FDA grants orphan drug status to new ‘Trojan Horse’ like Ewing’s Sarcoma drug



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The Cure Our Children Foundation, a nonprofit charitable foundation dedicated to children, announced today that the U.S. 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 incredible 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 that matter 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. Key elements of this drug technology are:

  • Fewer side effects may be possible
  • 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
  • This technology 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.

http://www.cureourchildren.org/

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Ewing sarcoma is the second most common type of primary bone cancer seen in children and young adults. Patients with relapsed or refractory Ewing sarcoma have a poor outcome with conventional therapies. Cytarabine decreases the levels of a certain key protein in Ewing sarcoma cells and has demonstrated preclinical activity against Ewing sarcoma cell lines

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