Millions of Americans get some form of cancer. The cost of treating and fighting cancer reaches into the billions of dollars. One source lists the cost of cancer care in 2010 at $157 billion. Imagine how much could be saved if researchers could find a successful way to fight cancer that didn’t require traditional chemotherapy or radiation therapy? Millions of lives could be saved and total healthcare costs in the United States could be lowered enough to almost make a national healthcare system affordable – ALMOST.

Researchers working for Novartis, a large drug company may have made what some are calling the most major breakthrough in cancer treatment in decades. Dr. David Lebwohl heads up the CAR-T Franchise Global Program that developed the breakthrough technology.

The process has been named CAR-T cell immunotherapy and the idea is brilliant. Important immune T cells are removed from a patient. The scientists then genetically modified them so that they would target and attack just cancer cells. Once the T cells have been genetically modified, they are injected back into the patient where they travel through the blood stream to the cancer and attack it. Using the patient’s own T cells to fight cancer eliminates many of the harsh and debilitating side-effects of most chemotherapies and/or radiation therapies.

Many cancer patients will tell you that many of the treatments they undergo are almost worse than the cancer itself. The nausea, weakness, headaches, aches and pains that go with most treatments, often leave cancer patients unable to function. They end up spending hours, days and even weeks feeling sicker than a dog before they feel better.

Dr. Lebwohl commented about the new CAR-T cell immunotherapy, saying:

“It’s truly a paradigm shift. It represents a new hope for patients.”

The FDA Advisory Panel has endorsed the use of the new immunotherapy, which was initially developed to treat children and young adults with B cell acute lymphoblastic leukemia, that either doesn’t respond to standard treatment of they have relapsed. This form of cancer is the most common cancer that affects children in the United States.

With standard treatment, many children become cancer free, but at some time down the road may suffer a relapse. Others don’t respond to the standard treatment or cannot tolerate it. When this form of leukemia relapses, quite often the standard treatment is not nearly as successful.

The initial study for the new treatment was conducted at 25 different locations on 88 patients in 11 different countries. The patients ran in age from 3 to 23 and all had experienced a relapse that did not respond to standard treatment or they failed to respond to standard treatment to begin with. The drug developed to help genetically modify the patient’s T cells is known as tisagenlecleucel or CTL019, or the ‘living drug’. In the studies, 83% of the patients that received CTL019 saw their leukemia go into total remission.

Dr. Stephen Hunger, a doctor at Children’s Hospital in Philadelphia assisted in the study on the new immunotherapy. He commented about the need for such a treatment, saying:

“There is a major unmet medical need for treatment options.” 

Other drug companies and researchers have tried to use similar forms of immunotherapy, but ran into a serious side-effect known as cytokine release syndrome. This is when the genetically modified T cells begin attacking some of the patient’s organs. In some cases, they attacked the brains, causing severe brain swelling and death.

So far in the study using CTL019, only a few patients developed the side effects, but none of those cases were fatal and all of the patients recovered. Dr. Timothy Cripe, an oncologist at the Nationwide Children’s Hospital in Columbus, Ohio commented about the drug, saying:

“This is the most exciting thing I’ve seen in my lifetime.”

Dr. Malcolm Smith, associate branch chief for the pediatric oncology at the Nation Cancer Institute, also commented about the study, saying:

“This is a major advance and is ushering in a new era in treating children.”

Since the researchers use a virus to help genetically modify the T cells, no one is sure if there are any long-term effects.

Getting the endorsement of the Advisory Panel, which voted 10-0 to endorse the new drug and treatment, is one of the first steps towards approval of the drug and treatment process. The FDA does not have to always take the endorsement of the Advisory Panel, but doctors and families of patients involved in the study are lobbying for approval.

While the new living drug, if approved, could save billions of dollars in cancer treatment, it’s probably not going to be cheap for patients to get. Novartis has not said how much the drug treatment will cost, but some analysts in the industry are estimating it could cost as much as $500,000 per infusion of the genetically modified T cells. Hopefully, it won’t cost nearly this much and is made readily available to the thousands of kids suffering from this form of leukemia.