Effective treatment of barotrauma: A Synthesis of Findings from 36 Studies

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Original Abstract of the Article

Major Findings

Riluzole is the only approved treatment for amyotrophic lateral sclerosis (ALS) and has been used since 1995, although its mechanism of action in slowing the progression of this disease remains unclear. 12 . A review of the literature found that riluzole had a wide range of effects on factors influencing neural activity, particularly the neuromotor system. 12 Treatment with riluzole in human ALS patients or transgenic rodent models of ALS most commonly produced a modest but significant extension of lifespan. 12 Riluzole treatment was well tolerated in humans and animals. 12 Riluzole 100mg daily is probably effective in prolonging median survival by about two to three months in patients with amyotrophic lateral sclerosis. 8 . In contrast, lithium in combination with riluzole did not slow ALS progression. 13 . This suggests that genotype-targeted treatments may be more effective. 22 Edaravone is approved for ALS treatment, but only short-term effectiveness has been demonstrated. 30 Edaravone has been shown to slow the decline in the ALS Functional Rating Scale-Revised (ALSFRS-R) score in patients with less severe ALS. 21 Ultrahigh-dose methylcobalamin may be effective in slowing functional decline in patients with early-stage ALS and moderate progression rate. 32 Tauroursodeoxycholic acid (TUDCA) shows promise as a neuroprotective agent for ALS. 36 RNS60 is an immunomodulatory and neuroprotective investigational product that shows promise in animal models of ALS. 34 Rapamycin could modify ALS expression by inhibiting the mTOR pathway and enhancing autophagy. 24 Various treatments are being developed to improve the quality of life for ALS patients, such as respiratory care, mental health support, communication skills training, and exercises. 27 Exercise therapy and cognitive behavioral therapy (CBT) may also improve function and quality of life in ALS patients. 14 In ALS treatment, non-invasive ventilation (NIV) has been shown to prolong survival and improve quality of life. 31

Treatment Summary

Treatments for ALS currently under research include riluzole, edaravone, methylcobalamin, TUDCA, RNS60, and rapamycin. Riluzole has been shown to extend survival by approximately 2-3 months. 8 Edaravone has been shown to slow the decline in the ALSFRS-R score in patients with less severe ALS. 21 Ultrahigh-dose methylcobalamin shows promise in slowing functional decline in early-stage ALS patients. 32 TUDCA is a neuroprotective agent with potential as an ALS treatment. 36 RNS60 is an immunomodulatory and neuroprotective investigational product that shows promise in animal models of ALS. 34 Rapamycin may modify ALS expression by inhibiting the mTOR pathway and enhancing autophagy. 24

Benefits and Risks

Benefit Summary

Riluzole has been shown to extend survival by approximately 2-3 months. 8 Edaravone has been shown to slow the decline in the ALSFRS-R score in patients with less severe ALS. 21 Ultrahigh-dose methylcobalamin shows promise in slowing functional decline in early-stage ALS patients. 32

Risk Summary

Riluzole may cause elevated serum alanine aminotransferase levels. 8 Lithium may cause falls or back pain. 13

Comparison of Studies

Commonalities

Many studies are focused on developing treatments to improve quality of life for ALS patients, including respiratory care, mental health support, communication skills training, and exercise. 27 Exercise therapy and cognitive behavioral therapy (CBT) may also improve function and quality of life in ALS patients. 14

Differences

Each study utilizes different treatment methods and assessment techniques.

Consistency and Discrepancies of Results

Riluzole has been shown to extend survival. 8 Lithium in combination with riluzole, however, did not slow ALS progression. 13 Edaravone has only demonstrated short-term effectiveness. 30

Considerations for Real-World Application

Many challenges still exist in ALS treatment. It is important to consult with a physician when choosing treatment options. The effectiveness of treatments can vary from person to person, so do not expect a cure or overly optimistic outcomes.

Limitations of Current Research

Research on ALS treatment is still insufficient. More research is needed.

Future Research Directions

The following research areas are crucial for developing ALS treatments:

  • Determining the cause of ALS
  • Developing new treatment methods
  • Validating the effectiveness and safety of existing treatments
  • Improving quality of life for ALS patients

Conclusion

Many challenges remain in the treatment of ALS. However, research is progressing, and the development of more effective treatments in the future is expected.

Treatment List

  • Riluzole
  • Edaravone
  • Methylcobalamin
  • TUDCA
  • RNS60
  • Rapamycin
  • Exercise Therapy
  • Cognitive Behavioral Therapy (CBT)
  • Non-Invasive Ventilation (NIV)
Literature analysis of 36 papers
Positive Content
28
Neutral Content
3
Negative Content
5
Article Type
13
7
22
19
35
1.

Low doses of TRH in amyotrophic lateral sclerosis and in other neurological diseases.

Author: CongiaS, TronciS, LeddaM, PorcellaA, CoppolaG

ALS treatment

This study evaluated the effects of low-dose thyrotropin-releasing hormone (TRH) treatment in 30 patients with various neurological conditions, including amyotrophic lateral sclerosis (ALS), Charcot-Marie Tooth atrophy, and spinal muscular atrophy. TRH treatment resulted in significant neurological improvement in 17 out of 23 patients with ALS, but showed little or no improvement in other patients with ALS or other neurological diseases. This suggests that TRH may be a potential treatment for some patients with ALS.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

2.

Dose-ranging study of riluzole in amyotrophic lateral sclerosis. Amyotrophic Lateral Sclerosis/Riluzole Study Group II.

Author: LacomblezL, BensimonG, LeighP N, GuilletP, MeiningerV

Improved ALS treatment

This study investigates the effectiveness of riluzole, a drug previously shown to slow muscle weakness and decrease mortality in ALS patients, in a double-blind, placebo-controlled, multicenter trial. The study aims to confirm the initial findings and assess the drug's effectiveness at different doses.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Comparative StudyResearch that compares multiple groups/interventions to determine differences/similarities in outcomes/characteristics.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

3.

Riluzole and ALS therapy.

Author: HugonJ

Improved ALS survival

Amyotrophic lateral sclerosis (ALS) is a serious neurological disorder characterized by progressive muscle weakness and nerve damage. While the cause is unknown, potential factors include genetic mutations, autoimmune problems, and excessive nerve stimulation (excitotoxicity). Riluzole, a medication that reduces nerve stimulation, was studied in 77 patients with ALS. After one year, 74% of those taking riluzole were still alive compared to 58% of those taking a placebo. The increased survival was significant for all patients and those with bulbar-onset ALS (ALS that starts in the brainstem).

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

4.

Selegiline is ineffective in a collaborative double-blind, placebo-controlled trial for treatment of amyotrophic lateral sclerosis.

Author: LangeD J, MurphyP L, DiamondB, AppelV, LaiE C, YoungerD S, AppelS H

Selegiline ineffective
Worsening depression

Selegiline treatment had no significant effect on the rate of clinical progression or outcome of amyotrophic lateral sclerosis (ALS) in a 6-month, double-blind, placebo-controlled study.

Clinical TrialA clinical trial is a planned study conducted on humans to evaluate the efficacy and safety of pharmaceuticals, medical devices, and treatment methods. It aims to explore the possibilities of new treatments and improve the health of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

5.

Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND).

Author: MillerR G, MitchellJ D, MooreD H

Prolonged survival
Increased alanine transferase

Riluzole 100 mg per day appears to be modestly effective in prolonging survival for patients with ALS.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

6.

Examining the evidence about treatment in ALS/MND.

Author: MillerR G

Enhanced quality of life
Prolonged survival

While evidence-based approaches to treating amyotrophic lateral sclerosis (ALS) are developing, research is limited. This review discusses current recommendations for ALS, including early use of feeding tubes for swallowing difficulties, non-invasive ventilation for breathing problems, and aggressive management of pain and shortness of breath. The review also describes a large database for tracking ALS outcomes and examines the evidence supporting riluzole, a medication that extends survival with few side effects.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

7.

Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND).

Author: MillerR G, MitchellJ D, MooreD H

Prolongs survival
Increased ALT

Riluzole 100 mg per day appears to be modestly effective in prolonging survival for patients with ALS.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

8.

Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND).

Author: MillerR G, MitchellJ D, LyonM, MooreD H

Prolonged survival
Alanine transferase increase

Riluzole 100 mg daily is reasonably safe and probably prolongs median survival by about two to three months in patients with amyotrophic lateral sclerosis.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

9.

Drug therapy for pain in amyotrophic lateral sclerosis or motor neuron disease.

Author: BrettschneiderJohannes, KurentJerome, LudolphAlbert, MitchellJ Douglas

Improved ALS pain treatment
Amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis (ALS), also called motor neuron disease (MND), is the most common neurological disorder affecting movement in adults. Pain is a common symptom, especially in the later stages, and can significantly affect quality of life and distress. Treating pain is a crucial part of providing supportive care for people with ALS.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

10.

Treatment for familial amyotrophic lateral sclerosis/motor neuron disease.

Author: BenatarMichael, KurentJerome, MooreDan H

Improved amyotrophic lateral sclerosis treatment
Amyotrophic lateral sclerosis

Amyotrophic lateral sclerosis (ALS), also called motor neuron disease (MND), is a rare neurological disease. About 5-7% of people with ALS/MND have a family history of the disease. A gene mutation is responsible for about 20% of familial cases. The cause of ALS/MND in people without a family history is unknown, as is whether people with familial and non-familial ALS/MND respond differently to treatment.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

11.

A systematic review of the effect of moderate intensity exercise on function and disease progression in amyotrophic lateral sclerosis.

Author: LuiAndrew J, BylNancy N

ALS treatment

Amyotrophic lateral sclerosis (ALS) is a progressive disease affecting nerve cells that control muscle movement, leading to weakness, stiffness, and breathing problems. While treatment options are limited to medication and supportive care, the potential benefits and risks of moderate exercise are debated. This review examined current research on the use of moderate exercise to maintain independence in people with ALS without accelerating disease progression.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

12.

A review of the neural mechanisms of action and clinical efficiency of riluzole in treating amyotrophic lateral sclerosis: what have we learned in the last decade?

Author: BellinghamMark C

ALS treatment

This systematic review examines the various effects of riluzole, the only approved treatment for amyotrophic lateral sclerosis (ALS), on neural activity. The review highlights the diverse effects of riluzole across a range of doses and discusses its potential benefits and mechanisms of action in slowing ALS progression.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

13.

Safety and efficacy of lithium in combination with riluzole for treatment of amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial.

Author: AggarwalSwati P, ZinmanLorne, SimpsonElizabeth, McKinleyJane, JacksonKatherine E, PintoHanika, KaufmanPetra, ConwitRobin A, SchoenfeldDavid, ShefnerJeremy, CudkowiczMerit,

Delayed elimination of methotrexate

Lithium in combination with riluzole did not slow progression of ALS more than riluzole alone.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, N.I.H., ExtramuralResearch funding provided by the NIH to external research institutions and researchers.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

14.

Effects of aerobic exercise therapy and cognitive behavioural therapy on functioning and quality of life in amyotrophic lateral sclerosis: protocol of the FACTS-2-ALS trial.

Author: van GroenestijnAnnerieke C, van de PortIngrid G L, SchröderCarin D, PostMarcel W M, GrupstraHepke F, KruitwagenEsther T, van der LindeHarmen, van VlietReinout O, van de WeerdMargreet G H, van den BergLeonard H, LindemanEline

Improved ALS management

This study discusses the importance of multidisciplinary care for patients with amyotrophic lateral sclerosis (ALS), focusing on the potential benefits of aerobic exercise therapy (AET) and cognitive behavioral therapy (CBT) in maintaining function and improving quality of life (QoL). Further research is needed to support these approaches.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

15.

Repetitive transcranial magnetic stimulation for the treatment of amyotrophic lateral sclerosis or motor neuron disease.

Author: GuoJian, ZhouMuke, YangMi, ZhuCairong, HeLi

Treatment of ALS

Amyotrophic lateral sclerosis (ALS), or motor neuron disease (MND), is a progressive disease without effective treatments. Some studies suggest that repetitive transcranial magnetic stimulation (rTMS) might be helpful for ALS. However, the effectiveness and safety of rTMS for ALS are still unclear.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

16.

Repetitive transcranial magnetic stimulation for the treatment of amyotrophic lateral sclerosis or motor neuron disease.

Author: FangJinghuan, ZhouMuke, YangMi, ZhuCairong, HeLi

Amyotrophic lateral sclerosis (ALS), a progressive neurological disease, currently has no effective treatment. While some research suggests that repetitive transcranial magnetic stimulation (rTMS) may be beneficial, its effectiveness and safety are uncertain. This updated review examines the latest research on this topic.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

17.

Drug therapy for pain in amyotrophic lateral sclerosis or motor neuron disease.

Author: BrettschneiderJohannes, KurentJerome, LudolphAlbert

ALS pain treatment

Amyotrophic lateral sclerosis (ALS), a debilitating nerve disease, commonly causes pain, especially in the later stages. Pain management is crucial for improving quality of life and reducing suffering in ALS patients. This review updates information on treating pain in ALS.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

18.

Therapeutic development in amyotrophic lateral sclerosis.

Author: BucchiaMonica, RamirezAgnese, ParenteValeria, SimoneChiara, NizzardoMonica, MagriFrancesca, DamettiSara, CortiStefania

ALS treatment

Amyotrophic lateral sclerosis (ALS) is a common nerve disease that is fatal within a few years. While there is no effective cure, recent research has focused on understanding the disease and potential treatments. The review examines new treatments under development, highlighting their specific targets within the body.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

19.

Cell-based therapies for amyotrophic lateral sclerosis/motor neuron disease.

Author: Abdul WahidS Fadilah, LawZhe Kang, IsmailNor Azimah, Azman AliRaymond, LaiNai Ming

ALS/MND treatment

Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND), is a fatal condition causing progressive weakness and paralysis. While there's no cure, current treatments focus on managing symptoms to improve quality of life. Research suggests that cell-based therapy could be a promising treatment option for ALS/MND based on preclinical studies.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

20.

Symptomatic treatments for amyotrophic lateral sclerosis/motor neuron disease.

Author: NgLouisa, KhanFary, YoungCarolyn A, GaleaMary

MND quality of life
MND survival

This overview highlighted the lack of robust evidence in Cochrane Systematic Reviews on interventions to manage symptoms resulting from motor neuron disease (MND). The overview found that there is a significant gap in studies assessing the effectiveness of interventions for symptoms relating to MND, such as pseudobulbar emotional lability and cognitive and behavioural difficulties. More research with appropriate study designs, robust methodology, and of sufficient duration is needed to address the changing needs of people with MND and their caregivers associated with MND disease progression and mortality.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

21.

Exploratory double-blind, parallel-group, placebo-controlled study of edaravone (MCI-186) in amyotrophic lateral sclerosis (Japan ALS severity classification: Grade 3, requiring assistance for eating, excretion or ambulation).

Author:

Adverse events

This double-blind, randomized study explored the efficacy and safety of edaravone in amyotrophic lateral sclerosis (ALS) patients with a Japan ALS severity classification of Grade 3. The study found no statistically significant difference in the change in the ALSFRS-R score between the edaravone and placebo groups, and the incidence of adverse events was similar in both groups.

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

22.

Meta-analysis of pharmacogenetic interactions in amyotrophic lateral sclerosis clinical trials.

Author: van EijkRuben P A, JonesAshley R, SprovieroWilliam, ShatunovAleksey, ShawPamela J, LeighP Nigel, YoungCarolyn A, ShawChristopher E, MoraGabriele, MandrioliJessica, BorgheroGiuseppe, VolantiPaolo, DiekstraFrank P, van RheenenWouter, VerstraeteEsther, EijkemansMarinus J C, VeldinkJan H, ChioAdriano, Al-ChalabiAmmar, van den BergLeonard H, van EsMichael A,

genotype-targeted treatments
no treatment effect

This study assessed the efficacy of lithium carbonate in treating amyotrophic lateral sclerosis (ALS) based on genetic subgroups. The study found no overall improvement in survival with lithium carbonate, but suggested that specific genetic subgroups may respond to the treatment.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

23.

Treatment of fatigue in amyotrophic lateral sclerosis/motor neuron disease.

Author: GibbonsChris, PagniniFrancesco, FriedeTim, YoungCarolyn A

ALS/MND treatment

Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND), is a fatal condition with no cure. Fatigue is a common and disabling symptom in ALS/MND. While there are treatments available, their effectiveness for fatigue in ALS/MND is not yet proven.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

24.

Rapamycin treatment for amyotrophic lateral sclerosis: Protocol for a phase II randomized, double-blind, placebo-controlled, multicenter, clinical trial (RAP-ALS trial).

Author: MandrioliJessica, D'AmicoRoberto, ZucchiElisabetta, GessaniAnnalisa, FiniNicola, FasanoAntonio, CaponnettoClaudia, ChiòAdriano, Dalla BellaEleonora, LunettaChristian, MazziniLetizia, MarinouKalliopi, SorarùGianni, de BiasiSara, Lo TartaroDomenico, PintiMarcello, CossarizzaAndrea,

Improved ALS treatment

Amyotrophic lateral sclerosis (ALS) is a serious brain disease caused by abnormal proteins and inflammation. Rapamycin, a medication that blocks a pathway called mTOR and helps cells break down waste products, has shown benefits in animal studies of ALS. It also increases levels of regulatory T lymphocytes (Treg), which are linked to slower progression of the disease in people with ALS. This study will investigate whether rapamycin can be an effective treatment for ALS in a randomized clinical trial.

Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

25.

Regulatory T cells for amyotrophic lateral sclerosis/motor neuron disease: A clinical and preclinical systematic review.

Author: RajabinejadMisagh, RanjbarSedigheh, Afshar HezarkhaniLeila, SalariFarhad, Gorgin KarajiAli, RezaiemaneshAlireza

Prolonged survival

This meta-analysis of preclinical studies and review of clinical trials suggests that regulatory T cells (Tregs) may be an effective treatment for amyotrophic lateral sclerosis (ALS). Preclinical studies showed that Tregs significantly prolonged survival in mice models of ALS. However, there is limited clinical data available, with only one RCT published as an abstract and three non-RCTs. Further research is needed to confirm the effectiveness of Tregs in treating ALS.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

26.

Cell-based therapies for amyotrophic lateral sclerosis/motor neuron disease.

Author: Abdul WahidS Fadilah, LawZhe Kang, IsmailNor Azimah, LaiNai Ming

Improved ALS/MND treatment

This updated review examined the potential of cell-based therapies for amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND), incorporating evidence from randomized controlled trials (RCTs).

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

27.

Quality of life and functional independence in amyotrophic lateral sclerosis: A systematic review.

Author: Rosa SilvaJéssica Paloma, Santiago JúniorJosé Bomfim, Dos SantosElizabete Lima, de CarvalhoFernanda Oliveira, de França CostaIandra Maria Pinheiro, MendonçaDeise Maria Furtado de

Improved ALS treatment

This systematic review analyzes treatment protocols and their outcomes in clinical trials focusing on ALS rehabilitation and its effects on quality of life and functional independence.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

29.

Perampanel for amyotrophic lateral sclerosis: A systematic review and meta-analysis.

Author: TuraldeChristian Wilson R, MoalongKevin Michael C, EspirituAdrian I, PradoMario B

ALS treatment

Amyotrophic lateral sclerosis (ALS) is a fatal and incurable disease that affects the nervous system. There's no effective treatment that can improve function or prolong life for people with ALS. A medication called perampanel might help slow the disease by protecting nerve cells.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Meta-AnalysisA statistical technique that integrates the results of multiple studies to draw more general conclusions about a particular issue.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.

Language : English

30.

Safety and Effectiveness of Long-term Intravenous Administration of Edaravone for Treatment of Patients With Amyotrophic Lateral Sclerosis.

Author: WitzelSimon, MaierAndré, SteinbachRobert, GrosskreutzJulian, KochJan C, SarikidiAnastasia, PetriSusanne, GüntherRené, WolfJoachim, HermannAndreas, PrudloJohannes, CordtsIsabell, LingorPaul, LöscherWolfgang N, KohlZacharias, HagenackerTim, RuckesChristian, KochBirgit, SpittelSusanne, GüntherKornelia, MichelsSebastian, DorstJohannes, MeyerThomas, LudolphAlbert C,

No disease modifying benefit

This multicenter, propensity score-matched cohort study found that, although long-term intravenous edaravone therapy for patients with amyotrophic lateral sclerosis (ALS) was feasible and mainly well tolerated, it was not associated with any disease-modifying benefit.

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.

Language : English

31.

Proposals from a French expert panel for respiratory care in ALS patients.

Author: GeorgesM, PerezT, RabecC, JacquinL, Finet-MonnierA, RamosC, PatoutM, AttaliV, AmadorM, Gonzalez-BermejoJ, SalachasF, Morelot-PanziniC

Improved quality of life
Prolonged survival

This study aimed to develop evidence-based guidelines for respiratory care in patients with Amyotrophic Lateral Sclerosis (ALS) in French tertiary care centers. The study highlighted the importance of non-invasive ventilation (NIV) in alleviating respiratory symptoms and prolonging survival but emphasized the need for further research on the optimal timing for NIV initiation.

Practice GuidelineGuidelines and recommendations for the diagnosis and treatment of specific medical conditions in medical practice.
Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

32.

Efficacy and Safety of Ultrahigh-Dose Methylcobalamin in Early-Stage Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial.

Author: OkiRyosuke, IzumiYuishin, FujitaKoji, MiyamotoRyosuke, NoderaHiroyuki, SatoYasutaka, SakaguchiSatoshi, NokiharaHiroshi, KanaiKazuaki, TsunemiTaiji, HattoriNobutaka, HatanakaYuki, SonooMasahiro, AtsutaNaoki, SobueGen, ShimizuToshio, ShibuyaKazumoto, IkedaKen, KanoOsamu, NishinakaKazuto, KojimaYasuhiro, OdaMasaya, KomaiKiyonobu, KikuchiHitoshi, KoharaNobuo, UrushitaniMakoto, NakayamaYoshiaki, ItoHidefumi, NagaiMakiko, NishiyamaKazutoshi, KuzumeDaisuke, ShimohamaShun, ShimohataTakayoshi, AbeKoji, IshiharaTomohiko, OnoderaOsamu, IsoseSagiri, ArakiNobuyuki, MoritaMitsuya, NodaKazuyuki, TodaTatsushi, MaruyamaHirofumi, FuruyaHirokazu, TeramukaiSatoshi, KagimuraTatsuo, NomaKensuke, YanagawaHiroaki, KuwabaraSatoshi, KajiRyuji,

Safety
Slowed functional decline

This randomized clinical trial evaluated the efficacy and safety of ultrahigh-dose methylcobalamin for early-stage amyotrophic lateral sclerosis (ALS). 130 patients were randomly assigned to receive either methylcobalamin or placebo. Methylcobalamin showed efficacy in slowing functional decline in patients with moderate progression rate and was well tolerated.

Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

33.

Associations between urate levels and amyotrophic lateral sclerosis functional score with edaravone treatment: Post hoc analysis of studies MCI186-16, MCI186-17, and MCI186-19.

Author: TakahashiFumihiro, KanoOsamu, NaganoYoshito, YoneokaTakatomo, NelsonSally, UshirogawaYoshiteru

ALS progression

This study investigated the effects of edaravone on patients with more severe amyotrophic lateral sclerosis (ALS) who progressed to grades 3 and 4 during the double-blind treatment period in two phase 3 studies. It also assessed the association between ALS Functional Rating Scale-Revised (ALSFRS-R) slope and biomarker changes after open-label edaravone initiation.

Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

34.

Effect of RNS60 in amyotrophic lateral sclerosis: a phase II multicentre, randomized, double-blind, placebo-controlled trial.

Author: BeghiEttore, PupilloElisabetta, BianchiElisa, BonettoValentina, LuottiSilvia, PasettoLaura, BendottiCaterina, TortaroloMassimo, SironiFrancesca, CamporealeLaura, ShermanAlexander V, PaganoniSabrina, ScognamiglioAda, De MarchiFabiola, BongioanniPaolo, Del CarratoreRenata, CaponnettoClaudia, DiamantiLuca, MartinelliDaniele, CalvoAndrea, FilostoMassimiliano, PadovaniAlessandro, PiccinelliStefano Cotti, RicciClaudia, Dalla GiacomaStefania, De AngelisNicoletta, InghilleriMaurizio, SpataroRossella, La BellaVincenzo, LogroscinoGiancarlo, LunettaChristian, TarlariniClaudia, MandrioliJessica, MartinelliIlaria, SimoniniCecilia, ZucchiElisabetta, MonsurròMaria Rosaria, RicciardiDario, TrojsiFrancesca, RivaNilo, FilippiMassimo, SimoneIsabella Laura, SorarùGianni, SperaCristina, FlorioLucia, MessinaSonia, RussoMassimo, SicilianoGabriele, ConteAmelia, SaddiMaria Valeria, CarboniNicola, MazziniLetizia,

ALS treatment

Amyotrophic lateral sclerosis (ALS) is a fatal disease with limited treatment options. This study highlights a new treatment, RNS60, which has shown promise in animal studies and early human trials for ALS and other neurological diseases. RNS60 has a good safety profile and seems well-tolerated by patients with ALS.

Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Language : English

35.

Sensory neuropathy in amyotrophic lateral sclerosis: a systematic review.

Author: BombaciAlessandro, LupicaAntonino, PozziFederico Emanuele, RemoliGiulia, ManeraUmberto, Di StefanoVincenzo

ALS treatment

This systematic review examines the presence and characteristics of sensory neuropathy in patients with amyotrophic lateral sclerosis (ALS). ALS is a fatal neurodegenerative disease primarily affecting motor neurons, but recent evidence suggests that sensory neuropathy may also be a significant feature of the disease.

Systematic ReviewA review that systematically collects, evaluates, and integrates relevant research to answer a specific research question.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
ReviewAn article that critically examines existing research on a particular topic and summarizes the current state of knowledge in the field.

Language : English

36.

A randomized double-blind clinical trial on safety and efficacy of tauroursodeoxycholic acid (TUDCA) as add-on treatment in patients affected by amyotrophic lateral sclerosis (ALS): the statistical analysis plan of TUDCA-ALS trial.

Author: LombardoFlavia L, Spila AlegianiStefania, MayerFlavia, CiprianiMarta, Lo GiudiceMaria, LudolphAlbert Christian, McDermottChristopher J, CorciaPhilippe, Van DammePhilip, Van den BergLeonard H, HardimanOrla, NicoliniGabriele, VanacoreNicola, DickieBrian, AlbaneseAlberto, PuopoloMaria,

ALS treatment

Amyotrophic lateral sclerosis (ALS) is a serious and debilitating brain disease with no cure. While a medication called riluzole can slow down the progression of ALS and slightly extend lifespan, we need new treatments. A medication called tauroursodeoxycholic acid (TUDCA) shows promise as a potential treatment for ALS due to its ability to protect nerve cells.

Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Multicenter StudyResearch conducted in collaboration with multiple research institutions. It aims to collect a wide range of data and obtain more generalizable results.
Clinical Trial, Phase IIIA Phase III trial is a trial that further rigorously verifies the efficacy and safety of a new drug or treatment, whose efficacy and safety have been confirmed in a Phase II trial, in a large number of patients, and evaluates whether it is superior to existing treatments.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.

Language : English

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