For 39 years, I have lived with sickle cell disease. As of late, the sickle cell community has heard a lot of excitement about gene therapy’s potential to be the cure we’ve all been waiting for. That potential took a step toward fulfillment Friday when the Food and Drug Administration approved Casgevy from Vertex Pharmaceuticals’ and Lyfgenia from Bluebird Bio, both gene therapy treatments for people with sickle cell.
Yet I find myself teetering between excitement and skepticism. I am thrilled that research for sickle cell disease treatments and possible cures has blossomed. However, I worry that the celebration around this approval is ignoring a significant barrier: a historic legacy of medical mistrust.
The Tuskegee Syphilis Study and the story of Henrietta Lacks are haunting chapters in the history of medical research. But as Harriet Washington’s excellent book “Medical Apartheid” describes, these are not the only historical medical traumas affecting the Black community’s relationship with health care, the drug industry, and providers alike. The unethical and inhumane treatment of our ancestors has sown seeds of doubt and mistrust that persist to this day, with three-quarters of Black Americans saying they are familiar with the misconduct in the Tuskegee Study and that it contributes to a collective hesitancy when it comes to embracing medical advancements. This backdrop cannot be ignored as we witness the rapid development and commercialization of gene therapies for sickle cell, a disease that, in the U.S., primarily affects Black people. One study found that in 2016-2018, more than 93% of those with sickle cell who were hospitalized were Black.
Many of us living with sickle cell have learned to live with the disease. We understand personalized triggers and how to handle them. As a mother, my primary goal is to maintain my health and avoid debilitating crises with the least possible risk. And, as I’ve heard many individuals people impacted by this disease say, sickle cell disease is the devil I already know and live with. Why would I risk exchanging it for another?
Opting for a new therapy is not an easy decision. Gene therapy requires long hospital stays, meaning time away from families, children, and work, constant support from staff and doctors, and astronomical medical bills, not to mention the associated medical risks, including infertility, increased cancer risk, and treatment-related mortality.
And those who live with sickle cell disease know that the more time you spend within the medical system, the more opportunities you will face for being misunderstood, mistreated, or overlooked. A recent study found that 50% of individuals people living with sickle cell disease wait more than 120 minutes in the emergency room before their pain is treated.
If the companies developing these gene therapies want to succeed, they need to understand and have compassion for the challenges patients face every day, both in receiving basic care and in navigating a health care system riddled with biases and systematic racism. Right now, I often see the health care system as a potential adversary instead of an ally in many places.
Building a stronger health care system for patients has to start nationally, with companies working outside of the research vacuum. It requires going beyond building an advocacy initiative where representatives come to wave at the community occasionally and donate small amounts of money to support events.
Instead, the medical and scientific communities must actively engage in open and transparent dialogues with the public. The relatively low trust that Black individuals have in the health care system puts them at greater risk of adverse outcomes such as infrequent doctor-patient interaction, reduced utilization of health care services, and poor clinical relationships. While a recent survey found that two-thirds of Black adults in the U.S. see clinical trials as very important, many still feel nervous about participating in them given the past mistreatment of Black Americans in medical research. Researchers must prioritize community involvement, seeking input and collaboration from those directly affected by genetic disorders, to ensure that the benefits of gene therapies are not only effective but also accessible and equitable.
It is also essential for health care professionals to educate the public about the rigorous ethical standards and oversight mechanisms in place today. Transparent communication about the safeguards in gene therapy research can help dispel fears rooted in historical injustices and empower individuals to make informed decisions about their health care. It is not enough to develop groundbreaking therapies in isolation; we must include the voices of those directly affected by the disease. Patients must have a seat at the table from the beginning. We cannot just be a box to check off in the process.
I am hopeful that all of this can happen across the nation. I’m hopeful because I’ve seen it firsthand. My organization, The Hills Tandem, has worked with the Sickle Cell Disease Foundation and the Center for Inherited Blood Disorders to create Networking California for Sickle Cell Care (NCSCC). NCSCC established 12 clinics across the state of California to deliver comprehensive care to adults with sickle cell disease. NCSCC is a patient-led network focused on advancing sickle cell care through collaboration, advocacy, community engagement, and innovation. The establishment of this network demonstrates a paradigm shift, proving that patient voices are integral to creating a health care system that truly meets the needs of those it serves. Patient trust has grown tremendously, and it shows in numbers. Currently the network serves about 1,000 adults, an 18% increase since the beginning of the year. This growth underscores the network’s commitment to providing compassionate and trusted care to the community and we expect to see this number continue to grow.
Ultimately, the success of gene therapy depends not just on scientific efficacy but also on the strength of the trust between the sickle cell community and the health care system as well. Only then can we truly celebrate the promise of a cure without leaving anyone behind.
Jennifer Fields is the founder and president of The Hills Tandem and the Implementation Strategy Consultant for the Sickle Cell Disease Foundation.