New short film showcases progress in Motor Neuron Disease research at UCL

A new short documentary showcasing the work into developing a treatment for Motor Neuron Disease (MND) has just been released.

The film features interviews with Professor Pietro Fratta and Professor Elizabeth Fisher (UCL Queen Square Institute of Neurology) about the progress in research for a new gene therapy that could be used to treat patients with MND.

In 2021, researchers worked out that a major cause of damage to motor neuron cells was the loss of a protein called UNC13A, which helps nerve cells pass messages to each other. In patients with MND, the instructions for how to make this protein becomes corrupted and the cell is no longer able to make UNC13A.

This discovery is now being used to develop treatments that target UNC13A with a £78m investment by a UCL spinout biopharmaceutical company, Trace Neuroscience. The company hopes the therapy will move to human safety trials next year, followed by full clinical trials.

The film explores a remarkable new technique for delivering gene therapy, devised by Professor Fratta and Dr Oscar Wilkins (Francis Crick Institute). The new delivery system ensures that the gene therapy only activates in cells affected by MND, while remaining inactive in other cells. This removes the risk of side effects in those cells.

Professor Pietro Fratta said: “If this gene therapy works, it would be incredible. It would bring lots of optimism to such a terrible disease. I really hope this can get to patients in less than two years from now”.

Professor Elizabeth Fisher said: “Going from bench to bedside, from basic research to treatments that work for patients, is usually a very long process. Watching the pace of change here is remarkable. This has been a very difficult field to be in for so many years. It’s astonishing.”

The documentary was produced by Quickfire Media. The filmakers will continue to follow the progress of the research team over the next few years.

Links

• Watch the documentary on Vimeo
• Motor Neuron Disease Centre
• Professor Pietro Fratta's academic profile
• Professor Elizabeth Fisher's academic profile

Image credit: Quickfire Media