The U.S. Food and Drug Administration (FDA) recently announced the approval of the first gene therapy that will be available in the United States. According to the agency’s press release, they approved Novartis Pharmaceutical’s Kymriah (tisagenlecleucel) as a treatment for B-cell Acute Lymphoblastic Leukemia.

According to FDA Commissioner Scott Gottlieb, the approval of the gene therapy is part of the agency’s commitment to “expedite the development and review of groundbreaking treatments” that could help save lives. Due to modern technologies and the latest medical innovations, the FDA stressed that they are boosting their efforts to initiate a new approach when it comes to treating cancer and other life-threatening illnesses.

Acute Lymphoblastic or Lymphocytic Leukemia (ALL)

Considered the most common and progressive form of childhood cancer in the United States, ALL is a type of cancer that begins from the primogenial stage of lymphocytes or white blood cells in the bone marrow, where new blood cells are produced. It is caused by the overproduction or having too many immature lymphocytes such as lymphoblasts, T or B lymphocytes.

ALL is both a cancer of the blood and the bone marrow, and if not treated immediately, it would be fatal.

As per the National Cancer Institute, these immature lymphocytes are considered cancer cells that do not function like the normal ones, hence, they aren’t able to fight infection.

Furthermore, the rise of these immature or cancer cells in the blood and bone marrow decreases the opportunity for the production of healthy white blood cells, red blood cells, and platelets.

This could result in easy bleeding, infection, and anemia.

Kymriah

Fortunately, a cell-based gene therapy is finally available in the United States. This one-time “CAR-T cells” infusion is intended for pediatric and young adult patients up to 25-years-old who have B-cell precursor ALL, which is “refractory or in second/later relapse.”

Even though the Kymriah treatment comes with a steep price of $475,000, Novartis Pharmaceuticals stressed that it would be free for patients who are unresponsive to the treatment within a month, ABC News revealed.

But each dose of this genetically-modified autologous T-cell immunotherapy treatment is customized with the patient’s own T-cells, along with a new gene that contains the specific CAR (chimeric antigen receptor) protein.

This CAR protein will regulate the T-cells to attack and destroy leukemia or cancer cells with the specific CD19 antigen on the surface. This therapy is reportedly a new approach to treat ALL by giving patients stronger T-cells that will spot and kill the cancer cells.

Novartis to provide hospitals with Kymriah

Despite the expensive cost for the one-time Kymriah treatment, Novartis stressed the significant benefits of the treatment are worth the price. The officials of the Switzerland-based drug company added that they will be collaborating with 20 hospitals to provide the treatment within a month, however, the company revealed that it will be offered to 32 sites.

In addition, the company revealed that they are “carefully training” the hospitals and its staff to provide the gene therapy, which can cause long-term complications and fatal immune reactions, USA Today reported. Meanwhile, advocacy group Patients for Affordable Drugs have met with Novartis officials to discuss setting a “fair price” for the treatment.
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