I’m handing out my “Golden Globule” awards to those sessions and presentations that stood out for me at this year’s ASH annual meeting. As fans of the much-anticipated Wicked: Part 1 or the grand finale of Taylor Swift’s “Eras Tour” know, not every leading actor or performance always wins an award — but here are the ones that shined the brightest for me.
All Too Well: Navigating the Realities of the Sickle Cell Era
In the shadow of emerging treatments, many newer gene therapies, while promising, are far from reach for most people with sickle cell disease (SCD). Optimizing Non-Curative Therapies for Sickle Cell Disease, moderated by Dr. Susan Elizabeth Creary, MD, MSc, of Ohio State University College of Medicine, presented attendees with practical guidance on getting the most out of current disease-modifying treatments, best practices on delivering acute pain management with different modalities, and real-world clinical care models. This session led to a healthy discussion regarding our current Sickle Cell Era. Dr. Creary emphasized that as providers for the SCD community, we should collaborate more with our patients, balancing optimal responses from disease modifiers and assessing for treatment failure.
As many of you know “All Too Well” (a nod to the Taylor Swift fans), managing hematologic conditions at the end of life presents challenges and opportunities for delivering evidence-based, compassionate care. Hematologic Challenges at the End of Life brought together multidisciplinary perspectives to explore key considerations such as life expectancy, symptom burden, and hospice care across hematologic diseases.
Family Matters: Breaking Barriers in Hemoglobinopathies and Health Equity
At the oral session on Determinants of Health Equity Across the Spectrum, presenters delved into the pressing issue of how socioeconomic and other determinants shape access to care and health outcomes, from newborn and maternal health to mental health and education. A unique highlight was the intergenerational collaboration between Jeffrey D. Lebensburger, DO, a passionate advocate for mentorship within ASH, and his teenage daughter, Hannah, who spoke about access to mental health care services for pediatric patients with SCD. As a mentee of Dr. Lebensburger, it was thrilling for me to see the legacy of his guidance — now a family affair — shine during this session. Not only did this discussion underscore the critical need for equitable mental health care, but it also served as a powerful reminder that it’s never too early to inspire the next generation of leaders in hematology.
The CRISPR Avengers: Gene Therapy Assembles
One of Sunday’s blockbuster sessions was Gene Editing and Replacement Therapies for Hemoglobinopathies: From Bench to Bedside. This high-powered lineup of presentations shed light on some of the latest “superhero-level” (or juggernaut?) advancements in gene therapies. Stacey Rifkin-Zenenberg, DO, shared seminal results on lovotibeglogene autotemcel for SCD. Up to 94% of patients achieved complete resolution of their vaso-occlusive events (VOEs), with the majority of patients demonstrating sustained improvement in hemoglobin (median: 12.3g/dl). Franco Locatelli, MD, spotlighted exagamglogene autotemcel for transfusion-dependent beta-thalassemia, reporting that more than 94% of patients achieved durable transfusion independence, as well as improved hemoglobin levels and quality of life — a true Iron Man move for β-thalassemia. Matthew W. Heeney, MD, introduced BEAM-101, a base-edited therapy for severe SCD. Early results have shown rapid induction of hemoglobin F (HbF), reduced hemolysis, and elimination of VOEs, positioning this therapy as a potential Infinity Gauntlet in the fight against SCD. This session left no doubt: gene therapies are transforming the landscape of hemoglobinopathy treatment, one CRISPR edit at a time.
Breaking Bad (Barriers): HbSC and the PIVOT Trial
While new therapies generate buzz, some treatments still face barriers to widespread adoption. Sunday’s plenary session, Double-Blind, Placebo-Controlled Randomized Controlled Trial of Hydroxyurea for HbSC: Results of the PIVOT Trial, aimed to change that narrative. The results are nothing short of transformative, revealing that hydroxyurea was well-tolerated, with mild and reversible side effects, patients experiencing significantly fewer VOEs and hospitalizations compared to the placebo group, and biomarkers improving markedly, including increased HbF and mean corpuscular volume. These findings underscore hydroxyurea’s potential as a game-changing treatment for hemoglobin SC (HbSC) and lay the groundwork for a future phase III trial.
Similarly, laying the red carpet for future innovation was A Novel Mouse Model of Hemoglobin-Sickle Cell Disease. Tahereh Setayesh, PhD, introduced a revolutionary HbSC mouse model, developed using CRISPR-edited humanized Townes HbSS mice to mimic the genetic and clinical traits of HbSC disease. These mighty mice are true trailblazers, exhibiting hallmark features like anemia and red blood cell xerocytosis, elevated blood viscosity, and organ damage, such as sickle retinopathy. While it may not be “Dino DNA,” this new model is a Jurassic Park-level breakthrough that will fill a critical research gap and pave the way for targeted therapies.
In my view, #ASH24 once again proved that hematology is having its own “Taylor’s Version” moment — reclaiming its narrative with bold discoveries, groundbreaking innovations, and a nod to future generations. From “Defying Gravity” at ASH-a-Palooza to wielding the “Infinity Gauntlet” of gene therapies, these sessions brought the drama, action, and heart of a cinematic blockbuster. Like the long-awaited sequel to a favorite franchise, this year’s highlights remind us why we show up: to learn, collaborate, and redefine what’s possible. Whether you’re team CRISPR Avengers or rooting for the underdog in sickle cell breakthroughs, the message is clear — hematology is not just advancing. It’s headlining.
