Calgary doctors are the first in the country to perform an experimental cell transplant that will help a little girl’s liver function better until she is big enough for a transplant.
Three-month-old Nazdana Ali from Winnipeg has a genetic disease called Urea Cycle Disorder or UCD.
The disorder causes ammonia to build up in the body and if left untreated would lead to brain damage and death. UCD patients have trouble converting ammonia to urea and when the ammonia builds up, there is a risk of neurological damage.
The condition is rare and incurable and without conventional treatment, most newborns with UCD die within the first two weeks of life.
A liver transplant is the only long-term treatment for patients like Nazdana but by transplanting liver cells doctors will be able to help her liver keep the ammonia down to safe levels.
Doctors at Alberta Children’s Hospital successfully completed a series of liver cell transplants earlier this month on Nazdana.
“We have been monitoring Nazdana closely and are happy to report that she has tolerated the liver cell transplant well, she is stable and can return home to Winnipeg,” says Dr. Aneal Khan, who is also an assistant professor of medical genetics and pediatrics at the University of Calgary, and a member of the U of C-AHS Alberta Children’s Hospital Research Institute for Child and Maternal Health.
The liver cell infusion will help to control the condition until Nazdana is big enough to undergo a transplant.
“This could represent a big leap forward in managing these cases. It’s a promising new bridge therapy that could improve the odds of Nazdana surviving until she is able to undergo a liver transplant.”
Doctors say little Nazdana currently weighs about 4.5 kilograms but she will have to grow to at least seven kilograms to improve her chances of a successful organ transplant.
Her father, Jouhar Ali, says he is grateful for the care his daughter has received.
“The health care people – the nurses and the doctors – they have all been outstanding. I have no words to explain it,” says Ali. “I really appreciate and am very thankful for what they have done.”
Up until now, the procedure has only been performed in Germany and a few centres in the U.S.
The study allows the enrollment of three patients and Dr. Khan expects the next candidate for the treatment will be a baby from southern Alberta whose high ammonia levels are currently being managed in hospital.
About 50 babies are born in Canada each year with UCD and the Alberta Children’s Hospital treats about two children with the condition annually.
