Wednesday, August 30, 2017

First Gene Therapy Treatment For Cancer

Kymriah, which scientists refer to as a "living drug" because it involves using genetically modified immune cells from patients to attack their cancer.

The drug was approved to treat children and young adults suffering from acute lymphoblastic leukemia, a cancer of blood and bone marrow that is the most common childhood cancer. About 3,100 patients who are 20 and younger are diagnosed with ALL each year.

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer,".

"New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses," he said.

The treatment involves removing immune system cells known as T cells from each patient and genetically modifying the cells in the laboratory to attack and kill leukemia cells. The genetically modified cells are then infused back into patients.

"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease," he said.

"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," he said in the statement.
 
The treatment, which is also called CTL109, produced remission within three months in 83 percent of 63 pediatric and young adult patients. The patients had failed to respond to standard treatments or had suffered relapses. Based on those results, an advisory panel recommended the approval. 

The treatment does carry risks, however, including a dangerous overreaction by the immune system known as cytokine-release syndrome. As a result, it is requiring strong warnings.

In addition, the treatment will be available only at hospitals and clinics that have been specially trained in administering the therapy.

Patient advocate groups have expressed concern that the treatment could be the most expensive drug ever sold in the United States. One group, Patients for Affordable Drugs, said the group met Tuesday to discuss the price.

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