IP Library Granted Patent US 9,168,257
Granted Patent B2
US 9,168,257 · App. 14/284,521 · Granted Oct 27, 2015

Combination therapy for MDS

Inventors: Daniel Starczynowski (Cincinnati, OH); Garrett W. Rhyasen (Cincinnati, OH)
Assignee: Children's Hospital Medical Center
A61K31/5377A61K31/4184A61K31/495C12N15/1137C12N2310/11C12N2310/531
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Quick Facts
Patent No.
US 9,168,257
App. No.
14/284,521
Granted
Oct 27, 2015
Kind
B2
Abstract

Disclosed are compositions and methods for the treatment of disorders such as myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). The disclosed methods include administering to an individual in need of such treatment a composition that may include an IRAK1/4 inhibitor. In other aspects, the method may include administration of a BLC2 inhibitor.

Claims (35)

1. A method of treating myelodysplastic syndrome (MDS) in an individual having an MDS characterized by increased IRAK1 activation, comprising the step of administering to said individual a composition comprising an IRAK1/4 inhibitor.

2. The method of claim 1 wherein said IRAK1/4 inhibitor is selected from N-acyl-2-aminobenzimidazoles, imidazo[1,2-a]pyridino-pyrimidine, imidazo[1,2-a]pyridino-pyridine, benzimidazolo-pyridine, N-(2-morpholinylethyl)-2-(3-nitrobenzoylamido)-benzimidazole,

or combinations thereof.

3. The method of claim 1 wherein said IRAK1/4 inhibitor comprises an RNAi sufficient to inhibit IRAK1 expression.

4. The method of claim 1 , further comprising the step of administering to said individual an apoptotic modulator.

5. The method of claim 1 , further comprising the step of administering to said individual an apoptotic modulator, wherein said apoptotic modulator comprises a BCL2 inhibitor.

6. The method of claim 1 wherein said myelodysplastic syndrome is selected from Fanconi Anemia, refractory anemia, refractory neutropenia, refractory thrombocytopenia, refractory anemia with ring sideroblasts (RARS), refractory cytopenia with multilineage dysplasia (RCMD), refractory anemia with excess blasts I and II (RAEB), 5q-syndrome, myelodysplasia unclassifiable, refractory cytopenia of childhood, or a combination thereof.

7. The method of claim 1 wherein said administering step is selected from orally, rectally, nasally, topically, parenterally, subcutaneously, intramuscularly, intravenously, transdermally, or a combination thereof.

8. The method of claim 1 wherein said administration decreases the incidence of marrow failure, immune dysfunction, transformation to overt leukemia, or combinations thereof in said individual, as compared to an individual not receiving said composition.

9. The method of claim 1 wherein said method decreases a marker of viability of MDS cells.

10. The method of claim 1 , wherein said treatment decreases a marker of viability of MDS cells.

11. The method of claim 1 , wherein said treatment decreases a marker of viability of MDS and/or AML cells, wherein marker is selected from survival over time, proliferation, growth, migration, formation of colonies, chromatic assembly, DNA binding, RNA metabolism, cell migration, cell adhesion, inflammation, or a combination thereof.

12. A method of treating myelodysplastic syndrome or acute myeloid leukemia in an individual, wherein said myelodysplastic syndrome or said acute myeloid leukemia is characterized by increased IRAK1 activation, comprising the step of administering to said individual

a) an IRAK1/4 inhibitor; and

b) an agent selected from an apoptotic agent, an immune modulating agent, an epigenetic modifying agent, and combinations thereof.

13. The method of claim 12 wherein said IRAK1/4 inhibitor is selected from N-acyl-2-aminobenzimidazoles, imidazo[1,2-a]pyridino-pyrimidine, imidazo[1,2-a]pyridino-pyridine, benzimidazolo-pyridine, N-(2-morpholinylethyl)-2-(3-nitrobenzoylamido)-benzimidazole,

or combinations thereof.

14. The method of claim 12 wherein said IRAK1 inhibitor comprises an RNAi sufficient to inhibit IRAK1 expression.

15. The method of claim 12 wherein said administration step includes administration of an apoptotic modulator.

16. The method of claim 12 wherein said administration step includes administration of an apoptotic modulator comprising a BCL2 inhibitor.

17. The method of claim 12 wherein said administration step includes administration of an immune modulator.

18. The method of claim 12 wherein said administration step includes administration of an immune modulator, wherein said immune modulator comprises lenalidomide.

19. The method of claim 12 wherein said administration step includes administration of an epigenetic modulator.

20. The method of claim 12 wherein said administration step includes administration of an epigenetic modulator, wherein said epigenetic modulator comprises a hypomethylating agent.

21. The method of claim 12 wherein said IRAK1 inhibitor comprises an RNAi sufficient to inhibit IRAK1 expression.

22. The method of claim 12 wherein said myelodysplastic syndrome is selected from Fanconi Anemia, refractory anemia, refractory neutropenia, refractory thrombocytopenia, refractory anemia with ring sideroblasts (RARS), refractory cytopenia with multilineage dysplasia (RCMD), refractory anemia with excess blasts I and II (RAEB), 5q-syndrome, myelodysplasia unclassifiable, refractory cytopenia of childhood, or a combination thereof.

23. The method of claim 12 wherein said administering step is selected from orally, rectally, nasally, topically, parenterally, subcutaneously, intramuscularly, intravenously, transdermally, or a combination thereof.

24. The method of claim 12 wherein said administration decreases the incidence of marrow failure, immune dysfunction, transformation to overt leukemia, or combinations thereof in said individual, as compared to an individual not receiving said composition.

25. The method of claim 12 wherein said method decreases a marker of viability of MDS cells and/or AML cells.

26. The method of claim 12 wherein said treatment decreases a marker of viability of MDS and/or AML cells.

27. The method of claim 12 wherein said treatment decreases a marker of viability of MDS and/or AML cells, wherein said marker is selected from survival over time, proliferation, growth, migration, formation of colonies, chromatic assembly, DNA binding, RNA metabolism, cell migration, cell adhesion, inflammation, or a combination thereof.

28. The method of claim 1 , comprising the step of administering to said individual an apoptotic modulator, wherein said apoptotic modulator comprises a BCL2 inhibitor selected from

and combinations thereof.

29. The method of claim 12 wherein said administration step includes administration of an apoptotic modulator selected from

and combinations thereof.

Assignments (5)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Apr 26, 2021
From: STARCZYNOWSKI, DANIEL; RHYASEN, GARRETT
To: CHILDREN'S HOSPITAL MEDICAL CENTER
Reel/Frame 056040/0043 →
CONFIRMATORY LICENSE Recorded Nov 4, 2016
From: CINCINNATI CHILDRENS HOSP MED CTR
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 040568/0860 →
CONFIRMATORY LICENSE Recorded Aug 9, 2016
From: CINCINNATI CHILDRENS HOSP MED CTR
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 039632/0062 →
CONFIRMATORY LICENSE Recorded Nov 28, 2014
From: CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER
To: NATIONAL INSTITUTES OF HEALTH (NIH), U.S. DEPT. OF HEALTH AND HUMAN SERVICES (DHHS), U.S. GOVERNMENT
Reel/Frame 034495/0227 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jul 8, 2014
From: STARCZYNOWSKI, DANIEL; RHYASEN, GARRETT W.
To: CHILDREN'S HOSPITAL MEDICAL CENTER
Reel/Frame 033259/0630 →
Continuity (2)
Provisional Application 61826211 · May 22, 2013
Related Publication 20140350070A1 · Nov 27, 2014