As part of its broader effort to identify new treatment strategies and secure a pathway to curing sickle cell disease (SCD) globally, ASH is in the process of establishing a clinical trials network (CTN) to identify high-priority research questions and support efforts to address these questions. In June 2018, ASH brought on Dr. Charles Chesson to serve on the ASH staff as director of the Sickle Cell Disease Clinical Trials Network, where he will be responsible for the network’s creation, implementation, and effective operation. Under his purview, the network will focus on infrastructure, programs, and services to help overcome the challenges of conducting clinical trials in SCD, including a comprehensive patient engagement strategy to increase understanding of both the disease and clinical trials overall.
In this Q&A, Dr. Chesson explains more about his role and the direction of the network in the near future.
Q: Describe what inspired you to join ASH to lead the CTN? A: SCD was first described in the scientific literature more than 100 years ago, and although it was the first “molecular disease” identified (in 1951 by Dr. Linus Pauling), there are still only two approved drugs for its treatment. These are exciting times in SCD research, and the goals of the CTN couldn’t be more admirable. I was inspired by ASH taking a leadership role in this area to improve the lives of patients living with SCD.
Q: Can you share with our readers some of the history of the network? A: In 2014, ASH leadership called for increased commitment to address the global SCD burden. As a first step in the development of this initiative, ASH hosted the Sickle Cell Disease Summit to develop strategies for improving outcomes for individuals living with this devastating disease in numerous priority areas. The initiative was defined as a multipronged approach consisting of access to care in the United States, training and professional education, research and clinical trials, and global issues related to SCD. In the research and clinical trials area, the SCD CTN Task Force was created under the leadership of Drs. Charles Abrams and Edward Benz, with the goal of identifying the scope, goals, and high-priority research objectives for the CTN. In the past year and a half, the task force has received feedback and advice from SCD clinicians and researchers, CTNs in other disease areas, leaders of pharmaceutical and biotech companies, and the National Heart, Lung, and Blood Institute, all of whom underscored the need for a CTN for SCD. In May 2018 the SCD CTN was approved by the ASH Executive Committee to address research and clinical trial needs. Moving quickly, ASH recruited me as director of the SCD CTN, and my first day on the job was in July.
Q: What makes SCD such a high-value priority in the clinical trials space?A: The simplest explanation is that there are now more than 40 new drugs in development to treat SCD, yet there is no cohesive path forward for recruiting and retaining the number of patients needed to determine which drugs will be the most beneficial to individuals living with SCD.
Q: In your view, what is the core of the CTN? What will hold it together and act to fuel its success?A: Entering the initial phases of forming the SCD CTN, we are ensuring that the network’s foundation is built with patients. We are not building a SCD CTN to approve drugs; rather we are building a network to find improved treatments and a cure for SCD. We seek meaningful involvement with patients as the SCD CTN is established and hope to from a collaborative relationship between patients, their caregivers, patient advocacy groups, physicians, and academic and industry sponsors of clinical trials.
Q: Now that you are on board here at ASH, what can we expect to see in the coming months?A: The next steps will be to advance the following four areas in establishing the SCD CTN:
Patient engagement. We are preparing a request for proposals to engage with a firm that will assist ASH in discussions with the community so that we can develop a long-term partnership that is grounded on mutual trust and collaboration.
Governance. We are examining the best structure for the CTN so we have a nimble organization with the appropriate inputs and without undue bureaucracy.
Sites. We need the appropriate blend of both pediatric and adult sites for enrolling patients. We will be soliciting proposals from sites before the end of this year.
Industry engagement. We will work with the patient and scientific communities to prioritize research needs and collaborate with industry to see how we can match those priorities.
Q: What is your message for members who would like to get involved and help spur progress in the arena?A: As we start to build this network, we want to hear from the SCD stakeholder community, especially those who are interested in SCD clinical trial activities and who would like to volunteer their expertise to assist with ongoing efforts. If you are interested in learning more about volunteer opportunities or how to become an official site for the CTN, please contact us at SCDCTN@hematology.org.
