Modeling and Bioinformatics Identify Responders to G-CSF in Patients With Amyotrophic Lateral Sclerosis

Frontiers in Neurology, 2021 · DOI: 10.3389/fneur.2021.616289 · Published: March 18, 2021

Simple Explanation

This study explores the use of filgrastim, a drug commonly used to stimulate stem cell production, as a potential treatment for ALS. Researchers compared the outcomes of ALS patients treated with filgrastim to a large database of ALS patient data (PRO-ACT) to assess the drug's effectiveness. The study used advanced data analysis techniques to identify which patients responded best to filgrastim and to identify biomarkers that could predict treatment response early on.

Study Duration

Variable, up to 78.8 months

Participants

36 ALS patients

Evidence Level

Not specified

Key Findings

1
Filgrastim-treated patients showed a significant survival benefit compared to the PRO-ACT database.
2
A model was developed to predict survival, and patients who responded to filgrastim based on this model showed less functional decline and longer survival.
3
Specific biomarkers, including younger age, increased stem cell mobilization, less aggressive inflammatory cytokine profiles, and preserved brain structure, were associated with a strong treatment response.

Research Summary

This study investigated the potential of long-term filgrastim treatment in a small cohort of ALS patients, using the PRO-ACT database as a historical control. The results suggest that filgrastim is safe and well-tolerated, with positive effects on disease progression and survival in some ALS patients. The study identified biomarkers that can help predict which patients are most likely to respond to filgrastim, paving the way for individualized treatment approaches.

Practical Implications

Personalized Treatment

Biomarker-based response classification allows for the identification of strong individual treatment responders at an early stage of therapy.

Drug Repurposing

Filgrastim, a well-established drug with a good safety profile, may be a viable therapeutic option for ALS.

Future Research

Further research is warranted to confirm these findings in larger, randomized controlled trials and to further explore the mechanisms of action of filgrastim in ALS.

Modeling and Bioinformatics Identify Responders to G-CSF in Patients With Amyotrophic Lateral Sclerosis

Simple Explanation

Key Findings

Research Summary

Practical Implications

Personalized Treatment

Drug Repurposing

Future Research

Study Limitations

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Was this summary helpful?

Modeling and Bioinformatics Identify Responders to G-CSF in Patients With Amyotrophic Lateral Sclerosis

Simple Explanation

Key Findings

Research Summary

Practical Implications

Personalized Treatment

Drug Repurposing

Future Research

Study Limitations

Your Feedback

Was this summary helpful?