Effects of photobiomodulation in mouse model of Sanfilippo syndrome

June 21, 2024

Grant Details

Project Title: Exploring novel photobiomodulation parameters in an animal model of Sanfilippo syndrome

Chief Investigator: Paul Austin, PhD

Study Time Period: July 2021- April 2023

Institution: University of Sydney and Flinders University

Types of Sanfilippo Studied: A

Types of Sanfilippo That Could Benefit: A, B, C, and D

Research Stage: Translational

Grant Information

Photobiomodulation (PBM) is a non-invasive therapy that uses low-level light in the visible red or infrared spectrum (600 – 1200 nm) to produce beneficial effects. There is evidence that PBM is neuroprotective and improves memory and movement performance in autism, traumatic brain injury, stroke, multiple sclerosis, Alzheimer’s and Parkinson’s diseases, and macular degeneration in both animal models and human trials.

PBM is inexpensive, can be combined with other treatments, has no known side-effects and, most importantly, it is available today.

Its mechanism of action is thought to be due to light being absorbed by special acceptor molecules within the neuron, with the most well-characterized being ‘cytochrome c oxidase’ within the mitochondria, the cells’ power station. This leads to increased neuronal ATP (“energy”) production, resulting in an increased capacity to resist cellular stress and dismantle clumps of protein, which are damaging to the cell.

Widespread beneficial effects of PBM act through changes in how genes are regulated to produce different proteins. PBM is associated with increased production of factors that promote neuronal health and new connections between nerve cells (synapses), and reduced production of inflammatory molecules.

The research team’s previous pilot study using a low dose of PBM at 670 nm for only 2-weeks in pre-symptomatic MPS IIIA mice provided evidence of reduced microglial activation in the retina and a 20% reduction in astrocyte activation in several brain regions, with both findings indicative of reduced neuroinflammation.

This project aims to investigate the beneficial effects of longer timespans and different doses of PBM in symptomatic MPS IIIA mice. This study will investigate different doses of 670nm light as well as the 904nm wavelength which can penetrate deeper into tissues.

The study will examine effects on the neurons, mitochondria, neuroinflammation, nerve growth factors, health of the retina, and abnormal protein clumping. Behavioral outcomes of movement performance and anxiety-like behaviors will also be measured in the mice.

The research team hopes the study will provide important learnings about the effects of PBM in MPS IIIA mice. If benefit is confirmed, this will lay the foundation for swift translation to children with Sanfilippo syndrome.

Grant Updates & News

June 2024

Investigators, who were supported by this grant, published their findings in the Journal of Neurochemistry on June 7, 2024. We encourage you to read more about their findings in the publication here.

April 2023

In April 2023, Cure Sanfilippo Foundation hosted a community webinar where the research team kindly shared information about photobiomodulation and updated the community research specific to Sanfilippo syndrome. We thank Dr. Paul Austin (University of Sydney, Australia), Dr. Adeline Lau (Flinders University, Australia), Prof. Kim Hemsley (Flinders Univ., Australia) and Prof. John Mitrofanis (Université Grenoble Alpes International, France) for their enlightening presentation and engaging discussion during the Q&A portion.

You can watch a replay of the webinar here.

Paul Austin (second from right) of The University of Sydney and members of his research team. Left to Right; Kevin Jin, Jackson Karrasch, Jayden O’Brien, and Dolores Huang.

A composite image of the Adelaide researchers involved with this study from both Flinders University and SAHMRI. Top row: Professort Kim Hemsley, Dr. Adeline Lau, Helen Beard, and Associate Professor Marten Snel. Bottom row: Thomas Windram, Barbara King, Daniel Neumann, and Dr. Paul Trim

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