Innovative brain cancer drug for non-treatable DIPG

Published on May 9, 2019

Researchers at the Cancer Research Institute in London, England, led an international drug development team that can treat DIPG, Diffuse Intrinsic Pontine Glioma. The development of this drug is critical in light of the fact that in the past 20 years, no new drug has been approved in the field. Doctors can treat the disease with radiation which is not curable and extends the child’s life by only 9-12 months after diagnosis. Chemotherapy is sometimes added to radiation.

The drug is in pre-clinical studies (not in humans) and has the potential to become the first treatment developed against DIPG.

The innovative drug works against a protein produced by mutations of the gene ACVR1, which controls about a quarter of DIPG cases. The mutations of ACVR1 are specific to this type of cancer.

The researchers found that the drug they developed reduced tumors and prolong life in mice by 25 percent, with minimal effect on healthy cells.

The drug company, M4K Pharma, Medicine for Kids Pharma, is expected to begin clinical trials (in humans) during 2021.

The study was published in the professional journal “Communications Biology”.

About DIPG

DIPG is a type of aggressive brain tumor in children that develops in the brain stem. The brain stem is located at the base of the brain and connects the brain to the spine. The brain stem is responsible for basic activities: vision, hearing, speech, walking, eating, breathing, pulse and more.
The diagnosis is usually done using CT or MRI, and not by taking a biopsy, which is the precise way to ensure that the lesion is cancerous. These tumors are difficult to take biopsy from by surgery because of their location such a procedure may pose a risk to the patient.

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