Neurological researchers study promising new treatment for deadly brain tumors

A promising new treatment for deadly brain tumors taken orally with minimal side effects is being studied by neurological researchers at Rush University Medical Center, Chicago. Rush is the first and only site in the world using the new drug treatment for deadly brain tumors.

The study focuses on treatment of all malignant astrocytomas that include glioblastomas, which are highly malignant and fast-growing brain tumors. The treatment uses AXL1717, an orally-administered small molecule, insulin-like growth factor 1 (IGF-1) receptor pathway inhibitor. IGF-1 is a hormone present in many tissues that acts similar to insulin in activating a signaling pathway between cells to promote cell growth.

"With this drug, we may be capable of killing cancer cells selectively without damaging other brain cells," said Dr. Robert Aiken, associate professor of neurology at Rush University Medical Center and principal investigator leading the study. Aiken is also the director of The Coleman Foundation Comprehensive Brain Tumor Center and the Section of Neuro-oncology at Rush.

"We have started this Phase I clinical trial based on substantial experimental evidence and knowledge about this particular pathway in cancer," said Aiken. "AXL1717 is a highly-selective small molecule to target a tumor to potentially drive it into remission without harming healthy brain cells or most other tissues in the body. This treatment has great implications for many cancers that express this particular receptor."

Most tumor cells are dependent on the IGF-1 receptor signaling pathway, which is why this treatment is regarded as a promising target for brain tumors.

Rush hopes to recruit 30 high-functioning patients from around the nation with recurrent malignant astrocytomas. The patients must be over the age of 18, experienced one or more failed therapies, and have an expected survival of at least three months. 

To date, there are no IGF-1 receptor antagonist drugs on the market. Axelar is currently conducting a randomized Phase II clinical trial in Europe with AXL1717 in non-small cell lung cancer patients. A Phase I/II clinical trial with AXL1717 including 49 patients has been completed and demonstrated a good tolerability profile of the compound, in addition to its superior preclinical efficacy against numerous tumors.

Glioblastomas are highly-malignant brain tumors that represent approximately 17 percent of all primary brain tumors. The cause of glioblastomas is unknown and they are made up of cells that respond differently to treatments. Because some cells respond well to certain treatment while others may not, treatment often includes a combination of several approaches that may include radiation therapy, radiosurgery, corticosteroids, antiangiogenic therapy and chemotherapy. Nevertheless, the median survival for adults is one to two years.