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Tankers will have to wait for a breakthrough against ALS as a major new trial led by The University of Texas Health Science Center at Houston (UTHealth) offers a unique therapy option.
That is the story from the TRAK-ALS trial a randomized controlled trial sponsored by Mayo Clinic published in the Journal of Clinical Investigation. This study tested the effect of a compound derived from rhodiola tree (RTR) a plant native to Asia and North America. It was conducted at 11 clinical trial sites in the United States and Canada.
TRAK-ALS is a preclinical trial that is now entering its second and final year. At approximately 14 months 90 percent of the study participants (105 patients) had responded to a maintenance phase. It is still unknown how long the response will last. Because of this The UTHealth researchers say the scientists are just beginning to definitively answer the question of whether the potential therapy as used in RTR cultivation could slow or stop the progression of death and cognitive decline in ALS and other neurodegenerative diseases.
Our aim is to determine if we can preserve or slow ALS progression and improve cognitive function in a group of patients each year who will never be able to drive or perform any day-to-day activities due to vascular dementia syndrome in the elderly lives said Dr. Irfan Rahman Khan the studys lead author a co-investigator of the trial at UT Health. The trial is a result of two multi-site but very similar randomized clinical trials that showed promise.
This is a really important breakthrough due to the possibility that this compound will be effective even in the reality of medical practice said Dr. John Doescher a Mayo Clinic Neurologist who is an internationally known expert on Parkinsons and ALS.
Lead author Dr. James Melendez Assistant Professor of Neurology says that if the compound proves to be safe for human use it is a significant breakthrough for our understanding of ALS. Also because the compound is active at multiple sites simultaneously and not used together the compound is possible to use parts to prevent disease progression and side effects as well as to optimize for treatment utilization and learning outcomes.
Senior Author Dr. Christopher Savage Professor of Neurology at UT Health said This new data from RTR-ALS offers an even stronger case for testing the safety and efficacy of a compound derived from rhodiola tree.
We found statistically significant improvements in scores on both the physical severity (physical function) scale and on the international classification of disease (AD) Savage said. We also performed a full Q-Test on 95 percent of the volunteers using the patient data to assess Alzheimers disease and Parkinsons disease.
One additional interesting observation is that the effects of RTR are but to date limited in preclinical studies of the compound and the effect on outcomes appears to be as strong as actually of RTR itself.