A baby in China has become the first child to receive a stem cell transplant to treat an inherited form of irritable bowel disease. It is a significant breakthrough because there is currently no cure for this often-fatal condition.
Zhao Jiaxin was born in 2015 and began to suffer from diarrhoea just 8 days after her birth. Her older sister had also suffered from severe diarrhoea shortly after being born and eventually passed away as a result. Zhao’s parents were alarmed at the prospect of losing another child to illness.
Zhao was taken to Children’s Hospital of Fudan University in Shanghai, where genetic testing was performed. It revealed that she had Very Early Onset form of Irritable Bowel Disease (VEO-IBS). VEO-IBS is caused by an inherited genetic mutation, which explained why two children in the same family had the disease.
VEO-IBS is caused when a deficiency in a gene called interleukin-10 leads to severe dysregulation of the immune system within the intestines, causing diarrhoea and other symptoms. This condition was first discovered in 2009. In addition to diarrhoea, it can cause fistulas and intestinal abscesses.
Huang Ying, MD PhD, director of the digestive disease department at Children’s Hospital of Fudan University informed Zhao’s parents that the only effective treatment was a stem cell transplant. He explained that stem cells could help to repair the genetic deformity and control the symptoms of the disease.
Zhao became the first of 9 patients to receive a stem cell transplant to treat VEO-IBS with IL10RA immune deficiency2 in the next 2 years. The patients ranged in age from 6 to 43 months.
Before receiving the transplant, each patient received an intense chemotherapy treatment, which destroyed their immune cells. This was followed by a transplant of umbilical cord blood stem cells, which allowed the body to produce healthy immune cells once more. This type of treatment “resets” the immune system, removing the genetically malformed immune cells from the patient’s body. All of the patients experienced normal stools within 4 to 12 weeks of the procedure. Little baby Zhao is now putting on weight and is healthier than ever.
Source: Cord Blood Transplant Corrects Very Early Onset Irritable Bowel Disease
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