Three new approaches for brain cancer treatment

Three new approaches for brain cancer treatment

A new targeted drug that more than doubles survival times, an mRNA vaccine and the use of Ultrasound to open the blood brain barrier to standard chemo, offer potential for tackling GBM. 

Vorasidenib - a completely new targeted drug

A new drug targeted at patients with an IDH1 or IDH2 mutation in their GBM, nearly tripled survival times in a trial involving 331 people over the age of 12, with recurrent grade 2 Glioma. All subjects had received surgery. The disease progressed in just 28% of people receiving Vorasidenib, compared to 54% of those receiving the placebo. As of September 2022, which was 30 months after the study began, 72% of patients who were in the Vorasidenib group were still taking the drug and their disease had not progressed.

Researchers were confident that this new drug could replace the standard Temozolomide/Radiotherapy combination, given for 6 weeks to almost everyone with brain cancer, although Temozolomide only works in the 20% of patients who are methylating.

The International study was published in the New England Journal of Medicine (1). Dr. Timothy Cloughesy, a professor of neuro-oncology and a member of the UCLA Jonsson Cancer Center, said that this drug can allow oncologists to hold off giving patients debilitating radiotherapy to the brain.

An mRNA vaccine for GBM

After preclinical tests in mice, then tests on 10 dogs with brain cancer, University of Florida Health Cancer Center trialled (2) their new mRNA vaccine in four humans to great effect although few details appear available. The vaccine involves using the patients’ own tumour cells to create the vaccine along with a change in the delivery system. A researcher described it as injecting a cluster of particles wrapped around each other ‘like onions’. 

In reality it’s an adaptation of mRNA technology and lipid nanoparticles, similar to those in COVID-19 vaccines. Next stop is a Phase 1 clinical trial with Children. The vaccine caused a very rapid and aggressive immune response to the tumours.

Ultrasound allows standard chemo to bypass the blood brain barrier

Doctors at NorthWestern in Chicago have used Ultrasound to temporarily open up the blood brain barrier allowing standard chemo drugs such as Paclitaxel and Carboplatin to attack the brain tumour. 

In the first in-human clinical trial (3), they used a novel, skull-implantable ultrasound device which created microbubbles to open the blood-brain barrier, allowing standard chemo drugs given by IV to permeate the tumour. The four-minute procedure to open the blood-brain barrier was used for up to six sessions of chemo.

The patient was awake, and could even go home after just a few hours. The results show the treatment is safe and well tolerated. The blood brain barrier is completely restored after 24 hours. There is now a Phase II Clinical Trial taking place to study increased survival times.

Founder of CANCERactive and Oxford University Biochemist Chris Woollams added, “We desperately need new options for brain cancer patients. Temozolomide only works in approximately 20% of patients and is really quite weak. It was approved for use by the FDA in 1986, almost 40 years ago. Clinical trials on Avastin have shown no real increases in survival times. And radiotherapy can cause damage. We’ve really gone nowhere since Catherine’s day.” 

Go to: Brain cancer - alternative treatments that work?




  1. Vorasidenib in IDH1- or IDH2-Mutant Low-Grade Glioma; Ingo K. Mellinghoff et al; June 4, 2023; N Engl J Med 2023;389:589-601, Vol 389; No 7

  2. UF-developed mRNA vaccine for GBM - 

  3. Powerful chemotherapy drug reaches brain tumors using novel ultrasound technology; Northwestern Now, May 3 2023 -




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