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Multidisciplinary Neurofibromatosis Program Research and Innovation

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Clinical Trials and Research

Boston Children’s Hospital /Dana-Farber Cancer Institute is one of nine sites in the national Neurofibromatosis (NF) Clinical Trials Consortium. As part of this group, we actively develop new treatment trials in NF. We also have several local research projects, initiated by our clinical investigators.

The following clinical studies are ongoing at Boston Children's Hospital:

  • Department of Defense Phase II Trial of R115777 in children and young adults with NF1, who have progressive, plexiform neurofibroma(s). PI: Widemann (NCI), site PI: Kieran. 
  • Department of Defense Phase II Trial of Pirfenidone in children and young adults with NF1, who have progressive plexiform neurofibroma(s). PI: Widemann (NCI), site PI: Kieran. 
  • A Phase II Trial of Peginterferon Alfa-2B (Pegintron) for Neurofibromatosis Type I Related Unresectable, Symptomatic or Life-Threatening Plexiform Neurofibromas
  • A randomized placebo-controlled study of lovastatin in children with Neurofibromatosis type 1.
  • A Phase II Study of Sirolimus in NF1-Related Plexiform Neurofibromas 
  • A Phase II study of RAD001 (Everolimus) for children with NF1 and chemotherapy-refractory radiographic progressive low-grade gliomas.
  • Pilot study of a novel computerized task to assess spatial learning in children and adolescents with Neurofibromatosis type 1.  
  • Genetics and molecular studies of Neurofibromatosis, Harvard NF center. PI: Irons. CHB protocol #: 04-05-066. 
  • Measurement of angiogenic growth factors in the blood and urine of patients with Neurofibromatosis type I and plexiform neurofibromas. PI: Irons. CHB protocol #: 04-10 113R.
  • Use of Matrix Metalloproteinases as non-invasive biomarkers of central nervous system disease. PI: Smith.  CHB protocol #:04-12-138. 
  • Moyamoya after cranial irradiation for primary brain tumors in children. PI: Ullrich. CHB protocol #:03-10-217. 

Specific research areas of interest to our team include:

  • Genomic analysis of NF1; genotype-phenotype correlations. In conjunction with the Harvard-wide NF center, we are trying to determine if particular mutations are associated with different clinical manifestations of NF1.
  • Dysregulation of angiogenesis in NF1 by evaluation of matrix metalloproteinases and angiogenic factors in patients with NF1, plexiform neurofibromas and optic pathway tumors.
  • Studying the process of new vessel formation as it pertains to the development of tumors in associate with NF1. This includes evaluating the small number of patients with moyamoya syndrome and vascular abnormalities.
  • Growth abnormalities in children with NF1, including delayed or precocious puberty. 
  • Abnormalities in bone development and fractures in children with NF1.

The future of pediatrics will be forged by thinking differently, breaking paradigms and joining together in a shared vision of tackling the toughest challenges before us.”
- Sandra L. Fenwick, President and CEO