Essay Example on Potential novel therapy for ALS

Future hopes rest upon longer term pre clinical trials to assess the tolerance and long term efficacy of the ataxin 2 ASO and subsequent human clinical trials if these prove successful As is the case in the wider field of neurodegenerative disease therapy questions surrounding the efficacy of the ataxin 2 ASO once disease symptom onset has occurred will need to be addressed The design of clinical and pre clinical trials to evaluate whether the ataxin 2 ASO is effective once ALS symptoms have manifested will be an important aspect of the assessment of the treatment s potential as detection of disease is often difficult meaning that it is unlikely for treatment administration to begin prior to disease onset In order for scientists to assay the ASO s action it will be crucial to develop ataxin 2 and or TDP 43 markers which can be used in the CNS to determine whether the ASO is interacting as expected This will not only provide confirmation of the action of the ataxin 2 ASO but could also provide useful insight if trial results are not as anticipated as has been the case when several promising neurodegenerative disease therapies have been tested in later stage trials Despite these substantial challenges Becker et al s paper represents a landmark achievement in ALS treatment research and provide significant hope for a broadly effective ASO therapy in clinical trials and beyond