Neurodegenerative disorders affect the structure and function of the brain and spinal cord.

The majority of these diseases are irreversible because the central nervous system doesn’t dispose of any capacity to restore damaged tissues. Current lack of effective treatment results in deterioration in quality of life, significant morbidity, and disability to participate and engage in daily life activities.

Amyotrophic lateral sclerosis – ALS is a late-onset neurodegenerative disease with preponderance in males and increasing incidence worldwide. This disease is characterized by the degeneration of both upper and lower motor neurons. Upper motor neurons in the brain send messages to lower motor neurons in the spinal cord and brainstem. The progressive upper and lower motor neuron degeneration lead- to signaling disruption, muscle weakness and physical disability. Amyotrophic lateral sclerosis results in loss of ability to initiate and control voluntary movement, affecting the muscles needed to breathe, swallow, and move arms and legs. Disease progression leads to symptom worsening which results in loss of coordination, decreased physical capacity and autonomy. Improving the quality of life requires a multidisciplinary approach to treatment and daily maintenance care.

Stem cells are being investigated as a potential therapeutic approach for central nervous system disorders. The majority of clinical trials remain in the early stages of development, attempting to establish proofofefficacy and hope in replacing damaged cells and tissues. These cells are a foundational buildingblock of regenerative medicine and tissue engineering. Stem cells therapy is a promising new approach to the treatment of amyotrophic lateral sclerosis by addressing the complex pathogenetic etiology through multiple mechanisms. This therapy will provide effective therapeutic potential due to its immune-modulating effects and production of growth factors that protect, support and repair neurons.

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The innovative stem cell-based therapy is a potential therapeutic approach that will provide immune modulation effects, neurotrophic properties, and capacity for neural tissue regeneration. Stem cells offer an attractive avenue to treat neurological disorders, including Parkinson’s disease and amyotrophic lateral sclerosis, where the leading cause of disability is due to localized degeneration of neurons. This novel approach represents a potential paradigm shift in neurodegenerative disease treatment as it provides remarkable regeneration of neural tissue, stabilization of neuronal networks and restoration of cognitive and functional capabilities. Stem cell-based therapy will contribute to neurodegeneration reversal which will offer multiple benefits to the healthcare system and clinical practice.

How will this approach help the patients?

Current therapies for amyotrophic lateral sclerosis are not providing the necessary efficacy and therapeutic potential. Effective care requires a multidisciplinary approach and extensive screening. Moreover, amyotrophic lateral sclerosis leads to significant quality of life and cognitive capacity depreciation, as well as physical disability and dependence on healthcare. Providing effective therapy requires a complex approach that incurs high healthcare resource utilization. The innovative stem cells-based therapy promises extended lifespans, restoration of cognitive capabilities, emotional well-being, and quality of social interactions. The high therapeutic potential of this novel approach is being investigated by numerous clinical trials, as it offers possibilities for tissue regeneration due to its immune modulation effects and multi-directional differentiation. Neurodegeneration reversal will provide improved treatment adherence and reduced burden and expenditure on healthcare. This novel approach will contribute to financial independence and enhanced productivity which will have a profound effect on the long-term economic contribution to society. The reduced dependency of patients on healthcare will improve caregiver quality of life and allocation of healthcare resources.

How many patients will be concerned?

The number of patients who have been diagnosed with amyotrophic lateral sclerosis in the European Union exceeds 32 thousand. Although this disease is predominantly idiopathic, some cases are due to genetic predisposition and various gene mutations, leading to disease development. There is an increasing reported incidence worldwide. The deteriorated quality of life requires raised expenditure on healthcare and daily care to withstand physical disability. The regeneration of damaged tissues in the brain will offer an enduring restoration of cognitive functions, as well as autonomy and better quality of relationships.

What will be the potential impact on healthcare systems in Europe?

This novel therapeutic approach will decrease the healthcare burden and healthcare system visits following sustained neural tissue regeneration. There is a potential for reduced expenditure on symptomatic therapy and medication for co-morbidities. The re-establishing of functional capabilities and autonomy will provide improved productivity and economic contribution to society. The alleviated socioeconomic burden and reduced healthcare utilization will lead to better working conditions for caregivers and effective allocation of healthcare resources.

DISCLAIMER

The therapies described above are currently under development and are not available to patients neither in Europe, nor in Bulgaria.