European Commission grants orphan drug status to Apogenix ’s asunercept for MDS

German biopharmaceutical company Apogenix ’s asunercept (APG101) has received orphan drug designation from the European Commission (EC) to treat patients with myelodysplastic syndromes (MDS).
Source: Pharmaceutical Technology - Category: Pharmaceuticals Source Type: news

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Publication date: Available online 13 August 2018Source: The Lancet HaematologyAuthor(s): Tapan M Kadia, Jorge Cortes, Farhad Ravandi, Elias Jabbour, Marina Konopleva, Christopher B Benton, Jan Burger, Koji Sasaki, Gautam Borthakur, Courtney D DiNardo, Naveen Pemmaraju, Naval Daver, Alessandra Ferrajoli, Xuemei Wang, Keyur Patel, Jeffrey L Jorgensen, Sa Wang, Susan O'Brien, Sherry Pierce, Carla TuttleSummaryBackgroundFront-line therapy for elderly or unfit patients with acute myeloid leukaemia (AML) remains unsatisfactory with poor outcomes and excessive toxicity. We studied a new low-intensity regimen of cladribine combin...
Source: The Lancet Haematology - Category: Hematology Source Type: research
Publication date: 13 August 2018Source: Cancer Cell, Volume 34, Issue 2Author(s): Stanley Chun-Wei Lee, Khrystyna North, Eunhee Kim, Eunjung Jang, Esther Obeng, Sydney X. Lu, Bo Liu, Daichi Inoue, Akihide Yoshimi, Michelle Ki, Mirae Yeo, Xiao Jing Zhang, Min Kyung Kim, Hana Cho, Young Rock Chung, Justin Taylor, Benjamin H. Durham, Young Joon Kim, Alessandro Pastore, Sebastien MonetteSummaryMutations affecting RNA splicing factors are the most common genetic alterations in myelodysplastic syndrome (MDS) patients and occur in a mutually exclusive manner. The basis for the mutual exclusivity of these mutations and how they co...
Source: Cancer Cell - Category: Cancer & Oncology Source Type: research
Source: Annals of Hematology - Category: Hematology Source Type: research
The prognostic significance of global aberrant alternative splicing in patients with myelodysplastic syndrome, Published online: 13 August 2018; doi:10.1038/s41408-018-0115-2The prognostic significance of global aberrant alternative splicing in patients with myelodysplastic syndrome
Source: Blood Cancer Journal - Category: Hematology Authors: Source Type: research
Authors: Tanaka H, Kuwabara C, Kayamori K, Shimizu R, Suzuki Y PMID: 30089748 [PubMed - as supplied by publisher]
Source: Journal of Clinical and Experimental Hematopathology : JCEH - Category: Hematology Tags: J Clin Exp Hematop Source Type: research
This study accessed the extent of contact system activation, the levels of extracellular traps, and coagulation activation in hematologic malignancies including acute leukemia. In 154 patients with hematologic malignancies (acute leukemia,n = 29; myelodysplastic syndrome,n = 20; myeloproliferative neoplasms,n = 69; plasma cell myeloma,n = 36) and 48 normal controls, the levels of coagulation factors (fibrinogen and factor VII, VIII, IX, and XII), D-dimer, thrombin generation, extracellular trap markers (histone–DNA complex, cell-free dsDNA, leukocyte elastase), ...
Source: Journal of Thrombosis and Thrombolysis - Category: Hematology Source Type: research
Myelodysplastic syndrome (MDS) is a heterogeneous group of clonal stem cell disorders with dismal prognosis,1 especially in patients with higher-risk MDS or those with lower-risk MDS having poor prognostic features.2 Despite recent advances in knowledges of genetic and molecular pathogenesis,3 therapies for MDS currently remain to use nonspecific approaches with unsatisfactory results. Among variable therapeutic options, allogeneic hematopoietic stem cell transplantation (HSCT) is peculiar in that it is the only considered option to cure MDS.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Hypermethylation has been documented in some myeloid disorders including myelodysplastic syndrome (MDS) and in renal cell carcinoma (RCC). A group at the Mayo Clinic noticed the epigenetic marking patterns between these vastly different conditions and wondered if there was a connection. The group found that there was a strong association.08/09/2018
Source: Kidney Cancer Association - Category: Urology & Nephrology Source Type: news
In conclusion, IMiD toxicity in PEL cell lines is independent of IKZF1 and IKZF3 but proceeds through degradation of the neosubstrate CK1α and downregulation of IRF4.
Source: Blood - Category: Hematology Authors: Tags: Immunobiology and Immunotherapy, Lymphoid Neoplasia Source Type: research
We describe a new conditional murine Srsf2P95H mutation model, where the P95H mutation is expressed physiologically and heterozygously from its endogenous locus after Cre activation. Using multiple Cre lines, we demonstrate that during native hemopoiesis (ie, no BM transplantation), the Srsf2P95H mutation needs to occur within the hemopoietic stem-cell–containing populations to promote myelomonocytic bias and expansion with corresponding transcriptional and RNA splicing changes. With age, nontransplanted Srsf2P95H animals developed a progressive, transplantable disease characterized by myeloid bias, morphological dys...
Source: Blood - Category: Hematology Authors: Tags: Hematopoiesis and Stem Cells, Myeloid Neoplasia Source Type: research
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