'Degen-lock' - a novel way of protecting the nervous system in MND
Research type
Research Study
Full title
'Degen-lock' - a novel way of protecting the nervous system in motor neuron disease
IRAS ID
284217
Contact name
T Newton
Contact email
Sponsor organisation
TMN Therapeutics Ltd
Clinicaltrials.gov Identifier
Not applicable, Not applicable
Duration of Study in the UK
3 years, 8 months, 15 days
Research summary
TMN Therapeutics is creating a cell therapy to treat neurodegeneration. Initially we will focus our therapy on motor neurone disease (MND). In this condition motor neurons die, causing patients to experience a progressive and irreversible paralysis. Current treatments for the disease are poor, with significant side effects and only a moderate delay in symptomatic progression.
One of the causes of neurodegeneration is neuroinflammation -- a chronic inflammation of the brain. A number of anti-inflammatory strategies has been attempted in MND research, but they have so far all failed. Strong anti-inflammatory effects suppress beneficial immune function in both the brain and the rest of the body, however, weak anti-inflammatory effects fail to be efficacious.
A promising approach to treat neuroinflammation is co-opting part of the immune system, regulatory T cells (Tregs). These anti-inflammatory cells are able to reduce inflammation in a local environment, without shutting down beneficial inflammation in other locations. Small scale clinical trials have been carried out with Treg cell therapy in MND. In this therapy Tregs from a patient's blood are purified, grown in a laboratory environment, and large numbers re-infused back into the patient. Results are promising, however, the amount of cells required is prohibitively costly, preventing their use as a mainstream therapy.
Our innovation is the invention of a targeting technology, 'degen-lock', which will guide Tregs to motor neurons which are suffering neuroinflammatory stress. This concentrates Tregs at sites where they are needed, greatly increasing the efficacy of therapy and reducing the number of cells needed. This reduces costs to viable levels.
Our key objectives for this project are to establish in vivo proof-of-concept for degen-lock allowing us to apply for IP protection. Conversations with investors has indicated this is a crucial step to receive further funding.
The experimental and legal strategy for this project has been developed since our foundation in October 2018 after extensive discussions with the Cell and Gene Therapy Catapult, patent lawyers Cooley LLC and our CRO partner, Aquila Biomedical. Aquila are based in Edinburgh and have expertise in both Treg biology and neurodegeneration, making them an ideal partner to carry out proof-of-concept work.
Once proof-of-concept has been established for 'degen-lock' we can start developing the technology as a therapy for MND. An exciting long term plan for degen-lock is to expand into other neurodegenerative diseases such as Alzheimer's and Parkinson's once clinical success has been established in MND.
REC name
North West - Preston Research Ethics Committee
REC reference
20/NW/0414
Date of REC Opinion
30 Oct 2020
REC opinion
Further Information Favourable Opinion