Children leave hospital after gene therapy trial success
The Children's Hospital of Fudan University announced the successful treatment of the first two children in a clinical trial of a domestically-developed innovative gene therapy for spinal muscular atrophy type 1.
SMA is a rare progressive disease characterized by the degeneration and loss of lower motor neurons, which leads to muscle atrophy. The disease is divided into four subtypes and type 1 is the most severe form and a leading genetic cause of infant death.
The two children, one from Vietnam, have been discharged after receiving the medication by intravenous injection and undergoing follow-up tests on safety and effects. The child from Vietnam is the first expat child to participate in China's clinical trial of gene therapy, the hospital said on Monday.
Doctors said the children both showed positive improvements after taking the medication without showing serious drug-induced complications, indicating the effects and safety of the therapy.
The Vietnamese boy's parents heard about the clinical trial from relatives and contacted Dr Wang Yi, chief expert of the clinical trial.
The Vietnamese boy is checked after receiving gene therapy at the Children's Hospital of Fudan University.
Wang's team spent much effort in streamlining the process for the child's participation in the trial within a short period of time. The medical evaluation and processes inside the hospital were also accelerated, as the child was already five months and 20 days old, very close to the criteria for the trial (which sets the age limit as six months after birth), while arriving in Shanghai.
"The parents bear a strong trust and hope for our hospital and have come all the way from Vietnam to Shanghai. We accelerated all necessary processes and were able to fulfill all the screening, preparation and drug injection within a week," Wang said.
The EXG001-307, a China self-developed gene injection, is a single-dose gene-replacement therapy for SMA.
"The participation of this expat child means our clinical trial has broken the barrier of border and language and China's SMA gene treatment trial is accessible to children all over the world, offering new thought for China's rare disease research, speeding up new drug trials and streamlining its official market," Wang said. "The so-far data and effects on the therapy give us strong confidence and we believe it is a very promising gene therapy."
The clinical trial is still recruiting patients, no matter what nationality. Anyone interested can contact the research team through Dr Zhu on 18101843510, the hospital said.
Doctors report positive improvement in children in the clinical trial.
