Although the entire details of the study have yet to be published, results of a new study testing a drug designed to halt Alzheimer's were released just last month at the Alzheimer's Association International Conference in Toronto, Canada. The drug, created by pharmaceutical company TauRX Therapeutics Ltd, is called LMTX. The drug's goal is to prevent or slow the progression of Alzheimer's on a neurological level by making the tau protein, which gathers in tangles in the brain and is suspected to be a key to curing the disease, unable to gather. In theory, this would make treating Alzheimer's disease that much easier by stopping its progression as soon as it is diagnosed. Yet much remains unknown about Alzheimer's progression. What should caregivers know about the efficacy of this drug? Here are a few considerations, caveats, and hopeful thoughts for future drug development.
This Is Not a Cure
Even if LMTX is effective in every patient every time, it will not (and does not seek to) cure Alzheimer's disease. Dementia and eventual death caused by Alzheimer's will not be erased through this treatment--it will merely be paused or slowed. Of course, this is not something to be overlooked. Slowing or pausing dementia progression can mean years of happy, healthy life for seniors with Alzheimer's that would otherwise have been spent coping with a debilitating disease that can cause a loss of memory, personality changes, and even dangerous aggression. Yet a cure has not yet been developed, and, in spite of media hype, anyone selling such a cure is being untruthful.
The Study Actually Failed
Although every detail about this new drug and its Phase 3 study are not known (only the abstract is available currently) the drug failed in its original design. When coupled with other treatments for Alzheimer's, the drug was ineffective. Experts agree that, in general, the trial saw little to no improvements for most of its patients, which means that many patients went into the trial hoping for a halt to their disease and found none with LMTX. This is significant because it means the hopeful results of the study (discussed below) come with a large caveat.
What the Study Did Show
The study about LMTX was conducted by Serge Gauthier, director of the Alzheimer's Disease Research Unit at McGill University, and others. The researchers found that LMTX does work in isolation. If seniors in the trial were on no other medications to manage their symptoms, LMTX did help in halting the development of the disease. This was only about fifteen percent of patients in the drug trial, making it uncertain exactly what the effect is--statistically, this is not enough to begin marketing the drug as a way to halt the development of Alzheimer's. However, it is encouraging--if the drug works in isolation in this trial, a larger trial of people taking LMTX in isolation is called for, and the drug has a promising future if the trial can replicate the results for this small percentage with a larger group.
The results were apparent not just in the tau protein tangles, but in the functional tests used by the scientists conducting the study. This means that, beyond just keeping the brain healthier on a cellular level, the actual symptoms of Alzheimer's progression, such as memory loss, language disruption, and confusion were maintained rather than worsened over the course of fifteen months. For caregivers, this is a sign of hope for future drug development--perhaps TauRX is on to something. Unfortunately, those with Alzheimer's often need their symptom-reducing drugs in order to keep them healthy and safe. Not managing the symptoms of Alzheimer's can lead to aggressive episodes, wandering, and even depression or anxiety as the brain becomes increasingly compromised. That is something very important to consider as the drug enters further trials, and as providers may offer the option of participation in the trial to patients with Alzheimer's.
Gallagher, James. (July 27, 2016). Drug "may slow" Alzheimer's brain death. BBC News, Available at http://www.bbc.com/news/health-36906554. Retrieved August 7, 2016.
Gauthier, Serge, et al. (July 27, 2016).. Phase 3 Trial of the Tau Aggregation Inhibitor Leuco-Methylthioninium-Bis(hydromethanesulfonate) (LMTM) in Mild to Moderate Alzheimer's Disease. Oral Session, Wednesday, July 27, 2016. Clinical Therapeutics: Clinical Trials-Tau and Inflammation. Alzheimer's Association International Conference. Toronto, Canada.
Lane, Lani. (July 28, 2016). Alzheimer's Cure Is Within Reach Through Drug Maintenance. Parent Herald. Available at http://www.parentherald.com/articles/57503/20160728/alzheimer-s-cure-is-within-reach-through-drug-maintenance.htm. Retrieved August 7, 2016.
Morin, Frances. (July 28, 2016). Anti-Tau Drug Fails to Demonstrate Benefit in Treating Alzheimer's Disease, Shows Some Promise as Monotherapy: Presented at Alzheimer's Association International Conference. FirstWordPharma.com, Available at https://www.firstwordpharma.com/node/1403523?tsid=28®ion_id=4. Retrieved August 7, 2016.