People living with motor neurone disease (MND) are often faced with a decision about whether to try a particular medication. You should discuss any proposed medication or therapy with your healthcare team.
Medications for MND may assist in the following ways:
In Australia, Riluzole is available through the Pharmaceutical Benefits Scheme (PBS). This means the medication is subsidised for eligible people, making it more affordable. To access Riluzole, you need a special kind of prescription, called an “authority script”, which means the doctor must apply for permission from the government.
Your first authority script for Riluzole must be written by the neurologist who diagnosed you with ALS or PBP. Once the initial prescription has been approved, your GP can issue repeat prescriptions.
Most side effects from Riluzole are relatively minor. The most common side effects are fatigue and nausea.
Riluzole can affect liver function. If you have pre-existing problems with your liver, your doctor can advise whether Riluzole is right for you.
Edaravone is thought to have antioxidant properties. These properties protect nerve cells by mopping up damaging “free radicals” in the body. Research suggests Edaravone has most benefit when used early. It is given as an intravenous infusion by a doctor.
Edaravone (RADICAVA®) is not yet readily available in Australia.
The Australian Therapeutic Goods Authority (TGA) has approved (edaravone) for the treatment of ALS in Australia. However, RADICAVA® (Edaravone) is not yet available for people to buy through the Pharmaceutical Benefits Scheme (PBS). It is expected to become available in 2025.
Edaravone has been approved for treatment of MND in countries including Japan, Canada and the USA. It is not yet readily available in Australia.
Common side effects of Edaravone are bruising, problems with walking and headache. Fatigue has also been reported as a side effect.
In Australia, tofersen is only available to people living with MND who carry mutations in the SOD1 gene. Biogen, the pharmaceutical company that developed the drug, has made it available through their compassionate access program.
Tofersen reduces activity of the SOD1 gene and decreases levels of the protein. It is the first gene-related therapy to be approved for treatment of MND.
To learn more talk to your neurologist.
People living with MND may also use medications to manage symptoms associated with the disease. This might include medications to relieve symptoms such as:
Be honest and open about your symptoms when you talk to your healthcare team. This way, they can help you explore medications that can help keep you more comfortable.
Scientists around the world continue to do research to learn about MND. Part of this research is to develop medications that may slow, and one day cure, the disease. The research could help find new drugs that make a positive difference to people living with MND.
Generally, clinical trials are a high-quality form of research. The trials follow strict scientific processes. Clinical trials try to find out if the proposed medication works, and is safe.
Figuring out if a medication can safely treat MND is important because it:
Finding effective drug treatments for MND takes a lot of time and resources. Advances in research, like platform trials, seek to address this challenge.
New research offers hope. With more flexible approaches to research, the process is quicker. Researchers can now test multiple drug treatments at once, which saves time.
Find out more about research in Australia and around the world
People living with MND and their families understandably want to know about treatments that claim to treat or cure MND. Often, people come across these treatments while searching for information about MND on the internet.
Sometimes it can be hard to tell the difference between good quality health information and paid advertising. A good place to learn about alternative treatments is to visit the ALS Untangled website, which helps people find better quality health information online.
ALSUntangled scientifically reviews alternative treatments and posts the outcomes on their website. This information supports people living with MND to make informed decisions about these therapies.
Learn more about ALS Untangled
Check with your doctor or pharmacist before taking any new medication, even over-the-counter remedies. Checking helps make sure any remedy or medication is safe to take with your current medication.
To learn more about off label unproven treatments and right to try, read the International Alliance of ALS/MND Associations Scientific Advisory Council (SAC) briefing notes.
You may also like to read this blog post about finding reliable information.