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.
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.”
Emily Whitehead
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
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.
Hugh and Beatrice Rienhoff
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.
Kareem Abdul-Jabbar
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
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.
