Health and Human Services Secretary Xavier Becerra, in remarks last month at the Congressional Black Caucus Foundation’s annual legislative conference, presented an audacious goal: to cure sickle cell disease by 2030. This was an enthusiastically welcomed statement, given that, for generations, sickle cell was largely an ignored disease, characterized by underfunded research, lack of accessible and available treatments, and poor understanding by medical professionals.
It is distressing that sickle cell disease has devastated and shortened lives for as long as it has, with minimal progress in alleviating the damage it causes. The 100,000 sickle cell patients in the United States, predominantly African-Americans, suffer chronic, often crippling pain, as misshapen blood cells block oxygen to joints and organs, and lifespans for sickle cell patients are 20 years shorter than that of the average American.
To ensure that Becerra’s goal is achieved in less than a decade, we must be realistic about the hard, focused work that has to happen, beginning immediately! We must optimize the scientific promise that exists today to develop a cure. We must ensure that any new treatments, therapies or cures for sickle cell are accessible to those needing them. And we need improved advocacy for health delivery equity for those suffering from this terrible disease.
Evolving science, however, is presenting an opportunity to strike a blow against this disease. Recent progress in gene therapy studies and clinical trials is demonstrating unprecedented promise in treating sickle cell disease. In years past, medical science could only attempt to treat symptoms with conventional drugs and chronic blood transfusions or a lucky few patients could undergo donor blood marrow transplants. Now, researchers have developed the ability to go right to a patient’s DNA and address the disease’s root cause. We’re seeing tangible results.
Clinical trials are having success with a single application of new gene therapies that restore blood cells to their normal shape in the patients participating in the trials, in many cases completely eliminating episodes of severe pain. Over the coming months and years, the FDA will have the opportunity to review these new treatments to assess their safety and efficacy.
But, as important as science will be in achieving Becerra’s goal, medical breakthroughs will do little good if patients can’t benefit from them. The history here, where sickle cell disease is concerned, is not a good one. In 2017, the Food and Drug Administration approved a new drug for sickle cell patients — the first in two decades — and insurers were reluctant to provide coverage, leaving it financially out of reach for most patients.
Leaders like Tennessee state senator Raumesh Akbari are taking the offensive to ensure this isn’t the case with future therapeutic breakthroughs. As Akbari said, “We live in too rich of a country for people who are fighting through this disease to have to worry how to pay for it.” She sponsored “Terrence’s Law,” named after someone who died of the disease and signed into law last year. It will pave the way for the state’s program to pay for future sickle cell treatments and cures. This kind of effort needs to be replicated nationwide.
And while we fight for the future, our healthcare system also needs to do a better job meeting the needs of today’s sickle cell patients. Too often, patients enter emergency rooms because of their acute pain and having delayed or denied treatments. They are being sent home to suffer because there aren’t clear protocols for supporting sickle cell disease, and doctors receive scant training on the condition in medical schools. Sickle cell patients need advocates in the healthcare system, not a lack of understanding.
All of us who have fought on behalf of sickle cell disease sufferers are thrilled by Becerra’s stated goal and the science that can make it a reality. Where the proverbial rubber meets the road, though, is that we must also be determined to make changes to a system that has been lacking in meeting the needs of this community.
People in pain should be treated with dignity and respect and shouldn’t have to fight to live a normal, healthy life when effective therapies are available.
