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Small molecule drugs to relieve EB-related symptoms

New scientific article from the EB House

While unfortunately no causal therapy is available to date to cure EB, treatment approaches using symptom-relieving drugs give promising perspectives to prevent or treat EB-related complications and increase the quality of life of the patients. Most of these drugs use small molecules, which are agents that can enter cells easily because of their small size. Once inside the cells, the drug can interact with other molecules such as proteins, which may reverse pathogenic processes or even cause cancer cells to die. In recent years, it has been found that chronic skin damage in EB leads to deregulated inflammatory reactions, which in turn contribute to severe EB symptoms. This opens new avenues for the use of drugs that modulate immune responses or decrease infections, which positively impact EB-related symptoms.

Symptom-relieving and disease-modifying drugs applied in EB are either specifically developed for a certain EB type, or use compounds that were initially approved for other diseases and have been repurposed for the treatment of EB. Drug repurposing for EB has been examined since many decades. It represents a much more cost-effective and rapid route to clinical application over a new compound, since the safety and interaction studies have already been done. This allows EB patients to benefit faster from a treatment. On the other hand, currently developed small molecule drugs directly interact with a defined EB-related cellular target.

EB House researchers published a review article on the current clinical development of small molecule-based drugs, including a summary of ongoing clinical studies in EB. Searching in online databases, they identified a total of 84 published studies where small molecule drugs were used to treat EB symptoms, predominantly in EBS and RDEB patients. 32 currently registered clinical trials investigate small molecules for their safety and efficacy for reducing disease manifestations, most frequently wounding, blister formation, itch and pain, and prevention of skin cancer. Another focus of these trials is to increase the safety of small molecules-based treatment approaches. While most of the earlier studies used oral administration, in currently performed studies a local application is preferred in order to avoid possible systemic side effects. It is expected that some of these tested repurposed or new drugs may receive market approval in the near future. The access to different symptom-alleviating drugs may result in improved treatment outcomes for individuals with EB.

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