Imagine holding a cube of ice with no way to relinquish it, or being unable to remove a knife that's constantly piercing an open wound.
These are some of the ways Marie Ojiambo described the "excruciating" pain of suffering from sickle cell disease, a disorder that causes red blood cells to change shape in ways that block proper blood flow and oxygen from traveling throughout the body. There are millions of black people around the world who are disproportionately affected by this disease.
World Sickle Cell Awareness Day, which was on Sunday, is meant to raise public knowledge and understanding of the crippling condition. In effort to carry on with that mission, Pfizer shared news with reporters at a roundtable discussion on June 6 in New York about promising new research it has conducted that could help millions of sickle cell patients around the globe.
Pfizer isn't the only company looking to improve treatments for the disease. Selexys and bluebird bio are two pharmaceutical companies that have both designed similar studies to treat sickle cell. But these companies all claim to run into a pretty significant problem: Difficulty enrolling black patients in the clinical trials.
These trials help to test the efficacy of the medications pharmaceutical companies hope to develop for the disease but because of a confluence of factors, from distrust of doctors to logistical difficulties, the enrollment rates of black patients have been low.
“Clinical trial participation is a challenge in general,” Dr. Freda Lewis-Hall, Pfizer's chief medical officer, said. “There are very special and unique problems in the sickle cell community. Only one in seven people in the U.S. know someone who has actually participated in a clinical trial and only one in five people say their physician has ever talked to them about a clinical trial. This is an elite experience, in general. When you get to the African-American community, it is an even more elite experience.”
The tendency of the black community to participate in clinical trials at far lower rates is arguably because of the community's mistrust of doctors, which was instilled from unethical studies done in the past. The Tuskegee experiment was one particular controversial and horrifically unethical study on Syphilis that played a major role in this divide. The experiment, which was conducted from 1932 to 1972, involved 600 impoverished black men who were intentionally withheld from treatment of the disease by doctors who sought to see the condition take its full course in black bodies.
“If this [trial] doesn’t make it because we can’t find and encourage enough patients to participate, for me, that would be a crying shame." Dr. Freda Lewis-Hall
The Tuskegee experiment, which was later turned into a film, forever left an impact on how black people perceive clinical studies and tainted their overall attitudes towards treatment from doctors, said Sonja L. Banks, president of the Sickle Cell Disease Association of America.
“There are lot of things I hear, one is of past studies and the history about the Syphilis study,” Banks said of the barriers to getting black people to participate in clinical trials. “But the majority of individuals with sickle cell are African-American or minority. Who’s gonna help? Who’s going to make these drugs come to fruition. It’s gotta be us. If we don’t participate, we will never find treatment or a cure.”
Banks added: “There’s not enough white people to get in trials to help you. Let’s get educated, [let’s] go back to the syphilis study and look at what happened. That will never happen again, because we’re too much of an educated community now. We can get educated enough to make an informed decision.”
Lewis-Hall stressed that at the end of the day, "we have got to find a way to increase diversity in clinical trials."
“There’s not enough white people to get in trials to help you." Sonja L. Banks on the desperate need for black participation in clinical trials
One of the ways to possibly alleviate the low participation from the black community and the cultural context behind mistreatment in the medical research community is to re-educate people about these past studies, according to Lewis-Hall.
“In the 100 years since sickle cell was discovered, there’s only one FDA approved drug and it’s not even for sickle cell,” Banks said. “Our people are still going to emergency room as their medical home. It’s all very heart-wrenching.”
Doctors said phase 2 of Prizer’s trial showed that the medication they developed decreased a patient's hospital stay by 55 hours and that individuals in the trial decreased their opioid use by 89 percent in the first 24 hours. However, the company is still seeking ways to enroll more patients to participate in the current and future phases of the study and help work towards a viable solution.
“If this [trial] doesn’t make it because we can’t find and encourage enough patients to participate, for me, that would be a crying shame,” Lewis-Hall said.
Ojiambo, who justly describes herself as a “sickle cell warrior,” said people in this particular patient population are “very, very desperate for a cure." She said she believes that advocates, like herself, play a crucial role in educating others about the way patient safety is prioritized in studies like Pfizer's.
“We need to talk more about the pros of the clinical trial studies," she said. "We need to demystify the process.”
Pfizer’s target is to enroll 350 patients in its study, which could take a few years, doctors said. They also said they plan to better fulfill patient's needs by providing comfort and ease throughout the process, should patients decide to participate in the trial.
"One of our my favorite advocacy statements is: 'Nothing about us, without us,'" Lewis-Hall said. “We need you, we don’t intend to do anything about you without you, but we need you here working with us.”