This week I’d like to shine the spotlight on a few companies who are making a difference in the field of orphan drug development.

1. PSR-Agility, a leading Dutch orphan-drug development expert, has been nominated for the category “Best CRO for Orphan Drugs” in the ROAR Awards.

2. On November 7 Prosensa, the Dutch biopharmaceutical company which focuses on RNA-modulating therapeutics for the treatment of rare diseases, announced the successful enrollment of the 100^th patient into their Natural History study of Duchenne muscular dystrophy (DMD).

3. The FDA announced on November 1 that Roche’s Gazyva (obinutuzumab) has been approved for the treatment of patients with previously untreated chronic lymphocytic leukemia (CLL), and that Gazyva has been

granted orphan drug designation.

Kudos to these companies who think outside the box, blaze new trails and change the blockbuster-dominated landscape to give hope to patients who don’t get a lot of good news.

University Hospital Maastricht (AZM) in the Netherlands reported last week that their researchers have found an explanation for the severe muscle atrophy and weight loss which plays a large part in the high mortality rate for lung cancer.

According to the AZM website, their studies on lung cancer showed a significant increase in the activity of NF-?B protein, which in turn is associated with increased breakdown of muscle protein and inhibition of protein production. Project leader Annemie Schols, Profession of Nutrition and Metabolism in Chronic Diseases, expresses her hope that this discovery will lead to more effective treatment of cachexic cancer patients, particularly at the pre-cachexic stage which has so far been largely been ignored in the treatment of cancer.

It’s not always just about the drugs that tackle a disease head-on; this is a great insight from an unexpected angle that should help these patients stay stronger and fight back harder. Read more about this discovery by Dr. Schols and her team on http://www.azm.nl/hetazm/nieuws/258927 (article in Dutch).