In 1817, James Parkinson expressed hope for the disease named after him. He thought that at some point a discovery would be made and “the progression of the disease might be halted.”
Almost 200 years after Parkinson's disease expressed hope, and after 40 years of failed clinical trials, a group of French researchers has produced the first sign of success: a one-year study that showed a gradual slowdown in the disease. I reported it.
And what drugs did they use? It is a so-called GLP-1 receptor agonist, similar to the popular diabetes drug Ozempic and the obesity drug Wegovy.
Half a million Americans have been diagnosed with Parkinson's disease, making it the second most common degenerative brain disease after Alzheimer's disease.
Symptoms include tremors, slowness and stiffness of movements, and difficulty with balance. This can make walking, talking, and swallowing difficult. Many patients develop dementia.
But Dr. David Standaert, a Parkinson's disease expert at the University of Alabama at Birmingham, said there are effective drugs and treatments, such as deep brain stimulation.
“You'll look and feel much better,” Dr. Standaert said. The problem is that the disease progresses inexorably.
“After five or 10 years of Parkinson's disease, a lot of problems start to emerge,” he says.
This new study gives researchers cautious hope.
Dr. Michael S. Okun, a Parkinson's disease expert at the University of Florida who was not involved in the study, said that while it's not a complete success, it's “on the edge of modifying the disease.”
Dr. Stendhart, who was not involved in the trial, said it was “a really encouraging step forward.”
“Too many trials have not shown success,” he added.
Dr. Hyun Joo Choo of the National Institute of Neurological Disorders and Stroke said the study was “very important,” although it was a Phase 2 study aimed at testing a hypothesis. He cautioned that the study was not large enough or of sufficient duration to draw definitive conclusions.
“There are many examples of very promising phase 2 trials,” she said. “People get really excited, but then things don't go well.”
The paper, published Wednesday in the New England Journal of Medicine, involved 156 patients with early-stage Parkinson's disease who were randomly assigned to receive Sanofi's lixisenatide or a placebo and followed for one year. It was done. The trial was funded by the French government and the British charity Cure Parkinson's.
Meanwhile, Parkinson's symptoms, such as tremor, stiffness, slowness, and loss of balance, worsened in patients who took the placebo but not in those who took the drug.
The drug also caused gastrointestinal side effects, such as nausea and vomiting, in more than half of the participants, likely because the researchers gradually increased the dosage like GLP-1 drugs such as Ozempic and Wigovy. This is likely due to starting at the highest dose instead of In one-third of participants whose side effects became intolerable, researchers cut the dose in half.
For European researchers led by Dr. Vasilios G. Meissner of the University of Bordeaux and Dr. Olivier Lascol of the University of Toulouse, it made sense to see if GLP-1 drugs could slow Parkinson's disease. .
Dr. Raskol said studies have repeatedly found that people with type 2 diabetes are at increased risk for Parkinson's disease. However, the increased risk is reduced in people taking GLP-1 drugs to treat diabetes.
He added that postmortem studies of brain tissue from Parkinson's disease patients have found abnormalities associated with insulin resistance, even if the patients did not have diabetes. GLP-1 drugs treat insulin resistance.
Finally, he said that GLP-1 drugs can bind to proteins in neurons, so they can affect the brain in different ways.
The French research group says it hopes to conduct larger, longer-term studies once funding and more drugs become available. Earlier this year, Sanofi announced it would stop selling the drug in the U.S. and began discontinuing it worldwide. A company spokesperson said the move was made for business reasons.
But what about Parkinson's patients who have diabetes or obesity? They are eligible to receive GLP-1 drugs. Should I take it in hopes of slowing the progression of Parkinson's disease?
Dr. Standaert, who wrote an editorial accompanying the study, said it was “rational” for them to take the drug.
However, he cautioned that it is impossible to determine whether the drugs slowed the progression of the disease because we do not know what would have happened if the patient had not taken the drugs.
“We can't learn anything from it,” he said.