Estate of prestigious neurologist funds vital VU research

Researchers from Victoria University’s Institute for Health & Sport (IHES) have received a gift in memory from the estate of US neurologist Dr Charles A Bonsett, which will fund a year of research into treatments for Duchenne muscular dystrophy.

After referencing his work in her 2008 PhD thesis, VU researcher Dr Emma Rybalka and Dr Bonsett formed a friendship and regular collaborations across the globe until his death at age 98 in 2020.

The generous gift from the neurologist's estate will enable Dr Rybalka, together with Dr Cara Timpani and the IHES team, to keep Dr Bonsett’s methodology alive, with the goal of find an effective treatment for the debilitating disease.

The late Dr Bonsett dedicated more than 50 years of his professional life to a metabolic approach for treating Duchenne and Becker muscular dystrophies.

These inherited diseases of the muscles are caused by a missing protein, which triggers extensive muscle wasting. The Duchenne type is debilitating and fatal, causing those with the condition to lose their ability to walk during childhood, and die from cardiorespiratory failure in early adulthood. There is no known cure.

The funds will be used to progress the PhD research of VU’s Dr Cara Timpani. The research was supervised by Dr Rybalka and Professor Alan Hayes, and guided remotely by Dr Bonsett and his non-profit organisation, Dystrophy Concepts, and published in Nature journal, 'Scientific Reports' in January 2020.

Dr Timpani’s project was conducted to re-invigorate clinical trials using the enzyme adenylosuccinic acid (ASA) – which has a protective effect on muscles – begun by Dr Bonsett in the 1980s.

Using Dr Timpani’s research, the IHES team and researchers in the Australian Institute for Musculoskeletal Science (AIMSS) discovered a novel mechanism of action of ASA, with results published in January 2021 in Current Medical Research Opinion.

Dr Rybalka says of the gift:

“We are deeply honoured to receive this contribution from the estate of Dr Charles A Bonsett, a pioneering research clinician of his era. We were saddened to hear of his passing, but feel fortunate to have gotten to know him and to be continuing the ASA work in his memory.

"The funding will allow us to gain a better understanding of how ASA works in severe bursts of muscle-wasting, which is what happens in children as they grow.

"Another aspect of our research is studying the genes that are switched on and off by the drug, leading to the benefits in muscles. This sort of information is important when seeking regulatory approval for a drug to initiate human clinical trials.”

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