Personalized Therapies Mark Significant Leap Forward in Fight Against Cancer

Personalized Therapies Mark Significant Leap Forward in Fight Against Cancer

http://www.innovation.org/index.cfm/NewsCenter/Newsletters/Newsletters?NID=191   October 12, 2011  This year marks the 40th anniversary of the signing of the National Cancer Act of 1971. Indeed, the 12 million cancer survivors living in the U.S. today attest to the significant progress in cancer prevention and treatment we have made over the past decades. Despite the remarkable advances we have made there are still more than 550,000 men and woman who lose their battle to cancer each year.   Recently released scientific data demonstrate that the collective commitment to cancer research is unwavering and our knowledge of the biology of cancer and ability to treat it continues to expand. One promising trend in cancer research: drug developers are harnessing an improved understanding of the molecular basis of many types of cancer to develop therapies uniquely targeted to these pathways.   For example, a newly approved drug for lung cancer called crizotinib is targeted to a mutation in a gene called anaplastic lymphoma kinase, or ALK. Mutations in the ALK gene are found in approximately 5% of patients with non-small-cell lung cancer. In data presented at this year’s meeting of the American Society of Clinical Oncology (ASCO), 54% of patients who received crizotinib were still alive after two years compared to just 12% in a control group. Crizotinib received fast-track review by the U.S. Food and Drug Administration (FDA) and was approved in August ahead of the six-month priority review schedule.   Dramatic advances are being made in the treatment of the skin cancer melanoma as well. More than 60 drugs are currently in development for the disease and this year two new medicines have been approved – the first approvals for the disease in 13 years. The first, ipilimumab, was approved in March and was the first treatment ever approved by FDA to show a survival benefit for patients with metastatic melanoma. In August the second, a new personalized medicine called vemurafenib, was approved to treat this deadliest form of skin cancer. This drug, which is taken orally, selectively inhibits a mutated form of the BRAF kinase gene. The mutated gene is associated with increased tumor aggressiveness, decreased survival, and is found in approximately half of all malignant melanomas. Recently reported clinical trials results demonstrate that the medicine reduces the risk of death by 63% percent.   Personalized medicine holds great potential beyond these two select examples in lung cancer and melanoma. MD Anderson Cancer Center recently reported on the results of a large-scale clinical trial examining the effect of matching targeted therapies with specific gene mutations across many cancer types. According to the results of the study, patients who received a targeted therapy demonstrated a 27% response rate compared to 5% for those whose therapy was not matched. This clinical trial marks the largest examination of a personalized approach to cancer care to date, and as principal investigator Apostolia-Maria Tsimberidou, M.D., Ph.D. concludes, "This study suggests that a personalized approach is needed to improve clinical outcomes for patients with cancer."   As these and many other studies illustrate, a dramatic transformation in cancer diagnosis and treatment is underway. Therapies targeted to the genetic and molecular underpinnings of disease are being developed, and patient outcomes are improving as a result. The studies highlighted above only begin to scratch the surface of the remarkable potential of personalized, targeted therapies, but are an indication of the great reward of years of research and investment, as well as great promise for continued innovation in the years to come.