What does “Disruptive” mean? Thoughts on the NIH SCI 2020 meeting
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摘要
The SCI 2020 meeting is a wakeup call for the SCI research community. The very large worldwide community of individuals with SCI and their family members are justifiably concerned about the pace of progress. But mismatches between research system realities("where is the innovation", grant durations versus the time it takes to do meaningful chronic studies in animals and people, etc.), clinical trial funding mechanisms, FDA approval processes, and patient needs and expectations will require dramatic changes in strategies and tactics. Those were not addressed at this meeting. If the SCI patient community wants to use its frustration to accelerate the development of specific therapies, itmay want to look at an aggressive top-down approach using a contract research organization model and have all studies done in parallel from the beginning to find robust therapies. Angel investors or foundations in it for the long haul will be needed. Alternatively, the National Center for Advancing Translational Sciences(NCATS) model of funding translational research might work, but rock solid therapeutic targets will be required. This strategy could lead to "disruptions" that could dramatically accelerate the pace of development of novel therapeutics.
The SCI 2020 meeting is a wakeup call for the SCI research community. The very large worldwide community of individuals with SCI and their family members are justifiably concerned about the pace of progress. But mismatches between research system realities("where is the innovation", grant durations versus the time it takes to do meaningful chronic studies in animals and people, etc.), clinical trial funding mechanisms, FDA approval processes, and patient needs and expectations will require dramatic changes in strategies and tactics. Those were not addressed at this meeting. If the SCI patient community wants to use its frustration to accelerate the development of specific therapies, itmay want to look at an aggressive top-down approach using a contract research organization model and have all studies done in parallel from the beginning to find robust therapies. Angel investors or foundations in it for the long haul will be needed. Alternatively, the National Center for Advancing Translational Sciences(NCATS) model of funding translational research might work, but rock solid therapeutic targets will be required. This strategy could lead to "disruptions" that could dramatically accelerate the pace of development of novel therapeutics.
The SCI 2020 meeting is a wakeup call for the SCI research community. The very large worldwide community of individuals with SCI and their family members are justifiably concerned about the pace of progress. But mismatches between research system realities("where is the innovation", grant durations versus the time it takes to do meaningful chronic studies in animals and people, etc.), clinical trial funding mechanisms, FDA approval processes, and patient needs and expectations will require dramatic changes in strategies and tactics. Those were not addressed at this meeting. If the SCI patient community wants to use its frustration to accelerate the development of specific therapies, itmay want to look at an aggressive top-down approach using a contract research organization model and have all studies done in parallel from the beginning to find robust therapies. Angel investors or foundations in it for the long haul will be needed. Alternatively, the National Center for Advancing Translational Sciences(NCATS) model of funding translational research might work, but rock solid therapeutic targets will be required. This strategy could lead to "disruptions" that could dramatically accelerate the pace of development of novel therapeutics.
引文
DiMasi JA,Grabowski HG,Hansen RW(2016)Innovation in the pharmaceutical industry:New estimates of R&D costs.J Health Econ 47:20-33.
Harkema S,Gerasimenko Y,Hodes J,Burdick J,Angeli C,Chen Y,Ferreira C,Willhite A,Rejc E,Grossman RG,Edgerton VR(2011)Effect of epidural stimulation of the lumbosacral spinal cord on voluntary movement,standing,and assisted stepping after motor complete paraplegia:a case study.Lancet 377:1938-1947.
Keller T,Krames ES(2009)“On the shoulders of giants”:a history of the understandings of pain,leading to the understandings of neuromodulation.Neuromodulation 12:77-84.
Kigerl KA,Hall JC,Wang L,Mo X,Yu Z,Popovich PG(2016)Gut dysbiosis impairs recovery after spinal cord injury.J Exp Med 213:2603-2620.
Lu P,Wang Y,Graham L,McHale K,Gao M,Wu D,Brock J,Blesch A,Rosenzweig ES,Havton LA,Zheng B,Conner JM,Marsala M,Tuszynski MH(2012)Long-distance growth and connectivity of neural stem cells after severe spinal cord injury.Cell 150:1264-1273.
McNamee LM,Walsh MJ,Ledley FD(2017)Timelines of translational science:From technology initiation to FDA approval.PLoS One12:e0177371.
Morrison SA,Lorenz D,Eskay CP,Forrest GF,Basso DM(2018)Longitudinal recovery and reduced costs after 120 sessions of locomotor training for motor incomplete spinal cord injury.Arch Phys Med Rehabil 99:555-562.
Park KK,Liu K,Hu Y,Smith PD,Wang C,Cai B,Xu B,Connolly L,Kramvis I,Sahin M,He Z(2008)Promoting axon regeneration in the adult CNSby modulation of the PTEN/mTOR pathway.Science 322:963-966.
Harkema S,Gerasimenko Y,Hodes J,Burdick J,Angeli C,Chen Y,Ferreira C,Willhite A,Rejc E,Grossman RG,Edgerton VR(2011)Effect of epidural stimulation of the lumbosacral spinal cord on voluntary movement,standing,and assisted stepping after motor complete paraplegia:a case study.Lancet 377:1938-1947.
Keller T,Krames ES(2009)“On the shoulders of giants”:a history of the understandings of pain,leading to the understandings of neuromodulation.Neuromodulation 12:77-84.
Kigerl KA,Hall JC,Wang L,Mo X,Yu Z,Popovich PG(2016)Gut dysbiosis impairs recovery after spinal cord injury.J Exp Med 213:2603-2620.
Lu P,Wang Y,Graham L,McHale K,Gao M,Wu D,Brock J,Blesch A,Rosenzweig ES,Havton LA,Zheng B,Conner JM,Marsala M,Tuszynski MH(2012)Long-distance growth and connectivity of neural stem cells after severe spinal cord injury.Cell 150:1264-1273.
McNamee LM,Walsh MJ,Ledley FD(2017)Timelines of translational science:From technology initiation to FDA approval.PLoS One12:e0177371.
Morrison SA,Lorenz D,Eskay CP,Forrest GF,Basso DM(2018)Longitudinal recovery and reduced costs after 120 sessions of locomotor training for motor incomplete spinal cord injury.Arch Phys Med Rehabil 99:555-562.
Park KK,Liu K,Hu Y,Smith PD,Wang C,Cai B,Xu B,Connolly L,Kramvis I,Sahin M,He Z(2008)Promoting axon regeneration in the adult CNSby modulation of the PTEN/mTOR pathway.Science 322:963-966.