Lysosomal Storage Diseases Therapeutics’ Demand Growing With Increasing Prevalence Of Lysosomal Storage Diseases

 Lysosomal storage diseases are inherited metabolic diseases caused by faulty activity of a number of enzymes in the lysosome. Patients with these diseases are likely to experience an array of symptoms, which may range from fatigue to loss of appetite. Clinical symptoms are dependent on the type of accumulated substrate, and the time it takes to develop symptoms is dependent on the disease. More than 40 types of lysosomal storage diseases have been identified by scientists. Each one affects different organs and tissues. Patients with Gaucher disease have enlargement of the spleen, problems with blood and bone development, and other symptoms that can be mild or severe. Type 1 Gaucher disease affects the central nervous system and can cause neurological symptoms.

Research on the development of treatments for Lysosomal Storage Diseases Therapeutics has yielded impressive results in the last two decades. Still, there are no treatments for almost two-thirds of lysosomal diseases. Despite their impact on the central nervous system, the disease is costly, limiting drug development. Further, the lysosomal storage diseases therapeutics remain expensive and have high development costs. The scientific community has identified a few genes associated with these disorders. According to the Rare Disease Database, these disorders affect approximately one in every 5,000 live births. Though they affect people from all over the world, Gaucher and Tay-Sachs are most common among Ashkenazi Jewish and Scandinavian populations. Other genetically related diseases that affect the lysosomal storage system include Hurler syndrome and Tay-Sachs disease.

Lysosomal Storage Diseases Therapeutics

Enzyme replacement therapy is another lysosomal storage diseases therapeutics option for patients suffering from lysosomal storage disease. In this therapy, a normal gene is substituted for the defective one, allowing the diseased body to produce the active enzyme. This therapy reduces toxic storage material and improves organ function. There are several benefits of enzyme replacement therapy, which are detailed below. These therapies have fewer side effects and are associated with a lower risk of complications. Currently, the treatment options for these diseases are not effective. The current standard of care for children suffering from MPS IH is bone marrow transplantation. However, bone marrow transplantation is less effective in correcting or preventing bone complications. This procedure involves the replacement of the entire blood system of the affected person. Further, it is also being investigated for other lysosomal storage diseases.

A new treatment for these diseases is awaited. Lysosomal Storage Disease Therapeutics are a growing area of pharmaceutical development. There are several promising treatments on the market. Clinical trials are ongoing and progressing at an impressive rate. A number of pharmaceutical companies are currently developing new drugs to treat these conditions. And as the field becomes more complex, more therapies will be developed. However, there is no single treatment for storage disease.

Currently, enzyme replacement therapy is the gold standard for treating some LSDs. However, enzyme replacement therapy is limited in its effectiveness as it cannot cross the blood-brain barrier. Furthermore, the enzyme infused does not match the native enzyme and may trigger an antibody response. The resulting antibodies reduce the efficacy of the therapy. And despite the advances in therapeutics, further research is necessary to understand the mechanisms behind these diseases.