Clostridium difficile, commonly shortened to C.diff is a bacteria present in the colons of 2-5% of the adult population. If able to become established (see risks below) C.Diff may disrupt the normal balance of gut flora and opportunistically dominate. Pathogenic strains of C. diff produce multiple toxin. Two of these toxins aptly named, Clostridium difficile A and Clostridium toxin B, produce diarrhea and inflammation in infected individuals.
Clostridium difficile infections (CDI) are among the most common health care associated infections in the United States. According to the Centers for Disease Control and Prevention (CDC) the risk for CDI increases in patients with:
- antibiotic exposure (people on antibiotics are 7-10 times more likely to experience CDI while on the drugs and during the month after)
- proton pump inhibitors
- gastrointestinal surgery/manipulation
- long lengthy stay in health care settings
- a serious underlying disease
- immunocompromising conditions
- advanced age
C. diff infections are extremely contagious, and are frequently passed to others through direct contact with fecal particulates directly by the patient or by care takers and medical personnel. The reason transmission rates are high is because C. diff is a spore forming bacteria.C. diff spores are hearty and can survive in environmentally stressful environments for extended periods of time that the active bacteria could not; for this reason C. diff may survive on surfaces even after thoroughly washing. To mitigate transmission, individuals with CDI shouldn’t share a bathroom (if possible) with others and thorough cleaning of rooms patient has resided with special spore-killing disinfectants must be performed regularly. Hand sanitizers do not kill C. diff, washing with warm soap and water is better, but still may remain insufficient alone. Loved ones, care takers and medical personnel should avoid touching surfaces whenever possible and wear gloves and gowns when contact with contaminated surfaces is possible.
How can I make sure a clinical trial is safe?
When deciding whether or not to participate in a clinical trial, safety is a key consideration.
CenterWatch estimates that the chances of due to an investigational drug during a clinical trial are about 1 in 10,000. Those odds need to be considered in light of the possible benefits of participation, such as helping future generations who have the same condition.
While the risk of dying as a result of a trial is low, the odds of experiencing a serious adverse event are significantly higher: about 1 in 30. Most serious adverse events happen in trials for people with cancer, heart disease and immunology/infectious diseases like AIDS. For these volunteers, trial participation often represents their best hope of survival.
Researchers work hard to keep participants safe. Before human trials can begin, investigational treatments are tested for safety in the lab or in animals and every clinical trial must follow a study plan or “protocol” to help ensure safety.
Regulators overseeing trials likewise work to keep participants safe. Every trial is overseen by a local independent review board (IRBs) that is in charge of making sure the trial is fair and ethical. Some trials are also overseen by a Data Safety Monitoring Board, which makes sure the research is valuable and patients aren’t exposed to unnecessary risk. Federal regulators, such as the Office of Human Research Protections and the Food and Drug Administration which has the final
say in whether or not a treatment is approved, also play a role. These regulators can shut down a trial if they consider it too risky.
While regulators safeguard volunteers, clinical research participants should take steps to protect themselves as well. As a research participant you should:
Educate yourself. Learn everything you can about the study treatment. Ask questions of study staff and outside healthcare professionals and do your own independent research about the study drug.
Check an organization’s certification. A number of organizations, such as the Association for the Accreditation of Human Research Protection Programs, the Association of Clinical Research Professionals, the Drug Information Association and the Academy of Pharmaceutical Physicians and Investigators, offer certification programs that
provide a level of quality assurance for would-be participants.
Speak up. If you ever sense that researchers are evading your questions, feel that you are not getting complete answers or do not feel comfortable in a trial, you need to raise your concerns. Talk to study staff, your doctor or the IRB monitoring your trial.
Put your own interests first. Remember, you are a volunteer and are free to drop out of a trial at any time for any reason.
Ultimately, the decision to participate in a trial is subjective: what may seem too risky to one person may be very promising to another. Researchers and watchdogs work hard to keep volunteers safe, but every investigation involves risks. As a participant you owe it to yourself to learn as much as you can about a trial and understand your rights as a volunteer.
History of Diabetes and Clinical Trials
by Evan D. Rosen, M.D., Ph.D, Medical Director, Diabetes, Veritas Medicine
The history of diabetes is rich with examples of the importance of clinical trials. Indeed, one of the most dramatic moments in medical history involved a research study testing a newly discovered hormone called insulin. Frederick Banting and Charles Best injected pancreatic extracts into a 14-year-old boy named Leonard Thompson, who lay dying of diabetes in a Toronto hospital in 1922. Amazingly, the boy’s high blood glucose levels improved, and he lived for an additional thirteen years. Further refinements in insulin and other drugs have led to even longer survival; all of these innovations had to be tested in humans before scientists and physicians could be sure that they worked.
Today, a patient diagnosed with type 1 diabetes can choose from a variety of insulin formulations, and patients with type 2 diabetes can choose from a rapidly expanding armamentarium of oral drugs. New developments are on the horizon, ranging from inhaled insulin to islet transplantation, and from gene therapy to stem cells. These will need to be formally tested in rigorous clinical trials before they can become part of the daily lives of people with diabetes.
If you have diabetes, the benefits of joining a clinical trial are several-fold. A trial might be the only way to obtain a new cutting-edge drug or procedure, and patients on trials often receive free expert advice about taking care of their diabetes. Finally, there is the satisfaction of knowing that because of your efforts, we will better understand diabetes, and you will have contributed in a real way to making diabetes a thing of the past.
John Crowley remembers hearing the late actor Christopher Reeve once say, “at the end of the day, biotechnology is really just a great big word for hope.”
John, who spoke at the CISCRP annual meeting in June, understands that better than most. In 1998 John’s toddler, Megan, and infant son, Patrick, were diagnosed with Pompe Disease, a nearly always fatal neuromuscular disorder. At the time, children with Megan and Patrick’s atypical strain of Pompe weren’t expected to live past five years old.
“You think, ‘this is not supposed to happen to us,’” says John, whose story is chronicled in the new movie Extraordinary Measures. “You go through the shock and denial and grief.”
In 2000 John teamed up with Dr. William Canfield, an Oklahoma-based biochemist who was developing an enzyme therapy for Pompe Disease but lacked funding for clinical trials. John, a Harvard trained MBA, left his job as an executive with Bristol-Myers Squibb Co. to become CEO of Dr. Canfield’s fledgling company.
He took out a second mortgage on his house to help finance the company, raised tens of millions of dollars from venture capitalists and ultimately sold the company to a larger firm, Genzyme, to help secure its future.
John’s story as a father turned advocate turned biotech CEO may be unique, but sadly his experience as a father desperate to obtain life-saving therapy for his sick children is not.
“The trial is the realization of hope,” he says. But that hope comes at a price. “The time it takes for individuals to become qualified for studies and for studies to advance through the system is brutally difficult” for patients and their families, says John, whose children were rejected from the first two phases of the enzyme therapy trial. And, of course, there are the risks.
Patrick barely survived having an infusion port inserted into his chest so he could receive the therapy during the third phase of the trial.
Hollywood loves a happy ending and the fact that Megan, now13, and Patrick, 12, are alive today is testament to the power of clinical research and their father’s unwavering dedication. The children continue to receive the enzyme therapy every other week, but their health is fragile: both depend on wheelchairs and ventilators and need full-time nurses.
For John, that means the search for a better treatment continues. Research is hope, he says, and hope does not stand still.
Article provided by CISCRP (Center for Information and Study on Clinical Research Participation). This article was featured in Volume 3, Issue 3 of the CISCRP Newlestter.