Page URL:

Gene therapy reverses multiple sclerosis in mice

25 September 2017
Appeared in BioNews 919

A novel gene therapy can reverse the symptoms and progression of disease in a mouse model of multiple sclerosis (MS).

The neurodegenerative illness is caused by the body's own immune system degrading and destroying nerve cells. US researchers have used a gene therapy approach in the livers of affected mice to produce more regulatory T-cells and reduce this autoimmune response.

'Using a clinically tested gene therapy platform, we are able to induce very specific regulatory cells that target the self-reactive cells that are responsible for causing multiple sclerosis,' said Dr Brad Hoffman at the University of Florida, College of Medicine in Gainesville, who led the study. 'Most current therapies for autoimmune diseases such as multiple sclerosis are based on general immune suppression, which has various side effects or complications.'

In MS, immune cells known as effector T-cells are thought to destroy myelin - the fatty sheath that protects nerve fibres. The study, published in Molecular Therapy, harnessed regulatory T-cells to reduce this behaviour. This approach has been shown to be safe and effective in patients with other autoimmune disorders such as type 1 diabetes, but the results are often insufficiently potent and short-lived.

To combat this issue, the researchers developed a strategy that utilised the liver's natural ability to induce immune tolerance. They injected the gene for a protein called myelin oligodendrocyte glycoprotein into the livers of mice, using a harmless virus vector; this then triggered the long-term production of regulatory T-cells.

Over seven months, symptoms were reversed in those mice that had already developed mild-to-moderate neurological deficits, and mobility restored in those with more severe symptoms such as hind-leg paralysis; by contrast, untreated mice developed neurological problems after 14 days.

'It's a very strong and potent result that we get from that treatment,' Dr Hoffman told The Gainesville Sun newspaper.

The gene therapy system alone did not fully reverse end-stage disease, however, but when combined with the immunosuppressive drug rapamycin, which allows the regulatory T-cells to proliferate, there was complete remission in the majority of the mice. And effects were long-lasting, with the mice protected from symptoms approximately 100 days later.

Though Dr Hoffman is optimistic about the potential of this approach in treating MS in humans, he cautions: 'This is done in a mouse model, and we always have to take that with a grain of salt.'

'This is a thorough and well-controlled set of experiments,' said Dr Gillies O'Bryan-Tear, from the Faculty of Pharmaceutical Medicine of the Royal College of Physicians of the United Kingdom, London. He noted that the study 'adds to the growing excitement being generated in the field of gene therapy'.

Gene immunotherapy protects against multiple sclerosis in mice
Medical Xpress |  21 September 2017
Gene Therapy-Induced Antigen-Specific Tregs Inhibit Neuro-inflammation and Reverse Disease in a Mouse Model of Multiple Sclerosis
Molecular Therapy |  21 September 2017
Gene therapy reverses symptoms, slows progression of MS in mice
FierceBiotech |  21 September 2017
UF researchers reverse disease in mice
The Gainsville Sun |  21 September 2017
Unique gene therapy prevents, reverses multiple sclerosis in animal model
University of Florida |  21 September 2017
16 July 2018 - by Dr Rosie Morley 
Gene therapy has been used in mice to successfully reverse obesity and insulin resistance, as well as other markers of type 2 diabetes...
12 March 2018 - by Martha Henriques 
Researchers have used genome editing to reveal genes that could be new therapeutic drug targets for the neurodegenerative condition amyotrophic lateral sclerosis (ALS)...
9 October 2017 - by Charlotte Spicer 
A new gene therapy has halted the progression of a fatal degenerative brain disease in a small study of affected boys...
4 September 2017 - by Meetal Solanki 
The world's first cancer treatment which uses a patient's own genetically modified immune cells has been approved...
31 July 2017 - by Jenny Sharpe 
A safe and effective gene therapy treating Duchenne muscular dystrophy in dogs has been demonstrated...
22 May 2017 - by Marcia Costa 
A new method combining stem cell and gene therapy with ultrasound and microbubbles has demonstrated an efficient way to heal severe bone fractures...
27 February 2017 - by Jamie Rickman 
A study has demonstrated that a new, one-off stem cell treatment for multiple sclerosis can 'freeze' progression of the disease for five years in some patients...
25 January 2016 - by Dr Lanay Griessner 
A stem cell treatment routinely used for bone and blood cancers is showing promise at reversing the effects of multiple sclerosis...
to add a Comment.

By posting a comment you agree to abide by the BioNews terms and conditions

Syndicate this story - click here to enquire about using this story.