Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative condition. The early signs of ALS include loss of upper and lower motor neurons, culminating in aggravated muscle weakness. ALS invariably results in paralysis, and respiratory failures, ultimately leading to death. Presently, there is no cure for ALS and novel treatments are required to slow down the progression of this deadly disease.

Stem cell therapy is acknowledged as one of the most remarkable and latest treatments for MND. Various pathogenic pathways could be aimed with this therapy to slow disease progression. Stem cell therapy has the ability to provide immunomodulatory support, potentially allowing for motor neuron regeneration. Many preclinical models have supported the use of stem cell therapy to control the progression of ALS.

The core idea behind using stem cells to treat ALS is to use them to regenerate motor neurons that had been lost as the disease progressed. Furthermore, transplanted cells act as a support system by secreting neurotrophic factors and boosting the motor microenvironment of neurons, slowing disease progression.

Lately, many studies have shown positive effects of stem cells in the treatment of ALS. Although the administration of SCs into the CNS presents numerous challenges. Multiple injections are required to deliver therapeutic doses to target upper motor neurons, lower motor neurons, or the spinal cord, which increases the risk of adverse events. A prudent risk-to-benefit review is necessary, especially for interventions addressing the brain. Furthermore, selecting the proper injection route is essential. Many studies have found SC intrathecal injections to be safe and effective.

In a study conducted by Ammar et al. (2021), 6 clinical trials were analyzed for their efficacy in ALS patients. Stem cell therapy for ALS had a beneficial impact on slowing the progression of the disease in all of the studies that were conducted. Out of which 3 studies that used bone marrow-derived mesenchymal stem cells (BM-MSC) found a significant reduction in disease progression. The results of these studies have shown that stem cell therapy, notably, intrathecal administration of MSCs indicated a benefit in preventing the development of ALS/MND.

Early clinical trials have made significant strides in determining the safety of SC therapy in the treatment of ALS. Articulating the treatment efficacy is absolutely essential, which includes determining who will benefit the most from such therapy and whether it is only available in the early stages of the disease. Due to the condition’s heterogeneity, future studies should subdivide their patients into multiple disease variants based on their assessment. The latest evidence on stem cell therapy for ALS shows great promise, but larger, better-designed clinical trials are required to truly validate its benefit.

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