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Thanks to a friend, the below report was brought to my attention yesterday. As the person who sent it to me said, "this document is very readable in layman's terms covering targets for intervention and pipeline improvements to bring potential therapies to fruition". To me, between the efforts of HDF, HDSA/CHDI and our international body of scientists working on HD, we can finally see some light towards getting a portfolio of therapeutic candidates.......or an HD cocktail..... in the not-to-distant future!
Please share this report with your HD families who may not have access to the internet.
Hereditary Disease Foundation's
PIPELINES AND PATHOGENESIS: NEW HORIZONS
PIPELINES AND PATHOGENESIS: NEW HORIZONS
download here: http://www.hdfoundation.org/PDF/PP_Report.pdf
January 20–21, 2007
Santa Monica, California
Report prepared by Marina Chicurel, Ph.D.
Santa Monica, California
Report prepared by Marina Chicurel, Ph.D.
Abstract - [excerpts - see document for full abstract]:
Inspired by a brave couple whose lives have been marred by Huntington’s disease (HD), participants at the “Pipelines and Pathogenesis: New Horizons” workshop identified key areas of research and strategies to help translate basic findings into treatment possibilities for HD. Although HD has presented a formidable challenge for scientists trying to understand its pathology and develop therapies, new results and approaches suggest effective treatments may emerge in the near future. In addition, the lessons learned from HD promise to help find treatments for other neurological disorders.
Inspired by a brave couple whose lives have been marred by Huntington’s disease (HD), participants at the “Pipelines and Pathogenesis: New Horizons” workshop identified key areas of research and strategies to help translate basic findings into treatment possibilities for HD. Although HD has presented a formidable challenge for scientists trying to understand its pathology and develop therapies, new results and approaches suggest effective treatments may emerge in the near future. In addition, the lessons learned from HD promise to help find treatments for other neurological disorders.
To make the most of these findings, participants agreed it will be important to prioritize the growing portfolio of therapeutic candidates. In addition, guidelines for translating the results of animal studies into clinical procedures will be needed, as well as new biomarkers to follow HD progression. Participants also emphasized the opportunity of applying lessons learned from HD to the study of other neurodegenerative disorders, and vice versa.
In sum, participants were optimistic about the future and hopeful they will soon be able to answer the plea for help put forward by the couple who generously shared their experiences at the meeting.
Concluding thoughts
In sum, participants were optimistic about the status of HD research. The development of strategies to combat HD at its primary source, including RNAi and intrabodies, is now well underway. In addition, multiple downstream pathways and molecules are emerging as feasible therapeutic targets. Modulating the cellular stress response, correcting the BDNF deficiency associated with HD, and targeting synaptic proteins such as glutamate transporters or calcium channels are some of the many encouraging candidates that may yield effective treatments. Furthermore, systematic, unbiased approaches to identify HD modifiers promise to further enrich the opportunities for therapeutic interventions.
In sum, participants were optimistic about the status of HD research. The development of strategies to combat HD at its primary source, including RNAi and intrabodies, is now well underway. In addition, multiple downstream pathways and molecules are emerging as feasible therapeutic targets. Modulating the cellular stress response, correcting the BDNF deficiency associated with HD, and targeting synaptic proteins such as glutamate transporters or calcium channels are some of the many encouraging candidates that may yield effective treatments. Furthermore, systematic, unbiased approaches to identify HD modifiers promise to further enrich the opportunities for therapeutic interventions.
A clear indicator of the recent progress made in the field was the priority participants gave to identifying concrete strategies to streamline the translation of lab results into clinical trials. A genuine interest in identifying key requirements and setting up guidelines for scientists to begin moving their data into the therapeutic pipeline was evident.
The optimization of this translational process, together with the recent strides in HD research, suggest it may not be very long before the plea for help put forth at the beginning of the workshop is answered.
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This document addresses:
Abstract
The far-reaching consequences of HD
Current status of the clinical pipeline
Current status of the clinical pipeline
Targets
Targeting mutant huntingtin mRNA: RNAi
Targeting mutant huntingtin protein
Targeting the cellular stress response
Targeting mutant huntingtin processing
Targeting mutant huntingtin’s disruption of transcription
Targeting the reduction of BDNF
Targeting cell-cell interactions: basal ganglia circuitry
Targeting cell-cell interactions:basal ganglia circuitry in HD
Targeting cell-cell interactions: synaptic disruptions in HD
Targeting modifiers of HD: Genetic screens
Targeting modifiers of HD: Aging and metabolism
Targeting environmental modifiers of HD
Accelerating flow through the pipeline
Streamlining the transition from animals to humans
Improving measures of HD in humans
Targeting mutant huntingtin mRNA: RNAi
Targeting mutant huntingtin protein
Targeting the cellular stress response
Targeting mutant huntingtin processing
Targeting mutant huntingtin’s disruption of transcription
Targeting the reduction of BDNF
Targeting cell-cell interactions: basal ganglia circuitry
Targeting cell-cell interactions:basal ganglia circuitry in HD
Targeting cell-cell interactions: synaptic disruptions in HD
Targeting modifiers of HD: Genetic screens
Targeting modifiers of HD: Aging and metabolism
Targeting environmental modifiers of HD
Accelerating flow through the pipeline
Streamlining the transition from animals to humans
Improving measures of HD in humans
Concluding thoughts [see above]
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