A new cancer treatment drug, CBL0137 (referred to as CBL) is currently being tested in adults and brings new hope to children with neuroblastoma. Researcher’s believe the drug will not only improve chances of survival, but may be able to prevent neuroblastoma if administered at birth. Best of all the new treatment does not damage DNA, which is the problem with most cancer treatments currently being used.
The MYCN gene is a known driver of neuroblastoma, but it also helps with the development of the sympathetic nervous system in the embryo. When the gene fails to shut off and continues to produce cells, tumors begin to develop. There is an essential feedback loop that accelerates the cancer involving the MYCN gene, MYCN proteins (a byproduct of the gene) and FACT, a DNA modifying agent.
FACT communicates with the MYCN gene to keep producing cells, which will result in even more MYCN proteins. FACT also prolongs the life of the MYCN proteins, which are what accelerates the progression of the cancer. CBL’s job is to target and block FACT, essentially stopping the progression of the cancer.
CBL, when used in combination with chemotherapy, can cut therapy time significantly and reduce the life-long side effects by blocking repairing mechanisms within the cancer cells, allowing the chemotherapy to work more efficiently.
Children’s oncology group, largest cancer study group in the world wants to take drug into clinical trial next year, but trials at Children’s Cancer institute in Australia will begin phase 1 trials soon.