With a grant of USD 475,000, the Michael J Fox Foundation for Parkinson's Research (MJFF) is funding preclinical development of a vaccine against Parkinson's disease by AFFiRiS AG, based in Vienna, Austria.
The vaccine, known as PD01, targets the protein alpha-synuclein and might offer for the first time a possibility for a treatment that can slow or stop the progression of Parkinson's disease. The basis of PD01 is the company's AFFITOME technology, which already delivered, among others, two vaccines from AFFiRiS AG for the treatment of Alzheimer's disease.
The American actor, who was diagnosed with Parkinson's disease in 1991, established the foundation in 2000. Its aim is to fund peer reviewed therapeutic development for the treatment of Parkinson's disease, a progressive disease of the nervous system.
The foundation will now support the development of the PD01 vaccine by AFFiRiS AG with USD 475,000. This sum has been committed for the completion of the preclinical development of the vaccine. The successful preclinical proof of concept was only recently announced by the company, in March 2010.
Dr Markus Mandler, Head of the department of Neurodegenerative Diseases at AFFiRiS AG, said: "As many as one in a hundred people over age 60 develops Parkinson's disease. Despite this large number, medical developments to date have mainly addressed the symptoms of the disease - a treatment that can slow or stop disease progression has not been successfully developed."
The vaccine PD01 targets the protein known as alpha-synuclein (alpha-syn), whose accumulation and associated deposition in the brain are considered to be key for the progression of Parkinson's disease. A reduction in the alpha-syn concentration in the brain should therefore have a positive effect on the clinical progression of Parkinson's. PD01 triggers an effective immune response to the harmful alpha-syn, to allow the patients' own immune system to reduce this protein.
The current industry report from GlobalData confirms that such new solutions are urgently needed for Parkinson's disease, an area which, according to the report, suffers from a lack of innovation and a focus on purely symptomatic treatments.
In its pursuit of breakthrough treatments for Parkinson's, MJFF has ranked alpha-syn one of its "top therapeutic targets". The Foundation works with top researchers in academic and industry settings around the globe to both develop new therapeutics that can modulate the activities of its top targets, and to increase biological understanding of Parkinson's in order to more effectively test potential new treatments.
In 2011 the Foundation will bring the Parkinson's Progression Markers Initiative (www.michaeljfox.org/PPMI), its landmark clinical study seeking biomarkers of Parkinson's disease, to Austria's University of Innsbruck and four other European sites. Alpha-synuclein will be one of the proteins tested in PPMI for its potential as a Parkinson's biomarker.
As in the case of the Alzheimer vaccines previously developed, AFFiRiS AG has once again succeeded in presenting a plan for developing a medical treatment that is urgently needed.
The company's CEO, Dr Walter Schmidt, comments on this repeated achievement: "Our AFFITOME technology continues to deliver vaccines with an extraordinary fine specificity. Consequently, these vaccines may very accurately reduce the protein type which contributes to a disease."
Dr Frank Mattner, CSO of AFFiRiS AG, explains why this fine specificity is particularly important for a Parkinson's vaccine: "The protein alpha-syn, for example, belongs to a family of proteins that also comprises neuroprotective factors, such as beta-synuclein (beta-syn). A vaccination aimed at reducing alpha-syn must therefore ensure that the triggered antibody immune response exclusively addresses alpha-syn, without affecting beta-syn. Our AFFITOME® technology does exactly ensure that."
The Michael J Fox Foundation