Commercialization deal boosts hope for new sickle-cell drug

A novel once-a-day treatment for sickle-cell disease, based on technology developed at Children's Hospital Boston (CHB) and Beth Israel Deaconess Medical Center (BIDMC), has taken an important step toward the clinic. McNeil Consumer & Specialty Pharmaceuticals and Icagen, Inc., a private biotechnology company, have agreed to jointly develop and commercialize the drug, known as ICA-17043. The agreement follows successful Phase II clinical trials in 90 adults with sickle-cell disease. The companies will collaborate on multicenter Phase III trials, the final step before seeking marketing approval from the U.S. Food and Drug Administration.

Current treatment options are few for sickle-cell disease, a genetic blood disorder that affects millions of children and adults worldwide, including about 70,000 Americans, primarily people of African descent (about 8 percent of African-Americans are carriers). The only approved sickle-cell treatment, developed at CHB 20 years ago, is hydroxyurea. Companies have been slow to invest in additional sickle-cell treatments because the potential market is relatively small. The FDA granted ICA-17043 Orphan Drug Designation in 2000 and Fast Track Status in 2002.

ICA-17043 is related to clotrimazole, an antifungal drug approved in 1975 and long used to treat vaginal yeast infections, jock itch, and athlete's foot. A team led by Dr. Carlo Brugnara, director of the Hematology Laboratory at CHB, discovered in the early 1990s that clotrimazole also reduces sickling of red blood cells.

In sickle cell disease, the body produces an abnormal form of hemoglobin, the compound in red blood cells that carries oxygen. The hemoglobin clumps up and polymerizes forms long rod shapes. This polymerization bends the red cells into sickle-shaped structures, and also causes the cell to lose water. This dehydration, in turn, hardens the cells and promotes sickling by hastening polymerization.


Contact: Mary-Ellen Shay
Children's Hospital Boston

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