The biotech’s ‘all new neurorestorative approach’ aims to rebuild and replace lost brain cells in Alzheimer’s that underlies clinical symptoms.
On the back of the trial, the company plans to launch a world-first human trial in 2024.
'Ray of hope'
The veterinary trial, led by Skin2Neuron and published this month in Stem Cell Research and Therapy, reversed the dementia-like syndrome that strikes down many older pet dogs with Alzheimer’s.
Dementia was reversed in more than half of the canine patients, with a ‘clinically meaningful’ improvement in 80%. Typically, improvement lasted around two years.
Skin2Neuron champions its new approach as a ‘ray of hope’ for Alzheimer’s disease: championing a completely different approach to the amyloid hypothesis of Alzheimer’s disease.
“Our target is the ultimate cause of dementia: lost neurons and synapses. We do this by microinjecting a patient’s own HFN cells directly into the hippocampus, the brain’s memory center and first area to be devastated by Alzheimer’s,” explains the company.
While its lead therapeutic target is Alzheimer’s, it says its technology also has potential to treat neurodegenerative conditions such as Parkinson’s disease, Amyotrophic Lateral Sclerosis and more.
Dog study
A dog’s thinking neocortex and hippocampus is similar to the human brain, says the company. Meanwhile, older dogs often develop a dementia syndrome similar to human dementia: becoming forgetful, irritable, lost, wandering around aimlessly, failing to recognize owners and experiencing disrupted sleep.
"Because of deep parallels between the canine brain and human brain, and canine Alzheimer's and human Alzheimer's, I started this trial 10 years ago with the assumption that if it's going to work in humans, then it needs to work in dogs first. And the results exceeded my wildest expectations,” said co-founder Professor Michael Valenzuela.
"The hippocampus, the memory center of the brain, was packed with baby neurons and new synapses, precisely where we delivered the cells. Compared to untreated dogs, it was like night and day".
Microscopic analysis confirmed the dogs had classic Alzheimer pathology: meaning the cell therapy worked in the setting of natural disease, a first of its kind, according to the company.
"Given our doggie patients also had many of the same health issues that older people face, it gives me even greater confidence," said Valenzuela.
Study: Valenzuela, M., Duncan, T., Abey, A. et al. Autologous skin-derived neural precursor cell therapy reverses canine Alzheimer dementia-like syndrome in a proof of concept veterinary trial. Stem Cell Res Ther 13, 261 (2022). https://doi.org/10.1186/s13287-022-02933-w