Promising 1-Shot Cancer Treatment Comes With $373,000 Price Tag

It may be scary, but the truth is cancers occur in our bodies all the time.

One study — on the tonsils of young people — found two-thirds contained abnormalities that could lead to cancer.

Obviously, two-thirds of kids don’t develop cancer. Instead, their immune systems recognize the disease and kill it.

But trouble can start when it’s the immune system that gets cancer.

In the most common form, relapsed or refractory large B-cell lymphoma, Dr. Richard Maziarz, with the OHSU Knight Cancer Institute, explains it’s one type of white blood cell, the B-cell, that gets sick.

“And the way I often describe this to patients is: The B-cells are like artillery, well behind the enemy lines, and you’re firing off shells at a distance, to kill at a distance,” Maziarz said.

When it's the B-cells that have cancer, they don’t fire off shells to kill themselves.

But thankfully there is another type of cancer-fighting white blood cell — the T-cell.

“And the T-cells are like infantry, they have to have hand to hand combat to deliver their lethal hit," Maziarz said.

So the T-cells should just go and kill the cancerous B-cells. But there’s a problem. The T-cells have to be taught what to target.

That’s how vaccinations work. A person is injected with a weak form of the measles for example, and the T-cells learn to attack it.

And that’s the basis of this new CART-T immunotherapy. A researcher draws blood from a lymphoma patient, sends it for manufacture where they stimulate growth in the T-cells, then genetically insert a new gene.

“What makes it unique is, it’s genetically constructed to have an antibody-binding site,” Maziarz said.

That allows the lethal T-cells to bind onto and kill the cancerous B-cells.

The treatment essentially reteaches the body's own immune system to fight cancer again. It’s literally one injection — tailor-made for the individual — that can cure their cancer.

“This is literally Star Wars-type science," said Stephanie Carlson, the executive director of the Leukemia and Lymphoma Society in Oregon.

“What we are doing is we unlock the body’s own immune system and use it to fight cancer cells,” Carlson said.

The new CART-T therapy is called Kymriah and this new clinical trial focused on patients with an advanced form of the disease and had already relapsed at least once. The trial found that 19 months afterward, 40 percent of patients were in remission, meaning their cancers can no longer be seen by imaging equipment.

Before immunotherapy treatments, there were really only three ways of battling cancer: poisoning it with chemotherapy, burning it with radiation or physically cutting it out.

With relapsed blood cancers like lymphoma, doctors would kill all the blood cells then do a complete bone marrow transplant. And in advanced, multiply relapsed patients, that success rate in the range of 15 percent.

“But with CART-T, instead of using those harsh chemicals, where we’re literally killing everything," Carlson said. "The immune system is able to identify the bad cells, the cancerous cells and it’s able to attack those cells only.”

The drug company Norvatis owns Kymriah and charges $373,000 for the single-injection treatment.

“It doesn’t do any good to invest millions in this research and then our patients can’t get access to it because of the cost of drugs,” Carlson said.

Sen. Roy Wyden, D-Ore., agrees. “What good is a cure if our people can’t afford them?” he said.

Wyden is the ranking Democrat on the Senate Finance Committee, with jurisdiction over more than a trillion dollars in national health care spending.

He thinks drug companies need to be more transparent about how much of their money goes into research and how much goes to profits and marketing. Wyden thinks insurance companies also need to step up to make sure drug prices are reasonable.

But he said there are things the government can do too.

“I would be in favor of say Medicare stepping in and negotiating to make sure that we strike the right balance between having access to cures and ensuring that they’re affordable,” Wyden said.

Back at the OHSU Knight Cancer Institute, Maziarz knows $373,000 is a lot for one injection.

But he thinks it needs to be seen in context. Lymphoma is expensive to fight. All the costs associated with a bone marrow transplant can drive the cost higher than $400,000, and there's still a risk of relapse.

“If ultimately this single therapy can eliminate the need for multiple, months of chemotherapy, or other type of therapy. If it can remove the ability to need a transplant and the complications of transplant, it may actually prove to ... have a value assessment,” Maziarz said.

Kymriah was developed by the University of Pennsylvania and the OHSU Knight Cancer Institute was an early adopter of the technology. Maziarz was one of the key leaders in the first global study examining a CART-T therapy for people with this kind of lymphoma.

Norvartis issued a statement saying, in considering how to price Kymriah, it looked at the value it provides patients, the cost of current standards of care, and the costs for production.

"We set the large B-cell lymphoma indication price at $373,000," the statement reads. "This is well below the value Kymriah delivers to patients and the broader healthcare system based on cost effectiveness modeling and significantly less than other approaches such as allogeneic stem cell transplant, which can cost upwards of $455,000 in the first year after treatment." [Copyright 2019 Oregon Public Broadcasting]