A 14-year-old boy with serious thalassemia has recovered due to a locally-developed innovative gene editing technology, the Children's Hospital of Fudan University said on Monday.
The boy from Jiangxi Province is the first child with serious thalassemia to be cured by the treatment after a new drug developed by Shanghai-based CorrectSequence Therapeutics Co Ltd was approved by the national authority for clinical trials in April this year.
The treatment CS-101 uses innovative transformer Base Editing (tBE) technology to help people with severe diseases such as transfusion-dependent thalassemia. The boy no longer needed blood transfusions just two weeks after receiving treatment.
His hemoglobin increased to within the normal level just five weeks after the treatment and the boy has been discharged and resumed a normal life, said doctors, adding he will no longer need regular blood transfusions to remain alive.
A 14-year-old boy with an inherited blood disorder has recovered after undergoing innovative gene editing therapy in Shanghai.
The boy's father expressed gratitude to the hospital's doctors and nurses and shared the family's long-time suffering over his son's condition. All patients with serious thalassemia require blood transfusions, which can cause multiple organ damage and lead to disability and a shorter life.
"The new treatment not only gives hope to patients, but also helps them reduce the dependence on blood transfusions, enjoying a better life," said the father, who gave all the doctors and nurses an award banner.
Thalassemia is an inherited blood disorder that affects the ability to produce hemoglobin and healthy red blood cells. It can cause anemia-like symptoms that range from mild to severe. Patients with a serious form of it need regular blood transfusions, which often leads to a large financial burden on families.
According to the World Health Organization, 7 percent of the world's population are carriers of mutated genes of thalassemia. There are 300,000 to 400,000 patients being diagnosed each year in the world.
In China, there are 30 million carriers and about 300,000 patients in a serious condition. Most of them live in the southern part of the country.
Genetic treatment brings new hope to thalassemia patients in recent years. This CS-101 is developed based on an innovative gene editing method, which collects a patient's own stem cells for precise gene editing to simulate naturally beneficial mutations to reactivate a certain protein to restore the function of hemoglobin. After the edited stem cells are transfused back into the patient's body, he or she can regain a healthy hemoglobin level and no longer need blood transfusions.
The new therapy's success can improve treatment outcomes for thalassemia patients and also speed up the clinical application of China's original gene editing technology to help patients all over the world, hospital officials said.
Doctors and the boy cut a cake to celebrate his recovery.
Source: SHINE Editor: Wang Yanlin
