Through Clinical Trials, You Can Help Introduce Much Needed Therapies for Sickle Cell

By Kate Silver

The road to treating disease is a complex one, requiring years of research and rigorous clinical trials. Sickle cell disease (SCD) is one condition that scientists are working to understand. As with all diseases, the quest for new SCD treatments depends heavily on volunteers. To gain FDA approval, therapies must be tested on willing participants who have the condition.

Thought to impact approximately 100,000 people in the United States, SCD is a group of blood disorders that are inherited. It’s a disorder that disproportionately impacts black people—about 1 in 12 people of African descent carry the trait.¹ People with SCD have red blood cells that take the crescent shape of a sickle, while healthy red blood cells are usually round. The sickle shape and the hard, sticky texture of the cells make it more difficult for them to move through the body’s tiny blood vessels. They tend to clog the blood flow or die off more easily.² This can lead to serious problems, such as chronic pain, anemia, acute chest syndrome, vision loss, deep vein thrombosis, pulmonary embolism, leg ulcers, infections, and other complications.³

Today, blood transfusions and bone marrow transplants are sometimes used to treat patients, but there is no cure for SCD.⁴ Pfizer is working on new therapies and conducting clinical trials to bring these therapies to the SCD community.

One challenge to enrolling patients in clinical trials is that patients often don't know about them or aren't discussing them with their doctors. According to a study by the organization Research America, a nonprofit education and advocacy alliance dedicated to research in health, 72 percent of Americans said that they would participate in a trial if their doctor recommended it, but only 22 percent said that a health care professional talked about clinical research.⁵

Another challenge to clinical trials is patients' attitudes. To understand that, Pfizer joined forces with the National Newspaper Publishers Association and academics from Howard University to conduct a poll on clinical trials and SCD, which found that more than three-quarters (76 percent) of respondents had positive or neutral attitudes toward SCD clinical trials.⁶ Further, the respondents indicated that they would be willing to participate in such a trial if they felt informed and if it was recommended by a health care professional. The results suggest that the long-standing historical perception of distrust toward medical professionals among African Americans is no longer true. In fact, 91 percent of those surveyed indicated that they believe health care professionals to be trustworthy.

This is all important news for future SCD clinical trials. Many people don’t realize that it typically takes a drug nearly 12 years to progress from its moment of discovery in a lab to the time that it reaches patients.⁷ In that time, there’s rigorous testing and screening that’s performed on the drug. When it reaches the clinical trial stage, the therapy is tested on volunteers to understand its safety and effectiveness, as well as risks. During that time, researchers adhere to strict guidelines to protect the safety of participants. Participants are provided with information about the trial that can help them understand the potential risks and possible benefits. Patients sign an informed consent document stating that they received information on the study and they understand the process.⁸ Often, the costs of the clinical tests are covered by insurance—just as any treatment would be.⁹ The patient will continue to see his or her usual health care provider during this time, as well.

Think of any prescription drug you’ve ever taken, and you can rest assured that it’s gone through a rigorous clinical trial and made its way to your medicine cabinet, thanks to volunteers who were willing to be a part of a study. If those people hadn’t participated, it’s possible that the drug would have languished on the lab floor, unable to help those who need it most.

In the case of SCD, there’s still work to be done. One out of 365 African Americans is born with SCD.¹⁰ Participation in a clinical trial could leave a lasting impact for generations to come and could help bring needed therapies to those living with SCD.

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¹ Centers for Disease Control and Prevention. Data & statistics. https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed August 23, 2018.

² Pfizer. Sickle cell disease. https://www.pfizer.com/health-wellness/disease-conditions/rare-diseases/.... Accessed August 23, 2018.

³ Centers for Disease Control and Prevention. Complications and treatments of sickle cell disease. https://www.cdc.gov/ncbddd/sicklecell/treatments.html#dvt. Accessed August 23, 2018.

⁴ Mayo Clinic. Sickle Cell Anemia. https://www.mayoclinic.org/diseases-conditions/sickle-cell-anemia/diagno.... Accessed August 23, 2018.

⁵ Research America. New national public opinion shows majority of Americans would participate in clinical trials if recommended by their doctor. https://www.researchamerica.org/news-events/news/new-national-public-opi.... Accessed August 23, 2018.

⁶ Pfizer. National sickle cell poll of African Americans dispels long-held views. https://www.pfizer.com/news/press-release/press-release-detail/national_.... Accessed August 23, 2018.

⁷ Greig, NH, Mohs, RC. Drug Discovery and Development: Role of Basic Biological Research. Alzheimer’s & Dementia: Translational Research & Clinical Interventions. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5725284/. Accessed August 23, 2018.

⁸ ClinicalTrials.gov. Learn about Clinical Studies: How are patients protected. https://clinicaltrials.gov/ct2/about-studies/learn#HowAreParticipants. Accessed August 23, 2018.

⁹ National Cancer Institute. Insurance coverage and clinical trials. https://www.cancer.gov/about-cancer/treatment/clinical-trials/paying/ins.... Accessed August 23, 2018.

¹⁰ Centers for Disease Control and Prevention. Data & statistics. https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed August 23, 2018.