Unrelated blood cord transplant was used at Sir Ganga Ram Hospital to treat 16-month-old Manya of Hurler’s Syndrome, a rare genetic disorder. Also called Mucoploysacharridosis Type I, the disease which causes deposition of abnormal mucopolysaccharides proteins in various parts of the body leading to bone deformity, big and abnormal face, thick skin and blindness.
In India, about 30,000-40,000 children are affected every year.
Experts say the treatment can also be used for leukemia, lymphomas, thalassemia, aplastic anaemia, immuno-deficiencies and other inherited metabolic disorders.
“Hurler’s syndrome is caused due to a defective enzyme. So far, to control the disease, enzyme therapy was used which had to be administered on a weekly basis, and the medication was life long,” said Dr Ratna Dua Puri, consultant, department of genetic medicine at the hospital .
“Manya alone needed enzymes worth Rs 50,000 every week, this was when she weighed only 10kg and the cost would have increased with weight gain,” she said. She underwent the double cord blood transplant on October 10.