I (Aunt L) just read an interesting article called Cracking the Code of NF1 , about research being performed by Dr. Nancy Ratner at Cincinnati Children's. The research is remarkable and promising--and yet, still so many unknowns! What struck me with this article is this is the first article I have read on a professional web page that expresses the frustration of all that remains to be understood about NF1 and what works and what doesn't work.
This particular research is studying a cancer drug that seems able to disrupt the molecular process of NF1 called MEK, showing the reduction of 80% of plexiform tumors in tested mice. Dr. Ratner believes that the reason the drug worked is that it comes the closest to the specific molecular defect of NF1. To try to put this in easier terms: the free running tumor creating process that goes on in patients with NF1 would be the bulls eye of a target. The most recent promising tumor treatments have come as close as 5 rings away from the bulls eye (but also at times misses completely), while this specific drug appears to get as close as 1 ring away from the center.
Risks involved with this new potential treatment are, as with so much of NF1, not clear. Neurofibromas are "tough", they are quite fibrous which is why chemo-therapies are often prescribed for such long periods of time (Ella's is 16 months). The longer the treatment the higher the risk, which leaves many doctors reluctant to advise strong and/or experimental therapies with NF1 patients. Dr. Ratner is hoping to have clinical trials with NF1 patients to be opening soon. Her entire career has been devoted to NF1 research and her goal is a cure, which is refreshing compared to so many articles about research that are purely symptomatic treatment centered. While she continues to focus on a cure, Dr. Ratner believes this treatment shows great promise and is another step towards a cure.
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