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Camptothecin-carrying bioadhesive nanoparticles for skin cancer

DISEASE CATEGORY: Cancer INDICATION: Skin cancer A Yale University team showed that bioadhesive nanoparticle delivery of topoisomerase inhibitor camptothecin could treat squamous cell carcinoma. The nanoparticle therapy was created by incubating non-adhesive hyperbranched polyglycerol-based nanoparticles...

PON-2 identified as B cell-ALL target

DISEASE CATEGORY: Cancer INDICATION: Acute lymphoblastic leukemia (ALL) Inhibiting PON2, a lactonase, could treat B cell ALL (B-ALL). Levels of PON2 mRNA were higher in Philadelphia chromosome-positive B-ALL cells from patients than in normal pre-B cells,...

Folate receptor-targeting CAR T for ovarian and other cancers

DISEASE CATEGORY: Cancer INDICATION: Ovarian cancer; melanoma; colorectal cancer CAR T cells targeting folate receptor FOLR2 could treat ovarian cancer, melanoma and colorectal cancer. Among patients with stage III or IV ovarian cancer, high FOLR2...

eIF4E identified as target in obesity, fatty liver disease

DISEASE CATEGORY: Endocrine/metabolic INDICATION: Obesity; liver disease Reducing levels of eIF4E or lowering the RNA cap-binding protein’s activity by blocking its phosphorylation could treat obesity...

A targeted CDK12 degrader for ALL

DISEASE CATEGORY: Cancer INDICATION: Acute lymphoblastic leukemia (ALL) A CDK12-targeted degrader, designed by a Dana-Farber Cancer Institute and Harvard Medical School team co-led by Nathanael Gray, could treat T cell ALL. The degrader’s targeting moiety was derived...

CXCR4 antagonist identified for hepatocellular carcinoma

DISEASE CATEGORY: Cancer INDICATION: Liver cancer An oxopropylaminoacetic acid-based CXCR4 antagonist could treat hepatocellular carcinoma (HCC). The CXCR4 inhibitor, optimized from a compound the...

Toxin-derived YBX1 inhibitor for ovarian cancer

DISEASE CATEGORY: Cancer INDICATION: Ovarian cancer A YBX1 inhibitor derived from azopodophyllotoxin could treat ovarian cancer. In a panel of five human ovarian cancer cell lines, the compound inhibited growth with IC50 values of 1.27-6.8...

RECK, an inhibitor of amyloid precursor protein cleavage, as a target for Alzheimer’s

DISEASE CATEGORY: Neurology INDICATION: Alzheimer’s disease Blocking RECK, a membrane-tethered inhibitor of APP-cleaving enzyme ADAM10, could treat Alzheimer’s disease. Levels of membrane-tethered...

A UTI vaccine that stimulates Th1 T cell responses

DISEASE CATEGORY: Infectious disease INDICATION: Urinary tract infection (UTI); Escherichia A vaccine containing uropathogenic E. coli antigens and an adjuvant that promotes Th1 immune responses could prevent UTIs. Mice immunized via intrabladder delivery of a lysate...

A neutralizing mAb against Junin virus for Argentine hemorrhagic fever

DISEASE CATEGORY: Infectious disease INDICATION: Viral hemorrhagic fever A mAb targeting Junin virus glycoprotein could treat infections by Junin virus, which causes Argentine hemorrhagic fever. In monkey kidney epithelial cells, a humanized mAb against Junin virus...

Herb-extracted compound triptonide identified as male contraceptive

DISEASE CATEGORY: Endocrine/metabolic INDICATION: Contraception Triptonide, a compound purified from Tripterygium Wilfordii Hook F, could serve as a non-hormonal male contraceptive. In vitro assays identified JUP as triptonide’s most likely target. In mice and cynomolgus monkeys, oral triptonide led to...

Targeting ferroptosis directly or via CD36 inhibition for melanoma, MM, NSCLC

DISEASE CATEGORY: Cancer INDICATION: Melanoma; multiple myeloma (MM); non-small cell lung cancer (NSCLC) Blocking ferroptosis directly or by inhibiting membrane glycoprotein CD36 could treat melanoma,...

Trastuzumab-RNA polymerase inhibitor conjugate for breast cancer with low HER2 expression

DISEASE CATEGORY: Cancer INDICATION: Breast cancer An antibody-drug conjugate comprising anti-HER2 antibody trastuzumab linked to POLR2A inhibitor α-amanitin could treat breast cancer with low HER2 expression and heterozygous loss of chromosome 17p. In a panel...

Sphingosine receptor agonist identified for cervical cancer, sarcoma

DISEASE CATEGORY: Cancer INDICATION: Cervical cancer; sarcoma A decahydroisoquinoline-based agonist of S1PR3, a sphingosine phosphate receptor, could treat cervical cancer and rhabdomyosarcoma. A pancreatic cancer cell-based screen of 696 compounds for differential suppression of proliferation in...

Antibody-drug conjugate targeting CLDN6 for liver cancer

DISEASE CATEGORY: Cancer INDICATION: Liver cancer An antibody-drug conjugate targeting the tight junction protein CLDN6 could treat hepatocellular carcinoma (HCC). In patients, high tumor levels of CLDN6 protein were associated with poor overall and disease-free...