Therapeutic and Biologic Studies in a Murine Model of NF1
Abstract
This report describes progress made during the first year of support for a translational research project involving Nf1 mutant mice with myeloid leukemia. This study has two Technical Objectives. First, examining the therapeutic efficacy of two agents: (1) mycophenolate mofetiel (MM), and (2) a fusion toxin that targets the GM-CSF receptor. These compounds represent rational new approaches for treating NF1-associated tumors. MM has been tested in the mouse model and our preliminary data indicate that it is unlikely to provide benefit to NF1 patients. We have also produced and purified the GM-CSF immunotoxin for use in preclinical studies during the next fund year. We are also performing correlative biochemical studies to elucidate the effects of these therapeutics on cellular GTP levels and Ras signaling. In aim 2, we are utilizing Nf1 mice to extend clinical observations suggesting that individuals with NF1 are susceptible to the development of therapy-associated second cancers. These studies were initiated over the past year. We will perform other correlative molecular studies in mice that develop tumors. We anticipate that the proposed experiments will yield novel data that may be of practical value to patients with NF1 and their physicians.
Document Details
- Document Type
- Technical Report
- Publication Date
- Oct 01, 1999
- Accession Number
- ADA385680
Entities
People
- Kevin Shannon
Organizations
- University of California, San Francisco