NBD Peptide - Using NF-ĸB blockers to Decrease Inflammation and Improve Muscle Function in Duchenne

 

Frequently Asked Questions (FAQs) about this research:

 

What stage is this research?

  • This research in Duchenne is pre-clinical, meaning it has not advanced to clinical trials involving people with Duchenne yet. 

 

Where is this research being done and who is funding this research?

  • This research is being done in Dr. Denis Guttridge’s laboratory at The Ohio State University in collaboration with colleagues at The Ohio State University and Nationwide Children's Hospital.  The work has been funded by the NINDS branch of the NIH.

 

What is the goal or purpose of this research?

  • The NF-ĸB pathway has been shown to be involved in promoting inflammation and compromising muscle function in response to the loss of dystrophin in Duchenne.  Dr. Guttridge’s group has used a small Glossary Link molecule called “NBD” to specifically block this pathway.  In mice that lack dystrophin, NBD significantly improves the function of breathing muscles and allowed the mice to maintain whole body function.  In addition, skeletal muscle limb function was also improved when NBD was administered to dogs lacking distrophin. Furthermore, in mice that lack dystrophin and utrophin, the drug significantly improved cardiac function.  

 

What is the current state of this research and what steps need to be completed before moving into a clinical trial?

  • Discussions have taken place with the FDA in order to submit a pre-Investigational New Drug application
  • Formal Non-GLP toxicology testing have been performed. GLP pharmacology toxicology studies are planned.

 

What is your best estimate for the length of time it will take to move this research into clinical trials?

  • Preparations are in place to complete the final studies leading to an IND submission.

 

Where would a clinical trial take place?

  • The phase I safety trial is planned for Nationwide Children’s Hospital, Columbus Ohio, directed by Dr. Jerry Mendell. For Phase II, it is too early to know where a clinical trial will take place and much will depend on the safety profile determined in Phase I.

 

Who would be eligible to participate in a clinical trial?

  • Beyond the Phase I safety study, it is too early to know what the inclusion criteria would be for a future clinical trial.

 

Where can I learn more about this research?

  • You can learn more about Dr. Guttridge’s research at The Ohio State University website (https://bme.osu.edu/).
  • www.ClinicalTrials.gov will post all clinical trials once they are actively recruiting patients.
  • Please check www.DuchenneConnect.org for updates to this FAQ sheet.                                                               

 

(Updated 4/24/2017)