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Current Repurposed Drug Trials in Parkinson’s Disease

Drugs that once treated conditions such as type 2 diabetes, cancer, high cholesterol and dementia are now being evaluated in repurposed drug trials in Parkinson’s disease. To find out more, you can read our summary here.

What is a repurposed drug?

A repurposed drug is a drug that has been developed to treat one type of disease, but is being utilised in the treatment of another disease.

The benefits of repurposed drugs are that they have already gone through the expensive and time-consuming clinical trial process to get them to market. Therefore, they can be less costly to develop and more readily available than novel therapies.

Many repurposed drug trials are currently underway in Parkinson’s disease. Examples include:

  • Exenatide – a drug currently used to treat type 2 diabetes, acts on the GLP-1 receptors in the pancreas and brain. Promising research from the UK has shown improvements in motor symptoms of PD in those participants receiving Exenatide, however more research is needed.
  • Liraglutide and lixisenatide – are newer drugs that also treat type 2 diabetes. Initial rodent studies found these drugs were more effective at preventing brain cell death than Exenatide. Currently, there are two trials underway to investigate the effects of these drugs on PDin the US and France.
  • Statins – are often prescribed to help lower cholesterol in people at risk of cardiovascular disease. Research in rodent models suggest statins have protective effects on PD progression, such as; reducing inflammation, oxidative stress damage, as well as reducing the build-up of alpha-synuclein proteins. Currently, a randomised control trial is underway in the UK to assess the effectiveness of a statin – Simvastatin on PD symptoms in 235 patients with mild PD symptoms.
  • Ursodeoxycholic acid (UDCA) – a drug typically used to treat liver disease has been implicated in the restoration of mitochondrial cells (which fuel our cells) in a UK based study. Impairments in mitochondrial function are a key contributor to brain cell death in PD.
  • Nilotinib – used traditionally to treat a form of leukaemia, however recent research has found Nilotinib reduces brain cell death by binding to specific proteins in the brain. A 2015 study found patients who took Nilotinib had a reduction in PD-related motor symptoms.
  • Deferiprone – is used to treat people with excess iron, a common symptom of PD, thought to be due to excess iron found in dopamine-producing brain cells. Promising research from animal models has led to a current European-based trial with over 300 participants to investigate Deferiprone in slowing the progression of PD.
  • Isradipine – is used to treat high blood pressure. It works by blocking the uptake of calcium into cells. Research has found Isradipine also works on dopamine-producing brain cells, leading to reduced calcium uptake and subsequent oxidative damage. A multi-centre clinical trial involving 336 newly diagnosed PD patients is currently underway across the US and Canada. The trial is expected to finish at the end of 2019.
  • Ambroxol – typically used as a cough medication to help reduce the build-up of mucus, has had positive effects in the treatment of PD through increasing the activity of a protein called glucocerebrosidase (or GCase), which control alpha-synuclein levels – a key protein implicated in PD. Current research is underway in the UK and Canada to investigate Ambroxol in PD patients.
  • Rivastigmine – is currently used to treat dementia but has shown promise in PD patients via its ability to increase acetylcholine levels, implicated in memory and concentration. A UK-based study recruiting 600 PD patients is currently underway to investigate the effect Rivastigmine has on PD symptoms.

Repurposed drug trials will soon be commencing in Australia through the Australian Parkinson’s Mission. See our next blog for further information.

The information provided in this article is of a general nature only and is not treatment advice. Recommendations regarding therapy can only be made on a case by case basis, discussed between a patient and their treating doctor/s.

The information in this article was current at December 2019.

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Drs Cook & Silberstein feature on the cover of the North Shore Times/Mosman Daily

Neurosurgeon Dr Raymond Cook and Neurologist Dr Paul Silberstein feature on the cover of the North Shore Times/Mosman Daily in the story ‘Brains trust: The north shore team taking the fight to Parkinson’s disease’.

The story talks about the work both doctors do for people that are living with Parkinson’s disease and features an interview with a Parkinson’s patient, Carole McFadden, whose life has profoundly changed after recently having deep brain stimulation surgery at North Shore Private Hospital.

Shake it Up Australia Foundation founder, Clyde Campbell, also discusses the journey that led him to launch the foundation, and the impressive work they do to fund medical treatments with the aim of finding a cure for Parkinson’s disease.

The full story can be accessed online at http://newslocal.smedia.com.au/north-shore-times. Click on ‘back issues’ to access archived editions of the weekly newspaper.

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The novel GDNF trial yields mixed results

The novel GDNF clinical trial offered hope for restoring brain cells in people with Parkinson’s Disease. Including 41 people with Parkinson’s Disease aged between 41 and 72, the clinical trial was designed to investigate whether GDNF could slow, stop or reverse Parkinson’s Disease. Results were recently published. Read our study summary here.

Neurotrophic factors are small proteins produced in the brain that support the growth, survival and differentiation of brain cells. These proteins are important for maintenance of brain health and consequently there has been considerable interest in whether these proteins might improve survival or indeed assist regeneration of brain cells that are affected by Parkinson’s disease. 

From 2012 to 2017, UK researchers conducted a novel clinical trial looking at the effects of delivering one such Neurotrophic factor, Glial Derived Neurotrophic Factor (GDNF) directly to the brain, using a new type of surgically implanted device. Link to article here https://www.parkinsonsmovement.com/the-gdnf-trial/ 

Including 41 people with Parkinson’s Disease aged between 41 and 72, the clinical trial was designed to investigate whether GDNF could slow, stop or reverse Parkinson’s Disease. 

The group, all of whom had been diagnosed around eight years ago, were split into two smaller groups who were allocated either a placebo or the GDNF treatment. 

Using a device mounted on the skull, the researchers were able to directly infuse GDNF into the putamen – an important brain region that is affected by PD. Study participants received either placebo or GDNF treatment every four weeks for 40 weeks. 

After the 40-week period, the researchers noted that there was “no overall difference between the GDNF and placebo group” in terms of their movement scores. However, nine people showed what researchers called a “clinically significant improvement”. 

The researchers then conducted an extension study for an additional 40 weeks and again found no clear difference between patients who had received GDNF versus placebo. 

Overall, neither the GDNF study nor the extension study helped to improve movement-related symptoms in people with Parkinson’s, ultimately leading to the conclusion that GDNF infusions at the dose and for the duration used did not improve Parkinson’s disease. 

Despite these disappointing results, the study demonstrated that direct monthly brain infusions of substances that might treat Parkinson’s disease is feasible and tolerable, thus providing an important platform for future trials into possible therapies for Parkinsons’s disease. 

A documentary regarding this trial has been produced by the BBC: https://www.bbc.com/news/av/stories-47483307/the-radical-drug-trial-hoping-for-a-miracle-parkinson-s-cure

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World Parkinson’s Day Fundraiser at NSPH


Thank you staff and visitors to North Shore Private Hospital for supporting Shake it Up Australia Foundation for Parkinson’s Research on World Parkinson’s Day. 100 beautifully decorated cupcakes on our cupcake stall sold out in no time!

All monies raised at the yearly April 11 World Parkinson’s Day fundraiser go to Shake it Up Australia Foundation to support the amazing work they do for Parkinson’s research.

For more information on Shake it Up, head to www.shakeitup.org or follow them on Facebook at https://www.facebook.com/shakeitupaust/https://www.facebook.com/shakeitupaust/

The Sydney DBS surgical team
North Shore Private Hospital Nurse Unit Managers, Karen Morrison and Morena Hill from our Neurological ward with NSPH CEO, Richard Ryan

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Travelling with Parkinson’s Disease

If you’ve been diagnosed with Parkinson’s Disease, you might be wondering about whether it’s safe to continue travelling – both at home, and overseas.

The good news is that most people living with Parkinson’s disease continue to be able to travel for many years after the diagnosis. Parkinson’s disease might mean you need to plan your trip in more detail than you have previously.

Here are some tips that you may find helpful for planning your vacations and staying safe while you travel.

Plan ahead

Before you book a holiday or short trip, you may wish to talk to your GP or specialist about your condition and ask if there is anything specific you need to keep in mind.

  • Consider asking your doctor to write a letter about your condition in case you need to visit a doctor while you’re travelling.
  • You may wish to arrange to have this letter translated into local languages if you’re travelling to countries where English is not spoken.
  • Learning how to translate Parkinson’s Disease can also be a helpful step to take before you travel.
  • Consider obtaining a written list of your medications from your doctor or pharmacist.
  • You might also want to investigate how to access medical services particularly if you are travelling to more remote or non-English speaking destinations.

Book in advance

Booking your accommodation and transport in advance can make travelling less stressful. If you are using a travel agent, you might want to ask about special assistance for people with Parkinson’s disease.

If you are flying to your destination, it’s worth thinking about whether it would be best for you to fly without a stopover, or if you would find it easier to break up your trip.

Some other points to keep in mind when you’re booking your hotel and flights include:

  • Location of hotel rooms – some people with Parkinson’s disease prefer to request ground floor rooms or rooms close to the lifts.
  • Rooms with extra space – wheelchair accessible rooms often provide more space, particularly in the bathroom.
  • Travelling to and from airports – plan your transport to and from the airport.
  • Travelling within airports or shipping terminals – using a wheelchair to travel through security, customs and immigration can reduce fatigue. Many airports provide ground staff that are able to assist with this.
  • Driving – if you are able to drive, check any restrictions in local areas.

Get travel insurance

Travel insurance is important for all travellers, but it can be especially important for travellers with special needs.

Consider talking to your insurer about your Parkinson’s disease, and enquire if there are any restrictions on your insurance cover. The extent of cover can vary widely between insurers, and you may find it useful to shop around for a policy that best covers your circumstances. Confirming your plan covers you for Parkinson’s disease internationally as well as locally can give you peace of mind before you travel, and can be very important in the event of an accident or illness while you are away.

Research your itinerary

If you’re driving, find out about car parking and how close this is to the transport terminal. Sketching out a rough itinerary can be helpful in thinking out if there is anything that might make it difficult for you to get around. If you’re driving, find out about car parking and how close this is to the transport terminal.

Medication planning

Talk to your doctor about how to take your medications while you’re travelling, particularly across time zones and consider finding out how to access medication overseas if necessary – including any restrictions you need to be aware of. A back up supply of medication is always a good idea.

For aircraft travel, you may wish to consider separating your two sets of medication between your carry-on bag and stowed luggage.

Further information about travelling with medication can be found on the Department of Foreign Affairs and Trade website https://smartraveller.gov.au/guide/all-travellers/health/Pages/medicine.aspx

References

The information provided in this article is of a general nature only and is not treatment advice. Recommendations regarding therapy can only be made on a case by case basis, discussed between a patient and their treating doctor(s).

The information in this article was current at January 2019.