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Success Stories


Publications
A High-Throughput Screening Platform Targeting PDLIM5 for Pulmonary Hypertension

Journal of Biomolecular Screening

Cheng H, Chen T, Tor M, Park D, Zhou Q, Huang JB, Khatib N, Rong L, Zhou G
Journal of Biomolecular Screening - vol. 21 333-341 (2016)

Pulmonary arterial hypertension is a complex disease with multiple etiologic factors. PDLIM5, a member of the Enigma subfamily of PDZ and LIM domain protein family, contains an N-terminal PDZ domain and three LIM domains at its C-terminus. We have previously shown that overexpression of PDLIM5 prevents hypoxia-induced pulmonary hypertension (PH), and deletion of PDLIM5 in […]

Publications
Repositioning « old » drugs for new causes: identifying new inhibitors of prostate cancer cell migration and invasion.

Clinical & experimental metastasis

Shah ET, Upadhyaya A, Philp LK, Tang T, Skalamera D, Gunter J, Nelson CC, Williams ED, Hollier BG
Clinical & experimental metastasis - vol. 33 385-399 (2016)

The majority of prostate cancer (PCa) deaths occur due to the metastatic spread of tumor cells to distant organs. Currently, there is a lack of effective therapies once tumor cells have spread outside the prostate. It is therefore imperative to rapidly develop therapeutics to inhibit the metastatic spread of tumor cells. Gain of cell motility […]

Publications
Benzbromarone, Quercetin, and Folic Acid Inhibit Amylin Aggregation.

International journal of molecular sciences

López LC, Varea O, Navarro S, Carrodeguas JA, Sanchez de Groot N, Ventura S, Sancho J
International journal of molecular sciences - vol. 17 (2016)

Human Amylin, or islet amyloid polypeptide (hIAPP), is a small hormone secreted by pancreatic β-cells that forms aggregates under insulin deficiency metabolic conditions, and it constitutes a pathological hallmark of type II diabetes mellitus. In type II diabetes patients, amylin is abnormally increased, self-assembled into amyloid aggregates, and ultimately contributes to the apoptotic death of […]

Publications
A Yeast/Drosophila Screen to Identify New Compounds Overcoming Frataxin Deficiency

Oxidative Medicine and Cellular Longevity

Seguin A, Monnier VV, Palandri A, Bihel FF, Rera M, Schmitt M, Camadro JM, Tricoire HH, Lesuisse E
Oxidative Medicine and Cellular Longevity - vol. ID 565140 1-11 (2015)

Friedreich’s ataxia (FA) is a rare neurodegenerative disease which is very debilitating for the patients who progressively lose their autonomy. The lack of efficient therapeutic treatment of the disease strongly argues for urgent need to search for new active compounds that may stop the progression of the disease or prevent the appearance of the symptoms […]

Publications
Deriving human ENS lineages for cell therapy and drug discovery in Hirschsprung disease.

Nature

Fattahi F, Steinbeck JA, Kriks S, Tchieu J, Zimmer B, Kishinevsky S, Zeltner N, Mica Y, El-Nachef W, Zhao H, de Stanchina E, Gershon MD, Grikscheit TC, Chen S, Studer L
Nature - vol. 531 105-9 (2016)

The enteric nervous system (ENS) is the largest component of the autonomic nervous system, with neuron numbers surpassing those present in the spinal cord. The ENS has been called the ‘second brain’ given its autonomy, remarkable neurotransmitter diversity and complex cytoarchitecture. Defects in ENS development are responsible for many human disorders including Hirschsprung disease (HSCR). […]

Publications
Chemical library screening and structure-function relationship studies identify bisacodyl as a potent and selective cytotoxic agent towards quiescent human glioblastoma tumor stem-like cells

PLoS ONE

Zeniou M, F??ve M, Mameri S, Dong J, Salom?? C, Chen W, El-Habr EA, Bousson F, Sy M, Obszynski J, Boh A, Villa P, Kahn SA, Didier B, Bagnard D, Junier MP, Chneiweiss H, Haiech J, Hibert M, Kilhoffer MC, Heeschen C
PLoS ONE 1-35 (2015)

Cancer stem-like cells reside in hypoxic and slightly acidic tumor niches. Such microenvironments favor more aggressive undifferentiated phenotypes and a slow growing « quiescent state » which preserves them from chemotherapeutic agents that essentially target proliferating cells. Our objective was to identify compounds active on glioblastoma stem-like cells, including under conditions that mimick those found in vivo […]

Publications
Development of a Cell-Based Fluorescence Polarization Biosensor Using Preproinsulin to Identify Compounds That Alter Insulin Granule Dynamics.

Assay and drug development technologies

Yi NY, He Q, Caligan TB, Smith GR, Forsberg LJ, Brenman JE, Sexton JZ
Assay and drug development technologies - vol. 13 558-569 (2015)

Diabetes currently affects 9.3% of the U.S. population totaling $245 billion annually in U.S. direct and indirect healthcare costs. Current therapies for diabetes are limited in their ability to control blood glucose and/or enhance insulin sensitivity. Therefore, innovative and efficacious therapies for diabetes are urgently needed. Herein we describe a fluorescent insulin reporter system (preproinsulin-mCherry, […]

Publications
Novel activities of select NSAID renantiomers against Rac1 and Cdc42 GTPases

PLoS ONE

Oprea TI, Sklar LA, Agola JO, Guo Y, Silberberg M, Roxby J, Vestling A, Romero E, Surviladze Z, Murray-Krezan C, Waller A, Ursu O, Hudson LG, Wandinger-Ness A
PLoS ONE - vol. 10 1-32 (2015)

Rho family GTPases (including Rac, Rho and Cdc42) collectively control cell proliferation, adhesion and migration and are of interest as functional therapeutic targets in numerous epithelial cancers. Based on high throughput screening of the Prestwick Chemical Library® and cheminformatics we identified the R-enantiomers of two approved drugs (naproxen and ketorolac) as inhibitors of Rac1 and […]

Publications
A chemical screen to identify inducers of the mitochondrial unfolded protein response in C. elegans

Worm

Rauthan M, Pilon M
Worm - vol. 4 e1096490 (2015)

Publications
Clobetasol and halcinonide act as smoothened agonists to promote myelin gene expression and RxRγ receptor activation

PLoS ONE

Porcu G, Serone E, De Nardis V, Di Giandomenico D, Lucisano G, Scardapane M, Poma A, Ragnini-Wilson A
PLoS ONE - vol. 10 1-22 (2015)

One of the causes of permanent disability in chronic multiple sclerosis patients is the inability of oligodendrocyte progenitor cells (OPCs) to terminate their maturation program at lesions. To identify key regulators of myelin gene expression acting at the last stages of OPC maturation we developed a drug repositioning strategy based on the mouse immortalized oligodendrocyte […]