AUGUST 24, 2009

The Rett Syndrome Research Trust announced today its financial support of an innovative research project with significant therapeutic potential. The project will be conducted in the laboratory of Stavros Lomvardas at the University of California, San Francisco.

Lomvardas discovered that the MeCP2 protein, associated with a variety of neurological disorders, including Rett Syndrome, regulates olfactory neuron differentiation in an unusual way. Olfactory receptor neurons, or ORNs, are crucial to our sense of smell. A differentiated, mature olfactory neuron expresses several molecular markers in a mutually exclusive manner.

But Lomvardas observed that in MeCP2 knockout mice these neurons co-express these molecular markers. Lomvardas will capitalize on the violation of the mutually exclusive expression pattern to develop a molecular assay for MeCP2 activity. The assay will then be used in a high throughput screen to identify chemical compounds that can reverse the molecular consequences of MeCP2 deletion in olfactory neurons. These experiments may be consequential to disease areas beyond Rett where epigenetic phenomena play a role.

"To my knowledge there are currently no cellular assays for MeCP2 dysfunction that are amenable to high throughput screening. Dr. Lomvardas is therefore uniquely positioned to develop the first in vitro assay that can be utilized to screen tens of thousands of chemical compounds in search of novel drugs to treat Rett Syndrome. Furthermore his research has the potential to contribute significantly to our understanding of MeCP2 function," said Adrian Bird, RSRT trustee and scientific advisor.

Lomvardas received his doctorate degree in biochemistry at Columbia University where he trained in the laboratory of Dimitris Thanos and Nobel Laureate Richard Axel. He established his independent lab at UCSF two years ago. Though at an early stage in his career, Lomvardas has already generated an impressive list of high-profile manuscripts.

"It is a great honor to be selected for funding from the Rett Syndrome Research Trust. I am fully committed to the goals of the Trust and I will work hard towards the better understanding of the causes of Rett Syndrome and the development of potential therapeutic approaches," said Lomvardas.

"Dr.Lomvardas is precisely the kind of out-of-the-box thinker and productive scientist RSRT seeks to recruit into the field. He has a track record of generating and executing on transformative ideas. I look forward with anticipation to the advances he will undoubtedly make," said Monica Coenraads, executive director of the Rett Syndrome Research Trust and mother to a daughter with the disorder.

An interview with Dr. Lomvardas is available online at the RSRT Blog.

About the Rett Syndrome Research Trust
The Rett Syndrome Research Trust is a nonprofit research organization formed in the wake of the dramatic pre-clinical reversal of Rett Syndrome symptoms. The Trust is intensively and exclusively focused on the development of treatments and cures for Rett Syndrome and related MECP2 disorders. Our short-term goal is to deliver clinical trials of a novel treatment strategy within five years. The strength of the Trust is based on the guidance of scientific advisors who are largely responsible for the major advances in Rett research over the past decade. To learn more about the Trust please visit www.ReverseRett.org.

About Rett Syndrome
Rett Syndrome is the most physically disabling of the autism spectrum disorders. Primarily affecting little girls, it usually strikes by 18 months of age, often just after a child has learned to walk and talk. As the cascade of Rett symptoms descends, she loses acquired skills, normal movement and speech, and begins the long regression that may leave her in a wheelchair, unable to communicate or use her hands. Extreme anxiety, Parkinson-like tremors, and orthopedic problems are common. Many children have intractable seizures. Some must be fed through tubes. Breathing, cardiac, circulatory and digestive functions are impaired. Most children live to adulthood, requiring total, 24-hour-a-day care.