Parkinson’s disease is a neurodegenerative disease that affects nearly 200,000 people and 26,000 new cases are diagnosed each year in France.
To date, only Levodopa (or L-Dopa) and dopaminergic agonists manage to limit the motor symptoms triggered by this pathology. Medications which, over time, lose effectiveness and cause undesirable side effects (behavioral disorders or dyskinesias). As a way out, three viable new approaches to Parkinson’s treatment could be a game changer…
1- Ambroxol: a new Parkinson’s treatment?
What if a cough medicine could slow the progression of Parkinson’s disease? This is the surprising hypothesis of clinical trials started in 2020 by British researchers. The latter looked at Ambroxol, a treatment against respiratory pathologies.
Cough medicine… And Parkinson’s
Known to treat cough, Ambroxol is part of the drugs of the class of GCase-modulators. A specificity that interests scientists for their ability to slow and stop Parkinson’s disease.
Confirmation received from preclinical data from phase 2, carried out on 17 people with dementia. Indeed, Professor Anthony Schapira’s team at UCL finds that the drug increases levels of GCase (glucocerebrosidase), a protein that enables cells to more efficiently eliminate protein waste products, in particular alpha-synuclein. , responsible for brain damage.
A 3rd clinical trial in progress
Following the good tolerance of Ambroxol by the participants, the phase 3 clinical trial, called ASPro-PD (led by the Queen Square Neurological Institute of UCL in partnership with the British organization Cure Parkinson’s and the Van Andel Institute), will be able to begin.
On the program: 330 Parkinson’s patients will be recruited to take Ambroxol for two years. Their results will be compared to a control group treated with placebo. The aim will be to validate the effectiveness of Ambroxol in terms of its ability to slow the progression of Parkinson’s disease, taking into account the quality of life and the involuntary movements of the participants.
After 10 years of detailed laboratory research, this is the first time that a drug applied specifically to a genetic cause of Parkinson’s disease has reached this level of testing.
Professor Anthony Schapira
2- Focused ultrasound to reduce the symptoms of tremors
Tremors are one of the main motor disorders of Parkinson’s disease.
Thanks to focused ultrasound, American researchers are using deep brain stimulation to significantly and immediately reduce this symptom in people with Parkinson’s.
A technique validated by the FDA
This clinical test involves directing ultrasound energy through the patient’s skull into the region of control of regular voluntary movements.
Among the 94 participants*, 69, placed awake with a transducer helmet in a magnetic resonance imaging scanner, received focused ultrasound. The other 23 patients underwent a sham procedure.
Using MRI images, scientists precisely locate the area to be treated and the temperature level for ablation.
The device used, called Exablate Neuro, has received approval from the United States Food and Drug Administration (FDA) to treat advanced forms of neurodegeneration in part of the brain.
*All participants had disabling tremors and had failed to respond to other treatments.
70% positive response to treatment
After 3 months of follow-up, 70% of patients treated with ultrasound responded successfully to the treatment. A year later, 2/3 of them continue to support this therapy with a significant improvement in:
- their tremors,
- their mobility,
- other physical symptoms.
According to study neurologist Howard Eisenberg, this type of therapy to manage the symptoms of Parkinson’s disease involves no incisions, which excludes any risk of serious infection, cerebral hemorrhage and other adverse effects.
3- Cell therapy to replace dopamine
On February 13, at Skane University Hospital in Sweden, a stem cell transplant was successfully performed on a patient with dementia. This clinical trial involved transplanting nerve cells derived from stem cells into 8 people with Parkinson’s to reduce their symptoms.
A stem cell transplant
Generated from embryonic stem cells, the transplant product STEM PD has the function of replacing lost dopaminergic nerve cells in the parkinsonian brain. A technique that seems to work:
The transplant proceeded as planned and the correct location of the cell implant was confirmed by magnetic resonance imaging.
The potential effects of the STEM PD product may take several years.
Patient has been discharged from hospital and assessments will be conducted as per study protocol
Gesine Paul-Visse, neurologist for the STEM-PD clinical trial
A clinical trial to watch
With an intervention of a few millimeters in the brain, the task remains delicate and the results are not expected for several years. In effect, further studies are needed to move STEM-PD from this first trial to a comprehensive treatment.
The selected patients were diagnosed with Parkinson’s disease at least ten years ago and are in a moderate stage of dementia. Researchers will carefully follow these patients and perform evaluations of cell survival and potential effects over the next few years.
In the meantime, the performance of this transplant is part of a revolution in the search for treatment against Parkinson’s disease.
From a cough medicine, to ultrasound, to cell transplantation, no approach is ruled out to find the next Parkinson’s treatment.
The one that will replace drug treatments like levodopa to act on dopamine. This ever more advanced and innovative research is accelerating progress towards a future where millions of patients will enjoy a better quality of life.
Sources :
https://cureparkinsons.org.uk/ambroxol-the-story-behind-the-medicine/
https://www.inserm.fr/dossier/parkinson-maladie
https://www.eurekalert.org/news-releases/981125
Etude du New England Journal of Medicine,
