Scientists in recent years have found a way to infuse stem cells into the brains of animals to repair damage to the central nervous system, offering some of the most encouraging news yet for multiple sclerosis patients.
Now, a key $12.1 million study soon will be under way in Buffalo and two other upstate medical centers that will for the first time begin to test the procedure in people.
The hope is that the stem cells will generate new myelin, the fatty substance that surrounds nerves like the insulation on a wire. Myelin is damaged in MS, leading to weak or lost signals between nerves. Eventually, the painful disease spreads in a slow, unpredictable path toward paralysis.
“This is a promising strategy. It has been extraordinarily effective in mice, and there is great hope the technique will be successful in people,” said Dr. Steven Goldman, co-principal investigator and co-director of the University of Rochester Center for Translational Neuromedicine.
The study by researchers in Rochester, the University at Buffalo and Upstate Medical University in Syracuse has far-ranging implications. It potentially could be applied to millions of patients with a host of other conditions, including Alzheimer’s and Parkinson’s disease.
Although stem cells show great promise, the approach is a ways from reality.
What works in mice does not always work in humans. In addition, scientists don’t know what causes MS, so they can’t exactly replicate MS in animals, complicating tests of the potential new treatment.
“Expectations have to be kept under control. You’re not going to implant stem cells in people and suddenly see them running around,” said Dr. Bianca Guttman-Weinstock, co-principal investigator at UB.
Stem cells are often referred to as master cells because they develop into the many different types of cells in the body that form organs and tissue. Stem cells also have the potential to repair or replace damaged cells.
Other scientists are looking at whether it may be possible to use certain stem cells to prevent the body’s immune system from attacking nerves.
“There is a lot happening in stem cell research, and it’s exciting because five years ago, these were just ideas. Now, they are reality,” said Dr. Timothy Coetzee, chief research officer at the National MS Society.
Until recently, scientists didn’t know exactly which master stem cells ultimately developed into cells that make myelin in a complicated process. They now know that cells called oligodendrocytes produce myelin. They also learned how to turn stem cells into a type of cell called glial progenitor cells. Glial progenitor cells are the cells that make oligodendrocytes.
These findings allowed scientists to transplant oligodendrocytes into the brains of mice that had no myelin. The result: The cells began to repair damaged areas.
“It was an extraordinarily effective strategy,” said Goldman.
All this progress has occurred in the last few years, including a study published late last year using stem cells transplanted into the brains of children with a rare genetic brain disease known as Pelizaeus-Merzbacher disease, in which myelin is lacking.
The significance of the research is that it indicates stem cells can be safely transplanted and may be effective at making myelin.
Children are different than adults, but Goldman and others say there is great hope the technique will succeed in adults.
The first step
The upstate consortium study for MS, which is funded by the Empire State Stem Cell Board, is the first step in a typical research process, which occurs in phases and usually takes many years to complete.
In the first two phases, scientists test the safety and effectiveness of an experimental treatment, and that’s what will happen with this stem cell trial for MS.
Patients enrolled in the study will be those with secondary progressive MS. These individuals no longer have periods of remission and, instead, experience a slow but steady worsening of symptoms with no approved therapy. Small holes will be drilled into their skulls and stem cells injected through catheters inserted in the holes.
The original proposal for the study recommended obtaining stem cells from discarded fetal brains. Technology has progressed enough that the current plan, dependent on government approval, is to start with stem cells from the patient’s own skin cells and reprogram them into cells useful for making myelin.
Before the first patients can receive the treatment in 2016, researchers must spend the next few years preparing for the trial. Among other things, they need to refine how they will measure the repair of myelin, as well as improvements in the patients. It’s a tricky issue because improvements are likely to occur slowly and will vary from person to person, said Guttman-Weinstock.
A key role
It seems fitting that the study will include Buffalo.
Research by the late Dr. Lawrence D. Jacobs, a Buffalo neurologist, played a key role in the development of Avonex, the drug most widely prescribed to treat relapsing MS.
Population studies show that higher rates of MS exist in temperate climates, in particular a geographic band in North America across the northern United States and southern Canada that includes Western New York.
If stem cells work, it could change the lives of millions of people worldwide who suffer from a disease that can be unbearable as patients decline in function and health.
That’s why many individuals like Shelly Boyle track research on potential MS treatments with a laser-like focus.
“I read everything I can read,” said Boyle, a 48-year-old Cheektowaga resident who was diagnosed with MS in 1990.
She stopped working as a chiropractor in 2004 as her symptoms worsened. She has trouble walking and coordinating the small muscles in her fingers. The medication she has been taking is no longer effective.
“The idea of drilling into my brain would scare me if I was in the trial, but the prospect of something new on the horizon is exciting,” she said. “I’m running out of options.”
Multiple sclerosis: The Basics
• Multiple sclerosis: A chronic, often disabling disease that attacks the brain, spinal cord, and optic nerves.
• What happens: The body’s immune system permanently damages the protective sheath called myelin that covers nerves.
• Types of MS: There are four types, including relapsing-remitting, which has flare-ups followed by periods of recovery; and secondary-progressive, in which the condition progressively worsens.
• Symptoms: Can range from mild numbness in limbs to paralysis and loss of vision.
• Cause: Still unknown.
• Treatment: There is no cure, but therapies exist to slow the disease’s progress and treat flare-ups.