Gene therapy injected into the brains' of mice with Huntington...( coordination balance and fatigue res...)

coordination, balance and fatigue, researchers evaluated mice walking on a rotating rod. Mice injected with the gene therapy performed significantly better than the other mice. These mice also showed diminished hind limb clasping, (a simulation of motor control behavior in HD patients). Perhaps most importantly, gene delivery of GDNF provided neuroprotection in the brain, with reduced density of brain inclusions and less cell death.

The authors wrote "Although GDNF's exact role in preventing cell death in mice modeled with HD remains to be established, we speculate the increase trophic support and inhibiting apoptosis (programmed cell death) via these two pathways likely played integral roles."

Kordower says the study suggests a new approach to forestall disease progression in newly diagnosed HD patients by delivering potent trophic factors with effects that are long-term and non-toxic." "If these results can be replicated in HD patients, it would represent a significant advance in the treatment of this tragic disease", agreed Dr. Jeffrey Ostrove, President and CEO of Ceregene.

"We are pleased with the results of this 'proof of concept' study with AAV-GDNF in HD mice", stated Raymond T. Bartus, Ph.D., Sr. Vice President, Clinical and Preclinical R&D and COO, Ceregene. "We now look forward to completing ongoing studies with our product, AAV-NTN (CERE-120), in HD mice, also performed in collaboration with Dr. Kordower and Rush University Medical Center", Bartus added.

Ceregene's lead program with CERE-120 is in Parkinson's disease (PD). The company completed enrollment of a Phase I trial with CERE-120 at UCSF and Rush University Medical Center, which was reported to be safe and well tolerated in PD patients at the American Association of Neurology meeting last spring. Initial efficacy results of this Phase I trial are expected to be presented this fall and a double-blinded, controlled Phase II trail in PD patients is planned for
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Contact: Mary Ann Schultz
mary_ann_schultz@rush.edu
312-942-7816
Rush University Medical Center
12-Jun-2006

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