Structure-Activity-Relationship Exploration and Animal Validation of Novel Assembly Modulator Small Molecule Chemical Series with Pan-Cancer Selective Cytotoxicity

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Gepubliceerd in:bioRxiv (Mar 4, 2025)
Hoofdauteur: Lingappa, Anuradha F
Andere auteurs: Mallesh, Suguna, Michon, Maya, Devaraju Shivarudraswamy, Lin, Jim, Prasad, M Dharma, Dey, Debendranath, Solas, Dennis, Lingappa, Vishwanath R
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Cold Spring Harbor Laboratory Press
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LEADER 00000nab a2200000uu 4500
001 3173595158
003 UK-CbPIL
022 |a 2692-8205 
024 7 |a 10.1101/2025.02.28.640839  |2 doi 
035 |a 3173595158 
045 0 |b d20250304 
100 1 |a Lingappa, Anuradha F 
245 1 |a Structure-Activity-Relationship Exploration and Animal Validation of Novel Assembly Modulator Small Molecule Chemical Series with Pan-Cancer Selective Cytotoxicity 
260 |b Cold Spring Harbor Laboratory Press  |c Mar 4, 2025 
513 |a Working Paper 
520 3 |a A novel class of protein assembly modulator chemical compounds that exhibit broad anti-tumor cytotoxicity has been recently described. Here we present a more detailed structure-activity-relationship exploration of the chemical series. PAV-0805, an advanced analog, was found to be safe in mice at 10mg/kg and nontoxic to normal human peripheral blood mononuclear cells (PBMC) cells. PAV-0805 has demonstrated broad anti-cancer activity in the NCI-60 cell lines, including against a diversity of brain, breast, colon, lung, ovarian prostate, renal, and skin cancers, and leukemias. In the aggressive 4T1 mouse allograft breast cancer model, PAV-0805 was effective in reducing tumor growth and metastasis when treatment was initiated early (primary tumor volume of 50mm3) or late (primary tumor volume of 500mm3), and under both treatment conditions showed inhibition of both tumor growth and metastasis comparable to paclitaxel. The novel multi-protein complex targeted by these compounds is enriched in gene products involved in the cancer hallmarks of resisting cell death and reprogramming energy metabolism.Competing Interest StatementVRL is CEO and AFL COO of Prosetta Biosciences, Inc. 
653 |a Cancer 
653 |a Cytotoxicity 
653 |a Cell death 
653 |a Tumors 
653 |a Metastasis 
653 |a Peripheral blood mononuclear cells 
653 |a Energy metabolism 
653 |a Metastases 
653 |a Leukocytes (mononuclear) 
653 |a Antitumor activity 
653 |a Paclitaxel 
700 1 |a Mallesh, Suguna 
700 1 |a Michon, Maya 
700 1 |a Devaraju Shivarudraswamy 
700 1 |a Lin, Jim 
700 1 |a Prasad, M Dharma 
700 1 |a Dey, Debendranath 
700 1 |a Solas, Dennis 
700 1 |a Lingappa, Vishwanath R 
773 0 |t bioRxiv  |g (Mar 4, 2025) 
786 0 |d ProQuest  |t Biological Science Database 
856 4 1 |3 Citation/Abstract  |u https://www.proquest.com/docview/3173595158/abstract/embedded/BH75TPHOCCPB476R?source=fedsrch 
856 4 0 |3 Full text outside of ProQuest  |u https://www.biorxiv.org/content/10.1101/2025.02.28.640839v1