Every student feels the pressure of assessments, deadlines and attending classes. But most don’t face the daily dilemma that I do living with sickle cell anaemia: do I settle for lower grades because the hard work needed to achieve the highest standards may result in me becoming sick? After hundreds of hospital visits and a double hip replacement before the age of 21, I know as well as anyone that living with sickle cell anaemia is challenging. But if I were given the choice to cure it, I would have to decline.

Prolong Pharmaceuticals’ investigational drug for treatment of sickle cell disease (SCD)-associated complications, Sanguinate, showed promising results in patients experiencing vaso-occlusive crisis (VOC). According to the research study, “SANGUINATE Returns RBCs To More Normal Morphology In Patients With VOC,” presented at the 2017 Annual Symposium of the Foundation for Sickle Cell Disease Research (FSCDR) in Fort Lauderdale, Florida, this new treatment was able to restore the shape of red blood cells, improving the VOC symptoms in SCD patients.

GlycoMimetics, in collaboration with Pfizer, is now enrolling participants for a Phase 3 clinical trial to test the effectiveness and safety of the drug candidate rivipansel (GMI-1070) for the treatment of vaso-occlusive crisis (VOC) in hospitalized patients with sickle cell disease (SCD). “We can report that the Phase 3 program to evaluate rivipansel for vaso-occlusive sickle cell crisis, according to our partner Pfizer, remains on track for completion in the second half of 2018,” Rachel King, CEO of GlycoMimetics, said in a press release.

Researchers at New York’s Weill Cornell Medicine have discovered an innovative method of producing healthy stem cells that could significantly improve treatment of sickle cell anemia and other blood disorders. A study describing the method, “Conversion of adult endothelium to immunocompetent haematopoietic stem cells,” appeared in the journal Nature.

A 13-year-old boy with sickle cell disease has become the world’s first patient to be successfully treated with gene therapy. The unidentified teenager remains free from sickle crises and other signs of the disease 15 months after his treatment, which was conducted as part of a Phase 1/2 clinical trial (NCT02151526). The research team, led by Marina Cavazzana, MD, PhD, a hematology professor at Paris-Descartes University in France, inserted an antisickling β-globin gene, with the help of a viral carrier, into the boy’s blood-producing stem cells. The report, “Gene Therapy in a Patient with Sickle Cell Disease,” appeared in the New England Journal of Medicine.

According to a study published in the American Journal of Hematology, the presence of a multidisciplinary clinical team can dramatically reduce maternal and perinatal mortality rates in pregnant women with sickle cell disease (SCD), especially in under-developed and low-income countries. In recent decades, medical advances have significantly improved the survival of those living with SCD, which now raises a new health challenge. Pregnant women with sickle cell disease have increased risks of complications and a higher risk of mortality.

Differences in comorbidities, pain, healthcare utilization and psychosocial outcomes were addressed in three groups of sickle cell disease (SCD) patients, distinguished by their respective ages, in the Pain in the Sickle Cell Epidemiology Study (PiSCES). Other studies are needed to determine if age-specific healthcare measures might improve these patients’ lives. The study, “Comorbidity, Pain, Utilization, and Psychosocial Outcomes in Older versus Younger Sickle Cell Adults: The PiSCES Project,” appeared in the journal BioMed Research International.

New guidelines for therapeutic strategies and pain management for sickle cell disease (SCD) should be considered by clinicians and healthcare teams, according to the author of a presentation made at the American College of Physicians (ACP) Internal Medicine Annual Meeting in San Diego. Controlling all the symptoms of SCD is a great challenge and is crucial for patients. The presentation titled “Sickle Cell Disease: New Guidelines for Therapy,” presented by Jennifer Yu, MD, from the department of radiation oncology at Cleveland Clinic, provided an overview of some of the major concerns in SCD disease management and improving patient care.

The inaugural World Cord Blood Day on Nov. 15 this year will educate parents and healthcare professionals about the current uses for cord blood transplants, a non-controversial source of stem cells which have the ability to treat more than 80 life-threatening diseases, including sickle cell anemia. To date, more than 35,000 umbilical cord blood transplants have been performed worldwide since 1988. In the emerging field of regenerative medicine, this approach holds great promise in treating autism, diabetes, stroke and many others. The procedure has also been performed in patients with thalassemia, lymphoma, and leukemia, among others. But umbilical cord blood is still thrown away as medical waste after most births across the world.

Intensifying treatment with hydroxyurea in young children with sickle cell anemia is safe and improves several disease parameters in the long run, researchers demonstrated in a clinical trial (NCT00305175) comparing toddlers with older children.

Based on the positive findings, researchers argue that the practice of giving small children higher doses of the drug needs to be further studied. A new clinical trial (NCT03020615) addressing these matters is in the planning stage.

The study, “Hydroxyurea at Maximal Tolerated Dose (MTD) Prior to Completion of the β-Globin Switch Has Additive but Not Sustained Benefits in Fetal Hemoglobin Production,” was presented at the ASH 2016 Annual Meeting.