Dana Farber. US: President Barack Obama is requesting an increase of $215 million in
the 2016 federal budget to launch the Precision Medicine Initiative.
This boost in funding for research will give genetic causes of cancer a
national focus specifically around precision or “personalized”
treatments for cancer in the future.
Here are some facts about precision medicine:
1) What is precision or “personalized” medicine?
Physicians
have long recognized that the same disease can behave differently from
one patient to another, and that there is no one-size-fits-all
treatment. Precision medicine
makes diagnosis and treatment of cancer and other diseases more
accurate, using the specific genetic makeup of patients (and, in cancer,
of their tumors) to select the safest and most effective treatments for
them.
In cancer, precision medicine involves testing DNA from
patients’ tumors to identify the mutations or other changes that drive
their cancer. Then a treatment for a particular patient’s cancer that
best matches, or “targets,” the culprit mutations in the tumor DNA is
used. While such therapies are not widespread yet, many cancer
specialists believe precision treatments will be central to the future
of cancer care.
2) Do all patients receive precision or targeted treatment?
Not
all patients need targeted therapy to treat their type of cancer. The
use of targeted therapies is meant for patients whose tumors have
specific gene mutations that can be blocked by available drug compounds.
Patients who have mutations in certain types of genes, who have
mutations that are beyond the reach of available drugs, or whose tumor
cells lack identifiable mutations generally would not be candidates for
personalized medicine treatments.
According to the National Cancer
Institute, a patient is a candidate for a targeted therapy only if he
or she meets specific criteria, which vary depending on the disease.
These criteria are set by the Federal Drug Administration (FDA) when it
approves a specific targeted therapy.
3) What is the precision medicine initiative at Dana-Farber?
The Profile project, launched in 2011 at Dana-Farber Cancer Institute and Brigham and Women’s Hospital,
is an example of precision medicine at work. All adult patients
diagnosed with cancer can consent to have samples of their tumors
analyzed for the presence of mutations and other cancer-related DNA
abnormalities.
“We’re really excited about Profile because it
represents our first foray into using molecular techniques, rather than
anatomical criteria, to understand cancers” said William Hahn, MD, PhD,
deputy chief scientific officer at Dana-Farber. “With all of the
knowledge we’ve gained over the last 30 or 40 years about what makes
cancers tick, we can now try to get at the basics of that within the DNA
of a tumor.”
Since the inception of Profile, more than 10,000
genetic profiles of patients' tumors have been completed, and 400
profiles are added each month to the database. Profile testing recently
expanded to pediatric patients at Boston Children’s Hospital.
4) What cancers have benefited from precision medicine research?
Ten years ago, researchers at Dana-Farber and in Japan published a study showing that non-small cell lung cancer
patients whose tumors had a malfunctioning version of a protein called
Epidermal Growth Factor Receptor (EGFR) responded dramatically to a drug
that specifically targets the EGFR protein.
“Prior to this research, lung cancer treatment had not made much progress,” says Bruce Johnson, MD, chief clinical research officer at Dana-Farber, who co-led the 2004 study with colleagues Pasi Jänne, MD, PhD, Matthew Meyerson, MD, PhD, and William Sellers, MD (now of the Novartis Institutes for BioMedical Research). “Today, patients with EGFR
mutations who are treated with targeted drugs have a year of remission
and survive an average of two to three years, with some making it to
five years or more.”
Besides lung cancer, Dana-Farber researchers
have made precision cancer discoveries in triple-negative and
HER2-positive breast cancer, colorectal cancer, neuroblastoma, small
cell lung cancer, and other malignancies.
Clinicians at
Dana-Farber/Brigham and Women's Cancer Center currently use targeted
therapies that pinpoint genetic mutations in a select number of other
cancers, including kidney, leukemia and sarcoma.
5) What is the future of precision medicine?
New
research in precision medicine is now focused on identifying a far
greater number of mutations for a wider array of cancers, ultimately
enabling clinicians to treat more cancers with targeted therapies in the
future.