Life on Wheels (82 page)

Determine the appropriate dosage of medications

Observe side effects or the possibility of death

Discover differences between results in humans and animals

Prove the value of the therapy

Decide whether or not to make the treatment widely available and for whom

A clinical trial is a carefully designed plan, prepared by doctors and scientists. The government’s FDA might participate in the development of a clinical trial, as might a commercial entity interested in being the producer of the drug. This is a very precise project, defining specific criteria and methods.

It used to be that any researchers could go out and try the next “miracle cure” on anybody they could convince to go along. Doctors were under no restrictions in their experiments, and there were plenty of “snake oil salesmen” out pitching the latest elixir to cure any and all ailments. Even Coca-Cola was first promoted as a curative.

People sometimes died in the process of this uncontrolled approach. Today, the standards are very high. Scientists who do not work within a very prescribed process risk wasting years of work if their data are not found to have been responsibly and consistently collected. More to the point, the scientists conducting the study will be just as concerned with your well-being as with their reputations and legal liability.

Most trials employ a Data and Safety Monitoring Board, which reviews the study as it progresses. The Board members have the power to end the study at any point if they find that the therapy is not effective or is causing harm rather than helping.

The hospital, clinic, company, or university conducting the study will also have an Institutional Review Board that has seen and approved the study protocol in advance. The Board members receive regular reports and also have the power to suspend a study.

The U.S. government keeps track of clinical trials—those currently enrolling participants and those that are complete—at www.clinicaltrials.gov. The Christopher and Dana Reeve Foundation formed the North American Clinical Trials Network in 2004 to create the infrastructure for the increasing number of clinical trials that they see coming on the horizon. According to Foundation:

The International Campaign for Cures of Spinal Cord Injury Paralysis has published a comprehensive guide to clinical trials for SCI (www. campaignforcure.org).

If you decide to participate in a clinical trial, you have the right to withdraw from a study at any time, but it may be possible that abruptly ending a therapy could itself be dangerous. You must understand that, when you agree to participate, you must follow the instructions absolutely and report any effects to your supervising physician. If you are considering withdrawing, discuss this with the doctor whose job it is to be your consultant, helping you to make the final decision. He or she should then advise you of the safest way to end the therapy and/or resume your previous treatment.

Many studies involve use of a placebo, an inert substance given to some subjects rather than the actual drug being tested. This is to reveal whether some of the results observed might be because a subject believes she has been given a beneficial drug. It is now increasingly understood that attitude and belief can have a real impact on the physical—the so-called mind-body connection.

It is important to compare people who are getting the drug to people who are not receiving the drug to understand what real impact the actual drug is having. In some studies, you might consistently receive either the active drug or the placebo. In others, you might be getting one or the other at any one time.

It is not possible to request that you be given the active form of the drug. It would alter the results of the study if you knew which you were getting. Again, your reason for participating is to assist research, not just to gain early access to an unproved treatment.

You might be asked to cease taking a drug that has already been prescribed for you. Drugs interact with each other in the body, and the needs of the trial might require that you end your current treatment. You must balance the possible risks of going off of your present therapy against the potential benefits of your participation.

Clinical trials are generally either single-blind or double-blind. If you are in a single-blind study, you do not know whether you are getting the active drug or the placebo, but your doctor does. In a double-blind study, neither you nor the doctor knows. However, other investigators involved in the study have that information. If you were to have a severe reaction to treatment, it would always be possible to find out what you actually received.

Clinical trials are performed in three phases:

Phase I. This study is sometimes referred to as a “safety study.” Its main purpose is to test the safety and effective dosage of a drug. This is the time of greater risk because less is known about reaction to the drug. High doses might be given to measure side effects of the drug. Only a few persons participate at this stage of drug testing.

Phase II. More people are included in the study, now that there is some sense of what the appropriate dosage should be. Now it is possible to begin to observe the effectiveness of the treatment. Several rounds of Phase II studies might be performed before the team feels ready for Phase III.

Phase III. Many more subjects are studied, often at a number of participating institutions. This is the time to validate the discoveries of the first two phases and to prepare statistical data to use in the process of gaining FDA approval for the potential commercial introduction of the therapy.

Some trials pay their subjects for their participation, and some don’t. There might be certain procedures that you would need to pay for, and you should check with your health insurer to see if they would be covered. There could be travel and accommodation expenses during periods in which you might need to stay near a medical center for initial tests and observation if you live away from where the study is centered. Or, your usual doctor might be able to participate and conduct some of the procedures.

On the other hand, you might benefit from additional coverage due to your participation in the study. You could be seen more regularly by doctors and, in effect, be getting more aggressive care of your condition at no extra expense.

You should not pay a fee to participate in a clinical study. These are always performed with funding from public, charitable, or commercial sources. The group conducting the study should not be in a position to profit from the actual study. They might, however, profit from future licensing or sale of the drug if it attains FDA approval.