Melbourne scientists find potential new therapy for Alzheimer’s and it will be trialled here first
Melbourne scientists have made a discovery that may be a “new frontier in the fight against Alzheimer’s disease” – and it could be as simple as a spray in the nose.
A world-first Alzheimer’s therapy with the potential to prevent or even repair the damage caused by the brain disease is being developed in Melbourne and could be ready for clinical trials within two years.
Internationally renowned IVF and stem cell pioneer Professor Alan Trounson will today launch a Melbourne-based biotech company to develop and trial the treatment to be delivered as a nasal spray.
“It may herald a new frontier in the fight against Alzheimer’s disease and other neuro-inflammatory conditions,” he said.
Professor Trounson said the company’s scientists believe the therapy will work by inducing the brain’s microglia (immune system) to actively clear and degrade amyloid protein aggregation and modulate brain immunity to reduce neuro-inflammation, which is present in Alzheimer’s.
“We are looking at the immune system in the brain and how we can do something about correcting the abnormal immune pathology,” he said. “There’s nobody else effectively taking this direct immune repair approach at present.”
Alzheimer’s disease is a condition that slowly damages memory, thinking, learning and other cognitive skills and is the leading cause of death in Australia.
This unique therapy will use extracellular vesicles (EVs) harvested from natural killer (NK) cells.
NK cells are the first responders of the immune system that seek out and destroy cells that shouldn’t be there. While this makes them ideal to fight cancer, they are prevented from crossing the blood-brain barrier, the fine membrane separating the two, which is crucial for treating neurological conditions like Alzheimer’s.
Professor Trounson is the inaugural chief executive and executive chair of the new biotech called Evinco Therapeutics which will develop the therapy and do safety studies in rodents starting next year.
Last week he stepped down as chief executive of Cartherics, moving into the role of deputy chair. Cartherics has made its name making and storing “billions” of frozen NK cells in its Clayton laboratory to treat cancer and endometriosis.
“We are pretty good at extracting EVs from NK cells and purifying them,” Professor Trounson said. “These EVs are tiny and will cross the blood-brain barrier to instruct microglia to destroy amyloid protein aggregates and dampen inflammatory cytotoxicity.”
He said the team will first look at it as a novel treatment for Alzheimer’s disease, but the potential for other neurological conditions such as Parkinson’s, concussion, traumatic brain injuries and the neurological pain condition fibromyalgia.
Professor Trounson said the idea came after he heard about a Korean company working with NK cells in the US to reduce infections in patients having radiotherapy or chemotherapy.
“Serendipitously, they treated an Alzheimer’s patient,” he said. “This patient hadn’t spoken for two years, and he started conversing normally.
“They then tried a younger patient, also with Alzheimer’s disease, and obtained a similar response.”
Professor Trounson said 13 Alzheimer’s patients have now been treated in FDA phase II studies in the US with this method and 12 had remarkably responded.
He said the Korean team were extracting the patients’ own NK cells, multiplying them in the laboratory and then injecting them back every couple of weeks into their vascular system.
“Probably the majority would end up in the body’s general circulation, concentrating in the liver and then destroyed,” Professor Trounson said.
“That’s why I want the delivery system to be primarily to the brain and the nasal route is the best way to effectively achieve this.”
He said the NK cells obviously do have some impact on microglia.
“(But) because of the blood-brain barrier, I don’t think these cells they’re giving to the patients can actually traffic to the brain, but it may be that the EVs that sprout off from these cells can enter the brain. They spit off a lot of these EVs to communicate with other cells,” Professor Trounson said.
“If you extract those EVs, which we’ve been doing, you can show they activate the microglia to destroy aggregated amyloid plaques and also have an impact on the tau proteins, both are connected to Alzheimer’s disease.
“We can see these having an impact in Alzheimer’s as we show their impact on inflammation and we know that they can pass through the blood-brain barrier because of their nature and tiny size.”
Professor Trounson said the next step was to establish proof of concept studies using EVs as a new therapy for Alzheimer’s and a major pharmaceutical company was already on board to help design these critical studies.
“Can this therapy reverse the effects of Alzheimer’s, we can’t be certain of that as yet; can it prevent it as a one-off therapy, that’s what we are also investigating,” he said.
“Since this isn’t going to be very expensive, EVs can be freeze-dried and made up into a nasal spray simply, multiple treatments are not really a problem for patients. I expect we will be able to try the treatment fairly soon in exploratory trials.
“I’m really excited about this because I can’t see any barriers to us finding out if it does work. And it is possible we will know within two years.”
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Originally published as Melbourne scientists find potential new therapy for Alzheimer’s and it will be trialled here first
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