First data from novel Parkinson’s vaccine

Phase I data for AFFiRiS’ PD01A suggests it is worth moving into Phase II.

A possible breakthrough in the treatment of Parkinson’s disease has been announced by Austrian company AFFiRiS – although it is still at the very early stages.

The Vienna-based company’s vaccine PD01A is in Phase I, the first therapy targeting the protein alpha-synuclein to enter clinical testing, and has shown enough promise to take the next steps towards Phase II.

The Michael J. Fox Foundation for Parkinson’s Research (MJFF) – named after the Hollywood actor who suffers from the neurodegenerative condition – provided a $1.5 million grant for the study.

Two different doses of PD01A were safe and well-tolerated by patients, thus meeting the trial’s primary endpoint, while secondary endpoints included the induction of an alpha-synuclein-specific antibody response.

Twelve patients in each group received four vaccinations at monthly intervals of 15 μg or 75 μg, while eight patients on best medical care, including standard symptomatic medication, served as a control group.

Half of the vaccinated patients generated alpha-synuclein-specific antibodies as measured in serum samples, and vaccine-induced antibodies were seen in cerebrospinal fluid.

This is important because it is thought that build-ups of alpha-synuclein in brain cells lead to the degrading and death of cells in Parkinson’s disease, which means that reducing alpha-synuclein accumulation could prevent this degeneration.

Mobilising antibodies against the protein “is strong preliminary evidence in support of the principle” of PD01A, the company says.

It adds that analysis of clinical endpoints revealed a trend towards stabilisation in the vaccinated groups as opposed to the control patients.

While this is far from conclusive, MJFF has committed to support a follow-up study testing a boost vaccination.

“The safety and tolerability observed in this study, especially in a protein such as alpha-synuclein where we do not yet know its normal function, are encouraging,” said Walter Schmidt, CEO of AFFiRiS.

The next study, for which recruitment is expected to begin next month, will be in Vienna and is set to focus on evaluating the immunological and clinical effects of the booster.

“A treatment that could slow or stop Parkinson’s progression would be a game changer for the five million worldwide living with this disease and the many more who will become at risk as our population ages,” explained MJFF CEO Todd Sherer. “The AFF008 trial is one of the most promising efforts toward that goal.”

Earlier this year, an early-stage gene therapy showed potential for the treatment of Parkinson’s in a study published in The Lancet.

Oxford BioMedica is behind the research to develop ProSavin. Parkinson’s disease is typically treated with oral dopamine replacement therapies, whereas the investigational gene therapy aims to restore dopamine production.


Early-stage gene therapy shows potential against Parkinson’s

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