Making new connections: Stem cells as treatment for ALS

By Kelly Lohr

The motor cortex in the human brain is mapped to match specific body parts. Body parts with more devoted cortex area are generally more sensitive or have finer motor control.

              Imagine slowly losing control of your muscles, first with a few twitches in your arms and legs or a slurred word here or there. Muscle failure will continue until it eventually stops your ability to move, speak, and breathe.  This is the life of a patient suffering from amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig’s disease, a progressive neurodegenerative disorder.  Currently, there is little treatment for the rapid course of this disease, but James Weimann, PhD, of Stanford Medical School provides a new hope.

            Weimann is part of a team of neuroscientists using transplanted neurons grown from embryonic stem cells to replace damaged cells in young animals.  This finding is the first of its kind in that the stem cells can be directed to take on the jobs of specific brain cells while also making the correct connections with other cells. Weimann’s cells transmit information from the cortex, the neural tissue that is outermost part the mammalian brain, specifically areas needed for motor function.

             Up until this point, the issue of stem cell transplantation in the brain was making the proper neuronal connections.  As an adult organism, creating the accurate connections in the nervous is extremely complex.  During development, superfluous neural connections deteriorate with lack of use. Only the pathways with the most activity remain in adulthood.  The chemical or physical signals that once lead the way in development are no longer present.  Without such cues, it is difficult for neurons to reach their target areas. For example, the stem cells created in Weimann’s lab must make connections with motor cortex in order to be an effective treatment for disorders like ALS or a traumatic brain injury.  Incorrect connections could result in further erratic brain function.

A step in the processing of human embryonic stem cells.

           While Weimann’s work holds a lot of potential for further progress and treatments, the studies have involved transplantation in young animal models.  Since the majority of neurodegeneration takes place in older adults, the next step will be to explore stem cell transplantation in adult animals.  Weimann and his team are hopeful that these newest findings will soon be used in treatment of neurons that are lost or damaged due to spinal cord injuries or diseases like ALS.

Uncovering a Missing Piece to the Puzzle

by Kristen Kocher

Researchers at the University of Edinburgh, have recently uncovered that the behavioral disorder, autism, is linked to abnormal brain development caused by Fragile X syndrome. Providing a critical clue into this puzzling disease, this research has begun to demystify the complexities of autism.

To begin unlocking the mysteries of autism, Professor Peter Kind at the University of Edinburgh, began research in an attempt to locate the differences between a normal brain and a brain with Fragile X. Through the use of a mouse model, certain sensory regions of the brain were found to react differently to stimuli, such as touch. Kind and his team believe that these differences may be found in other regions of the brain, which would aid in explaining the effects of Fragile X in patients. This discrepancy, it was also found, is caused by certain irregularities in brain development caused by the Fragile X mutation. Further studies by Kind and his associates also showed that abnormal brain development occurs during development in the womb. The identification of this window of time in which autistic brain development occurs may provide a more tangible and effective option for treatment methods to combat the disease.

Fragile X syndrome affects approximately one in every 4,000 males and one in every 8,000 females around the world and is the leading cause of autism. In terms of genetics, Fragile X is caused by a mutation within a gene sequence of the X chromosome. Autism presents itself in early childhood and is usually identified in a child that slow speak and does not interact with others. Compulsive, ritualistic, self-injury behavior are also characteristic of autism. As a result, this condition severely inhibits an affected individual’s ability to communicate with the outside world, causing numerous social, language and behavioral problems.

In the past, autism has proved difficult to study because it affects the inner workings of the brain without having any visible pathogenesis. In addition, those affected by Fragile X/autism are unable to reveal hints about the disease because they are unable to communicate with others. Therefore, without a fundamental understanding of the disease, treatment and therapy options are extremely limited, making autism a frustrating condition for the individual, the family, and the doctor. However, thanks to the research of Professor Kind and his team, the autism puzzle is one piece closer to being solved.

Original Press Release

Act Early, Spread Awareness

Running in Genes

By Abby Larson

Can someone really be born to be an athlete?  Science says so.  The idea of a genetic basis to exercise is a fairly new area of science, but it makes sense based on how the human body works. The expression of genes controls the function of human physiology: muscle development, capillary growth, hemoglobin concentration in red blood cells, etc.   After strenuous exercise, gene expression fires up to control muscle tissue repair due to increased forces on the body and tissue metabolic demand.  Capillaries feeding the muscles grow and become more efficient at delivering oxygen to tissues.  All of this is controlled by gene expression, the cellular switchboard of the human body.

Recent studies have identified over 200 genes that can determine the body’s ability to adapt quickly to exercise.  Based on this, training and conditioning could only take an athlete up to his or her genetically predetermined potential.  Does this mean that children can be genetically tested to see if they will be good at sports?  Is there a gene that makes a good football player versus a good runner? It’s more complicated than saying if a person has a specific gene, he or she can be a top athlete.  Like all processes in the human body, multiple genes are involved in adaptation to exercise and gene interactions play a large role.   Gene products don’t interact in a linear fashion, but in pathways and networks.  This makes genes harder to understand, and our knowledge of the interactions is in its infancy.  Once these pathways are discovered, scientists can begin to understand the extent to genetic determination of athletic ability.

These studies on the genetic basis of exercise are not going to benefit  just athletes—physical activity is one of the greatest preventative medicines for obesity, diabetes, and heart disease.  It is likely that genes correlated with exercise response could be mutated in people that have obesity or heart disease, which proposes new options of drug and gene therapy as preventative medicine.  The more we understand the benefits and mechanisms of exercise, the better we can understand how exercise can be used to improve public health.  So next time you go to the gym or run outside, think to yourself, “this is science.”

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Diabetic Blindness, a Light at the End of the Tunnel?


Susanne Mohr

Many of you, like me, may know some one close to you that has diabetes. Diabetic retinopathy is very prevalent, not only in the diabetic community but in the population as a whole. Roughly forty-five percent of diabetics are affected by diabetic retinopathy and it is among the leading causes of blindness among American adults. In this particular disease, existing blood vessels may swell, or new ones may form, both of which result in the obstruction of the retina. It is this obstruction of the retina, which is vital for human sight, that causes the  blindness associated with diabetic retinopathy.

Until now, this disease has gone untreated for the most part. That is until Susanne Mohr, a researcher at Michigan State University, made a major breakthrough in identifying the primary cause of diabetic retinopathy. In her research, she has found that a protein, siah-1, is produced in the body when blood sugar levels are correspondingly high, as you would find in a diabetic. She found that the siah-1 protein could be used to indicate the levels of a different protein, glyceraldehyde-3-phosphate dehydrogenase (GAPDH). 

This second protein, GAPDH, is the real culprit in diabetic retinopathy. When the levels of this protein are high in the blood, they accumulate in special cells in the eyes called Müller cells. Müller cells live on the blood vessels in the retina and when these cells die, it causes the blood vessel damage in the eyes that is associated with diabetic retinopathy. 

GAPDH is necessary throughout the body for energy among other things, so regulating the production of that is not possible. However, siah-1 is only produced when the blood sugar levels are high, so the regulation of that protein may be possible. Although this is one of the first studies with these results, and subsequent research is not yet known concerning the regulation of siah-1, the news is promising. This may have enormous impact in the scientific community and American society as a whole.

Justin Williams ’13


New Drug Shown to Heal Back Pain in 3 Days

By Johnathan Nieves

Source: Belmar-Fitness

Lower back pain is the fifth leading cause for doctor visits in the U.S. and over 85 percent of people suffer at least one bout of lower back pain in their lifetime. SOMA®, a new drug under development by, Meda Pharmaceuticals, Inc., a Somerset, N.J. based company, has been shown to improve functionality and reduce disability associated with lower back pain in as few as three days as confirmed by patient outcomes data.

The “outcomes data differentiates SOMA® 250 mg among the diverse treatment choices for patients with acute low back pain,” said Steven M. Simon, MD, RPh, Clinical Assistant Professor at the University of Kansas School of Medicine and Biosciences.

“Almost all acute low back pain is mechanical in origin and one in five patients with this condition suffers from significant limitations in activity.  Treatment of acute low back pain with SOMA® 250 mg has been shown to improve functionality, as measured by an internationally validated tool.”

Meda Pharmaceuticals, Inc. claims that SOMA® is the only skeletal muscle relaxant proven to significantly improve functionality in patients with acute low back pain as measured by the Roland-Morris Disability Questionnaire (RMDQ), an internationally validated standard for measuring the degree of disability and functionality in patients with lower back pain.

The Company’s approach to assessing patient symptom progression during SOMA® treatment via the RMDQ is unique in that it is a form of outcomes-based healthcare. Outcomes-based health care has become an increasingly popular and comprehensive approach to healthcare with goals of providing high quality care and reducing treatment costs.

“Overall, the greatest cost savings from a societal perspective may be obtained from interventions that promote early return to work and minimize lost productivity,” said Al Moorad, MD, Medical Director, Integris Jim Thorpe Rehabilitation, Oklahoma City. “This may be accomplished by appropriate drug utilization to allow patients to actively participate in rehabilitation therapy and return to daily activities.”

Meda Pharmaceuticals’ will present its findings this week at the 26th annual meeting of the American Academy of Pain Medicine in San Antonio, TX.

To learn more about SOMA®, you may visit The original press release pertaining to this article may be viewed at