When will this trial begin?
This trial has started! We have grafted our first four subjects and are looking for additional subjects.
There is a two-step process required to enter the gene transfer trial. We will first need to see if you meet the criteria for the study, described above. We do this by having you participate in our “Characteristics” study.
The Characteristics study will involve a trip to Stanford, in which we will perform blood tests, skin biopsies, and genetic testing. We will pay for the laboratory expenses and reasonable travel expenses related to this visit. You can contact our research coordinator to find out more about study enrollment. Melissa Barriga at (650) 850-1674 or firstname.lastname@example.org.
Who will be the first subjects in the trial?
The first four subjects were 18 years old or older, but now patients must be now at least 13 years or older, have RDEB, and be healthy enough to come to Stanford University for evaluation. We have additional requirements, which we will find out from results of the Characteristics study. A list of criteria for the gene transfer trial can be found at the top of this page.
After a subject is invited to enroll in the gene transfer study, we may need to perform additional tests.
If any other enrollment criteria change, we will update this website.
Are there any risks with a gene transfer trial?
We are concerned about many risks. In one gene transfer trial for children with a lethal blood disease, several of the children developed leukemia. In a previous trial for a metabolic disease, one child died. In some of the trials of gene therapy for cancer some of the subjects have shown benefit and some have not benefited at all.
We also want to make sure that grafted subjects do not have an immune reaction. Our enrollment criteria has been designed to minimize this risk.
It is important to realize that a Phase 1 trial is designed to protect the subject while looking for any risks or illnesses that may develop.
RDEB affects infants and children. When can we try to help them?
Since we were able to show some benefit in four adults with small risks, we can now enroll adolescents 13 years and older. We hope to continually lower the inclusion age as we demonstrate benefit.
Are we working on any other EB treatments?
We recently gained FDA approval for a Phase 2 study of an anti-itching drug.
We are participating as a site in Scioderm’s study of SD-101 cream. (http://www.sderm.com/)
We are still examining the potential of protein therapy for EB. Protein therapy involves introducing type VII collagen directly to a participant’s skin.
In addition we continue to examine the relationship between RDEB and squamous cell carcinoma.
Is there someone I can speak to in order to understand more about EB, gene transfer, and cancer?
As in the case of therapy efforts, this web site is maintained to provide the most current public information on this subject related to the research effort ongoing at Stanford University. This website was designed to both inform the community and to prevent staff from being diverted from research efforts to repeat the same answers to similar questions arising from EB patient families around the world. In the event that further information becomes available, this site will be updated to reflect this new information. Also your dermatologist who cares for you or your relative may be able to explain the details of this information to you.