Anti-inflammatory biologics for lysosomal diseases

Lysosomal storage diseases (LSDs) are a group of inherited metabolic disorders caused by defects in lysosomal homeostasis. There are over 60 LSDs, affecting 1 in 5000 people worldwide, and yet most LSDs are without treatment.

Oxford researchers have identified that anti-TNF biologics could provide a treatment that is applicable to many LSDs. The researchers identified novel mechanisms that lead to inflammation in lysosomal diseases, and have demonstrated proof-of-concept for using anti-TNF therapy in preclinical models for two such diseases – Sandhoff and Niemann-Pick type C diseases.

Rare but severe diseases

Lysosomal Storage Diseases (LSDs) are rare inherited disorders afflicting between 5,000 and 10,000 people worldwide. Over 60 diseases belong to the LSD group and they are challenging to diagnose and treat. There is currently no approved treatment for many LSDs.

LSDs affect many of the body’s systems but mainly attack the nervous system. They tend to present in the first few years of life and the rapid progression results in frequent hospitalisation. If left untreated, patients often die in their mid-teens. Therapeutic approaches for LSDs are limited and many of the available options only serve to improve a quality of life. In particular, some enzyme-replacement therapies are available, but they suffer from an inability to cross the blood-brain barrier and are therefore unable to address the central nervous system symptoms of the disorders.

Anti-TNF Therapy for LSDs

Anti-TNF therapy is one of a new class of biologic agents that target specific proteins in the immune system known to increase arthritis and other inflammatory diseases. Anti-TNF therapies have been used to treat many autoimmune diseases, including rheumatoid arthritis, juvenile rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis and Crohn’s disease.
Anti-TNF therapy for LSDs has the potential to reduce the side effects that come with treatments that block other important processes. Oxford researchers have identified novel mechanisms that lead to inflammation in LSDs, and have demonstrated proof-of-concept in preclinical models that anti-TNF therapy can be used to target this inflammation. Whilst the current studies focussed on Sandoff disease and Neimann-Pick type C disease, the therapy could potentially benefit patients with over 70 of the lysosomal diseases that trigger inflammation.
Oxford University Innovation has filed a patent application and is interested in speaking to parties about licensing this technology.

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