Parents agonize as trip delayed

Parents agonize as trip delayed

Three-month wait for infant son’s experimental gene therapy for a rare disease leaves Parker’s parents fearful for his health

Nov. 7, 2006. 06:42 AM

Nine-month-old Parker DesLauriers’ trip to Italy for life-saving gene therapy has been delayed until January.”We want to go as soon as we can,” his mother, Tracy DesLauriers, said in an interview last week at the Hospital for Sick Children.Right now would be the optimal time since he is the picture of health. But DesLauriers is resigned to the wait, even if it means enduring the cold and flu season, which could be devastating for her only child.”Everything with Parker seems to happen for a reason,” his mother said.DesLauriers inherited a rare disease, called ADA-deficient severe combined immunodeficiency (SCID), from his parents, who both carried a copy of a defective gene. Parker’s illness, also known as boy-in-the-bubble disease because patients have to be isolated, is so rare that Sick Kids has treated only 15 patients with it in the past two decades.The baby spent most of his first five months at the downtown Toronto hospital, but was allowed to go home to Ajax in June. Tracy and her husband Kevin were hoping to take him to Milan for experimental gene therapy in September, but the clinical trial has been delayed.Parker travels to the hospital twice a week to get an injection of an enzyme that his body can’t make. Without the adenosine deaminase (ADA), he is unable to filter out toxins that would kill off his immune system and eventually attack his organs. The injections work for only about two to three years.When Parker goes to the hospital, Tracy puts him in his stroller and fastens a clear plastic weather guard over it to protect him from germs.To look at the baby, you would never guess the smiling, robust boy is seriously ill and must spend most of his days inside the house and out of contact with the general public. A simple cold could jeopardize his health. He’s thriving now because of the enzyme injection he receives at the hospital, but the shot he receives in his thigh isn’t a cure.In order to help Parker, Italian doctors, led by Dr. Alessandro Aiuti, will remove stem cells from his bone marrow and introduce a normal version of the ADA gene. These will be reinjected and it is hoped they will migrate to the bone marrow and begin making ADA.A cell’s genetic material is stored on chromosomes in the nucleus on long molecules of deoxyribonucleic acid or DNA, which contain our genes. The genes dictate an organism’s characteristics.Dr. Roderick McInnes, scientific director for the Institute of Genetics at the Canadian Institutes of Health Research, said gene therapy holds much promise for the future. So far, the one place it appears to be working well is in the treatment of ADA-deficient SCID patients. “The Italian team are amongst the world’s best gene therapists,” he said. Not one of the handful of children treated by the Italian team seems to have any side effects. “It appears to be a safe `cure,'” said McInnes. Since this is a new therapy and the team is dealing with children, no one quite knows if the treatment will last forever or for only five or 10 years. Gene therapy for SCID is also being done in England and Japan, noted Kevin DesLauriers. “We were kind of bummed out when they told us we weren’t going until January,” he said. “But we can now have Christmas at home, perhaps his first birthday here.”Tracy agreed. “When you go through something like this, you learn not to question as much,” she said. They’ve learned to enjoy every moment they have with their son. “We just have to try and keep him healthy.”

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