Cyclophosphamide: a Therapeutic Option for Amyotrophic Lateral Sclerosis

Introduction: Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease, with about 3–4 years median survival from the onset of symptoms. Only two therapies for ALS have been proven effective in slowing down this condition, riluzole and edaravone. Recently approved edaravone, either original or generic, is not registered in Poland. It can only be used after special approval of Polish Ministry of Health. The costs of payment have to be covered by patient in full. Aim: In this article we will describe the public demand of edaravone in Poland for ALS treatment and problems related to prescribing the drug, like procedures and prices. Material and methods: This article is based on the available literature and on data obtained from Polish Ministry of Health. Results and discussion: The high cost of therapy affects the relatively widespread use of generic medicines not registered in Europe. We identify mechanisms of the demand and supply for various formulations of edaravone in Poland. These information should be of relevance to many other countries, especially within the European Union. Conclusions: Medicines available from licensed manufacturer are more expensive than offers available on-line. Although original chain of distribution presents with unrivaled warranty to avoid purchasing of adulterated drug we report the challenges of the edaravone treatment initiation in ALS patients in Poland.

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“Who Will I Be?”: Relational Identity, Living with Amyotrophic Lateral Sclerosis, and Future-Oriented Decisionmaking

Cambridge Quarterly of Healthcare Ethics ◽

10.1017/s0963180120000365 ◽

2020 ◽

Vol 29 (4) ◽

pp. 617-629

Author(s):

ERIKA VERSALOVIC ◽

ERAN KLEIN

Keyword(s):

Amyotrophic Lateral Sclerosis ◽

Family Involvement ◽

Therapeutic Option ◽

The Other ◽

Relational Identity ◽

Relational Approach ◽

Caregiver Relationship ◽

Shed Light ◽

Lateral Sclerosis ◽

Critical Aspects

AbstractPatients with amyotrophic lateral sclerosis (ALS) face many difficult, timing-sensitive decisions over the course of their illness, weighing present versus future harms and benefits. Supplemented by interviews with people with ALS, we argue for a relational approach to understanding these decisions and their effects on identity. We highlight two critical aspects of the patient–caregiver relationship: (1) the extent to which each may rely on the other leaves their wellbeing intimately intertwined and (2) patients often require others to help with the imaginative task of considering possible futures for each therapeutic option. We show why family involvement in decisionmaking practices can be so critical, and shed light on the ways intimate others help preserve and protect people’s identities amidst the destabilizing uncertainty illness and treatment can bring.

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The use of mesenchymal stem cells (MSCs) for amyotrophic lateral sclerosis (ALS) therapy – a perspective on cell biological mechanisms

Reviews in the Neurosciences ◽

10.1515/revneuro-2017-0018 ◽

2017 ◽

Vol 28 (7) ◽

pp. 725-738 ◽

Cited By ~ 6

Author(s):

Bor Luen Tang

Keyword(s):

Stem Cells ◽

Amyotrophic Lateral Sclerosis ◽

Mesenchymal Stem Cells ◽

Immune Cells ◽

Therapeutic Option ◽

Mechanisms Of Action ◽

Biological Mechanisms ◽

Beneficial Effects ◽

Motor Neuron Survival ◽

Lateral Sclerosis

AbstractRecent clinical trials of mesenchymal stem cells (MSCs) transplantation have demonstrated procedural safety and clinical proof of principle with a modest indication of benefit in patients with amyotrophic lateral sclerosis (ALS). While replacement therapy remained unrealistic, the clinical efficacy of this therapeutic option could be potentially enhanced if we could better decipher the mechanisms underlying some of the beneficial effects of transplanted cells, and work toward augmenting or combining these in a strategic manner. Novel ways whereby MSCs could act in modifying disease progression should also be explored. In this review, I discuss the known, emerging and postulated mechanisms of action underlying effects that transplanted MSCs may exert to promote motor neuron survival and/or to encourage regeneration in ALS. I shall also speculate on how transplanted cells may alter the diseased environment so as to minimize non-neuron cell autonomous damages by immune cells and astrocytes.

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Quality of life and muscle strength improvement in an amyotrophic lateral sclerosis patient after nutraceuticals

Vestnik of Saint Petersburg University Medicine ◽

10.21638/spbu11.2020.308 ◽

2020 ◽

Vol 15 (3) ◽

pp. 221-227

Author(s):

Jozélio Freire de Carvalho ◽

◽

João Ricardo Yamasita ◽

Leonid Churilov ◽

◽

...

Keyword(s):

Quality Of Life ◽

Amyotrophic Lateral Sclerosis ◽

Muscle Strength ◽

Therapeutic Option ◽

Amyotrophic Lateral Sclerosis Patient ◽

Well Being ◽

Marrow Transplant ◽

Tea Extract ◽

Lateral Sclerosis

The objective of the article was to describe a patient with amyotrophic lateral sclerosis (ALS) treated with nutraceuticals who had a better quality of life and improved muscle strength. A 65-year-old male patient with a past medical history of gout was diagnosed with amyotrophic lateral sclerosis in 2016. He received intravenous immunoglobulin and prednisone, but no improvement was observed. In 2018, he was diagnosed with multiple myeloma, submitted to a bone marrow transplant, and initiated riluzole. After 2 months, he came to our private clinic; he could not walk anymore and used outpatient nasal ventilator equipment. Beck anxiety inventory (BAI) was 20 [normal range (nr): < 8], Beck depression inventory (BDI) was 12 (nr: < 10), Bristol stool form scale (BSTS) was 5 (nr: 3–4), and there were 7 symptoms of dysbiosis. Analogic visual scale (Lickert scale) for well-being was 5.0. We suspended the colchicine, added vitamin D3, creatine, vitamin C, N-acetyl cysteine, 5-hydroxytryptamine, B1, and B6 vitamins. Dysbiosis was also treated. After 2 months, he returned feeling much better, BAI and BDI reduced BDI to 11, and BSFS normalized. Following this evaluation, a nutraceutical prescription was added: methylfolate, zinc, magnesium, green tea extract, Ginkgo biloba, lipoic acid, pyrroloquinoline quinone, vitamin E, coenzyme Q10, and resveratrol. After 7 months, he was feeling very well; BAI and BDI were normal, he gained weight. He felt a marked improvement in his muscle strength, and he gained again the capacity of eating alone, his quality of life improved, and AVS well-being was 8.0. This report illustrates a demonstrative case of a patient with ALS treated with nutraceuticals and improved his quality of life and muscle strength. It may be an alternative therapeutic option for such patients.

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Riluzole Oral Suspension for the Treatment of Amyotrophic Lateral Sclerosis: Texture and Compatibility with Food Thickeners Evaluation

J ◽

10.3390/j3030021 ◽

2020 ◽

Vol 3 (3) ◽

pp. 275-288

Author(s):

Giuseppe Colombo ◽

Roberta Artico ◽

Daniele Barbareschi

Keyword(s):

Amyotrophic Lateral Sclerosis ◽

Therapeutic Option ◽

Oral Suspension ◽

Test Results ◽

Related Substances ◽

Flow Test ◽

Oral Liquid ◽

Als Patients ◽

Liquid Medicine ◽

Lateral Sclerosis

Riluzole 5 mg/mL oral suspension is the only licensed liquid medicine to treat Amyotrophic Lateral Sclerosis (ALS) orally. As more than 80% of ALS patients develop dysphagia, an oral liquid formulation provides an important therapeutic option. The Riluzole 5 mg/mL oral suspension is administered by means of the graduated oral dosing syringe included in the medicine package. Its concentration (5 mg/mL) is consistent with a small and easy to measure volume (10 mL) to deliver the prescribed 50-mg dose twice daily. This work had a dual objective. The first was to evaluate the texture of the Riluzole 5 mg/mL oral suspension according to the International Dysphagia Diet Standardisation Initiative (IDDSI) flow test. Results of this experiment indicated that Riluzole 5 mg/mL oral suspension would basically fall under the “mildly thick” IDDSI descriptors. This is an important feature because thick fluids facilitate a safer swallow in patients with dysphagia. As a second objective, we evaluated for scientific purposes the compatibility of Riluzole 5 mg/mL oral suspension with some of the most common food thickeners available on the market. Intimate mixtures of the Riluzole 5 mg/mL oral suspension with thickeners were evaluated for appearance, pH, Riluzole assay and Riluzole related substances immediately after preparation and after two hours at room temperature. Riluzole 5 mg/mL oral suspension resulted to be compatible with all the marketed thickeners tested.

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Automated Acoustic Analysis of Oral Diadochokinesis to Assess Bulbar Motor Involvement in Amyotrophic Lateral Sclerosis

Journal of Speech Language and Hearing Research ◽

10.1044/2019_jslhr-19-00178 ◽

2020 ◽

Vol 63 (1) ◽

pp. 59-73 ◽

Cited By ~ 3

Author(s):

Panying Rong

Keyword(s):

Amyotrophic Lateral Sclerosis ◽

Acoustic Analysis ◽

Speaking Rate ◽

Disease Stage ◽

Healthy Controls ◽

Tongue Movement ◽

Major Barrier ◽

Syllable Repetition ◽

Oral Diadochokinesis ◽

Lateral Sclerosis

Purpose The purpose of this article was to validate a novel acoustic analysis of oral diadochokinesis (DDK) in assessing bulbar motor involvement in amyotrophic lateral sclerosis (ALS). Method An automated acoustic DDK analysis was developed, which filtered out the voice features and extracted the envelope of the acoustic waveform reflecting the temporal pattern of syllable repetitions during an oral DDK task (i.e., repetitions of /tɑ/ at the maximum rate on 1 breath). Cycle-to-cycle temporal variability (cTV) of envelope fluctuations and syllable repetition rate (sylRate) were derived from the envelope and validated against 2 kinematic measures, which are tongue movement jitter (movJitter) and alternating tongue movement rate (AMR) during the DDK task, in 16 individuals with bulbar ALS and 18 healthy controls. After the validation, cTV, sylRate, movJitter, and AMR, along with an established clinical speech measure, that is, speaking rate (SR), were compared in their ability to (a) differentiate individuals with ALS from healthy controls and (b) detect early-stage bulbar declines in ALS. Results cTV and sylRate were significantly correlated with movJitter and AMR, respectively, across individuals with ALS and healthy controls, confirming the validity of the acoustic DDK analysis in extracting the temporal DDK pattern. Among all the acoustic and kinematic DDK measures, cTV showed the highest diagnostic accuracy (i.e., 0.87) with 80% sensitivity and 94% specificity in differentiating individuals with ALS from healthy controls, which outperformed the SR measure. Moreover, cTV showed a large increase during the early disease stage, which preceded the decline of SR. Conclusions This study provided preliminary validation of a novel automated acoustic DDK analysis in extracting a useful measure, namely, cTV, for early detection of bulbar ALS. This analysis overcame a major barrier in the existing acoustic DDK analysis, which is continuous voicing between syllables that interferes with syllable structures. This approach has potential clinical applications as a novel bulbar assessment.

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