Identification and Characterization of Class II HDACs: Lead Targets for Treatment of Cancer and Neurodegenerative Diseases

Summary Background: One promising target of recent cancer therapy research involves a family of proteins primarily involved in gene regulation known as histone deacetylases or HDACs. Currently there are over 15 separate clinical trials ongoing with HDAC inhibitors. A major drawback with the use of present HDAC inhibitors in clinical trials is their toxicity. HDAC inhibitors target specific classes of HDACs (HDACs consist of three classes and a total of at least eighteen known isoforms) rather than individual members, which is believed to contribute to the toxic side effects and diminish the therapeutic window. The promise of more effective treatments with HDAC inhibitors lies in the discovery of agents that can target isoform specific HDAC’s. The recent discoveries of the role of class II HDACs, particularly HDAC 6, in motility, aggresome formation, and microtubule assembly holds the promise of developing more targeted therapies for treatment of cancer and neurodegenerative diseases.

Invention: The first discovery, isolation, and characterization of a subset of class II histone deacetylase—HDAC4, HDAC5, HDAC6, and HDAC7. One of the four isoforms, HDAC6, has also been shown by the inventors (See Harvard Cases 2436, 2672, and 2673) to play a prominent role in both aggresome formation and cell motility, both of which contribute to cancer and neurodegenerative diseases, respectively. This data demonstrates the prominent role of HDAC 6 in disease progression and allows for efforts to discovery small molecule HDAC inhibitors for the treatment of diseases.

Applications Advantages: HDACs are capable of modulating proliferation, survival and/or differentiation of cells. HDAC proteins also have the ability to modulate cell growth by influencing cell cycle progression or to modulate gene transcription.

Applications: -A method for modulating the growth, differentiation, or survival of a mammalian cell by modulating HDAC bioactivity -Antibodies and antibody preparations specifically reactive with an epitope of the class HDAC II epitope -A transgenic animal can serve as an animal model for studying cellular and tissue disorders comprising mutated or mis-expressed class II HDAC alleles for use in drug screening. -Probes of the invention can be used as a part of a diagnostic test kit for identifying dysfunctions associated with mis-expression class II HDAC proteins, such as for detecting in a sample of cells isolated from a patient. -The so called "probes/primers" can also be used as a part of "antisense" therapy--by inhibiting transcription and/or translation. -Method is used to treat tumor cells by antagonizing HDx activity and blocking cell cycle progression

Inventor(s): Schreiber, Stuart L

Type of Offer: Licensing



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