Neurofibromatosis frustrates doctors because there is currently no effective treatment. Now, however, researchers have identified a promising inflammatory cell target to treat the symptoms of neurofibromatosis. The researchers are experimenting with potential drug compounds and hope to begin preliminary testing in humans by the end of this year.
One target will be mast cells, immune system cells that are involved in asthma and allergic reactions. Mast cells play a role in neurofibromatosis because they also are involved in blood vessel formation – and tumors need blood vessels in order to grow.
“We are beginning to have a better understanding of cell to cell interactions that lead to the development of tumors and are poised to answer some of the most perplexing questions that underlie neurofibromatosis,” said IU School of Medicine scientist Dr Wade Clapp. “Our work may well be applicable to other types of tumors such as breast and ovarian cancers, because inflammatory cells play an important role in tumor formation in these malignancies.”
The IU investigators now are identifying drugs that can disrupt the function of mast cells and their proteins in ways that they believe will starve the tumors.
By using drugs that target the mast cells’ ability to promote blood vessel growth, Clapp and his colleagues hope to prevent tumors from getting larger. Eventually, with continuing treatment, they hope the tumors will shrink or die from lack of blood vessel support.