Clinical Trial to Explore Therapeutic Potential of Bone Marrow Cells for Crohn's Disease
A new study at Emory University in Atlanta, Georgia will examine whether patients’ own bone‐marrow cells can be used to effectively treat Crohn's disease.
The phase 1 clinical trial is being led by Subra Kugathasan, MD, professor of pediatric gastroenterology at Emory and is supported by the Crohn's and Colitis Foundation of America and the Atlanta Clinical and Translational Science Institute.
Crohn's is an inflammatory bowel disease characterized by severe abdominal pain, diarrhea, fever, weight loss, and inflammation within the digestive system. Existing treatments are not always effective and immunosuppressant therapies often have other side effects. Bowel resection is sometimes required to treat the disease.
Dr. Kugathasan's study involves harvesting mesenchymal stromal cells from the bone marrow of Crohn's patients ages 18 to 65 and developing “personalized” disease‐fighting cells in the lab to reinfuse into ‐patients.
“We believe these cells have high amounts of anti‐inflammatory cytokines that reduce inflammation and put the disease into remission,” Dr. Kugathasan says. “Our preclinical work has been very promising, and everyone is excited about this evolving into a new therapeutic platform. The only question is whether this therapy can repair the patient's damaged system and how long it will work,” he adds.
Blood and bone‐marrow cells have been used for more than 25 years to treat life‐threatening hematological conditions like leukemia, but this study is the first to apply the personalized process in Crohn's disease. Using patients’ own cells to defeat the disease is not expected to cause problems, says Dr. Kugathasan. Investigators hope to enroll a total of 16 patients within the next year.
Drug Therapy May Hold Promise for Treating Alcohol‐Use Disorders
Two California‐based universities are collaborating on research around the antiparasitic drug ivermectin and its potential to treat alcohol‐use disorders, which affect 18 million people and cause 100,000 deaths annually in the United States.
“We've identified a target in the central nervous system that can be worked on to develop new therapies for alcoholism, and we are repurposing a drug that has been used in humans for about 30 years,” says Daryl Davies, PhD, associate professor of clinical pharmacy at the University of Southern California (USC) School of Pharmacy.
Working with investigators at the University of California Los Angeles (UCLA), Dr. Davies' research is being funded through a $50,000 pilot grant from the Southern California Clinical and Translational Science Institute (SC–CTSI) at USC and Children's Hospital Los Angeles, as well as a $30,000 pilot award from UCLA. The funds will support a phase 1, 10‐person clinical trial of the US Food and Drug Administration‐approved drug to determine its safety and therapeutic value.
In the study, researchers will combine ivermectin with moderate doses of alcohol to see whether the drug reduces alcohol craving. Investigators hope the study also will set the stage for an additional research on the long‐term use of ivermectin.
“Right now, the drug is only approved for acute or intermittent use, but we know that alcoholism is a chronic disease and it will need to be treated long term,” Dr. Davies says. “There are additional safety and pharmacology studies we have to do.”
Investigators will study whether the craving for alcohol can be diminished by the antiparasitic drug invermectin.
Clinical Data Research Network Merges Healthcare Systems' Information
Seven New York City‐based healthcare systems plan to share data to improve clinical research and patient recruitment to clinical trials. Their effort, known as the New York City Clinical Data Research Network (NYC‐CDRN), has received a $7 million contract from the Patient‐Centered Outcomes Research Institute (PCORI) to fund their goal.
“The network is an incredibly exciting opportunity to connect 6 academic medical centers and one practice‐based research center in order to create a comprehensive, longitudinal database of minimally one million patients over 18 months,” says principal investigator Rainu Kaushal, MD, MPH, chair of healthcare policy and research for Weill Cornell Medical College in New York City.
Dr. Kaushal says she expects the new network will help facilitate patient‐centered clinical research, develop a healthcare system that more rapidly translates scientific findings into practice, and improve the management and delivery of clinical care for specific patient populations.
Rainu Kaushal, MD, MPH, says a new data‐sharing network linking seven New York City‐based healthcare systems will create a database of information for at least one million patients.
Web‐Based “Launch Pad” Offers Career Guidance, Advice for Researchers
A new Website developed by the Clinical Translational Science Institute (CTSI) at the University of California San Francisco (UCSF) features a series of videos designed to help young clinical and translational investigators.
The videos and site, found at launchpad.ucsf.edu, are the brainchild of Ruben Rathnasingham, PhD, associate director of early translational research at CTSI and head of the institute's catalyst awards program, which fosters early interaction between product development experts and UCSF researchers.
“We discovered that we were repeating a lot of the same advice,” says Dr. Rathnasingham. “I developed a proposal and received funding to put together brief videos of our principal investigators (PIs) and industry advisors to discuss challenges and opportunities in translational research.”
Dr. Rathnasingham and colleagues developed 72 videos centered around 5 main themes: identifying and addressing unmet patient needs; defining desired clinical and commercial attributes of various products; meeting challenges and finding opportunities in clinical and translational science; identifying and evaluating product development plans; and identifying, nurturing, and growing promising innovations.
The site launched in January 2014 and new videos will be added monthly. Investigators featured in the videos discuss how—as early‐career researchers—they needed help navigating regulatory pathways and attracting potential investors. They also offer tips on how best to present products.
“We got some really great videos of PIs and advisors talking about how naive they were when they started and what they learned,” says Dr. Rathnasingham. “We want investigators to see themselves in these videos and say, ‘I know I need help. Now I know what questions I need to ask,’ and then come to us for help.”
The Website, launchpad.ucsf.edu, serves as a resource for early‐career researchers looking for help in tackling challenges and finding opportunities in clinical and translational science.
IDeA Program Expands Reach of Translational Research
A nationwide program designed to broaden the geographic distribution of federal funding for competitive biomedical research is benefiting researchers in Western and Northwestern regions of the United States.
Among them, Ken Izoura, MD, assistant professor of internal medicine at the University of Nevada School of Medicine in Las Vegas, is working with colleagues to explore possible links between periodontal disease, diabetes, and osteoporosis. Suzanne Christopher, PhD, a professor in the community health department at Montana State University in Bozeman, has partnered with the Crow Nation of Native Americans in Crow Agency, Montana to develop a model to treat such chronic conditions as diabetes, arthritis, and asthma; and Kristynia Robinson, PhD, associate professor at New Mexico State University in Las Cruces, is testing the potential of mobile technology to deliver community health education to underserved rural populations along the US–Mexico border.
The researchers are part of the Mountain West Clinical and Translational Research Infrastructure Network (CTR‐IN). Based at the University of Nevada, the regional consortium encompasses 13 universities across 7 states, including Alaska, Idaho, Montana, Nevada, New Mexico, Hawaii, and Wyoming.
Each of their projects are supported through the federal Institutional Development Award (IDeA) program, an initiative funded by the National Institute of General Medical Sciences, a division of the National Institutes of Health (NIH). The IDeA program is designed to develop and expand research capabilities in states that traditionally have not received significant amounts of NIH research funding.
“The amount of clinical or translational research happening across our 13 institutions is a tiny fraction of what you'd find if you walked down a 30‐foot hallway passing 5 faculty offices at a major academic medical center,” says Robert Langer, MD, MPH, principal investigator of the Mountain West CTR‐IN. “There are a variety of reasons for this, but some are lack of role models and mentors, sparse resources, and institutional cultures that have been built on success in basic science with relatively little expertise in translational and clinical domains.”
“The CTR‐IN will provide critical expertise and resources in mentoring, education, practical research experience, biostatistics, and study design for clinical translational research that have historically been largely unavailable within our partner institutions,” he added.
Diabetes is one of several diseases being explored by a consortium of federally funded researchers in the Western and Northwestern United States.