美国监管机构批准白血病基因疗法

US regulators on Wednesday approved the first gene¹ therapy against cancer - a treatment that uses a patient's own immune cells to fight leukemia - opening a new era in the fight against one of the world's top killers². 

 

美国监管机构于8月30日批准了首个对抗癌症的基因治疗手段——使用患者的免疫细胞对抗白血病。白血病是世界上致死率最高的疾病之一，该治疗手段开启了对抗白血病的新纪元。

 

The treatment is made by Novartis and is called Kymriah. 

 

This type of anti-cancer immunotherapy, known as a CAR-T cell therapy, was known by CTL019 until now. 

 

"This marks the first-ever CAR-T cell therapy to be approved anywhere in the world," Novartis CEO Joseph Jimenez told reporters on a conference call. 

 

"It uses a new approach that is wholly personalized by using a patient's own T-cells." 

 

Kymriah was approved by the US Food and Drug Administration for children and young adult patients up to age 25 with a form of acute lymphoblastic leukemia (ALL). 

 

To qualify for treatment, patients must have B-cell precursor³ ALL that is refractory⁴, or the patient has relapsed at least twice. 

 

The FDA described the approval as "a historic action" and a "new approach to the treatment of cancer and other serious and life-threatening diseases," said a statement.

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