Day 1 P1 Blumenstein Why CHDI Reached Out to C-Path · Why CHDI reached out to C-Path Robi...
Transcript of Day 1 P1 Blumenstein Why CHDI Reached Out to C-Path · Why CHDI reached out to C-Path Robi...
Why CHDI reached out to C-Path
Robi BlumensteinPresident, CHDI Management, [email protected]
Silver Spring, MDNovember 6, 2017
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How learning to play chess can inform efforts to bring effective
treatments for HD to patients
Acknowledgments
Vahri BeaumontJonathan Bard
Doug MacdonaldLadislav Mrzljak
Larry ParkRoger CachopeDavid Howland
Seung KwakRobert Pacifici
Celia DominguezLeticia Toledo-Sherman
John WityakMichel MaillardBeth BorowskyAndrew Wood
Valentina DildaCheryl Fitzer-AttasCristina Sampaio
Andrea VarroneChrister HalldinJenny Häggkvist
P. Fazio
Steve ZhangPhilip GregoryEd RebarFyodor Ursinov
Nikisha CartyYalda SedaghatKarsten TillackAndreas WeissMichael PrimeFrank HerrmannSabine SchaertlChristopher BrownDaniel Clark-FrewSamuel CoePaul GilesSamantha GreenSarah HayesDuane HigginsRichard MarstonMatthew MillsAnthony PadfieldMadlen HotzeKathrin PetersenManuela HeßmannGlauco TarozzoStefanie JahnMiriam BaritschTom SchwagarusNicole Höschen,Marta KotasinskaKarsten KottigGalina BursowChristina ThiedeLisa Mettler
Sarah TabriziEd Wild
Blair Leavitt
Frank BennetHolly Kordasiewicz
Laura OrsattiRoberto SpezialeAlessandra CeccacciChantal PaoliniLicia TomeiAlberto Bresciani
Czech Institute of Animal Physiology Zdenka EllederovaJan Motlik
University of AucklandSusanne ReidRussell Snell
Aeash RassoulpourMariette Heins
David FischerMichela Tessari
George McAllister
Acknowledgements
Science AdminRobertPacifici RuthBasuCristinaSampaio DavidRankinCeliaDominguez EileenNeacyIgnacioMunoz-SanjuanThomasVogtAndrewWood
ControlPreManifestHD
A last word on strategy… and hope
PreManifestControl HD
CONFIDENTIAL INFORMATION11
CAG age Model, 80% power, alpha = .05, 5% annual censoring
10% 20% 30% 40% 50%3 19,509 4,690 1,997 1,071 649 4 13,669 3,297 1,409 759 462 5 10,321 2,496 1,070 578 353 6 8,196 1,986 853 462 283
Estimates for a group that is all gene-expanded, with CAG lengths known
Magnitude of Therapeutic Effect
Year
sfo
llow
ed
circa 2004
Phenotype progression through States 1 - 9
Distance between states proportional to empirical durations in years
1 2 3 4 5 6 7 8 9
Motor, cognition & GCI
Motor, cognition, GCI+ occupation
Cognition, motor + moderate activities (walking, climbing)
Function, SF & Caregiver survey
Motor, cognition + MMSE
Cognition, motor& function
Function, cognition,motor, physio & MMSE
abc: new outcome measures
10#$
10%#$
A word on strategy… and hope
Number of half moves played
Expe
cted
hal
f mov
es re
mai
ning
CONFIDENTIAL INFORMATION20