A family of drugs used to treat muscular dystrophy could improve treatment of brain tumors

A drug from the same family licensed for use in patients with Duchenne muscular dystrophy and blood cancer could transform the treatment of meningioma—the most common form of primary brain tumor in adults. Scientists at the Brain Tumor Research Center of Excellence at the University of Plymouth carried out the research using patient samples in the laboratory, and the results have been published in the journal Cell Death & Disease.

About 3,500 adults are diagnosed with meningiomas each year, and with no chemotherapy drugs proving effective, the main treatment is surgery. However, some people cannot have surgery safely because of the size or location of the tumor, and for those patients, radiotherapy is the only treatment option. Unfortunately, in more aggressive forms of the disease, the tumor can become resistant to damage caused by radiotherapy, reducing its effectiveness.

As part of their ongoing work to uncover new treatments, the scientific team—led by Dr. Juri Na and Professor Oliver Hanemann—explored whether a drug called dacinostat could help improve the impact of radiotherapy on meningioma.

Dacinostat belongs to a family of drugs known as HDAC (Histone Deacetylase) inhibitors, which affect how cells control their DNA and respond to damage. HDAC inhibitors have already shown promise in cancer therapy, with three currently approved by the FDA for the treatment of various blood cancers and Duchenne muscular dystrophy.

Using patient samples to create models of meningioma in the laboratory, Dr. Na and her team demonstrated that treating tumor cells with low doses of dacinostat before radiotherapy increased DNA damage and cell death, and reduced tumor growth.

"For the first time, we show how dacinostat works in detail, revealing its potential as a new treatment for aggressive meningioma. Outside surgery, these patients have very limited options, and radiotherapy alone is often insufficient for aggressive or recurrent meningiomas. This research provides strong evidence that combining radiotherapy with drugs such as dacinostat could improve tumor control while minimizing treatment-related toxicity, with the potential to enhance patient quality of life."

In addition to identifying dacinostat as a potential route to boosting the effectiveness of radiotherapy, the team has uncovered for the first time the process that meningioma tumors use to repair DNA after radiotherapy. This has opened up a completely new area of research that could lead to more treatments to increase the effectiveness of radiotherapy.

Based on these findings, Dr. Na and her team are now testing new treatment approaches that combine radiotherapy with approved anti-cancer drugs. After screening more than 7,000 drug combinations in meningioma models, they are now studying the most promising candidates in greater detail. The aim is to identify safe, effective combinations that can move rapidly into clinical use.

This article was originally published on MedicalXpress Breaking News-and-Events.