ALS treatment 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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New Brain-Computer Interface Allows Man with ALS to ‘Speak’ Again

The AHA! Team — Fri, 11 Oct 2024 08:20:41 +0000

UC Davis Health via Newswise – Technology developed by UC Davis Health restores interpersonal communication A new brain-computer interface (BCI) developed at UC Davis Health translates brain signals into speech with up to 97% accuracy — the most accurate system of its kind. The researchers implanted sensors in the brain of a man with severely impaired speech due to amyotrophic lateral sclerosis (ALS). The man was able to communicate his intended speech within minutes of activating the system. A study about this work was published today in the New England Journal of Medicine. ALS, also known as Lou Gehrig’s disease, affects the nerve cells that control movement throughout the body. The disease leads to a gradual loss of the ability to stand, walk and use one’s hands. It can also cause a person to lose control of the muscles used to speak, leading to a loss of understandable speech. The new technology is being developed to restore communication for people who can’t speak due to paralysis or neurological conditions like ALS. It can interpret brain signals when the user tries to speak and turns them into text that is ‘spoken’ aloud by the computer. “Our BCI technology helped a man with paralysis to communicate with friends, families and caregivers,” said UC Davis neurosurgeon David Brandman. “Our paper demonstrates the most accurate speech neuroprosthesis (device) ever reported.” Brandman is the co-principal investigator and co-senior author of this study. He is an assistant professor in the UC Davis Department of Neurological Surgery and co-director of the UC Davis Neuroprosthetics Lab. The new BCI breaks the communication barrier When someone tries to speak, the new BCI device transforms their brain activity into text on a computer screen. The computer can then read the text out loud. To develop the system, the team enrolled Casey Harrell, a 45-year-old man with ALS, in the BrainGate clinical trial. At the time of his enrollment, Harrell had weakness in his arms and legs (tetraparesis). His speech was very hard to understand (dysarthria) and required others to help interpret for him. In July 2023, Brandman implanted the investigational BCI device. He placed four microelectrode arrays into the left precentral gyrus, a brain region responsible for coordinating speech. The arrays are designed to record the brain activity from 256 cortical electrodes. “We’re really detecting their attempt to move their muscles and talk,” explained neuroscientist Sergey Stavisky. Stavisky is an assistant professor in the Department of Neurological Surgery. He is the co-director of the UC Davis Neuroprosthetics Lab and co-principal investigator of the study. “We are recording from the part of the brain that’s trying to send these commands to the muscles. And we are basically listening into that, and we’re translating those patterns of brain activity into a phoneme — like a syllable or the unit of speech — and then the words they’re trying to say.” Faster training, better results Despite recent advances in BCI technology, efforts to enable communication have been slow and prone to errors. This is because the machine-learning programs that interpreted brain signals required a large amount of time and data to perform. “Previous speech BCI systems had frequent word errors. This made it difficult for the user to be understood consistently and was a barrier to communication,” Brandman explained. “Our objective was to develop a system that empowered someone to be understood whenever they wanted to speak.” Harrell used the system in both prompted and spontaneous conversational settings. In both cases, speech decoding happened in real time, with continuous system updates to keep it working accurately. The decoded words were shown on a screen. Amazingly, they were read aloud in a voice that sounded like Harrell’s before he had ALS. The voice was composed using software trained with existing audio samples of his pre-ALS voice. At the first speech data training session, the system took 30 minutes to achieve 99.6% word accuracy with a 50-word vocabulary. “The first time we tried the system, he cried with joy as the words he was trying to say correctly appeared on-screen. We all did,” Stavisky said. In the second session, the size of the potential vocabulary increased to 125,000 words. With just an additional 1.4 hours of training data, the BCI achieved a 90.2% word accuracy with this greatly expanded vocabulary. After continued data collection, the BCI has maintained 97.5% accuracy. “At this point, we can decode what Casey is trying to say correctly about 97% of the time, which is better than many commercially available smartphone applications that try to interpret a person’s voice,” Brandman said. “This technology is transformative because it provides hope for people who want to speak but can’t. I hope that technology like this speech BCI will help future patients speak with their family and friends.” The study reports on 84 data collection sessions over 32 weeks. In total, Harrell used the speech BCI in self-paced conversations for over 248 hours to communicate in person and over video chat. “Not being able to communicate is so frustrating and demoralizing. It is like you are trapped,” Harrell said. “Something like this technology will help people back into life and society.” “It has been immensely rewarding to see Casey regain his ability to speak with his family and friends through this technology,” said the study’s lead author, Nicholas Card. Card is a postdoctoral scholar in the UC Davis Department of Neurological Surgery. “Casey and our other BrainGate participants are truly extraordinary. They deserve tremendous credit for joining these early clinical trials. They do this not because they’re hoping to gain any personal benefit, but to help us develop a system that will restore communication and mobility for other people with paralysis,” said co-author and BrainGate trial sponsor-investigator Leigh Hochberg. Hochberg is a neurologist and neuroscientist at Massachusetts General Hospital, Brown University and the VA Providence Healthcare System. Brandman is the site-responsible principal investigator of the BrainGate2 clinical trial. The trial is enrolling participants. To learn more about the study, visit braingate.org or contact braingate@ucdavis.edu. A complete list of coauthors and funders is available in the article. Caution: Investigational device. Limited by Federal law to investigational use. 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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