Small Molecule Publications
Acknowledging ICCB-L in Publications
Researchers using the services of ICCB-L staff or equipment agree to acknowledge the support of 'ICCB-Longwood Screening Facility at Harvard Medical School' in publications arising from your use of the facility. If an ICCB-L member has made an important intellectual contribution to the project, as determined by generally accepted criteria for academic collaborations, then that person should be considered for authorship on the publication. If your project was supported by a grant that also directly supports the ICCB-L, please consult the ICCB-L Director regarding the appropriate grant citation. Please notify Jen Smith upon public release of your publication.
Below is a list of publications that have resulted from small molecule screening efforts at ICCB-Longwood. Please notify us of your publications that involve use of the ICCB-Longwood Screening Facility.
2026
Petrova V, Mills CE, Hug C, Cetinkaya-Fisgin A, Splaine J, Fouladzadeh S, Hakim S, Powell R, Zhen S, Chung M, Bradshaw GA, Deng T, Singec I, Wang Q, Kawaguchi R, Jonnagaddala H, Barrett LB, Smith JA, Kalocsay M, Gyori BM, Hoke A, Sorger PK, Woolf CJ. A human iPSC-derived sensory neuron platform for high-throughput discovery of neuroprotectants against chemotherapy-induced peripheral neuropathy.
Cell Reports Medicine. Elsevier; 2026;
Petrova V, Mills CE, Hug C, Cetinkaya-Fisgin A, Splaine J, Fouladzadeh S, Hakim S, Powell R, Zhen S, Chung M, Bradshaw GA, Deng T, Singec I, Wang Q, Kawaguchi R, Jonnagaddala H, Barrett LB, Smith JA, Kalocsay M, Gyori BM, Hoke A, Sorger PK, Woolf CJ. A human iPSC-derived sensory neuron platform for high-throughput discovery of neuroprotectants against chemotherapy-induced peripheral neuropathy.
Cell Reports Medicine. Elsevier; 2026;
2025
Bharath N, DiPietro E, Durfee O, Kycia I, Splaine J, Sethupathy P, Rogers MS, Vakili K. A novel high-throughput screening platform to identify inhibitors of DNAJB1-PRKACA-driven transcriptional activity in fibrolamellar carcinoma.
SLAS discovery : advancing life sciences R & D. 2025;32:100221. PMID: 39947627
Bharath N, DiPietro E, Durfee O, Kycia I, Splaine J, Sethupathy P, Rogers MS, Vakili K. A novel high-throughput screening platform to identify inhibitors of DNAJB1-PRKACA-driven transcriptional activity in fibrolamellar carcinoma.
SLAS discovery : advancing life sciences R & D. 2025;32:100221. PMID: 39947627
Law JD, Gao Y, Kovvali S, Thirugnanasambantham P, Wysocki VH, Ahmer BMM, Gopalan V. Identification of inhibitors of the Salmonella FraB deglycase, a drug target.
FEBS open bio. 2025; PMID: 39945759
Law JD, Gao Y, Kovvali S, Thirugnanasambantham P, Wysocki VH, Ahmer BMM, Gopalan V. Identification of inhibitors of the Salmonella FraB deglycase, a drug target.
FEBS open bio. 2025; PMID: 39945759
Quek RT, Shirazinejad CR, Young CL, Hardy KS, Lim S, Elms PJ, McSwiggen DT, Mitchison TJ, Silver PA. Comparative evaluation of cell-based assay technologies for scoring drug-induced condensation of SARS-CoV-2 nucleocapsid protein.
SLAS discovery : advancing life sciences R & D. 2025;31:100220. PMID: 39894078
Quek RT, Shirazinejad CR, Young CL, Hardy KS, Lim S, Elms PJ, McSwiggen DT, Mitchison TJ, Silver PA. Comparative evaluation of cell-based assay technologies for scoring drug-induced condensation of SARS-CoV-2 nucleocapsid protein.
SLAS discovery : advancing life sciences R & D. 2025;31:100220. PMID: 39894078
2024
Niu X, Melendez DL, Raj S, Cai J, Senadeera D, Mandelbaum J, Shestopalov IA, Martin SD, Zon LI, Schlaeger TM, Lai LP, McMahon AP, Craft AM, Galloway JL. A conserved transcription factor regulatory program promotes tendon fate.
Developmental cell. 2024;59(23):3106–3123.e12. PMID: 39216481
Niu X, Melendez DL, Raj S, Cai J, Senadeera D, Mandelbaum J, Shestopalov IA, Martin SD, Zon LI, Schlaeger TM, Lai LP, McMahon AP, Craft AM, Galloway JL. A conserved transcription factor regulatory program promotes tendon fate.
Developmental cell. 2024;59(23):3106–3123.e12. PMID: 39216481
Kassu M, Zulauf KE, Ross JN, Kirby JE, Manetsch R. Development of CGS-15943 Adjunctives for the Disruption of Plasmid Maintenance in Multidrug Resistant .
ACS infectious diseases. 2024; PMID: 39741382
Kassu M, Zulauf KE, Ross JN, Kirby JE, Manetsch R. Development of CGS-15943 Adjunctives for the Disruption of Plasmid Maintenance in Multidrug Resistant .
ACS infectious diseases. 2024; PMID: 39741382
Hammel F, Payne C, Marando V, Mazitschek R, Walker S. Identification of a Polypeptide Inhibitor of -GlcNAc Transferase with Picomolar Affinity.
Journal of the American Chemical Society. 2024;146(38):26320–26330. PMCID: PMC11440498
Hammel F, Payne C, Marando V, Mazitschek R, Walker S. Identification of a Polypeptide Inhibitor of -GlcNAc Transferase with Picomolar Affinity.
Journal of the American Chemical Society. 2024;146(38):26320–26330. PMCID: PMC11440498
Petrova V, Snavely AR, Splaine J, Zhen S, Singh B, Pandey R, Chen K, Cheng A, Hermawan C, Barrett LB, Smith JA, Woolf CJ. Identification of novel neuroprotectants against vincristine-induced neurotoxicity in iPSC-derived neurons.
Cellular and molecular life sciences : CMLS. 2024;81(1):315. PMCID: 11335239
Petrova V, Snavely AR, Splaine J, Zhen S, Singh B, Pandey R, Chen K, Cheng A, Hermawan C, Barrett LB, Smith JA, Woolf CJ. Identification of novel neuroprotectants against vincristine-induced neurotoxicity in iPSC-derived neurons.
Cellular and molecular life sciences : CMLS. 2024;81(1):315. PMCID: 11335239
Shehadeh F, Felix L, Kalligeros M, Shehadeh A, Fuchs BB, Ausubel FM, Sotiriadis PP, Mylonakis E. Machine learning-assisted high-throughput screening for Anti-MRSA compounds.
IEEE/ACM transactions on computational biology and bioinformatics. 2024;PP. PMID: 39058605
Shehadeh F, Felix L, Kalligeros M, Shehadeh A, Fuchs BB, Ausubel FM, Sotiriadis PP, Mylonakis E. Machine learning-assisted high-throughput screening for Anti-MRSA compounds.
IEEE/ACM transactions on computational biology and bioinformatics. 2024;PP. PMID: 39058605
Rawat V, DeLear P, Prashanth P, Ozgurses ME, Tebeje A, Burns PA, Conger KO, Solís C, Hasnain Y, Novikova A, Endress JE, González-Sánchez P, Dong W, Stephanopoulos G, DeNicola GM, Harris IS, Sept D, Mason FM, Coloff JL. Drug screening in human physiologic medium identifies uric acid as an inhibitor of rigosertib efficacy.
JCI insight. 2024;9(13). PMID: 38815134
Rawat V, DeLear P, Prashanth P, Ozgurses ME, Tebeje A, Burns PA, Conger KO, Solís C, Hasnain Y, Novikova A, Endress JE, González-Sánchez P, Dong W, Stephanopoulos G, DeNicola GM, Harris IS, Sept D, Mason FM, Coloff JL. Drug screening in human physiologic medium identifies uric acid as an inhibitor of rigosertib efficacy.
JCI insight. 2024;9(13). PMID: 38815134
Issa H, Fléchon L, Laine W, Ouelkdite A, Gaggero S, Cozzani A, Tilmont R, Chauvet P, Gower N, Sklavenitis-Pistofidis R, Brinster C, Thuru X, Touil Y, Quesnel B, Mitra S, Ghobrial I, Kluza J, Manier S. MYC dependency in GLS1 and NAMPT is a therapeutic vulnerability in multiple myeloma.
iScience. 2024;27(4):109417.
Issa H, Fléchon L, Laine W, Ouelkdite A, Gaggero S, Cozzani A, Tilmont R, Chauvet P, Gower N, Sklavenitis-Pistofidis R, Brinster C, Thuru X, Touil Y, Quesnel B, Mitra S, Ghobrial I, Kluza J, Manier S. MYC dependency in GLS1 and NAMPT is a therapeutic vulnerability in multiple myeloma.
iScience. 2024;27(4):109417.
Deblais L, Drozd M, Kumar A, Antwi J, Fuchs J, Khupse R, Helmy Y, Rajashekara G. Identification of novel small molecule inhibitors of twin arginine translocation (Tat) pathway and their effect on the control of Campylobacter jejuni in chickens.
Front. Microbiol. 2024;(16 April). PMCID: PMC11061419
Deblais L, Drozd M, Kumar A, Antwi J, Fuchs J, Khupse R, Helmy Y, Rajashekara G. Identification of novel small molecule inhibitors of twin arginine translocation (Tat) pathway and their effect on the control of Campylobacter jejuni in chickens.
Front. Microbiol. 2024;(16 April). PMCID: PMC11061419
Soni D, Borriello F, Scott D, Feru F, DeLeon M, Brightman S, Cheng W, Melhem G, Smith J, Ramirez J, Barman S, Cameron M, Kelly A, Walker K, Nanishi E, vanHaren S, Phan T, Qi Y, Kinsey R, Raczy M, Ozonoff A, Pettengill M, Hubbell J, Fox C, Dowling D, Levy O. From hit to vial: Precision discovery and development of an imidazopyrimidine TLR7/8 agonist adjuvant formulation.
Sci Adv. 2024;10(27):eadg3747.
Soni D, Borriello F, Scott D, Feru F, DeLeon M, Brightman S, Cheng W, Melhem G, Smith J, Ramirez J, Barman S, Cameron M, Kelly A, Walker K, Nanishi E, vanHaren S, Phan T, Qi Y, Kinsey R, Raczy M, Ozonoff A, Pettengill M, Hubbell J, Fox C, Dowling D, Levy O. From hit to vial: Precision discovery and development of an imidazopyrimidine TLR7/8 agonist adjuvant formulation.
Sci Adv. 2024;10(27):eadg3747.
Shamsabadi M, Jia X. A fluorescence polarization assay for high-throughput screening of inhibitors against HIV-1 Nef-mediated CD4 downregulation.
J Biol Chem. 2024;300(8):107528.
Shamsabadi M, Jia X. A fluorescence polarization assay for high-throughput screening of inhibitors against HIV-1 Nef-mediated CD4 downregulation.
J Biol Chem. 2024;300(8):107528.
Chen Y, Chaudhari S, Harris D, Roberts C, Moscalu A, Mathur V, Zhao L, Tavakkoli, Devlin A, Sheu E. A small intestinal bile acid modulates the gut microbiome to improve host metabolic phenotypes following bariatric surgery.
Cell Host Microbe. 2024;17.
Chen Y, Chaudhari S, Harris D, Roberts C, Moscalu A, Mathur V, Zhao L, Tavakkoli, Devlin A, Sheu E. A small intestinal bile acid modulates the gut microbiome to improve host metabolic phenotypes following bariatric surgery.
Cell Host Microbe. 2024;17.
Do B, Hsu P, Vermeulen S, Wang Z, Hirz T, Abbott K, Aziz N, Replogle J, Bjelosevic S, Paolino J, Nelson S, Block S, Darnell A, Ferreira R, Zhang, Milosevic J, Schmidt D, Chidley C, Harris I, Weissman J, Pikman Y, Stegmaier K, Cheloufi S, Su X, Syke D, Vander-Heiden M. Nucleotide depletion promotes cell fate transitions by inducing DNA replication stress.
Dev Cell. 2024;May.
Do B, Hsu P, Vermeulen S, Wang Z, Hirz T, Abbott K, Aziz N, Replogle J, Bjelosevic S, Paolino J, Nelson S, Block S, Darnell A, Ferreira R, Zhang, Milosevic J, Schmidt D, Chidley C, Harris I, Weissman J, Pikman Y, Stegmaier K, Cheloufi S, Su X, Syke D, Vander-Heiden M. Nucleotide depletion promotes cell fate transitions by inducing DNA replication stress.
Dev Cell. 2024;May.
Elfawal M, Goetz E, Kim Y, Chen P, Savinov S, Barasa L, Thompson P, Aroian R. High-Throughput Screening of More Than 30,000 Compounds for Anthelmintics against Gastrointestinal Nematode Parasites.
ACS Infect Dis. 2024;(Dec 9). PMID: 39653369
Elfawal M, Goetz E, Kim Y, Chen P, Savinov S, Barasa L, Thompson P, Aroian R. High-Throughput Screening of More Than 30,000 Compounds for Anthelmintics against Gastrointestinal Nematode Parasites.
ACS Infect Dis. 2024;(Dec 9). PMID: 39653369
Fontana P, Du G, Zhang Y, Zhang H, Vora S, Hu J, Shi M, Tufan A, Healy L, Xia S, Lee DJ, Li Z, Baldominos P, Ru H, Luo H, Agudo J, Lieberman J, Wu H. Small-molecule GSDMD agonism in tumors stimulates antitumor immunity without toxicity.
Cell. 2024;187(22):6165–6181. PMID: 39243763
Fontana P, Du G, Zhang Y, Zhang H, Vora S, Hu J, Shi M, Tufan A, Healy L, Xia S, Lee DJ, Li Z, Baldominos P, Ru H, Luo H, Agudo J, Lieberman J, Wu H. Small-molecule GSDMD agonism in tumors stimulates antitumor immunity without toxicity.
Cell. 2024;187(22):6165–6181. PMID: 39243763
Saffari A, Brechmann B, Böger C, Saber W, Jumo H, Whye D, Wood D, Wahlster L, Alecu J, Ziegler M, Scheffold M, Winden K, Hubbs J, Buttermore E, Barrett, Borner G, Davies A, Ebrahimi-Fakhari D, Sahin M. High-content screening identifies a small molecule that restores AP-4-dependent protein trafficking in neuronal models of AP-4-associated hereditary spastic paraplegia.
Nat Commun. 2024;15(1):584.
Saffari A, Brechmann B, Böger C, Saber W, Jumo H, Whye D, Wood D, Wahlster L, Alecu J, Ziegler M, Scheffold M, Winden K, Hubbs J, Buttermore E, Barrett, Borner G, Davies A, Ebrahimi-Fakhari D, Sahin M. High-content screening identifies a small molecule that restores AP-4-dependent protein trafficking in neuronal models of AP-4-associated hereditary spastic paraplegia.
Nat Commun. 2024;15(1):584.
2023
Ajay A, Chu P, Patel P, Deban C, Roychowdhury C, Heda R, Halawi A, Saad A, Younis N, Zhang, Jiang X, Nasr M, Hsiao LL, Lin G, Azzi J. High-Throughput/High Content Imaging Screen Identifies Novel Small Molecule Inhibitors and Immunoproteasomes as Therapeutic Targets for Chordoma.
Pharmaceutics. 2023;15(4):1274.
Ajay A, Chu P, Patel P, Deban C, Roychowdhury C, Heda R, Halawi A, Saad A, Younis N, Zhang, Jiang X, Nasr M, Hsiao LL, Lin G, Azzi J. High-Throughput/High Content Imaging Screen Identifies Novel Small Molecule Inhibitors and Immunoproteasomes as Therapeutic Targets for Chordoma.
Pharmaceutics. 2023;15(4):1274.
Nguyen L, Wei Z, Silva M, Barberán-Soler S, Zhang J, Rabinovsky R, Muratore C, Stricker J, Hortman C, Young-Pearse T, Haggarty S, Krichevsky A. Small molecule regulators of microRNAs identified by high-throughput screen coupled with high-throughput sequencing.
Nat Commun. 2023;14(1):7575.
Nguyen L, Wei Z, Silva M, Barberán-Soler S, Zhang J, Rabinovsky R, Muratore C, Stricker J, Hortman C, Young-Pearse T, Haggarty S, Krichevsky A. Small molecule regulators of microRNAs identified by high-throughput screen coupled with high-throughput sequencing.
Nat Commun. 2023;14(1):7575.
Ryan C, Brander D, Barr P, Tyekucheva S, Hackett L, Collins M, Fernandes S, Ren Y, Zhou Y, McDonough M, Walker H, McEwan M, Abramson J, Jacobsen E, LaCasce A, Fisher D, Brown J, Davids M. A phase 1b study of ibrutinib in combination with obinutuzumab in patients with relapsed or refractory chronic lymphocytic leukemia.
Leukemia. 2023;(Jan 30).
Ryan C, Brander D, Barr P, Tyekucheva S, Hackett L, Collins M, Fernandes S, Ren Y, Zhou Y, McDonough M, Walker H, McEwan M, Abramson J, Jacobsen E, LaCasce A, Fisher D, Brown J, Davids M. A phase 1b study of ibrutinib in combination with obinutuzumab in patients with relapsed or refractory chronic lymphocytic leukemia.
Leukemia. 2023;(Jan 30).
Mitchell D, Kuljanin M, Li J, VanVranken J, Bulloch N, Schweppe D, Huttlin E, Gygi S. A proteome-wide atlas of drug mechanism of action.
Nat Biotechnol. 2023;41(6):845–857.
Mitchell D, Kuljanin M, Li J, VanVranken J, Bulloch N, Schweppe D, Huttlin E, Gygi S. A proteome-wide atlas of drug mechanism of action.
Nat Biotechnol. 2023;41(6):845–857.
Chew K, Lee, Ozonoff A, Smith J, Levy O, Dowling D, Haren S. A protocol for high-throughput screening for immunomodulatory compounds using human primary cells.
STAR Protoc. 2023;4(3):102405.
Chew K, Lee, Ozonoff A, Smith J, Levy O, Dowling D, Haren S. A protocol for high-throughput screening for immunomodulatory compounds using human primary cells.
STAR Protoc. 2023;4(3):102405.
Kurmi, Liang D, vandeVen R, Georgiev P, Gassaway B, Han S, Notarangelo G, Harris I, Yao, Park J, Hu SH, Peng J, Drijvers J, Boswell S, Sokolov A, Dougan S, Sorger P, Gygi S, Sharpe A, Haigis M. Metabolic modulation of mitochondrial mass during CD4+ T cell activation.
Cell Chem Biol. 2023;30(9):1064–1075.
Kurmi, Liang D, vandeVen R, Georgiev P, Gassaway B, Han S, Notarangelo G, Harris I, Yao, Park J, Hu SH, Peng J, Drijvers J, Boswell S, Sokolov A, Dougan S, Sorger P, Gygi S, Sharpe A, Haigis M. Metabolic modulation of mitochondrial mass during CD4+ T cell activation.
Cell Chem Biol. 2023;30(9):1064–1075.
Du T, Song Y, Ray A, Wan X, Yao, Samur M, Shen C, Penailillo J, Sewastianik T, Tai YT, Fulciniti, Munshi N, Wu, Carrasco R, Chauhan D, Anderson K. Ubiquitin receptor PSMD4/Rpn10 is a novel therapeutic target in multiple myeloma.
Blood. 2023;(Jan 11).
Du T, Song Y, Ray A, Wan X, Yao, Samur M, Shen C, Penailillo J, Sewastianik T, Tai YT, Fulciniti, Munshi N, Wu, Carrasco R, Chauhan D, Anderson K. Ubiquitin receptor PSMD4/Rpn10 is a novel therapeutic target in multiple myeloma.
Blood. 2023;(Jan 11).
Yang JH, Petty C, Dixon-McDougall T, Lopez M, Tyshkovskiy A, Maybury-Lewis S, Tian X, Ibrahim N, Chen Z, Griffin P, Arnold M, Li J, Martinez O, Behn A, Rogers-Hammond R, Angeli S, Gladyshev V, Sinclair D. Chemically induced reprogramming to reverse cellular aging.
2023;15(13):5966–5989.
Yang JH, Petty C, Dixon-McDougall T, Lopez M, Tyshkovskiy A, Maybury-Lewis S, Tian X, Ibrahim N, Chen Z, Griffin P, Arnold M, Li J, Martinez O, Behn A, Rogers-Hammond R, Angeli S, Gladyshev V, Sinclair D. Chemically induced reprogramming to reverse cellular aging.
2023;15(13):5966–5989.
Potter D, Du R, Bohl S, Chow KH, Ligon K, Bueno R, Letai. Dynamic BH3 profiling identifies pro-apoptotic drug combinations for the treatment of malignant pleural mesothelioma.
Nat Commun. 2023;14(2897).
Potter D, Du R, Bohl S, Chow KH, Ligon K, Bueno R, Letai. Dynamic BH3 profiling identifies pro-apoptotic drug combinations for the treatment of malignant pleural mesothelioma.
Nat Commun. 2023;14(2897).
Ralvenius W, Mungenast A, Woolf H, Huston M, Gillingham T, Godin S, Penney J, Cam H, Gao F, Fernandez C, Czako B, Lightfoot Y, Ray W, Beckmann A, Goate A, Marcora E, Romero-Molina C, Ayata P, Schaefer A, Gjoneska E, Tsai LH. A novel molecular class that recruits HDAC/MECP2 complexes to PU.1 motifs reduces neuroinflammation.
J Exp Med. 2023;220(11):e20222105.
Ralvenius W, Mungenast A, Woolf H, Huston M, Gillingham T, Godin S, Penney J, Cam H, Gao F, Fernandez C, Czako B, Lightfoot Y, Ray W, Beckmann A, Goate A, Marcora E, Romero-Molina C, Ayata P, Schaefer A, Gjoneska E, Tsai LH. A novel molecular class that recruits HDAC/MECP2 complexes to PU.1 motifs reduces neuroinflammation.
J Exp Med. 2023;220(11):e20222105.
Quek R, Hardy K, Walker S, Nguyen D, Magalhães T, Salic, Gopalakrishnan S, Silver P, Mitchison T. Screen for Modulation of Nucleocapsid Protein Condensation Identifies Small Molecules with Anti-Coronavirus Activity.
ACS Chem Biol. 2023;18(3):583–594.
Quek R, Hardy K, Walker S, Nguyen D, Magalhães T, Salic, Gopalakrishnan S, Silver P, Mitchison T. Screen for Modulation of Nucleocapsid Protein Condensation Identifies Small Molecules with Anti-Coronavirus Activity.
ACS Chem Biol. 2023;18(3):583–594.