In a new study, researchers have discovered a world-first new treatment that reverses the effects of memory loss associated with Alzheimer’s disease in a study of mice with advanced dementia.
The work builds on their work begun in 2016 involving a ground-breaking gene therapy that uses an enzyme that is naturally present in the brain, known as p38gamma.
The researchers found that p38gamma, when activated, can modify a protein such that it prevents the development of Alzheimer’s disease symptoms.
This latest finding has gone a step further and showed that the gene actually improved or restored much of the memory in advanced Alzheimer mice.
Importantly, their findings also suggest that gene therapy may be effective in other forms of dementia, including frontotemporal dementia which presents in much younger patients in their 40s and 50s.
The research was conducted by a team from Macquarie University in Australia.
Gene therapy is a process whereby genetic material is introduced into cells to replace abnormal genes or to make a beneficial protein.
The team showed that the new gene therapy is safe even at high doses and when applied long term, with no adverse events observed during the study.
The next step will be to transition to testing safety and efficacy in humans.
Macquarie University is currently undertaking a detailed assessment of the development and regulatory pathway required to evaluate gene therapy in human patients.
Partnerships with potential investors and pharmaceutical partners are also being actively explored.
The team says it will be exciting to see how over 10 years of basic research to understand the mechanisms of Alzheimer’s disease will finally transition into clinical development to eventually benefit those most in need, people living with dementia.
The researchers predict that the possible success of this new therapy could be within reach of humans in less than 10 years.
One author of the study is Dr. Arne Ittner.
The study is published in Acta Neuropathologica.
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