|
 |
Bone marrow transplants have also been successful in some cases, but they pose serious risks of their own. There was no available donor for the two children in the study, who were seven months-old, and two years and six months old.
"With gene therapy you can treat every patient, and the toxicity is enormously lower than for bone marrow transplants. We propose at this stage, that every patient lacking a donor with an identical tissue type should be directed to gene therapy," said Bordignon.
Bordignon's team must test their therapy on more volunteers before it will be approved for researchers to use in Italy or abroad. They will start with patients without a matched donor for a bone marrow transplant, and may then include patients for whom a donor is available. Because ADA-SCID is a relatively rare disorder, these studies would function as phase I and II clinical trials, meaning they aim to demonstrate that the procedure is both safe and effective, Bordignon said.
In order to extend this method to a larger population of Italian patients, the group is seeking the approval of Italy's Istituto Superiore di Sanità . A similar application process will be necessary for performing the therapy elsewhere in Europe and in the United States. Because the therapy could be considered under the "orphan drug" category, meaning a treatment for a rare disease, the approval process in Europe should likely be relatively fast, lasting a few months, according to Bordignon.
Teacher: Michael
Many articles taken from 'A word with the doctor', by Dr. John Windsor.
|
Search
