The first human trial on the cause of Parkinson’s disease vaccine has begun. Called PD01A, the vaccine targets a protein – alpha-Synuclein – which experts say causes the onset and development of the disease. The Phase I trial is being conducted by AFFiRiS AG, an Austrian pharmaceutical company.

The company hopes the vaccine may deliver a causative treatment for Parkinson’s. Its development has been generously supported by the Michael J. Fox Foundation. The trial’s primary endpoints are tolerability and safety of PD01A.

According to AFFiRiS, the vaccine candidate, PD01A, represents “..the first agent worldwide aiming disease modification of Parkinson’s rather than addressing symptomatic improvement only. PD01A targets a protein called alpha-Synuclein (alpha-syn) which plays a key role in the onset and progression of Parkinsons.”

PD01A is designed to teach the body’s immune system to generate antibodies that target alpha-syn. The trial, involving 32 patients, will be carried out at the Confraternität Privatklinik Josefstadt in Vienna, Austria.

AFFiRiS CEO, Dr. Walter Schmidt, said:

“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.

Even in its preliminary stages this new treatment concept was highly appreciated, as the renowned Michael J. Fox Foundation assented financial support to a total of 1,5 Mio. USD. Hence, this is one of the few projects outside of the USA considered worthy of support by the foundation.”

The company says this is the first vaccine to target the cause of Parkinson’s disease. For the last five decades, all therapeutic interventions on Parkinson’s have sought to substitute dopamine, a neurotransmitter.

Drugs used for treating patients with Parkinson’s have only been able to address the symptoms. There is not one drug today that can modify the course of the disease.

Experts say that Parkinson’s disease is caused by pathological forms of alpha-syn depositing in the brain. If the brain’s accumulation of alpha-syn can be reduced, they believe the course of the disease can be altered.

AFFiRiS claims that PD01A can reduce the brain’s alpha-syn aggregates by getting the body’s immune system to target alpha-syn, and thus stop its toxic impact.

Dr. Frank Mattner, CSO of AFFiRiS, said:

“PD01A is based on our AFFITOME®-technology, which already identified our lead vaccine developments in the field of Alzheimers. 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.”

Written by Christian Nordqvist