New Gene Therapy Sparks Hope Against Deadly Brain Cancer

A groundbreaking gene therapy is making waves in the fight against glioblastoma (GBM), a ruthless brain tumour known for shrugging off treatment. Scientists at Brigham and Women’s Hospital have unveiled a novel oncolytic virus that not only attacks cancer cells but also jolts the immune system into action. Their promising findings were published in Nature.

Beating Glioblastoma’s Defences

GBM is a brutal killer with a grim survival time of under 10 months once it recurs. Traditional immunotherapy has failed because the tumour creates an immune-resistant environment, making the body’s defenders useless. The researchers set out to flip the switch on this immunosuppressive fortress, turning it into a hotspot for immune attack.

The secret weapon? CAN-3110, an engineered herpes simplex virus (oHSV) designed to infect cancer cells. Unlike previous versions, this virus includes the ICP34.5 gene—usually dropped due to safety concerns—but crucial here to trigger the all-out inflammatory onslaught needed to fight the tumour without damaging healthy brain tissue.

Promising Trial Results

  • 41 patients with high-grade gliomas took part in the phase I trial, 32 had recurrent GBM.
  • Only two experienced seizures as serious side effects.
  • Patients with pre-existing HSV1 antibodies (66%) lived a median of 14.2 months—surpassing expectations.
  • Tumours showed signs of a ramped-up immune attack, with increased diversity of tumour-fighting T cells.

Experts believe the virus kills tumour cells, releasing cancer antigens that rev up the immune system. These immune changes linked to longer survival provide fresh hope for a disease long resistant to treatment.

What’s Next for CAN-3110?

The Gene and Cell Therapy Institute at Mass General Brigham is charging ahead, planning bigger trials to test CAN-3110 in patients both with and without HSV1 antibodies. They also aim to explore the effects of up to six injections in four months, hoping to boost the therapy’s power. Funding from Break Through Cancer backs this next phase.

“The virus provoked a strong immune response with infiltration of tumour-killing T-cells,” said Dr E. Antonio Chiocca, Chair of BWH Neurosurgery and lead author. “These results open a new door for treating GBM—something traditional immunotherapy hasn’t been able to do.”

This breakthrough gene therapy offers a genuine shot at beating one of the deadliest brain cancers. GBM’s days of dodging treatment may finally be numbered.

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