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  4. Amyotrophic Lateral Sclerosis - Cell Based Therapy and Novel Therapeutic Development

Amyotrophic Lateral Sclerosis - Cell Based Therapy and Novel Therapeutic Development

Exp Neurobiol, 2014 · DOI: http://dx.doi.org/10.5607/en.2014.23.3.207 · Published: September 1, 2014

Regenerative MedicineNeurology

Simple Explanation

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the loss of motor neurons, leading to premature death. Current treatments offer minimal delay in disease progression, prompting the search for novel therapies. Cell-based therapy, particularly stem cell transplantation, has emerged as a potential regenerative approach for ALS by replacing damaged motor neurons. The advent of induced pluripotent stem cell (iPSC) technology offers possibilities for patient-specific disease modeling and autologous cell transplantation, broadening therapeutic options for ALS.

Study Duration
Not specified
Participants
ALS patients
Evidence Level
Review Article

Key Findings

  • 1
    Mesenchymal stem cell (MSC) transplantation has shown minor improvements in ALS patients, with some studies suggesting that pre-selecting MSCs based on biomarkers could optimize treatment outcomes.
  • 2
    Neural stem cell transplantation, particularly using human fetal spinal cord-derived neural stem cells (HSSC), has demonstrated potential for replacing dying motor neurons in ALS.
  • 3
    Induced pluripotent stem cell (iPSC) technology enables the generation of patient-derived iPSCs for disease modeling and drug screening, offering personalized therapeutic approaches for ALS.

Research Summary

The review paper discusses recent accomplishments in cell transplant treatment for ALS and future options with iPSC technology. The paper highlights the potential of cell-based therapies, including MSC and neural stem cell transplants, in treating ALS and improving patient outcomes. iPSC technology offers a promising avenue for personalized ALS treatment through disease modeling, drug screening, and autologous cell transplantation.

Practical Implications

Personalized Medicine

iPSC technology enables the creation of patient-specific disease models for drug screening and personalized therapeutic regimens.

Regenerative Therapy

Stem cell transplantation, particularly with neural stem cells, holds potential for replacing damaged motor neurons and restoring function in ALS patients.

Drug Discovery

iPSC-based drug screening can identify novel therapeutic targets and compounds for treating ALS by focusing on individual patient conditions.

Study Limitations

  • 1
    Limited efficacy of current cell transplant therapies for ALS.
  • 2
    Challenges in achieving successful differentiation and integration of transplanted stem cells into the spinal cord.
  • 3
    Potential for immune rejection and tumorigenesis with non-autologous stem cell transplants.

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