In August 2017, the treatment of childhood leukemia saw a new breakthrough. The U.S. health officials approved a treatment that could genetically engineer patients with childhood leukemia. The U.S. Food and Drug Administration called this approval as a historic one because this is the first gene therapy to get into the U.S. market. This treatment is the result of joint efforts taken by the “University of Pennsylvania” and the pharma giant “Novartis”.

An Introduction to CAR-T Cell Therapy

The infusion that is going to be in the U.S. market is called “CAR-T cells”. This, which can be expanded as “Chimeric Antigen Receptor T”, would leverage the patients’ own immune systems to fight cancers. Thus, this therapy makes use of gene therapy techniques not to really fix the disease-causing genes. But, the idea here is to turbocharge the patients’ T cells that will fight against cancers.

In this line, the researchers have filtered those cells from the blood of the patient and reprogram it to harbor a receptor called “chimeric antigen receptor (CAR)”. This antigen would zero-in on cancer cells and will kill them. Thus, this can be seen as a form of immunotherapy that is growing in the field of cancer and treatment today. However, this technique is found to be harnessing the patient’s immune system in a completely different way than the popular immunotherapy drugs.

Information on Therapy Approval

The first CAR-T version that has been developed by Novartis along with the University of Pennsylvania was approved for use by hundreds of patients every year. In other terms, it can be said that it can be used by the patients who are desperately affected by acute lymphoblastic leukemia. A statistical report shows that childhood leukemia strikes over three thousand children, as well as young adults in the country every year. While most of these children survive, around 15% of the cases relapse in spite of giving the best treatments.

Evidence for the Effectiveness of CAR-T Cell Therapy

In one of the major studies that involved the CAR-T cell therapy, it has been shown that 63 advanced patients, 83% went into remission immediately after receiving the CAR-T cells. However, it’s to be noted that it’s not clear how long these benefits would last. It’s because some patients were found to relapse months after the treatment, while others are being tracked to see how long they fare.

In this line, Dr. Ted Laetsch, University of Texas said that a far higher percentage of patients went into remission with this CAR-T therapy when compared to all other treatments that are in use till date.

Are There Any Side Effects Involved?

The answer to this question will be an obvious “YES”. Most of the patients who received the treatment suffered side effects that are grueling or even life-threatening. This treatment, which can lead to an immune overreaction called “cytokine release syndrome”, could stimulate the effects like high fevers, decreasing blood pressures, and in some cases, severe organ damages. It’s to be noted here that these are the side effects that need sophisticated care in order to help the patients when at the same time keep the cancer treatment going. However, the good news is that the FDA has designated a treatment for these side effects last week.

Cost of the CAR-T Therapy

The pharma giant, Novartis has set the cost of its one-time infusion of CAR-T cells at $475,000. However, the company also said there it would not charge anything for the patients who don’t show any response within one month of infusion.

The process would involve the collection of immune cells from the patients, which will be frozen thereafter and sent to a Novartis factory in NJ. Here, each dose will be created, which will take about three weeks. Here, it’s to be noted that the cost that is stated above doesn’t include the costs that are needed for hospitalizations, travel to certified hospitals, and other expenses.

Yes, the costs are found to be huge for now. For this, the Novartis executives said that they are working closely with the Medicaid program, as well as the private insurers and expects some broader coverage. They also said that they are looking to offer some financial assistance with the other things like travel expenses. But, the company didn’t give any assuring word that all the patients with childhood leukemia will be able to get this therapy.

For some patients, this new therapy with CAR-T therapy might even replace the bone marrow transplants that are priced at more than half a million dollars today. Thus, if this is the last treatment a patient needs, that’s going to be a one-time investment for the patients’ wellness, especially for the kids who have their entire life ahead of them.

First Treated Case

The first child treated with CAR-T cell therapy is a girl named “Emily Whitehead”, who had been near her death. With this treatment, she has been free of cancer for half a decade now.