FDA orphan status for children's cancer drug

The Los Angeles, USA-based Cure Our Children Foundation, a non-profit charitable foundation dedicated to children, says that the Food and Drug Administration has approved the Orphan Drug Designation of its unique drug product for children with Ewing's sarcoma cancer. The efforts to develop this drug were made possible by volunteers and researchers in private industry and at two universities, says the Foundation.

This ground-breaking new drug combines two modern technologies and is analogous to the concept of a Trojan Horse, the Foundation notes. The product consists of cell matter that is modified to have the same genetic code as the cancer cells, but that matter is not viable food for the tumor cells. The matter is then placed in a nanotechnology formulation which allows it to migrate through the body's own vessels directly to the tumor cells. When the tumor cells uptake the matter, they cannot replicate and die.

Key elements of this drug technology are: fewer side effects may be possible; the agent is directed only at the tumor cell and not at healthy ones; the product is so small that it migrates right through blood vessels and cell walls; and this technology may be applied to other diseases in the future that have a genetic component.