Título: Identification of copy number alterations as targets for available drugs involved in T-ALL leukemogenesis and prognosis.
IP: Dra. Eulalia Genescà
Resumen del proyecto: The survival rates for Acute Lymphoblastic Leukemia (ALL) are lower in adults than in children. The long-term disease free survival (DFS) is about 80% in children and only 35%-45% for adults. The reasons for this survival difference lie largely in the higher frequency of poor prognosis subtypes of ALL in adults and a poorer tolerance of the treatment as age increases. Except for selected subgroups (i.e., Ph+ ALL), the current therapeutic protocols for ALL do not take into account the differences in the molecular background of the disease. This is especially evident in T-ALL, in which the same protocols for B-precursor ALL is used, in spite of its different molecular basis. The T-ALL subtype also has fewer new therapeutic alternatives available. Therefore, if we want to improve the survival of patients with ALL and especially those with T-ALL, we need to focus translational research on two main aspects: firstly on obtaining detailed and relevant molecular information to enable doctors to accurately define the risk and decide on the treatment. Secondly, we need to have specific therapeutic alternatives available to apply to these new oncogenetic T-ALL subgroups. Accordingly, we want to assess the prognosis value of different molecular markers that likely contribute to clinical decisions at the time of the diagnosis of T-ALL and assess the anti-leukemic effect of the available drugs that specifically target these genes in an in vivo model of T-ALL. We also want to check if other new drugs currently being assessed in preclinical phases or in clinical trials and that target other CNAs important for the development of other leukemia and solid cancers could be also a therapeutic alternative in T-ALL.
Entidad financiadora: CELGENE