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Arrowhead Pharmaceuticals, Inc., a Pasadena, California, company, today announced ARO-DUX4 as Arrowhead’s first muscle targeted investigational RNAi therapeutic candidate. Using the company’s proprietary Targeted RNAi Molecule (TRIMTM) platform, ARO-DUX4 is designed to target the gene that encodes human double homeobox 4 (DUX4) protein as a potential treatment for patients with facioscapulohumeral muscular dystrophy (FSHD). Pending abstract acceptance, Arrowhead intends to present preclinical data on ARO-DUX4 at the 28th Annual FSHD Society International Research Congress being held virtually on June 24-25, 2021. Arrowhead is currently conducting IND/CTA enabling toxicology studies and intends to file for regulatory clearance in the third quarter of 2021 to begin clinical studies of ARO-DUX4.
“In our various animal models, ARO-DUX4 reduced DUX4 expression by greater than 70%, prevented body weight loss associated with tamoxifen-induced DUX4 expression, and prevented loss of muscle function,” said Chris Anzalone, Ph.D., president and chief executive officer at Arrowhead. (In these studies, tamoxifen, a drug used to treat tumors, is used as an artificial switch to turn on the DUX4 gene in mice that have been genetically engineered to do so. Switching on DUX4 in the mice leads to muscle degeneration and weakness, similar to that seen in the human disease. Normally, tamoxifen does not affect DUX4 expression, so individuals with FSHD should not be concerned that taking tamoxifen might trigger the progression of symptoms.)
Me interesa ya que padezco FDH,al igual que mis hermanos.
This sounds like the most targeted potential treatment we’ve seen announced.
This sounds very promising!! So as a person that has FSHD how do we get involved in any clinical trials, etc. Please provide any such contact information!!! Thank You!!!!
As potential affected, I’d like to take part of this clinical trials or wherever…
Congratulations for your job, our solution smells it’s near…
I would be interested in pursuing this for my adult son with FSHD. What are the next steps? He does not live on the west coast, so geography may be an issue.
Please make sure your son is signed up too, because clinical trial alerts are usually targeted to the geographic region where a prospective volunteer is living. Here’s the link: https://www.fshdsociety.org/for-patients-families/join-fshd-research-contact-registry/
How does one get involved with these trials
Clinical trials are typically restricted to individuals who meet the inclusion criteria for the trial and are in the geographic area of a trial site, or who are willing to commit to traveling to participate in the trial. To make sure you are notified about opportunities to volunteer for a trial, be sure to join our Research Contact Registry:
https://www.fshdsociety.org/for-patients-families/join-fshd-research-contact-registry/
This is really interesting, but I’m sorry to say I got confused. Are the mouse given tamoxifen only to simulate or provoke symptoms similar to FSHD in humans? And then does this mean that people with FSHD would not need to take tamoxifen when given the TRIM therapeutic? Thanks for any clarification!
Let me suggest the last sentence be rewritten as “Normally, tamoxifen does not affect DUX4 expression, so individuals with FSHD who take tamoxifen should not be concerned it might trigger the progression of symptoms.” Without revision, the sentence seems to imply tamoxifen is given as part of treatment.