NeuRA Magazine #23

NEW DISCOVERY IN MOTOR NEURONE DISEASE SPARKS NEW HOPE

Senior Principal Research Scientist, Professor Lars Ittner is working to understand Motor Neurone Disease (MND), and trying to develop novel therapeutic approaches for this devastating condition.

Specifically, Prof Ittner develops disease models that allow his team to understand the detailed cellular events that lead to the dysfunction and death of neurons and therefore to the loss of motor function. These models also allow him to test new therapies, which he hopes will delay and eventually prevent this disease.

In a recent interview, Prof Ittner shared details of his current research work, touching on new therapeutic directions and some exciting indicators.

Where is the future of MND research?

“I think that the future of MND has actually already started. It started with the bringing together of basic researchers like myself and clinicians, who deal on a day-to-day basis with the patients and their loved ones. We now develop research strategies together, instead of researching in separate streams, as it has happened for many years.”

What was it like when you first came in contact with someone with MND?

“The first time I was in contact with MND I remember it vividly. It was when I was in my surgical training to become an orthopaedic surgeon, many years ago. A relatively young woman with knee pain came to our clinic and we diagnosed problems with reflexes which unfortunately led very rapidly to a diagnosis of MND. Her whole world came tumbling down. It was a very emotional time for all of us in the medical team as we tried to support her grief. This event really ignited my research passion to want to understand what is really going on that leads to such drastic and rapid loss of motor functions and what can we do to stop it?“

Where has your research passion led you, and is there hope for any future breakthroughs?

“We have discovered in our animal models that there is potential for significant recovery from MND which is an exciting step, but we still have a long way to go.

Over the past 5 years or so, working with these models, it has been our clear intention to understand the mechanics that lead to MND. We actually got quite far on our journey to reveal the mechanisms of the degenerative process, when we made a discovery that really sparked hope for future treatments.

Specifically, we were able to genetically modify our disease models and that they actually stopped producing the toxins that lead to neuronal dysfunction. Amazingly, in two weeks these models recovered lost motor functions to a significant degree, and changed their behaviour back to normal.

So, while this is obviously not yet a treatment for patients, it clearly illustrates that we should be able to develop a treatment that is able to remove the toxins. We are excited to be at the beginning of understanding what drives MND, to allow the brain and spinal cord to recover function it has previously lost.”

This suggests that the brain is capable of recovering partially from MND, which in itself is a profound outcome of our research using models.

What are your future research plans?

“To develop a treatment for MND or even a cure, it is essential that we understand the mechanisms that underlie this devastating disease, so that needs intensive basic research in cellular models, animal models and translational research in patients, and a close collaboration with the pharmaceutical industry.

Research involving relevant disease models is unfortunately horrendously expensive. Therefore, we need the support of government bodies and industry, but also by the community to drive research in the direction of developing a new remedy and a cure for MND.”

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