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:
- by slowing down progression of MND (Riluzole and Edaravone)
- managing symptoms associated with the disease (e.g. pain, drooling).
What is riluzole?
Riluzole is the active ingredient in medicines sold under various names, including Rilutek® and Teglutik®. Riluzole is a prescription medicine that may be prescribed for people with some forms of MND. It may be taken as a tablet or liquid.
How does riluzole work?
Research shows that riluzole can slow down the progression of amyotrophic lateral sclerosis (ALS) and progressive bulbar palsy (PBP), the most common types of MND. Recent research suggests that taking riluzole may prolong life [GS2] by 6 to 19 months. Riluzole is most effective when started as soon as possible after diagnosis.
In ALS, it is thought that motor neurons release too much of a neurotransmitter called glutamate. Glutamate stimulates and excites motor neuron cells. When too much glutamate is released by the neuron sending a signal, it can overstimulate the neuron. This process is called excitotoxicity.
Riluzole lowers the amount of glutamate around neurons. It protects motor neuron cells from excitotoxicity and further damage.
How do I get riluzole?
Riluzole is listed on the Australian Pharmaceutical Benefits Scheme (PBS). It is available from pharmacies on prescription.
Your first prescription for riluzole must be written by the neurologist who diagnosed you with ALS or PBP. After that your GP can also write repeat prescriptions.
How do I take riluzole?
Your neurologist will give you instructions on how to take riluzole. It is normally taken every 12 hours as either a tablet or a liquid, orally or through a PEG.
What are the side effects of riluzole?
Most side effects from riluzole are minor. The most common side effects are fatigue (tiredness) and nausea. If you have side effects, speak to your doctor.
Riluzole can affect liver function. If you have pre-existing problems with your liver, your doctor will advise whether riluzole is right for you.
Where can I get more information about riluzole?
Your neurologist and GP can give you advice about riluzole. If you have been refused access to riluzole under the PBS and think you are eligible, talk with a neurologist, your MND clinic or state MND Association.
What is edaravone (RADICAVA®)?
Edaravone is the active ingredient in a treatment sold under the product name RADICAVA®. It was developed in Japan and originally used to treat stroke patients. In 2015 edaravone was tested for use in people with ALS. Results suggest it slows the progression in a small group of people with ALS, with the otential to help these people preserve function longer.
How does edaravone work?
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.
How do I get edaravone?
RADICAVA® IV infusion (edaravone) is listed on the Australian Pharmaceutical Benefits Scheme (PBS) and available on prescription.
RADICAVA® IV must be first prescribed by a neurologist. It can only be prescribed to people with ALS (the most common form of MND) within two years of symptom onset.
To find out whether edaravone is right to you, talk with your neurologist.
How do I take edaravone?
RADICAVA® IV (edaravone) is not a tablet. It is given as an intravenous (IV) infusion by a health professional, usually a nurse. An IV infusion is a way of giving medicines, fluid, blood or nutrition directly to the bloodstream via a vein.
Infusions are usually given in an infusion clinic at a public or private hospital. Some people may also be able to receive edaravone infusions at home. This is through a private provider, so can be expensive. However, costs may be covered by private health insurance. To find out if this service is available in your area, visit chemo@home.
What else do I need to know about the treatment?
RADICAVA® IV is given over a 28-day cycle.
In the first cycle, infusions are given every day for 14 days in a row. This is followed by 14 days of no treatment. In subsequent cycles, 10 infusions are given over 14 days, followed by 14 days of no treatment.
Each infusion takes around an hour.
What are the side effects of edaravone?
Common side effects of edaravone are bruising, tiredness, headaches and problems with walking.
Where can I get more information?
To find out more about RADICAVA® IV infusion, talk to your neurologist or visit the RADICAVA® Consumer Medicine Information (CMS) summary on the TGA website
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:
- drooling
- cramping
- pain
- shortness of breath
- constipation
- anxiety
- depressed mood.
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:
- reduces the risk of side effects, illnesses and other more serious health problems from taking a new kind of medication
- helps find the right dosage and best way to take a medication
- finds out if a medication works for broad range of people
- makes sure research focuses on drug treatments that are more likely to benefit people living with MND.
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.
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.
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.
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