One of the best things about the December edition of Advance is that we get to announce the fantastic research projects and brilliant researchers we will be supporting in 2025. Once again, we have a strong focus on supporting up-and-coming Australian researchers and it is immensely gratifying to see a host of new names on the list of successful grants for this year. Projects we are funding include identifying environmental risk factors, developing new genetic therapies, how we can better track progression of the disease, how we can monitor patient care at night, and better manage cognitive change. This support totalled $2.5M and every one of those dollars came from the massively generous and resilient MND community. To ensure we spend these funds as well as possible we have expert researchers reviewing the grant applications and our august Research Committee finalise the grant selection. For the first time this year we had a person with lived experience participate in the Research Committee final discussions which provided a valuable perspective to aid the decision-making process.
Treatment options around the world continue to expand. Biogen's genetic therapy for SOD1, Qalsody (tofersen), has continued to gain regulatory approvals with China giving conditional approval following earlier approvals in the US, Canada and Europe. Biogen are currently preparing an application to the TGA for approval in Australia. Tofersen is currently available in Australia for eligible patients through Biogen's Early Access Program. A Japanese company, Eisai, was granted approval for a MND treatment “Rozebalamin® in Japan. This drug is a very high dose of the biologically active form of vitamin B12 and is thought to protect motor neurons. Neurizon (formerly PharmAust) are planning a Phase 2/3 trial for their treatment NUZ001 (formerly Monepantel) with the US-based Healey Platform Trial. Although this will mean the trial will not have any Australian sites, this approach allows for multiple investigational drugs to be tested and evaluated simultaneously which accelerates the outcomes from trials.
Although not technically a “treatment”, Synchron have announced positive results of their COMMAND brain computer interface (BCI) device trial, demonstrating that simple, thought-derived expressions of intent could be converted into digital actions on computers. The Synchron BCI was supported in its early days by one of our Innovator grants, so it is gratifying to see our support contributing to real clinical advances.
Another marker of how our approach of funding early career stage researchers and new ideas to build world-class Australian MND research is the progression of projects through to larger grants. The recent FightMND grant announcement features many projects initiated with MND Australia funding that have moved through the development pipeline towards potential clinical use.
On the 24th of June 2024, MND Australia hosted an event at Australian Parliament House in Canberra and awarded lifetime achievement medals to 30 people who have made a significant contribution to the MND community over the last 30 years. Many researchers were included in this list as well as Janet Nash who headed up MNDRA from its inception to 2019.
In a busy year, MND Australia also hosted the very successful 3rd Australian and New Zealand MND Research Symposium in Melbourne on the 27th and 28th of August. We had a record 300+ attendees. Highlights included talks from people with MND and world-leading international researchers, ground-breaking research from fantastic Australian researchers and some great debates.
In these tough economic times, we continue to be amazed and humbled by the depth and loyalty of your support.
Thank you.
Dr Gethin Thomas, PhD
