Clofarabine
Clofarabine is a next-generation purine nucleoside antimetabolite that is currently under review by the FDA for the treatment of relapsed or refractory acute leukemias in children. Nucleoside analogs are antimetabolites that affect DNA synthesis. For more information about the clofarabine clinical trials, please visit www.clinicaltrials.gov.
Pediatric Leukemia
In October 2003, the first part of a New Drug Application (NDA) was submitted to the U.S. Food and Drug Administration (FDA) for clofarabine for the treatment of refractory or relapsed acute leukemias in children. The remainder of the clofarabine NDA application was submitted to the FDA in March 2004. The FDA has stated that it expects to respond to the marketing application on, or before, December 30, 2004.
Acute leukemia consists of acute lymphoblastic leukemia (ALL) and acute myelogenous leukemia (AML). ALL is the most common cancer in children. Up to thirty percent of children with ALL relapse despite initial therapy and fail to recover. AML is the second most common cause of pediatric cancer. Although most children with AML can achieve initial remissions with intensive chemotherapy, approximately 30-60 percent relapse and eventually succumb to their disease.
Both diseases are rapidly progressing cancers that accumulate in the marrow and quickly infiltrate the marrow space, preventing the formation of healthy, infection-fighting blood cells. ALL and AML differ based on the cell type that becomes cancerous. Symptoms of ALL and AML are similar to the signs of the flu, making them often difficult to recognize. The most common symptoms are persistent fever, weakness, frequent infections, bleeding, bruising and bone and joint pain.
Adult Leukemia
As part of Genzyme Oncology’s development program, the combination of clofarabine and ara-C is also being studied in a Phase I acute leukemia trial in adults over the age of 50 years.
Solid Tumors
Two Phase I clinical trials are ongoing to evaluate the safety and tolerability of clofarabine in adult patients with locally advanced or metastatic solid tumors who have either failed standard therapy or for whom no standard therapy exists.