Abstract: Provided are methods for treating a disease, disorder, or condition associated with an acid ceramidase (AC) biological activity. The methods include administering to a subject in need thereof a composition including an AC inhibitor and at least one additional active agent, such as a C6-ceramide nanoliposome (CNL); an inhibitor of a Bcl-2 family protein; a hypomethylating agent; an intensive chemotherapeutic agent such as cytarabine (AraC) and/or daunorubicin; a Hedgehog pathway inhibitor; a targeted agent, such as a FLT2 inhibitor or a EDH1/2 inhibitor; and/or an antibody drug conjugate that targets, for example, CD-33. The composition can include N-[(2S,3R)-1,3-dihydroxyoctadecan-2-yl]2-chloroacetamide (SACLAC) or a pharmaceutically acceptable salt thereof and at least one additional active agent. The disease, disorder, or condition associated with the AC biological activity can be a cancer, such as acute myeloid leukemia (AML).

Abstract: Provided are methods for treating diseases, disorders, and conditions associated with undesirable cellular proliferation in subjects in need thereof. In some embodiments, the methods include administering to a subject in need thereof a therapeutically effective amount of a composition comprising, consisting essentially of, or consisting of a chemotherapeutic agent and a short chain ceramide. Also provided are methods for increasing total ceramide levels in cells, for increasing long chain ceramide to a very long chain ceramide ratios in cells, methods for enhancing apoptosis of cells, for prognosing subjects with diseases, disorders, and conditions associated with undesirable cellular proliferation with respect to treatments, for increasing sensitivities of drug-resistant tumor and/or cancer cells to chemotherapeutics, and compositions that have one or more short chain ceramides and one or more chemotherapeutically active agents.

Abstract: The present application provides novel compositions and methods for treating acute myeloid leukemia (AML). Compounds of the invention are acid ceramidase inhibitors, reduce AML cell viability, inhibit AML cell proliferation, increase cell death of AML cells, and induce apoptosis in AML cells. A primary compound of the invention is SACLAC: 2-chloro-N-((2S,3R)-1,3-dihydroxyoctadecan-2-yl)acetamide. The bromine analog of SACLAC (SABRAC: 2-bromo-N-((2S,3R)-1,3-dihydroxyoctadecan-2-yl)acetamide) is also useful for treating AML. SACLAC has much better activity than other know drugs used to treat AML.

Abstract: The present application provides novel compositions and methods for treating acute myeloid leukemia (AML). Compounds of the invention are acid ceramidase inhibitors, reduce AML cell viability, inhibit AML cell proliferation, increase cell death of AML cells, and induce apoptosis in AML cells. A primary compound of the invention is SACLAC: 2-chloro-N-((2S,3R)-1,3-dihydroxyoctadecan-2-yl)acetamide. The bromine analog of SACLAC (SABRAC: 2-bromo-N-((2S,3R)-1,3-dihydroxyoctadecan-2-yl)acetamide) is also useful for treating AML. SACLAC has much better activity than other know drugs used to treat AML.

Abstract: The present application discloses compositions and methods utilizing a combination of Pert and ddPCR technologies that yield, for example, the unexpected result of absolute quantification of the presence of RT enzymes. The novel technology disclosed herein is referred to as ddPERT. This is an advance over prior technology such as PERT.