David Hirsch Perlmutter

US Patent:

6403646, Jun 11, 2002

Filed:

Jun 30, 2000

Appl. No.:

09/608324

Inventors:

David H. Perlmutter - Pittsburgh PA 15213
Jon A. J. Burrows - St. Louis MO 63110
Lauren K. Willis - Norfolk VA 23510-1001
Jeffery H. Teckman - St. Louis MO 63110

International Classification:

A61K 31192

US Classification:

514570, 514569, 514568

Abstract:

A method for the treatment of alpha-1-antitrypsin deficiency caused by the protease inhibitor type Z mutation by administration of phenylbutyric acid derivatives. Also disclosed are methods for the prevention and treatment of liver injury and emphysema associated with alpha-1-antitrypsin deficiency by administration of phenylbutyric acid derivatives.

US Patent:

20110154510, Jun 23, 2011

Filed:

Sep 14, 2010

Appl. No.:

12/881976

Inventors:

Stephen C. Pak - Pittsburgh PA, US
David H. Perlmutter - Pittsburgh PA, US
Gary A. Silverman - Pittsburgh PA, US

International Classification:

G01N 33/00
C07H 21/04
C12N 15/63
A01K 67/00

US Classification:

800 3, 536 231, 4353201, 800 13, 800 9, 800 12

Abstract:

The present invention relates to methods and compositions for high content drug screening in which may be used to identify compounds that treat disorders associated with protein aggregation.

US Patent:

20120129839, May 24, 2012

Filed:

Jan 31, 2012

Appl. No.:

13/362606

Inventors:

David Hirsch Perlmutter - Pittsburgh PA, US
George Konstantine Michalopoulos - Pittsburgh PA, US
Tunde Hidvegi - Gibsonia PA, US

International Classification:

A61K 31/55
A61P 25/16
A61P 25/28

US Classification:

514217

Abstract:

The present invention relates to methods of treatment of clinical disorders associated with protein polymerization comprising administering, to a subject, an effective amount of carbamazepine, oxcarbazepine or another carbamazepine-like compound. It is based, at least in part, on the discovery that, in cells having a genetic defect in α1-antitrypsin, carbamazepine was able to decrease levels of the mutant protein. Furthermore, carbamazepine reduced the hepatic load of mutant α1-antitrypsin and the toxic effect of that mutant protein accumulation, hepatic fibrosis, in vivo using a mouse model of the disease. As patients having this defect in α1-antitrypsin exhibit toxic accumulations of the protein, treatment according to the invention may be used to ameliorate symptoms and signs of disease.

US Patent:

20130024953, Jan 24, 2013

Filed:

May 3, 2012

Appl. No.:

13/463638

Inventors:

Stephen C. Pak - Pittsburgh PA, US
David Hirsch Perlmutter - Pittsburgh PA, US
Gary A. Silverman - Pittsburgh PA, US

Assignee:

University of Pittsburgh - of the Commonwealth System of Higher Education - Pittsburgh PA

International Classification:

A61K 31/454
A61P 25/16
A61K 31/5415
A61K 31/138
A01K 67/033
A61K 31/34
A61K 31/704
A61K 31/545
C07H 21/00
A61K 49/00
A61P 25/28
A61K 31/337

US Classification:

800 3, 514322, 5142258, 514646, 514449, 514468, 514 27, 514209, 536 235, 800 9, 800 12

Abstract:

The present invention relates to methods of treatment of clinical disorders associated with protein aggregation comprising administering, to a subject, an effective amount of an anti-protein aggregate (“APA”) compound selected from the group consisting of pimozide, fluphenazine (e.g., fluphenazine hydrochloride), tamoxifen (e.g., tamoxifen citrate), taxol, cantharidin, cantharidic acid, salts thereof and their structurally related compounds. It is based, at least in part, on the discovery that each of the aforelisted compounds were able to promote degradation of aggregated ATZ protein in a model system. According to the invention, treatment with one or more of these APA compounds may be used to ameliorate the symptoms and signs of AT deficiency as well as other disorders marked by protein aggregation, including, but not limited to, Alzheimer's Disease, Parkinson's Disease, and Huntington's Disease.

US Patent:

51752530, Dec 29, 1992

Filed:

Apr 24, 1991

Appl. No.:

7/690284

Inventors:

Robert J. Fallon - Chesterfield MO
Joseph W. Bulock - St. Peters MO
Steven P. Adams - St. Charles MO
David H. Perlmutter - Clayton MO

Assignee:

Washington University - St. Louis MO

International Classification:

C07K 706
A61K 3700

US Classification:

530330

Abstract:

Novel short peptides of up to about 20 amino acid residues are disclosed that bind to the SEC receptor. These novel peptides preferably are pentapeptides which are synthetic analogs of a pentapeptide domain in the carboxy-terminal fragment of. alpha. sub. 1 -antitrypsin (amino acids 370-374, Phe-Val-Phe-Leu-Met [SEQ ID NO:2]). A preferred binding peptide is Phe-Val-Ala-Leu-Met [SEQ ID NO;6].

US Patent:

20160361319, Dec 15, 2016

Filed:

Aug 25, 2016

Appl. No.:

15/247518

Inventors:

- Pittsburgh PA, US
David Hirsch Perlmutter - Pittsburgh PA, US
Gary A. Silverman - Pittsburgh PA, US

Assignee:

University of Pittsburgh - Of the Commonwealth System of Higher Education - Pittsburgh PA

International Classification:

A61K 31/5415

Abstract:

The present invention relates to methods of treatment of clinical disorders associated with protein aggregation comprising administering, to a subject, an effective amount of an anti-protein aggregate (“APA”) compound selected from the group consisting of pimozide, fluphenazine (e.g., fluphenazine hydrochloride), tamoxifen (e.g., tamoxifen citrate), taxol, cantharidin, cantharidic acid, salts thereof and their structurally related compounds. It is based, at least in part, on the discovery that each of the aforelisted compounds were able to promote degradation of aggregated ATZ protein in a model system. According to the invention, treatment with one or more of these APA compounds may be used to ameliorate the symptoms and signs of AT deficiency as well as other disorders marked by protein aggregation, including, but not limited to, Alzheimer's Disease, Parkinson's Disease, and Huntington's Disease.

US Patent:

20150265626, Sep 24, 2015

Filed:

Jun 2, 2015

Appl. No.:

14/728619

Inventors:

- Pittsburgh PA, US
David Hirsch Perlmutter - Pittsburgh PA, US
Gary A. Silverman - Pittsburgh PA, US

Assignee:

University Of Pittsburgh - of the Commonwealth System of Higher Education - Pittsburgh PA

International Classification:

A61K 31/5415

Abstract:

The present invention relates to methods of treatment of clinical disorders associated with protein aggregation comprising administering, to a subject, an effective amount of an anti-protein aggregate (“APA”) compound selected from the group consisting of pimozide, fluphenazine (e.g., fluphenazine hydrochloride), tamoxifen (e.g., tamoxifen citrate), taxol, cantharidin, cantharidic acid, salts thereof and their structurally related compounds. It is based, at least in part, on the discovery that each of the aforelisted compounds were able to promote degradation of aggregated ATZ protein in a model system. According to the invention, treatment with one or more of these APA compounds may be used to ameliorate the symptoms and signs of AT deficiency as well as other disorders marked by protein aggregation, including, but not limited to, Alzheimer's Disease, Parkinson's Disease, and Huntington's Disease.

US Patent:

20140331341, Nov 6, 2014

Filed:

Jul 18, 2014

Appl. No.:

14/335413

Inventors:

- Pittsburgh PA, US
David H. Perlmutter - Pittsburgh PA, US
Gary A. Silverman - Pittsburgh PA, US

Assignee:

University of Pittsburgh - of the Commonwealth System of Higher EdUcation - Pittsburgh PA

International Classification:

A61K 49/00
C12Q 1/68
A01K 67/027
G01N 33/50

US Classification:

800 12, 4353201, 506 10, 435 613, 800 9

Abstract:

The present invention relates to methods and compositions for high content drug screening in which may be used to identify compounds that treat disorders associated with protein aggregation.