May is ALS awareness month and Waisman Center investigator Su-Chun Zhang, MD, PhD, uses stem cells from ALS patients to uncover the cause of ALS and screen drugs to treat the disease.
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord that control muscle movement. When nerve cells die, the brain’s ability to control muscles is lost. A breakthrough discovery, made possible by the Waisman Center’s induced Pluripotent Stem Cell (iPS) Core, has identified a likely cause for this cell death.
Zhang pinpointed an error in protein formation that could be the root cause of ALS using iPS cells—stem cells derived from reprogrammed skin cells—from patients with ALS to identify a reduction of a protein, neurofilament (NF-L), critical for proper nerve cell function.
“Neurofilament moves throughout the cell. If NF-L proteins can’t travel properly within the cell, they form tangles that have a negative ripple effect on nerve cell development,” says Zhang.
This scientific advance allows scientists to study how to restore normal levels of this protein that could help prevent ALS tangles from forming in the cells.
Zhang continues to develop stem cell-based therapies to protect and repair diseased and damaged motor neurons.
“iPS cells are integral to understanding ALS. With iPS cells, we’re able to look at the disease in a new way, from the very start, and see where things go awry.”
In the past, scientists thought that once nerve cells were destroyed, there was no hope for recovery. They now believe the brain is much more “plastic” than anyone imagined, with the potential to enable scientists to repair or replace damaged cells in neurodegenerative diseases such as ALS.
“We’re in the process of testing FDA-approved drugs to see which has positive effects on the disease. This is a promising approach because it will speed up potential treatments when we find the right drug,” says Zhang.
Complementing these research efforts is the Waisman Center’s Augmentative Communication Aids and Systems Clinic (CASC), which conducts in-depth evaluations of the communication needs of people with ALS. A speech pathologist and occupational therapist work with patients to help identify assistive devices that not only meet their current needs, but are flexible enough to adapt to their changing abilities as their illness progresses.
CASC staff collaborate with UW Hospital’s Multidisciplinary ALS Clinic. Many of the patients seen in the ALS clinic receive follow-up services from CASC. There are also patients with ALS who are referred to CASC by the UW Department of Neurology, other healthcare facilities, support groups, or other sources. The team at CASC works together with the patient, and any people who are supporting him/her, in order to make sure that everyone is comfortable with the recommendations that are made related to assistive technology.
More on ALS research and activities at the Waisman Center
- Laying a foundation for treating ALS, spinal cord injuryby Kelly Tyrrell
- ALS community suddenly awash in awarenessby David Wahlberg
- Study helps unravel the tangled origin of ALS by David Tenenbaum