A new and exciting clinical trial has become the first multiple sclerosis (MS) research study to receive funding from the Federal Government’s newly created Medical Research Future Fund. The trial will be led by Professor Anne-Louise Ponsonby of the Murdoch Children’s Research Institute in Melbourne. The team will investigate whether a nutritional therapy that targets mitochondria, the energy producing factories in cells, can reduce fatigue and depression in people with MS.
Nearly all cells in the human body contain hundreds if not thousands of little structures known as mitochondria, these are the power plants of cells. They use some of the calories from the food we eat and the oxygen we breathe to generate the chemical energy needed to power all of our biological processes. If mitochondria are not working properly, our cells cannot function.
However, in the process of providing our cells with chemical energy, these powerhouses can also produce toxic by-products such as free radicals (highly reactive chemicals which can damage DNA and other parts of the cell). These free radicals are normally kept in check by natural antioxidants found in our bodies, but sometimes, if mitochondria become damaged, they can leak some of these by products into the rest of the cell.
In MS, these power plants are thought to get damaged in the brain cells. The damaged mitochondria can’t produce as much energy as the cells require, putting more stress on both the damaged nervous system and the mitochondria. The mitochondria may then add to the damage by leaking free radicals. Problems with mitochondria may underlie some of the common symptoms of MS, and are also considered to play a particular role in progressive forms of MS.
Australian scientists, led by Professor Anne-Louise Ponsonby, will now target mitochondria with a novel Australian-developed nutritional therapy. They have secured funding for a clinical trial of the therapy in one of the first rounds of funding released by the Federal Department of Health’s new Medical Research Future Fund (MRFF).
The trial will test whether this nutritional supplement can help alleviate fatigue and depression in people with MS. The therapy is a unique combination of around 20 ingredients that are specifically designed to support, protect and bolster the function of mitochondria.
The ingredients include biotin, folic acid and other vitamins and minerals. A number of the ingredients have already been shown to have some benefits in MS or in other health conditions. However, the combination is designed to be a multi-pronged approach to specifically support the function of the mitochondria to target the symptoms of MS.
The trial will randomly assign 150 people into two groups. One group will receive the treatment and the other will receive a mock treatment (placebo). All participants will be in the study for 16 weeks. Neither doctors nor patients will know who is getting the active treatment or placebo. This is known as a randomised, double-blind placebo controlled trial, which is considered to be the gold-standard method to test the efficacy of experimental new treatments.
However, the trial also incorporates many innovative features to ensure the team can gain the maximum information from a short clinical trial. They hope to reveal not just the efficacy of the treatment, but also shed further light on the biology of MS and the role that mitochondria play.
To do this they are using precision medicine and powerful MS clinical databases to track the participant’s outcomes. The team will sequence the DNA of the participants and use cutting edge technologies to detect chemical changes in the blood which help reveal whether the therapy is working as expected and, more importantly, how it is working and for whom it might be most beneficial. By linking with international MSBase clinical database and MS Research Australia’s Australian MS Longitudinal Study, the researchers will also be able to track any long term affects associated with the treatment.
This is an exciting trial as it is tackling one of the most significant symptoms for people with MS – fatigue. It is also uses cutting-edge technologies to scientifically test the outcomes of taking a combination nutritional supplement.
If the trial is successful it could be translated very quickly into clinical practice as the majority of the components are readily available in Australia, and the safety and tolerability has already been established in earlier studies.
As the funding announcement has only just been made, the team will now swing into action to get the trial up and running. Once it is ready to start recruiting we will bring you further news via our website, newsletters and on our MS Trials website.