GBM is so difficult to treat partly because the greatest challenge for new neuro-oncology therapies is simply gaining access to the brain, which the body treats as its most protected organ within what’s called the blood brain barrier. The blood brain barrier is a highly specialized network of cells that both actively and passively protect the brain and maintain the brain's microenvironment. One of its primary functions is limiting toxins from entering the brain. This is a key reason why current chemotherapies are ineffective against GBM; they are not even able to reach the tumor. 

CNS Pharmaceuticals (NASDAQ:CNSP) is a biopharmaceutical company focused on developing innovative therapies for central nervous system disorders, specifically aggressive brain cancers. One of their leading treatment candidates is Berubicin, a chemotherapy drug that interferes with the DNA of cancer cells to inhibit their growth and potentially shrink tumors. Berubicin is an anthracycline, a clinically trusted class of drugs in use for 60 years against dozens of common cancers. However, unlike all other anthracyclines and other traditional chemotherapy drugs, CNS Pharmaceuticals reports that Berubicin has the ability to penetrate the blood-brain barrier and directly target cancer cells in the brain, increasing its potential effectiveness.

CNS Pharmaceuticals has received FDA Orphan Drug Designation and Fast Track Designation, which together grant them exclusive marketing rights and expedited development and review of drugs for serious conditions. The company is conducting a potentially pivotal clinical trial designed to evaluate the safety and effectiveness of Berubicin. Results from an earlier trial showed a clinical response in 44% of patients. With no approved treatment, the company’s dedication to developing novel therapies and the unique mechanism of Berubicin offers hope for patients whose brain cancer has returned – and options have all but disappeared.

In October, the company announced that 229 out of 243 expected patients had already been enrolled in their ongoing study evaluating the effectiveness of Berubicin for recurrent GBM, a highly aggressive and incurable form of brain cancer. This global study of Berubicin is a critical and adaptive trial that involves multiple centers and is open-label. It is randomized and controlled, comparing Berubicin to Lomustine – a drug unapproved for this indication but still used as the standard of care – in adult patients with recurrent GBM (WHO Grade IV) after standard first-line therapy has failed. The primary goal of the study is Overall Survival (OS), a rigorous measure that the FDA recognizes as fundamental for approving oncology drugs when there is a statistically significant improvement compared to a randomized control arm. Currently, the company has opened 46 clinical trial sites out of approximately 60 selected locations across the United States, Italy, France, Spain and Switzerland.

The company reports that its safety analysis is also showing that the drug is well tolerated by patients. Dr. Sandra Silberman, MD, PhD, the Company’s Chief Scientific Officer, will deliver a poster regarding the positive safety and toxicity data recorded so far at the Society for Neuro-Oncology Annual Meeting November 16-19 in Vancouver. CNS Pharmaceuticals had set a specific goal to analyze the efficacy data after more than half of the patients enrolled in the study reached the primary endpoint. This represents a significant portion of the total patient population in the trial. During this analysis, an independent data safety monitoring board (DSMB) closely examines the number of deaths in each treatment group to ensure that patients receiving Berubicin show a significant improvement in overall survival compared to those receiving Lomustine. They also look at other important factors like how long patients live without the cancer progressing, response rates and safety. The interim analysis will conclude if Berubicin is at least as effective as Lomustine, in which case the DSMB will make a recommendation that the trial continue.