Pioneering Cancer Gene Therapy Gets Green Light-And a $475000 Price Tag

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Kymriah is the first CAR-T cell therapy to get approved, and several more are in the works.

Known as gene therapy, the treatment involves removing immune cells called T cells from the patient's blood and then genetically altering them in a lab. This revised forecast encompasses the expected cross-industry reaction to the approval, and envisions the next stages in the development of cell therapies and the industry.

"This represents the progression of the field of gene therapy, which has been developing over the last 30 years", says gene therapy pioneer David A. Williams, MD, who is chief scientific officer of Boston Children's Hospital and president of the Dana-Farber/Boston Children's Cancer and Blood Disorders Center. This is the first type of gene therapy to hit the US market called the CAR-T cell treatment that was developed by Novartis Pharmaceuticals and the University of Pennsylvania.

Gene therapy has been heralded as a powerful new weapon in the fight against cancer with some experts even claiming that eventually it could signal the end of the disease.

Mitchell said the campaign group thought a price below $300,000 was more appropriate, but said the estimate came with a "huge disclaimer" because Novartis has not shared information about its costs. The new treatment can act as a lifesaver for those patients who did not recover through first line drug treatments.

"Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials". Treatment with tisagenlecleucel has the potential to cause severe adverse effects.

Of more than 60 ALL-suffering subjects in a clinical trial for Kymriah, 83 percent were cancer-free within three months.

Dana-Farber/Boston Children's has recently participated in a similar clinical trial of vehicle T-cells - led by Silverman and pediatric oncologist Steven Margossian, MD, PhD - that evaluated the efficacy of auto T-cell therapy (short for chimeric antigen receptor T-cell therapy) in treating relapsed or treatment-resistant B-cell ALL. This therapy - known as chimeric antigen receptor T-cell therapy, or CAR-T - gives the cells the ability to recognize and kill the source of the cancer. For others, Kymriah serves as a "bridge to transplant", keeping them alive longer enough to undergo this therapy. The engineered T-cells can lead to what is called cytokine release syndrome, a life-threatening condition that is triggered by an overactive immune system. This also applies to those patients of this particular leukaemia who have suffered a relapse of the cancer.

Most patients with ALL recover through other treatments such as radiation, chemotherapy and stem cells. About 3100 people under age 21 are diagnosed with ALL each year in the United States, though most respond to standard therapy. Instead of getting a better, updated therapy for a disease every decade or so, we might begin to see second-generation cell therapies in a few years.

The treatment is the first of its kind approved in the U.S. "The lessons learned from the clinical trials of auto T-cell operations will assist us in being able to provide to the larger population".



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