FDA Approval of the Second Living CAR-T Cancer Therapy Sparks Excitement

With advances in genetic techniques, genetically based treatments targeting cancer more effectively have begun to be used clinically. A development concerning CAR-T cell therapies, the leading approach in this field, came from the United States Food and Drug Administration, the FDA. FDA, on 18 October 2017, approved the CAR-T treatment Yescarta for use in diffuse B-cell lymphoma. Yescarta (axicabtagene ciloleucel) is the second CAR-T treatment drug to receive approval.

For Which Cancer Will Yescarta Be Used?

It is approved for adults diagnosed with diffuse large B-cell lymphoma. The treatment was considered appropriate for patients who had received two previous lines of treatment without responding and whose disease had relapsed.

Diffuse large B-cell lymphomais the most common subtype of non-Hodgkin lymphoma in adults. Non-Hodgkin lymphomas, cancers of immune cells called lymphocytes, may progress rapidly (aggressively) or slowly. In our country, approximately 5400 new lymphoma diagnoses are made every year, and one in every 3 lymphoma diagnoses is diffuse large B-cell lymphoma.

Which Clinical Trials Led to Yescarta’s Approval?

Treatment effectiveness and safety were evaluated in a multicenter study involving 100 patients, a clinical trial . A complete response was observed after treatment in 51 percent of patients.

What Are Yescarta’s Side Effects?

Yescarta can potentially cause serious side effects. One of the most important is cytokine release syndrome. Proliferation and activation of T lymphocytes modified through chimeric antigen receptor treatment (CAR-T) can cause excessive cytokine release. This can produce high fever, flu-like symptoms and neurological toxicities. When uncontrolled, cytokine release syndrome leads to life-threatening conditions. Tocilizumab (Actemra) is used against this type of side effect.

Other side effects include reduced blood cell counts, a decreased immune response and serious infections. Side effects generally occur during the first 2 weeks of treatment.

What Does This Development Mean for Cancer?

• We are living through one of the most exciting periods in the history of cancer research. We have begun using drugs and treatments prepared against cancer through “modern genetic techniques.”
• CAR-T cell therapy, in which the immune system is programmed to find and destroy cancer and which is therefore a form of cancer immunotherapy , is regarded by many scientific authorities as one of the most important developments of the century.
• Because CAR-T cell therapy is a high-technology treatment carrying significant risks, it can be performed in specially certified hospitals and clinics. Healthcare personnel delivering the treatment must be trained and certified.
• Yescarta follows Kymriah as the second approved CAR-T cell therapy. Yescarta is the first CAR-T cell therapy approved for non-Hodgkin lymphoma.
• CAR-T cell therapies are developed using advanced genetic engineering technology. The treatment is therefore very expensive. The cost for a single patient is 373.000 dollars. Indeed, whether treatment costs overshadow its success, and how the financial problem can be overcome, are currently being intensely debated.

It is important to emphasize that modern medicine uses drugs and treatments developed through years of careful research. Cancer arises from numerous genetic and epigenetic abnormalities. Modern medicine seeks the most appropriate treatment with support from genetic and biochemical analyses. Cancer treatment is possible only through modern medicine.