Computational Drug Repurposing for EBS



Status:Active, not recruiting
Conditions:Healthy Studies, Skin and Soft Tissue Infections, Skin and Soft Tissue Infections
Therapuetic Areas:Dermatology / Plastic Surgery, Other
Healthy:No
Age Range:Any
Updated:9/8/2018
Start Date:November 28, 2017
End Date:December 31, 2018

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Computational Drug Repurposing for Epidermolysis Bullosa Simplex

The study will compare gene expression differences between blistered and non-blistered skin
from individuals with EBS, as well as normal skin from non-EBS subjects. State of the art
computational analysis will be performed to help identify new drugs that might help EBS wound
healing and reduce pain. Researchers will focus on drugs that have already been approved for
treatment of other dermatologic or non-dermatologic diseases, and therefore be repurposed for
treatment of EBS. Drug development is a very expensive process taking decades for execution.
Drug repurposing on the other hand, significantly reduces the cost and shortens the amount of
time that is needed to bring effective treatments to clinical use. To date, there is no
specific treatment targeting the physiology and immunologic response in EB patients during
wound healing. Market availability of repurposed medications will provide EBS patients rapid
access to treatments, thus improving their quality of life.

Although gene, cell, and protein-based therapies are in development for patients suffering
from epidermolysis bullosa simplex (EBS), new pharmacological treatments are in dire need.
Characterizing molecular changes in EBS, including gene expression, can identify new
therapeutic targets and drugs that modulate those targets. However, sifting through gene
expression information to identify the most promising drug targets is a complex data
challenge. The goal of the study will identify a computational approach to evaluate and
identify existing drugs approved for other diseases that can be repurposed for EBS patients.
The study will perform an unprecedented characterization of gene expression changes in EBS
patients compared to healthy, non-EBS individuals across multiple tissues. Using a validated
computational drug discovery platform, researchers will analyze gene expression and drug data
using unique algorithms. In the first year, a list of ten, safety drugs more probable to
treat the EBS disease state will be identified. The most promising drugs discovered will then
be tested in the clinic setting.

Inclusion Criteria:

- Subjects of all ages

- Diagnosis of EBS subjects

- Healthy, non-EBS subjects

- Ability to complete study visit to collect tissue and blood specimen

Exclusion Criteria:

- Pregnancy, breast feeding

- Prior history of liver disease

- Serious known concurrent medical illness or infection, which could potentially present
a safety risk and/or prevent tissue collection from subjects
We found this trial at
1
site
Palo Alto, California 94304
Principal Investigator: Joyce M Teng, MD, PhD
Phone: 650-723-0636
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Palo Alto, CA
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