FDA approves Orphan Drug status for nano-based pediatric cancer drug

October 27, 2008 The Cure Our Children Foundation, a nonprofit charitable foundation dedicated to children, announced today that the US Food and Drug Administration (FDA) has approved the Orphan Drug Designation of the foundation’s unique drug product for children with Ewing’s Sarcoma cancer. The efforts to develop this drug were made possible by the generous volunteers and researchers in private industry and at two universities.

Orphan Drug status allows for recognition of the potential viability of a drug therapy while providing a variety of benefits during the drug approval process. These benefits include waivers of certain FDA fees, the availability of government grants, and FDA attention and assistance during the review process.

This groundbreaking new drug combines two modern technologies: biotechnology and nanotechnology. This technology is analogous to the concept of a Trojan Horse, and is expected to have very far reaching implications for other cancer treatments. The product consists of cell matter that is modified to have the same genetic code as the cancer cells, but is not viable food for the tumor cells. The cell matter is then placed in a nanotechnology formulation which allows the matter to migrate through the body’s own vessels directly to the tumor cells. When the tumor cells uptake the matter, they cannot reproduce, and they die.

Benefits of this drug include the possibility of fewer side effects; the drug is directed only at the tumor cell and not at healthy cells; the product is so small that it migrates right through blood vessels and cell walls; and it may be applied to other diseases in the future that have a genetic component.

The president of the foundation, Barry Sugarman, a 30-year veteran executive and consultant in the pharmaceutical industry — and father of son who has survived Ewing’s Sarcoma — will continue the development of the drug product by raising money from individuals and foundations.

The Cure Our Children Foundation identifies important under-researched children’s issues and devotes extensive resources to educate and guide parents, professionals, government and the public. The results of the research are provided as a public service, and are supported by donations to the foundation. The foundation has a number of other research projects underway that will continue to benefit children and families.


Lainie, Alon, Lilah and Barry Sugarman. Alon Sugarman was diagnosed with Ewing’s Sarcoma of the Distal Femur on March 6, 1998. He is now 16 years old.

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