Will you buy a new drug/treatment that costs US $475,000 with potentially fatal side effects?

Yet that is what The Food and Drug Administration (FDA) approved not more than 3 months ago when they gave the go-signal to pharmaceutical drug giant Novartis to market Kymriah, the new gene-altering therapy drug for leukemia.

Kymriah is the first-ever treatment drug to genetically change a cancer patient’s genes to fight cancer. This kind of treatment will allegedly start a new revolutionary trend in cancer treatment for the coming years.

This new therapy technically called “gene therapy”, is part of the growing study and practice of immunotherapy. Immunotherapy uses the natural immune system of the body along with drugs or other alternative therapies in order to fight cancer and other life-threatening diseases. With Kymriah, a patient’s cells become the drug itself, training them to recognize, attack, and exterminate the attacking disease.

Kymriah was developed and approved for children and young adults in fighting B-cell acute lymphoblastic leukemia, a deadly type of leukemia that has been largely resistant to a lot of treatments or prone to patient relapse.

Novartis, like all other big pharma companies, have been in a race against time to develop gene therapies for a host of cancers and other diseases. Currently, more than 550 gene therapies are being studied for approval, says the FDA.

A Cancer Cure at the Cost of What?

If you’re thinking that U.S.-based B-cell acute lymphoblastic leukemia patients are so fortunate to have Kymriah launched in their midst, think again! This gene-altering drug has its downsides.

Firstly, it poses life-threatening side effects ranging from deep dives in blood pressure, lung congestion, high fever, neurological problems, and other potentially fatal complications.

That is why medical centers and medical staff administering this drug need to be trained and certified to do so, at least in the U.S.

Secondly, the prohibitive cost of the drug (US$475,000) is a deterrent to people who don’t have the resources, nor the medical insurance, to afford such a drug. Yes, Novartis said they will provide monetary help to families who are uninsured or underinsured. I wonder if we can do the same thing here in the Philippines?

Novartis said if the patient did not respond favorably within the first month of treatment, they will not charge the patient for the treatment.

Initial beneficiaries of this drug are about 600 potential young adults and children in the US in one year.

A Bit of History & Administering Kymriah

This kind of gene therapy (formerly identified as CAR-T cell therapy, CTL019, OR tisagenlecleucel), was originally developed by University of Pennsylvania scientists who licensed it to Novartis.

To be able to tailor Kymriah for the needs of individual patients, a person’s white blood cells called T cells are removed from a patient’s bloodstream at a certified medical center, frozen and shipped to Novartis HQ for genetic engineering and replication. After the process, the T cells are frozen again and sent back to the Medical Center they come from where it is intravenously dripped into the patient. It takes 22 days to complete the process.

Novartis said Kymriah and its accompanying treatment will be made available to an initial network of 20 certified medical centers in the U.S., and will be expanded to 32 by end-year.

We at the Dr. Farrah Agustin-Bunch Natural Medical Center is against the use of ANY FORM of synthetic drug in the treatment of cancer and other debilitating, chronic diseases. However, we respect the patient’s choice in terms of the treatment they will decide to use. So, we advise patients to practice “discernment” when choosing the right kind of treatment so that you can heal or prolong your life at least longer than the usual maximum 5 years life expectancy for cancer victims.

We also believe that if you are paying almost half a million dollars to prolong your life, then better choose a treatment that you will be wholeheartedly happy about when executed.

So, what are your opinions about this super expensive, gene-altering therapy for a rare leukemia disease? Share your opinions in the comments section below.

Image by Thor_Deichmann / CC0 1.0