June 11, 2012

Clinical Trial Underway -- Using Humans -- on New Vaccine for Parkinson's Disease

There is intriguing news from Vienna about a new clinical trial involving a vaccine for Parkinson’s disease (PD).

The Michael J. Fox Foundation is jazzed enough about the trial to have committed one and a half million dollars to it. This support may in part explain Fox’s comments several weeks ago, when he suggested that the most likely opportunity for progress against PD would probably NOT emerge from stem cell research, which for years has been a primary focus.

This new study is small: no more than 32 people. But it’s an important first step in moving vaccine studies from rodent to human participants. On June 5, AFFiRiS AG – a biotechnology company based in Austria – announced the start of Phase 1 in its study of the PD vaccine “PD01A.” By introducing antibodies that target the protein alpha-synuclein (alpha-syn), the new vaccine aims to disrupt and neutralize the possible toxic effect of this clumping protein – which seems to be a pathological “hallmark” of PD. (During his interview, Fox repeatedly mentioned the promise of working with "biomarkers." Alpha-syn accumulation is such a biomarker.)

Going Beyond Dopamine Replacement
So far, PD meds – like L-DOPA, introduced half a century ago – impact only the disease’s symptoms. The recent excitement about the gel pump therapy essentially uses that “old” technology: introduce a substance into the body which the brain then converts into dopamine, to offset dying neurons and improve “communication” between brain and body. (Yes, the pump at least has introduced the novel element of “continuous” medication into the mix – a step forward.)

But to date, no intervention has been shown effective in altering or halting the progress of the disease. Leaders of this new trial hope the vaccine will change all that. Said AFFiRiS CEO Walter Schmidt, "Worldwide, for the first time immunotherapy is applied for the treatment of Parkinson's. It is a so-called ‘First-in-Man’ and ‘First-in-Kind’ trial, because PD01A is the first medication worldwide aiming for clinical efficacy by modulating the metabolic pathway of alpha-syn.” 

Other Vaccines in the Works?
PD isn’t the only disease the company is addressing. Based on AFFiRiS’s own patent positions, it is developing “tailor-made” vaccines for Alzheimer's disease, atherosclerosis, hypertension and several other conditions. The new PD vaccine in trial now, PD01A, “is based on our AFFITOME®-technology, which already identified our lead vaccine developments in the field of Alzheimer's,” said AFFiRiS CSO Dr. Frank Mattner. He added:
This technology delivers not only a single vaccine for the treatment of a certain disease but a whole pool of product candidates with excellent safety profiles and exactly fine tuned specificities. Therefore, we apply our strategy of ‘clinical maturation,’ meaning that we investigate several vaccines against a certain disease in clinical testing to ensure that the best vaccine for humans will be developed.
It will be fascinating to watch this trial – small as it is -- play out. Are we on an important new road toward a real treatment for Parkinson’s? 

More info: 
The Michael J. Fox Foundation on the protein alpha-synuclein.
NIH's U.S. National Library of Medicine on AFFiRiS's AFFITOME® technology.

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