Cambridge Scientist on Verge of Curing Multiple Sclerosis

Multiple Sclerosis (MS) affects approximately 2.3 million people worldwide. This auto-immune condition attacks the cells within both the spinal cord and the brain. This chronic, unpredictable disease currently has no cure.

MS can cause many symptoms, including:

  • Blindness
  • Blurred vision
  • Extreme fatigue
  • Loss of balance
  • Numbness
  • Paralysis
  • Poor coordination
  • Problems with memory and concentration
  • Slurred speech
  • Tremors

These problems may either come and go or persist for a lengthy time, continually getting worse. According to the National MS Society, the average age of diagnosis is 20-50, however, there have been recorded cases of people diagnosed as old as 75, and as young as 2. More than 2-3 times as many women have been found to develop MS than men, and this divide between the genders has been increasing gradually over the past 50 years.

Multiple Sclerosis, MS

Dr. Su Metcalfe and her company, LIFNano have devoted themselves to the search for a cure. Dr. Metcalfe states:

“Some people get progressive MS, so go straight to the severe form of the disease, but the majority have a relapsing or remitting version. It can start from the age of 30, and there’s no cure, so all you can do is suppress the immune response, but the drugs that do that have side effects, and you can’t repair the brain. The cost of those drugs is very high, and in the UK there are a lot of people who don’t get treated at all.”

Dr. Metcalfe was working at the Department of Surgery at the University when she made an important discovery. She explains, “I was looking to see what controls the immune response and stops it auto-attacking us.” She has now brought together one of the bodies cleverest functions, the stem cell particle, with a cutting-edge technology called a LIF. She went on to explain:

I discovered a small binary switch, controlled by a LIF, which regulates inside the immune cell itself. LIF is able to control the cell to ensure it doesn’t attack your own body but then releases the attack when needed.”

“That LIF, in addition to regulating and protecting us against attack, also plays a major role in keeping the brain and spinal cord healthy. In fact, it plays a major role in tissue repair generally, turning on stem cells that are naturally occurring in the body, making it a natural regenerative medicine, but also plays a big part in repairing the brain when it’s been damaged.”

“So I thought, this is fantastic. We can treat auto-immune disease, and we’ve got something to treat MS, which attacks both the brain and spinal cord. So, you have a double whammy that can stop and reverse the auto-immunity, and also repair the damage caused in the brain.”

 Multiple Sclerosis, MS

Unfortunately, Dr. Metcalfe was faced with an unexpected challenge. The LIF was only able to survive outside the cell for 20 minutes before the body would break it down. This meant that there was not enough time in order to deploy it during a therapy treatment. It was at this stage of the journey that she introduced the technology of nanoparticles. She went on to describe what happened next:

They are made from the same material as soluble stitches, so they’re compatible with the body and they slowly dissolve. We load the cargo of the LIF into those particles, which become the delivery device that slowly dissolve and deliver the LIF over five days. The nanoparticle itself is a protective environment, and the enzymes that break it down can’t access it. You can also decorate the surface of the particles with antibodies, so it becomes a homing device that can target specific parts of the brain, for example. So you get the right dose, in the right place, and at the right time.”

The nanoparticles were developed at Yale University, the co-inventor listed on the IP that was filed. The exciting news is that LIFNano has worldwide license to deploy these particles. Dr. Metcalfe believes that she is on the verge of a breakthrough in medicine and the treatment of MS, stating:

Nanomedicine is a new era, and big pharma has already entered this space to deliver drugs while trying to avoid the side effects. The quantum leap is to actually go into biologics and tap into the natural pathways of the body.”

“We’re not using any drugs; we’re simply switching on the body’s own systems of self-tolerance and repair. There aren’t any side effects because all we’re doing is tipping the balance. Auto-immunity happens when that balance has gone awry slightly, and we simply reset that. Once you’ve done that, it becomes self-sustaining and you don’t have to keep giving therapy because the body has its balance back.”

To date, LIFNano has been the recipient of two major funding awards, from the Government’s Innovate UK agency as well as from the drug firm Merck. Realizing that she needed someone with business knowledge to run that side of the company, Dr. Metcalfe recruited in the company’s Chairman Florian Kemmerich and CEO Oliver Jarry. They hope to attract even more investment and are aiming for a clinical trial start date of 2020. Dr. Metcalfe went on to say:

The 2020 date is ambitious, but with the funding we’ve got and the funding we’re hoping to raise, it should be possible. We’ve got everything we need in place to make the nanoparticles in a clinically compliant manner, it’s just a case of flicking the switch when we have the money. We’re looking at VCs and big pharma because they have a strong interest in this area. We’re doing all our pre-clinical work concurrently while bringing in the major funds the company needs to go forward in its own right.”

When asked about her interest and fascination with immune cells, Dr. Metcalf explained, “I wanted to understand something that was so simple on one level but also so complex. The immune cell is the only single cell in the body that is its own unity, so it functions alone. It’s probably one of the most powerful cells in the body because it can kill you, and if you haven’t got it you die because you haven’t got it.”

She continued, eluding to a longer-term goal of taking the technology even further than the treatment of MS, adding, “MS is our key driver at the moment, but it’s going to be leading through to other major auto-immune disease areas. Psoriasis is high up on our list, and diabetes is another. Downstream there are all the dementias because a LIF is a major health factor for the brain. So if we can get it into the brain we can start protecting against dementia.”

via EWAO

Leave a comment