Groundbreaking Leukemia Gene Therapy Carries $475k Price Tag

The FDA has just approved Kymriah, a new form of genetically modified T-cell immunotherapy for treating young patients with a type of blood and bone marrow cancer.

The Food and Drug Association has approved the first cell-based gene therapy in the United States, opening the door for therapy that will attack and destroy cancer cells.

"While both external and Novartis' quantitative assessments of these values indicate that a cost-effective price could be $600,000 to $750,000, we recognize the importance of this paradigm-shifting therapy and are setting the price at $475,000 for this one-time treatment", Dana Cooper, a spokesman for Novartis, said in an interview with OncLive. The FDA approval of this therapy is based on the results of the Phase II ELIANA trial, which was sponsored by Novartis and included 25 centres in the US, EU, Canada, Australia and Japan.

The treatment, called Kymriah, aims to give some patients a second chance after first-line drugs have failed.

The treatment is made from scratch for every patient and costs $475,000 but would be no charge if the patient didn't show a response within a month. Treatment with tisagenlecleucel has the potential to cause severe adverse effects.


The modified cells are then transfused back into the patient. In a key clinical trial of 63 people, 83 per cent of participants were cancer-free after three months.

Cooper said fewer inpatient and outpatient visits for treatment and fewer hospitalizations are likely to result from treatment with vehicle T-cell therapy, which he said would allow parents and other family members and caregivers "to resume a normal life, including the possibility to return to work".

Epstein, the former CEO of Novartis' pharmaceuticals division, left in 2016 and now serves as executive chairman at Rubius Therapeutics, a biotech that's also working with cell therapy to develop treatments like Kymriah that don't have to be as personalized.

In CAR-T cell treatment the Patients own blood cells or immune cells are filtered out and sent to lab where they get reprogrammed into a CAR (chimeric antigen receptor) which makes the T cells super active or turbocharge and after that the T cells are again infused into the body so that they could combat with the cancerous cells more efficiently. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of auto T-cells causing high fever and flu-like symptoms, and for neurological events.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells. "The established path to commercialization taken by Novartis will provide considerable motivation for cell therapy scientists and development companies as well as investors looking to benefit from the ongoing success in the industry".


Epstein said he envisioned cell therapies having much shorter life cycles than traditional drugs.

For one, the treatments will have to treat more types of cancer than the one approved on Wednesday.

There could be side effects.

With the treatments available now, fewer than 10 percent of patients with the relapsed, hard-to-treat leukemia are alive five years after diagnosis.



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