Clinical translation of EB research
EB is rare, and some subtypes are classified as very rare. Recognition of a condition as rare is compensated by financial and regulatory schemes to encourage investment and facilitate approval and market entry. However, the rarity of EB poses challenges to the development of treatments, as does the severity of some EB subtypes in patient recruitment to clinical trials.
Developing therapies from successful research is expensive: clinical trials are at least 10 times more expensive than laboratory research. EB-ResNet members need partners from industry to help develop therapies – not only to help fund the research and clinical trials but also for their expertise in translating research into clinical treatments. However, extensive investment over 40 years by EB-ResNet members has significantly de-risked development for industry partners.
Main challenges for EB patient organisations
- Selecting which research to develop: with competing technologies and finite financial resources, EB-ResNet is likely to have to choose to develop one type of therapy over another.
- Feasibility: in practice, therapies selected for development are likely to be those where there is the opportunity to create a safe, effective therapy that is affordable, for any type of EB. Consideration of issues such as market size, practicality of delivery and affordability under various reimbursement schemes have to be taken into account.
- Financial: to date, EB-ResNet members have funded research up to the point of early-stage clinical trials (phase I/ 2); further development requires much greater funding, and this will require working in partnership with others, notably industry.
- Expertise: EB-ResNet will benefit not only from increased financial resources through partnership, but also the advice of individuals and companies with experience and expertise in taking research through clinical trials, and regulatory approval, into the clinic.
EB-ResNet seeks industry and academic partners to overcome these challenges. Contact email@example.com if you want to partner.
What would a possible cure look like?
EB is a rare condition, but presents opportunities for companies with interests in traumatic, burn and congenital-defect wound repair or small molecule drugs to modify the skin microenvironment and the course of the disease, or the chronic inflammation which predisposes the skin to aggressive cancer.
The nature and severity of symptoms and the prognoses, vary widely across the main EB types and subtypes. The important cells for therapy development are the keratinocytes in the epidermis and/or the fibroblasts in the dermis – these are the cells that make the proteins that hold the layers of skin together and are faulty in EB.