Nov. 27, 2018 / 12:27 PM
CST
By Lauren Dunn and Maggie Fox
The Food and Drug Administration has approved a new cancer drug
that is the first to be designed from the start to fight a specific genetic
mutation, not a traditional cancer type.
The new drug, named Vitrakvi, is not approved to fight breast
cancer or lung cancer or colon cancer. Instead, it’s designed and approved to
treat cancers that arise anywhere in the body that carry a certain genetic
characteristic.,
201801:26
“Traditionally in cancer therapy, we've treated patients based
on where their cancer came from, what part of the body. What makes Vitrakvi
unique is that it works regardless of where the cancer came from as long as it
has the specific mutation,” said Dr. David Hyman, chief of early drug
development at Memorial Sloan Kettering Cancer Center in New York.
While several drugs are approved to treat a variety of different
cancers based on genetic mutations, Vitrakvi, known generically as
larotrectinib, is the first that is approved from the beginning to treat
cancers solely based on the mutation.
“Today’s approval marks another step in an important shift
toward treating cancers based on their tumor genetics rather than their site of
origin in the body,” FDA Commissioner Dr. Scott Gottlieb said in a statement.
“We now have the ability to make sure that the right patients
get the right treatment at the right time. This type of drug development
program, which enrolled patients with different tumors but a common gene
mutation, wouldn’t have been possible a decade ago because we knew a lot less
about such cancer mutations.”
Keytruda was the first example of a drug approved to treat a
genetic mutation. Originally FDA approved in 2014 to treat melanoma that had
spread, the makers of Keytruda showed it could treat other tumor types, if they
had the same mutation that drives some cases of melanoma. Now it’s used against
a range of tumors, including breast cancer.
Vitrakvi treats a different genetic mutation involving genes
called NTRK genes. Rarely, they can fuse together and cause the out-of-control
growth that results in tumors.
“Prior to today’s approval, there had been no treatment for
cancers that frequently express this mutation, like mammary analogue secretory
carcinoma, cellular or mixed congenital mesoblastic nephroma and infantile
fibrosarcoma,” the FDA said.
Anna Plaza says her daughter, Rihanna, is living proof of how
well the drug can work if given to the right patient. When Rihanna was born in
Bridgeport, Connecticut last year, she had an enormous mass on her arm.
“They didn't know what was wrong. They just knew it was a mass
and they covered it up with a Pamper and we needed to get transferred to
another hospital because nobody knew what to do with us,” Plaza told NBC News.
“I was like, ‘oh my God, what am I going to do? What are we
going to do?" she added. "It was horrible."
Biopsies showed Rihanna had a rare tumor known as infantile
fibrosarcoma, a malignant soft tissue tumor typically found in children under
one year of age.
Chemotherapy did little to help the tiny baby. The family
eventually was referred to the clinical trials being done to test the drug at
Memorial Sloan Kettering.
“It was a syrup. She would take it once in the morning, once at
night.” Surprisingly, the baby liked it.ted
Three days later, the tumor was already shrinking.
“When we went back to Memorial Sloan Kettering, they were even
more shocked,” said Anna Plaza’s husband Enrique. “They were like ‘Whoa, we've
never seen this process go fast. We've seen it in months, but so fast, within a
week?’” he added.
“We were happy because they didn't have to cut her arm off.”
Surgeons could much more easily remove the tumor afterwards.
"If we hadn't shrunk it first, she could had ended up with a forearm and a
hand that didn't work the way they should for the rest of her life," said
Dr. Todd Heaton, a pediatric oncologist who helped treat her.
Rihanna still goes for follow-up treatments and, as with so many
targeted cancer therapies, only a small percentage of cancer patients are
helped.
“For patients that have this mutation — it is a revolution for
these patients,” Hyman said.
“But what we really have recognized about cancer is that it's
not a one-size-fits-all approach, and we need to apply the best therapy for
every patient.”
Another roadblock — health insurance companies don't always pay
for the genetic tests needed to guide doctors to the targeted drugs. Hyman hopes
having a specific FDA approval can help change that.
And these drugs are not cheap. Bayer, which partners with the
company that makes Vitrakvi, says it will cost $393,600 a year, while the
pediatric syrup formulation will cost $11,000 a month.
Lauren
Dunn is a producer with the NBC News medical unit in New York.
Maggie
Fox is a senior writer for NBC News and TODAY, covering health policy, science,
medical treatments and disease.
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