There are a number of very positive findings in this study. First, the study was well-performed, with no deviations from the protocol. The treatment appears to be safe and well-tolerated and there were increases of PRX002 in the cerebrospinal fluid, suggesting, although not proving, that this immunotherapy will have access to the brain. Also, robust and dose-dependent decreases in α-synuclein in the serum indicate that following treatment, the immunotherapy is working in a cell type that is accessible for examination. These data are encouraging, and I look forward to a study designed to test efficacy.
I have received conference/lecture honorariums from Prothena and have been a scientific consultant to Roche.
This year represents the 10th anniversary of the birth of the concept of prion-like propagation of α-synuclein pathology, and it is gratifying to see that it has resulted in numerous clinical research programs. Several immunotherapy programs, at various stages of clinical development, target α-synuclein in different ways. I think it is truly exciting that so many efforts are underway to mitigate the cell-to-cell transfer propagation of α-synuclein pathology. In just a few years from now, we should know if this is a viable approach to significantly slow progression of Parkinson’s disease. This would be a much-needed success in a disease where there still only exist symptomatic therapies.
It is encouraging to see that PRX002 had a good favorable safety and tolerability profile in this Phase 1b trial in people with Parkinson’s disease, so the clinical testing of this approach can continue as planned in Phase 2. While it is unclear that there is any value of decreasing α-synuclein in serum in Parkinson’s disease, it nevertheless demonstrates biological activity of the therapeutic. Furthermore, the antibodies did enter the CNS at levels that appear biologically relevant.
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Rush University, Chicago, and Van Andel Research Institute, Grand Rapids, Michigan
There are a number of very positive findings in this study. First, the study was well-performed, with no deviations from the protocol. The treatment appears to be safe and well-tolerated and there were increases of PRX002 in the cerebrospinal fluid, suggesting, although not proving, that this immunotherapy will have access to the brain. Also, robust and dose-dependent decreases in α-synuclein in the serum indicate that following treatment, the immunotherapy is working in a cell type that is accessible for examination. These data are encouraging, and I look forward to a study designed to test efficacy.
View all comments by Jeffrey H. KordowerAssociate Director of the Van Andel Research Institute, and Director of the Center for Neurodegenerative Science
I have received conference/lecture honorariums from Prothena and have been a scientific consultant to Roche.
This year represents the 10th anniversary of the birth of the concept of prion-like propagation of α-synuclein pathology, and it is gratifying to see that it has resulted in numerous clinical research programs. Several immunotherapy programs, at various stages of clinical development, target α-synuclein in different ways. I think it is truly exciting that so many efforts are underway to mitigate the cell-to-cell transfer propagation of α-synuclein pathology. In just a few years from now, we should know if this is a viable approach to significantly slow progression of Parkinson’s disease. This would be a much-needed success in a disease where there still only exist symptomatic therapies.
It is encouraging to see that PRX002 had a good favorable safety and tolerability profile in this Phase 1b trial in people with Parkinson’s disease, so the clinical testing of this approach can continue as planned in Phase 2. While it is unclear that there is any value of decreasing α-synuclein in serum in Parkinson’s disease, it nevertheless demonstrates biological activity of the therapeutic. Furthermore, the antibodies did enter the CNS at levels that appear biologically relevant.
View all comments by Patrik BrundinMake a Comment
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