Cancer breakthrough drug approved by FDA

September 5, 2017

In a historic moment, the US Food and Drug Administration (FDA) has approved the first treatment that engineers a cancer patient’s own immune system to treat the disease. According to BBC News, the multinational pharmaceutical company Novartis will charge $475,000 for this breakthrough “living drug” therapy called Kymriah, which works against acute lymphoblastic leukemia, a type of blood cancer.

 

Tailor-made for each patient unlike other treatments like chemotherapy or radiotherapy, Kymriah is what is called a CAR T-cell therapy: white blood cells are extracted from the patient's blood, then are genetically reprogrammed to seek out and kill cancer. When reintroduced in the body, the cells find their target and multiply. This new approach to fighting cancer is a very exciting “because of the remarkable responses they have produced in some patients—both children and adults—for whom all other treatments had stopped working”, according to the National Cancer Institute (NIH) website. Out of 63 patients treated with CAR-T therapy, 83% were in complete remission within three months.

 

Dr Scott Gottlieb from the FDA stated, “We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer. 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 therapy can cause potentially life-threatening cytokine release syndrome from the rapid proliferation of the CAR-T cells in the body. This, however, can be controlled with drugs.

 

The potential of CAR-T technology goes beyond one type of cancer. Dr. David Maloney, medical director of cellular immunotherapy at the Fred Hutchinson Cancer Research Center, said the FDA's decision was a "milestone," and believes that this is just the first of what will soon be many new immunotherapy-based treatments for a variety of cancers. While it has struggled against "solid tumors" such as lung cancer or melanoma, the CAR-T treatment has shown most promise against different blood-based cancers. In the near future, however, this early stage-technology is expected to get better, as boosting the immune system is already a cornerstone of modern cancer treatment.

 

Professor Peter Johnson, chief clinician at the charity Cancer Research UK, declared, "The first genetically modified cell therapy to be approved by the FDA is an exciting step forward. We still have a lot to learn about how to use it safely and who might benefit from it, so it is important to recognize this is just a first step."

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