Accelerating Breakthrough Discoveries into Medicines

Susan P. Perrine, MD

Dr. Perrine is developing a small molecule therapeutic for treatment of beta-thalassemia and sickle cell anemia.

Susan Perrine SM, MD is a pediatric hematologist who has dedicated 25 years to developing patient-friendly treatments for serious blood diseases and cancers. She is a professor of pediatrics, medicine, pharmacology and experimental therapeutics at Boston University School of Medicine.

Beta-thalassemia and sickle cell anemia are serious blood diseases and are World Health Organization (WHO)-classified as a global health burden. Dr. Perrine’s lab has developed a therapy that activates expression of genes for another type of hemoglobin, fetal globin or HbF, which functions well before birth but is suppressed during infancy. Increased expression of HbF reduces the severity of these diseases.

She became intrigued with reactivating alternate genes as a treatment approach, from seeing patients in the Middle East who had few or no symptoms of sickle cell disease because of high levels of fetal hemoglobin. She found an infant population who also has high HbF levels, associated with circulating high levels of a short chain fatty acid, and developed similar small molecules into drugs.

Her compound was identified in a high-throughput screen as a potent HbF inducer, validated in vitro and in vivo, including in non-human primates. The drug targets 3 repressors of the gene promoter, offering high potential for benefit in patients. With 40 years of clinical use for another condition as a combination therapy, PB-04 has never been formulated as a single agent drug. Dr. Perrine and her lab are completing final experiments and expect to begin clinical trials in patients soon.

Dr. Perrine received her BA from Mills College, Masters degrees from Harvard School of Public Health and Robert Wood Johnson School of Medicine, and her M.D. from Tufts University School of Medicine. She completed her Pediatric Residency training at Tufts-New England Medical Center and a Hematology-Oncology fellowship at The Children’s Hospital, Boston, Dana Farber Cancer Institute, and Harvard Medical School. She is Board Certified in Pediatrics and Pediatric Hematology-Oncology.

Dates to Know

April 5, 2017
Letter of Intent Deadline

May 31, 2017
Full Application Deadline (For those invited)

December 2017
Awardees Notified

Harrington Project

The Harrington Discovery Institute is the nonprofit arm of the Harrington Project for Discovery & Development, an international initiative supporting breakthrough research by physician-scientists. The Project is a new and powerful approach to address some of the challenges hindering advancement of medicine today.

The Harrington Grant is an important part of the overall pharmaceutical research initiative created by The Harrington Project.

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