A “revolutionary” treatment”, as called by Dr. Prakash Satwani, a pediatric hematologist at New York-Presbyterian/Columbia University Medical Center, has just been approved by the U.S. Food and Drug Administration (FDA) for treatment of leukemia. This new medication, called CTL019 is considered a breakthrough therapy for leukemia.

Survival rates for leukemia patient vary depending on the type of leukemia a patient is diagnosed with. The prognosis is unfavorable overall and treatment options most often consist of toxic treatments like chemotherapy and radiation. CTL019 is recommended for the treatment of relapsed or refractory pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL). Novartis, the company that developed CTL019, is now poised to be the first to develop a treatment of this kind and is working on similar types of treatments in hundreds of patients with another form of the disease, as well as multiple myeloma and aggressive brain tumors.

How Does It Work?

This new form of treatment uses the patient’s own immune system to help fight the disease. The treatment requires removing millions of a patient’s T-cells, a type of white blood cell often called soldiers of the immune system, and genetically modifying them to kill the patient’s cancer cells. The treatment uses a disabled form of H.I.V. to carry new genetic material into the T-cells and consequently reprograms them. The process causes the T-cells to attack B-cells, a normal part of the immune system that turn malignant in leukemia. The T-cells seek a protein called CD-19 that is found on the surface of most B-cells. The now altered T-cells are then put back into the patient’s veins, where they are able to multiply and start fighting the cancer cells. It is estimated that a single treatment can kill 100,000 cancer cells.

Price Concerns

Leonard Saltz, chief of gastrointestinal oncology at Memorial Sloan Kettering Cancer Center in New York, thinks that this treatment will be a significantly more expensive cancer medication, compared to current therapies. British authorities have said a price just shy of $650,000 would be justified given the significant benefits from just a single treatment. Others have predicted a cost of around $300,000 for a single treatment. Norartis has declined to give a price at this point. Given this estimate and the recent history of pharmaceutical companies, much fear remains over the affordability of the treatment. The patient advocacy group names Patient’s for Affordable Drugs has already asked for Novartis to meet with them to discuss a fair price. Much of the hope for this medication is coupled with significant fear over its affordability. David Mitchell, the president of Patient’s for Affordable Drugs, notes that “drugs don’t work if patients can’t afford them.”

Current cancer therapies easily cost $100,000 and combination therapies can cost around $250,000. These types of prices seem outrageous given the fact that much of the research that helped to find these therapies was provided through taxpayer dollars, notes Mitchell. Unfortunately, there are more costs associated with chemotherapy and radiation such as lengthy hospital stays, hotel expenses for families, and medication expenses for drugs to reduce side effects from treatment. It is too early to tell how CTL019 will compare – the hope is for fewer side effects and shorts stays at the hospital compared to traditional treatment options.

Clinical Trials

Emily Whitehead, age 12, is walking proof of the effectiveness of CTL019. Diagnosed at age 6 with leukemia, Emily was the first patient to try this treatment and it nearly killed her. After coming through the treatment, Emily was cancer free and continues to be. Conor, another patient who received the treatment battled leukemia for 12 years. After treatment, Conor was able to get back to playing hockey. His dad, an advocate for CTL019, noted the grueling effects that standard treatment had on his son leaving him infertile from the chemotherapy. The pain of the bone marrow transplants and countless spinal taps leave a painful reminder of how harsh traditional treatments were. Conor and his dad note that the CTL019 treatment was far better tolerated by Conor.

The main evidence presented to the FDA by Novartis was a study consisting of 63 patients given CTL019 for treatment of leukemia. 52 of the patients or 82.5 percent of them went into remission – this percentage is considered extremely high for such a potent disease. Amy Kappen’s daughter, Sophia, died at age 5 of leukemia after receiving CTL019. However, Amy notes how well Sophia’s symptoms were relieved by treatment and how it added extra months to her life. Amy believes that if treatment had started sooner, Sophia might have survived.

CTL019 is the first treatment of its kind and holds much promise for leukemia patients and patients with various other types of cancer. With further research and learning this treatment could prove to shape the landscape for cancer treatments both now and in the future.