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Towards new drug treatments

Towards new drug treatments

Research to find drug treatments that slow, stop or even reverse MND.

A drug that slows the progress of MND. Even better, a treatment that stops the progress of MND or even promotes repair. This is what most people with MND want, and it is our ultimate goal too.
At the Euan MacDonald Centre, we believe that rigorous scientific research is the only way to ensure no opportunity is missed or stone unturned in our search for a cure. All our research is based on solid and hard-won evidence.

Clinical Trials

In January 2020, we launched MND-SMART, an innovative clinical trial for MND. MND-SMART is a pioneering clinical drugs trial in its design and reach. It will allow hundreds of people living with motor neurone disease (MND) across the UK to take part in tests of potential treatments.
For more information, visit the trial website at www.MND-SMART.org.

Repurposed drugs

The first drugs being tested in MND-SMART are repurposed drugs. Repurposing means using a drug that is already licesnsed for a different condition, which is already safety-tested and approved for use in people. This can save years, and it’s a lot cheaper too! The drugs are chosen in a systematic and unbiased screen of the scientific literature. For more information about drug choice, see the FAQs on the MND-SMART website.

 

Towards new drugs

At the same time, we are working towards finding completely new treatments for MND. Most of this research takes place in the lab, studying cells and the intricate mechanisms that go on within them (read more in our ‘Understanding motor neurons’ theme). This knowledge will be invaluable in developing new ‘designer’ drugs that might slow or halt MND, but it is inevitably slow.

While cell research is ongoing, we’re taking an additional approach that works much faster. We can use stem cells donated as a blood or skin sample by people with MND, and grow them in the lab. Then, in a series of "chemical cookery" steps over many months, we can turn these stem cells into living, functioning motor neurons to use in experiments. See more about this in our Stem cells and MND 'in a dish' research case study.

We have established a high-throughput drug screening platform that uses robotics and automated imaging to test thousands of 'small molecules' on these stem cells, to see if any make the motor neurons more healthy. These 'small molecules' can be obtained from pharmaceutical companies or from our own experiments. If one of these small molecules changes the rate at which a neuron in a dish dies, it might be able to be fast-tracked to be tested in people in MND-SMART.

 

Making a difference

The first gene therapy for motor neurone disease

Spinal muscular atrophy (SMA) is an infant-onset form of MND. Because scientists have pinpointed the exact genetic error that causes SMA, progress towards treatments is relatively advanced.

Meet the researchers

Watch MSc student Pratap Harbham talking about his research setting up drug screening on stem cell-derived motor neurons.

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