New Personalized Medicine Products Signal Progress for Patients

The approvals of new treatments for two different types of cancer indicate that we are making significant progress in personalized medicine. Although the U.S. Food and Drug Administration (FDA) could further define a more transparent and predictable regulatory environment for personalized medicine products, the agency is clearly serious about personalized medicine. In August it cleared Xalkori® (crizotinib), which targets a rare form of lung cancer, ahead of its own priority six-month review goal and Zelboraf® (vemurafenib), which treats patients with melanoma also well ahead of schedule.

These combination products represent a harbinger of the future of medicine. By applying new genetic understanding of tumors to target treatments, drug and diagnostic companies working together are able to develop drugs that are safer and more effective, offering hope to cancer patients where none existed.

In the case of Xalkori®, Pfizer and Abbott Molecular co-developed the drug and a companion diagnostic test (Vysis ALK Break Apart FISH Probe Kit) to identify the subset of patients who are most likely to respond to the drug.  Around five percent of patients diagnosed with non-small cell lung cancer, the most common form of the disease, have tumors with an anaplastic lymphoma kinase (ALK) gene defect that responds to the drug – about 6,000 patients per year in the United States.  Xalkori® works by blocking proteins produced by the abnormal ALK gene.  It is the first lung cancer treatment developed and approved with a diagnostic test.

Stafford O’Kelly, Abbott Molecular Vice President and President Molecular Diagnostics, noted, “[This collaboration] marks a breakthrough in the advancement of personalized medicine…that will help a subset of lung cancer patients get treatment tailored to their unique genetic profile.”

Also recently approved is a targeted therapy to treat melanoma – the deadliest form of skin cancer.  Zelboraf® is indicated for treatment of patients with melanoma whose tumors express a gene mutation called BRAF V600E, which the drug blocks.  Approved with a diagnostic test (cobas® 4800 BRAF V600 Mutation Test) that determines which patients carry the mutation, the drug was clinically effective in 50 percent of these patients.

These new models for collaborative research and development of treatments are yielding positive results. They should keep us focused on the emerging field of personalized medicine and its promise to deliver the right treatment to the right patient with improved diagnosis, more efficient drugs, and better medical outcomes.