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New therapy in the pipeline for Parkinson's disease?

Posted 14 March 2019 in Men's Health, Womens health

A brain scan showing substantia nigra in red. Source: medicalimages.comParkinson’s disease affects one in every 500 people, with symptoms beginning to show over the age of 50 in most cases, but for 5% of people with the condition, symptoms will first appear before the age of 40. It can have a devastating impact on a person’s quality of life, with movement, balance, memory, mental health and sleep all having the potential to be affected. Current treatment aims solely to ease symptoms and does not cure the condition. But a recent trial of an innovative therapy has hinted at new hope for those with Parkinson’s disease. (1, 2)

What is Parkinson’s disease?

Parkinson’s disease is a neurodegenerative disease involving damage and death of dopamine-releasing nerve cells in a region of the brain known as the substantia nigra, which regulates body movements. Dopamine is a neurotransmitter, which is a chemical which acts as a communicator between nerve cells. Therefore, the diminution in dopamine levels in the brain caused by the death of these cells reduces intercellular communication relating to movement and leads to tremor, sluggishness and stiffness. Symptoms do not usually show until only around a fifth of these nerve cells remain. (2, 3)

In addition to the primary movement symptoms, people with Parkinson’s disease can also experience a deterioration in mental health, difficulty keeping balance (and therefore greater risk of falling), insomnia, reduced sense of smell and poor memory. (2)

What is the outcome of the recent trial?

The results of a recent trial of a radical treatment for Parkinson’s disease have been released. The treatment, known as glial cell line-derived neurotrophic factor (GDNF), is an entirely new approach, administered via a ‘port’ that is embedded in the side of the patient’s head. This allows the drug to be delivered directly into the brain, where it then acts on dopamine nerve endings. (1)

Before the trial, the 35 participants had surgery which involved embedding four tubes into their brains. These tubes would be the route of administration of the drug into the brain, with the outer end being attached to the port where the drug would enter the tube. (1)

The trial lasted nine months, during which half of the participants were administered with a dose of GDNF every month, with the other half being administered a placebo infusion (containing no drug). For those given the drug, brain scans after the trial suggested that the progression of the disease had been significantly reversed. The average length of time since diagnosis for the trial’s participants was eight years, but the post-trial scans appeared to show features typical of brains just two years after diagnosis. This implies that GDNF can revitalise dying nerve cells and restore their function. (1)

However, it is too early to know whether GDNF will become a routine treatment at this stage – more trials involving larger numbers of participants, longer treatment courses and different doses will need to be performed before its licensing is considered. The placebo effect will need to be ruled out (this is when a participant being given a placebo experiences an improvement in their condition or symptoms). (1)

Regardless, it is very exciting that new modes of drug delivery such as this are being developed and tested. If successful, the approach may even be used to develop treatments for other conditions affecting the brain, including Alzheimer’s disease and brain tumours. (1)

References

  1. Therrien A (2019). ‘Radical Parkinson’s treatment tested in patients’, BBC News. Available at: https://www.bbc.co.uk/news/health-47370498

  2. NHS. Parkinson’s disease – Overview [cited 1 March 2019]. Available at: https://www.nhs.uk/conditions/parkinsons-disease/

  3. NHS. Parkinson’s disease – Causes [cited 1 March 2019]. Available at: https://www.nhs.uk/conditions/parkinsons-disease/causes/

    Author
    Gabby Gallagher MPharm

    Medically reviewed by
    Superintendent pharmacist Margaret Hudson BSc(Hons)MRPharmS 
    14/03/19

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