VJHemOnc Podcast

Key updates in thalassemia at ASH 2023: gene therapies, ongoing trials & the value of precision medicine

Jan 31, 2024

Thalassemia experts discuss gene therapies, ongoing trials, and precision medicine for thalassemia treatment. Updates on pharmacologic therapy, complications in adult patients, and treatment options for aging patients. Precision therapy and stem cell depletion are also explored.

12:22

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Episode guests

Maria Domenica Cappellini

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Quick takeaways

Novel gene therapies, such as CRISPR-Cas9 and CRISPR-Cas12a, show promise in improving quality of life and potential transfusion independence for thalassemia patients.

Pharmacological treatments like luspatercept and PKE activators offer potential benefits in improving outcomes and quality of life for thalassemia patients with ineffective erythropoiesis.

Deep dives

Advancements in Gene Therapy for Thalassemia

The podcast highlights the exciting updates in thalassemia presented at the 2023 Ash annual meeting. Experts discuss the promise of novel gene therapies, such as CRISPR-Cas9 and CRISPR-Cas12a, and their long-term benefits, including improved quality of life and potential transfusion independence. The recent FDA approval of CRISPR-Cas9 for sickle cell disease suggests it may also be approved for thalassemia. Additionally, pharmacological treatments like luspatercept and PKE activators show promise in improving outcomes and quality of life for patients with ineffective erythropoiesis.

Introduction

3min

Updates on Pharmacologic Therapy for Thalassemia and the Importance of Precision Medicine

3min

Complications and Morbidities in Adult Thalassemia Patients

3min

Treatment options for aging thalassemia patients and the importance of selecting the right candidates

2min

Precision Therapy and Depletion of Stem Cells in Thalassemia

2min

In the last decade, the treatment landscape of thalassemia has been transformed with the introduction of novel therapies. With several agents being evaluated in clinical trials, the future looks promising for patients. Despite these advances, there are challenges and unmet needs which remain, and both clinicians and researchers are working to address these, with the goal of improving outcomes for patients.

In this podcast, you will hear from experts Maria Domenica Cappellini, MD, University of Milan, Milan, Italy, and Kevin Kuo, MD, MSc, FRCPC, University Health Network, Toronto, Canada, who discuss recent updates in thalassemia presented at the 2023 ASH Annual Meeting. The experts cover several topics, including the promise of novel gene therapies, the BEYOND trial (NCT03342404), and more.

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