Edge of Tomorrow
Cedars-Sinai’s vice dean of research argues clinical trials are essential for patients, the community, and the future of medicine.
Patients rely on leading academic medical centers like Cedars-Sinai for today’s most effective treatments—and for tomorrow’s as well. Through clinical trials, we develop breakthrough therapies, novel devices, and new approaches to solving intractable health challenges. Academic medical centers must embrace and promote clinical research as the path to knowledge that serves patients locally and, ultimately, globally.
In 1946, the first modern-day clinical trial provided the original antibiotic for tuberculosis. Ever since, clinical trials have been breaking down doors marked “hopeless,” providing patients with access to new drugs, therapies, devices, procedures, and dietary approaches. Today, nearly 300,000 clinical trials are registered at ClinicalTrials.gov, a comprehensive database sponsored by the U.S. National Library of Medicine, part of the National Institutes of Health (NIH). At Cedars-Sinai, we conducted more than 500 clinical trials in fiscal year 2018, with hundreds more in the pipeline. Why?
For some diseases, the standard of care is a clinical trial. Many rare diseases, like amyotrophic lateral sclerosis (ALS) and certain types of cancer—such as malignant brain or pancreatic cancers—have no viable treatments. Rare or “orphan” diseases (affecting fewer than 200,000 individuals in the U.S.) historically have been ignored by pharmaceutical companies because of the lack of financial incentives. Collectively, however, orphan diseases affect some 25 million Americans.
Academic medical centers like Cedars-Sinai help bridge that gap by going beyond treating common maladies to also seek cures for underrepresented and challenging diseases. This effort is what draws patients to our doors from across Los Angeles and around the world.
Take one of the most common causes of death in pregnant women: preeclampsia. Maternal mortality in the U.S. is three to five times higher than in any other developed country, yet we have had no effective treatments for preeclampsia, which begins simply with high blood pressure. Private industry is unlikely to invest greatly in this complex and non-lucrative space.
Through research in Europe, our team has developed a test for early detection of preeclampsia, and we are now investigating an experimental therapy. We plan to launch a Food and Drug Administration-guided study in the U.S. in early 2019 in partnership with Sarah Kilpatrick, MD, PhD, chair of the Cedars-Sinai Department of Obstetrics and Gynecology and the Helping Hand of Los Angeles Chair in Obstetrics and Gynecology.
When standard care fails, patients need viable alternatives. For many diseases, the standard of care encompasses therapies that work for many patients but not all, or not permanently. Heart failure, for example, is met with various successful interventions: medications, bypass surgery, valve repair or replacement, and mechanical pumps. Yet about half of those with the condition die within five years of diagnosis. In short, we have a reliable standard of care, but it doesn’t always work.
Regardless of the condition—a rare disease with no treatments or a common malady with many interventions that may or may not be effective—the rubber meets the road when patients ask where they can get the maximum number of options with the greatest chance of achieving optimal health. By developing and participating in clinical trials, we can adopt new experimental therapies and deliver them to our patients more quickly.
If Cedars-Sinai and other academic medical centers fail to offer the full spectrum of experimental therapies, we limit our ability to offer hope and care to those who most need it.
Developing and participating in clinical trials benefits our community, but the relationship isn’t one-sided. To receive support for clinical trials from traditional funders—like the NIH—investigators must show background data indicating a likely benefit from the research. This creates a chicken-and-egg scenario. Researchers cannot approach funding agencies without supportive data but cannot gather data without support. Philanthropic gifts are vital to collecting initial data and launching us on the path to new breakthroughs and federal funding to extend our research.
Clinical trials also represent a two-way handshake between physicians and patients to advance better healthcare for all. Participating in a clinical trial involves inherent risk, as the treatment offered is, by definition, unproven. The burden is on investigators to be transparent about potential risks and benefits, and to ensure that patients are entering a properly conducted clinical trial. I have been impressed and moved over the years by the patients who have graciously partnered with us—not just because they hope for a cure. Many are driven by altruism. One young woman who participated in my preeclampsia research told me: “My sister had this condition. My mother had this condition. Even though this treatment might not help me personally, I don’t want women to suffer from this any longer.”
To remain relevant to their peers and vital to their patients, academic medical centers must foster a robust research practice that recognizes that the clinical trials of today will become the standard of care tomorrow.
About Dr. Thadhani
Thadhani serves as vice dean of Research and Graduate Research Education and chair of Biomedical Sciences. In addition, he is a visiting professor at Harvard Medical School and former executive director of the Clinical Trials Office at Partners HealthCare in Boston.
A world-renowned expert on vitamin D metabolism, kidney dialysis, and preeclampsia, he is also an internationally recognized clinical and translational leader in nephrology. His work has been published more than 250 times in top-tier scientific journals, including the New England Journal of Medicine, the Journal of the American Medical Association, and The Lancet. As a reflection of the highest peer recognition, he is an elected member of the American Society for Clinical Investigation and the Association of American Physicians.