Potential drug therapy for fatal childhood cancer

Sydney, February 14: A team of researchers has identified a potential new drug treatment for a deadly childhood cancer, diffuse pontic intrinsic glioma (DIPG).

The study, published in the journal Nature Communications, reveals a potential combination of drugs that – in animal studies and in the world’s first 3D models of the tumor – is “spectacularly effective in eradicating cancer cells,” according to the researchers. researchers

In preclinical trials on mouse models, the researchers found that the promising drug combination led to the survival of two-thirds of the mice and that the drug combination completely stopped the growth of these very aggressive tumors in these mice.

Importantly, drug therapy, which is currently in early trials in adult cancer, is the most effective treatment ever tested in laboratory models of this incurable childhood cancer, the researchers said. , including David Ziegler of the Children’s Cancer Institute in Australia.

The treatment is a combination of two drugs – difluoromethylornithine (DFMO), an established drug, and AMXT 1501. DFMO is gaining more and more attention as a treatment for hard-to-control cancers like neuroblastoma, another cancer. aggressive childhood and colorectal cancer in adults. DFMO works by targeting the polyamine pathway – an important mechanism that allows tumor cells to grow, the team said.

For the study, the team developed Australia’s first DIPG research program using tumor cells donated by parents of children who died of the disease. From these, they created the first laboratory models of the tumor to test new drugs. These models were used to show that DIPG can bypass DFMO activity by pumping polyamines into the cancer, essentially allowing the tumor to continue to grow despite treatment with DFMO, the team said.

They have now made the breakthrough discovery that treatment with a new developmental drug, AMXT 1501, potently blocks the transport of polyamines into the DIPG cancer cell.

Treatment with AMXT 1501 re-sensitized DIPG cells to DFMO, which led to what Ziegler said, “was a dramatic response in animal models, with dramatically increased survival and minimal toxicity (side effects). ) ”.

Disclaimer: This story is auto-generated from the IANS service.