For The Sake Of Science

BY CYNTHIA MCFARLAND

are used, as well. The cardiovascular system in pigs, for example, is similar to that in humans.

But testing with animals has its limits, so before a new drug or medical device is available to the public, it must go through even more intensive testing. This is when clinical trials—and human volunteers—play a vital role.

Scottish surgeon James Lind is generally credited with conducting the first controlled trial. This happened all the way back in 1747—while at sea, no less. Lind, who was a surgeon in the British Royal Navy, undertook his trial of 12 sailors su ering from scurvy on the vessel Salisbury. Lind divided the men into six groups of two, giving each group di erent daily treatments but keeping other factors (diet and environment) as similar as possible.

Among the treatments were vinegar, cider, citrus fruit, sea water, dilute sulfuric acid and a paste made from garlic, mustard seed, dried radish root and gum myrrh. (In case you’re wondering, the sailors who received the oranges and lemons fared the best, proving that citrus fruit can be successful in treating scurvy. By the end of the 18th century, the Royal Navy routinely provided its sailors with lemon juice.)

“Clinical trials are absolutely essential to the introduction of newer and better therapies for cancer patients,” says Hatem Soliman, M.D., who works in the Breast Oncology Clinic at Mo tt Cancer Center in Tampa and is the Clinical Trials O ce Medical Director.

“Approved drugs like Herceptin have revolutionized the treatment of women with HER2+ breast cancer and would never have made it to the clinic without the evidence provided by brave patients participating in well-designed clinical trials,” notes Soliman. (Herceptin gained United States Food and Drug Administration (FDA) approval in September 1998.)

TYPES & PHASES

Clinical trials can vary significantly depending on the type of trial, which include:

› Treatment: Testing new drugs, treatments or methods

› Screening: Testing new ways to detect a disease or condition

› Diagnostic: Comparing or studying testing/ procedures to diagnose a certain disease or condition

› Prevention: Looking at ways to prevent disease

› Behavioral: Comparing/evaluating ways of promoting behavioral changes to improve health

› Quality of life/supportive care: Evaluating ways of improving quality of life and comfort in people with a certain illness/condition

A trial may be sponsored by a company, government agency or other organization that funds and manages the study but doesn’t conduct the actual research.

Researchers use di erent phases of clinical trials for di erent purposes. There are typically four phases of trials, as defined by the FDA. Each phase is designed to help researchers gain more information about the drug or treatment being studied, including risks and e ectiveness.

The first phase involves the smallest number of participants, with increasing numbers used in subsequent phases. If you are considering participating in a clinical trial, you’ll want to ask which phase you will be in.

› Phase I: A small number (generally 20 to 80) of people are used to study the safety and dosing of a drug.

› Phase II: The same drug or treatment is given to a larger number (100 to 300) of people with a focus on e cacy (how active the drug or treatment is).

› Phase III: An even larger number (1,000 to 3,000) of people receive the same drug or treatment to further study e ectiveness and to determine whether the new treatment is better than the standard of care.

› Phase IV: The drug or treatment has already been approved by the FDA and made available to the public, but this continued research is used to track safety and gain more information about benefits and use.

Some studies are randomized, meaning participants are randomly assigned to either the treatment group or the control group. Neither you (nor your doctor) can choose which group you’ll be in.

In a blind study, the participants don’t know which treatment they’re receiving. In the case of a double-blind study, neither the participants nor their doctors know. Blinding can be helpful in that it lowers the chance of bias during evaluation of participant outcomes, which helps make study results more reliable.

In the United States, a clinical trial “must have an institutional review board (IRB) if it is studying a drug, biological product or medical device that the Food and Drug Administration (FDA) regulates or it is funded or carried out by the federal government.”

An IRB is comprised of an independent group of doctors, statisticians and community members who review, monitor and approve the study. It’s the task of the IRB to make sure the study is ethical, that volunteers’ rights and welfare are protected and that risks are reasonable as compared to possible benefits.

Risk Factors

All you have to do is read the fine print on a drug label to realize some amount of risk is inherent with any medication—even for the drugs that have already been FDAapproved for use.

Is there risk involved in a clinical trial?

The short answer is “yes.” The mere fact that this is a trial means there are more unknowns than with an established medication or treatment. If researchers were absolutely sure of what to expect, there would be no need for a study.

Understandably, the earliest phases of a trial may carry greater risk, simply because the drug or treatment is so new.

That said, your physician should be able to help you decide whether the possible benefits outweigh potential risks. For example, some cancer patients are willing to participate in a clinical trial because of the chance of help or improvement.

Questions to consider before deciding to participate in a clinical trial include:

› What is the required time commitment?

› Is travel involved?

› Will I be reimbursed for time and travel?

› Will I have to pay any costs?

› What are possible short-term benefits and risks?

› What are possible long-term benefits and risks?

› What are the side e ects?

› Can I expect any discomfort as part of the process?

› Am I entitled to treatment if I am harmed as a result of the trial?

› Are other treatment/medication options available?

› If so, how do the potential risks and benefits of those options compare with those of the clinical trial?

Anyone who enrolls in a clinical trial is required to sign an informed consent form, which includes details about the study, including risks, as well as contact information. Once chosen, you are not required to remain in the study. You are a volunteer and as such are allowed to withdraw at any time.

WHY PARTICIPATE?

The groundbreaking advances made possible by clinical research depend on “regular” people—just like you and me—to volunteer for clinical trials.

There are a variety of reasons someone is willing to participate. If you have an illness or condition (this makes you a “patient volunteer”), being part of a clinical trial can o er an opportunity to receive the latest treatment, medication or procedure. If you’re in good health, volunteering for a trial can be your way of making a contribution to science in hopes of helping others in the future, or maybe you’re doing it because someone you love is su ering from a specific disease or condition.

Patient volunteers may or may not actually benefit personally from the trial. In the case of a drug study, some may receive a placebo or a test dose of the drug that is not at a level which can actually treat the illness or condition. (In a cancer trial, placebos are not usually used unless given along with an active drug.)

Healthy volunteers are vital in that they help develop a baseline for comparison with patient volunteers. Both healthy and patient volunteers receive the same drug, test or procedure, allowing researchers to learn while comparing the two groups.

Patient volunteers should ask if they’re able to continue getting the drug or treatment once the study ends if it’s been helpful.

And just because you volunteer for a study doesn’t mean you will be accepted to participate. There are guidelines for every clinical trial that take into consideration a wide range of inclusion and exclusion criteria. Age, gender, health, past treatment history and existing medical conditions are all factors that may allow or prevent participation.

If rejected for a trial, you shouldn’t take it personally. Researchers must use inclusion and exclusion criteria to identify appropriate volunteers who can safely participate in the study.

What To Expect

If you meet the criteria to be accepted into a trial and have signed the informed consent form, you may be asked to visit the clinic or hospital where the study will take place. During this time, a sta member will discuss study details and answer any questions you may have.

The amount of time involved and what is required of you can vary widely and will depend on the particular study. You may have frequent physical exams and blood draws for testing, which require regular appointments that typically fall into a narrow window of time, generally three to seven days. These appointments may be days, weeks or months apart. There may be follow-up appointments or contact with the research team after the study ends.

In this day of identity theft, it’s understandable that someone volunteering for a clinical trial wants to know their information will remain confidential. To protect your personal and medical information, your name is not included on test result forms and information that is entered into computer databases for the study. You are identified by a number or code, so researchers analyzing the results don’t see your name and your personal information will never appear in any published study results.

How To Find A Trial

Cancer, Alzheimer’s disease, neurological disorders, infectious diseases and allergies are among the illnesses and conditions frequently studied in clinical trials. If you are already being treated for one of these illnesses or conditions, your doctor may suggest participating in a specific clinical trial.

“Speak with your doctor if you’re wondering whether or not a clinical trial is right for you,” says Soliman.

You can also find out about clinical trials on your own and contact them directly to see if you meet the criteria for inclusion. Then, you can discuss the details with your health care provider before deciding to join a study.

ClinicalTrials.gov is an excellent resource, as it features a database of federally and privately supported clinical trials in the United States and worldwide. When you visit the website, you can narrow your options by entering a type of condition or illness and location. You can then view the various trials that pertain to your search. The site provides information about the trial’s purpose, who may participate and phone numbers to call for further information.

For cancer trials, visit Mo tt Cancer Center website and click on the “research” tab.

The National Institutes of Health (NIH) conducts clinical research trials for many diseases and conditions at its clinical center in Bethesda, Maryland.

You can also join a national registry of research volunteers. The NIH has funded a free registry, Research Match, to help connect researchers and volunteers looking to participate in clinical research studies. Who knows? You could be part of the process that saves a life—your own, a loved one or someone you may never meet.