Roche signs agreement with Harvard and affiliated hospitals to develop cell models for autism spectrum disorders (ASDs)

Basel, Switzerland, 24 February 2012

Roche (SIX: RO, ROG; OTCQX: RHHBY), together with the Harvard Stem Cell Institute, Harvard Medical School, Children’s Hospital Boston and Massachusetts General Hospital (MGH), announced today a research and development agreement to create in-vitro drug screening assays for the modeling of autism spectrum disorder (ASD). The ultimate aim is to screen and identify new drugs for the treatment of the condition.

In a unique partnership, researchers from Roche, working with Children’s Hospital Boston, will obtain skin biopsies from suitable individuals after genetic screening of patients with specific genetic conditions causing ASDs. These skin samples, along with comprehensive clinical data gathered by Children’s researchers, will be provided to researchers at MGH and the Harvard Stem Cell Institute for fibroblast isolation and reprogramming into induced pluripotent stem cells (iPSCs). Researchers at Harvard Medical School will, in turn, differentiate the iPSCs into lines of functional neurons that will be used to identify potential therapeutic targets and for a variety of drug screening assays.

Luca Santarelli, Global Head of Roche Neuroscience, said: “Roche is committed to harnessing the potential of emerging technologies to develop new treatment options for diseases with a high unmet medical need. Using patient- derived stem cells will help us better understand the cellular phenotype of ASD and holds the promise of developing truly personalized therapies.”

Anirvan Ghosh, Head of CNS Discovery at Roche, said: “Despite a high unmet need, ASD is characterized by a lack of suitable cellular models for drug development. Our collaboration with Harvard, Children’s and MGH represents an important step towards this goal. Our intention is to generate cell lines from ASD patients, which will be used to identify signaling and synaptic defects associated with ASD, and to develop drug screening platforms. This should greatly facilitate the discovery of the next generation drugs to fight the condition. ”

“This study is a great step toward possible treatment of genetic forms of autism and gives hope to patients and families,” said Mira Irons, MD, associate chief of genetics at Children’s Hospital Boston and co-principal investigator in the collaboration. “If specific patterns can be identified that are similar in the different genetic conditions that cause ASDs, this knowledge may more quickly lead researchers to identify targets for pharmacological therapy.”

“This collaboration brings together outstanding science at Roche with a wide range of expertise in autism research at Children's and Harvard,” added co-investigator Michael Greenberg, PhD, professor and Chair of Neurobiology at Harvard Medical School. “It makes me optimistic that important advances will be made in understanding and developing therapies for autism.”

ASD is a group of cognitive disorders - which include Autism, Asperger Syndrome and Pervasive Developmental Disorder Not Otherwise Specified - that affect and impair patients’ social interaction and communication. Although a specific cause of ASD has not been found, several studies suggest ASD has a genetic component and may be caused by abnormal neuronal growth during the early stages of brain development.