Why Reinvent the Wheel?
Drug Repurposing is considerably faster and cheaper than normal drug discovery.
With a $44,000 grant from FRAXA, awarded in September, 2016, University of Cambridge (England) startup Healx is taking this approach to identify novel therapies based on existing drugs to help those with fragile X syndrome.
“We use genomic information to help identify which drug will be best suited to counteract the effects of the fragile X condition,” said Narissa Gipp, Head of Patient Engagement, Healx. “Literature mining techniques are used to search through the entire scientific literature to identify additional information that can help match existing drugs to rare diseases.”
Sophisticated drug-matching technologies, which originate from the University of Cambridge, are used to identify hidden links between existing drugs and rare diseases. The most promising drugs are then tested experimentally in fragile X models. In case an approved drug is indeed safe, a pivotal clinical trial can be developed in a small group of patients to assess efficacy.
Given a successful result, doctors could then consider “off-label” prescription of the medicine to improve quality of life for fragile X patients, while the final stages of clinical development are being conducted.
“Overall, drug repurposing is a quick and cheap way of delivering potential treatments,” Gipp said.
Healx’s three-phase analysis process includes dataset searches, incorporating gene expression quality checks, and candidate compound list derivation.
“Application of novel drug-matching technologies can significantly help accelerate this process,” Gipp said. “The generation of excellent gene expression data is important. It helps understand the effects in detail and therefore increase the likelihood of performing successful drug-matching.”
Gipp said working with FRAXA is “fantastic.”
“FRAXA is incredibly well organized and established,” she said. “Its program of research and drug discovery can very effectively help progress successful drug candidates to the next stages of development. The resulting findings will also greatly help progress other rare diseases, such as Pitt Hopkins syndrome among others.
“We are incredibly excited about working together with FRAXA to help identify new effective treatments.”
Attention, Scientists: Request for Transcriptomic Data
FRAXA awarded $44,000 to Healx for the project, Identifying Novel Therapies based on Existing Drugs for Fragile X Syndrome. This drug repurposing project aims to use advanced data analytics and machine learning to predict which approved drugs could have a therapeutic effect on Fragile X patients. As part of this study the investigators are using transcriptomic data to facilitate the drug/disease matching process. Data has been obtained through open sources, however the more data we can add to this study the greater likelihood we have of identifying a new potential treatment.
In particular we are looking for transcriptomic data and good case controls in the condition. If you have data available and are interested in joining this project don’t hesitate to get in touch.
This additional data will help us to identify potential drugs which can be repurposed for the treatment of Fragile X Syndrome.
Head of Patient Engagement