Progeria, a disease causing some aspects of premature aging, is so rare that only about 40 children worldwide are known to have it. Children with progeria often develop cardiovascular disease and osteoporosis within their first few years of life, and most die of heart attacks, at an average age of 13.
Now, Children's is treating as many of these children as possible in a clinical trial—the first ever launched for progeria. Led by Mark Kieran, MD, of Children's Division of Hematology/Oncology, the study is backed by a multidisciplinary team including David Miller, MD, PhD (Genetics) and Monica Kleinman, MD (Critical Care Medicine).
The children, aged 3 to 16 years and representing 16 different countries, have been flown to Boston, all costs paid, by the Progeria Research Foundation. "When I first started, I had never seen a child with progeria, and now I have seen 28, which is 27 more than any physician in the world," Kieran says.
All participants are receiving an experimental cancer drug called lonafarnib, normally used for brain tumors. In mice with progeria-like disease, lonafarnib and similar drugs prevented some signs of premature aging—the mice gained weight and had fewer bone fractures.
The clinical trial is starting with doses of lonafarnib known to be tolerated by children with cancer, to be increased if no serious side effects occur. Completion is scheduled for 2009.
"These families have never had treatment options, so they've gone from nothing to hope," says Kieran. "But it's important to have realistic expectations."