A leading scientist battling to halt the development of Multiple Sclerosis has revealed that clinical human trials will start by 2020, after her company was honoured with a major nano medicine award. Dr Su Metcalfe's regenerative nanomedicine company, LIFNano™, was named Most Promising Nanomedicine Project at the International Nano Medicine Awards in Berlin on Tuesday, November 7.
Founded by Dr. Metcalfe, LIFNano™ slowly releases nanoparticles which are forecast to trigger natural healing in the brain. They contain Leukaemia Inhibitory Factor (LIF), to counter the disease process as it attacks the brain and spinal cord.
Dr. Metcalfe, who earned a PhD in pathology at the University of Cambridge, launched the technology company LIFNano™in 2013 and has been recognised many times already in the last few years. She said, upon receiving this global award “It has always been about getting this to patients. We have found evidence in our animal trials showing significant healing of damaged brain tissue in addition to the potent protection of brain cells.” Dr Metcalfe, who is based in Cambridge, England went on to say, “We are honoured to have been selected for this international award, but our absolute focus is ensuring patients see the benefits through clinical trials in 2020.”
Battling with Multiple Sclerosis
Multiple sclerosis (MS), is a neurological disease that attacks the brain and spinal cord. It affects more than 2.3 million people around the world and occurs when the immune system attacks the myelin sheath, the protective coating that encases nerve cells. This triggers symptoms such as vision problems, poor balance and muscle spasms. It could lead to total disability and dementia. The disease is forecast to cost in excess of $100 billion annually around the globe.
The issue with current treatments has been that they typically greatly diminish the activity of immune cells and they need to be used on a regular basis. Although these treatments prevent those immune cells from ravaging myelin within the brain, they also leave the body vulnerable to infection. None protects the brain. Metcalfe’s nanoparticles reset and correct the fault that causes MS as they deliver the natural stem cell growth factor LIF, directly and specifically to the sites of nerve damage, and keep the immune system from attacking myelin. They do not impair immunity and allow immune cellsto mobilize against harmful invaders when needed. What’s more, LIF can actually help repair damaged myelin within the brain.
If LIFNano™ holds up in clinical trials, “that would be a really significant addition to the treatments we have,” explained Bruce Bebo, executive vice president of research at the National Multiple Sclerosis Society, in an interview in October with OZY. Another benefit is its targeted approach which means fewer side effects expected, using the naturally occurring LIF.
For patients with MS, the potential of LIFNano™ could be profound once clinical trials have been successfully completed. Indeed, in addition to regulating immunity, a further key role of LIF is to sustain a healthy central nervous system, protecting nerves and maintaining myelin. LIFNano is therefore a powerful candidate for treatment of patients suffering from MS, providing a triple action for healing the brain and protecting nerves, repairing myelin and resettingself-tolerance. The beauty of the technology is its simplicity as it lets the body do what nature intended, slowly releasing LIF via the nanoparticles. Time really matters for MS sufferers, as early treatment is known to slow the course of the disease.
Dr Metcalfe's preclinical research has already found no toxicity of the LIF nanoparticles. Remarkably, LIF itself, the PLGA nanoparticles, as well as the anti-body fragment used to direct them have all been already used, separately, in various clinical trials.
Quantum Leap Nanomedicine
The nanoparticle manufacture has originally been developed jointly by Dr. Metcalfe and Yale University. Ultimately, Yale granted an exclusive license to LIFNano™ to develop the platform for MS, and for other degenerative diseases such as dementia and for auto-immune conditions, for years to come. The company, which has been self-funded to date, uses safe and natural ingredients rather than synthetic drugs.
LIFNano™ has a stellar team and advisers who are now working with Dr Metcalfe, including its Chairman, Florian Kemmerich, who also heads up the Europe-based NanoMed. The company's CEO, Olivier Jarry, joined the company with executive experience at Novartis, Bayer and Bristol-Myers Squibb, in addition to having consulted for giants such as Pfizer, GSK and Sanofi. Commenting on the award Olivier said, “Our treatment has the curative potential to deal with the root cause of MS, rather than just addressing symptoms and we hope to prove this in our human trials.” He went on to say, “the delivery of LIF across the blood brain barrier could profoundly improve therapeutic outcomes, reducing costs and potentially increasing convenience and safety.”
The Chairman of the company, Florian Kemmerich, said, “We have great partners, solid patents and exclusive licenses. This platform technology could go on to have profound impacts on other diseases such as Alzheimer's. We have received a lot of support and more is coming due to the scalability of the platform. We believe that, by successfully delivering these LIF nanoparticles to the brain, we will enable cells damaged by 'rogue' white blood cells to be repaired. This should then re-balance the body, using the natural reparative immune system we all have.”
The EU's European Technology Platform Awards honour firms that have developed a nanomedicine solution “that could bring significant benefits to patients, changing the way diseases are treated or diagnosed or providing new tools for physicians.” It further supports our drive to reach people with MS quickly. The award has previously been won by the Merck group, for a project aiming to develop a nanomedicine oncology product for mucosal therapeutic vaccination.
LIFNano™ and Dr. Metcalfe can add this to several other awards they have won or have been shortlisted for, including the UK government-backed Innovate UK Smart Award, the Merck Serono GMSI award, the Rolex Award for Enterprise and Business Weekly's Woman Entrepreneur of the Year Award.
The company is currently securing investment to complete GMP manufacturing, and secure authorizations to start clinical trials, aiming at generating a ‘proof of concept’ (Phase IIa) in 2020.