Less than two months after agreeing to buy Israeli company, Kate Pharma for $11.9 billion in cash, Pharma giant Gilead is making a breakthrough in the anti-cancer drug market.
The U.S. Food and Drug Administration today approved Yescarta (axicabtagene ciloleucel), a drug that mobilizes the immune system to identify and destroy cancer cells.
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YESCARTA developed on the basis of the knowledge of Prof. Zelig Ashchar, Prof. Gideon Gross and Dr. Daniel Schindler of the Weizmann Institute and Tova Wax, their laboratory manager.
The drug is intended for patients aged 18 years with certain types of large B-cell lymphoma whose disease who have not responded to or who have relapsed after at least two other kinds of treatment.
YESCARTA is the second approved drug based on the breakthrough technology CAR-T, which deals with the difficulty of the immune system to identify and attack the cancer cell. It is also the first drug approved for certain types of non-Hodgkin lymphoma (NHL).
“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases. In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer,” said FDA Commissioner Scott Gottlieb, M.D. “This approval demonstrates the continued momentum of this promising new area of medicine and we’re committed to supporting and helping expedite the development of these products. We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine. That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies. We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”
The difficulty in this method is that a cancerous cell, unlike a virus, for example, is a cell of the body itself and has mechanisms to evade the immune system.
The technology developed by Buckthorn together with Dr. Stephen Rosenberg of the National Cancer Institute (NCI) is based on cells of the immune system undergoing special “training” through genetic engineering, and as a result, are equipped with a unique combination of navigation and combat means that increases their ability to identify and kill the cancer cell metastases.
This is a combination of the two main tools used by the immune system in its fight against foreign invaders and pathogens that penetrate the body.
The first is the antibodies that identify specific molecules that are shown on the outer membranes of cells infected with viruses, original tumor cells, cancer cells traveling in the bloodstream on the way to metastases, as well as disease-causing bacteria.
The other is white T-blood cells, which can kill cells that are identified as “enemies” but in fact they are able to identify as an enemy only a minority of cancerous tumors.
In other words, the antibodies excel in identifying the enemy but find it hard to kill him, while the T cells excel in killing enemy cells, but have difficulty identifying.
Buckthorn found a way to combine these two abilities. To do so he created – in methods of genetic engineering – a special receptor that is shown on the membranes of T cells and has a similar recognition ability as an antibody.
Thus, in fact, he was able to create fighting cells that could effectively identify the cancer cells (original or grafted) and kill them.
Approximately 72,000 new cases of NHL are diagnosed in the U.S. each year, and DLBCL represents approximately one in three newly diagnosed cases.
The price of YESCARTA in the US is $373,000.
Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma.
Each dose of Yescarta is a customized treatment created using a patient’s own immune system to help fight the lymphoma. The patient’s T-cells, a type of white blood cell, are collected and genetically modified to include a new gene that targets and kills the lymphoma cells. Once the cells are modified, they are infused back into the patient.
“The approval of Yescarta brings this innovative class of CAR-T cell therapies to an additional group of cancer patients with few other options – those adults with certain types of lymphoma that have not responded to previous treatments,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER).
The safety and efficacy of Yescarta were established in a multicenter clinical trial of more than 100 adults with refractory or relapsed large B-cell lymphoma. The complete remission rate after treatment with Yescarta was 51 percent.
Treatment with Yescarta has the potential to cause severe side effects. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR-T cells causing high fever and flu-like symptoms, and for neurologic toxicities. Both CRS and neurologic toxicities can be fatal or life-threatening. Other side effects include serious infections, low blood cell counts and a weakened immune system. Side effects from treatment with Yescarta usually appear within the first one to two weeks, but some side effects may occur later.
To further evaluate the long-term safety, the FDA is also requiring the manufacturer to conduct a post-marketing observational study involving patients treated with Yescarta.
The FDA granted Yescarta Priority Review and Breakthrough Therapy designations. Yescarta also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
