Sickle-cell, Gene Fixes, and Health Justice

Ideas and Issues

New Orleans       Gene therapy is both the wave of the future and a huge ethical quandary of almost unimaginable complexity.  A young Chinese doctor, who attracted international publicity by becoming the first to perform gene therapy in the womb on a set of twins to potentially provide immunity to a disease to which they were genetically susceptible, is now facing sanctions and legal problems as the Chinese government reacts to protests from virtually all quarters.  There are debates that surround all of these issues.  Will the rich be allowed to pay to have “perfect” babies?  Will genetic tests be allowed for all and therapies provided for genetic diseases from in utero forward?  The questions are thorny and endless at this point, especially since so few outside of the medical field have a firm grasp of the potential and the current state of play.

It was exciting to read an article in Scientific American that focused on the devastating sickle-cell disease and quoted researchers and doctors arguing that, “Gene therapy might offer a cure for sickle-cell disease, and clinical trials are already underway.”  Wow!  Anyone who has worked in lower income communities heavily populated by minorities, both in the US and globally, knows people well who have suffered – and died – from this disease.  No one is immune.  I first learned of this disease at twenty years old from an early president of Massachusetts Welfare Rights Organization’s son who was afflicted.   Later, I was close at hand when the grandchild of the president of our union local in Louisiana, and the daughter of one of ACORN organizers and leaders in New Orleans, were afflicted and endured constant crises.  One was fortunate enough to have a bone marrow transplant, which is about the only current cure, but gene therapy could help many more victims of the disease since it would rely on stem-cells from the individual rather than a donor.  Because the therapy involves only essentially one gene, a cure is fully realizable, everything being equal.

Unfortunately, nothing is equal, leading one doctor to be quoted saying “sickle-cell disease represents the best and worst of health care in the United States.”  The disease itself is ghettoized, making demographics destiny in a deadly way.  One expert “estimates that at least 30% of his adult patients with sickle-cell disease die from preventable causes…but the lack of infrastructure, such as care centers” leaves gaps in monitoring and treatment with fatal consequences.  The hope of gene therapy for sickle-cell is then deflated by concerns that the infrastructure that doesn’t exist now, would also not exist in the future to achieve the cure.

And, then there’s the cost.  Experts estimate that gene therapy would run from $500,000 to $700,000 over a couple of years.  It would save money over time, but obviously the costs would come on the frontend in achieving the cure.  It is also a huge public policy problem because 50% of the patients in the US being treated for sickle-cell are covered by Medicare or Medicaid.  As one doctor says, the problem is “money and ethnicity.”

The Center for Disease Control estimates that 100,000 Americans suffer from sickle-cell.  In urban communities where many of us work, we know the pain is permanent, and then there is death.  Increasingly, we are also going to know that there is a pathway to a cure here that could upend a tragic inheritance now and in the future.

There needs to be equity and justice in healthcare,  just as we demand it everywhere else.