United States finally approves the breakthrough Gene Therapy for the treatment of Leukemia
- Author: Santos West Sep 01, 2017,
Sep 01, 2017, 1:42
The Food and Drug Administration (FDA) has called this a historic moment because it is considered to be the first gene therapy hitting the USA markets. The treatment is called a living drug because involves using genetically modified immune cells from patients to attack their own cancer. The FDA itself called the approval a "historic action". The first child that was treated with this therapy was near death but is now cancer free for five years and counting.
Additionally, those who are paying such a high price to receive this drug will have to travel to one of 32 sites across the country to have their cells collected and then shipped to Novartis' facility in New Jersey for the drug to be specified for that patient, according to Stat News.
A spokeswoman for Switzerland-based Novartis declined to say how much the drug will cost, though analysts have estimated a price of $500,000 or more. He's now the executive chairman of Rubius Therapeutics, a biotech firm that's also working with cell therapy to develop treatments like Kymriah that don't have to be as personalized. That's why Novartis has approached the Centers for Medicaid and Medicare Services and insurers about schemes where only patients who have responded to Kymriah in a month will incur a charge, despite the high cost of administering the treatment. Treatment with tisagenlecleucel has the potential to cause severe adverse effects. Once the cells are modified, they are infused back into the patient to kill the cancer cells.
The FDA said it has granted 76 applications for trials involving experimental CAR-T therapies.
Tisagenlecleucel (Kymriah), Novartis' newly approved auto T-cell therapy, will be put on the market with a price of $475,000 for a single infusion, an amount that is within the range anticipated by oncologists and that Novartis characterized as well below a price level that could be justified on cost.
The treatment will only be used once and is made custom for each patient.
The CAR-T treatment is aimed to treat patients desperately ill with common pediatric cancer.
"This transformational therapy for patients is the result of true collaboration between industry, academia, healthcare professionals, patients and caregivers", said Bruno Strigini, CEO, Novartis Oncology.
Kymriah does have side effects, causing cytokine release syndrome, a systemic response to the proliferation of auto T-cells that can cause flu-like symptoms such as a high fever, and neurological events. Novartis also considered the cost of bone-marrow transplants, which are now given to many leukemia patients whose cancer relapses. About 3100 people under age 21 are diagnosed with ALL each year in the United States, though most respond to standard therapy.
For one, the treatments will have to treat more types of cancer than the one approved on Wednesday. This allows the cells to replicate quickly and zero in on cancer cells, fighting the disease for years.
Other drugmakers have been developing Car-T products, including Kite Pharma, which was snapped up for $11.9 billionby Gilead Sciences earlier this week. And past year, another company, Juno Therapeutics, said five people in its clinical trials had died, all from cerebral edema.