In April, Genzyme (www.genzyme.com) reported that it established a program to participate in efforts to discover and advance novel treatments for neglected diseases affecting the developing world. The company’s new Humanitarian Assistance for Neglected Diseases initiative (HAND) will serve as a vehicle to identify, evaluate, and manage scientific projects and partnerships focused on diseases that collectively affect hundreds of millions of people. These could include malaria, tuberculosis, leishmaniasis, Chagas disease, sleeping sickness, and other diseases.
Under the program, the company earlier this year began collaborating with the Broad Institute of MIT and Harvard University to discover and advance new therapeutic candidates for malaria. Approximately 300–500 million cases of malaria occur each year, killing more than one million people annually, with young children and pregnant women being the most vulnerable. The majority of malaria cases occur in sub-Saharan Africa, but the disease also remains a significant health problem in Asia and South America.
According to Roger Wiegand, Ph.D., director of infectious diseases at the Broad Institute, almost all of the drugs currently in clinical trials for malaria were discovered 30 or more years ago. Artemisinin, the most promising new drug, was first isolated from the Chinese medicinal plant in 1972, he notes.
“Until recently there has been relatively little application of the newest technologies to antimalarial drug discovery,” says Dr. Wiegand, adding that the Broad Institute has advanced drug discovery capabilities that are being employed in this project. These include high-throughput screening, chemical genomics, sequencing and genetic mapping, and genome annotation.
“With respect to high-throughput screening, we use an assay in which we monitor the growth of parasites in infected red blood cells,” explains Ted Sybertz, Ph.D., senior vp of scientific affairs at Genzyme.
“Through work at the Broad, we have access to several different strains of parasite with different drug sensitivity profiles. The Broad has identified the gene sequence on some of these. By looking at patterns of drug sensitivity across different strains and attempting to match drug sensitivity with known genetic differences in the strains, we hope to identify new targets of drug action. In addition, we can utilize techniques of affinity labeling and proteomics to define targets of unknown drugs.”