The invention relates to novel compositions and to methods for the pulsed administration of compositions to a patient or to cells in vitro for the treatment of human blood disorders. Compositions contain chemical compounds that stimulate the expression of fetal hemoglobin and/or stimulate the proliferation of red blood cells, white blood cells and platelets in patients and ex vivo for reconstitution of hematopoiesis in vivo. These methods are useful to treat or prevent the symptoms associated with anemia, sickle cell disease, thalassemia and other blood disorders. The, invention also relates to methods for the pulsed administration of compositions to patients for the treatment and prevention of cell proliferative disorders including deficiencies such as cytopenia and malignancies such as viral-induced tumors, other forms of neoplasia and for expansion of cells for hematopoietic transplantation. Pulsed administration has been shown to be more effective than continuous therapy in patients tested.
Compositions And Administration Of Compositions For The Treatment Of Blood Disorders
The invention relates to novel compositions and to methods for the pulsed administration of compositions to a patient or to cells in vitro for the treatment of human blood disorders. Compositions contain chemical compounds that stimulate the expression of fetal hemoglobin and/or stimulate the proliferation of red blood cells, white blood cells and platelets in patients and ex vivo for reconstitution of hematopoiesis in vivo. These methods are useful to treat or prevent the symptoms associated with anemia, sickle cell disease, thalassemia, blood loss, and other blood disorders. The invention also relates to methods for the pulsed administration of compositions to patients for the treatment and prevention of cell proliferative disorders including deficiencies such as cytopenia and malignancies and for expansion of cells for hematopoietic transplantation. Pulsed administration has been shown to be more effective than continuous therapy in patients tested.
Compositions And Methods For The Treatment Of Cystic Fibrosis
Douglas V. Faller - Weston MA, US Susan P. Perrine - Weston MA, US George Stamatoyannopoulos - Seattle WA, US
International Classification:
A61K 31/19
US Classification:
514557
Abstract:
The invention is directed to novel pharmaceutical compositions comprising chemicals agents that are useful in the treatment and prevention of cystic fibrosis and the prevention of signs and symptoms of this disease. These pharmaceutical compositions are surprisingly successful in the treatment disorders related to cystic fibrosis including disorders of blood production. Many of these compositions of the invention are even more effective when administered to a patient in pulses. Pulse therapy is not a form of discontinuous administration of the same amount of a composition over time, but comprises administration of the same dose of the composition at a reduced frequency or administration of reduced doses.
Susan P. Perrine - Braintree MA, US Douglas V. Faller - Braintree MA, US
Assignee:
The Trustees of Boston University - Boston MA
International Classification:
A01N 37/00 A61K 31/33
US Classification:
514557, 514183
Abstract:
The invention relates to compositions containing chemical compounds and compositions containing steel factor which stimulate the expression of hemoglobin or globin protein such as embryonic or fetal globin, or the proliferation of hemoglobin expressing and other cells. These compositions can be used to treat or prevent the symptoms associated with anemia, sickle cell diseases, thalassemia and other blood disorders. The invention also relates to methods for administering these compositions to patients and to medical aids for the treatment and prevention of blood and other disorders.
The invention is directed to novel pharmaceutical compositions comprising chemicals agents that are useful in the treatment and prevention of cystic fibrosis and the prevention of signs and symptoms of this disease. These pharmaceutical compositions are surprisingly successful in the treatment disorders related to cystic fibrosis including disorders of blood production. Many of these compositions of the invention are even more effective when administered to a patient in pulses. Pulse therapy is not a form of discontinuous administration of the same amount of a composition over time, but comprises administration of the same dose of the composition at a reduced frequency or administration of reduced doses.
Methods And Low Dose Regimens For Treating Red Blood Cell Disorders
Susan P Perrine - Weston MA, US Douglas V Faller - Weston MA, US Ronald J Berenson - Mercer Island WA, US
Assignee:
Trustees of Boston University - Boston MA Hemaquest Pharmaceuticals, Inc. - Seattle WA
International Classification:
A61K 31/19 A61K 31/706 A61K 31/4406 A61P 7/06
US Classification:
514 43, 514357, 514557
Abstract:
Disclosed herein are methods and low dose regimens for increasing fetal hemoglobin levels in patients with red blood cell disorders, such as beta thalassemia, sickle cell disease, other anemias, or blood loss. Fetal and total hemoglobin levels and red blood cell counts are increased by administering 2,2-dimethylbutyrate (DMB) alone or in combination with hydroxyurea, decitabine or an HDAC inhibitor. Treatment can be continued for at least two weeks.
Compositions And Methods For The Treatment Of Cystic Fibrosis
Douglas Faller - Weston MA, US Susan Perrine - Weston MA, US George Stamatoyannopoulos - Seattle WA, US
International Classification:
A61K031/216 A61K031/22
US Classification:
514/543000, 514/546000
Abstract:
The invention is directed to novel pharmaceutical compositions comprising chemicals agents that are useful in the treatment and prevention of cystic fibrosis and the prevention of signs and symptoms of this disease. These pharmaceutical compositions are surprisingly successful in the treatment disorders related to cystic fibrosis including disorders of blood production. Many of these compositions of the invention are even more effective when administered to a patient in pulses. Pulse therapy is not a form of discontinuous administration of the same amount of a composition over time, but comprises administration of the same dose of the composition at a reduced frequency or administration of reduced doses.
Compositions And Methods For The Treatment Of Cystic Fibrosis
Douglas Faller - Weston MA, US Susan Perrine - Weston MA, US George Stamatoyannopoulos - Seattle WA, US
International Classification:
A61K 31/215 A61K 31/19 A61P 1/00
US Classification:
514529000, 514557000, 514568000, 514574000
Abstract:
The invention is directed to novel pharmaceutical compositions comprising chemicals agents that are useful in the treatment and prevention of cystic fibrosis and the prevention of signs and symptoms of this disease. These pharmaceutical compositions are surprisingly successful in the treatment disorders related to cystic fibrosis including disorders of blood production. Many of these compositions of the invention are even more effective when administered to a patient in pulses. Pulse therapy is not a form of discontinuous administration of the same amount of a composition over time, but comprises administration of the same dose of the composition at a reduced frequency or administration of reduced doses.
Solomon Center Hem/Med Onc 830 Harrison Ave Suite 3200, Boston, MA 02118
Boston Medical Center Corporation 1 Boston Medical Center Place, Boston, MA 02118
Education:
Medical School Tufts University Graduated: 1976 Medical School Tufts-New England Mc Graduated: 1976 Medical School Harvard Med Sch-Dana Farber Graduated: 1976
Center For Hemoglobin Research In Minorities Howard University Nov 2014 - Jan 2016
Program Director
Tufts Medical Center Feb 2011 - Dec 2015
Pediatric Hematologist Oncologist
Hemaquest Pharmaceuticals Nov 2007 - Dec 2010
Chief Scientific Officer, Vice President, Clinical Affairs
Phoenicia Biosciences Nov 2007 - Dec 2010
President and Chief Medical Officer
Boston University School of Medicine Nov 2007 - Dec 2010
Professor, Medicine, Pediatrics, Pharmacology and Experimental Therapeutics
Education:
Tufts University School of Medicine 1974 - 1976
Doctor of Medicine, Doctorates, Medicine
Harvard T.h. Chan School of Public Health 1970 - 1972
Master of Science, Masters, Microbiology, Public Health
Skills:
Clinical Trials Pharmaceutical Development Research Scientist Clinical Trials Design and Management Biotechnology Oncology Clinical Research