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03-02-2012, 04:20 PM | #1 | |||
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http://blog.michaeljfox.org/2012/02/...irst-patients/
From mjff website:This week, Austria-based biotech AFFiRiS AG enrolled the first patients in an early stage clinical trial of a first-of-its-kind vaccine approach to treating Parkinson’s disease, called PD01. Just this past summer, The Michael J. Fox Foundation for Parkinson’s Research (MJFF) awarded $1.5 million to AFFiRiS for their vaccine targeting alpha-synuclein, a protein implicated in Parkinson’s whose clumping is the pathological hallmark of the disease. The Phase I study is testing the safety and efficacy of PD01. The hope is that this agent will simulate the production of antibodies that bind to alpha-synuclein, clearing it from the brain and slowing disease progression. Alpha-synuclein is a high-priority target for MJFF, largely because there is compelling evidence that it may play a role in both genetic and idiopathic cases of Parkinson’s disease. It is also the major component in the Lewy bodies that are the pathological hallmark of Parkinson’s. The trial, led by AFFiRiS chief medical officer Achim Schneeberger, MD, will engage 24 subjects with mild Parkinson’s disease over two years at a single clinical site in Austria. Following the MJFF award, AFFiRiS secured 30 million Euros in additional funding from two investors to develop its portfolio of Parkinson’s and Alzheimer’s vaccines.
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Sim00 Born in 1969, diagnosed PD in 2007, first symptoms 2004. DBS in July 2016. |
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03-02-2012, 05:57 PM | #2 | ||
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We are pretty excited about this...finally, we are in the clinic testing a therapy for alpha synuclein. I think that gene was discovered in 1996--16 years ago! Despite having the strongest relevance for therapeutic value (Asyn rationale comes both from it's role in "cause" of PD -- rare familial cases as well as in pathology -- it is present in post mortem tissue of PD patients) it has taken way too long to get to the clinic. My great hope is not only for this or trophic factors to prove disease modifying (these both have strategies in clinical testing now) but also that we can get much more strategic in what actions can be taken upon discovering a new target...that is something we've been working on. The LRRK2 discovery was published in 2004. I believe we have made more progress faster on LRRK2--at least that is our intention.
Debi |
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03-02-2012, 07:41 PM | #3 | ||
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I normally think of a vaccine as something to be given a person to prevent the onset of a problem, such as the polio vaccine. Is this a vaccine that can be given to people who already have the disease with the expectation that it will slow or reverse the course?
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03-02-2012, 10:18 PM | #4 | |||
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Quote:
I have been following the Affiris research and awaiting this news! Nan from what I recall, there is also a team at University of Nebraska at work on research here (as of 2010) but has stalled. I actually cried when I read this could be disease modifying; no matter what the outcome it is a watershed in PD research. Maybe Deb has some insight into why this is receiving no press? It also lends support to the theory that PD is primarily an auto immune dysfunction involving a prion and that it most likely is result of a long-standing untreated infection elsewhere. Their is a research group in the UK believe PD stems from bacterial infection H. pyllori and have dedicated much of their research efforts into this theory. Researchers proceed to develop vaccination to reverse neurological damage seen with Parkinson's disease I read that in very early PD it may reverse and in rest of us slow down the process. Dr. Gendelman also thinks this may work for ALS too! They have a patent but need corporate partnership; I can't believe they are having trouble funding this?!? See What is driving Parkinson's |
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03-03-2012, 01:22 AM | #5 | |||
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So what about Paula_W's post:
The pathology of Parkinson's disease is characterized by a loss of dopamine-producing neurons in the pars compacta of the substantia nigra (SN), an area of the brain associated with motor control, along with the development of α-synuclein (αS) protein in the form of Lewy bodies (LB) in the neurons that survive. The spread of LB pathology is thought to progress along with the clinical course of Parkinson's disease, although recent studies suggest that they are not the toxic cause of cell death. A new study published in The Journal of Parkinson's Disease finds no support for a primary pathogenic role of LBs, as neither their distribution nor density was associated with the severity of nigral cell loss. http://www.news-medical.net/news/201...LBs-in-PD.aspx __________________ |
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03-04-2012, 09:12 AM | #6 | |||
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Gerry,
Lewy Bodies are the end result of the toxic cascade; it comprises the junk protein and dead cell mass that results from the aggregation of alpha-Synuclein. Lewy Bodies are also known as the end stage of alpha-syn toxicity called fibrils. However, scientists have su bstantiated that the toxic stage is in the middle when alpha-Synuclein forms oligomers that spread throughout our system. Also note that Lewy Bodies are present elsewhere in our bodies. Please note that Lewy Bodies are found in so-called normal brains upon autopsy with some way worse than a typical PD brain, yet those people never experienced any clinical sign of PD. If we look at genetic forms of PD, those with a Parkin 1 (I think- one of the Parkins) has mitochondrial dysfunction and no alpha-Synuclein toxicity at all. Others with PD who have a longstanding chronic bacterial infection in the stomach called H. pylori have had their PD symptoms improve markedly when the bacteria is eradicated. Some have a vascular form of PD who have been "cured" when under the knife for a vascular condition. This is why we are all over the place with symptoms and reactions to meds, imho. So this tells us what? There are many different main causes or triggers for PD. Yet, they essentially test us for nothing? This means that whenever a cure or new treatment is introduced it may work for some of us, not all. What is really bothersome is why are we presumed to all have alpha-syn pathology? Why the idiopathic label with no testing? We are all left with highly addictive prescription drugs and a load of questions that no one can seem to answer. Makes one wonder...and Lewy Bodies are supposed to be "the Hallmark" of idiopathic PD?!? Laura |
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"Thanks for this!" says: | GerryW (03-04-2012) |
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