SAU Buzz

Cancer Treatment Breakthrough

by Jessica Lewis
Posted on Sep 21, 2017

On August 30th, the FDA announced that the first cell-based gene therapy drug in the United States had been approved for use. This means that cells are reprogrammed based on the patient’s genetics and sent to attack cancer cells The medical field is buzzing with excitement at the doors that this drug, Kymriah, has opened.

“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,” FDA commissioner Scott Gottlieb said.

Currently, pharmaceutical companies are working to develop treatments similar to Kymriah that do not have to be personalized to the patient. Treatments like those in development would allow more people to be treated each year and make the treatments safer for use in patients.

Kymriah works by the extraction of T-cells, an important piece of the immune system, and modifying those T-cells to attack leukemia cells specifically. T-cells are responsible for attacking bacteria, viruses, and foreign tissues, and producing cytokines that direct responses and activities in other immune cells. This can also be called CAR-T cell therapy.

The drug is currently approved to treat children and young adults up to 25 with acute lymphoblastic leukemia. This cancer is the most common childhood cancer, with over three thousand patients under twenty years old diagnosed each year. With this new therapy, it is estimated that only 600 patients can be treated each year. More patients may be able to receive treatments in the future.

According to Business Insider, out of those tested, the treatment was able to provide remission after three months to 83% of patients and 64% were still in remission after a year. All patients had originally been unresponsive to other treatments or had suffered relapses with their prior treatments.

The Kymriah therapy does have many risks. For example, the treatment can cause an extreme overreaction of the immune system and other side effects that may be deadly. The treatment is only recommended with great caution.

The treatment is a one-time procedure, with a controversial cost of $475,000 if the drug works. David Mitchel, the founder and president of Patients for Affordable Drugs, weighed in on this saying, “while Novartis’ [the pharmaceutical developer] decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive.” Although the cost is high, Kymriah Therapy raises hopes that we are on the way to improved cancer treatments.