News

March 6, 2009

Baby Amy Homeward-Bound

Infant Girl from Belfast First Child in World to Receive ENB-0040 Clinical Drug Trial

The 11-month-old baby girl who received a world first experimental treatment at Children's Hospital - Health Sciences Centre Winnipeg is now well enough to fly home to Belfast.

Baby Amy Tinsley was flown with family into Winnipeg on October 2, 2008 to receive experimental "enzyme replacement" treatment for a rare bone disorder known as infantile hypophosphatasia. The often fatal condition affects only one in every 100,000 children.

This rare disease leads to severe rickets in infants or children, and causes debilitating osteomalacia - "soft bones" - in adults. The earlier the symptoms appear in the patient, the more severe they will be. When symptoms appear in infancy, up to 50 per cent of hypophosphatasia patients die. Baby Amy's symptoms occurred with bones breaking while she was in the womb.

Montreal-based biotechnology company Enobia recently initiated clinical testing of the drug called ENB-0040 in Winnipeg after receiving the appropriate approval from Health Canada for a Phase 1 clinical trial in adults. In addition, Enobia has regulatory approval to enrol several additional severely affected infants who meet study criteria in a parallel study. In October, Winnipeg was the only approved study site for the infantile treatment protocol (now approved at sites in US, UK and UAE).

Dr. Cheryl Rockman-Greenberg, Medical Director for the Child Health Program at the Winnipeg Regional Health Authority and HSC, worked closely with the Tinsley family during the drug trial.

Rockman-Greenberg says Baby Amy was initially started off on a low dose of the enzyme - which was given intravenously; the dose was increased according to the study protocol. Rockman-Greenberg says, "We are excited by the results to date in Amy and so pleased that we were able to help this little girl."

In total, Baby Amy spent 22 weeks in Winnipeg, celebrating her first birthday at Children's Hospital on February 20, 2008.

"Doctors in Belfast didn't actually expect her to survive," Liane Tinsley, the baby's mother says. "I'm so very thankful for the kindness and care Amy and our family received from everyone at Children's Hospital. I feel so blessed to be able to take Amy home now."

The Tinsleys leave Winnipeg for Belfast on Saturday, March 7, 2009.