Mixed Feelings Over Sickle Cell Cure
Fears of losing one’s identity, and facing the ‘unknown.’
Terry Jackson lives a life dominated by sickle cell disease. The genetic disorder, in which misshapen red cells become wedged in blood vessels, causes him daily bone pain and lower back pain and has sent him to the hospital for pain treatment and life threatening emergencies for five decades. He has frequent transfusions of fresh blood.
The disease has directed his every choice. It led him to get a doctorate in genetics from Duke University in North Carolina because he became fascinated with the science behind it. It is central to his identity.
“I ask myself, ‘Who am I without sickle cell?’ ” said Dr. Jackson, who owns a science communication business. “It’s hard to even imagine what I would do, what would I be, if I don’t have it any more.”
This year, Dr. Jackson and others may have the option of finally living without the damage the disease causes. Two drug companies are seeking approval from the U.S. Food and Drug Administration for gene therapies that may provide what amounts to a cure. But the decision to undergo the therapy, should it become available, is not so simple.
After a life adapted to their illness, some like Dr. Jackson are unsure of how to begin again as healthy people. Others fear that the logistical complexities of gene therapies may imperil their ability to access them.
Sickle cell disease affects millions worldwide. It mostly strikes Black and Hispanic or Latino people, but it also occurs in people with Mediterranean and Indian ancestors. People with the disease face searing pain, stroke, damage to tissues and organs and often death at an early age.
Two gene therapy drugs, one from Bluebird Bio and the other by Vertex and CRISPR Therapeutics, have been in clinical trials. Both efforts reported that patients were freed of debilitating episodes of pain. Their blood was no longer flooded with misshapen red cells.
Gene therapy must be paid for all at once. Concerns about the high price worry many people with sickle cell, as well as their families. The modified virus that delivers gene therapy is expected to cost at least $1 million, based on what other gene therapy vectors cost.
But that is only part of the price.
Treatment starts with intense chemotherapy in a hospital to wipe the bone marrow clean, leaving space for genetically modified red blood cells that result from the treatment. Patients then spend about a month in the hospital waiting for the modified cells to grow. After they are discharged, the patients are immunocompromised for about six months.
Insurers in the U.S. are likely to pay for the part of the treatment that involves using a disabled virus to deliver the therapy. But whether they will pay for most of the chemo, hospitalization and other costs is uncertain.
And how easy will it be for patients and family members who assist them to take off a month or more for the treatment?
Without the disease, said Teonna Woolford, 31, a sickle cell patient and advocate who lives in Owings Mills, Maryland, patients “don’t know who they are” and many worry about “trading one familiar issue — sickle cell — with something else and overall fear of the unknown.”
Dr. Jackson said he would reject the cure in part because sickle cell is so much a part of his identity that he cannot imagine life without it.
But others cannot wait for gene therapy. Jimi Olaghere of Atlanta, Georgia, participated in the Vertex and CRISPR trial about three years ago, when he was 35. His life is transformed, he said: “The daily pain I used to face — literally round-theclock pain — that’s gone.”
Mr. Olaghere did experience “an identity crisis,” when he had to make a life without sickle cell, he said. But now all of his suffering, except, he says, for “mental health challenges,” is gone.
“It feels,” he said, “like a miracle.”