PHILADELPHIA — This morning’s event at the White House to highlight the investments that are needed to improve public health and treat the myriad of diseases we call cancer underscores the nation’s opportunities to exploit genomics in clinical medicine. This topic is a significant focus of the American Association for Cancer Research’s (AACR) Cancer Progress Report 2014.
“We live in an extraordinary time when the scientific opportunities and our ability to translate this new knowledge into ways to both save and improve the quality of life of patients are simply astounding,” said Margaret Foti, PhD, MD (hc), chief executive officer of the AACR. “This is why we are so excited about today’s event at the White House and specifically about President Obama’s major investment in the enormous potential of precision medicine, which is in the very early stages of transforming health care.”
The president’s FY 2016 budget request will include $215 million for the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), and the National Coordinator for Health Information Technology (ONC), to support and foster the goals of precision medicine. Of the $215 million, the NIH will receive $130 million to recruit 1 million or more people to participate in a national volunteer research cohort that will help increase our understanding of disease. A total of $70 million will be provided to the National Cancer Institute (NCI), NIH, to scale up efforts to identify the genomic drivers in cancer and to translate this information into more effective strategies to prevent and treat cancer, which is the second-most common cause of death within the United States. The FDA will receive significant dollars to ensure that regulations are modernized.
The AACR Cancer Progress Report 2012 includes the personal story of one of the guests who was invited to attend the White House event, Melanie Nix, a survivor of triple-negative breast cancer. Less than 10 percent of all cancers are inherited; however, Melanie inherited a predisposition to developing breast and ovarian cancer from her mother in the form of an alteration in a gene called BRCA1. Genes like BRCA1 and the related BRCA2 gene produce proteins that normally prevent cancer-causing mutations from accumulating in our DNA.
Women, such as Melanie, her mother, and her aunts, who inherit mutated forms of these genes are at increased risk for breast and ovarian cancer, and affected men are at greater risk for breast and pancreatic cancer in addition to an aggressive form of prostate cancer. When Melanie was diagnosed, there were no precision medicines available to her, and she is with us today thanks to surgery, radiation, and traditional chemotherapy. However, in December 2014, the FDA approved olaparib (Lynparza), the first-ever molecularly targeted drug for treating women with ovarian cancer who carry a BRCA gene mutation. This is the first step toward precision medicine for a group of cancer patients like Melanie, who traditionally were left with few treatment options.
Another example of the power of precision medicine is the story of Zach Witt, featured in the AACR Cancer Progress Report 2014. Zach is a 10-year-old boy from Barto, Pennsylvania, who has a blood cancer called anaplastic large cell lymphoma (ALCL). When Zach was first diagnosed in 2010 at the age of five, he was given standard-of-care treatment for ALCL consisting of traditional chemotherapies that made him sick. Initially, this treatment helped to reduce the extent of Zach’s cancer, but eventually he relapsed. Fortunately, genetic testing done at the Children’s Hospital of Philadelphia identified the mutation driving Zach’s cancer, and more importantly, a drug called crizotinib (Xalkori) had been developed to treat a similar mutation in adult non-small cell lung cancer that might benefit Zach. In 2011, Zach enrolled in a clinical trial testing the efficacy of crizotinib in treating the between 10 and 15 percent of children who have ALCL that harbors the same genetic mutation. Zach has been in remission for four years and as his mother, Pam Witt, says, is “living the life of a normal 10-year-old.”
Stories like Zach’s, and those of many other cancer patients who have been fortunate enough to be treated with such novel strategies, underscore the true power of precision medicine, which seeks to develop therapeutics that treat the underlying cause of a disease while sparing normal tissue, and deliver them to the individuals who will benefit from these therapeutics.
In addition to the personal stories of many stunning examples of effective precision medicine, the AACR Cancer Progress Report 2014 chronicles the progress that has been made against the more than 200 diseases we call cancer and details how federal investment in the NIH, the NCI, and the FDA is transforming cancer care and the lives of patients in the United States and around the world.
Additional information regarding the Precision Medicine Initiative is available on The White House Blog and the NIH website.