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Publications (ALS)

The importance of non-motor symptoms in the clinical picture of motor neuron disease

Non-motor symptoms are an obligate sign of motor neuron disease and can often serve as a rather sensitive indicator of the general functional state of ALS patients. Gastrointestinal disorders, weight loss, pain, sweating and anxiety prevail in the structure of non-motor symptoms in ALS patients

The presence of non-motor symptoms in patients indicates the multisystemic nature of this fatal disease. The use of Non-Motor Symptom Scale (NMS) in patients with ALS in the early stages will allow neurologists to detect non-motor symptoms timely.

Authors: V. V. KOSTENKO, Y. I. GOLOVCHENKOP. L. Shupyk National Medical Academy оf Postgraduate Education Kyiv
Posted: Ukrainian Neurological Journal 2020, №3
How is edaravone effective against acute ischemic stroke and amyotrophic lateral  sclerosis?

Abstract. Edaravone isa low-molecular-weight antioxidant drug targeting peroxyl radicals among many types of reactive oxygen species. Because of its amphiphilicity, it scavenges both lipid- and water-soluble peroxyl radicals by donating an electron to the radical. Thus, it inhibits the oxidation of lipids by scavenging chain-initiating water-soluble peroxyl radicals and chain-carrying lipid peroxyl radicals. In 2001, it was approved in Japan as a drug to treat acute-phase cerebral infarction, and then in 2015 it was approved for amyotrophic lateral sclerosis (ALS). In 2017, the U.S. Food and Drug Administration also approved edaravone for treatment of patients with ALS. Its mechanism of action was inferred to be scavenging of peroxynitrite. In this review, we focus on the radical-scavenging characteristics of edaravone in comparison with some other antioxidants that have been studied in clinical trials, and we summarize its pharmacological action and clinical efficacy in patients with acute cerebral infarction and ALS.

Authors: Kazutoshi Watanabe, Masahiko Tanaka, Satoshi Yuki, Manabu Hirai, Yorihiro Yamamoto
Posted: J. Clin. Biochem. Nutr. January 2018. Vol. 62, № 1. Р. 20-38
Ukrainian legislation on the prevention and treatment of rare (orphan) diseases

From 01 January 2015, amendments to the Fundamentals of Legislation of Ukraine on Health Care implemented by the Law of Ukraine No. 1213-VII dated 15 April 2014, have become effective. According to the implemented amendments to the legislation, patients with rare (orphan) diseases shall be provided, on a continuous and free basis, with medicinal products for the treatment of these diseases and appropriate food products for special dietary consumption in accordance with their list and volumes approved by the central executive body responsible for the formation of the national health care policy, in accordance with the procedure established by the Cabinet of Ministers of Ukraine.

At the same time, on 15 April 2015, the CMU Resolution No. 160 of 31 March 2015 “On approval of the procedure for providing citizens suffering from rare (orphan) diseases with medicines and appropriate food products for special dietary consumption” became effective. According to the specified Procedure, provision of the citizens suffering from rare (orphan) diseases with medicines and food products is performed by respective healthcare institutions according to the place of residence or treatment of such citizens.

References to laws:

Authors: Верховна Рада України; Кабінет міністрів України.
Posted: Відомості Верховної Ради (ВВР), 2014, № 26; Постанова Кабінету міністрів України №160 від 31.03.2015 р.
Extended revised ALS Scale (ALSFRS-R)

Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) is a tool for monitoring the progression of disability in patients with amyotrophic lateral sclerosis (ALS). The disadvantage of ALSFRS is that its rating of the bulbar functions and limb functionality is overweighted compared to the rating of respiratory dysfunction.

A revised ALS functional rating scale (ALSFRS-R) retains the properties of the original scale, while demonstrating a valid structure and internal weighting.

The revised ALS functional rating scale (ALSFRS-R) is useful for physicians and scientists to diagnose patients, track disease progression, study and compare the results of different ALS treatment regimens in clinical trials.

ALSFRS-R includes 12 questions scored from 0 to 4. 0 means no function, whereas 4 means full functionality.

Authors: ALS Functional Rating Scale Revised (ALS-FRS-R).
Posted: version of May 2015.
Safety and efficacy of edarowon in patients with amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled study

This randomized double-blind parallel trial (Phase III) was attended by patients aged 20-75 years with grade 1 or 2 ALS from 31 hospitals in Japan. Patients had a score of at least 2 for each of all 12 points of ALSFRS-R, a forced vital lung capacity of ≥ 80% and an established or suspected ALS according to the revised El Escorial criteria. Patients with duration of the disease not exceeding 2 years were subject to inclusion.

Between 28 November 2011 and 03 September 2014, 213 patients were examined and 192 potential participants were selected. Of these, 137 patients completed the full period of follow-up: 69 were randomized to receive edaravone, 68 were randomized to receive placebo. 68 patients who were administered edaravone and 66 who received placebo were included in the primary analysis of efficacy. As a result, the change in ALSFRS-R was -5.01±0.64 in the edaravon group and -7.50±0.66 in the placebo group. The difference between the groups was -2.49 (p=0.0013) in favor of edaravone. 58 (84%) patients treated with edaravone and 57 (84%) patients on placebo reported treatment-related side effects. 11 (16%) patients receiving edaravone and 16 (24%) receiving placebo had serious side effects; 1 (1%) patient receiving edaravone and 4 (6%) patients receiving placebo had adverse events (one case of dysphagia in the edaravone group versus one case of dyspnoea, two cases of respiratory disorders and one case of rash in the placebo group), which resulted in withdrawal of these patients from the study.

Therefore, edaravone has proven its effectiveness in patients with amyotrophic lateral sclerosis (ALS) who met the inclusion criteria, demonstrating a significantly lower rate of reduction in the assessment of ALS progression according to ALSFRS-R versus placebo.

Authors: The Writing Group on behalf of the Edaravone (MCI-186) ALS 19 Study Group.
Posted: сайт thelancet.com/neurology, 15 травня 2017.
In 2017, the FDA approved a new drug for the treatment of lateral amyotrophic sclerosis (press release, in English)

On 05 May 2017, US Food and Drug Administration (FDA) approved edaravone as an officially recommended drug product for the treatment of patients with amyotrophic lateral sclerosis (ALS).

The U.S. Food and Drug Administration today approved Radicava (edaravone) to treat patients with amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig’s disease.

“After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States,” said Eric Bastings, M.D., deputy director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option.”

ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles. Voluntary muscles produce movements such as chewing, walking, breathing and talking. The nerves lose the ability to activate specific muscles, which causes the muscles to become weak and leads to paralysis. ALS is progressive, meaning it gets worse over time. The Centers for Disease Control and Prevention estimates that approximately 12,000-15,000 Americans have ALS. Most people with ALS die from respiratory failure, usually within three to five years from when the symptoms first appear. 

Radicava is an intravenous infusion given by a health care professional. It is administered with an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period. Subsequent treatment cycles consist of dosing on 10 of 14 days, followed by 14 days drug-free.

The efficacy of edaravone for the treatment of ALS was demonstrated in a six-month clinical trial conducted in Japan. In the trial, 137 participants were randomized to receive edaravone or placebo. At Week 24, individuals receiving edaravone declined less on a clinical assessment of daily functioning compared to those receiving a placebo.

The most common adverse reactions reported by clinical trial participants receiving edaravone were bruising (contusion) and gait disturbance.

Radicava is also associated with serious risks that require immediate medical care, such as hives, swelling, or shortness of breath, and allergic reactions to sodium bisulfite, an ingredient in the drug. Sodium bisulfite may cause anaphylactic symptoms that can be life-threatening in people with sulfite sensitivity.

The FDA granted this drug orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted approval of Radicava to Mitsubishi Tanabe Pharma America, Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency is also responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Authors: The U.S. Food and Drug Administration.
Posted: офіційний сайт FDA, травень 2017.