Researchers from the MGH and Children’s Hospital recently reported the successful treatment of a child with a recurrent jaw tumor using the anti-angiogenic drug interferon alfa-2a. The article in the June issue of Pediatrics describes the first such treatment of a bone tumor, called a giant cell lesion, using the concept of anti-angiogenesis, blocking the growth of new blood vessels. This strategy of treating tumors was developed in the laboratory of Judah Folkman, MD, of Children’s Hospital, senior author of the paper.

The girl was first seen in 1994 at the age of 5 for a large, painful growth on her left jaw by Leonard Kaban, MD, DMD, chief of Oral and Maxillofacial Surgery at the MGH and lead author of the Pediatrics paper. He removed the growth — a blood-filled cyst within her jawbone — twice, but the tumor recurred within months. Recognizing that the tumor behaved very much like the blood vessel tumors called hemangiomas, Kaban consulted Folkman’s team at Children’s, which previously had found that yearlong treatment with interferon alfa caused hemangiomas to disappear. Based on that similarity, and the fact that the girl was found to have abnormally high levels of bFGF, a substance known to stimulate blood vessel growth, the physicians started the child on interferon alfa treatment.

The tumor began to shrink within weeks and had disappeared when treatment ended a year later. There has been no recurrence in the three years since therapy was completed. In addition, the child’s left jaw, most of which had been removed, completely regenerated, something that is rare but not unknown in children who have had bone tissue removed. While the researchers believe they are defining a set of blood-vessel-related tumors that can be treated with interferon alfa, they stress that the substance is a very weak angiogenesis inhibitor and would not be a candidate to treat more virulent forms of cancer.

Additional MGH co-authors of the Pediatrics paper include R. Alan Ezekowitz, MBChB, DPhil, chief of Pediatrics, and David Ebb, MD, of Pediatric Hematology/Oncology.