# US 2019/0167711 A1 · Methods for diagnosis and treatment of acute lymphoblastic leukemia > Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt - Number: US 2019/0167711 A1 - Document ID: US20190167711A1 - Status: Published application - Kind: A1 - Application number: 16/272,352 - Filed: 2019-02-11 - Published: 2019-06-06 - Earliest claimed: 2014-07-30 - Applicants: Mor Research Applications Ltd. - Inventors: Smadar AVIGAD; Isaac YANIV; Keren SHICHRUR - Web page: https://patentgrep.com/patent/US20190167711A1 ## CPC - A61K 31/7105: Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links - A61P 35/02: specific for leukemia - C12Q 1/6886: for cancer (immunoassay for cancer G01N33/575) - C12Q 2600/106: Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism - C12Q 2600/118: Prognosis of disease development - C12Q 2600/158: Expression markers - C12Q 2600/178: miRNA, siRNA or ncRNA - A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D) - A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS - C12Q: MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS (immunoassay G01N33/53); COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES ## Abstract Provided herein is the use of miR-451 as a biomarker in prognosis of anti-ALL treatment modalities, in early diagnosis of ALL relapse risk, and/or in identifying an ALL patient that can benefit from a treatment modality that affects one or more miR-451-related metabolic pathways. ## Claims ### Claim 1 A method for identifying acute lymphoblastic leukemia (ALL) patients most likely to benefit from treatment with a nicotinamide phosphoribosyl transferase (NAMPT) inhibitor, comprising: obtaining a biological sample from an ALL patient; determining a level of expression of miR-451 in the biological sample; and if the level of expression of miR-451 is below a predetermined level, identifying the patient as being likely to benefit from treatment with a NAMPT inhibitor. ### Claim 2 (depends on 1) The method of claim 1, wherein the biological sample is selected from the group consisting of bone marrow, lymph fluid, whole blood, plasma, CNS fluid or serum. ### Claim 3 (depends on 1) The method of claim 1, wherein the subject is an adolescent, child, or infant. ### Claim 4 (depends on 1) The method of claim 1, wherein ALL is pediatric B-cell ALL. ### Claim 5 A method for prognosis of a treatment modality that inhibits nicotinamide phosphoribosyl transferase (NAMPT) in a subject afflicted with acute lymphoblastic leukemia (ALL), the method comprising: obtaining a biological sample from the subject; determining a level of expression of miR-451 in the biological sample; and if the level of expression of miR-451 is below a predetermined level, correlating the level of expression of miR-451 with a positive prognosis of a treatment modality that inhibits NAMPT. ### Claim 6 (depends on 5) The method of claim 5, wherein the biological sample is selected from the group consisting of bone marrow, lymph fluid, whole blood, plasma, CNS fluid or serum. ### Claim 7 (depends on 5) The method of claim 5, wherein the subject is an adolescent, child, or infant. ### Claim 8 (depends on 5) The method of claim 5, wherein ALL is pediatric B-cell ALL. ### Claim 9 A method for treatment of acute lymphoblastic leukemia (ALL) in a patient suffering from ALL, comprising: obtaining a biological sample from the ALL patient; determining a level of expression of miR-451 in the biological sample; and if the level of expression of miR-451 is below a predetermined level, treating the patient with a nicotinamide phosphoribosyl transferase (NAMPT) inhibitor. ### Claim 10 (depends on 9) The method of claim 9, wherein NAMPT inhibition is by one or more inhibitors selected from the group consisting of a small molecule inhibitor, antibody, antisense nucleic acid, micro-RNA (miRNA) and RNA interference agent, with the proviso that the inhibitor of NAMPT is not miR-451. ### Claim 11 (depends on 9) The method of claim 9, wherein the NAMPT inhibitor is FK866 or a functional variant thereof. ### Claim 12 (depends on 9) The method of claim 9, wherein the patient is an adolescent, child, or infant. ### Claim 13 (depends on 9) The method of claim 9, wherein the treatment of ALL includes reducing risk of relapse in a patient. ### Claim 14 A method for early diagnosis of acute lymphoblastic leukemia (ALL) relapse risk in a subject, comprising: obtaining a biological sample from the subject when first diagnosed as inflicted with ALL; detecting a level of expression of miR-451; and diagnosing ALL relapse risk if the level of expression of miR-451 is below a predetermined level. ### Claim 15 (depends on 14) The method of claim 14, wherein the biological sample is selected from the group consisting of bone marrow, lymph fluid, whole blood, plasma, CNS fluid or serum. ### Claim 16 A method for treatment of pediatric acute lymphoblastic leukemia (ALL) in a subject, comprising: administering to the subject a therapeutically effective amount of an inhibitor of nicotinamide phosphoribosyltransferase (NAMPT), selected from the group consisting of an antibody, antisense nucleic acid, microRNA (miRNA) and RNA interference agent, thereby treating the patient, with the proviso that the inhibitor of NAMPT is not miR-451.