consists of surgical resection, followed by radiation and chemotherapy in selected patients (3). These treatments are highly toxic in the body and have many side effects. Therefore, there is a desperate need for new and more targeted treatment options with fewer side effects.

Is vorasidenib currently available?

Vorasidenib is not currently available to treat low-grade glioma.

It has been granted fast tracked designation by the Food and Drug Administration (FDA) in the US in March 2023. Details for the timelines for drug approval are being determined (4).

What does our expert think?

Dr Becky Birch, Head of Research at The Brain Tumour Charity, said:

“Research into low-grade gliomas is vitally important. Finding new, targeted treatments is essential if we are to improve the quality of life for those living with a brain tumour and accelerate towards a cure.

“This study shows promising results, and seeing that vorasidenib can more than double the time of progression free survival in patients is very encouraging.

“Understanding brain tumours at a molecular level and exploiting this knowledge to find targeted treatments will help those with this devastating diagnosis live longer, better lives.

“We hope that more people can have access to innovative treatments such as this because we urgently need kinder, more effective treatments for those facing this devastating diagnosis.

“Vorasidenib has been granted fast tracked designation by the Food and Drug Administration (FDA) in the US, and we’d like to see it receive a timely review by the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK to ensure this treatment is made available to patients as quickly as possible.

“Anyone affected by a brain tumour diagnosis can speak to us for support and information on 0808 800 0004 or by emailing support@thebraintumourcharity.org. If you need someone to talk to, we’re here for you.”

  1. Cohen, A.,Holmen S., and Colman, H. (2013). ‘IDH1 and IDH2 Mutations in Gliomas’, Current Neurology and Neuroscience Reports. 13(5):345. Access: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4109985/
  2. Mellinghoff, IK., et al. (2023). ‘Vorasidenib in IDH1- or IDH2-Mutant Low-Grade Glioma’, The New England Journal of Medicine. Access: https://www.nejm.org/doi/full/10.1056/NEJMoa2304194
  3. Youssef, G and Miller, J J. (2020). ‘Lower Grade Gliomas’, Current Neurology and Neuroscience Reports. 20, 21. Access: https://link.springer.com/article/10.1007/s11910-020-01040-8
  4. ‘Vorasidenib Significantly Improves Progression-free Survival in Grade 2 Glioma with IDH Mutation’. Read here. (Last accessed 29/06/23)

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