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Stem Cell Research for Treating MS

Improving hematopoietic stem cell transplantation (HSCT) through Dr. Danwei Wu’s research of the immune system could enhance treatment for many neurological disorders. 

Dr. Danwei Wu is a Neuroimmunology Fellow at Stanford University and a 2023 Next Generation Research Grant Recipient for her research into potential therapy for aggressive forms of Multiple Sclerosis (MS). After witnessing her grandfather going through Alzheimer’s and dementia, she was inspired to enter the field of neurology and research. 

During her residency, Dr. Wu was moved by the young patients at the hospital who were diagnosed with MS. She calls MS, “a disorder of the young,” as peak onset for patients is when they are in their twenties to early forties. She explains, “I was really struck by how well people did with MS if they were treated early, diagnosed early, and managed aggressively.” Therapy is crucial to maintaining quality of life for people with MS, especially those who are diagnosed in the peak years of their life.

More than 1 million people in the United States live with MS and 10,000 new cases are diagnosed each year. Multiple sclerosis (MS) disrupts the communication between the brain and the body, causing muscle weakness, fatigue, and balance problems. In MS, the body’s immune system attacks its own tissues—specifically myelin, the protective coating that surrounds nerve cells—which disrupts communication between the brain and body. Dr. Wu’s most immediate research goal is to achieve a better understanding of the way in which MS wreaks havoc on the body in order to find potential drug targets. Her research begins with investigating immune systems hijacked by MS. 

Resetting the Immune System

When a person has MS, their immune system, which intends to protect and heal, causes inflammation that leads to the destruction of tissues and a lack of communication from the brain. Dr. Wu’s research centers on resetting the immune system so that it can heal the body once again. She focuses on a treatment called autologous hematopoietic stem cell transplantation (HSCT) or bone marrow transplant. She explains this treatment is “where we take out your own immune cells from the bone marrow, we wipe out the immune cells that are in your blood causing disease, and we replace your bone marrow to help reset the immune system.” 

HSCT is already used as treatment for MS and other neurologic disorders, such as ALD or myasthenia gravis. Her research will study ways to make HSCT more effective, which in turn may guide new treatments to repair damage to the nervous system for many types of disorders beyond MS. 

Why We Study Neuroinflammation and the Immune System

Neuroinflammation is inflammation that is taking place in the brain or spinal cords and complex changes in the brain’s response to this inflammation is an element of nearly every neurological and neuropsychiatric disorder. MS is a neuroinflammatory disorder, meaning that the body’s immune system overreacts and attacks healthy cells by mistake. 

There are many immune components to disorders that are not primarily considered neuroinflammatory. For example, the immune system is linked to brain aging, and therefore linked to neurodegenerative disorders, like Alzheimer’s, Parkinson’s and all types of dementia. Dr. Wu, a neuroimmunologist, explains, “If you can really understand how the immune system in the bone and blood marrow responds to any damage to the brain and spinal cord, then you can apply these techniques and approaches to all different types of disorders.” 

Used in HSCT treatment, stem cells are powerful because they are the building blocks for all tissues, blood, and the immune system. They can solve problems from within the body. Take for instance, breaking your bone. Instead of fixing the bone with external bandages, stem cell treatment would be like fixing it from within to create a stronger bone and prevent it from breaking again in the future. 

Dr. Wu explains, “These cells will propagate throughout an individual’s life. So you’re now creating a reservoir where you can, in theory, have a very sustainable long-term effect on an individual’s health.” In prior studies, researchers have investigated how to incorporate stem cells to repair specific central nervous system cells, such as glia cells. Dr. Wu said that the limitation to stem cell treatment is the difficulty of incorporating stem cells equally across all different tissues in an organ system. 

HSCT is already used as a mechanism for hematologic (blood) cancer treatment, where the immune system can be hijacked to fight the disease. Research into the immune system has the potential to address many types of neurologic disorders, even those that are not inflammatory. 

Building a Foundation for Treating Other Brain Disorders 

Dr. Wu’s research aligns with our “Cure One, Cure Many” philosophy. In researching MS and HSCT, a treatment that is already useful across many disease states, she is making strides for many other related brain diseases. Her research is targeting one disease, but a natural byproduct is a clearer picture of the brain as a whole. Dr. Wu explains, “I think that my project can be directly translated into treatment for a lot of different therapies or a lot of different disorders.” 

To further explore the mysteries of the brain, Dr. Wu’s Next Generation Research Grant allows  her to have protected time for lab research and to shift clinical duties towards crucial research. In her opinion, investigating brain disease is “the last frontier for medicine,” as the biggest strides in neuroscience have only been made in the last decade. 

She believes our desire to fund and further brain disease research is a consequence of our humanity. “We want to preserve our personality, our cognition, our mobility,” said Dr. Wu. “Understanding how all components of our nervous system age and [are] affected with disease is also fundamental to preserving independence.”

The American Brain Foundation is committed to supporting the next generation of brain disease researchers. By donating today you can help us achieve our vision of life without brain disease.