Glioblastoma Multiforme

Glioblastoma Multiforme (GBM), a type of central nervous system cancer, is the most common and most aggressive form of primary brain cancer. Globally, over 241,000 people die each year as a result of brain or nervous system cancer, with GBM being the most common form of the disease. In the United States alone, approximately 13,200 new cases are diagnosed annually, making it a significant public health concern. Many prominent political figures, including John McCain, Ted Kennedy, and Beau Biden, have battled this deadly disease.

​

The prognosis for GBM remains dire. The five-year survival rate is less than 4 percent and even lower in older patients—only 1.7 percent for those aged 65 and above. The median age at diagnosis is 58, with a median age at death of 65. These sobering statistics reflect the aggressive nature of this cancer and the limited effectiveness of current treatment approaches.

​

Historically, radical surgical resection was the primary treatment offered to GBM patients. However, studies revealed no significant outcome benefit—patients died within the same timeframe whether or not they underwent radical resection. The cause of rapid mortality is the quick recurrence of tumors in other areas of the brain. It took scientists considerable time to discover that the tumor has already disseminated throughout the entire brain long before it is diagnosed at a single location. As Dr. Mark Gilbert of the National Cancer Institute stated, "Removing all the visible tumor does not cure this cancer—ever." Years ago, surgeons even removed the entire half of the brain containing glioblastoma, yet this "did not markedly increase survival." Patients still died because malignant cells had already invaded the rest of the brain.

​

Current chemotherapy options offer limited hope. Only 30 percent of GBM tumors respond to first-line chemotherapy with temozolomide, and those who do respond gain only an additional three months of survival compared to untreated patients. The tumor readily becomes resistant due to mechanisms involving cancer stem cells and epithelial-mesenchymal transition (EMT). Despite advances in recent years—including targeted drugs, immunotherapies, vaccines, and leading-edge radiation techniques—outcomes remain poor. The average cost of care for a refractory patient to live 12 months can reach $780,000 for a single drug alone, with hospitalizations and disability costs adding significantly to the financial burden.

​

Despite exhaustive efforts to treat this disease, the complex biology of glioblastoma cells has defeated even the most sophisticated scientific expertise. Glioblastoma cells move quickly, transition to mobile mesenchymal forms, and disseminate throughout the brain, making it virtually impossible to effectively treat a single localized tumor site.

​

There is hope on the horizon. MTET effectively reduces the ability of glioblastoma cells to migrate and disseminate. It directly kills cancer cells and specifically targets glioblastoma stem cells. In various in vivo models, epigenetic therapies have demonstrated the ability to drastically shrink tumors where chemotherapy has been ineffective or shown minimal efficacy. Epigenetic therapies are also considered a cornerstone of secondary prevention, reducing the risk of recurrence in this devastating disease.

The Cancer Research Life Foundation is currently working to sponsor and implement an epigenetic therapy trial using MTET as part of a clinical study for refractory glioblastoma. The foundation aims to fully cover the estimated $8 million cost of this groundbreaking trial, offering new hope to patients facing this aggressive cancer.