Sickle Cell Disease: Promises for a brighter future

Sickle Cell Disease: Promises for a brighter future | Image Credit: © Oleksandr - © Oleksandr - stock.adobe.com.

Children with a diagnosis of Sickle Cell disease (SCD) or transfusion dependent Beta-thalassemia (TDT) face a challenging lifestyle with potential for immediate medical interventions at any given moment in time, that disrupts their physical and emotional health, their day to day lives, their family lives, and creates a steady state of stress. Ehmsen, Weinstock, & Simeonova discuss the new genetic therapies in their article, “Novel genetic therapies for sickle cell disease and B-thalassemia,” that were recently approved for children and adults by the FDA.1 The authors further report that the two FDA approved drugs are the first gene-editing therapy to treat human diseases.1 The drugs, exagamglogene sutotemcel and lovotibeglogene automecel were both approved for children 12-years and older and adults with a diagnosis of SCD, while lovotibeglogene automecel was investigated for treatment of TDT.1 I highly recommend reading the article to better understand these new drug therapies and the long term follow-up studies that will continue to investigate the clinical outcomes for those who receive the drugs.

The promise of hope for a cure

How promising and elating it must feel for parents of children with diagnosis of SCD or TDT to hear the words, “the goal is a cure”. The safety and efficacy profiles for the drugs during clinical trials for both drugs met the strict FDA criteria for approval even while a 15-year follow-up study continues.1 I am confident that the health care providers who provide care for the children with a diagnosis of SCD or TDT every day, also feel elated for this breakthrough. In sharp contrast, how devastating will the outcomes of this goal be, if it is not attainable for all children and eligible adults if they cannot even dream of a personal cure due to the extraordinary expense of the drugs, currently estimated to be over $2 million dollars per patient? 1 As health care providers we understand the time, effort, and expertise required to develop such novel, innovative, life altering therapies, but we also understand the adverse consequences for those who are unable to access treatments that are cost prohibitive. Herein, also is the time to implement strategies for the hope of health equity for all.

It is time to be a strong child advocate

The first thought that emerges is: how can we, as strong and persistent advocates for children and health equity, effectively remove barriers for accessing care that is driven by a dependency on financial resources? Child advocacy requires each one of us, our organizations, our legislatures, the children and families, and our communities to join together to solve the financial restraints for access to care in which “the goal is a cure”! How many times in our lifetime will we be able to proactively work together to achieve the promise of hope for a cure and a life without pain and suffering for children in our nation and worldwide?

Reference:

1. Ehmsen JT, Weinstock NI, & Simeonova P. Novel genetic therapies for sickle cell disease and B-thalassemia. Contemporary Pediatrics, 2024;40(6):10-12.