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US approves first gene therapy against cancer.
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The United States has approved the first gene therapy against cancer — a treatment that uses a patient’s own immune cells to fight leukemia — opening a new era in the fight against one of the world’s top killers.
Flash Points:
The treatment is made by Novartis and is called Kymriah (tisagenlecleucel).
This type of anti-cancer immunotherapy, known as a CAR-T cell therapy, was known by CTL019 until now.
This marks the first ever CAR-T cell therapy to be approved anywhere in the world.
It uses a new approach that is wholly personalized by using a patient’s own T-cells.
Kymriah was approved by the US Food and Drug Administration for children and young adult patients up to age 25 with a form of acute lymphoblastic leukemia (ALL)
The FDA described the approval as “a historic action” that would usher “in a new approach to the treatment of cancer and other serious and life-threatening diseases.
Procedure of treatment:
To qualify for treatment, patients must have B-cell precursor ALL that is refractory, or the patient has relapsed at least twice.
The treatment is not a pill or a form of chemotherapy, which can weaken the body’s natural defenses.
Instead, it harnesses a patient’s own immune cells, called T-cells and white blood cells, to recognize and fight cancer.
The patient’s immune cells are removed with a special blood filtration process, sent to a lab, and genetically encoded with a viral vector to be able to hunt down cancer cells.
These re-engineered T-cells are then transfused back into the patient, where they can begin attacking leukemia.
Studies have shown that 83 percent of patients responded to the treatment, achieving remission within three months.
Viability:
According to Novartis the price of Kymriah — which is delivered to a patient just once — is $475,000.
Patients who do not respond to the treatment within the first month would not be expected to pay, he told reporters.
The more common treatment for leukemia — bone marrow transplants — can cost between $540,000 and $800,000 the first year in the United States.
Meanwhile, outside analyses set a cost-effective price for Kymriah between $600,000 and $750,000.
Most patients who fit the criteria for treatment would likely be covered by insurance, since they are under 25 and would either be on their parents’ insurance or covered by Medicaid.
Breakthrough therapy.
The treatment was pioneered by Carl June at the University of Pennsylvania.
Its most high-profile patient is Emily Whitehead, now 12, who six years ago was the first child to receive what was widely considered a risky treatment.
She has been cancer free ever since.
In 2014, US regulators designated CTL019 as a “breakthrough therapy” and put the experimental immunotherapy agent on the fast track to market approval.
It was the first cancer immunotherapy to receive the breakthrough designation.
About two dozen centers in the United States are expected to be able to treat patients seeking Kymriah, with as many as 35 up and running by the end of the year.
