GTC Biotherapeutics Inc announced that the National Institute of Allergy and Infectious Diseases (NIAID) has approved a proposal to fund development of clinical-grade production for a recombinant form of the Merozoite Surface Protein 1 (MSP-1) antigen of the Plasmodium falciparum parasite as a vaccine for malaria. The development work will be performed under the existing NIAID Contract No. NO1-A1-05421 managed by Science Applications International Corporation (SAIC). The scope of work includes developing founder goats that express the MSP-1 antigen in their milk as well as the downstream purification process and final product formulation. The approved scope of work also includes the submittal of an Investigational New Drug (IND) application to the Food and Drug Administration (FDA). GTC's portion of this project will be supported completely with Federal funds amounting to at least $4.9 million.

"GTC is very pleased that its transgenic technology has made the development and production of this difficult-to-express vaccine candidate possible. GTC's technology has the added benefit of being highly cost effective, an important factor for the developing countries where malaria is prevalent," stated Geoffrey F. Cox, GTC's Chairman and CEO. Dr. Cox continued, "We are pleased that the NIAID has recognized and supports the commitment GTC has made to develop the MSP-1 vaccine."

In preclinical studies involving scientists from the NIAID and GTC working under a Cooperative Research and Development Agreement, four out of five Aotus nancymai monkeys were successfully protected from developing malaria after exposure to the parasite using MSP-1 material that GTC developed in the milk of transgenic mice. By comparison, six out of seven unvaccinated animals had to be treated for virulent malaria. This study was published December 18, 2001 in the Proceedings of the National Academy of Sciences online.

The World Health Organization (WHO) estimates that 300-500 million people are infected annually worldwide, with over one million malaria-attributable deaths each year, mostly among infants and children. Mosquitoes that transmit the disease have become resistant to insecticides, and the most lethal form of the parasite, Plasmodium falciparum, has become resistant to the major drug used to treat the disease, chloroquine. It is widely recognized that development of effective malaria vaccines would constitute a significant global public health advance. Commercial-scale production of potential MSP-1 antigen-based vaccines has been hampered by the inherent difficulty in expressing these proteins in other recombinant systems, where they have been produced at limited levels or in forms that may not induce the necessary immune response.