In the largest report on acute stroke in sickle cell disease to date, researchers at the Medical University of South Carolina and elsewhere provide the first systemic evidence for the safe use of tPA in stroke patients with sickle cell disease. Adult patients with sickle cell disease (SCD) who experience a stroke caused by a clot (i.e., ischemic strokes or IS) can be treated safely with tissue plasminogen activator (tPA) if they qualify, report investigators at the Medical University of South Carolina (MUSC) and elsewhere in the March 2017 issue of Stroke.

License of Gamma-Globin from Cincinnati Children’s Hospital Medical Center to be used in combination with Calimmune’s Select+™ Technology. Calimmune, Inc., a clinical-stage gene therapy company, today announced a license agreement with Cincinnati Children’s Hospital Medical Center (Cincinnati Children’s) to develop and commercialize gene therapies combining Calimmune’s Select+™ technology with Cincinnati Children’s proprietary gene therapy construct for the treatment of patients with sickle cell disease and beta thalassemia. Calimmune’s Select+™ platform aims to improve engraftment of stem cell gene therapies through a post-cellular infusion selection process.

Research team establishes biomarkers for the inherited blood disorder. Researchers have found new biophysical markers that could help improve the understanding of treatments for sickle cell disease, a step toward developing better methods for treating the inherited blood disorder that affects an estimated 80,000 to 100,000 Americans each year.

Highlights · The global apheresis equipment market is expected to reach USD 3.7 billion by 2024, according to a new report by Grand View Research, Inc. · The apheresis equipment industry is expected to be impacted by factors such as rising demand for plasma, platelets and other blood components, increasing incidence of chronic illnesses, presence and development of ideal reimbursement policies across various regions, and supportive government initiatives. · Growing incidence of blood related disorders leading to the rise in demand for platelets and plasma is expected to serve the market as a high impact rendering driver. · Disposable apheresis kits are anticipated to dominate the product segment by 2024. · The photopheresis procedure is anticipated to witness lucrative growth owing to its rising demand in treatment.

A study of children with sickle cell disease (SCD) referred for an evaluation of neuropsychological deficits found that while 19 of 89 patients (25%) were diagnosed with attention deficit hyperactivity disorder (ADHD), only 21% with ADHD had been prescribed medication for it. The study, “Attention Deficit Hyperactivity Disorder in Children With Sickle Cell Disease Referred for an Evaluation,” was published in the Journal of Pediatric Hematology/Oncology.

Tim Townes has a dream. He has a dream that one day in Alabama, he will see four little girls sitting on the steps of the Sixteenth Street Baptist Church in Birmingham. Townes dreams that on Martin Luther King Jr. Day two years from now, national television news shows will trumpet the story of those girls, healthy and no longer ravaged by sickle cell disease. He dreams of seeing that kind of good news on an important civil rights anniversary – instead of well-worn footage of the Sixteenth Street bombing in 1963 that killed four little girls, or marchers beaten on Selma’s Edmund Pettus Bridge. “I think Alabamians could really be proud of that and I think would want to work toward that,” says Townes, a Ph.D., director of the UAB Stem Cell Institute and chairman of the Department of Biochemistry and Molecular Genetics.

With drug overdoses now the leading cause of accidental death in the U.S., efforts are underway in many states to limit abuse and access to prescription opioids — steps that, some say, could cause unnecessary suffering among sickle cell disease (SCD) patients who rely on these medications to treat their symptoms. “I’m seeing more and more patients calling me or coming in and saying they’re in so much pain and have been taken off the narcotics they have been receiving for years,” said Judy Anderson, executive director of the Sickle Cell Association in Norfolk, Virginia, an education and advocacy organization.

Sancilio Pharmaceuticals Company, Inc. (SPCI) today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to Altemia™ Soft Gelatin Capsules for the treatment of sickle cell disease (SCD) in children. Altemia™, a leading development program for SPCI, is being developed as an oral, once-a-day formulation for children between the ages of 5 – 17 with SCD. "The FDA's Rare Pediatric Disease designation for Altemia™, in addition to the previously granted Orphan Drug Designation, further stresses the Agency's recognition that the SCD community faces a critical need for new treatments like Altemia™," said Dr. Frederick Sancilio, President and Chief Executive Officer of SPCI. "We continue to make important progress in our clinical trial; a randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of Altemia™ in pediatric patients. We look forward to reporting top-line results from the study early in the fourth quarter, this year."

The Patient-Centered Outcomes Research Institute (PCORI) Board of Governors today approved about $18 million to fund two studies comparing different ways to manage the transition of people with sickle cell disease from pediatric to adult care.

Although children with sickle cell disease generally receive the care they need to manage their condition, the transition to adult care can be hazardous. Many primary care clinicians who treat adults are unfamiliar with caring for this rare condition, and patients can find it challenging to access care across specialists. Losing a source of care, such as pediatric specialty care, decreases the likelihood that young adults with sickle cell disease will receive preventive care and screening visits, and it increases the chance they will seek emergency department care when painful symptoms flare.

Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) announced today that the independent Data and Safety Monitoring Board (DSMB) for the Phase 3 HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study evaluating voxelotor (previously called GBT440) for the treatment of sickle cell disease (SCD), has completed its first scheduled clinical safety review. No drug-related serious or severe adverse events were reported in either of the voxelotor treatment arms (900 mg and 1500 mg), and there were no discontinuations due to adverse events. Based on this review, the DSMB did not identify safety concerns with voxelotor at either dose level across all patients dosed in the ongoing SCD clinical program, including adult patients in the Phase 3 HOPE Study and adolescent patients in the HOPE-KIDS 1 (GBT440-007) Study. Based on the available safety data to date, including at the high dose level of 1500 mg, the DSMB recommended that the pivotal study continue as planned and approved initiation of enrollment in adolescent patients age 12 to 17.