Repurposing Drugs for Glioblastoma

Glioblastoma is a stage IV glioma – a cancer of the glial cells in the brain. It is the most common type of primary (arising in the brain) brain tumor. While it is confined to the brain, by no means does this designate it as a less lethal cancer. Some refer to glioblastoma as the emperor of all cancers, and this title is very fitting. Glioblastoma is a deadly tumor, and the current treatments we have for it (surgery, radiation, chemotherapy) are inadequate. While these treatments may prolong a patient’s life by a couple of months, what they offer in quantity of time, they rob in quality of time.

While the median age of those affected by glioblastoma is around 59 years with males being 1.5x more likely to be afflicted, glioblastoma is starting to be seen more commonly in younger individuals.  Specifically, in an age group referred to as AYA (adolescents and young adults), individuals between the ages of 15-39. Researchers have found that glioblastoma in these individuals is distinctly different from glioblastoma in older adults. A focus of Glioblastoma Foundation is to support research to study glioblastomas that arise in AYA individuals.

Temozolomide, an oral chemotherapeutic agent, remains the current mainstay of therapy; however, some patients are not candidates for this drug - and even for those that are - resistance is starting to develop. There have been no new drugs for glioblastoma in over 30 years. This is unacceptable. 

Small molecules (drugs) present a compelling, and as yet, unexamined therapeutic option for glioblastoma.

The current standard of care is not working. With the current standard of care, patients can expect to live an average of 15 months after diagnosis. This is appalling, and Glioblastoma Foundation believes that we need to transform therapy for glioblastoma.  Small molecules may offer the best chance for doing so by attacking the tumor from within. However, resistance will inevitably occur, and the only way to out run this cancer is to develop several different drugs. We see a future in which glioblastoma is no longer a terminal diagnosis but a chronic condition that can be managed by drug therapy.

The current lack of therapeutic options for glioblastoma is leading researchers to examine opportunities for repurposing existing drugs currently used for other conditions. Glioblastoma Foundation supports this initiative, as there are several drugs currently available that possess characteristics that may make them effective agents for glioblastoma.

To be an ideal drug candidate for glioblastoma, a drug must penetrate the brain and exhibit appropriate accumulation to be effective but also not cause significant toxicity, because patients may not be willing to take a drug regularly if it causes significant side effects.

Overall, drug repurposing presents a compelling avenue for discovering effective therapies for glioblastoma. Drug repurposing could very well lead to novel therapeutic paradigms for glioblastoma.  

We may not have a cure for glioblastoma in the next five years, or the next ten years - but we will have a cure one day - and Glioblastoma Foundation will be a part of it by supporting research into novel small molecule therapies for glioblastoma. If you would like to get involved and help us transform glioblastoma therapy, please contact us.