By Rebekah Arsenault

Joseph Caffey* has probably never heard of J.A. Lind, who served in the British Navy in the mid 1700s. Aboard the H.M.S. Salisbury on the English Channel, Lind carried out what is commonly accepted as the first clinical trial.

Because of his work, individuals such as Joseph, who spent years on dialysis prior to receiving a kidney transplant, have increasingly higher chances of prolonging their organ health and beating their disease.

From the effective treatment of scurvy at sea almost 260 years ago, clinical trials, also known as drug studies, have come a long way. This article will help you navigate the details of drug studies and determine if participation in a trial is right for you.

There are numerous choices available to kidney patients. Some choose to remain on dialysis rather than seek transplantation. Some wish to take part in clinical trials. Joseph participated in one study while on dialysis and began another immediately after his transplant surgery. The "right thing" will vary from person to person, and situation to situation.

Who’s In Charge?

Clinical trials are an area of medicine where there are clear delineations regarding who is "in charge." In 1962, the U.S. Congress made amendments to the Food, Drug and Cosmetic Act, paving the way for the regulation of organizations conducting clinical trials. Institutional Review Boards – made up of physicians, scientists, clergy members and others – determine ethical value of trials and protect the rights of participants.

Within the trials is a hierarchy of care, as well. Physicians known as "principle investigators" oversee the study, sign off patient orders and are responsible for the general maintenance of protocol. Additional physicians serve as "co-investigators" and provide routine care to participants. Nurses – often Certified Clinical Research Associates – enroll patients in the study, coordinate with the Institutional Review Board, prepare study budgets and provide patient care from the first day of participation to the study’s conclusion.

Benefits vs. Risks

"I do this job because of the relationships I get to build with patients," says Debbie Fernandez, an RN with LifeLink HealthCare Institute’s Clinical Research Department. "I follow patients for one to five years. I find it rewarding to provide ‘state-of-the-art’ medications to patients, giving them improved care and the best medicine available to them." Joseph says the benefits of participation include free medications, the chance at improved health and the opportunity to do something which could benefit future kidney patients. "The longevity of kidney function is the mostobvious benefit," reiterates Fernandez. "Butthere’s also the increased involvement patientshave in their own care." Having likely heardthe question, "Who’s in charge?" many times,Fernandez sees clinical trials as a way forpatients to maintain control of their health. "Patients are more aware of their medications,and how those medications are affecting them … they’re more connected to the management oftheir disease because of their participation in thestudies."

When asked about the risks, Joseph says he knew he could face side effects and Fernandez feels medicinal effects are the greatest risk posed to participants. Her role includes monitoring patient response. "It’s challenging, because you adjust the medications to coordinate with their levels for the best possible patient outcome." Fernandez works with both FDA-approved and non-approved medications, in three of four possible phases of trials.

After a medication has been evaluated according to these three standards, it can be marketed. At this point, a fourth phase may be introduced, wherein more information regarding effects in various populations is gathered. Individuals who would not qualify for other studies, because of age, health indications or other reasons, often take part in this phase. Side effects from long-term use are also determined during this phase.

The "Sugar Pill"

Placebos generate interest because of their psychological impact. It’s been said a "sugar pill" works because the person swallowing it expects it to work. First used in the mid 1800s, placebos were given to participants in the form of cocoa pills or breadcrumb concoctions. Scientists did this to reassure these individuals and give the illusion everyone was receiving the same care.

Placebos are not used in immunosuppression studies, as the delaying of treatment can be harmful to patients. Their use is not required by the FDA. Often times the "placebo" in blinded studies is a conventional, readily-available drug which has already received FDA approval. Joseph says participants might never know if the medication they take is "doing what it’s meant to do ... you never know, in the studies, if you’re reaping all the benefits." He’s enjoyed great physical health since his transplant three years ago, however, and says before that time, he was one of the few dialysis patients who worked full-time throughout treatment. He would like to credit the clinical trial medications for allowing him to live such a full life, but acknowledges the mystery.

And while Joseph’s mystery has resulted in only positive things since his transplant three years ago, it’s important you make your own decisions. Is a clinical trial, pre or post, right for for you? Participation may provide you with life-enhancing opportunities and the chance to feel more in control of your body.

You’re encouraged to discuss these possibilities with your entire healthcare team. Additional information, including the regulation of clinical trials and specific study details, can be found at www.fda.gov.

Clinical Trial Phases

This article originally appeared in the July 2007 issue of aakpRENALIFE.