SCSAP SPEAKER SPOTLIGHT

Roel Verhaak, Ph.D.

 Harvey and Kate Cushing Professor of Neurosurgery

Department of Neurosurgery

Yale School of Medicine

Short Bio

Roel Verhaak, PhD., is a Professor in the Department of Neurosurgery at the Yale School of Medicine. Following graduation with a Ph.D. in medicine from the Erasmus University Medical Center in Rotterdam, the Netherlands, Roel joined the Broad Institute/Dana-Farber Cancer Institute as a postdoctoral associate, supported by a fellowship from the Dutch Cancer Society. During his time at the Broad, he was part of the team analyzing data from The Cancer Genome Atlas. He led the identification and characterization of gene expression subtypes in glioblastoma, work that resulted in a seminal Cancer Cell 2010 publication.

Roel moved to MD Anderson Cancer Center in Houston, TX, in 2010 to start his own lab. Since then, the Verhaak lab has studied tumor evolution and mechanisms of therapy resistance in low- and high-grade gliomas. The group was foundational in establishing the Glioma Longitudinal Analysis Consortium, which has established a resource of molecular profiles over time on a large cohort of patients with a glioma. They identified and described genetic ‘scars’ and cellular phenotypes associated with glioma progression and disease recurrence. Extrachromosomal DNA amplifications were discovered as critical drivers and are now a major part of the team’s research. After being affiliated with the Jackson Laboratory for Genomic Medicine from 2016, the Verhaak lab joined the School of Medicine, Department of Neurosurgery, in 2023. Roel Verhaak is a recipient of the AAAS Wachtel Award, the Agilent Early Career Professor Award, and the Peter Steck Memorial Award, and is a co-founder of Boundless Bio.

Abstract

Despite standard treatment, IDH-mutant gliomas inevitably recur. Therapeutic resistance may result from a combination of intratumoral cellular heterogeneity, epigenetic evolution, and acquired genetic alterations. We charted evolution of IDH-mutant glioma through multi-platform single-nucleus and bulk characterization of longitudinally collected specimens. In addition to defining IDH-mutant malignant cellular states that were reminiscent of IDH-wild type glioma, we analyzed open chromatin accessibility data to define state-specific differentially accessible peaks which supported that the malignant states are epigenetically encoded. Between the time points of collection, we found that there was a longitudinal increase in the cycling, undifferentiated, and mesenchymal-like populations with a corresponding decrease in the astrocyte-like population.  Longitudinal genetic analysis revealed that 19 of 35 tumors acquired at least one of the following genetic alterations: treatment-associated hypermutation, cell cycle alteration, or large changes in copy number alterations. Importantly, tumors that acquired these key genetic alterations demonstrated significant shifts towards reduced differentiation and increased cycling populations while those tumors that did not had more stable malignant profiles. Collectively, our results suggest a common cellular hierarchy across IDH-mutant gliomas with a shift towards reduced differentiation, increased cycling populations during disease progression that is driven by acquired genetic alterations.

Publications:

A brave new framework for glioma drug development.

Hotchkiss KM, Karschnia P, Schreck KC, Geurts M, Cloughesy TF, Huse J, Duke ES, Lathia J, Ashley DM, Nduom EK, Long G, Singh K, Chalmers A, Ahluwalia MS, Heimberger A, Bagley S, Todo T, Verhaak R, Kelly PD, Hervey-Jumper S, de Groot J, Patel A, Fecci P, Parney I, Wykes V, Watts C, Burns TC, Sanai N, Preusser M, Tonn JC, Drummond KJ, Platten M, Das S, Tanner K, Vogelbaum MA, Weller M, Whittle JR, Berger MS, Khasraw M.Lancet Oncol. 2024 Oct;25(10):e512-e519. doi: 10.1016/S1470-2045(24)00190-6.PMID: 39362262 Review.

Single nucleus transcriptomics, pharmacokinetics, and pharmacodynamics of combined CDK4/6 and mTOR inhibition in a phase 0/1 trial of recurrent high-grade glioma.

Johnson KC, Tien AC, Jiang J, McNamara J, Chang YW, Montgomery C, DeSantis A, Elena-Sanchez L, Fujita Y, Kim S, Spitzer A, Gabriel P, Flynn WF, Courtois ET, Hong A, Harmon J, Umemura Y, Tovmasyan A, Li J, Mehta S, Verhaak R, Sanai N.medRxiv [Preprint]. 2024 Jun 7:2024.06.07.24308439. doi: 10.1101/2024.06.07.24308439.PMID: 38883740 Free PMC article. Preprint.

The Epigenetic Evolution of Glioma Is Determined by the IDH1 Mutation Status and Treatment Regimen.

Malta TM, Sabedot TS, Morosini NS, Datta I, Garofano L, Vallentgoed W, Varn FS, Aldape K, D’Angelo F, Bakas S, Barnholtz-Sloan JS, Gan HK, Hasanain M, Hau AC, Johnson KC, Cazacu S, deCarvalho AC, Khasraw M, Kocakavuk E, Kouwenhoven MCM, Migliozzi S, Niclou SP, Niers JM, Ormond DR, Paek SH, Reifenberger G, Sillevis Smitt PA, Smits M, Stead LF, van den Bent MJ, Van Meir EG, Walenkamp A, Weiss T, Weller M, Westerman BA, Ylstra B, Wesseling P, Lasorella A, French PJ, Poisson LM; Consortium The GLASS; Verhaak RGW, Iavarone A, Noushmehr H.Cancer Res. 2024 Mar 4;84(5):741-756. doi: 10.1158/0008-5472.CAN-23-2093.PMID: 38117484 Free PMC article.

IDHwt glioblastomas can be stratified by their transcriptional response to standard treatment, with implications for targeted therapy.

Tanner G, Barrow R, Ajaib S, Al-Jabri M, Ahmed N, Pollock S, Finetti M, Rippaus N, Bruns AF, Syed K, Poulter JA, Matthews L, Hughes T, Wilson E, Johnson C, Varn FS, Brüning-Richardson A, Hogg C, Droop A, Gusnanto A, Care MA, Cutillo L, Westhead DR, Short SC, Jenkinson MD, Brodbelt A, Chakrabarty A, Ismail A, Verhaak RGW, Stead LF.Genome Biol. 2024 Feb 7;25(1):45. doi: 10.1186/s13059-024-03172-3.PMID: 38326875 Free PMC article.

Longitudinal characteristics of T2-FLAIR mismatch in IDH-mutant astrocytomas: Relation to grade, histopathology, and overall survival in the GLASS-NL cohort.

van Garderen KA, Vallentgoed WR, Lavrova A, Niers JM, de Leng WWJ, Hoogstrate Y, de Heer I, Ylstra B, van Dijk E, Klein S, Draaisma K, Robe PAJT, Verhaak RGW, Westerman BA, French PJ, van den Bent MJ, Kouwenhoven MCM, Kros JM, Wesseling P, Smits M.Neurooncol Adv. 2023 Nov 9;5(1):vdad149. doi: 10.1093/noajnl/vdad149. eCollection 2023 Jan-Dec.PMID: 38024241 Free PMC article.

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