Turning science into hope: MND research update

Motor neurone disease (MND) research is advancing at pace, with new discoveries bringing hope to those affected. The team at the University of Auckland’s MND Research Lab is working to uncover the genetic causes of MND in New Zealanders, develop targeted therapies, and improve support for families. Thanks to the generosity of MND NZ and the Walk to Defeat community, significant progress has been made—from identifying genetic markers to testing promising new treatments.

As they continue this vital work, they are pleased to share some of their latest findings and advancements, which have been made possible through your ongoing support.

Kia ora fellow walkers and the MND community,

It's our pleasure to update you on our recent progress in MND research and to express our gratitude for the support of MND NZ, which was made possible by the success of the Walk to Defeat MND events.

Advancing genetic research to uncover MND’s causes

Our team of 11 researchers—including postdoctoral fellows, PhD candidates, Masters students, a research technician, and A/Prof. Scotter—is working to identify genetic causes of MND in New Zealanders. By understanding the underlying genetics, we aim to develop targeted therapies and provide meaningful support to families affected by MND and related diseases.

We have performed genetic testing for 197 people with MND, helping them understand whether gene errors may have played a role in their condition. Knowing their genetic status can be crucial—some may gain access to emerging gene therapies, while others can find reassurance about the risks for family members.

Solving genetic mysteries and shaping testing guidelines

Our research discovered gene errors in approximately 5% of people with no family history of MND. Additionally, we have solved the mystery gene for 12 families, including three families with extremely rare gene errors that are now under further investigation. These findings directly shape genetic testing guidelines for the public health service, ensuring that more people receive timely and accurate genetic insights.

Progress in genetic therapies and drug development

Beyond identifying genetic causes, we are actively working on gene-targeted treatments. Our research focuses on small DNA-like molecules, similar to the MND drug Tofersen, that are designed to ‘switch off’ harmful gene errors.

By testing these molecules in cells grown from MND patients, we have found that some can partially or even fully suppress faulty genes—a promising step toward future treatments. Our work greatly benefits from collaboration with Professor Chris Shaw, whose UK-based gene therapy company runs clinical trials on similar therapies. With his expertise and our team's dedication, we are making meaningful strides toward new treatment options.

A partnership driven by knowledge, action, and hope

At the heart of our research is a deep commitment to those living with MND. By working closely with families, we are gaining insights that help shape better testing, treatments, and support systems. Every discovery we make is driven by a shared mission: to bring knowledge, action, and hope to those affected by MND.

We are incredibly grateful for the support of MND NZ, and all Walk to Defeat participants. Your fundraising efforts directly fuel our research, making breakthroughs possible.

Thank you for walking with us on this journey. We look forward to sharing more of your progress soon.

Ngā mihi nui,

The MND Research Team
Centre for Brain Research, University of Auckland