Detection of Niemann-Pick disease comprising detection of lysozyme and cathepsins
Provided herein are methods for detecting an increased probability or risk of neurodegeneration in a subject. In some embodiments the method comprises assaying a sample from the subject for lysozyme and/or cathepsin S and detecting the lysozyme and/or cathepsin S from the sample, wherein an increased lysozyme and/or cathepsin S compared to levels in a control subject sample indicate an increased probability or risk of neurodegeneration. Methods of treatment and screening assays for determining drug effectiveness are also provided herein.
1. A method for detecting an increased probability or risk of neurodegeneration caused by Niemann-Pick disease in a subject and treating said subject comprising detecting lysozyme and cathepsin S levels in a sample from the subject, wherein an increased ratio of lysozyme:cathepsin S levels in the subject compared to the ratio of lysozyme:cathepsin S levels in a control subject sample indicates an increased probability or risk of the neurodegeneration; and
administering to the subject an effective amount of a drug for the treatment of the neurodegeneration in the subject;
wherein the drug comprises hydroxy propyl beta cyclodextrin (HPBCD).
2. The method of claim 1 , wherein the Niemann-Pick disease is selected from the group consisting of Niemann-Pick Disease, Type A, Niemann-Pick Disease, Type B, and Niemann-Pick Disease, Type C.
3. The method of claim 1 , wherein the subject is a mammal selected from the group consisting of humans, primates, monkeys, chimpanzees, dogs, cats, sheep, cattle, goats, pigs, horses, chickens, mice, rats, rabbits, and guinea pigs.
4. The method of claim 1 , wherein the drug further comprises Vorinostat.
5. A method for detecting an increased probability or risk of neurodegeneration caused by Niemann-Pick disease in a subject and treating said subject comprising detecting in a sample from the subject lysozyme and cathepsin S levels, wherein an increased ratio of cathepsin S:lysozyme in the subject compared to the ratio of cathepsin S:lysozyme in a control subject sample indicates an increased probability or risk of neurodegeneration; and
administering to the subject an effective amount of a drug for the treatment of the neurodegeneration in the subject;
wherein the drug comprises hydroxy propyl beta cyclodextrin (HPBCD).
6. The method of claim 5 wherein the Niemann-Pick disease is selected from the group consisting of Niemann-Pick Disease, Type A, Niemann-Pick Disease, Type B, and Niemann-Pick Disease, Type C.
7. The method of claim 5 wherein the subject is a mammal selected from the group consisting of humans, primates, monkeys, chimpanzees, dogs, cats, sheep, cattle, goats, pigs, horses, chickens, mice, rats, rabbits, and guinea pigs.
8. The method of claim 5 wherein the drug further comprises Vorinostat.