CureDuchenne Research Portfolio
| Drug/Project Name |
Status |
| Description |
Optimization/Preclinical Phase I Phase II Phase III |
|
| Human Clinical Trials |
|
Glaxo Smith Kline: GSK2402968
|
|
|
Exon Skipping: GSK is developing Prosensa's exon 51 skipping drug that would be applicable to approximately 15% of Duchenne patients if successful.
|
|
|
|
Exon Skipping: Prosensa is testing a compound for exon 44 skipping which, if successful will restore some dystrophin to muscle cells.
|
|
Ataluren: PTC Therapeutics
|
|
|
This compound addresses 10%-13% of Duchenne patient who have a premature stopcondon mutation.
|
|
AVI - Biopharma: AVI-4658
|
|
|
Exon Skipping: AVI is testing a different chemistry than GSK/Prosensa. This trial is for exon 51, results should be available mid-2012
|
|
Ibuprofen + Isosorbide Dinitrate: Dr. Clementi
|
|
|
FDA approved drugs that show some potential to mitigate some of the effects of Duchenne
|
|
Sildenafil: Kennedy Krieger Institute
|
|
|
Study of FDA approved drug (Viagra) for its potential to improve the health of muscle cells
|
|
Adult Stem Cell: Dr. Cossu
|
|
|
Dr. Cossu will study adult stem cells in a very small group of Duchenne patients to test for safety
|
|
VPB 15: ReveraGen Biopharma
|
|
|
The only approved treatment for Duchenne boys are corticosteroids, which carry significant side effects. This project is developing a drug that, if successful, would have the benefits of steroids without the side effects.
|
|
|
|
This is a quick and inexpensive test for toxicity and effectiveness of potential drugs.
|
|
| Preclinical |
|
Gene Therapy: Dr. Chamberlain
|
|
|
Gene therapy replaces the faulty gene with micro-dystrophin which could potentially repair the muscle cells and provide a long term benefit.
|
|
Spironolactone + lisinopril/losartan: Rafael-Fortney
|
|
|
These FDA drugs are being tested in the mouse dystrophin model (mdx) to ascertain if a combination of these drugs provide cardiac and skeletal muscle benefit
|
|
Optimize exon 44 & 53: Dr. Qi Lu
|
|
|
Dr. Qi Lu is optimizing the best sequence for morpholino exon skipping of exons 44 and 53. This project will provide options for the best compounds to take to trial.
|
|
Duplication Mouse Model: Dr. Flanigan
|
|
|
Dr. Kevin Flanigan is developing a new mouse model that will be able to test potential drugs for the rare cases of Duchenne that have duplication mutations
|
|
Utrophin: PTC Therapeutics
|
|
|
Utrophin is another muscle protein that could compensate for the lack of dystrophin. PTC is investigating an oral drug to upregulate utrophin to compensate for the lack of dystrophin
|
|
PTC Therapeutics: Myostatin and Insulin-Like Growth Factor 1 (mIGF1)
|
|
|
Drugs to modulate these muscle proteins were selected as targets to increase muscle growth and regeneration. Drugs that target these proteins are expected to increase muscle mass.
|
|
| Clinical Trials Centers |
|
Center for Duchenne Muscular Dystrophy at UCLA
|
|
|
This is the first Duchenne center of excellence to serve patients in Southern California
|
|
