The Cure Sickle Cell Initiative (CureSCi; ) was created by the National Heart, Lung, and Blood Institute to accelerate the development of treatments aimed at a genetic- based cure for sickle cell disease (SCD), and ASH has partnered with the Initiative in an effort to drive this goal forward. The mission of CureSCi is to use emerging safe and promising genetic therapies to improve the lives of individuals with SCD; to actively engage the SCD community of patients, family members, caregivers, and advocates to work together on a path to the cure; and encourage collaboration among researchers, industry, non-profit organizations, and policy-making agencies who will play a role in curing SCD. Edward J. Benz, MD, is the Initiative’s executive director, and he shared with ASH News Daily his thoughts on what lies ahead for the Initiative and its role in moving toward a cure, plus what researchers and clinicians can do to make an impact.
ASH News Daily: Why is now the time for CureSCi?
Dr. Edward Benz: My answer is that it is about time, because we have understood the fundamental genetic cause for over 50 years, but still have only two drugs each only partly effective for treating sickle cell disease. Fortunately, our ability to control how globin genes are expressed in a therapeutic fashion has advanced to a stage that we can actually conduct clinical studies of gene therapy and gene editing for curing sickle cell disease. CureSCi is focused on accelerating the advancement of genetic therapies by mobilizing novel and flexible convening and funding mechanisms to support practical efforts to develop cures.
AND: Who needs to be included in this effort and why is their involvement so critical?
EB: This effort requires involvement and active, high-impact participation of many, many stakeholders and constituencies: investigators, patients and patient advocacy groups, providers, government agencies, payors, and the biotechnology and pharmaceutical industries. We have formed working groups both to engage each of these constituencies, and to promote interactions and cross-fertilization among them. CureSCi has also engaged experts to conduct a comprehensive clinical and economic impact analysis to enhance our understanding of the benefits and risks as well as health status for individuals receiving currently available treatments, as well as emerging therapies, which, hopefully, can be administered just once for a durable cure. Each of our constituencies, and the Initiative itself, needs to be informed about the needs, concerns, and possible contributions of the others in order to address these complex interlocking challenges.
AND: What are some the technological and scientific opportunities that the Initiative can help advance?
EB: Advances in gene therapy and gene editing technologies make the ultimate goal of durable curative therapies possible in the foreseeable future. Our job at CureSCi is to use our convening power and strategic application of material resources to support efforts that will accelerate progress in these areas to promote the safety, efficacy, and wide applicability of these modalities. We are supporting the most impactful applied efforts such as biomarker and endpoint research; clinical and eco- nomic analysis; resources for data collection, warehousing, and stakeholder access; development of improved delivery vectors and mechanisms; and access to erythroid stem cells from both non-SCD individuals and patients with the disease. We are also supporting efforts to advance some of the more regulatory and administrative aspects of rapid deployment of these therapies by engaging regulatory agencies and payers.
AND: What are some the key activities for the next few years?
EB: CureSCi has organized a comprehensive set of resources in the form of expert panels, outreach, and review mechanisms, to advance on a broad front. We have formed collaborative partnerships with key groups sharing our goals such as ASH and the California Institute for Regenerative Medicine while also increasing our engagement with advocacies and community groups to provide education and enhanced access to trials. We anticipate that the clinical and economic impact analysis will require about another year to complete. We have already awarded support for some key technology and applied research projects necessary for accelerating scientific and clinical elements of therapies, such as better vector design and delivery, analysis of clinical, biochemical, and hematologic and genetic endpoints, and superior clinical trial design.
AND: How can researchers and providers help advance the work of the Initiative?
EB: It is gratifying to see the tremendous amount of work focused on genetic therapies that is already underway. The initiative must leverage, support, and synergize these many activities, and not reinvent the wheel. We know that it is unlikely that there will be just one curative strategy that is suitable for all patients. Therefore, we encourage investigators and providers to be in touch with us about potential innovative approaches, ideas, and proposals. We also urge patients and providers to engage with us so we can be certain that efforts that we support and foster are responsive to the needs and concerns of the people who will ultimately be most affected.
The Cure Sickle Cell Initiative welcomes input from the sickle cell community. For more information on activities and funding opportunities, please visit www.curesickle.org.