An experimental pill meant to calm inflammation in the brain did better than a dummy pill on Parkinson’s symptom scores in a small trial, but mostly in one group of patients: those with a higher number of platelets, tiny cell fragments in the blood1. The drug, called bezisterim and made by the US company BioVie, was tested in 57 people with early Parkinson’s disease12. The results were shown as a poster at the 151st Annual Meeting of the American Neurological Association in San Diego1.
What happened
The trial, called SUNRISE-PD, was a Phase 2 study, a mid-stage test that looks for signs a drug helps before a larger trial1. People were picked at random to take either 20 mg of bezisterim or a placebo (a dummy pill) twice a day for 12 weeks, about three months1. Neither patients nor researchers knew who got which, according to the trial’s public registry entry2.
Everyone in the study had early Parkinson’s and had not yet been treated with levodopa, the common drug for movement symptoms. Brief use of levodopa for diagnostic testing was allowed1. The registry describes them as people aged 45 to 80 who were nearing the need for symptom treatment2.
The new results focus on 27 people whose platelet count at the start was above 230 billion per liter of blood1. In this group, those on bezisterim improved significantly more than those on the dummy pill on scores for movement symptoms, non-movement symptoms, and the overall score1. Parkinson’s News Today reported that this subgroup was identified before the data were analyzed1.
In the broader group used to judge whether the drug helped, people on bezisterim were also more likely than those on the dummy pill to show meaningful improvement on a combined score of symptoms and daily functioning, which the researchers treated as exploratory1.
How the symptoms were measured
The researchers used the MDS-UPDRS, a standard checklist doctors use to rate Parkinson’s symptoms and how they affect daily life. Its parts cover non-movement symptoms, trouble with everyday activities, and movement symptoms1. For the movement exam, they used a modified version that left out tests of muscle stiffness and balance1.
They also used a “Composite Benefit Score”, an exploratory measure that combines several aspects of symptoms and daily functioning. The high-platelet group did significantly better on it with bezisterim than with the dummy pill1. The trial also tracked inflammation markers, sleep and overall clinical benefit1.
Why platelets? Higher platelet counts may be a sign of inflammation throughout the body1. The idea behind the subgroup is that a drug aimed at inflammation might help most in people who have more of it.
What it does not show yet
Several limits matter here.
- It is small and short. 57 people took part2, and the subgroup with the clearest results had 271. The treatment lasted about 12 weeks1. A three-month study cannot show whether a drug changes how Parkinson’s progresses over years.
- The main goal was a blood measure. The registry lists the trial’s main planned outcome as the monocyte-lymphocyte ratio at 12 weeks, a blood test that relates to inflammation2. The coverage of the poster does not say whether that goal was met. Symptom scores were among the other things measured1.
- Subgroup findings are exploratory. Parkinson’s News Today described the platelet results as coming from an exploratory subgroup analysis1. Results in a slice of a small trial can appear by chance and need a bigger study to confirm.
- The full numbers are not public. The registry shows that results have not yet been posted there2, and the reports read for this article do not give the size of the score changes, side effects, or how many people dropped out.
- Not peer reviewed. The data were presented as a conference poster1, not a journal paper checked by outside experts.
There is also a small difference between sources on length of treatment. The poster coverage says 12 weeks1, while the registry’s plain-language summary says participants would take the drug or dummy pill for four months2.
Who is behind it
BioVie sponsored the trial2. The drug has been in the company’s pipeline for years under the code NE3107; a 2023 industry pipeline report already listed BioVie and NE3107 among Parkinson’s drug candidates3.
The upbeat interpretation comes from the company. Clarence Ahlem, BioVie’s senior vice president and program lead for Parkinson’s disease, said in a company press release:
These results provide evidence that an anti-inflammatory treatment can benefit both motor and, importantly, non-motor symptoms of Parkinson’s disease.
He added that “because non-motor symptoms remain a significant unmet need, the breadth of activity observed with bezisterim is particularly encouraging”1.
How it fits the field
Parkinson’s slowly destroys nerve cells that make dopamine, a chemical messenger that helps control movement. As dopamine falls, people can develop tremor, stiffness and slowed movement1. Symptoms appear after 80% or more of these cells in a brain area called the substantia nigra have been lost, according to a 2023 industry report3. The disease also causes non-movement problems such as poor sleep, trouble with thinking and memory, depression and anxiety1.
Bezisterim rests on two ideas. One is that inflammation in the brain may help damage and kill dopamine-making cells. The other is that the brain’s response to insulin, the hormone that helps cells use blood sugar, may be impaired in Parkinson’s, which could affect how nerve cells use energy1. The pill is designed to reach the brain, reduce inflammation and improve how well cells respond to insulin1.
It joins a crowded field. Other experimental approaches include ambroxol, an old cough medicine being tested for two years in a UK trial of about 330 people; prasinezumab, a lab-made antibody aimed at a clumping protein called alpha-synuclein; and bemdaneprocel, a stem-cell therapy that tries to replace lost nerve cells4. Prasinezumab’s history is a reminder of how mid-stage results can look: its Phase 2 trial came up just short of its main goal, yet signs in several patient groups led developers into a larger Phase 3 trial4.
What to watch next
BioVie says the new data support a Phase 3 trial, the larger kind of test usually needed before a drug can be approved. The company has asked to meet with the US Food and Drug Administration about a proposed study that could enroll about 175 people with early Parkinson’s1.
Points to follow:
- Whether the full results, including the main blood-marker goal and side effects, are published or posted to the trial registry2.
- Whether the larger trial selects patients by platelet count, the group where the clearest differences appeared1.
- What the FDA says at the requested meeting1.
For now, the study shows a signal in a small, pre-chosen slice of patients over about three months. Whether bezisterim helps people with early Parkinson’s in a lasting way is still unknown.
