UW to provide promising and expensive cancer drug
Last-chance $475,000 treatment to be offered at 20 hospitals
The University of Wisconsin-Madison’s American Family Children’s Hospital will be one of 20 sites to offer one of the most promising and expensive new cancer drugs, one that will come with what amounts to a limited warranty.
UW took part in the clinical trial of the drug Kymriah, which just won approval as a type of gene therapy that harvests a person’s immune cells. The drug treats a rare form of cancer known as acute lymphoblastic leukemia in patients under age 25 who have relapsed or not responded to other treatment.
The one patient UW treated during the clinical trial, a young woman who had been given less than six months to live, went into remission and has remained so for nearly six months, said Christian Capitini, a pediatric cancer doctor with UW Health.
“After all the patient has been through, it is amazing to say she is now in remission,” he said.
In the clinical trial, 83% of the 63 patients who got the drug were in remission within three months. However, the drug also can cause serious or even fatal side effects, though it is hoped those can be controlled with other drugs.
“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” FDA Commissioner Scott Gottlieb said in a statement. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.”
The drug also carries a $475,000 price tag for the one-time infusion.
However, the drug’s maker, Novartis, said it has worked out a deal with the U.S. Centers for Medicare and Medicaid that allows for payment only when the patient responds within one month of treatment.
That’s not good enough, said Vinay Prasad, a cancer specialist with Oregon Health & Science University, who said the drug’s price was “cruel.”
He noted that more than $200 million in federal funding had gone into helping develop the therapy.
“For the first five years after (CAR)-T (therapy) any patient who dies, who does not respond, or who responds, but has cancer return, should be issued a full refund,” he said.
The treatment is known as chimeric antigen receptor (CAR)-T cell therapy. It works by engineering a person’s immune cells, known as Tcells, to express a CAR and, when infused back into the body, to attach to and kill those leukemia cells.
It is estimated that about 1,000 people a year will be candidates for the drug, but it is hoped that the therapy can be adapted to other cancers.
Capitini guessed that UW might see about five patients a year.
He said UW already has received certification to administer the drug and is waiting final approval from Novartis and the U.S. Food and Drug Administration.
The drug can cause toxicity to the brain as well as a side effect known as cytokine release syndrome, which can range from high fever and body ache to life-threatening problems that might require a ventilator. There are drugs to treat the syndrome.