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Parkinsonís Disease Foundation Comments on Failed Trial of Experimental Treatment for Parkinsonís

Ceregene, Inc., a company testing the potential of a gene therapy, CERE-120 (AAV-neurturin), has announced that its Phase 2b clinical trial failed to meet the end point that would have demonstrated the effectiveness of the treatment in easing the motor symptoms of Parkinson’s disease.
 
This is the second time in five years that AAV-neurturin, which delivers a neurotrophic factor to the brain, has failed in clinic trials. After the first trial, which ended in 2008, scientists found encouraging evidence of later improvement in the motor symptoms of participants. They felt this justified a second trial, targeting an additional and larger area of the brain. 
 
Christopher Goetz, M.D., Head of the Section of the Movement Disorders at Rush University Medical Center, Chair of the Parkinson’s Disease Foundation (PDF) Medical Policy Committee and member of PDF’s Scientific Advisory Board, issued the following statement: 
 
“The negative results of this second trial of neurturin are disappointing both to the people with Parkinson’s and their physicians, all who had invested their hopes in its success. However, we need to make a crucial distinction between the poor performance of this particular gene and the potential of gene therapy as a whole. This gene may have failed, but the excellent safety profile and successfully executed technical aspects of the trial suggest great potential for the testing of other genes. This area of science continues to be one of the most promising potential approaches to reaching into the cell machinery of Parkinson’s disease and easing its symptoms — especially those involving movement.  We now know, from this trial and others, that the technique can be conducted safely, and we understand much better than we used to the nature of the brain targets, the delivery systems and the surgical techniques. The study, even though it failed to prove that this gene manipulation was successful, allows us to move to the next promising gene rapidly, because we have resolved a number of questions on technique and safety.”
 
Robin Elliott, PDF’s Executive Director, noted PDF’s emphasis on training and mobilizing advocates to play leadership roles in research, including helping to fill trials, saying, “The failure of any trial in which so many hoped for so much is of course a big disappointment, but it challenges us all, at PDF and among our hundreds of volunteer research advocates, to try and try again. The progress of scientific research is never a straight shot, and those of us who support it need to be prepared to face the disappointments along the way — and to learn from them for the next time around.”
 
“The results are very disappointing for the nearly one million of us in the US living with Parkinson’s, including friends of mine who participated in the study, who all saw CERE-120 as an important, promising new treatment,” added Linda Morgan, M.B.A., R.Ph., a person living with Parkinson’s, member of PDF’s Advisory Council and PDF Research Advocate. She continued, “Although the study was unsuccessful, it points to the opportunity for us as people with Parkinson’s — those with the most to gain or lose — to help scientists bring about better treatments at a faster pace. It is crucial that people with Parkinson's continue to be involved in research not only as participants but as integral contributors to the entire process."

Source Date: Apr 19 2013