Healthcare systems around the world struggle to manage the socio-economic burden posed by neurodegenerative diseases. Huntington’s Disease uniquely disrupts patients’ voluntary and involuntary control of movement, cognitive faculties, and even their emotional state. Those who suffer from this genetic disease experience a rapid deterioration of the quality of their lives and of their finances. As medium spiny neurons continue to die and shrivel in the striatum, brain atrophy becomes a permanent condition beyond treatment. The pace of research into neurodegeneration today seeks to understand and develop therapy to slow down this rapid cell death. In the race to find potential candidates for neuroprotective therapies, one Stem Cell type from the regenerative branch of cellular therapy has emerged as an excellent candidate. Recent research focuses on UC-MSCs as a promising clinical candidate with the potential to restore neurological function.
Huntington’s Disease mechanism
An analysis of the aberrations in Huntington’s Disease at the molecular level reveals a great deal about the disease’s pathology. The mutated huntingtin protein’s aberrations exemplify a highly defective and toxic protein that accumulates in fragile neurons. These aberrations also demonstrate cellular impairment, the dysfunction of mitochondria, and the death of the neuron by the process of apoptosis. The formation of toxic aggregates within the cell disrupts the process of transcription that is crucial to supplying the neuron with neurotrophic factors essential for the survival of the neuron. There is no other means to counter the destruction of the neuron’s ecosystem apart from introducing an ample supply of a robust Stem Cell population. The majority of such populations, particularly UC-MSCs, demonstrate therapeutic effects by exhibiting dynamic paracrine signaling. They accomplish this by establishing a microenvironment that is rich in neurotrophic factors, extracellular vesicles, and modulators of the immune system. Stem Cells propagate a supportive and localized network of neurogenesis and enhance angiogenesis while also regulating neuroinflammation. Secreted target molecules from such Stem Cell populations assist in clearing protein aggregates.
Conventional Huntington’s Disease Treatments
The treatment options for Huntington’s Disease have centered largely around the management of symptoms. This is primarily due to the lack of options available to clinicians to assist their patients. Management options for other neurodegenerative diseases have been created to restore the balance of neurotransmitters. However, in the case of Huntington’s Disease, the drugs have been created to palliate symptoms. For the chorea movements exhibited by patients, clinicians may prescribe tetrabenazine. Antipsychotics may also be prescribed to assist with some of the more concerning behavioral symptoms. Even though symptom management and palliation is the goal of the prescribing physician, clinicians are focusing more and more in recent years on the consequences of prolonged pharmacological management. In more recent literature, issues such as the exacerbation of depression, Parkinson’s-like symptoms, and increased sedation have become more concerning. This has become the focus in the treatment of the disease, in the absence of curative options. The lack of research on other treatment options, like a Stem Cell approach utilizing UC-MSCs, and the lack of creative treatment strategies has left clinicians with a massive therapeutic gap.
Rationale for Using UC-MSCs
The critical inadequacies of current methods for daily symptomatic treatment create an impetus for researchers to find better cellular options. The use of UC-MSCs provides significant and unprecedented advantages when compared to other non-specific therapies for Huntington’s Disease. These cells have unique properties of intrinsic immune system modulation, allowing them to avoid strong immune rejections from the host and the use of strong supportive immunosuppressive therapies. This unique property of UC-MSCs makes them ideal for allogenic use on a wide scale. The use of UC-MSCs leads to the resolution of the hostile inflammatory environment, which is of a great concern in the case of the transiting degeneration of the striatum. When UC-MSCs are carefully transplanted, this Stem Cell is able to migrate towards the site of severe neural injury and remain there permanently, releasing protective factors for the survival of the highly endangered medium spiny neurons. These Stem Cells are also able to donate healthy, functioning mitochondria to neurons that are in the need of cellular energy.
Future Trends in Thailand
Southeast Asia is witnessing a fast regional uptake of biotherapeutics. The broader deployment of clinically available UC-MSCs in Thailand will represent a major advance in the field of specialized neurological care for Huntington’s Disease in that country. Thailand has a well-developed and sophisticated medical system that is complemented by a well-structured and supportive national regulatory system for clinical trials. Major research institutions in the emerging science corridors of Pathum Thani have the extensive knowledge and expertise in modern biotechnology, and are in the process of developing highly pure Stem Cell lines, maintained under optimal laboratory conditions. The combination of the sophisticated services available in the country and the stable and cost-affordable environments provides the country with the potential to be the world leader in developing novel therapeutic procedures for this serious and challenging neurological condition. The combination of its strategic location, advanced infrastructure, and affordable healthcare further positions Thailand as an emerging leader in global medical tourism.
Figure 1: Biotherapeutics and Regional innovation in the Future trends
Conclusion
The relentless and severe progression of Huntington’s Disease suggests that a big step away from simple symptom control and toward real structural repair of the central nervous system is imperative. Along this line, the focused application of a particular Stem Cell type offers a strong multidimensional biological defense against the cellular degeneration of this serious genetic disorder. Taking advantage of the strong neuroprotective, anti-inflammatory, and restorative regenerative potential of UC-MSCs, the medical community will be in a better position to design an effective permanent solution. Rapid positive changes in Thailand show that there is a future where there is much more hope* for patients with severe conditions.