According to a story from Oncology News, a team of researchers affiliated with UNSW Sydney have made a discovery that could have major implications for the rare cancer neuroblastoma, a type of cancer that primarily affects children. The findings could lead to new approaches that could prevent the disease from appearing and provide effective treatment.
Neuroblastoma is a type of cancer that appears in nerve tissue. Children are more frequently affected than adults. The direct cause of neuroblastoma is not known in most cases; in a small number of patients, an inherited mutation ALK gene has been implicated, but this only explains around two percent of cases at the most. Symptoms of neuroblastoma include a lump in the affected area which may be blue in color, bone and joint pain, loss of appetite, fatigue, and fever. Other symptoms vary depending on where the tumor is located; it typically originates in the adrenal glands but can also appear on the spine, neck, abdomen, or chest. Treatment approaches include immunotherapy, radiation therapy, chemotherapy, surgery, and stem cell transplant. Outcomes vary considerably; the five year survival rate is 68 percent for patients between age one and fourteen. To learn more about neuroblastoma, click here.
The Role of MYCN
The discovery is related to the MYCN oncogene. This gene has been linked to neuroblastoma and several other types of solid tumor cancers. The research has helped the scientists more clearly understand the link between the MYCN oncogene and certain molecules that can facilitate the growth of cancer cells. In about a quarter of cases, cancer tumors contain more copies of MYCN. These cases are deemed high risk and are associated with worse than average outcomes.
The researchers found that the polyamine molecular pathway, which helps cancer cells grow, is almost entirely controlled by the MYCN oncogene. A drug targeting a gene that is associated with the synthesizing of polyamines, called DFMO, is currently being tested, but the scientists also identified the gene that allows these molecules to be utilized by neuroblastoma cancer cells. The team determined that the activity of this gene could be inhibited by another therapy called AMXT-1501.
In a neuroblastoma mouse model, the researchers combined these two drugs alongside a standard chemotherapy regimen. The drug was able to increase survival in mice with the disease and was also capable of preventing mice who were genetically predisposed to neuroblastoma from developing tumors. This combination is currently being tested in human patients in a Phase 1 clinical trial in several different cancer types.
The results of this trial could indicate a new and highly effective treatment option for neuroblastoma and other cancers linked to MYCN.