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Prohost Biotech - Tuesday, August 19, 2014

Researchers at Columbia University Medical Center were able to identify the immune cells responsible for causing the baldness known as alopecia areata. The researchers chose to use  ruxolitinib -- a JAK1 and 2 inhibitor marketed under the trade name Jakafi by the biotech firm Incyte (INCY). The drug is currently approved for intermediate or high-risk myelofibrosis (MF), including primary MF, post–polycythemia vera MF and post–essential thrombocythemia MF.

Alopecia areata is recognized as an autoimmune disease caused by the immune system’s attack on people’s own cells and organs, in this case, the immune system attacks the hair follicles.  The researchers tested ruxolitinib on a few people suffering from the alopecia baldness with stunning positive results. Many of the participants regrew hair in a short period of time – results never experienced before in moderate to severe baldness.Patients experienced total regrowth of hair in less than six months following the drug's administration.   

"We still need to do more testing to establish that ruxolitinib should be used in alopecia areata, but this is exciting news for patients and their physicians," Raphael Clynes, the leader of the trial  said. "This disease has been completely understudied – until now, only two small clinical trials evaluating targeted therapies in alopecia areata have been performed, largely because of the lack of mechanistic insight into it."

Published in Nature Magazine, Mr. Clynes said: "We've only begun testing the drug in patients, but if the drug continues to be successful and safe, it will have a dramatic positive effect on people with this disease."

Another JAK inhibitor, Tofacitinib, seems to have also succeeded in stopping the immune system attack on the hair follicles. Tofacitinib, developed by Pfizer, inhibits the enzyme Janus kinase 3 (JAK3), hence interferes with the JAK-STAT signaling pathway, which transmits extracellular information into the cell nucleus, influencing DNA transcription. The drug has good results in arthritis as it inhibits the production of inflammatory mediators and suppressing STAT1- dependent genes in the joints. This efficacy correlated with the inhibition of both JAK 1 and JAK  3 signaling pathways, suggesting that tofacitinib may exert therapeutic benefit via pathways that are not exclusive to inhibition of JAK3.

Prohost Comments: This is good news for people who suffer from alopecia areata and for Incyte. As we mentioned before, targeted drugs will not stop at treating one disease. Continued genetic research on the origin of various diseases is expected to pinpoint and confirm a single target as an important protein or enzyme in the pathway of many diseases. That’s what make companies such as Incyte, Exelixis, Synta, Sunesis, Array and others (see prohost portfolio) exciting.  

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