In the recent EB Research Partnership Town Hall, we heard powerful testimonies and updates on the fight against epidermolysis bullosa (EB) and the work being done to find a cure.
Humphrey Hanley, Vice President of DEBRA International, shared his personal experiences living with EB, particularly the challenges of squamous cell carcinoma (SCC). His resilience in the face of frequent surgeries and health complications was inspiring, as was his advocacy for EB patients worldwide.
"It's almost a monthly occurrence that something needs an excision… I guess I call this the price of admission to the show known as life. And honestly, given the alternative, I think it’s a price that I’m more than willing to pay for every day I get to spend with the little cheeky monkey that is my daughter."
Humphrey emphasized the importance of having a strong community who understands what you are going through and offers support. He also empowers EB patients to have a voice in their treatment and care, urging the medical community to listen closely to those living with the disease.
"Nothing about us without us. Most of us with EB are too busy fighting to survive to have energy left to fight for our voices to be heard as well."
Dr. Andrew South, from Thomas Jefferson University, gave an in-depth presentation on his groundbreaking research focused on squamous cell carcinoma (SCC), a common complication of EB. His team has been studying the efficacy of Rigosertib, an experimental drug, in treating EB-related SCC. Early trials have shown promising results, with some patients experiencing significant reduction in their tumor cells. Dr. South highlighted that Rigosertib targets a molecule called PLK1, which plays a critical role in cell division. Inhibition of PLK1 in cancer cells leads to their death, offering hope for a new treatment approach. While some patients responded well to the treatment, Dr. South noted that others with more advanced cancers had less favorable outcomes, emphasizing the importance of early detection and intervention.
"We are incredibly grateful to the community that joins clinical trials, donates skin and material, and supports research foundations like EBRP. This progress would not be possible without you."
EBRP is honored to have funded Dr. South and his team at Thomas Jefferson for various projects, under our Venture Philanthropy model, totaling over $1.7M, for many years now.
Rigosertib is currently in clinical trial early phase 1 and is being offered to patients who have run out of other SCC treatment options. If you would like to get in touch with Dr. South and his team at Thomas Jefferson, please email [email protected] and we will put you in touch.
For more information on the trial, see below.
Rigosertib is currently in clinical trial early phase 1 and is being offered to patients who have run out of other SCC treatment options. If you would like to get in touch with Dr. South and his team at Thomas Jefferson, please email [email protected] and we will put you in touch.
For more information on the trial, see below.
