Clinical Trials: The Lifesaving Power of Increased Treatment Options
Feb 21, 2016 04:55PM
By MED Magazine
By Alex Strauss
Biomedical research studies conducted on human participants are universally known in the profession as “clinical trials”. But for the President of Research at Sanford Health, the term is just too limited.
“I prefer the phrase ‘increased treatment options’, because that’s what clinical trials really are,” says David Pearce, PhD, Senior Scientist with Sanford Children’s Health Research Center and director of the Pearce Lab.
“Biomedical science and medicine are always advancing. When you are treating a patient, you have the standard of care. But [through clinical trials] you also have the ability to offer them the most up-to-date technology or intervention for their malady that may eventually become the standard of care.”
While bench research can take place almost anywhere innovative thinkers and a well-equipped laboratory exist, clinical trials require a sufficient number of eligible human participants and, often, significantly more funding. Less than a decade ago, there were few of these “increased treatment options” available in the South Dakota region. But as the area’s population has grown and the number of qualified physician scientists and researchers has increased, institutions like Sanford have committed more resources to clinical trials and have been rewarded for their efforts.
“Sanford has expanded Sanford Research because there was a feeling that our research needs to impact patients,” says Dr. Pearce whose own research has focused on the molecular basis of inherited pediatric neurodegenerative diseases. “We have basic and translational research, but we have also invested in advancing our clinical research.”
The effort got a major boost when Sanford was admitted into an elite group of 34 healthcare institutions that are part of the National Cancer Institute’s Community Oncology Research Program. NCORP sites participate in NCI cancer prevention, control, screening and post-treatment surveillance clinical trials. Sanford Health’s large footprint means that many of the region’s cancer patients are able to take part in large-scale national trials they might never have been able to access just a few years ago .
Clinical trials lead to more clinical trials in a sort of snowball effect. An institution’s involvement in NCORP-style community based clinical trials can, as they have in Sanford’s case, open the door to what Dr. Pearce calls commercial trials. A growing number of pharmaceutical companies and device manufacturers now look to South Dakota as a good place to run an FDA trial. “Reputation is everything in research,” says Dr. Pearce.
The third category of trials happening with increasing frequency are those initiated by local clinicians or researchers. “Every year, we have had more applications to the FDA generated by us, our own intellectual ideas,” says Pearce. “In these cases, one of our own investigators has developed an approach or a compound.”
Beyond the obvious advantages of increased treatment options for area patients and their physicians, the availability of more clinical trials has had another positive effect. “It allows us to attract some fantastic physicians in oncology, radiation oncology, breast surgery, and a number of other areas. These doctors want to be able to offer the very latest treatment options to their patients. So it really has a knock-on effect,” says Dr. Pearce.
Although Sanford makes an effort to keep providers up-to-date on the various open clinical trials, like other area institutions, it also lists all available trials on its website for easy reference by any physician looking for an alternative or, in some cases, an add-on to the standard of care for patients who have few options.
“It is important to remember than a clinical trial is not an experiment and that a treatment has already been extensively tested in the laboratory before it is even approved to be tried in people,” says Dr. Pearce. “My message to clinicians is that you should always consider these additional treatment options and clinical trials as possibilities for your patients who qualify.”
New and Notable Sanford Research Trials
A Vaccine for Ebola
With ebola continuing to resurface in the wake of last year’s outbreak in Africa, research being conducted by infectious disease physician and researcher Gerard David, MD, is especially timely. David and his colleagues are conducting a Phase 3 randomized placebo controlled multicenter safety study on the immunogenicity of an ebola vaccine.
“The vaccine uses a recombinant vesicular stomatitis virus in which the protein of this virus is replaced with ebola protein,” explains Dr. David. “Our goal is to determine whether this is immunogenic and whether it is safe.”
The study, sponsored by Merck Pharmaceuticals, had met its 1125 patient enrollment goal (including 17 participants from Sanford) just a month after it opened in August 2015. Most participants are healthcare workers. They will have three follow-up visits to report side effects and be tested for antibody titers over the course of the six month study.
“There is currently no vaccine for Ebola,” says Dr. David. “This trial has very good potential, although it is too early to say how effective the vaccine will be.”
The Role of Genetics in Cancer
The role that a patient’s genes play in the development, progression or treatment response of their disease is another major focus for area researchers. The recently-concluded GEMMA (Genetic Exploration of the Molecular Basis of Malignancy in Adults) trial utilized next-generation gene sequencing technology to analyze tumor samples and provide real-time clinical information to the care teams of 120 adults with rare or recurrent cancer.
“One thing we learned from the GEMMA trial is that patients really want access to these kinds of treatments but that insurance doesn’t always want to pay for it and physicians don’t always know how to interpret it,” says Sioux Falls oncologist and clinical researcher Steven Powell, MD.
For this reason, the next generation of GEMMA - COMPASS (Community Oncology use of Molecular Profiling to Personalize the Approach to Specialized Cancer Treatment at Sanford) will offer genetic testing to any children or adults with incurable cancer, with the goal of helping to direct them toward the most effective treatment.
Immunotherapy is another area of interest for Dr. Powell, who is leading a trial of the PD-1 inhibitor pembrolizumab as a way to enhance the effects of standard chemotherapy for head and neck cancer. “This allows us to use a lower dose of chemotherapy. So we are hoping for higher cure rates with less toxicity,” says Powell.
A New Stent Graft for TAAA
Sanford physician inventor Patrick Kelly, MD, recently operated on the first patient treated as part of the clinical study of a new kind of stent graft for thoracoabdominal aortic aneurysm. Dr. Kelly says the Medtronic Valiant TAAA Stent Graft System, which he developed, allows for minimally invasive repair of a type of aneurysm for which the open-procedure mortality rate is 25 percent.
“As an artery becomes diseased, it becomes deformed. To develop a graft that is going to match everyone’s anatomy is virtually impossible,” says Dr. Kelly who designed his alternative stent when faced with a particularly challenging case of aortic disease. “Our design makes no attempt to try to match people’s anatomy so we are not limited by their anatomy.”
Kelly credits his ability to “think outside the box” and design a creative solution to his background as a structural engineer. Based on the positive responses of the first 29 patients treated with the new stent, Sanford entered into a license agreement with Medtronic to produce the final design which is now the subject of the multi-center clinical trial.
“This procedure marked an important first step in the process to obtain FDA approval,” says Dr. Kelly.
Kelly was also recently involved in a collaboration with USD to create drug coated balloons for the treatment of peripheral artery disease.
Open Clinical Trials in South Dakota
Although clinical trials are often associated with cancer treatment, hundreds of trials in a variety of disciplines are underway in the state of South Dakota alone and many more in the surrounding states. Below is a partial list of disease processes being studied by researchers in our region. A full list of open clinical trials and their eligibility criteria are available on each institution’s website.
Acute Myeloid Leukemia
Coronary Artery Disease
Type 1 Diabetes
Type 2 Diabetes
Head and Neck Cancer
Inflammatory Bowel Disease
Neurological Disorders (Huntington’s, MS, Neuropathy)
Pulmonary Arterial Hypertension
Squamous Cell Carcinoma
Various pediatric cancers
Vitamin D Deficiency