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Overview of Current and Emerging Therapies for Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis is a fatal neurodegenerative disorder characterized by the loss of cortical and spinal motor neurons, leading to weakness, muscle atrophy, and, in a substantial number of patients, cognitive impairment. Patients with older age, bulbar-onset, early respiratory dysfunction, and a lower score on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale have a poorer prognosis. A post hoc analysis of an open-label follow-up of 65 edaravone-treated patients and 58 placebo-treated patients who then received edaravone for 24 weeks showed continued benefit out to 48 weeks in the 93 patients who completed the follow-up. Practice parameter update: the care of the patient with amyotrophic lateral sclerosis: drug, nutritional, and respiratory therapies: report of the Quality Standards Subcommittee of the American Academy of Neurology. Chen JJ. Examining best practices for specialty pharmacists to improve the care of patients with amyotrophic lateral sclerosis. Meng L, Bian A, Jordan S, Wolff A, Shefner JM, Andrews J. Profile of medical care costs in patients with amyotrophic lateral sclerosis in the Medicare programme and under commercial insurance. Practice parameter update: the care of the patient with amyotrophic lateral sclerosis: multidisciplinary care, symptom management, and cognitive/behavioral impairment: report of the Quality Standards Subcommittee of the American Academy of Neurology. Hinchcliffe M, Smith A. Riluzole: real-world evidence supports significant extension of median survival times in patients with amyotrophic lateral sclerosis. Confirmatory double-blind, parallel-group, placebo-controlled study of efficacy and safety of edaravone in amyotrophic lateral sclerosis patients. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. On survival of patients with amyotrophic lateral sclerosis. Masitinib as an add-on therapy to riluzole in patients with amyotrophic lateral sclerosis: a randomized clinical trial.

Keywords: [“patient”, “ALS”, “disease”]

ALS Biorepository Brain Bank

The VA Biorepository Brain Bank is a human tissue bank that collects, processes, stores and gives out research specimens for future scientific studies. Veterans without ALS or other neurological diseases are also eligible to participate in the VABBB, because it is important to also study Veterans without neurological disease in order to learn more about the causes of ALS. ALS is a neurologic disease affecting the central nervous system, specifically the brain and spinal cord. The VA ALS Registry enrolled 2050 Veterans with ALS aged 23 to 93 years from combat eras spanning World War II to the 1990-1991 Gulf War. In addition to the VA ALS Registry, VA started the VABBB in 2006 to collect brain and spinal cord tissue from Veterans in the Registry. The VABBB has opened enrollment to all Veterans with ALS regardless of whether they were in the original VA ALS Registry. Weekends, or holidays please call our 24/7 cell phone for an on-call ALS Brain Bank researcher at 857-214-0992 if death is imminent. The Veterans Affairs Biorepository Brain Bank was established in 2006 to promote and support research in amyotrophic lateral sclerosis and other illnesses that affect Veterans by collecting brain and spinal cord tissues, along with relevant clinical data, essential for such research. Principal investigators who have the necessary preliminary data to support the use of greater numbers of specimens should submit a request using the Tissue Request Form. Requests for access to tissue for pilot projects are limited to the amounts described in the VABBB Tissue Request Guidelines. Justification of Tissue Requests: Applications should be carefully prepared to justify and fully describe the tissue requested. In certain situations, if a request has been made for highly sought after tissue the VABBB may suggest alternative tissue. Final decisions about access will be made on the basis of qualifications of the investigator, scientific merit of the proposed research utilizing the tissue, quantities of tissue requested, statistical issues and availability of tissue.

Keywords: [“tissue”, “ALS”, “request”]

New treatment may help slow progression of ALS, research shows

An experimental medication may slow the progression of amyotrophic lateral sclerosis, or ALS, researchers reported Wednesday. The drug is not a cure, but it may help slow the inexorable disability caused by ALS, which rapidly destroys the nerve cells that control the muscles that allow us to move, speak, eat and even breathe. “Patients keep telling me their No. 1 goal is to be able to retain physical function for as long as possible,” said the study’s lead author, Dr. Sabrina Paganoni, a neuromuscular specialist at Massachusetts General Hospital’s Sean M. Healey & AMG Center for ALS. “They want to be able to continue to walk and to use their hands.” About 20,000 people in the U.S. have ALS at any given time, according to the ALS Association. The treatment studied by Paganoni and her colleagues targets two cellular structures damaged by the disease: the mitochondria, which are the cells’ power plants, and the endoplasmic reticulum, the cellular dump trucks that cart away waste that can gunk up the cells’ machinery. The multicenter, randomized, double-blind study is the second step – a phase 2 trial – in a three-step process required by the Food and Drug Administration for drug approval. In a double-blind study, neither the patients nor the researchers know who is receiving the drug. If a phase 2 study generates positive results, the FDA typically requires a larger and longer phase 3 trial. To test the effectiveness of the two-drug combination, the researchers recruited 137 ALS patients who had become symptomatic within the previous 18 months. About two-thirds of the patients received the drug, while the remaining third were given a placebo. “By the time they entered the trial, on average, patients had already lost 12 points. Their baseline score was about 36, on average,” Paganoni said. During the six months of the study, patients taking the medication lost an average of 2.32 points less than those receiving placebos, a 25 percent better functional outcome.

Keywords: [“ALS”, “study”, “Patients”]
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