Margarida Maia, PhD,  science writer—

An ongoing, early-phase clinical trial of pociredir, an oral small molecule being developed by Fulcrum Therapeutics as a possible treatment for sickle cell disease (SCD), is still recruiting adult patients at locations in the U.S. and one in South Africa. This Phase 1b trial, called PIONEER (NCT05169580),…

Vascarta is launching a proof-of-concept Phase 1 clinical study to test its topical curcumin gel VAS-101 — which uses a component of turmeric — in people with sickle cell disease (SCD). The experimental therapy, also known as Vascepto, is designed to be applied onto the skin. It contains curcumin,…

People with sickle cell disease (SCD) who are hospitalized for opioid overdose have fewer serious complications than those without the condition, but face longer hospital stays, higher costs, and unique healthcare challenges that require specialized care, a study found. “A multidisciplinary approach to pain management, tailored to individual patient…

It may be possible to collect nearly three times as many stem cells for gene therapy for sickle cell disease (SCD) with BioLineRx’s motixafortide than with standard plerixafor, and even more when it’s combined with natalizumab, a Phase 1 study shows. The proof-of-concept clinical trial (NCT05618301) is…

The U.S. Food and Drug Administration has granted orphan drug status to Kind Pharmaceuticals’ investigational oral small molecule AND017 to treat sickle cell disease (SCD). This designation is granted to potential treatments for rare diseases, or those affecting fewer than 200,000 people in the U.S. It provides incentives like…

Preschool-aged children with sickle cell disease (SCD) living in so-called food deserts, or areas where access to healthy food is limited, and those without reliable transportation are at a higher risk of experiencing health issues than other youngsters with SCD, even when they receive good medical care, a study…

The number of stroke cases among adults and children with sickle cell disease (SCD) continues to increase in at least one U.S. state, indicating a need for greater adherence to standard-of-care blood transfusions and more attention paid to factors that can be controlled to reduce the risk of…

Using lactated Ringer’s solution instead of normal saline to replace lost fluids in adults admitted to the hospital for vaso-occlusive crises, that is, pain episodes caused by sickle cell disease (SCD), allows for shorter stays, fewer readmissions, and a reduced need for opioids, a study finds.  Dehydrated…

Low-intensity stem cell transplants, which use milder conditioning regimens, appear not to damage the lungs and may even improve lung function in adults with sickle cell disease (SCD), according to a new study led by scientists from the National Institutes of Health (NIH). “By using a low-intensity blood…

A second clinical study to test how well BioLineRx’s motixafortide helps mobilize hematopoietic stem cells for use in gene therapies in sickle cell disease (SCD) is expected to begin enrolling in the next few months. The planned Phase 1 study, called SCDSTEMM (NCT06442761), will run as a…