scholarly journals We need to conduct clinical trials of disease-modifying therapy in pregnancy to optimize care of women with MS – Yes

2018 ◽  
Vol 25 (2) ◽  
pp. 187-188 ◽  
Author(s):  
Enrique Alvarez ◽  
Ellen M Mowry
Keyword(s):  
Clinical Trials ◽  
Disease Modifying Therapy ◽  
Disease Modifying ◽  
In Pregnancy

Cutaneous cryptococcosis in a patient taking fingolimod for multiple sclerosis: Here come the opportunistic infections?

2017 ◽  
Vol 23 (2) ◽  
pp. 297-299 ◽  
Author(s):  
Adam F Carpenter ◽  
Shikha J Goodwin ◽  
Peter F Bornstein ◽  
Andrew J Larson ◽  
Christine K Markus
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trials ◽  
Case Report ◽  
Skin Lesion ◽  
Opportunistic Infections ◽  
Oral Disease ◽  
Disease Modifying Therapy ◽  
Post Marketing ◽  
Disease Modifying ◽  
Cutaneous Cryptococcosis

Background: Fingolimod is an oral disease-modifying therapy for relapsing forms of multiple sclerosis, which acts by sequestering lymphocytes within lymph nodes. Objective: To describe a case of extrapulmonary cryptococcosis in a patient taking fingolimod. Methods: Case report. Results: A 47-year-old man developed a non-healing skin lesion approximately 16 months after starting treatment with fingolimod. Biopsy revealed cryptococcosis. Fingolimod was discontinued and the lesion resolved with antifungal therapy. Conclusion: Despite few reported opportunistic infections in the pivotal clinical trials and first few years post-marketing, there has been a recent increase in reported AIDS-defining illnesses in patients taking fingolimod. Neurologists should be alert for opportunistic infections in their patients using this medication.

scholarly journals HAVING A GO AT SPINAL MUSCULAR ATROPHY WITH SPINRAZA

2017 ◽  
Vol 10 (6) ◽  
pp. 16
Author(s):  
Balaji O ◽  
Amita D ◽  
Sereen Rt ◽  
Navin Ap
Keyword(s):  
Cystic Fibrosis ◽  
Clinical Trials ◽  
Spinal Muscular Atrophy ◽  
Drug Administration ◽  
Muscular Atrophy ◽  
Food And Drug Administration ◽  
Neurological Condition ◽  
Disease Modifying Therapy ◽  
Different Types ◽  
Disease Modifying

Spinal muscular atrophy (SMA), a neurological condition which is genetically mediated is the second most common infantile disease causing morbidity and mortality next to cystic fibrosis. It is of five different types with each type having different severity outcomes. For almost three decades, only supportive measures were advocated in the treatment of SMA. Recently, Biogen’s Spinraza came out as the first disease modifying therapy to treat infantile as well as adult SMA. This review throws light on the pharmacological aspects of the drug; its approval by Food and Drug Administration and various completed clinical trials as well ongoing clinical trials.

scholarly journals Toward a Disease-Modifying Therapy of Alpha-Synucleinopathies: New Molecules and New Approaches Came into the Limelight

Molecules ◽  
2021 ◽  
Vol 26 (23) ◽  
pp. 7351
Author(s):  
Matthew Upcott ◽  
Kirill D. Chaprov ◽  
Vladimir L. Buchman
Keyword(s):  
Clinical Trials ◽  
Lewy Bodies ◽  
Alpha Synuclein ◽  
Therapeutic Window ◽  
Disease Modifying Therapy ◽  
Stage Of Development ◽  
Phase Ii Clinical Trials ◽  
Modifying Effect ◽  
Disease Modifying ◽  
Alpha Synuclein Aggregation

The accumulation of the various products of alpha-synuclein aggregation has been associated with the etiology and pathogenesis of several neurodegenerative conditions, including both familial and sporadic forms of Parkinson’s disease (PD) and dementia with Lewy bodies (DLB). It is now well established that the aggregation and spread of alpha-synuclein aggregation pathology activate numerous pathogenic mechanisms that contribute to neurodegeneration and, ultimately, to disease progression. Therefore, the development of a safe and effective disease-modifying therapy that limits or prevents the accumulation of the toxic intermediate products of alpha-synuclein aggregation and the spread of alpha-synuclein aggregation pathology could provide significant positive clinical outcomes in PD/DLB cohorts. It has been suggested that this goal can be achieved by reducing the intracellular and/or extracellular levels of monomeric and already aggregated alpha-synuclein. The principal aim of this review is to critically evaluate the potential of therapeutic strategies that target the post-transcriptional steps of alpha-synuclein production and immunotherapy-based approaches to alpha-synuclein degradation in PD/DLB patients. Strategies aimed at the downregulation of alpha-synuclein production are at an early preclinical stage of drug development and, although they have shown promise in animal models of alpha-synuclein aggregation, many limitations need to be resolved before in-human clinical trials can be seriously considered. In contrast, many strategies aimed at the degradation of alpha-synuclein using immunotherapeutic approaches are at a more advanced stage of development, with some in-human Phase II clinical trials currently in progress. Translational barriers for both strategies include the limitations of alpha-synuclein aggregation models, poor understanding of the therapeutic window for the alpha-synuclein knockdown, and variability in alpha-synuclein pathology across patient cohorts. Overcoming such barriers should be the main focus of further studies. However, it is already clear that these strategies do have the potential to achieve a disease-modifying effect in PD and DLB.

DEMENTIA PREVENTION BY DISEASE-MODIFICATION THROUGH NUTRITION

2017 ◽  
pp. 1-2
Author(s):  
A.D. Smith ◽  
H. Refsum
Keyword(s):  
Clinical Trials ◽  
Cognitive Impairment ◽  
Mild Cognitive Impairment ◽  
Good Evidence ◽  
Disease Modification ◽  
Disease Modifying Therapy ◽  
Dementia Prevention ◽  
Disease Modifying

It is agreed that disease-modification is the most desirable approach to tackling dementia (1), but we are often told that it is not yet feasible and that “no disease modifying therapy has proven effective in clinical trials” (2). The purpose of our Editorial is to show that disease-modification is not only feasible but that there is good evidence that it has already been achieved in subjects with Mild Cognitive Impairment (MCI).

Promising disease-modifying therapies for Parkinson’s disease

2019 ◽  
Vol 11 (520) ◽  
pp. eaba1659 ◽  
Author(s):  
Valina L. Dawson ◽  
Ted M. Dawson
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Clinical Trials ◽  
New Agents ◽  
Disease Modifying Therapy ◽  
Disease Modifying Therapies ◽  
Disease Modifying

To date, there is no disease-modifying therapy for Parkinson’s disease; however, promising new agents have advanced into clinical trials.

A Review of Friedreich Ataxia Clinical Trial Results

2012 ◽  
Vol 27 (9) ◽  
pp. 1217-1222 ◽  
Author(s):  
Susan L. Perlman
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Friedreich Ataxia ◽  
Therapeutic Agents ◽  
National Institutes Of Health ◽  
Disease Modifying Therapy ◽  
Disease Modifying ◽  
Clinical Trial Results ◽  
Research Alliance

There are now 21 agents or classes of therapeutic agents in the Friedreich ataxia research pipeline (http://www.curefa.org/pipeline.html) that have been developed in the 15 years since the discovery of the frataxin gene, with the ongoing characterization of its mutations and the resulting molecular pathology. Twenty-four studies are currently posted on ClinicalTrials.gov. Twenty-seven works discussing the results of clinical trials in Friedreich ataxia have been published. In 2010, 42 public (National Institutes of Health) and private (Friedreich Ataxia Research Alliance, Muscular Dystrophy Association, and National Ataxia Foundation) grants were funded for translational and clinical research in Friedreich ataxia. Millions of dollars from public, private, and industry-based initiatives have been dedicated to research in Friedreich ataxia therapeutics. Despite this vigorous international effort, there is as yet no proven disease-modifying therapy for Friedreich ataxia.

Implications of FDA Approval of a First Disease-Modifying Therapy for a Neurodegenerative Disease on the Design of Subsequent Clinical Trials

Neurology ◽  
2021 ◽  
pp. 10.1212/WNL.0000000000012329
Author(s):  
Joshua D Grill ◽  
Jason Karlawish
Keyword(s):  
Clinical Trials ◽  
Placebo Control ◽  
Controlled Trials ◽  
New Drug ◽  
Disease Modifying Therapy ◽  
Fda Approval ◽  
Background Therapy ◽  
Disease Modifying ◽  
Disease Modifying Treatment ◽  
Practical Implications

The goal of clinical research is to improve clinical practice. In progressive neurodegenerative conditions without any disease-slowing therapies, this will result in eventual approval of a first disease-modifying treatment. Clinical trials will still be needed to discover treatments that are more effective, safer, or more convenient. This will generate controversies over how to design these trials, specifically, controversies about the use of a placebo control. We consider ethical guidance for these studies with attention to three designs: placebo-controlled trials in the absence of the new drug, placebo-controlled trials with the approved drug as background therapy, and trials with the new drug as an active-control. To understand the practical implications of these designs, we examine experiences in drug development in Multiple Sclerosis (MS). We conclude by contemplating the future of clinical trials in Alzheimer’s disease (AD).

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