Clinical trials of sickle cell disease (SCD) treatments that use the gene-editing technology CRISPR could start as soon as this year. However, signing up African-Americans, a population with a legacy of medical disenfranchisement, may be more difficult than researchers realize, according to a STAT News investigation. SCD affects roughly 100,000 Americans – mostly individuals of African descent – and is the country’s most common genetic disorder. Yet, the U.S. Food and Drug Administration (FDA) has approved only two treatments: hydroxyurea in 1998 and L-glutamine oral powder nearly 20 years later in July 2017. In contrast, hemophilia B, which affects approximately 20,000 Americans (mostly white men) has about 30 approved drugs.
African-Americans are also haunted by a history of unethical medical experimentation typified by the infamous Tuskegee syphilis study, in which the U.S. Public Health Service misled black male participants and allowed them to go untreated for decades in order to study the disease’s progress. Although the project was shut down in 1972, African-American turnout for clinical trials has remained low.
Earlier this year, ministers and researchers met at the “Freedom with CRISPR” conference in Berkeley to discuss how to best reach out to African-Americans and assuage their unease about enrolling in clinical trials. Mark A. Dewitt, PhD, lead author of a 2016 paper showing that CRISPR could be used to correct sickle cell mutation in mice, attended the conference with an open mind. He is part of a team hoping to ready the therapy for human trials in the next few years. Spending a few days with faith leaders, he told STAT News, “made him realize that his team needs to start early to get out the word about CRISPR clinical trials and must engage potential volunteers in a dialogue.”
Switzerland’s CRISPR Therapeutics plans to begin clinical trials this year for an SCD therapy that edits a patient’s stem cells so they produce more fetal hemoglobin, which can reduce the severity of the disease.
Source: STAT News, February 21, 2018.
For an in-depth look at CRISPR and the concerns about unintended side effects of gene editing, see the ASH Clinical News feature, “Demystifying Gene Editing With CRISPR.”