The decision-making process and criteria in selecting candidate drugs for progeria clinical trials

EMBO Molecular Medicine

Volumn 8, Issue 7, 2016, Pages 685-687

  Gordon, Leslie B ^a,b   Kieran, Mark W ^c   Kleinman, Monica E ^a   Misteli, Tom ^d  

^a BOSTON CHILDREN S HOSPITAL   (United States)

^b BROWN UNIVERSITY   (United States)

^c DANA FARBER CANCER INSTITUTE   (United States)

^d NATIONAL CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

LAMIN; LAMIN A; LONAFARNIB; PROGERIN; UNCLASSIFIED DRUG;

CLINICAL DECISION MAKING; CLINICAL TRIAL; DRUG DEVELOPMENT; DRUG TOXICITY; HIGH THROUGHPUT SCREENING; HUMAN; NONHUMAN; NOTE; PRIORITY JOURNAL; PROGERIA; PROTEIN LOCALIZATION; RARE DISEASE; TOXICITY TESTING;

EID: 84977615740     PISSN: 17574676     EISSN: 17574684     Source Type: Journal    
DOI: 10.15252/emmm.201606280     Document Type: Note

References (8)

1. De Sandre-Giovannoli A, Bernard R, Cau P, Navarro C, Amiel J, Boccaccio I, Lyonnet S, Stewart CL, Munnich A, Le Merrer M et al (2003) Lamin A truncation in hutchinson-gilford progeria. Science 300: 2055
2. Eriksson M, Brown WT, Gordon L, Glynn MW, Singer J, Scott L, Erdos MR, Robbins CM, Moses TY, Berglund P et al (2003) Recurrent de novo point mutations in lamin A cause Hutchinson-Gilford progeria syndrome. Nature 423: 293–297
3. Gordon LB, Kleinman ME, Miller DT, Neuberg DS, Giobbie-Hurder A, Gerhard-Herman M, Smoot LB, Gordon CM, Cleveland R, Snyder BD et al (2012) Clinical trial of a farnesyltransferase inhibitor in children with Hutchinson-Gilford progeria syndrome. Proc Natl Acad Sci USA 109: 16666–16671
4. Gordon AS, Gordon LB (2014) The Progeria Research Foundation: its remarkable journey from obscurity to treatment. Expert Opin Orphan Drugs 2: 1–9
5. Gordon LB, Massaro J, D'Agostino RB Sr, Campbell SE, Brazier J, Brown WT, Kleinman ME, Kieran MW, Progeria Clinical Trials C (2014a) Impact of farnesylation inhibitors on survival in Hutchinson-Gilford progeria syndrome. Circulation 130: 27–34
6. Gordon LB, Rothman FG, López-Otín C, Misteli T (2014b) Progeria: a paradigm for translational medicine. Cell 156: 400–407
7. Herter-Sprie GS, Kung AL, Wong KK (2013) New cast for a new era: preclinical cancer drug development revisited. J Clin Invest 123: 3639–3645
8. Zhang H, Kieckhaefer JE, Cao K (2013) Mouse models of laminopathies. Aging cell 12: 2–10