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National Brain Tumor Society Provides Funding to Expand the Potential of a Promising Therapeutic in Development for Glioblastoma

2020-05-21

$100,000 grant will support researchers at Cleveland Clinic and Curtana Pharmaceuticals investigating the possible dual action of a novel small molecule drug


National Brain Tumor Society (NBTS), a leader in brain tumor patient advocacy, research, information, and support, today announced that it’s providing $100,000 to support research on the novel drug candidate, CT-179, which is being developed as a potential treatment for glioblastoma (GBM) by Curtana Pharmaceuticals. The grant will support preclinical studies of CT-179 in the laboratory of Justin Lathia, PhD, principal investigator in Cleveland Clinic’s Lerner Research Institute and co-director of the Brain Tumor Center of Excellence.

“There is quite a bit already known about CT-179 that we found encouraging,” said Kirk Tanner, PhD, Chief Scientific Officer, National Brain Tumor Society. “We hope now, with the additional funding from NBTS’s Defeat Brain Tumors research program, that we can learn more about the latent capability of this compelling drug, including if it’s ultimately amenable to combinations with immunotherapeutics in order to generate potentially more effective and durable responses for patients.”

Preclinical studies have shown CT-179 to be a potent inhibitor of Olig2, a member of a family of proteins known as transcription factors, which is found in markedly high quantities in nearly all cancer stem cells within GBM tumors, but not in normal adult brain cells. A prominent theory in cancer research holds that cancer stem cells are resistant to chemotherapy and radiation therapy, and thus survive initial treatment attempts and may ultimately be main drivers of tumor recurrence and mortality. Thus, the researchers’ approach is to add CT-179 to the standard of care therapy for GBM patients at diagnosis to eliminate the cancer stem cells, potentially improving the survival of patients. 

Additionally, Dr. Lathia has hypothesized that over-expression of Olig2 in myeloid-derived suppressor cells (MDSCs) may modulate the GBM tumor microenvironment resulting in insensitivity to immunotherapy drugs. MDSCs hamper the ability of the immune system to mount an attack on tumor cells. By blocking Olig2 with CT-179, the drug may reduce immune suppression mediated by MDSCs and pave the way to combine CT-179 with immune system-activating treatments like checkpoint inhibitors to generate a two-pronged attack on tumors.

“There is accumulating evidence MDSCs can potently suppress immune activation in glioblastoma, leading to tumor growth and resistance to immune-activating strategies,” reports Dr. Lathia. “We are actively evaluating approaches to target MDSCs to increase immune activation, and are excited to  study the potential of targeting Olig2 in MDSCs via CT-179.”

In prior studies, CT-179 has demonstrated the ability to successfully cross the blood-brain barrier and extend survival in animal models of GBM. The work funded by NBTS will allow Dr. Lathia to test his hypothesis that targeting Olig2 with CT-179 could also complement immunotherapy strategies by reversing the immunosuppressive nature of the tumor microenvironment to potentially increase an anti-tumor immune response, and enhance the effect of immune-activating therapies, such as anti-PD1 or anti-CTLA-4 drugs.

“We already know that CT-179 extends survival in relevant animal models, particularly when combined with standard of care chemotherapy and radiation,” said Gregory Stein, MD, CEO of Curtana Pharmaceuticals. “However, to-date we’ve not been able to fully test our hypothesis that killing cancer stem cells should make drugs that activate the immune system more effective. The research proposed by Dr. Lathia will allow us to answer this question and, potentially, provide us with much better strategies to treat this horrible disease and improve the lives of GBM patients. We are tremendously grateful for the support of the NBTS and Dr. Lathia. Without their leadership and expertise, this important research could not be done.”

ABOUT GLIOBLASTOMA

Glioblastoma is the most commonly occurring primary malignant brain tumor (brain cancer), accounting for nearly half of all new brain cancer diagnoses. With a five-year relative survival rate of only approximately six percent, they are also the deadliest of adult brain tumors, and, indeed, one of the most complex, aggressive, and heterogeneous of all cancers. More than 13,000 estimated new cases of GBM will be diagnosed in 2020. 

About National Brain Tumor Society

National Brain Tumor Society invests in, mobilizes, and unites the brain tumor community to discover a cure, deliver effective treatments, and advocate for patients and care partners. We are the largest patient advocacy non-profit solely dedicated to the brain tumor community and a leader in collaborating with the neuro-oncology field. Headquartered in Newton, Massachusetts, our organization raises funds to invest in accelerating brain tumor treatments, prepare the community to navigate their unique brain tumor experience, and convene stakeholders while changing public policy to improve the lives and survival of brain tumor patients. Visit us at www.braintumor.org.

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