Clinical Trials (Part 1) – Platform Presentations: Highlights from Perth

This blog is part of the ‘Highlights from Perth’ collection of articles, where you can read about the content of some of the talks and posters presented at the 30th International Symposium on ALS/MND. All presentations have a code beginning with ‘C’ followed by a number (e.g. C50). This will help you locate the specific abstract mentioned throughout the post in the official abstract book.

Researchers are invited to present their work as either a platform (oral) presentation or as a poster. Session 2B of the Symposium’s platform presentations looked at clinical trials. Here we summarise the session with four talks reporting concepts, updates and results of the trials being investigated. This includes the Healey ALS Platform Trial, Ibudilast, NurOwn, and Tofersen.

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How animals are helping to improve our understanding of MND

‘From antibiotics and insulin to blood transfusions and treatments for cancer or HIV, virtually every medical achievement in the past century has depended directly or indirectly on research using animals’ – from the Royal Society’s position statement on the use of animals in research.

We know that talking about using animals in research is an emotive topic. We appreciate that some people will never accept that using animals in research is necessary, and we understand that it is not our place to try and influence anyone’s opinion on the use of animals in research. The purpose of this blog is to explore how using animal models of MND can further our understanding of this devastating disease, and how animals make it possible for potential new treatments for the disease to move forward into clinical trials in people.Read More »

Tofersen: antisense oligonucleotide drug shows promising results in Phase 1/2 trial

A recent press release by the pharmaceutical company Biogen reported preliminary results from an ongoing clinical trial investigating a form of precision therapy in people with SOD1-related MND. This drug, known as tofersen, is now in the final stages of Phase 1/2 testing in centres across the world, including Sheffield in the UK.

Tofersen is an antisense oligonucleotide (ASO), designed to prevent the faulty disease-causing protein from being made. Proteins, the building blocks of the body, are created from our genetic information (DNA) via its photocopy (RNA). If a piece of DNA is damaged, the RNA will also be damaged, leading to formation of a faulty protein and creating issues in the body. Tofersen is a synthetically-created RNA directed to stick to the faulty photocopy (RNA) preventing it from making faulty proteins.Read More »