amyotrophic lateral sclerosis Archives - Amazing Health Advances

Targeting the Brain Should Be the Focus of ALS Therapy, Not Just Spinal Cord

AHA Publisher — Mon, 06 Dec 2021 08:00:40 +0000

Northwestern University via News-Medical – The brain is indeed a target for treating ALS (amyotrophic lateral sclerosis), Northwestern Medicine scientists have discovered. This flips a long-standing belief that the disease starts in the spinal motor neurons and any therapy would need to target the spine as the key focus. A new Northwestern study shows the degeneration of brain motor neurons (the nerve cells in the brain that control movement of the limbs) is not merely a byproduct of the spinal motor neuron degeneration, as had been previously thought. “We have discovered that the brain degenerates early in diseases like ALS, sends us warning signals and shows defects very early in the disease. Therefore, we need to repair the brain motor neurons if we want long-term and effective treatment strategies. The brain is important in ALS.” Hande Ozdinler, lead study author, associate professor of neurology, Northwestern University Feinberg School of Medicine The paper will be published Dec. 2 in Gene Therapy. ALS is a swift and fatal neurodegenerative disease that paralyzes its victims. Upper motor neuron diseases, such as ALS, hereditary spastic paraplegia and primary lateral sclerosis affect more than 250,000 people a year in the U.S. alone. There is no cure and no effective long-term treatment strategy. This is the first study to clearly reveal the brain motor neuron degeneration is not a consequence of spinal motor neuron degeneration but is independent of the spinal motor neuron degeneration. The research also is the first to show that the gene UCHL1 is important for maintaining the health of brain motor neurons that are diseased due to two independent underlying causes. One is the accumulation of badly folded proteins and the other is the accumulation of sticky protein clumps inside the cells. These problems are observed in more than 90% of all ALS cases and also in other cases of upper motor neuron diseases. “Our findings not only give legitimacy for targeting brain motor neuron health in ALS as a therapeutic intervention, it also reveals the first target gene that can help these neurons be revitalized,” Ozdinler said. “This has huge clinical implications,” Ozdinler said. “Being able to modulate gene expression in diseased brain motor neurons in upper motor neuron disease patients is mind boggling. Since movement starts in the brain, if we can make the brain motor neurons happy and healthy, if we can boost their health and integrity with directed gene delivery, we may begin to develop personalized treatment options especially for patients with upper motor neuron disease, who currently have no effective treatment options. Northwestern University scientists have previously identified NU-9, the first compound that eliminates the ongoing degeneration of upper motor neurons that become diseased and are a key contributor to ALS. Now, this study reveals the importance and significance of treating upper motor neurons in ALS and identifies the first genetic target. The next step is to determine the best dose and the best site of injection with respect to improvement of movement and reduction of disease conditions in at least two different ALS disease models. After preclinical toxicology studies, scientists will move to translate these results into a clinical trial, a process that likely will take several years. To read the original article click here.

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Potential ALS Drug Gets Boost Toward Preclinical Trials

AHA Publisher — Fri, 05 Nov 2021 07:00:58 +0000

Abigail Klein Leichman via Israel21c – A new class of small molecules for treating amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease, and other neurodegenerative diseases, is being developed at Neuromagen Pharma of Beersheva with several million dollars in seed money raised from local private investors. “Currently there are no drugs available to treat ALS, so our drug candidate presents a new treatment paradigm and could be both first and best-in-class,” said Dr. Gil Ben-Menachem, founder and chief executive officer of Neuromagen. The preclinical-stage company – whose name means “neuro shield” in Hebrew — was incubated at the Ben-Gurion University of the Negev (BGU) Oazis accelerator and venture builder. It grew out of research conducted by BGU Prof. Esther Priel and her team at the university’s Laboratory for Nucleic Acids Topology. She is the company’s cofounder and chief scientific officer. Priel and her team have published papers describing how the family of novel small molecules they developed activates the transcription of a major surviving enzyme, telomerase reverse transcriptase, which protects and rehabilitates neuronal cells. When tested in ALS animal models, these drug candidates demonstrated delayed onset as well as delayed progression of the disease, and increased survival of the neurons by 60 percent. Neuromagen Pharma’s drug is not expected to be a cure; the goal is to delay the onset and progression of neurodegenerative diseases, thereby improving the quality and length of life for individuals with such diseases. Ben-Menachem said the funding “will enable us to jumpstart the company and initiate the preclinical work towards developing our promising drug candidates.” This work is a step that is necessary before proceeding to human clinical trials for regulatory approval. In December, the company will present its findings at the virtual 32nd International Symposium on ALS/MND. To read the original article click here.

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URI Engineering Professor Helping ALS Patients Use Their Brains to Communicate

AHA Publisher — Wed, 01 Jul 2020 07:00:20 +0000

University of Rhode Island via Newswise – Doug Sawyer was diagnosed with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, 11 years ago. His only muscles that still function are those that control eye movement. Despite his disability, Sawyer still works as an engineer from his home, designing electronics for Hayward Industries. Using only his eyes, the 57-year-old writes reports and other papers, draws pictures and schematics, talks on the phone, sends text messages and emails, and attends meetings online multiple times a week. However, Sawyer’s gaze weakens as he gets tired, causing the technology he currently uses to become ineffective. That’s why the Seekonk, Massachusetts resident was eager to work with University of Rhode Island Assistant Professor Yalda Shahriari to develop a new way for ALS patients to communicate. Shahriari and her team of student researchers in URI’s College of Engineering are developing a way for those with severe motor deficits such as ALS to communicate using brain signals, eliminating the need for patients to maintain fine eye-gaze control. Her project, funded by a National Science Foundation (NSF) grant, has two main goals. The first is to develop multimodal personalized algorithms to improve the robustness of the brain-computer interface (BCI) systems for patients with severe motor deficits. The second is to develop an autonomous hybrid system for non-communicative patients who are without residual motor control, such as those who lose their fine eye-gaze control in the late stages of ALS. Through longitudinal recordings taken of several patients with ALS during this and previous projects, Shahriari and her group have noticed significant day-to-day variations in brain-computer interface performance. “These variations are speculated to be associated with several factors, including cognitive fluctuations and environmental factors,” said Shahriari. “Developing personalized algorithms will enable us to predict these fluctuations and optimize performance based on each patient’s specifications and needs.” To ensure more accurate readings of brain activity, two non-invasive techniques are implemented simultaneously: electroencephalogram (EEG) and functional Near Infrared Spectroscopy (fNIRS) signals. EEG detects electrical activity in the brain using small, metal discs called electrodes. Functional Near Infrared Spectroscopy is an optical imaging technique in which an emitter transmits near infrared light and a detector detects the light reflected from the surface of the brain. This technique measures oxygen changes in the concentration of hemoglobin in the brain. The higher the concentration, the more activity is taking place. “We will use a hybrid of EEG and fNIRS signals to compensate for each neuroimaging modality shortage and use the complementary features obtained from each modality to improve our system,” said Shahriari. For patients in the later stages of ALS who experience cognitive dysfunction, such as memory loss and the inability to maintain eye gaze on objects, Functional Near Infrared Spectroscopy has shown to be a more accurate method of measurement. Shahriari and her students have developed a visuo-mental dual task paradigm which relies on conventional oddball-based protocols, but require the subjects to do some mental arithmetic tasks. This BCI approach is accomplished by displaying a grid of letters and numbers and intermittently flashing an image (matrix of digits) over each row and column. “By giving the patient higher demanding tasks to focus on, we can trigger several cognitive functions and extract the associated signatures or neural biomarkers,” said doctoral student Bahram Borgheai. “The computer can then decode the pattern of neural activities that appear after the patient performs the tasks. The patterns can be used for diagnostic and communication purposes.” Shahriari has collaborated with the National Center for Adaptive Neurotechnologies on projects since 2012. With the support of the national center, the Rhode Island Chapter of the ALS Association and Rhode Island Hospital, the professor would like to add more patients to the study. “Our analysis of the data becomes much more powerful if we can significantly increase the number of patients in the study,” said Shahriari. Patients will be asked to wear a cap with sensors attached that can record brain activity in the comfort of their homes or at a care center. Recordings of those with healthy brains will take place in Shahriari’s Neural Processing and Control Laboratory in URI’s Fascitelli Center for Advanced Engineering. All data processing and analysis will be conducted in the lab. Once enough patients have volunteered to participate in the research project, Shahriari plans to partner with more local hospitals and medical schools to take advantage of their clinical expertise. Sawyer has relished the opportunity to participate in the study. “Taking part in the brain activity study has been very rewarding,” said Sawyer. “I enjoy learning new things and staying abreast of the latest technology. Dr. Shahriari and her team have been willing to share their progress. They make me feel as if I’m part of their team and not just a test number.” Sawyer hopes that his participation will help Shahriari develop a way for ALS patients to work and communicate after their motor functions have ceased. “I don’t consider myself a victim of ALS and I don’t consider myself handicapped,” Sawyer said. “I just need help sometimes. There are people out there far worse off than me. Hopefully the time I give to Dr. Shahriari will someday improve their lives.” To read the original article click here.

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