(A) BPDCN cell lines CAL-1 and GEN 2.2 (n= 9) and (B) primary cells (12 patients) are sensitive to SL-401 (2.7 fM) treatment (18 h) in vitro. **p<0.001. (C) BPDCN cells were cultured alone or with SL-401 (2.7 fM) or ERW (erwinase, 10 UI/ml), KID (kidrolase, 10UI/ml), MTX (methotrexate, 4.5µg/ml), CYC (cyclophosphamide, 100 µmM), CYT (cytarabine, 80 µg/ml), DEX (dexamethasone, 250 µg/ml), VIN (vincristine, 20 ng/ml), or IDA (idarubicin, 0.078 µg/ml). The viability was evaluated by cytometry using AV/7AAD staining after 18 h of culture. Results (mean + SEM) of 4 experiments.*p<0.05. (D) Survival rate of 2 Gy irradiated NSG mice injected IV with 1 x 106 GEN 2.2 cells on day 0. SL-401 treatment IP (2 µg) daily for 5 days (pink area) was initiated on day 7. The solid and dotted lines show saline-treated controls (n = 3) and SL-401 treated animals (SL-401, n=4), respectively. Results from one representative experiment out of 3. (E) GEN 2.2 cells detected in a node 2 weeks post-injection. Cells were stained with standard MGG solution and show a blastic morphology.
Frankel:Stemline Therapeutics: Patents & Royalties, Research Funding. Brooks:Stemline Therapeutics: Employment, Equity Ownership. Rowinsky:Stemline Therapeutics: Employment, Equity Ownership.
Asterisk with author names denotes non-ASH members.