LMTX is the first drug trying to undo so-called tau tangles in the brain, caused by Alzheimer’s disease. As it reached the final stage of clinical trials, results of which were eagerly awaited, there was no effect in patients taking LMTX as an add-on. The initial reaction to the outcome was disappointment since the patients who received LMTX did not have a slower rate of decline in mental ability or daily functioning than those in the control group. One hypothesis why LMTX did not work with other drugs was that the other drugs set off a mechanism that cells use to expel drugs. LMTX was caught up in this and also expelled from brain cells; this has yet to be proven. However, there is a chance that LMTX may be effective for certain patients if used alone, without additional medications.
For those taking the drug as monotherapy, there were large effects that were clinically meaningful. Dr. Claude Wischik, founder and chief executive of TauRx Therapeutics, developer of LMTX, said a second clinical trial sponsored by another company found the same phenomenon and planned to apply for approval to be used by itself. There was no obvious explanation why it would be expected to work only in patients not getting other drugs. The participants in the monotherapy trial had essentially no decline in cognition or daily functioning for the entire 15 months of the trial, said Dr. Serge Gauthier, director of the Alzheimer’s disease research unit at McGill University and the principal investigator of the trial. He is also chairman of TauRx’s scientific advisory board. Conclusions were drawn from only a small subset of patients and it is thought that regulators might require another large study, using participants not using other drugs. There may be some other factor and not the drug itself that could explain why these patients did better. More tests and results to come. Read more at: http://www.nytimes.com/2016/07/28/business/alzheimers-drug-lmtx-trial.html
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