Drug Discovery

Colleen Niswender, Ph.D.

Research Professor of Pharmacology

23rd Avenue at Pierce, Nashville, TN, 37212-6600

Research Description

I am an Associate Professor of Pharmacology and also currently serve as the Director of Molecular Pharmacology for the Vanderbilt Center for Neuroscience Drug Discovery (VCNDD). My work focuses on high throughput screening, assay development, and in vitro pharmacology experiments to investigate the effects of novel modulators on cellular signaling pathways. I have been or am currently involved in each of the main VCNDD collaborations, including those partnered with the Michael J. Fox Foundation, Johnson and Johnson, Astrazeneca, the William K. Warren Foundation, and the NIH. One of these projects has progressed to a clinical trial. I am also the Biology Team Leader for the development of potential clinical candidates for metabotropic glutamate receptor 4 (mGlu4) modulation as a treatment of Parkinson's disease, a program that is now moving into IND-enabling work. Most recently, I have initiated a new area of study for the VCNDD: that of disorders related to mutations or overexpression of the protein Methyl CpG Binding Protein 2 (MeCP2). Mutations in the MECP2 gene cause the neurodevelomental disorder Rett syndrome, while gene duplications cause MECP2 Duplication syndrome. These disorders result in a constellation of symptoms, beginning in childhood, including regression and loss of previously acquired skills, stereotypic movements, difficulty in walking and speaking, apneas, recurrent infections, and debilitating seizures. It is our goal to explore the therapeutic potential of modulation of metabotropic glutamate receptors 3, 5 and 7, as well as the M1 and M4 muscarinic receptors, in the treatment of Rett and MECP2 Duplication symptoms using small molecule allosteric modulators.

Selected Publications