The NLRP3 inflammasome (green) is expressed by immune cells (red) in the brains of people with Parkinson's disease.
Photo: University of Queensland The NLRP3 inflammasome (green) is expressed by immune cells (red) in the brains of people with Parkinson's disease.
Photo: University of Queensland
Life Wellbeing Our scoop on experimental Parkinson’s treatment fuels hope – and a little anxiety too Updated:

Our scoop on experimental Parkinson’s treatment fuels hope – and a little anxiety too

Share
Tweet Share Reddit Pin Email Comment

Early in November, The New Daily published a story about a new therapy that appears to stop Parkinson’s disease “in its tracks”.

Our report drew hundreds of thousands of readers, scores of comments from people directly or indirectly affected by the disease, and led to a flood of emails to the researchers from people wanting to volunteer in human trials.

The therapy – which will begin phase-one clinical trials next year – was developed by researchers at the University of Queensland and is a world-first because it stops the death of brain cells in Parkinson’s disease (PD) sufferers, rather than managing symptoms.

You can read the original story here.  And the technical abstract of the research paper here.

Michael J. Fox foundation concerned by the fervour

So fervent was the response to the story that the Michael J. Fox Foundation for Parkinson’s Research – which is partly under-writing the new research – posted a blog from a US doctor cautioning people not to get their hopes up while acknowledging the breakthrough is a promising one.

The writer also admonished us for over-hyping the story – while the researchers were happy with how we characterised their work. The drug company working on the therapy, Inflazome, provided the following explainer video:

The foundation’s broader concern is obvious and reasonable: there are millions of people desperate for a cure, and the idea that a single pill, taken daily, could bring to an end the inflammation in the brain that causes much of the neuro-degeneration and loss of motor skills borders on the miraculous.

On the flip-side, a failure to deliver means one more bitter disappointment, one more dashed hope.

Any hope will do

We put this quandary to a University of Queensland Faculty of Medicine researcher, Associate Professor Trent Woodruff, who was quoted in our piece last week.

He said: “I don’t like to over-promise what may or may not happen … and drug development is a risky business. But what people really want to see is research that is intent on finding a cure.

“They worry the treatments they’re undertaking are not effective, which is why they want to try something new, anything. When there’s a lot of desperation, people want hope.”

He said that most of the emails he’d received were from family members of PD sufferers wanting to sign up their loved ones to the human trials. If phase one works well and the drug proves safe in healthy people, then phase-two trials with PD-afflicted people will start in 2020 – probably in Britain, says Dr Woodruff, where the drug company Inflazome is based.

Moving on from mice

Dr Woodruff said the company was currently doing its pre-clinical safety trials with higher mammals. The initial work, which involved three different models of Parkinson’s, was done with mice.

The University of Queensland and Inflazome are closely connected. Matt Cooper, professor at UQ’s Institute of Molecular Bioscience is co-founder and CEO of Inflazome.

On the company website he explains why the new therapy is different to current treatments: “Drug companies have tried for decades to develop medicines to combat neurodegenerative diseases by blocking the many different neurotoxins and amyloids that build up in the brain as we age.

“The problem is that if one toxin is blocked by a drug, another may still build up and cause disease. In contrast, our line of research focuses not on individual toxins, but instead on the immune cells in our brains that clear amyloid and other neurotoxins. These cells, called microglia, normally protect us from infections that can lead to encephalitis and meningitis.

“However, as we age, our immune system can become over-activated, which leads to neuro-inflammation. Over-active microglia can no longer function as efficient ‘cleaners’ of neurotoxins, but instead contribute to long-term damage in the brain.”

As professors Cooper and Woodruff explained in our story, the new therapy is a small molecule, MCC950, that appears to effectively cool this inflammation.

Dr Thomas Jung is Inflazome’s clinical development adviser and, suggested Professor Woodruff, a better target for emails from potential volunteers. He can be reached at t.jung@inflazome.com