Why this statement?
As neurologists specialising in the care of people living with motor neuron disease, we are receiving increasing enquiries about a drug called ellorarxine. Many of our patients are asking for access, when it might be available, or why we are not trialling this drug.
How are new treatments approved?
To understand the answer, it is helpful to know what is needed for a drug to be available either in a trial or for prescription.
For a drug to be authorised for a clinical trial there needs to be evidence that the drug might work and that it is safe in the doses needed to reach the nervous system in humans.
A drug cannot be prescribed unless clinical trials have been performed to confirm safety, to understand the side effects, and to test if there is an effect on MND in people. Gathering this evidence is essential to allow approval for a drug.
This process typically takes many years. To speed it up, it is important to collect the right evidence. A group of patients, patient charities, medicine discovery experts, MND scientists, and MND doctors came together to create a roadmap for the first part of this process: showing that the drug might work. You can read about it here.
At the UK MND Research Institute, which is a disruptive, nationwide, patient-led partnership of scientists and doctors, we are building a set of tools to support the whole pipeline needed for drug development and testing, making it more reliable and faster. You can read about what we are doing here.
What about Ellorarxine?
Ellorarxine is just one of many early-stage compounds being developed as possible treatments for MND. Hundreds reach this early stage of promising results. Of those, only some can be used safely in people without causing intolerable side effects and can be tested in clinical trials.
To put this in perspective, over a 27-year period from 1996, of those hundreds of possible treatments, only 38 made it to a clinical trial involving more than 100 people. Less than half that number reached the final step before approval, with nearly all failing to show a benefit. A drug showing some effect on MND from those trials is riluzole.
There is currently no evidence that ellorarxine is safe in humans. However, if this evidence becomes available, we would be delighted to work with the company involved to advise on the fastest way to assess it in clinical trials.
Testing a drug takes a lot of time, a lot of commitment from people living with MND, and a lot of money. We must be sure that we put our efforts in the right place to find new and effective treatments as quickly as possible.
