crenolanib (ARO-002) / AROG - LARVOL DELTA

Profiling drug sensitivity in CLL B cells after BTK inhibitor progression using a novel drug panel (ASH 2025) - " Using the Mayo Clinic CLL Database and the Mayo Clinic CLL Tissue Bank, we identified 47 patients with RR CLL, all of whom experienced disease progressionon a BTKi (35 ibrutinib +/- CD20 antibody, 10 acalabrutinib +/- CD20 antibody, 2 other)...We also observed that RR CLL cohort exhibited significantly increased drug resistance to most of the tested drugs in the presence of BMSCs with corresponding increases in IC50s (with vs. without BMSCs, fold-change respectively): LP-118 3.92-fold, venetoclax 1.98-fold, carfilzomib 2.58-fold, TP-0903 1.65-fold, panobinostat 204.23-fold, TCIP1 1.91-fold, crenolanib 1.31-fold, idasanutlin 1.69-fold, belinostat 6.14-fold, LP-168 1.29-fold, eprenetapopt 3.44-fold and GTE 1.08-fold (11 out of 12 with p -values <0.05 except for GTE which was not significant, Mann-Whitney U test)... Our results highlight the spectrum of currently available drugs that show promise for therapeutic use in patients with RR CLL who experience disease..."

IO biomarker • Chronic Lymphocytic Leukemia • Hematological Malignancies • Leukemia • AXL • BCL2 • STAT6 • TP53

Comparative safety and efficacy of first- and second-generation FLT3 inhibitors in newly diagnosed and relapsed AML: A meta-analysis stratified by molecular biomarkers (ASH 2025) - "First-generation (sorafenib and midostaurin) and second-generation (gilteritinib, quizartinib, and crenolanib) FLT inhibitors demonstrated efficacy and safety across disease stages and are now used as a standard of treatment for FLT3-mutated AML. Conclusions Second-generation FLT inhibitors exhibit enhanced efficacy in relapsed-refractory AML scenarios and comparable outcomes to first-generation inhibitors in newly diagnosed AML.Stratification using ITD allelic ratio and NPM1 co-mutation is emerging as a critical factor. In light of these findings, it's imperative to integrate second-generation inhibitors in salvage settings and Biomarker-guided treatment decisions in front-line FLT3 mutant-AML"

Biomarker • Retrospective data • Acute Myelogenous Leukemia • Cardiovascular • Hematological Disorders • Hypertension • Neutropenia • FLT3 • NPM1

Unveiling FLT3 in B-ALL: Molecular drivers and targeted treatment opportunities (ASH 2025) - "In vitro assays withincreasing concentrations of 6 FLT3i for 24, 48, 72h [Gilteritinib (Gil), Midostaurin, Crenolanib, Sorafenib,Quizartinib, Ponatinib (Pon)] and Venetoclax (Ven) were performed on primary patient samples (n=29; FLT3-mut n=5/29), 11 B-ALL wt cell lines, 2 B-ALL mut (NALM6-mut, KASUMI-10), and 4 AML cell lines (OCI-AML3 wt; MV-4-11, MOLM-13, MONO-MAC6 FLT3-mut). FLT3 alterations define a novel Ph-negative B-ALL subgroup withtherapeutic relevance. Given the responses in FLT3-wt in addition to the mutated models, FLT3i may alsobenefit patients without FLT3 muts. Thanks to: Ricerca Corrente by the Italian MoH L3P1946 andAILTreviso."

Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • T Acute Lymphoblastic Leukemia • FLT3 • IKZF1 • KMT2A • PBX1 • TCF3 • TP53 • ZNF384

Crenolanib plus salvage chemotherapy improves outcomes in FLT3-mutant and NPM1 co-mutated relapsed/ refractory (R/R) Acute Myeloid Leukemia (AML): Results from a randomized, placebo-controlled, double-blind trial (ASH 2025) - P3 | "2017-001600-29).Materials and Fit adults with R/R FLT3m AML were randomized 1:1 to salvage induction chemo (FLAG-IDA/HAM) +crenolanib (100 mg TID) or placebo, followed by intermediate-dose cytarabine consolidation + crenolanibor placebo, and (if eligible, hematopoietic stem cell transplantation (HSCT)) followed by crenolanib orplacebo monotherapy for up to 1 year. In this randomized, placebo-controlled, double-blind study, the addition of crenolanib to salvagechemotherapy met its primary endpoint, demonstrating a statistically significant improvement in EFS aswell as an improved CR/CRi rate and a trend toward improved OS over chemotherapy alone in 106 fitadult pts with heavily pre-treated R/R FLT3m AML. The benefit was more pronounced in pts withNPM1/FLT3 co-mutated disease (n=59), with statistically significant improvement seen in not onlyremission rate and EFS but in overall survival as well. This data supports the further development ofcrenolanib in adults with..."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Gastrointestinal Disorder • Hematological Malignancies • Infectious Disease • Leukemia • Pneumonia • Respiratory Diseases • Septic Shock • FLT3 • NPM1

Determining sensitivity to FLT3 inhibitors prior to therapy in FLT3 mutant acute myelogenous leukemia (ASH 2025) - "For ex-vivo sensitivity assays, AML cells were treated with several dilutions of a FLT3i(Sorafenib, Midostaurin, Crenolanib, Quizartinib, Gilteritinib, Tuspetinib and MAX-40279 (a dualFLT3/FGFR inhibitor)) and cell viability was assessed with CellTiter-Glo® (Promega). Using our MS-based proteomics measurements and sensitivity results with our proprietary MLmodel, we processed both AML patient samples (N=57) and patient-derived cell lines (N=59), and wewere able to identify 7 distinct clusters. Previously we demonstrated that RPPA proteomics could discriminate FLT3i sensitive andresistant cases and here we present orthogonal confirmation by MS proteomics and ex-vivo sensitivityassays. Moreover, we show that the expression of only three proteins forms a robust biomarker topredict FLT3i sensitivity of FLT3-MUT AML patients prior to therapy. We also identified AML cell lines thatmimic both FLT3i resistance and sensitivity, and combined with deep proteomics data, these..."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • CD34 • FLT3 • PXN • SMARCA2

Cladribine, idarubicin, and AraC (CLIA) combined with a FLT3 inhibitor in patients with newly diagnosed FLT3-mutated Acute Myeloid Leukemia: A pooled analysis of prospective trials (ASH 2025) - "Introduction The incorporation of FLT3 inhibitors, cladribine, and high-dose cytarabine have been shownto improve outcomes in pts with newly diagnosed FLT3-mutated AML...We present a pooled analysis of frontline trials ofintensive chemotherapy (IC) with CLIA in combination with FLT3 inhibitors, focused on outcomes by co-occurring mutations.Methods Patients (pts) 18-65 years with newly diagnosed FLT3-mutated AML, fit for IC, were enrolled onprotocol with CLIA (cladribine 5 mg/m2 IV D1-5, araC 1.5-2 g/m2 IV D1-5, idarubicin 10 mg/m2 IV D1-3) plusgilteritinib (gilt) or sorafenib (soraf)...31 pts (78%) underwent allogeneic stem cell transplant (SCT) in CR1 and 19 pts (61%) receivedpost-SCT maintenance: 8 soraf, 6 gilt, 4 crenolanib, 1 AZA/VEN.After a 68-month median follow-up for all pts, median OS was not reached (NR), with 2- and 4-year OS of67% and 62%...The mostcommon AE for CLIA/gilt were elevated ALT/AST (n=17), febrile neutropenia (n=11), and rash (n=11); themost..."

Retrospective data • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • ASXL1 • DNMT3A • FLT3 • IDH1 • IDH2 • KRAS • NPM1 • RUNX1 • SF3B1 • SRSF2 • TET2 • U2AF1 • WT1

Negative feedback regulation of nfkb, MAPK, and JAK-STAT drives adaptive resistance to gilteritinib in AML (ASH 2025) - "Patients treated with highly selective inhibitors such asgilteritinib, crenolanib, and quizartinib usually relapse within eight to twelve months. These findings explain how elevated AP1 and NF-κB/JAK-STAT activitiestranscriptionally induce negative regulators to favor cellular survival, as unchecked MAPK signaling wouldotherwise trigger apoptosis. Collectively, these data strongly support the rationale for evaluating thecombination of gilteritinib and venetoclax with momelotinib as a therapeutic strategy to achieve effectiveand durable responses in AML."

IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • FLT3 • IRAK1 • MAPK8 • STAT5 • TET2

MODULE 3: Role of FLT3 Inhibitors in AML Management (ASH 2024) - "This program is supported by educational grants from AbbVie Inc, Astellas, and Daiichi Sankyo Inc.Pharmacologic similarities and differences among available FLT3 inhibitors for AML; implications for potency, activity and tolerability Principal findings from the Phase III QuANTUM-First study evaluating the addition of quizartinib to chemotherapy and its continuation as a single agent for patients with newly diagnosed AML with a FLT3-ITD mutation FDA approval of quizartinib for previously untreated AML with a FLT3-ITD mutation; clinical role Long-term outcomes reported with gilteritinib versus salvage chemotherapy for patients with relapsed/refractory (R/R) AML with a FLT3 mutation; optimal integration into routine practice Early data with gilteritinib combined with standard intensive chemotherapy or venetoclax-based therapy for AML with a FLT3 mutation Available data with and ongoing investigations of other novel FLT3 inhibitors, such as crenolanib and BMF-500, for AML..."

Acute Myelogenous Leukemia • FLT3

Optimizing FLT3 Inhibitor Use in Adult Acute Myeloid Leukemia with FLT3 Mutations Using Proteomics (ASH 2023) - "Of 137 with FLT3 ITD or D835 mutations, 54 who were treated with FLT3I (Sorafenib=22, Quizartinib=11, Midostaurine=6, Gilteritinib=4 and Crenolanib=1) combined with other agents (Hypomethylating agents (HMA)=23, HMA+Venetoclax (VTX)=5, Idarrubicin+Cytarabine (AraC)+Purine Analogs (IA+Pur)=19, IA+Pur+VTX=2, AraC=3, Fludarabine+AraC (FA)=1, none=1) were selected for analyses. Protein profiling identified cohorts of FLT3 mutant patients that achieved the highest benefit from FLT3I addition, regardless of all other clinical or molecular characteristics. We identified targetable proteins that are elevated in adverse signatures, and hypothesized that these may be targets for modulation to improve FLT3I response. Moreover, a small set of 6 proteins accurately classifies patients and could be used clinically to triage patients for treatment recommendation."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • AIF1 • AKT1 • AKT2 • ASXL1 • BBC3 • BCL2L11 • CD34 • CDKN2A • CEBPA • DDB1 • DNMT3A • FLT3 • IDH1 • NPM1 • PTPN11 • RHEB • SDHA • SIRT6 • SLFN11 • SMARCA2 • TET2 • TP53 • WT1

Efficacy and Safety of FLT-3 Inhibitors in Newly Diagnosed FLT-3 Mutated AML Patients: A Systematic Review of Clinical Trials (ASH 2023) - "85) significantly in favor of quizartinib (Qui) + low dose cytarabine (LDAC) vs. LDAC alone. In two nRCTs (N=63) on unfit patients, Qui + venetoclax (Ven) +Aza/LDAC showed complete response (CR)/overall response (ORR) of 42%/72% and Qui+Aza/LDAC showed CR/ORR of 44%/56%...5% with midostaurin (Mid)+ chemotherapy and chemotherapy, respectively...9% and 73% with Gil+chemotherapy and crenolanib (Cren) + chemotherapy, respectively... Addition of Gil, Qui, Lus, Mid, or Cren to standard therapy was well tolerated by most of the patients with FLT-3 mutation. Addition of Mid or Qui to chemotherapy significantly improved survival and response rates as compared to chemotherapy alone in fit patients with FLT-3 mutation and the results were consistent in both young and old fit patients. In early phase trials, Gil and Cren were effective in combination with chemotherapy in fit patients."

Clinical • Review • Acute Myelogenous Leukemia • FLT3

Crenolanib improves outcomes for AML patients with FLT3 and NPM1 mutations (Roswell Park Comprehensive Cancer Center) - "The findings of both studies will be presented...at the 67th annual meeting of the American Society of Hematology (ASH)....Of 106 patients enrolled in the study, 52 were assigned to receive crenolanib in addition to intensive chemotherapy, while 54 received chemotherapy and placebo. Those in the crenolanib group experienced a higher overall response rate (60% vs. 39%) and lower rate of treatment resistance (24% vs. 59%). At a median follow-up of 37.3 months, those who received crenolanib also had both longer event-free survival (3.4 vs. 0.0 months) and longer overall survival (10.4 vs. 8.7 months)."

P3 data • Acute Myelogenous Leukemia

High-Throughput Drug Repurposing Identifies SN-38 As a Potent Inhibitor of AML with Synergistic Effects in Combination with PARP and BCL-2 Inhibitors for Treating KMT2A-Rearranged Leukemias (ASH 2024) - "Results : High Throughput Drug Screening identified the top five most active FDA-approved drugs across 6 AML cell lines : 1) SN-38 (a topoisomerase I inhibitor and the active metabolite of irinotecan), 2) Triplotide (an NF-κB inhibitor), 3) Mitoxantrone (a topoisomerase II inhibitor), 4) Idarubicin (a topoisomerase II inhibitor), and 5) Bortezomib (a proteasome inhibitor)...Drugs that exhibited greater selectivity, meaning greater responses in KMT2Ar cell lines, included : 1) Ingenol Mebutate (a PKC inhibitor), 2) a BET bromodomain inhibitor, 3) MIK665 (an MCL1 inhibitor), 4) Prexasertib (a CHK inhibitor), and 5) Crenolanib (an FLT3/PDGFR inhibitor)...Moreover, the observed synergy with PARP and BCL-2 inhibitors reveals strategies to overcome AML resistance mechanisms to SN-38. However, rigorous preclinical and clinical studies are essential to validate its efficacy, safety, and optimal dosing regimens."

Combination therapy • IO biomarker • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Pediatrics • T Acute Lymphoblastic Leukemia • AFF1 • FLT3 • KMT2A • MLLT3

Roswell Park Experts Share Latest Hematology Research at 67th ASH Annual Meeting (Roswell Park Comprehensive Cancer Center)

Clinical data • Acute Myelogenous Leukemia • B Acute Lymphoblastic Leukemia • Blastic Plasmacytoid Dendritic Cell Neoplasm • Chronic Lymphocytic Leukemia • Diffuse Large B Cell Lymphoma • Multiple Myeloma

Stag2-Cohesin Loss Attenuates Flt3ITD Myeloid Blast Expansion Yet Preserves Mutant HSC (ASH 2023) - "To understand the mechanistic contribution of STAG2-cohesin loss with FLT3ITD, we generated sequential mutagenesis murine models where the order of Stag2 and Flt3ITD mutation is set as either ITD1st Stag22nd (de novo like) or Stag21stITD2nd (sAML like) using tamoxifen-inducible Cre/Flpo recombinase or pIpC-inducible Mx1Cre...STAG2 mutations are more likely to be identified in poor responders to FLT3 inhibition as both reported by us with Pexidartinib treatment, as well as in the setting of Crenolanib treatment where expansion of the STAG2-mutant clone was observed during treatment...Our data highlights an important regulatory role of Stag2-cohesin in Flt3ITD mediated leukemogenesis, while generating a model that mimics the genetic evolution of sAML. This model will not only shed light on the sAML pathogenesis but also with creates a pre-clinical testing platform with potential therapeutic relevance."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • FLT3 • SOCS2 • STAG2 • STAT5 • STAT5AWqe

Developing and validating a sensitive and fast UPLC-MS/MS method for estimating the in vitro metabolic stability of crenolanib in HLMs: identification of structural alarms related to the in silico toxicity and metabolic lability. (PubMed, Anal Methods) - "CLB and encorafenib (EFB as the internal standard, IS) were separated utilizing an isocratic mobile phase method on a reversed-phase (SB-C18) column. The intrinsic clearance of CLB was computed to be 26.79 mL min-1 kg-1, while the in vitro half-life was estimated to be 30.27 min. In silico analysis indicated that slight structural changes to the piperidin-4-amine moiety (99%) during drug design may increase the metabolic stability and improve the safety compared to the case with CLB."

Journal • Preclinical

Long-Term Follow-up of Crenolanib Maintenance after Allogeneic Transplantation in Newly Diagnosed and Relapsed FLT3 Mutated AML (ASH 2024) - P2 | "20 patients had prior exposure to at least one TKI, with 16 receiving sorafenib. The RFS of 78.9% and OS of 84.2% in pts transplanted in CR1 with FLT3-ITD is encouraging. Trials are being planned to formally assess the potential benefit of crenolanib in preventing relapse in the post-transplant setting."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Oncology • Transplantation • DEK • DNMT3A • ETV6 • FLT3 • IDH1 • IDH2 • MLLT3 • NPM1 • NUP214 • RUNX1 • TET2 • WT1

Gilteritinib Augments Preclinical FLT3 and CD19 CAR T Cell Immunotherapy in High-Risk Pediatric Leukemias (ASH 2024) - "In vitro exposure of FLT3CART to other second generation FLT3i crenolanib and quizartinib interestingly did not increase T cell numbers, suggesting a gilteritinib-specific phenomenon that may be mediated in part by inhibition of other T cell kinase(s) not targeted or less targeted by crenolanib and quizartinib. Conclusions : Our data suggest that gilteritinib co-treatment directly enhances desired anti-leukemia activity of FLT3CART immunotherapy against FLT3-activated AML and KMT2A-R ALL. More broadly, these studies also highlight the potential for gilteritinib co-therapy to augment the efficacy of other clinically-in-use CAR T cell immunotherapies and present a promising combinatorial translational strategy for prospective clinical investigation."

CAR T-Cell Therapy • IO biomarker • Preclinical • Hematological Malignancies • Leukemia • Oncology • Pediatrics • CD4 • CD8 • FLT3 • KMT2A

FLT3-ITD Driven CMTM6 Expression Contributes to Immune Escape of Acute Myeloid Leukemia (ASH 2024) - "FLT3 kinase inhibition assays were performed using the FLT3 inhibitors tandutinib, crenolanib, and quizartinib. Taken together, our study identifies a new mechanism of oncogene-induced stabilization of CMTM6 in leukemia cells, resulting in tumor immune escape. Inhibiting the CMTM6/PD-L1 axis could enhance the GVL effect post allo-HCT in patients with FLT3-ITD+ AML."

IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor • CD34 • CD69 • CD8 • CMTM6 • FLT3 • IFNG

Epitope Edited Hematopoietic Stem Cells to Enable Synergistic Immunotherapy Combinations for Acute Myeloid Leukemia (ASH 2023) - "FLT3 tyrosine kinase inhibitors (e. g. , Crenolanib) have the potential to enforce surface expression of FLT3 by impairing its intracellular recycling, thus enhancing CAR-T mediated killing, but their use may be limited by overlapping toxicities on healthy hematopoiesis, in particular in the post-HSCT setting...In conclusion, we believe that epitope edited HSPCs may not only enable safe and effective CAR-T immunotherapies for AML, but also allow their combination with pharmacological blockade of leukemia survival/proliferative pathways to achieve synthetic lethality mechanisms, while still avoiding dose-limiting toxicities. Further exploration of immunotherapy-synergistic combinations will be fundamental to improve the outcomes of difficult-to-treat high-risk AML patients."

IO biomarker • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Transplantation • CD123 • CD34 • FLT3 • IL3RA

Selective EV Protein Sorting and Pathway Perturbation in AML Upon Synergistic FLT3 and Hedgehog Pathway Inhibition. (PubMed, J Extracell Vesicles) - "These findings were corroborated by comparative proteomics of EVs derived from AML patients and healthy donors. Ribosomal and ErbB signalling pathway proteins may play an important role in microenvironmental modulation by EVs, and Crenolanib treatment potentially acts by interfering with leukaemia niche formation."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • FLT3 • GAB1 • RPL27A • RPS26

Signature of leukemia stem cell death pattern predicts prognosis and therapeutic response of acute myeloid leukemia patients. (PubMed, Sci Rep) - "Additionally, predictions regarding FDA-approved drugs indicated that the high LSCD score group is less sensitive to Venetoclax but more sensitive to Crenolanib, Tandutinib, or Midostaurin. In summary, we developed an LSCD model that shows the predictive potential of clinical prognosis and drug sensitivity. This model provides meaningful insights for personalized treatment of AML patients."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • CDK6 • HOXA9 • IL2RA • S100A4 • XIRP2

Tyrosine Kinase Inhibitors for Gastrointestinal Stromal Tumor After Imatinib Resistance. (PubMed, Pharmaceutics) - "Sunitinib, regorafenib, and ripretinib are currently approved as standard second-, third-, and fourth-line therapies, each demonstrating efficacy against distinct mutational profiles. Avapritinib, notably effective against PDGFRA D842V mutations, represents a milestone for previously untreatable subgroups. Several alternative agents-such as nilotinib, masitinib, sorafenib, dovitinib, pazopanib, and ponatinib-have shown varying degrees of success in refractory cases or specific genotypes. Investigational compounds, including crenolanib, bezuclastinib, famitinib, motesanib, midostaurin, IDRX-42, and olverembatinib, are under development to address resistant or wild-type GISTs...Future strategies include precision medicine approaches such as ctDNA-guided therapy, rational drug combinations, and novel drug delivery systems to optimize bioavailability and reduce toxicity. Ongoing research will be crucial for refining treatment sequencing and expanding therapeutic options,..."

Journal • Review • Gastrointestinal Cancer • Gastrointestinal Disorder • Gastrointestinal Stromal Tumor • Oncology • Sarcoma • KIT • PDGFRA

TARGETING FLT3 IN B-ALL: FROM GENOMIC INSIGHTS TO THERAPEUTIC APPLICATIONS (EHA 2025) - "In vitro studies using 6 FLT3i [Gilteritinib (Gil), Midostaurin, Crenolanib, Sorafenib, Quizartibin and Ponatinib (Pon)] and Venetoclax (Ven) were conducted on pt primary cells and on 11 B-ALL wt and 4 AML cell lines (OCI-AML3 wt; MV-4-11, MOLM-13, MONO-MAC6 FLT3-mut).Data analyses highlight the heterogeneity in FLT3i sensitivity across pediatric ALL cell lines with midostaurin, Pon and quizartinib were among the top scoring drugs for most cell lines (Fig. FLT3 alterations identify a novel subgroup of TN B-ALL with therapeutic potential also in combination regimens."

Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • Pediatrics • T Acute Lymphoblastic Leukemia • ABL1 • FLT3 • IKZF1 • KMT2A • NUP214 • ZNF384

Cytomolecular mechanisms of relapse after frontline FLT3 inhibitor (FLT3i)-based therapy in FLT3-mutated (mut) acute myeloid leukemia (AML). (ASCO 2025) - "Induction therapy was intensive chemotherapy (IC) in 107 pts and low intensity therapy (LIT) in 165 pts [including HMA+venetoclax(VEN)+FLT3i in 93 pts]. FLT3i's used were gilteritinib (n=105), sorafenib (n=96), quizartinib (n=54), midostaurin (n=16), and crenolanib (n=1)... Loss of FLT3 mut at relapse occurred in almost 50% of pts receiving frontline FLT3i and was more common in pts receiving IC+FLT3i or HMA+VEN+FLT3i. Common mechanisms of clonal evolution included emergent mutations in RAS pathway, WT1, and DNA methylation genes (TET2, IDH1/2). Mutational clonal evolution was less frequent in post-ASCT relapses."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ABL1 • FLT3 • IDH1 • IDH2 • TET2 • WT1

Establishment and characterization of NCC-PS2-C1: a novel cell line of high-grade pleomorphic spindle cell sarcoma, most consistent with myxofibrosarcoma. (PubMed, Hum Cell) - "Drug screening using NCC-PS2-C1 cells revealed that cobimetinib, crenolanib, and ixazomib were effective against PS. In conclusion, we established NCC-PS2-C1 cells from primary tumors of PS. This cell line is a valuable resource for developing novel chemotherapies."

Journal • Preclinical • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor • Spindle Cell Sarcoma