Paper Details

  1. Home
  2. Paper Details

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

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

Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
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.
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.

Overview

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.
Paper Details　
Original Abstract of the Article :
Misfolded aggregated proteins and neuroinflammation significantly contribute to amyotrophic lateral sclerosis (ALS) pathogenesis, hence representing therapeutic targets to modify disease expression. Rapamycin inhibits mechanistic target of Rapamycin (mTOR) pathway and enhances autophagy with demonst...See full text at original site
Dr.Camel IconDr.Camel's Paper Summary Blogラクダ博士について

ラクダ博士は、Health Journal が論文の内容を分かりやすく解説するために作成した架空のキャラクターです。
難解な医学論文を、専門知識のない方にも理解しやすいように、噛み砕いて説明することを目指しています。

* ラクダ博士による解説は、あくまで論文の要点をまとめたものであり、原論文の完全な代替となるものではありません。詳細な内容については、必ず原論文をご参照ください。
* ラクダ博士は架空のキャラクターであり、実際の医学研究者や医療従事者とは一切関係がありません。
* 解説の内容は Health Journal が独自に解釈・作成したものであり、原論文の著者または出版社の見解を反映するものではありません。


引用元：
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6024184/

データ提供：米国国立医学図書館（NLM）

Rapamycin: A Potential Oasis for Amyotrophic Lateral Sclerosis

[Amyotrophic lateral sclerosis (ALS)] is a devastating neurodegenerative disease. This study outlines a [phase II randomized clinical trial] to investigate the [potential benefits of rapamycin] in [ALS patients]. The researchers aim to determine if [rapamycin, a drug that inhibits the mTOR pathway and enhances autophagy], can [modify the disease expression] and [improve clinical outcomes] in patients with [ALS].

A New Path Through the Desert: Exploring Rapamycin's Potential

This study highlights the [rationale for testing rapamycin] in [ALS]. The researchers cite [preclinical studies] that demonstrate [rapamycin's positive effects] on [neurodegeneration] in [cell line and animal models]. They also highlight the [potential of rapamycin] to [modulate immune responses] and [reduce inflammation], which are key factors in [ALS pathogenesis].

Finding Hope in the Desert: A Clinical Trial for ALS

This study represents a [hopeful step forward] in the [search for effective treatments] for [ALS]. The researchers' commitment to [conducting a rigorous clinical trial] to evaluate [rapamycin's efficacy] is a testament to the [ongoing efforts] to [find new therapies] for this debilitating disease.

Dr.Camel's Conclusion

This study outlines a [promising clinical trial] to investigate the [potential benefits of rapamycin] in [ALS]. It's like a camel seeking a hidden oasis in a vast desert, driven by the hope of finding a source of relief and healing. The findings of this trial could have a significant impact on the [treatment and management] of [ALS], offering new possibilities for [improving the lives] of individuals living with this challenging condition.
Date :
  1. Date Completed 2018-06-21
  2. Date Revised 2022-04-19
Further Info :

Pubmed ID

29901635

DOI: Digital Object Identifier

PMC6024184

SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

This site uses cookies. Visit our privacy policy page or click the link in any footer for more information and to change your preferences.