A new drug that attacks the precise form of deadly brain cancer that has stricken GOP Sen. John McCain of Arizona is being hailed as a potentially life-saving medical breakthrough.
Called Berubicin, it belongs to a class of drugs known as anthracyclines, which are among the most powerful anti-tumor medications ever discovered.
Unlike other drugs in this category, Berubicin is able to pass through the blood barrier that protects the brain from dangerous toxins, striking directly at the cancer. That means Berubicin may prove uniquely able to halt the cancer's deadly progression.
Pharmpro.com reports: "Fighting brain cancer has been particularly difficult because of the blood-brain barrier, which is made up of special endothelial cells that pump potentially harmful substances out, keeping any disease-causing pathogens from entering the brain.
"But this property has also kept most anticancer drugs that could effectively kill these tumors from reaching the brain."
A Phase 1 clinical trial already has been conducted. It triggered substantial, anti-tumor effects in 44 percent of patients. CNS Pharmaceuticals' chief medical officer, Dr. Sandra Silberman, called those results "extremely encouraging and exciting," and added the drug "may change the way glioblastoma is treated worldwide."
Glioblastoma, one of the most virulent malignancies known to man, has long been considered a virtual death sentence. The median survival rate is about 15 months. About 13,000 cases are diagnosed in the United States annually.
The disease strikes the brain's supportive tissues, sending out eerie tentacles that snake through the brain. Surgeons say it is virtually impossible to remove all of the cancer surgically.
Among its notable victims: Liberal lion Sen. Ted Kennedy and Beau Biden, the son of former Vice President Joe Biden. McCain was diagnosed with the disease last July. Current therapies have limited effectiveness.
Waldemar Priebe, an internationally known expert on cancer treatments, is credited with the discovery of Berubicin at the University of Texas MD Anderson Cancer Center. He is the founder of CNS Pharmaceuticals.
Phase One trials are primarily intended to determine whether a given medication is safe. The next step in the development of Berubicin will be a Phase Two trial.
Phase Two trials are focused more on evaluating a drug's effectiveness, usually via a more expensive trial involving a larger group of subjects. CNS has announced it will defray the cost of the drug's development via a unique equity crowdfunding campaign.
The New York Times reports that in the past five years, the frequency of patients surviving two years or longer following a glioblastoma diagnosis tripled from 8 percent to 25 percent. The development of Berubicin suggests that more help from the frontier of medical science may soon be on the way.
According to Informa Pharma Intelligence, a market research firm, there are currently 87 experimental therapies in clinical trials that target glioblastoma. Some drugs are vaccines, some involve gene therapy, some activate the immune system to fight the cancer and some rely on monoclonal antibodies.
The other new medicines and treatments that are either in use or moving through the medical-trial pipeline include:
- A cocktail of Avastin, an anti-angiogenic drug that restricts blood-vessel growth and helps control brain swelling, is being combined with an immunotherapy medicine called Rintega that helps the body recognize the cancer the immune system needs to target.
- On the surgery front, StatNews.com reports a new "imaging agent" won approval in June that could make a difference. Patients drink a product called Gleolan three to five hours before surgery. It turns the tendrils of glioblastoma pink, enabling doctors to find and remove much more of it.
- M.D. Anderson Cancer Center in Houston is collaborating with Moleculin Biotech on a drug known as WP1066, a STAT3 inhibitor, that can stimulate a natural immune response against tumor cells to halt their progression.
- A study published by the Journal of Experimental Medicine suggests a derivative of the Zika virus, which induces cell death in the brains of developing fetuses, may one day be used to attack glioblastomas. The trick is to get the Zika to attack the cancer cells, while leaving normal brain cells alone. The study reports: "Genetically modified strains that optimize safety could have therapeutic efficacy for adult glioblastoma patients."