An innovative treatment could change how we treat multiple sclerosis (MS), but are we any closer to a cure?
The work of Dr. Su Metcalfe, founder and chief scientific officer of the biotech company LIFNano, appears to be breathing new life into that hope.
Metcalfe and her team developed a way to fight MS by using the body’s own natural mechanisms — but it hasn’t been tested in humans yet.
MS is an inflammatory and neurodegenerative autoimmune disease that can result in an array of neurological symptoms including fatigue, muscle spasms, speech problems, and numbness. It is caused by the immune system attacking myelin, the insulating coating that runs along the outside of nerve cells. The result is damage to the brain and central nervous system.
The disease currently affects roughly 2.5 million people worldwide. About 200 new cases are diagnosed each week in the United States.
Flipping the switch on the immune system
LIFNano uses a new treatment based on LIF — a stem cell protein that forms naturally in the body — to signal and regulate the immune system’s response to myelin.
“LIF, in addition to regulating and protecting us against attack, also plays a major role in keeping the brain and spinal cord healthy,” Metcalfe recently told Cambridge News.
“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,” she said.
Metcalfe has spent years studying LIF, but only recently realized its potential for treatment — likening it to an on/off switch for the immune system.
However, once she discovered its potential, there were almost immediate problems in its application. One of the earliest was how quickly LIF breaks down once it is administered into the body.
“If you try just to inject it into a patient, it dissipates or disappears in about 20 minutes,” Olivier Jarry, CEO of LIFNano, told Healthline.
“That makes it unusable in a clinic. You would have to have some kind of pump and inject it continually.”
A novel delivery system
A breakthrough came for Metcalfe when she took findings from her studies of LIF and applied them to nanotechnology. The treatment she is now developing relies on nanospheres derived from a well-established medical polymer known as PLGA, which is already used in materials like stitches. And because it is biodegradable, it can be left to dissolve inside the body.
Storing LIF inside these PLGA nanospheres before administering them into the bloodstream allows for a sustained dose over the course of several days.
The process differs significantly from the current drugs used to treat MS. These treatments most often fall under the category of drugs known as immunosuppressors, which inhibit the body’s overall immune system response.
LIF is theoretically much more precise than immunosuppressors, and should keep the immune system functioning against harmful infections and disease.
“We’re not using any drugs,” said Metcalfe. “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. Autoimmunity happens when that balance has gone awry slightly, and we simply reset that.”
Tempering expectations for a ‘cure’
The team cautions that LIF therapy is still several years away.
While some outlets have run wild with Metcalfe’s research, announcing that a “cure” for MS is right around the corner, those headlines are speculative.
Some MS advocacy groups have even made public statements calling coverage of her work “premature and irresponsible.”
Jarry told Healthline that LIFNano is expecting to enter phase I trials in 2020. This would be the first time that it is used in human subjects. But even if the treatment proves to be safe and effective, the soonest it could be on the market is 2023, he estimated.
The main focus of LIF therapy is now on MS. But it has potential for treating other autoimmune diseases including psoriasis and lupus.
“We are optimistic in the sense that we may provide a long-term remission for patients with MS,” said Jarry.
“Is it a cure? We’d love at some point to use the term ‘cure,’ but we are very cautious.”