the Food & Drug Administration campus in Silver Spring

FDA Approves Groundbreaking Cancer Treatment With A Bank-Breaking Price Tag

In a landmark achievement for the much-maligned pharmaceutical industry, the American Food and Drug Administration (FDA) has given the green light to begin dispensing a revolutionary new cancer treatment that turns the body’s immune system into a cancerous-cell-fighting superhero.

Novartis, a Swiss big-pharma corporation, has developed the first-ever gene therapy that causes a cancer sufferer’s own antibodies to attack malignant cells aggressively. So far, the technology is being specially employed in the treatment of acute lymphoblastic leukemia.

The gene therapy was originally called CLT019 as it was being developed but has since been renamed as Kymriah following the FDA approval. For young people fighting a particularly pugnacious type of leukemia, Kymriah is a name that will bring them hope.

Some biological therapies — also known as immunotherapies — are already being used to treat cancer. Monoclonal antibodies (MAbs), for example, are produced in laboratories using mice as incubators for humans cells containing the appropriate antigens.

Once they are ready, biomedical engineers harvest the cells — now able to produce the necessary antibodies — and fuse them with a myeloma cell, also known as a cancerous B cell, to form a monoclonal antibody.

In simple terms, a MAb is merely a custom-built antibody used by medical doctors and scientists to produce brand new ways to combat specific diseases. Until recently, the only FDA-approved MAb used to treat leukemia was called alemtuzumab.

What makes Kymriah so unique is that it’s a chimeric antigen receptor T cell (CAR-T) therapy. Instead of using generic human cells that have been modified and incubated in mice, the CAR-T method uses each patient’s own cells.

The treatment works by modifying a patient's own immune cells to make them more effective at hunting down and killing tumor cells
CAR-T cell therapies are manufactured by extracting a patient’s T cells to reprogram them to recognize and fight cancer cells. [Infographic by Novartis]

Oncologists collect blood samples from a patient that will, in turn, be sent to a biomedical facility where scientists can extract the patient’s T cells and manually reprogram them. Once the process is completed, the patient’s very own cells will be able to express the chimeric antigen receptor that is necessary to recognize, attack, and kill cancer cells.

In the United States, acute lymphoblastic leukemia (ALL) accounts for roughly 25 percent of cancer diagnoses among pre-adolescent children, making it the most common pediatric cancer. Until now, patients diagnosed with ALL have not had many treatment options.

However, despite the arrival of this groundbreaking therapy, the process will only be available in a select number of medical facilities in the U.S. Due to the highly personalized nature of the procedure, it takes nearly a month to complete.

But that’s not all that is standing in the way of broad access to the drug. Kymriah does not come cheap. In fact, at this stage, most Americans won’t be able to afford the treatment without the help of subsidies

Novartis announced that it would be charging up to $475,000 per treatment. Although this figure is less than what experts had anticipated, it’s still much more than what the average Joe and Jane Soap can afford.

The big question, then, is whether the benefit to the patient will justify the price tag.

Bill Hinshaw, the head of oncology at Novartis U.S., was quick to point out that independent cost analysis has shown that the company could charge hundreds of thousands of dollars more. Because most patients will be children, Hinshaw believes that Medicaid would cover most of the bill while government subsidies could augment the rest.

Furthermore, Hinshaw says that Novartis will only accept payment if patients have responded to Kymriah within the first month of treatment. The pharmaceutical giant has also pledged to offer additional financial assistance to help families with travel and accommodation expenses during treatment.

However, according to the Wall Street Journal, Novartis has come under fire for the price of their new drug in spite of their offers to assist sufferers financially. David Mitchell, of the Patients for Affordable Drugs campaign, believes the cost is unreasonable.

“While Novartis’ decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive. Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more.”

Kymriah comes with a few caveats, including severe potential side effects such as dangerous drops in blood pressure. Therefore, the FDA has insisted that Novartis provides strategic training to doctors who will be involved in the treatment program. Hospitals have also been instructed to stock a particular drug that can counter the serious side effects.

Nevertheless, Novartis is confident about the future of Kymriah in light of the FDA’s Oncologic Drugs Advisory Committee’s (ODAC) approval of their pioneering CAR-T therapy.

Kymriah was born at the University of Pennsylvania, where the chimeric antigen receptor — then still known as CTL019 — was first used to enhance cellular responses after it is infused into the patient. In 2012, Novartis approached Penn University to enter into a large-scale research partnership.

The next step will include additional applications filed by Novartis to expand the Kymriah treatment plan to the European Union, as well as to broaden the scope of the drug to include the treatment of adults with diffuse large B-cell lymphoma (DLBCL).

Moreover, even though Kymriah is most effective in blasting blood cancers, Novartis is currently testing CAR-T technologies in the treatment of lung and brain cancer.

The therapy holds out the promise of a significant advance in the treatment of children and young adults with a form of blood cancer known as B-cell acute lymphoblastic leukemia
The treatment works by modifying a patient’s own immune cells to make them more efficient at hunting down and killing tumor cells. [Infographic by Novartis]

Acute lymphoblastic leukemia is a malignant form of cancer that is found in bone marrow and blood. About 3,100 children and young adults are diagnosed with ALL each year in the United States. An estimated 600 patients don’t respond to traditional treatments and will thus be eligible for the Kymriah program.

[Featured Image by Andrew Harnik/AP Images]