New 'vaccine style' treatment shows promise for slowing Parkinson's
Early results from a study of the drug developed by AC Immune offer hope that it could slow the progression of Parkinson's symptoms.
The drug works in a similar way to a vaccine, training the immune system to prevent clumps of a troublesome protein called alpha-synuclein forming and damaging brain cells.
What is immunotherapy?
Our immune system consists of cells which help our bodies fight challenges from viruses, bacteria and other things that could make us unwell. Our immune system learns to fight these challenges, by producing antibodies which can recognise and attack bacteria or viruses and prevent them from causing harm.
The idea behind an immunotherapy for Parkinson’s is a bit like a vaccine. The drug trains the body to recognise and break down toxic clumps of a protein called alpha-synuclein. This protein forms clumps inside the cells affected by Parkinson’s and is believed to contribute to the loss of brain cells.
Read more about why brain cells die in Parkinson’s on our research blog.
What did the researchers do?
In this study, 34 people with Parkinson’s received either the drug, or a placebo (dummy) drug, by injection into a muscle over a period of 12 months. 20 of these participants went on to receive the drug for a further 6 months.
Researchers then gathered samples of blood and cerebrospinal fluid (CSF, taken via a needle inserted into the spine) and looked for changes in the amount of alpha-synuclein clumps.
They also asked participants to perform movement tasks, assessed by the MDS-UPDRS score, and have a brain scan to see how the brain cells were changing over time.
What do the results show?
AC Immune, the company that ran the research study, have shared their interim results which show that the drug is safe and gets to where it needs to be in the body.
They also report that the amount of alpha-synuclein in the CSF was significantly higher in people who had received the drug, compared to those on the placebo. This suggests that the drug is sticking to and clearing away the alpha-synuclein clumps, so that they aren’t able to enter the brain and damage brain cells.
Finally, they looked at the movement symptoms of participants by comparing the differences in their MDS-UPDRS scores. Although the results weren’t significantly different, people who had received the active drug did tend to have similar scores at the beginning and end of the study, suggesting that there had been a slowing of symptoms getting worse. While people who had received the placebo drug had worse scores, as would be expected as Parkinson’s movement symptoms get worse over time.
Read a summary of these results in the AC Immune press release.
What happens next?
Dr Becky Jones, Research Communications Manager, said: "So far these are very early results, based on a small group of people. But they offer encouraging signs that the drug is safe and effective.
"It’s particularly interesting to see that the people who received the active drug showed some stabilising of their movement symptoms. While this certainly needs more exploring, it’s positive that this could be an early sign that the drug is slowing down symptoms getting worse.
"The company will release further results from the study early next year, and then the next step would be a bigger trial including more people, to see whether the drug could have potential for people with Parkinson’s."
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Read our research blog to find out what we know so far about why brain cells die in Parkinson's, and what researchers are doing to try and stop it.