SYT13: A protective gene against motor neuron degeneration?
The protein synaptogamin (SYT13), which was found to be strongly activated in neurons that control eye movement (which are largely unaffected in MND), protected vulnerable motor neurons from degeneration in cell and animal models of the disease. This suggests a gene therapy based on this protein’s related gene SYT13 could be a therapy for the disease. The data collected suggests that the protective effects of SYT13 are intrinsic to neurons, meaning they occur naturally. This study serves as proof-of-concept for a strategy of identifying factors that intrinsically promote survival as a means of finding new therapeutic targets for MND
Reldesemtiv granted orphan drug status by the EMA
The European Medicines Agency (EMA) has granted orphan drug status to reldesemtiv, developed by Cytokinetics in collaboration with Astellas Pharma, for the treatment of MND. This follows orphan drug designation of reldesemtiv by the US Food and Drug Administration (FDA) in December last year. Orphan drug status means that the company will have access to assistance in developing trial protocols and a 10-year period of market exclusivity once the treatment is approved. Reldesemtiv is a fast skeletal muscle troponin activator which means it can ease muscle contraction with minimal nerve stimulation to slow the decline of muscle function associated with MND. A Phase 2 trial – FORTITUDE-ALS – compared the effects of reldesemtiv in people who were within two years of diagnosis before entering the study to a placebo group. It showed that the drug slowed decline in lung function and ALSFRS scores in participants with middle- to fast-progressing MND but not in those with a slow-progressing disease. Orphan drug status is given to drugs that are being developed to treat rare diseases and assist companies with development costs which would not be recouped once the drug is licensed. This acts as an incentive for the companies developing drugs to continue through the clinical trials and marketing process.
Clinical trials begin for new combination therapy
PrimeC, a new combination treatment designed to slow down or halt progression of MND, has received orphan drug status from the US Food and Drug Administration (FDA), and is being investigated in two new clinical trials. The decision to begin testing in people came after a zebrafish model of MND significantly regained the ability to swim after treatment with PrimeC. The two trials, being held in the USA and Italy, are investigating the safety, tolerability and effectiveness of PrimeC in people with MND.
MND-SMART is led by a team of researchers based in Edinburgh and will simultaneously whether two existing treatments for other conditions could have some benefit for people living with MND. Funding is provided by Euan MacDonald Centre, substantial private donations, MND Scotland and My Name’5 Doddie Foundation and we’re pleased a number of Association-funded care centres will be involved in recruiting people with MND to take part. There are more trials in the pipeline than ever before – the Association is currently involved in two trials, MIROCALS and TUDCA, across a number of our care centres. We’re also working with neurologists on two additional trials through an international consortium called TRICALS that we hope will start in 2020 and work in a similar way to MND-SMART. If you would like more information about MND SMART, visit our website.
Latest news about NurOwn from BrainStorm
NurOwn is a treatment that uses bone marrow-derived stem cells, taken from a person with MND, that are re-programmed into neurone-supporting cells which are then transplanted back to the same person. They can then begin to secrete neurotrophic factors to protect and promote growth of neurones.
BrainStorm Cell Therapeutics, developers of NurOwn, met recently with the US Food and Drug Administration (FDA) to discuss the potential of NurOwn as an approved treatment for MND. NurOwn is currently being evaluated in a fully enrolled Phase 3 clinical trial in the USA. The FDA will look at all the data gathered in this clinical trial and will work with BrainStorm to identify a regulatory pathway forward, which will include an accelerated review of the data collected.
Both the FDA and BrainStorm acknowledge the urgent unmet need and the hard goal of moving much needed therapies for MND forward as quickly as possible. The Research team will be following this trial with interest and will further report when new information is released.
Healthtalk: Understanding experiences of inherited MND
A new study, funded by the MND Association, aims to improve out understanding of what it is like to live with inherited MND. Information gathered from interviews will be used to produce a new section on the research-based website healthtalk.org which helps support others going through similar experiences, and will inform healthcare professionals about inherited MND. The study is open to anyone living with, or at risk from, inherited MND. You can find out more on our website
New MND gene identified
Professor Ammar Al-Chalabi, Professor of Neurology and Complex Disease Genetics and King’s College London tweeted about the discovery of a new MND gene – DNAJC7. The DNAJC7 protein helps other proteins to fold into the correct shape and helps to clear proteins that have been damaged. When this process goes wrong, proteins build up in nerve cells and they die. This discovery means we now understand a little bit more about what causes MND, and this gives us new ways to think about developing an effective treatment. It also opens up the possibility of a specific future therapy for people with this genetic change. Unfortunately, open access to the full paper is not yet available but you can read the abstract here.
Another International Symposium has drawn to a close
It’s all over! Another successful International Symposium on ALS/MND has now reached an end and what a fantastic 3-days it was! Bringing together the brightest minds in MND research and healthcare, over 850 delegates enjoyed 110 talks over 26 sessions, 22 plenary speakers, more than 420 posters and five fantastic awards.
At the end of the first day, everyone was encouraged to take part in a Global Walk to D’feet MND, wearing a t-shirt representing their countries ALS/MND association or institution, along Perth’s picturesque Swan River to raise awareness of the disease. Following this, Cytokinetics invited participants to join them for an Australian BBQ to honour and recognise the contribution of advocacy groups worldwide. All Symposium news will be reported on our Periodic Table of MND Research and blog so keep checking these as we will continue to update them over the coming weeks. #alssymp #alsmndsymp.
2019 UK Biobank of the Year
We are delighted to share that the MND Association has been awarded the prestigious accolade of 2019 UK Biobank of the Year. The award recognises our work on the UK MND Collections, a bank of biological samples from people living with MND and controls, used by researchers to study MND. Kirsten Kelly, Research Projects and Partnerships Manager and Sarah Thompson, Research Co-Ordinator (Abstracts and Collections), from the Research Development Team at the Association, attended the event to receive the award. Our heartfelt thanks go to all who provided samples without whom this resource would not exist.
FDA approves dissolvable from riluzole
A dissolving film form of riluzole, called Exservan, has been approved by the US Food and Drug Administration (FDA). The film is placed onto the tongue where it dissolves and is absorbed into the body. Exservan is formulated to be taken without the need for water, making it particularly useful for people who are experiencing difficulties with swallowing. Aquestive Therapeutics, who developed Exservan, has granted a license to Zambon Pharma for the drug as a treatment for MND in the EU. Zambon will still need to obtain regulatory approval from the European Medicines Agency (EMA) before the Exservan can be prescribed in the EU. Hopefully this will then be picked up by the Medicines and Healthcare Products Regulatory Agency (MHRA) for approval in the UK. You can read more about this is ALS News Today.
MND Register hits the 2,000 mark!
Over 2,000 people have now joined the MND Register of England, Wales and Northern Ireland.
The MND Register is funded by the MND Association and is led by Prof Ammar Al-Chalabi at King’s College London and Prof Kevin Talbot at University of Oxford. It aims to collect information about every person living with MND. This will help plan the care for people living with MND and tell researchers more about what might be causing the disease.
For more information, and to register, see our website.