Research updates

Get involved in research - TUDCA-ALS Phase 3 clinical trial

TUDCA-ALS is a European run clinical trial taking place in centres across the UK and Ireland as well as Italy, Germany, France, Belgium and the Netherlands. This trial will test the effectiveness and safety of using TUDCA in people with MND. The trial will look at the effects, both good and bad, of TUDCA on people with MND and if it slows down the advance of the disease.

TUDCA

TUDCA is a substance naturally produced by the body and found in trace amounts in bile. TUDCA works by camouflaging a stress chemical that triggers a chemical cascade that results in the death of a distressed or damaged cell. Although MND is not a disease of the liver and bile duct, there is evidence that increasing the levels of TUDCA can slow the rate of death of motor neurones.

As with all clinical trials, there are inclusion and exclusion criteria but people who are living with MND who have less than 18 months since symptom onset and have no swallowing difficulties are invited to take part. Recruitment is via a neurologist who will refer to one of the recruitment centres in Sheffield, Preston, Liverpool, Plymouth, Salford or Dublin. You can find out more in our Information Sheet DB: TUDCA-ALS or at clinicaltrials.gov. For more information about all of our research opportunities, visit our website.

New gene therapy targeting C9orf72-ALS begins Phase 1 clinical trial in the UK

Researchers at King’s College Hospital, led by Professor Christopher Shaw, have embarked on the first gene therapy clinical trial for people affected by a specific form of ALS, the most common type of MND.

In 2011, researchers discovered that mistakes in the C9ORF72 gene cause up to 40% of all inherited cases of MND and frontotemporal dementia (FTD). Since the mutated gene produces toxic products, blocking the gene with gene therapy might be a useful approach to treatment. The gene therapy is a short DNA molecule called an antisense oligonucleotide (ASO) which is capable of selectively binding to and degrading toxic products made from the C9orf72 mutation.

DNA

The clinical trial is taking place across multiple site in the USA, Canada and Europe and aims to recruit up to 80 patients to test the safety and tolerability of the ASO. Recruitment is via referral by a neurologist. However, places are very limited so it is likely that this will be confined to patients who are local to the site. The first dose for the study at King’s College Hospital was successfully delivered by Dr Keith Mayl on Monday 16th September 2019 and marks a key moment for the MND community.

If the therapy is successful it will have wider implications for neurodegenerative diseases. Read more in our blog article written by Dr Keith Mayl and Dr Ahmad Al Khleifat of King’s College London.

An update on RCH4

There are literally thousands of alternative or ‘off-label’ therapies advertised on the internet and one such treatment, that repeatedly prompts questions and forum debates, is RCH4. We are very keen to understand more about RCH4 and to engage with the people behind it and would welcome the chance to talk to them about ongoing research they are undertaking and any validated data they are able to present. This validation is standard international protocol for researchers to ensure transparency and safety.

ALS Untangled

ALSUntangled reviews alternative and off-label treatments for people with MND. RCH4 is described as ‘an investigative new drug’ that ‘does not have Regulatory Authority marketing approval in any country’. With the information available to them, ALSUntangled concluded that as they were unable to determine RCH4’s structure or chemical class; that its claimed mechanism is one that has never been shown to be useful in treating people with ALS before; and they have been unable to independently verify RCH4’s reported effectiveness or safety they cannot, at this time, advise people living with MND to use this product.

You can read the full ALSUntangled review here.

The Research Development team be reached by phone 01604 611880 or by email on research@mndassociation.org. If you’d like to chat about any of these articles, please get in touch.

For further information and support contact our MND Connect helpline

mndconnect 0808 802 6262