Embryonic Stem Cells Offer Treatment Promise for Multiple Sclerosis

Scientists in the University of Connecticut’s Technology Incubation Program have identified a novel approach to treating multiple sclerosis (MS) using human embryonic stem cells, offering a promising new therapy for more than 2.3 million people suffering from the debilitating disease.

The researchers demonstrated that the embryonic stem cell therapy significantly reduced MS disease severity in animal models, and offered better treatment results than stem cells derived from human adult bone marrow.

The study was led by ImStem Biotechnology Inc. of Farmington, Conn., in conjunction with UConn Health Professor Joel Pachter, Assistant Professor Stephen Crocker, and Advanced Cell Technology (ACT) Inc. of Massachusetts. ImStem was founded in 2012 by UConn doctors Xiaofang Wang and Ren-He Xu, along with Yale University doctor Xinghua Pan and investor Michael Men.

“The cutting-edge work by ImStem, our first spinoff company, demonstrates the success of Connecticut’s Stem Cell and Regenerative Medicine funding program in moving stem cells from bench to bedside,” says Professor Marc Lalande, director of the UConn’s Stem Cell Institute.

The research was supported by a $1.13 million group grant from the state of Connecticut’s Stem Cell Research Program that was awarded to ImStem and Professor Pachter’s lab.

“Connecticut’s investment in stem cells, especially human embryonic stem cells, continues to position our state as a leader in biomedical research,” says Gov. Dannel P. Malloy. “This new study moves us one step closer to a stem cell-based clinical product that could improve people’s lives.”

The researchers compared eight lines of adult bone marrow stem cells to four lines of human embryonic stem cells. All of the bone marrow-related stem cells expressed high levels of a protein molecule called a cytokine that stimulates autoimmunity and can worsen the disease. All of the human embryonic stem cell-related lines expressed little of the inflammatory cytokine.

Another advantage of human embryonic stem cells is that they can be propagated indefinitely in lab cultures and provide an unlimited source of high quality mesenchymal stem cells – the kind of stem cell needed for treatment of MS, the researchers say. This ability to reliably grow high quality mesenchymal stem cells from embryonic stem cells represents an advantage over adult bone marrow stem cells, which must be obtained from a limited supply of healthy donors and are of more variable quality.

“Groundbreaking research like this furthering opportunities for technology ventures demonstrates how the University acts as an economic engine for the state and regional economy,” says Jeff Seemann, UConn’s vice president for research.

The findings also offer potential therapy for other autoimmune diseases such as inflammatory bowel disease, rheumatoid arthritis, and type-1 diabetes, according to Xu, a corresponding author on the study and one of the few scientists in the world to have generated new human embryonic stem cell lines.

There is no cure for MS, a chronic neuroinflammatory disease in which the body’s immune system eats away at the protective sheath called myelin that covers the nerves. Damage to myelin interferes with communication between the brain, spinal cord, and other areas of the body. Current MS treatments only offer pain relief, and slow the progression of the disease by suppressing inflammation.

“The beauty of this new type of mesenchymal stem cells is their remarkable higher efficacy in the MS model,” says Wang, chief technology officer of ImStem.

The group’s findings appear in the current online edition of Stem Cell Reports, the official journal of the International Society for Stem Cell Research. ImStem is currently seeking FDA approval necessary to make this treatment available to patients.

Study Suggests Immune System Can Boost Nerve Regrowth

Modulating immune response to injury could accelerate the regeneration of severed peripheral nerves, a new study in an animal model has found. By altering activity of the macrophage cells that respond to injuries, researchers dramatically increased the rate at which nerve processes regrew.

Influencing the macrophages immediately after injury may affect the whole cascade of biochemical events that occurs after nerve damage, potentially eliminating the need to directly stimulate the growth of axons using nerve growth factors. If the results of this first-ever study can be applied to humans, they could one day lead to a new strategy for treating peripheral nerve injuries that typically result from trauma, surgical resection of tumors or radical prostectomy.

“Both scar formation and healing are the end results of two different cascades of biological processes that result from injuries,” said Ravi Bellamkonda, Carol Ann and David D. Flanagan professor in the Wallace H. Coulter Department of Biomedical Engineering and member of the Regenerative Engineering and Medicine Center at Georgia Tech and Emory University. “In this study, we show that by manipulating the immune system soon after injury, we can bias the system toward healing, and stimulate the natural repair mechanisms of the body.”

Beyond nerves, researchers believe their technique could also be applied to help regenerate other tissue – such as bone. The research was supported by the National Institutes of Health (NIH), and reported online Sept. 26, 2012, by the journal Biomaterials.