During his State of the Union Address, President Obama announced that he is launching a new initiative that will help deliver the right treatment to the right patient at the right time. Precision medicine gives medical professionals the resources they need to target the specific treatments of the illnesses we encounter, further develops our scientific and medical research, and keeps our families healthier. As the country that eliminated polio and mapped the human genome, America is well-positioned to lead in a new era of medicine.
While most of today's medical treatments have been designed for the average patient, precision medicine allows us to be more effective than a "one-size-fits-all" technique. It's an emerging approach to promoting health and treating disease that takes into account individual differences in people's genes, environments, and lifestyles. This makes it possible to design targeted treatments for cancer and other diseases. As the President noted in his speech, this revolutionary approach has even reversed cystic fibrosis, an illness once thought unstoppable.
This approach is already saving lives, giving those in the medical field better options, and helping keep families healthy. Read a few of their stories:
William Elder, Jr. was diagnosed with cystic fibrosis (CF) at the age of eight, when the life expectancy for CF patients was very low. Now at 27, Bill is alive thanks to Kalydeco, a treatment of a particular form for his cystic fibrosis and a remarkable drug that treats the underlying cause of his CF, rather than the symptoms.
At a congressional briefing in 2013, Bill told members of the U.S. Senate that just knowing that there were individuals who were researching his condition gave him hope and the strength to continue his treatments and work to be healthier every day. Bill described waking up in the middle of the night after taking his new treatment for the first time. “I sat on the floor of my room for a while slowly breathing in and out through my nose, and then I realized that was it. I had never been able to easily breathe out of my nose before. This was something profound,” he said. He recalls telling his parents, "For the first time in my life, I truly believe that I will live long enough to be a grandfather.”
At age six, Emily Whitehead was the first pediatric patient to be treated with a new kind of cancer immunotherapy and was cancer free only 28 days later. “If you didn’t know what happened to her, and you saw her now, you would have no idea what she has been through,” says Emily's Mom.
Her parents decided to enroll her in a pioneering cancer immunotherapy trial at the Children’s Hospital of Philadelphia. Emily’s T-cells were collected from her blood and re-engineered in the lab to recognize a protein found only on the surface of leukemia cells. Those T-cells were then infused back into Emily’s blood, where they circulated throughout her body on a mission to seek and destroy her leukemia. Knowing how to turn these T-cells into what Emily called “ninja warriors” required big investments in basic biomedical research. In fact, Science Magazine named it a 2013 Breakthrough of the Year -- Emily's family couldn't agree more.
Melanie Nix's family has a history of breast cancer -- a history that Melanie couldn't escape when she tested positive for the BRCA gene mutations linked to breast cancer in 2008. After 16 rounds of chemotherapy and breast reconstruction surgery, she had to have both ovaries removed to further reduce risks of cancer in the future. But Melanie is now cancer free thanks to precision medicine.
Melanie's positive test results for the BRCA gene mutations instantly concerned her medical team. BRCA gene mutations are linked to breast and ovarian cancers. Further tests confirmed that she had triple-negative breast cancer, a very aggressive form of breast cancer that disproportionately affects African-American women. Her best chance for cancer-free survival was to have a bilateral mastectomy. Melanie says that this type of tailored treatment gave her hope. "Precision medicine offers the hope that by the time my daughter is at an age when she considers genetic testing, new, targeted treatments will be available to give her additional choices for preserving her health," she said.
Beatrice Rienhoff's eyes were spaced wider than usual, her leg muscles were weak, and she couldn't gain weight. Her father, a trained clinical geneticist, took notice and wanted to help. After six years, he and his team of scientific volunteers identified the cause of her condition.
Beatrice's original medical team had thought her condition resembled Marfan syndrome, a genetic disorder that can cause tears in the human heart. It's typically a fatal syndrome. However, the doctors couldn't fully diagnose Beatrice with Marfan -- or any other known disease. Acting as "Super Dad," Hugh lead his team to identifying a variant responsible for his daughter's condition and this research gave rise to the description of a whole new syndrome. The team continues to use precision medicine to learn more about the new syndrome and further study genetic variation to help those like his daughter. Today, Beatrice is living a full life.
Six-time NBA Most Valuable Player, Kareem Abdul-Jabbar was diagnosed with a form of leukemia in 2008. Known to be lethal, leukemia is a cancer of the blood and bone marrow. It caused the basketball great to slow down, fall ill, and worry. A few years later, he credits precision medicine for helping him to be well today.
Keith Yamamoto has dedicated his life to medicine and research. Well-known for his molecular biology and biochemistry research, Keith leads a major precision medicine effort at the University of California, San Francisco (UCSF). He continues to be a leader in the precision medicine field.
Keith currently serves as the Vice Chancellor for Research and Executive Vice Dean of the School of Medicine at UCSF. He also continues to teach, allowing younger generations to learn from his research. While promoting up-and-coming methods for targeting the specific treatments needed to help patients, Keith also chaired the 2011 National Academies Report on Precision Medicine.
We've only begun to tap into potential for precision medicine to improve care and produce new treatments. Translating initial successes to a larger scale will require a coordinated and sustained national effort -- an effort the President wants America to lead. Learn more at www.whitehouse.gov/precision-medicine