Phase 1 study of UB-312 shows promise in Parkinson’s disease

In a study published in Nature Medicine on 18th September 2024, Parkinson’s patients reported improved daily movement with Vaxxinity’s Parkinson’s drug UB-312. The small trial, which included work from researchers at the University of Texas, the Mayo Clinic, and the Michael J. Fox Foundation for Parkinson’s Research, included only 20 people with Parkinson’s.

However, it describes what could potentially be two firsts for Parkinson’s disease: a diagnostic test and a potential immune-based treatment that works in a similar way to a vaccine. 

The target of the test and treatment is alpha synuclein, which aggregates abnormally in PD brains and destroys nerve cells.

Researchers have been investigating ways to measure and target these proteins. The company Vaxxinity developed an ‘active immune medicine’, to train the immune system to attack only abnormal versions of alpha synuclein —which are improperly folded.

“The idea is that patients should recognise their own misfolded proteins, and it is personalised because their own immune systems are doing the work,” says Dr Mark Frasier, chief scientific officer at the Michael J. Fox Foundation for Parkinson’s Research, which funded the testing part of the study.

Overall, people receiving the vaccine generated more antibodies against the abnormal alpha synuclein protein than those vaccinated with placebo, as measured by the Parkinson’s test. The test uses samples of cerebrospinal fluid to measure a person’s levels of abnormal alpha synuclein.

“What is unique about our technology is that it can stimulate the immune system to produce very, very specific antibodies against toxic forms of alpha synuclein, and do it in a safe way, which is reassuring,” says Jean-Cosme Dodart, senior vice president of research at Vaxxinity and senior author of the paper.

About half of the patients in the trial showed high levels of antibodies against the misfolded alpha synuclein, and most of these patients received the highest dose of the vaccine. They also scored the highest on motor and cognitive tests. There were too few patients to adequately assess any changes of Parkinson’s symptoms, but the researchers believe that longer follow-up with those tests, and potentially more frequent or higher doses of the vaccine, could lead to improvements in scores.

Overall, people receiving the vaccine generated more antibodies against the abnormal alpha synuclein protein than those vaccinated with placebo