Lou Gehrig's Disease Archives - Amazing Health Advances

Promising New Drug Could Slow Progression of ALS

The AHA! Team — Mon, 25 Nov 2024 06:46:21 +0000

John Jeffay via Israel21c – A chance encounter with an inspiring ALS patient, prompted Alon Ben-Noon to set up NeuroSense Therapeutics, a startup developing a drug combo that aims to slow progression of ALS and other neurodegenerative diseases. It was a life-changing moment for Alon Ben-Noon when he first met Shay Rishoni. Rishoni, once a keen runner and cyclist, had been robbed of all movement by the devastating and degenerative disease ALS. It was 2016 and Rishoni was by then immobile, only able to communicate by staring at an eye-tracking computer screen. But that didn’t stop him working as CEO of Prize4Life, a nonprofit founded by other ALS patients to help find a treatment for the disease. Ben-Noon was profoundly moved by the meeting. “I looked at myself and I thought: You’re completely functioning, but you’re not doing half of what he’s doing. He’s completely paralyzed and he’s moving mountains.” Ben-Noon was already working in the pharmaceutical industry, as a consultant, but decided to change track and do everything he could to allow ALS patients to live longer and live better. And so NeuroSense Therapeutics was born. The pharmaceutical startup, based in Herzliya, central Israel, aims to slow the progress of ALS (amyotrophic lateral sclerosis) as well as Alzheimer’s and Parkinson’s, which are also neurodegenerative disorders. PrimeC ALS is a rare and incurable disease caused by the death of motor neurons, the nerve cells that send messages from the brain and spinal cord to our muscles and glands. ALS (also known as Lou Gehrig’s disease) leads to complete paralysis, followed by death, usually with two to five years. Rishoni, married with two sons, was diagnosed when he was 45 and survived another seven years, which is longer than most. At the time, the only medication available was a drug called Riluzole, approved for use in 1995, which extended patients’ lives by around three months. Ben-Noon was determined to do better. He gathered a team of experts to identify molecules in existing drugs that could be combined to attack multiple targets associated with ALS. Previous attempts to treat the disease had focused on single targets. The team succeeded in addressing a number of distinct problems, including the degeneration of motor neurons. In clinical trials in Israel, Canada and Italy, the drug that NeuroSense developed has been shown to give ALS patients, on average, an extra 18 months. Patients experienced a 36% slower disease progression and a 43% better survival rate over 12 months compared to control subjects. The drug is named PrimeC – “prime” is English for “Rishoni” — and could be available for patients within three and a half years. The patented drug combines the antibiotic ciprofloxacin and the anti-inflammatory agent celecoxib, both already approved by the US Food and Drug Administration (FDA) for unrelated conditions. Fast progression ALS is a highly aggressive and complex disease that affects around one in 10,000 people. Initial symptoms are mild, such a weakness in a finger, or dragging a leg, but it can progress at an alarming rate. “Quality of life in terms of functionality is usually quite good at the beginning and then it declines as the disease progresses,” says Ben-Noon. “One day a patient can still eat by himself; the next day they’ll need assistance. One day a patient can walk independently and the next day they’ll have difficulties walking without a cane and soon they’ll need a wheelchair. “We understood quite quickly that we cannot reverse the disease, but we can stop it and make a meaningful change to people’s lives.” But he hopes to do even more. “Eventually, we will create a world where ALS is a non-fatal disease. Patients will live life to the full, happily, maybe with a very small dysfunction. That’s it, that’s the vision,” Ben-Noon says. Orphan drug designation NeuroSense has received orphan drug designation in the US and Europe, recognizing its potential to treat a rare condition (which means tax breaks and other benefits for the company) though it still needs to gain regulatory approval pending further clinical trials. The company, which went public on NASDAQ in December 2021, has so far attracted $30 million in funding and has a US office in Cambridge, Massachusetts. “We are only 16 employees but we work with dozens of consultants and vendors who are assisting us in advancing our programs,” says Ben-Noon. Dr. Vivian Drory, director of the ALS clinic at Tel Aviv Sourasky Medical Center, said that promising results from the company’s 12-month clinical study highlight the significant potential of PrimeC as a disease-modifying drug for ALS. “These findings underscore the importance of early intervention, which can lead to more substantial benefits, and provide valuable insights that will inform the design of the company’s Phase 3 study, increasing the likelihood of success,” she said. It’s often small companies, like NeuroSense, that pioneer new drugs, Ben-Noon notes. “Nowadays the ratio is about 60/40 — 60 for the small companies 40 for big pharma,” says Ben-Noon. “In many cases it starts in a very small company like ours and then a big pharma looks at the outcomes and decides to buy out the company and continue the development.” Looking back to his first meeting with Rishoni, back in 2016, does he feel he’s done what he set out to achieve? “Yes, absolutely,” he says. “We always keep in touch with Tami [Rishoni’s widow]. We talk, we meet and every time we reach a new milestone is very fulfilling.” “If I hadn’t bumped into Shay,” he reflects, “I probably would still be doing medical consulting work. But now I’m very proud to say we’re creating change in the world.” For more information, click here. 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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