Strength in Numbers

Jeanne Moses didn’t have a history of cancer in her family. She didn’t have symptoms—just backache and a bit of weight loss. Nothing unusual for a 45-year-old mother working two jobs. So she was stunned when her doctor delivered the news: Jeanne Moses—technical writer, theatrical costumer, daughter of the director, emeritus, of the Vanderbilt-Ingram Cancer Center—had Stage IV colon cancer.

Moses immediately underwent surgery at Vanderbilt to remove a portion of her colon and ovaries—a procedure that can halt colon cancer if performed in time. But her cancer had metastasized to her lymph nodes, and three large liver tumors could not be removed without chemotherapy to reduce their size. The prognosis was not good.

“I was not at my best,” Moses recalls dryly. “I was in shock, losing blood, and on heavy pain medication. But I knew if I had to have this disease, Vanderbilt was the place to have it.”

That’s because Vanderbilt offered a ray of hope, she says, in the form of oncologist Dr. Jordan Berlin and the Phase II clinical trial he was conducting to test an investigational drug that showed promise in halting tumor growth.

“I didn’t know a thing about cancer trials, which is odd when you consider my father’s work,” says Moses. “But I thought this was my best chance. Just the hope was enough for me to sign up, even if I got the placebo. But I would have done it anyway. That’s what you do: give back.”

Every year Vanderbilt investigators conduct more than 620 clinical research studies and drug trials with the goal of finding new treatments and cures for the diseases that plague us. Their work is that ray of hope—hope in science, discovery, and the belief that every person can help mankind.

And Vanderbilt investigators do mean every person.

“When we evaluate vaccines, it’s in healthy people between the ages of 18 and 49 first,” says Dr. Kathryn Edwards, director of the Vanderbilt Vaccine Research Program, who has conducted successful vaccine trials for 43 years. “They have no underlying illness and can tolerate it the best.”

Edwards also heads the Vanderbilt Vaccine Treatment and Evaluation Unit, a $24 million program funded by the National Institutes of Health (NIH) to test vaccines rapidly for a variety of developing infectious diseases. The Centers for Disease Control and Prevention have tapped Edwards and her team to test the H1N1 (swine) flu vaccine.

If swine flu becomes a bigger problem, “it will hit in all people, not just healthy adults,” Edwards explains. “We need to study it in children, then healthy people over 65, then the infirm over 65. We need Hispanics and African Americans, to make sure there aren’t differences in the way people respond. We need it all.”

Edwards, who has conducted major vaccine trials for smallpox, avian flu, malaria, cytomegalovirus and pertussis, actually welcomes the challenge of preventing swine flu.

“Swine flu is a good issue to engage people in research,” she says. “Pandemics or diseases that appear suddenly and infect a large number of people point out that you can do social distancing and hand washing, but ultimately you need a prevention strategy. You need a vaccine.”

For that you need a clinical trial—a regulated, systematic way to answer a question related to health care. Also called medical research or research studies, clinical trials are used to determine the safety and effectiveness of new drugs or treatments.

Edwards points out that volunteering for a research study offers benefits beyond the rewards of altruism. Trial participants receive a vaccine for a disease before it is made available to the public. Volunteers may be paid for their time and, depending on the study, may also receive free medical tests or treatment.

Risks also may be involved with clinical research. These risks must be spelled out in simple language on the research consent form—a federal requirement that applies to all studies involving humans. Trained nurses and physicians go over the risks with each potential volunteer and answer all questions before the person signs the form and enrolls in the study.

“People need to fully embrace and understand all the potential benefits and risks,” Edwards says. “If people can’t understand what’s involved, we don’t enroll them.”

Research volunteer Jennifer Gilbert, BS’06, admits she was scared at the thought of volunteering for a Phase I clinical trial to evaluate the safety and effectiveness of an HIV vaccine in healthy, non–HIV-positive adults. A recent Vanderbilt survey showed that safety concerns are the most common reason people hesitate to join a clinical trial.

“I worried. I didn’t want to get AIDS,” says the 25-year-old Peabody College doctoral candidate. “But the HIV vaccine is different. No HIV enters participants’ bodies. There is zero percent chance of contracting HIV from participating in the study.”

Ultimately, Gilbert’s concern for others overcame her safety worries. “The church I attend does work in Africa, and I became aware of how devastating HIV is,” she explains. “As a grad student I won’t get to do work in those areas, but I thought if I could help in any small way, I would.”

Gilbert met with the HIV vaccine research coordinator, who explained benefits and risks and answered all her questions. She enrolled in the study, which required her to receive three vaccine injections and monthly follow-ups during the course of a year. She was paid $390 and experienced no side effects.

“Two or three nurses would work on me at one time to get me in and out as soon as possible,” Gilbert says. “They were amazing to work with. They cared and treated me with respect. They are the kind of people you want to help.”

This personalized service can be crucial in cancer trials, says Dr. Jeffrey Sosman, director of the melanoma program and co-leader of the signal transduction program at the Vanderbilt-Ingram Cancer Center.

Cancer patients, by their very definition, are seriously ill. They need not only the best treatment, but also the best possible guidance regarding whether or not they should participate in a clinical trial.

“For the patient, a clinical trial offers a number of advantages,” says Sosman. “But they should not be in a trial where there’s better available therapy that the patient’s not getting. They should always get the option of standard of care, or it must be included in the trial.”

That said, Sosman believes the only way the best cancer treatments can ever be found is for more patients to participate in clinical trials.

“I tell my patients that my care comes independent of a clinical trial. But this is the way I can offer the best treatment,” he explains. “By going into a trial, you’ll have a team of people taking care of you. You have backup checks and balances. If I forget to do a test, they remind me. If I don’t have time to check the result, they check it. The patient gets a much closer, more thorough evaluation.”

Sosman says his melanoma and kidney cancer patients always ask him the same question when he suggests a clinical trial: “What would you do if your wife had cancer?”

“I give them the same answer,” he says. “If my wife had cancer, I would find the place that was most comfortable, that had the best care. I wouldn’t go blindly into a clinical trial, but I would take input from the physician I felt most comfortable with.”

Although Sosman and Edwards perform different research on different people, their work faces the same challenge: Not enough people are willing to volunteer for research studies.

According to CenterWatch, only about 2 percent of the U.S. population gets involved with clinical trials each year. Among people who suffer from severe, chronic illnesses, only 6 percent participate. Ninety-four percent of the public recognizes the importance of participating in clinical research, and more than 70 percent say they would consider volunteering but don’t know how to find information about research studies.

These percentages keep Edwards awake at night. “That’s one of the things I worry about—where I’ll get enough people to fulfill our mission,” she says. “We feel frustrated that we go to the same well for volunteers. We recruit heavily at Vanderbilt, but we need engagement in the broader community.”

Vanderbilt is taking steps to solve this problem. In late summer the medical center will launch a Web site for investigators that will help them harness professional marketing and communications techniques to recruit research participants. And a major marketing campaign is in the works, with the goal of raising awareness and educating the public about the importance of volunteering for medical research.

Vanderbilt also is using resources from the largest federal grant in its history [see related story at the top of this page] to make it easier for people to find and enroll in a research study. A portion of the NIH Clinical and Translational Science Award (CTSA) funding supports the Vanderbilt Clinical Trials Web site and its recruitment registry—a searchable database where volunteers may sign up to be contacted for research studies.

“Right now anyone can access Vanderbilt’s recruitment registry,” says Dr. Gordon Bernard, assistant vice chancellor for research and principal investigator for the CTSA grant. “They don’t have to be in Nashville or Middle Tennessee. When they register they can specify how far they are willing to travel to take part in a study.”

Bernard is cautiously hopeful that more federal funding will be available in the near future so Vanderbilt can invest more in community engagement and educating the public about why and how to volunteer for research.

“We’ve really been hurting with the flat NIH funding,” he explains. “For the past three or four years, research funding has actually fallen 3 to 4 percent.”

Now, President Obama’s American Recovery and Reinvestment Act (ARRA) legislation is providing an unprecedented $8.2 billion to advance scientific research.

“That sure got people excited,” Bernard says, noting that the NIH has received approximately 16,000 applications for the 600 grants that will be funded. He points to the length of time for which grants will be funded as another downside to ARRA stimulus money.

“Research programs will only receive two years of funding rather than the standard five years,” he says. “You can do some things in two years, but it’s not the answer to everything. What happens when you hire people but can only keep them two years? But I think it will help loosen the belt at the NIH.”

Whether or not Vanderbilt receives a windfall of ARRA money, its research studies will move forward and expand as the institution’s research enterprise grows.

“We have a long and successful track record of clinical trials at Vanderbilt,” Edwards says. “We have an incredible safety record and lots of experience. We enter into very close relationships with our volunteers. In the past we’ve risen to the challenge of smallpox, pertussis and avian studies. We’re poised to do it again.”

But, Edwards cautions, Vanderbilt and other research institutions cannot meet that challenge without research volunteers.

“Each volunteer is important,” Edwards says. “The impact you can have on the world is enormous. We all have a responsibility to others. If we can help in terms of answering research questions, that is our responsibility.”