It's very early (so we are not publicly talking about the details until this has passed peer review), but we are very excited that it looks like we may have multiple small molecules which appear to inhibit toxicity of Abeta, the protein which is the toxic element in Alzheimer's Disease.
This is exciting in many ways. It's been a long road for FAH to get to this point, but we are starting to see the possibility of seeing these results published easily before our 10th birthday (October 2010). Considering all the technology development that had to be done in the first five years, these results have come very quickly (in the last 3 years), which is exciting. In particular, we are now looking to apply these methods to other protein misfolding diseases (we have pilot projects for Huntington's Disease underway).
Finally, I should stress that while we're very excited about this, it's still early and a lot can go wrong between where we are and having a drug that doctors can prescribe. Over the holidays, we will be double checking the experimental data, crossing t's and dotting i's to make sure there is nothing missed before we think about submitting this for peer reviewed publication. Also, there is still a long way from an interesting possible drug (where we are now) to something which has passed
FDA clinical trials (where we'd love to be), and a lot can go wrong in clinical trials in particular.
Thus, this is an important milestone for FAH and we are very grateful to all who have contributed."