Could a repurposed drug break a centurylong stalemate in the war on cancer?

A pharmaceutical compound approved 21 years ago to treat breast cancer in postmenopausal women is showing fresh promise as a therapy for glioblastomas, the usually deadly brain tumors against which little progress has been made over the past 100 years.

The science is advancing at the University of Cincinnati, where pharmaceutical scientist Pankaj Desai, PhD, have received a $1.19 million grant from NIH’s National Institute of Neurological Disorders and Stroke to dig deeper into the use of letrozole for glioblastomas (GBMs).

A news item posted by the university’s UC News notes that the drug works by targeting aromatase, and enzyme implicated in the flourishing of breast cancer cells.

Upon finding the enzyme presented in brain tumor cells as well, a former doctoral student in Desai’s lab hypothesized letrozole might work off-label in cancers of the brain.

That was in 2012, according to UC News.

Since then Desai’s team has studied the drug first in animal models and then in cell lines drawn from human brain tumors.

“What we saw in the patient-derived cells is that letrozole is very effective in killing the tumor cells in cell culture models,” Desai says in the item.

More from UC News:

Desai said early results have shown the drug is ‘unequivocally’ reaching its target of the brain tumor tissue safely. Preliminary results also show that doses of letrozole higher than those needed for breast cancer treatment can be safely achieved in GBM patients.”

Desai acknowledges that, even if found effective against GBM, letrozole likely wouldn’t suffice as a standalone therapy.

To find out how much it could contribute to the cause of advancing against GBM, Desai and colleagues will work under the NIH grant for the next three years, initially combining letrozole with other chemotherapy compounds in new animal studies.

“I think finding a cure for a disease like GBM is like finding a needle in a haystack, and we hope that it’s going to really work, and that’s what we are all striving for,” he says in the UC news piece. “This is a disease where an urgent breakthrough is absolutely needed.”

He’s not overstating the matter. According to the National Brain Tumor Society, the five-year survival rate for glioblastoma patients is only 6.8%, and the average length of survival for glioblastoma patients is only around eight months. That’s despite GBM’s maintaining a steady presence in the scientific literature since the 1920s.

Read the full UC News item.

Dave Pearson

Dave P. has worked in journalism, marketing and public relations for more than 30 years, frequently concentrating on hospitals, healthcare technology and Catholic communications. He has also specialized in fundraising communications, ghostwriting for CEOs of local, national and global charities, nonprofits and foundations.

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