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June 25, 2018
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The U.S. Food and Drug Administration approved the first-ever prescription drug made from marijuana Monday, The Associated Press reports. The medication, Epidiolex, is an oral treatment for seizures associated with two rare forms of childhood epilepsy. "This approval serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies," said the FDA commissioner, Scott Gottlieb.

Epidiolex uses cannabidiol (CBD), one of the chemicals in the cannabis plant, not tetrahydrocannabinol (THC), which causes the "high" associated with the drug.

"The difficult-to-control seizures that patients with Dravet syndrome and Lennox-Gastaut syndrome experience have a profound impact on these patients' quality of life," said Billy Dunn, the director of Division of Neurology Products at the FDA's research wing. "In addition to [Epidiolex being] another important treatment option for Lennox-Gastaut patients, this first-ever approval of a drug specifically for Dravet patients will provide a significant and needed improvement in the therapeutic approach to caring for people with this condition."

The medication, which comes in the form of a strawberry-flavored syrup from Britain's GW Pharmaceuticals, was tested in trials with more than 500 patients. Read about its common side effects and the approval process at the FDA's website. Jeva Lange

October 18, 2017
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The Food and Drug Administration on Wednesday approved a one-time treatment for lymphoma in adults, only the second time a gene therapy for blood cancer has been given the okay in the United States.

This is the first gene therapy approved for adults, and it involves removing a patient's T cells, reprogramming them to find and kill cancer cells, then putting the cells back into the patient, The Associated Press reports. The treatment uses the same technology as a gene therapy recently approved in the U.S. for childhood leukemia, and will cost $373,000 per patient, its manufacturer said. Catherine Garcia

July 12, 2017
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A new cancer treatment endorsed Wednesday by the Federal Drug Administration's Oncologic Drugs Advisory Committee is being described by one panel member as "the most exciting thing I've seen in my lifetime."

CAR T-cell therapy, made by Novartis, retrains a patient's immune cells so they target and attack cancer; the cells are removed from the body, sent to Novartis' plant so they can be genetically modified, and then shipped back to the patient for infusion. Novartis is seeking approval for use in children and young adults with leukemia who have relapsed despite undergoing chemotherapy. Stephen Grupp, the oncologist who led Novartis' trial, said most of the patients experienced cytokine release syndrome, which results in fever and flulike symptoms, and some had seizures and delirium.

If the FDA approves CAR T-cell therapy, which could happen by the end of September, it would be the first gene therapy cleared for use in the United States. Thomas Whitehead's daughter, Emily, was the first child to go through the treatment, at age six. She had leukemia and was close to death, he said, and while the CAR T-cell therapy nearly killed her, she survived and no longer has cancer, The Washington Post reports. He spoke in front of the Oncologic Drugs Advisory Committee on Wednesday and told them, "If you want to see what a cure looks like for relapsed ALL [acute lymphoblastic leukemia], she's standing right beside me." Catherine Garcia