FEB 24, 2019 7:30 PM PST

Possible Therapeutic for Duchenne Muscular Dystrophy (DMD)

WRITTEN BY: Nouran Amin

Individuals diagnosed with Duchenne muscular dystrophy (DMD) face few treatment options. DMD is a progressive muscle weakness and degeneration with loss of contractibility caused by one of several mutations in the DMD gene that leads to the chronic activation of a transcription factor called NF-κB—leading to the loss of the dystrophin protein in muscle cells.

DMD affects 3,500-6,000 male births and is the most common genetic neuromuscular disease. It is caused by one of several mutations in the DMD gene. No matter what the particular mutation, a key driver of muscle degeneration and suppression of muscle regeneration in DMD is chronic activation of the transcription factor NF-κB, which causes loss of dystrophin, a protein which helps keep muscle cells intact. Current therapeutics either assist only a subset of DMD patients who have a particular genetic mutation or cause significant side effects.

Learn more about Duchenne Muscular Dystrophy (DMD):

However, a recent investigational drug called ‘edasalonexent’, an oral NF-κB inhibitor, may hold the potential to slow the progression of the disease. The drug was recently examined to hold no safety issues during a Phase I clinical trial. Paving the way for more clinical testing, results of the study were recently published in the Journal of Neuromuscular Diseases.

"In addition to being well tolerated in pediatric patients with DMD, our Phase 1 data demonstrated that edasalonexent (CAT-1004) inhibited NF-κB. This is important because NF-κB is a key link between the loss of dystrophin and disease progression in DMD. This would mean that edasalonexent has the potential to limit disease progression for all patients affected by DMD, regardless of their underlying mutation," explains Joanne Donovan, MD, PhD, Chief Medical Officer of Catabasis Pharmaceuticals, Inc. (Cambridge, MA, USA).

The drug, edasalonexent, is orally administered and that contains two active substances, salicylic acid and the omega-3 fatty acid docosahexaenoic acid (DHA). These two substances separately are inhibitors of NF-kB, but together they provide a more potent inhibition. "This shows that with short-term dosing, edasalonexent can directly reduce the levels of elevated NF-κB in circulating DMD mononuclear cells prior to any changes observable in muscles," notes Dr. Donovan.

Source: Science Daily

About the Author
  • Nouran earned her BS and MS in Biology at IUPUI and currently shares her love of science by teaching. She enjoys writing on various topics as well including science & medicine, global health, and conservation biology. She hopes through her writing she can make science more engaging and communicable to the general public.
You May Also Like
APR 15, 2020
Chemistry & Physics
APR 15, 2020
Could this drug help people with SAD?
Have you ever been sad in the winter? If so, you’re not alone. Medaka fish feel it too. Seasonal affective disorde ...
APR 11, 2020
Drug Discovery & Development
APR 11, 2020
Promising Experimental Anti-Malarial Drug
At St. Jude Children’s Research Hospital, researchers discovered a fast-acting anti-malarial compound with promisi ...
APR 22, 2020
Drug Discovery & Development
APR 22, 2020
Using Colors To Track The Spread of Drug Particles
Forensic scientists from the National Institute of Standards and Technology (NIST) are relying on color to see the sprea ...
MAY 06, 2020
Drug Discovery & Development
MAY 06, 2020
Why Doctors are Repurposing Drugs to Treat COVID-19
Creating a drug from scratch is a lengthy process. Often taking years, given the rate of infection and deadliness of COV ...
MAY 14, 2020
Immunology
MAY 14, 2020
Antibody-laden Nanoparticles to Help COVID Patients Breathe Easy
Antibodies are a powerful countermeasure against COVID-19. Not only can they relieve symptoms in patients experiencing s ...
MAY 23, 2020
Cancer
MAY 23, 2020
A New Biomarker to Identify a Triple Negative Breast Cancer Prognosis
Breast cancer, one of the most common cancers in the world, are commonly separated into one of several sub-types. These ...
Loading Comments...