Rare Disease Report

New Developments in Managing Rare Diseases

DECEMBER 06, 2014
Christina Loguidice

The American Society of Hematology (ASH) 56th Annual Meeting and Exposition is expected to draw more than 20,000 attendees from around the world. The meeting, which was first held in in 1958 and included just 300 attendees, has grown to become the premier annual event for healthcare providers in hematology. The meeting is known for featuring breakthrough research that advances the care of patients with hematologic disorders, including rare diseases such as Gaucher disease, sickle cell anemia, and various malignancies.

During the ASH meeting, Rare Disease Report (RDR) will keep you apprised of the latest research on various rare hematologic diseases through video interviews, daily news highlights, and live tweets. Here are a few hematology experts we have lined up for our ASH video series and a summary of the exciting research they will be presenting during the meeting:

Jawed Fareed, PhD, from Loyola University Medical Center, Maywood, IL, will discuss the impact of an investigational agent known as MST-188 (a triblock polymer formulated using a purified form of poloxamer 188) on circulating microparticles in the setting of sickle cell anemia and sepsis-associated coagulopathy. These microparticles are generated through the fragmentation of blood and endothelial cells and contribute to inflammatory and thrombogenic responses. In an in vivo study to determine whether MST-188 can decrease these effects, Fareed and colleagues found that adding MST-188 to blood drawn from individuals with sickle cell anemia and sepsis-associated coagulopathy resulted in a marked decrease of functioning microparticles and thrombin generation. Abstract 2816.

Neal J. Weinreb, MD, from the University Research Foundation for Lysosomal Storage Diseases, Coral Springs, FL, will review new treatment options for Gaucher disease. In particular, he will focus on the literature regarding imiglucerase, a treatment that reportedly improves bone density, decreases the occurrence of bone crisis, and ameliorates bone pain in patients with Type 1 Gaucher disease. To determine the real-world clinical effects of imiglucerase, Weinreb and colleagues reviewed US claims data for patients treated between January 1, 1997, and June 30, 2013. Their findings corroborated previous reports, leading them to conclude that “early treatment [with imiglucerase] can mitigate the development of chronic bone disease and can result in a reduction of associated medical claims [in adult and pediatric patients].” Abstract 4837.

John Mascarenhas, MD, MS, from The Icahn School of Medicine at Mount Sinai, New York, NY, will discuss Janus kinase (JAK) inhibitors for myelofibrosis, focusing on the results of an open-label phase 2 study of INCB039110, a selective JAK1 inhibitor. In this study of 87 patients, those treated with INCB039110 at a dose of 200 mg twice daily or 600 mg daily had meaningful improvements in myelofibrosis-related symptoms, modest decreases in spleen size, and stable mean hemoglobin levels through week 24. The findings indicate that JAK1 inhibition may produce less myelosuppression than JAK1/JAK2 inhibition. Abstract 714.

Lee Greenberger, PhD, chief scientific officer, Leukemia & Lymphoma Society (LLS), will review key points from the LLS symposium, with an emphasis on emerging immunotherapies for hematologic malignancies. Dr. Greenberger will discuss immune checkpoint inhibitors, which are novel agents designed to suppress the release of immunosuppressive proteins. Blockading the immune checkpoint pathway essentially unharnesses the immune system, enabling unimpeded expression of cancer-fighting antibodies. Dr. Greenberger will also present encouraging data on the outcomes of patients treated with chimeric antigen receptor therapy, sometimes referred to as CAR-T. This is an investigational cellular therapy in which a patient’s T cells are harvested, genetically re-engineered to target the patient’s specific cancer cells, and reinserted to begin their attack.

Other speakers scheduled for our video series include Guillermo Garcia-Manero, MD, from the Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX, who will discuss pracinostat plus azacitidine to treat acute myeloid leukemia; Barbara Pro, MD, from the Department of Medical Oncology, Thomas Jefferson University, Philadelphia, PA, who will review the efficacy of brentuximab in anaplastic large cell lymphoma; and Marina Cavazzana, MD, PhD, from Hôpital Universitaire Necker -Enfants Malades, Paris, France, who will talk about the use of gene therapy to treat sickle cell anemia.

For these videos and news from ASH, we encourage you to regularly visit our conference portal and to follow us on Twitter (@raredr), where we will be tweeting live from the meeting.
We look forward to updating you on the latest advances in diagnosing and treating rare hematological conditions.

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