Research Roundup: Compound Reverses ALS Nerve Damage and More
New Compound Appears to Reverse Neuron Damage Caused by ALS. Researchers at Northwestern University identified a compound that appears to reverse the ongoing degeneration of upper motor neurons associated with amyotrophic lateral sclerosis. ALS is a progressive neurodegenerative disease affecting nerve cells in the brain and spinal cord. “We have identified the first compound that improves the health of upper motor neurons that become diseased.” The compound can reduce protein misfolding in critical cell lines. ALS is linked to protein misfolding and protein clumping inside the brain. In about 90% of ALS patients, proteins aggregate inside the cell and cause what is called as TDP-43 protein pathology. Glaucoma affects more than 75 million people globally, and about 3 million in the U.S. Painless, glaucoma is an increase of ocular pressure that can cause irreversible damage to the optic nerve. There is no cure or way of stopping the disease, but the eye drops reduce eye pressure to slow disease progression and prevent permanent damage to the optic nerve. An international team of researchers compared the genes of 34,179 people with glaucoma to 349,321 control subjects and identified 44 new gene loci and confirmed 83 previously reported loci associated with glaucoma. Researchers out of Rice University published research that identifies a genetic mechanism that allows Mycobacterium tuberculosis, which causes tuberculosis, to respond to stress quickly based on “Memory” of previous stress. Scientists with the Stanley Mannen Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital in Chicago showed that a nanotherapy decreased intestinal inflammation and shrank lesions in a rodent model of severe Crohn’s disease. Researchers at the University of Buffalo studied the HAND2 gene, which evolved to be switched on in the uterine linings of placental mammals during pregnancy, but not in other species the researchers studied.
NIH News in Health
The worldwide phenomenon raised awareness-and millions of research dollars-for a fatal disease called ALS. ALS stands for amyotrophic lateral sclerosis. ALS attacks motor neurons, the nerve cells responsible for regulating “Voluntary” muscles that we’re aware of controlling, such as those in our arms, legs, and face. “At first only a single limb may be affected, such as some weakness in a hand or a leg, or a person may have problems speaking or swallowing,” explains Dr. Amelie Gubitz, who oversees much of NIH’s ALS research. Most people with ALS die from respiratory failure, usually within 3 to 5 years after symptoms first appeared. About 10% of people with ALS survive for 10 or more years. Nationwide, more than 12,000 people have ALS. It affects people of all races and ethnicities. ALS most commonly strikes between the ages of 40 and 60, but younger and older people also can develop the disorder. In most ALS cases, the underlying causes are unknown. Were associated with some cases of ALS. Since then, mutations in over a dozen genes have been found to cause familial ALS. These discoveries provide new information about the disease that will help guide future research. In 1995, the U.S. Food and Drug Administration approved a medication called riluzole as the first drug treatment for ALS. The drug prolongs life by several months, and it can extend the time before someone needs ventilation support. An FDA-approved drug combination of dextromethorphan and quinidine helps manage intense emotional changes, such as uncontrollable laughing or crying, that are often seen with ALS. Because the mind remains relatively intact, people with ALS may be keenly aware of their continued loss of function. While much is still unknown, scientists are working to gain new insights into ALS. “I believe potentially promising approaches for treatment are in the pipeline,” Gubitz says.
ALS neuron damage reversed with new compound
New compound targets neurons that initiate voluntary movement After 60 days of treatment, diseased brain cells look like healthy cells More research needed before clinical trial can be initiated. CHICAGO and EVANSTON– Northwestern University scientists have identified the first compound that eliminates the ongoing degeneration of upper motor neurons that become diseased and are a key contributor to ALS, a swift and fatal neurodegenerative disease that paralyzes its victims. In addition to ALS, upper motor neuron degeneration also results in other motor neuron diseases, such as hereditary spastic paraplegia and primary lateral sclerosis. “Even though the upper motor neurons are responsible for the initiation and modulation of movement, and their degeneration is an early event in ALS, so far there has been no treatment option to improve their health,” said senior author Hande Ozdinler, associate professor of neurology at Northwestern University Feinberg School of Medicine. “We have identified the first compound that improves the health of upper motor neurons that become diseased.” The NU-9 compound addresses two of the important factors that cause upper motor neurons to become diseased in ALS: protein misfolding and protein clumping inside the cell. Proteins fold in a unique way to function; when they misfold they become toxic to the neuron. The research team began to investigate whether NU-9 would be able to help repair upper motor neurons that become diseased due to increased protein misfolding in ALS. The results in mice were positive. Both the mitochondria and the endoplasmic reticulum began to regain their health and integrity resulting in improved neuron health. “Improving the health of brain neurons is important for ALS and other motor neuron diseases,” Ozdinler said. Upper motor neurons are the brain’s commanders-in-chief of movement. Thus, the lower motor neuron activity is in part controlled by the upper motor neurons.