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Developmental disorders drug research bolstered

BY: BILL SNYDER

1/08/2010 - Vanderbilt University Medical Center has entered into a collaborative research agreement with Seaside Therapeutics LLC to discover and develop potential drugs to treat developmental disorders such as Fragile X syndrome and autism.

The targets of this effort are muscarinic acetylcholine subtype 1 (M1) receptors, which are known to regulate learning and memory.

Jeffrey Conn, Ph.D.

Jeffrey Conn, Ph.D.

Research conducted by Seaside Therapeutics suggests that inhibiting M1 receptor signaling could provide therapeutic benefit for individuals with disorders of brain development.

Vanderbilt scientists led by Jeffrey Conn, Ph.D., director of the Vanderbilt Program in Drug Discovery and a member of Seaside's Scientific Advisory Board, have identified novel small molecule compounds that are M1 antagonists.

Vanderbilt will work exclusively with Seaside on the discovery, development, optimization and eventual selection of lead compounds for continued study in disorders of brain development.

“Our focus on identifying the molecular pathophysiology of single-gene disorders associated with autism has provided insights for developing targeted therapeutics with the potential to correct or fundamentally alter the course of brain development and function,” said Randall Carpenter, M.D., president and CEO of Seaside Therapeutics, based in Cambridge, Mass.

“Vanderbilt's expertise in drug discovery and their continued commitment to developing novel therapeutics for brain development disorders makes them an exceptional partner in this initiative,” Carpenter said.

“Selectively inhibiting M1 receptors represents an innovative and very promising approach to treating brain development disorders,” Conn added.

“We believe the combined expertise of the two teams may play an important role in advancing research in the field and, hopefully, in the long-term, bringing disease-modifying therapeutics to individuals suffering from Fragile X syndrome, autism and other brain development disorders.”

Joining Conn in the research will be teams of Vanderbilt scientists led by Craig Lindsley, Ph.D. (Medicinal Chemistry), Carrie Jones, Ph.D. (Behavioral Pharmacology), and by Colleen Niswender, Ph.D., and David Weaver, Ph.D. (Molecular Pharmacology).

This agreement is the second between Seaside Therapeutics and the Vanderbilt Program in Drug Discovery (VPDD).

In 2008, Vanderbilt and Seaside entered into a collaboration to develop compounds that inhibit excessive signaling through the metabotropic glutamate receptor subtype 5 (mGluR5).

Research conducted by founders of Seaside Therapeutics and others indicates that excessive signaling through mGluR5, a receptor for the neurotransmitter glutamate, may be responsible for the neurological and psychiatric consequences of Fragile X syndrome, the most common inherited form of mental retardation and the most common genetic cause of autism.

Selective inhibition of the receptor potentially could reduce or eliminate these devastating effects, company officials said.

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