Targeting Medium-Chain Acyl-CoA Dehydrogenase for Glioblast…

Targeting Medium-Chain Acyl-CoA Dehydrogenase (MCAD) for Glioblastoma (GBM)

Developing potent, selective MCAD inhibitors

Hits identified by high-throughput screen of 278k compounds based on RF-MS.

MCAD structures with screening hits guides small molecule optimization.

Example of a partially optimized screening hit with cellular potency < 1 µM.

Crystal structure of MCAD:Octanoyl-CoA

Properties

CmpdID: 75915

MCAD IC 50 (nM)

37

Octanoyl-CoA (substrate)

SCAD IC 50 (nM)

>5,600

LCAD IC 50 (nM)

>5,600

VLCAD IC 50 (nM)

>5,600

Flavin adenine dinucleotide (FAD, cofactor)

MCAD CETSA IC 50 (nM)

~200

MCAD OCCT (nM)

540

Pampa Pe (x10 -6 cm/s (% rec))

11(60%)

100

Plasma St. (m/r/d/h t 1/2 min)

330/100/360/99

Cryo-EM structure of MCAD

MW/cLogP/TPSA

459/2.28/116

50

MPO/BBB

3.83/3.16

0

Refined map at 3.3 Å Using 88k particles

F Yu 1 , P Leonard 1 , M Hamilton 1 , F Puca 2 , N Pham 2 , N Rogers 1 , F Alvarez 1 , C Rodriguez 1 , V Nair 1 , N Akkaladevi 1 , R Thapar 1 , S Vaccaro 1 , A Mendiola 1 , Q Xu 1 , M Geck Do 1 , J Cross 1 , M Soth 1 , Y Jiang 1 , G Draetta 2 , and P Jones 1

0.0001

0.01

1

100

Conc. (nM)

IACS-050595-000-2, IC50 = 1000 nM IACS-075710-000-1, IC50 = 55 nM IACS-135587-000-1, IC50 = 8238 nM

1 Institute for Applied Cancer Science 2 Department of Genomic Medicine

Nov. 17-18, 2022

3

MD Anderson Cancer Center

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