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ALS Center & Lou Gehrigs Treatment Clinic; WashU Neurology

We provide quality care, state-of-the-art treatment, precision medicine, and research opportunities to patients diagnosed with amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig’s disease. Our St. Louis ALS team, consisting of physicians, clinicians, researchers and experts, collaborates to evaluate patient needs, create individualized clinical care plans, and implement recommended clinical services. Clinical trials transform promising laboratory research into life-enhancing treatments. Patients at our Center have access to leading-edge clinical trials which focus on personalized medicine to improve the understanding, treatment, and prevention of ALS. Research & Precision Medicine. Our world-class team transforms cutting-edge translational research into new therapies, accelerating treatment and the search for a cure. Dr. Miller is an international leader in precision medicine for neurologic disease. Meet Roger; Clinical Trial Participant When Roger was diagnosed with ALS, a debilitating neurodegenerative disorder, he decided to participate in a clinical trial at the ALS Center at Washington University School of Medicine in St. Louis. Help us develop new treatments, therapies and cures. Participate in a trial or study that relate to the cause and treatment of ALS. “The staff are compassionate and caring. They understand that they’re working with people, not just subjects. I pray that with their assistance one day there will be a cure for ALS.”. “Working with Dr. Miller and his team has been awesome. I was able to participate in studies, which made me feel that I was fighting the disease. I felt part of a team.”

Keywords: [“ALS”, “clinical”, “treatment”]
Source: https://alscenter.wustl.edu/

Angel Fund contributes $1 million for ALS research at UMass Medical School

“We can do this because of the tremendous support of our donors, our supporters and our board. Without research, there is no cure,” said Richard Kennedy, president of the Angel Fund, a nonprofit organization that is deeply committed to supporting Dr. Brown’s research. Kennedy was diagnosed with ALS in 2016 at the age of 56. ALS has been a part of his family since 1989 when the disease claimed his father. His youngest brother, Jimmy, was diagnosed several years later and died from ALS at the age of 31. “At a time when funding is so crucial, we are pleased to honor our commitment to Dr. Brown’s research with our most recent donation,” Kennedy said “We can do so because we are fortunate to have supporters that care so much and help us by hosting events, participating in the events, donating and volunteering. All for our mission-to find a treatment and cure for ALS.”. ALS is a progressive neurodegenerative disease that affects neurons in the brain and the spinal cord. People with ALS slowly lose the ability to initiate and control muscle movement, which often leads to total paralysis and death within two to five years of diagnosis. While 10 percent of ALS is familial in origin because of a genetic defect, the other 90 percent of ALS cases are considered sporadic, or without a family history. A study published last year by Brown showed that a single transplantation of a bone marrow-derived mesenchymal stem cell treatment for ALS called NurOwn was safe and well tolerated by patients. The results were published in the journal Neurology. Developed by BrainStorm Cell Therapeutics Inc., NurOwn cell therapy is the furthest advanced autologous stem cell treatment in development for ALS..

Keywords: [“ALS”, “support”, “cell”]
Source: https://www.umassmed.edu/news/news-archives/2020/02/angel-fund-contributes-$1-million-for-als-research-at-umass-medical-school/

First-of-its-kind trial in ALS spurs hope for brutal disease

Morris said she has reason for hope, stemming from a first-of-its-kind study in ALS that will test five experimental drugs simultaneously in a platform trial. Five drugmakers selected for the trial were announced last week. This trial aims to accelerate the pace of ALS research and may prove to be a template for other rare diseases as well, experts said. Morris, who is also a leader of the patient group I Am ALS, said the trial can bring hope to future patients, even if it comes too late to help her. Now, slightly less than a year after being established, the Sean M. Healey & AMG Center for ALS Research is aiming to start the platform trial in the first few months of 2020. Cudkowicz, who leads the study, as well as the Healey Center and Mass General’s neurology department, said in an interview the trial aims to have 160 patients for each of five treatments. While smaller biotechs have bought into the platform trial and its efficiencies, missing from the list of initial participants are industry leaders. Cudkowicz noted larger drugmakers have shown interest in roundtable discussions that shaped the trial’s design, and there’s always potential to add additional therapies after the trial starts. The main hang-up for these companies is ceding control, said Scott Berry, a senior statistical scientist and co-founder of Berry Consultants, which worked on the ALS platform trial. Beyond ALS, adaptive trials have been started in breast cancer, Alzheimer’s disease and glioblastoma. Just this month, the agency finalized guidance on ALS research that advised companies to consider adaptive trial designs. Morris said the platform trial has struck her with its compassion.

Keywords: [“trial”, “study”, “ALS”]
Source: https://www.biopharmadive.com/news/als-platform-trial-mass-general-healey-center/563595/

‘First Step’ Mass. Research Turns Off Key Gene In ALS That Runs In Families

He died in late 2019 at age 34.Researchers at the University of Massachusetts Medical School and Massachusetts General Hospital report progress on a potential new weapon against the fatal nerve disease ALS: they used a virus to deliver small molecules called micro-RNAs to block a gene that causes about 2% of ALS cases. UMass Neurology Professor Bob Brown says the method, reported in The New England Journal of Medicine, shows promise for treating the types of ALS that run in families, “Because almost all of them are caused by toxic genes that misbehave. And what these treatments do is turn off those misbehaving genes.” We have used a virus to deliver into the spinal fluid a small DNA molecule that can turn off an ALS gene. The clinical result is that we see a reduction in the spinal cord of the activity of the gene that we have targeted. To my knowledge, this is the first time that a virus has been used with this DNA molecule, called a micro-RNA, to turn off a gene that causes a degenerative disease in the brain. It’s the first time that a virus and a micro-RNA have ever been used to turn off a disease gene – not just with ALS, with any disease. What these treatments do is turn off those misbehaving genes. It was back in 1993 that you discovered the ALS gene at issue here, called SOD1. In 1994, we published a paper with colleagues at Johns Hopkins showing we could turn off the gene in a petri dish. Usually, when we think about gene therapy, the term often conveys putting back a missing gene. Here what we’re doing is suppressing a misbehaving gene. It certainly is the onset of gene suppression therapy.

Keywords: [“gene”, “disease”, “virus”]
Source: https://www.wbur.org/commonhealth/2020/07/08/als-lou-gehrig-disease-silence-gene-treatment
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