Researchers at the Translational Genomics Research Institute (TGen) have identified a drug called propentofylline, or PPF, that might help treat individuals with deadly brain tumor. The significant breakthrough reports that the drug could control the spread of glioblastoma multiforme, or GBM (the most common basic tumor of the brain and central nervous system) by targeting a protein known as TROY.

“PPF reduced glioblastoma cell expression of TROY, inhibited glioma cell invasion, and made brain cancer cells more susceptible to TMZ and radiation”, stated Dr Nhan Tran, Associate Professor and head of TGen’s Central Nervous System Tumor Research Lab. The study was funded, in part, by The Ben & Catherine Ivy Foundation.

Prevent Brain Tumor: Explaining Glioblastoma

GBM is among the most aggressive forms of cancer, affecting people of all ages. The median survival for newly diagnosed cases of GBM is only 14.6 months, and only five percent of patients survive more than 5 years. “Funding research that focuses primarily on helping patients survive longer is essential. Studies such as this fulfill this goal of not only improving treatments for brain cancer, but ultimately hoping to find a cure.

The basic treatment of GBM involves surgical excision of the brain tumor. However, due to the aggressive nature of the tumors and their progressive invasion into surrounding brain tissue, complete removal of the tumor is not possible. Thus, the cancer eventually returns and spreads, adversely affecting the effectiveness of chemotherapy and radiation therapy.

Preventing Brain Tumor: Propentofylline The Remarkable Drug

This small molecule has been tested in clinical trials and has proved to be successful in treating Alzheimer’s disease and dementia. What’s great is that the drug has already been approved by the FDA. “Our data shows that PPF, in combination with TMZ and radiation, could control glioblastoma invasion and significantly improve the clinical outcome of patients with brain tumors”, said senior author Dr Tran.

PPF works by targeting and decreasing the expression of TROY protein. Researchers associated this protein to the cellular mechanisms that assist GBM tumors to invade healthy brain cells and defy anti-cancer drugs.

“New therapeutic regimes targeting molecular aspects of invasion are needed for improving clinical outcomes”, highlighted lead author Dr Harshil Dhruv, a TGen Research Assistant Professor. “Propentofylline (PFF) may prove to be a pharmacologic approach for targeting TROY, inhibiting cell invasion and decreasing therapeutic resistance in glioblastomas”.

Crossing The Blood Brain Barrier

Among the main challenges in treating brain cancer using drugs is the blood-brain barrier that separates circulating blood from the brain’s extracellular fluid. This mechanism protects the brain from exposure to toxins. However, this safety mechanism also shields the brain from various life-saving drugs, hence making it difficult to treat brain tumor and other diseases of the brain.

“Clinical trials have demonstrated that PPF can cross the blood-brain barrier, that too with minimal side effects. Moreover, PPF could easily be translated to clinics as an adjuvant therapy in combination with the regular care treatment regime for GBM patients”, concluded Dr Tran. The study was published in the Journal of NeuroOncology.