It's the first type of gene therapy to hit the US market. 5 years ago, for the first time, he used the CAR-T cell therapy to treat a girl child who was close to death but now she is completely ok.
Once transfused back into the patient, the immune cells then attack leukemia cells for months or even years.
The Kymriah is intended for children and young adults up to 25 who have resisted other therapies against this leukemia or relapse, which occurs in 15% to 20% of cases.
Novartis AG on Wednesday won highly anticipated U.S. approval for the first of a new type of potent gene-modifying immunotherapy for leukemia, a $475,000 treatment that marks the start of a potential new treatment paradigm for some cancers. Priced at $475,000, it will be offered at just 32 sites around the country.
"In the next few years", he said, "I think we're going to see dramatic progress and push the boundaries of what many people thought was possible with these adoptive cell transfer-based treatments". That company picked up CAR-T technology in its Monday acquisition of Kite Pharma for $11.9 billion.
Novartis' price tag for the treatment was met with criticism from David Mitchell, Founder and President of Patients For Affordable Drugs. In June, a clinical trial in China showed promise in treating another blood cancer, multiple myeloma, using reprogrammed T-cells in much the same way Kymriah does.
"The FDA is requiring that hospitals and their associated clinics that dispense Kymriah be specially certified", it added.
Prior to the FDA approval, outside estimates for what Novartis' CAR T-cell therapy might cost ranged as high as $700,000. At the one year mark, most patients would be relapse-free.
Kymriah works through a process of collecting a patient's cells - specifically T cells that work as part of the immune system - and genetically modifying them to recognize and attack cancer cells. The company announced that Kymriah would ring in at $475,000 for a treatment course (and that's actually a bit less expensive than what many analysts had expected).
The therapy would cost $475,000 (400,000 euros) per dose, but Novartis said anyone who didn't respond within a month would not have to pay.
The therapy does have side effect for which the FDA has created a treatment.
As with any drug, there are potential side effects, including cytokine release syndrome (CRS) and "neurological events", both severe and possibly life-threatening.
An Australian representative for Novartis Pharmaceuticals, which sponsored the US-approved treatment, would not comment on whether it planned to seek approval from the TGA to make the treatment commercially available in Australia.
Other genetic therapies for cancer are also in the research pipeline.