A new therapy that appears to stop Parkinson’s disease “in its tracks” will begin phase one clinical trials in humans next year. Researchers at Australia’s University of Queensland (UQ) have published extremely promising results from their work demonstrating that a molecule called “MCC950” is capable of stopping the development of Parkinson’s in a mouse model of the disease.
Trent Woodruff, associate professor in the UQ Faculty of Medicine, explained in the video below that “Current therapies for Parkinson’s disease tackle the symptoms but not so much the progression of the disease, the underlying cause.”2
Prof. Woodruff explained that “The disease is characterised by the loss of brain cells that produce dopamine, which is a chemical that co-ordinates motor control, and is accompanied by chronic inflammation in the brain.
We found a key immune system target, called the NLRP3 inflammasome, lights up in Parkinson’s patients, with signals found in the brain and even in the blood. MCC950, given orally once a day, blocked NLRP3 activation in the brain and prevented the loss of brain cells, resulting in markedly improved motor function.”3
According to UQ, “There are no medications on the market that prevent brain cell loss in Parkinson’s patients, with current therapies focusing on managing symptoms rather than halting the disease. UQ Institute for Molecular Bioscience researcher Professor Matt Cooper said drug companies had traditionally tried to treat neurodegenerative disorders by blocking neurotoxic proteins that build up in the brain and cause disease.” 3
Toxins Set the “Brain on Fire”
According to another team member, Dr. Richard Gordon, “The approach that we’ve taken for this disease is to target microglia, the immune cells in the brain. With Parkinson’s and most diseases of aging there is an accumulation or buildup of these toxic protein aggregates in the brain.
What that does is trigger inflammation which sets the brains on fire, so to speak. What we did was this drug is target the immune system and it blocked the immune response and the inflammation associated with these protein aggregates. What we found was surprisingly that when you block this pathway these immune cells are possibly able to clear these toxic protein aggregates and that actually enables the neurons to function and survive better.” 2
Dr. Gordon observed, “We’re quite hopeful that this drug will provide a new treatment therapy for Parkinson’s disease symptoms and actually potentially slowing the progression of disease or even halt it in its tracks.”2 Phase 1 clinical trials with healthy volunteers are anticipated to start in 2019. Phase 2 trials in Parkinson’s patients will follow, hopefully in 2020 with patients in the UK. 3
The current study was funded by the Michael J. Fox Foundation for Parkinson’s Research and the Shake It Up Australia Foundation. According to The New Daily, the Ireland-based drug company Inflazome is also involved in the work to bring a potential treatment to market. 4
The Michael J. Fox Foundation “is enthusiastic about new targets and positive results,” but warns, “the therapy needs to go through several steps (and many years) of testing before it may be ready and valid for human use. (Unfortunately, clinical trials are the steps at which many therapies fail.)” 5
In the meantime, as millions of Parkinson’s patients wait for a therapy that prevents the debilitating loss of brain cells, more and more are turning to alternative therapies to manage the symptoms of their disease.
One area where there is great interest is medical cannabis, especially as more and more states legalize its use. According to the Parkinson’s Foundation, there are risks and benefits for people with Parkinson’s considering the use of medical cannabis. “Benefits include: Possible improvement in anxiety, pain management, sleep dysfunction, weight loss and nausea. Potential adverse effects include: impaired cognition (impairment in executive function), dizziness, blurred vision, mood and behavioral changes, loss of balance and hallucinations.”6
The Parkinson’s Foundation offers this final guidance: “Research is still needed to determine how medical marijuana should be administered and how its long-term use can affect symptoms of PD. To keep patients safe, states that legalize medical marijuana will eventually need to develop training programs for doctors and medical teams that prescribe medical marijuana. Consult your doctor to see if medical marijuana is an option for you.”6
1Gordon R, Albornoz EA, Christie DC, Langley MR, Kumar V, Mantovani S, Robertson AAB, Butler MS, Rowe DB, O’Neill LA, Kanthasamy AG, Schroder K, Cooper MA, Woodruff TM. Inflammasome inhibition prevents α-synuclein pathology and dopaminergic neurodegeneration in mice. Science Translational Medicine 2018 Oct 31;10(465). pii: eaah4066.
2University of Queensland Faculty of Medicine. “Cooling “brains on fire” to treat Parkinson’s. Video. 31 October 2018.
3University of Queensland. News. Cooling ‘brains on fire’ to treat Parkinson’s. Press Release. 1 November 2018.
4Elder J. World-first Pill May Stop Parkinson’s. 8 November 2018. The New Daily.
5Dolhun R. A Pill to Stop Parkinson’s? Foxfeed Blog. 9 November 2018.
6Parkinson’s Foundation. Medical Marijuana.
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