Lysosomal Storage Disease Treatment Opportunities

One of the dangerous genetic diseases is lysosomal storage disease (LSD) which is caused by the lack of the lysosomal enzyme. Lysosomes are responsible for complicated processes of protein, fat, and carbohydrate cleaving. When the enzyme is defective, some of the cells cannot function properly. As a result, the body tissues are highly affected, causing the degradation of the vital organs. The lysosomal storage disease causes the surplus of the metabolites in the cells, which damage the tissue and disturb the normal functioning of the organs. As far as this disease impedes normal cleaving, the metabolism fails to fulfill the synthesis appropriately. Thus, the lysosomal storage disease disrupts the metabolism process in the body’s tissues and cells.

The lysosomal storage disease is perilous due to its genetic path of transferring. As far as the prevention of this disease is practically impossible, scientists put a lot of effort into developing the ways of treatment or at least alleviating the symptoms regarding the destruction processes. Thus, Li (2018) considers enzyme replacement therapy as a possible way to treat the lysosomal storage disease. The scientist emphasizes that enzyme replacement cannot treat LSD entirely but “at least increase enzyme activity for a period of time” (Li, 2018, p. 192). The fundamental principle of this therapy is to provide the affected body with additional enzymes to replace the lack of lysosomes.

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The main problem with this treatment is that it is pretty challenging to direct the enzymes to the lysosomes. The author of the article emphasizes that there were various failures in therapy because of this problem (Li, 2018). However, nowadays, this therapy can be used as an efficient tool to lower the risks for the patient for a particular period. The “limited bioavailability in certain tissues” is another problem that can negatively affect the therapy’s outcomes (Li, 2018, p. 192). Particular neurological symptoms, such as blood-brain barrier and membranes modifications, prevent enzyme delivery to the damaged cells (Li, 2018). Therefore, when adjusted correctly, enzyme replacement therapy can reduce the destruction of the tissues and improve the general physical state of the patients diagnosed with LSD.

Another strategy to maintain the body’s normal functioning with LSD is supportive therapy, which includes medicaments prescriptions, surgery interventions, and other activities. These practices strive to eliminate the symptoms of the disease while the root cause is intact. Almeciga-Diaz et al. believe that substrate reduction therapy is the most efficient way of treatment LSD (2020). This type of therapy aims to reduce the storage metabolites by implementing the small molecules, which inhibit the synthesis of substrates (Almeciga-Diaz et al., 2020). The opportunity to treat LSD by transplanting the healthy cells to the patient is called hematopoietic stem cell transplantation (Almeciga-Diaz et al., 2020). The main problem here is the necessity to choose the appropriate donor with similar human leukocyte antigens. Moreover, this method increases the risk of rapid death due to the failure of transplantation.

The mentioned above resources included various methods of LSD treatment. However, medicine still does not have an instrument to cure LSD completely. One of the possible perspectives in treating this dangerous genetic disease is gene therapy. Genome editing is still in the stage of development but has promising outcomes in treating different genetic illnesses such as LSD. Moreover, thanks to genetic modifications, specialists can regulate the regular storage of the metabolites in the tissues before the child’s birth. All mentioned above ways of treating LSD can also be applied to improve the health outcomes for patients. However, the prognosis for such patients is adverse in most cases because all the treatment opportunities are costly and have significant risks.

References

Almeciga-Diaz, Cruz, J., C., Espejo-Mojica, A., Leal, A., Sanchez, O., Ramirez, C., Reyes, L. (2020). Lysosomal storage diseases: Current therapies and future alternatives. Journal of Molecular Medicine, 98, 931–946.

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Li, M. (2018). Enzyme replacement therapy: A review and its role in treating lysosomal storage diseases. Pediatric Annals, 47(5), 191–197.

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