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GENE THERAPY
Dec 19, 2006

HELLO DR. BOB,

I AM ABOUT TO PARTICIPATE IN GENE THERPY IN ABOUT A MONTH. THERE ARE ONLY(5) PEOPLE IN MY OFFICE THAT WILL GET THIS OPPORTUNITY AT THIS POINT IN TIME. MY DOCTOR WANTED ATLEAST (1) PATIENT HE KNEW WAS 100% COMPLIANT WITH THEIR MEDS. IM NOT SURE IF THIS PART OF THE TRIAL REQUIRES SOMEONE WHO IS 100% COMPLIANT WITH HIS/HER MEDS.

I'M NOT SURE WHO ELSE WILL BE IN THE STUDY, MEANING I DONT KNOW IF THE OTHERS IN THE STUDY ARE RESISTANT TO MEDS. MY DOC IS WELL KNOWN THROUGHOUT THE COUNTRY AND HAS PARTICIPATED IN OVER 100 TRIALS. HE IS TRULY GOD SENT AND SPECIALIZES IN HIV.

MY CD4 ARE 480 AND MY VL IS -50 COPIES.

I HAVE NEVER MISSED A DOSE IN THE 3 YEARS I HAVE BEEN HIV+. HIV WAS A GOING AWAY GIFT FROM MY EX GF...LOL. SHE HAD NO IDEA SHE HAD IT SO THERE ARE NO HARD FEELINGS OF COURSE. SHOULD HAVE COVERED UP, CAN'T BLAME ANYONE BUT ME. GRRRRR! ANYWAY, WE ARE STILL GOOD FRIENDS. I HAVE MADE STEADY PROGRESS FROM THE START.

THIS WILL BE PHASE II OF THE TRIAL. MY QUESTION IS IF THIS GENE THERAPY DOES NOT KEEP MY VL DOWN AND MY CD4 UP WILL I BEABLE TO GET BACK ON MY MEDS WITHOUT ANY IRREVESIBLE EFFECTS? WILL I BEABLE TO GET BACK TO AS HEALTHY AS I CURRENTLY AM AND STILL GET HEALTHIER IF I STOP MY MEDS FOR THIS TRIAL AND THEN RESTART THEM?

THANX...

Response from Dr. Frascino

Hello,

The question of whether to participate in a clinical trial is a difficult one. There are risks and unknowns by the very nature of it being a scientific investigation, a clinical "trial" of something new or different. Consequently, there are never guarantees that it will work and there are always risks involved. No one can guarantee that if your viral load shoots up and CD4s plummet, you'll be able to go back on your current medications and have the same excellent response you currently enjoy. There are indeed excellent reasons to consider participating in clinical trials, including altruism and a desire to help further HIV/AIDS research. However, before committing to a clinical trial, you should thoroughly understand both the potential risks and benefits. I'll post some information below that addresses the issue. Also, I'm not sure which gene therapy study you are considering, but I'll also repost several questions from the archives that discuss recent gene therapy trials.

Good luck.

Dr. Bob

Should You Participate in Clinical Trials? By Kevin J. Armington December 1997 Once there was only AZT. Now there are a dozen drugs that can be used to combat HIV, and new drugs are being developed, tested, and approved at an unprecedented rate. Last October the Food and Drug Administration approved Combivir®, the first two-in-one pill for antiretroviral therapy. As its name suggests, Combivir combines two widely prescribed antiretroviral agents, AZT and 3TC, in a single tablet. These two drugs are routinely prescribed together because the addition of 3TC to an AZT-containing regimen results in the reversal of high-level resistance to AZT and leads to more durable suppression of viral load. The particular advantage of Combivir, which is now on pharmacy shelves, is that it allows patients to take fewer pills -- two tablets a day, instead of the four to eight pills per day that patients assigned AZT and 3TC must take. This simplified dosing will make it easier for people with HIV who are taking these two drugs to remain compliant with their daily pill-taking schedule. Last month the F.D.A. approved a new formulation of the protease inhibitor Invirase®. This version, a soft-gel capsule, has eight or nine times the bioavailability of the old formulation. Low bioavailability has been the chief limitation of this otherwise well-tolerated protease inhibitor, and the new formulation has compared favorably with Crixivan® in head-to-head studies. With the wealth of new anti-HIV drugs now on pharmacy shelves, it is easy to forget that a relatively short time ago AZT was the only therapeutic option for people with HIV. Our current good fortune is the direct result of years of hard and often frustrating effort on the part of physicians, pharmaceutical companies, and their partners in the healthcare profession. These partners include unnamed and uncounted thousands of people with HIV who volunteered to participate in so-called clinical trials of new AIDS drugs. Some of these drugs were found to be highly effective in combating HIV infection, especially when used in combination with other agents. Others, like suramin, were found to be not only ineffective but highly, even fatally, toxic. All of these experimental drugs -- the good, the bad, and the ugly -- were tested on human subjects before they were approved by the F.D.A. Those human subjects, the often unsung heroes of the AIDS epidemic, participated in the clinical trials of these drugs knowing the risks involved. You owe it to yourself to know the risks -- and the potential benefits -- of such trials before you consent to participate in one or more of them. Despite the dramatic advances that have been made in treating HIV, the need for solid clinical research -- and for volunteers to participate in that research -- is as strong as ever. Researchers have not yet hit upon a combination of drugs that works for everyone. Indeed, the plain truth is that we have only learned how to slow the spread of HIV in the body. We still don't know how to eradicate it. And until we do, clinical trials of anti-HIV drugs are essential in the war against this cunning and persistent enemy.

What is a clinical trial? Clinical trials are not for everyone. Sometimes a particular clinical trial is not in a particular patient's best interests. Sometimes a given clinical trial is a given patient's best treatment option. How do you know the difference? To help you decide, this article will explain what clinical trials are, and it will explore some of the issues that people should consider before joining a trial. Because the language of clinical trials is highly technical -- and most of it is unfamiliar to those who are not members of the healthcare profession -- we have defined some of the terms that are commonly used to describe clinical trials (see "The Terms You Need to Know," below). These terms also appear in the Pull Out and Save section of this issue, "From AZT to ZDV, a Glossary for People with HIV." You might start by asking: What is meant by the phrase "clinical trial"? Basically, that means a drug trial that involves people. "Pre-clinical trials" are those conducted in test tubes and in animals. Test-tube studies are done to see if a potential anti-HIV drug kills the virus or, failing that, dramatically slows its growth. Animal studies provide all-important information about the toxicity and safety of an experimental compound. (Because few laboratory animals become infected when they are exposed to HIV, it has been difficult to determine how safe new anti-HIV drugs are before they are tested in human beings.) If the results from pre-clinical studies are encouraging, a drug is next tested in clinical studies. There are four phases to all clinical studies, but when we are dealing with anti-HIV drugs we are mainly concerned with the first two. Phase I clinical trials focus primarily on safety and on what is called the "pharmacokinetics" of the drug in question -- that is, on how the drug is distributed throughout the body and how the body metabolizes the drug. These studies help researchers determine where the drug becomes concentrated after it is taken and how long it lasts in the blood stream. Usually, only a few patients are recruited for Phase I trials, and such trials last a short period of time -- typically from a few weeks to a few months. Phase II trials are larger and longer. They provide additional information about a drug's safety and new information about its efficacy -- that is, how well the drug works. Some anti-HIV drugs have been approved or made available through large early-release programs after successful Phase II trials. However, the norm is for drugs to go on to still larger and longer Phase III trials before they are licensed. In the United States, a drug can only be licensed by the Food and Drug Administration, which also defines the specific medical conditions for which a drug may be prescribed. Let's focus a little more on Phase II trials. Usually, they compare two or more groups of participants. The idea is to make the groups as similar as possible, so that the only variable is the experimental drug; this way, researchers can be reasonably sure that differences between the groups are due to the drug. For instance, the organizers of a trial might set up two groups of 50 HIV-positive patients who have roughly the same CD4 counts and are in about the same state of health. Typically, one group receives the experimental drug and the other gets either a placebo (a dummy pill that looks like the trial drug but contains no active ingredients) or another standard medication (like AZT or ddI). The group that receives the placebo or the standard treatment is called the "control" group. Not all Phase II trials are controlled; some are designed so that everyone receives the experimental treatment. These trials are called "open-label" studies. Today, of course, very few trials look at only one drug; most test several combinations of several drugs in several groups -- which makes conducting the trials, and analyzing their results, exceedingly complicated. "If you are not doing well on the currently available treatments, clinical trials are a way of gaining access to new drugs that might help you. Sometimes these experimental treatments are available outside of clinical trials, and you may want to explore this option after you have discussed the matter with your care-providers." Objectivity is extremely important in clinical research. For this reason, controlled trials are often "blinded." This means that patients do not know which drug (or drug dose) they are receiving. If a study is described as "double-blind," this means that no one -- not even the researchers who are conducting the trial -- knows who is getting what drug until the end of the trial. Long clinical trials have a mechanism built into their design, however, to ensure the safety of the participants. Halfway through the study an independent group of researchers "unblinds" the results, to see if there is a real advantage to the experimental drug. If a significant difference is found, the trial may be stopped and the new drug offered to everyone.

Should you participate in a clinical trial? What do you have to gain -- or lose -- by entering a clinical trial? For some people with HIV, there are real advantages to participation. For others, the benefits are not so clear. Let's start with the potential advantages: If you are not doing well on the currently available treatments, clinical trials are a way of gaining access to new drugs that might help you. Sometimes these experimental treatments are available outside of clinical trials, and you may want to explore this option after you have discussed the matter with your care-providers. The F.D.A. has permitted some pharmaceutical companies to set up "early release" programs for promising new drugs, programs that give people with HIV direct access to experimental drugs if they meet the criteria of the program. In most cases, however, a clinical trial is the only way to get an unapproved drug. If you agree to participate in a clinical trial you often receive extra medical care, and it is usually free or offered at very low cost. Keep in mind that the care you are being given by researchers should not replace regular visits to your primary care-provider. Think of this extra care as a bonus, not a substitute. Some patients with little or no health insurance can get better medical care by participating in clinical trials. Since drugs that are already approved are sometimes studied in clinical trials, participation can be a way to gain access to a treatment that you otherwise could not afford, if your insurance does not cover prescriptions for the drugs being used in the study. Some people feel a sense of heightened power, and of closer control over their clinical condition, when they seek out the latest treatments. For these individuals, participation is a way of ensuring that they have left no stone unturned in their efforts to achieve clinical stability and maintain their health. Participating in a trial "helps the cause." It is no exaggeration to say that a large share of the credit for current treatment successes belongs to the people who participated in the clinical trials conducted over the last decade. Still, it is important that you do not lose sight of the fact that your first responsibility is to yourself and to your own health. Some primary care-providers participate in clinical trials, and they may be able to give you the study drugs in their own offices. This eliminates the need to go to someone else's office, or to a clinic or hospital, for testing and treatment. Now, let's consider the possible disadvantages of participation in a clinical trial: If it is a controlled trial, you may not get the experimental drug. Indeed, if it is a placebo-controlled trial, you may not get any drug at all. There is no guarantee that an experimental treatment will help. In fact, it might even hurt you -- despite whatever evidence has been gathered from test-tube studies that the experimental treatment is beneficial. Some trials require that you stop current treatments. If you are stable on your current regimen, changing it is probably unwise. Even if a drug has proven to be safe when taken in short-term Phase I trials, this does not guarantee that side effects won't develop with long-term use. On rare occasions these unanticipated side effects can be serious, and on very rare occasions they can even be life-threatening. Some trials require you (or your insurance carrier) to bear some of the costs of the study. Clinical trials that entail significant costs to participants should generally be avoided. Trials can be inconvenient. They usually require extra visits to the doctor's office or to a clinic or hospital. Some may even require you to be admitted to the hospital for a time. Transportation and/or child-care may be a problem for some patients. Ask if any of these costs are covered by the trial. Trials can be unpleasant. They may require that you undergo a lot of tests, and these tests may be uncomfortable or even painful. It is not uncommon for such studies to require that you have blood samples taken on a regular basis, and some trials may also require stool samples, sputum samples, bone-marrow testing, and other laboratory tests. Although the list of disadvantages is longer than the list of advantages, this does not mean that the scales usually tip against joining a trial. What this does mean is that entering a clinical trial is not a decision that should be taken lightly. Don't sign up on the spur of the moment. Talk your options over with your regular healthcare providers, and listen carefully to their advice. Discuss these options with your friends and with fellow patients -- especially those who have had experience participating in clinical trials. "Clinical trials can be inconvenient. They usually require extra visits to the doctor's office or to a clinic or hospital. Some may even require you to be admitted to the hospital for a time. Transportation and/or child-care may be a problem for some patients. Be sure to ask if any of these costs are covered by the trial." Next, you should discuss the pros and cons of participation with the researchers who are conducting the trial. Until they have allayed any anxieties you may have -- and have answered whatever questions you have about the risks and benefits of participation -- don't agree to enroll. To help you reach a decision, we have prepared a list of questions that you may want to ask the organizers of the study (see "The Questions You Need to Ask," below). Last but certainly not least, ask yourself if you are absolutely certain that you are willing and able to fulfill the requirements of participation before you sign up. At the time of your formal enrollment in the trial, you will be asked to sign an "informed consent" form. By signing it, you affirm that the organizers of the trial have explained all of the potential risks to you and outlined all of your responsibilities as a participant. Do not join a trial that does not require an informed-consent form, and keep in mind that informed consent is not a contract. You have the right to leave a trial for any reason whatsoever at any time you choose. It is appropriate to become suspicious of the objectives of a clinical trial if its organizers seem to be pulling you away from your regular care-providers or the type of medical care you have been receiving. Their "trial" may be a money-making scheme rather than serious science. Ideally, a clinical trial should be beneficial to researchers who conduct it, to patients who participate in it, and to the patients, the healthcare professionals, and the pharmaceutical companies who later benefit from the information it generates. To maximize their utility, clinical trials need to be carefully designed, so that they both accommodate the needs of participants and yield useful data. Over the last fifteen years, AIDS activists have become intimately involved in the planning and execution of clinical trials in a way that has enhanced the state of AIDS research. Today, many well-designed trials are available to patients at all stages of HIV disease. If the idea of participating in one or more of these trials appeals to you, talk the matter over with your primary care-providers and investigate all of your options. You can get information about clinical trials -- anywhere in the country -- by calling the National Clinical Trials Hotline at 1-800-TRIALS-A.

The Terms You Need to Know Arm: all of the study subjects taking a particular drug or drug dose are said to be in that "arm" (or "cohort") of the study Bioavailability: how much of the drug is absorbed into the bloodstream Blinded: participants do not know which arm of the study they are in (i.e. they do not know what drug or drug dose they are getting) Control group: the trial participants who do not receive the experimental treatment Crossover: a trial in which the experimental and control groups switch treatments Data Safety Monitoring Board (DSMB): a group of independent researchers who review the data while the trial is going on, to determine if one drug or drug dose is markedly safer or more effective than another Double-blinded: neither the trial participants nor the researchers know who is receiving which drug or drug dose Exclusion criteria: list of reasons why some prospective participants should not be included in the study (e.g. a trial may not want people with CD4 counts above 500, or pregnant women, or patients who have previously used the drug being studied) Experimental group: the trial participants who do receive the experimental treatment Half-life: the time it takes for the body to eliminate half of the drug that reaches the bloodstream Inclusion criteria: list of things (clinical state, blood values, etc.) that are required of every participant in a clinical trial at time of entry Open-label: a trial in which everyone gets the experimental treatment Pharmacokinetics: the study of how the body metabolizes a drug, which tissues the drug penetrates, its half-life, and other variables Placebo: a pill that looks like the experimental drug but does not contain any active ingredients Protocol: the overall design, or blueprint, of the study Randomized: distributed by chance

The Questions You Need to Ask Here are some of the issues to consider when you meet with the study coordinators: Will you need to alter -- or stop -- your current treatment? (Be sure to bring to your first meeting with the study organizers a list of all drugs you are taking.) What is the evidence that this treatment might work against HIV? What are the potential risks? How long will the study last? How often do you need to come in for visits, and how many blood samples and/or other specimens will be needed? Are evening/weekend appointments available? Are there any costs to you? Can you be reimbursed for travel expenses or child care? Who will take care of you if you have side effects? Will you have 24-hour access to a doctor involved in the study, in case you have an emergency that could be related to the experimental drug? Will you have access to the drug after the study is over, and will you be eligible for future studies of the drug?

Clinical Trials: Making Them Work for You By Sarah Biel-Cunningham, M.S.W. January/February 2004

In the early 1980s, when AIDS began to descend upon our community, fear was everywhere. Innocent people were dying and modern day medicine failed at saving lives. The medical community began working to improve what was available in the way of medicines to help those who were fighting this disease. It was known from the start that a cure may not be found right away, but researchers were driven by the fact that therapies could be discovered to restore health and slow the disease process. With these newly discovered experimental treatments came opportunities for individuals to partake in clinical trials to help test the validity of the newly developed drug therapies. A lifeline was born, and for the first time there was hope through access to new therapies not widely available. Lives were saved and the success of the clinical trials benefited the community through the approval of the first drug therapy in 1987, AZT. Clinical trials played an important role in the history of AIDS, but it is even more crucial to understand that clinical trials are just as important to this fight as they were 20 years ago. It is through clinical trials that individuals receive access to options, ultimately influencing the direction of drug therapies for the greater community. Ironically, many people are still unsure what clinical trials are and how one can participate in clinical trials. This is a brief overview of clinical trials and how to decide if participating in one is right for you. What Is a Clinical Trial? AIDS clinical trials are research studies in which new therapies for HIV/AIDS are tested in humans. Before new drug therapies can become available to the community to treat HIV, they must be proven both safe and effective. Clinical trials are designed to test these therapies on humans in order to provide results to the Food and Drug Administration (FDA) for approval. However, the first step is not to test in humans. New therapies are subjected to laboratory tests and tests that involve animals. If the results from these tests are good, the drug treatment is then moved into a clinical trial to be put through more testing involving human beings. Clinical trials are divided into three steps or phases. The first phase determines the treatments' safety, which is assessed by using a small group of people. Phase two tests if the treatment works. Phase three assesses the long-term results of the treatment in humans. Clinical trials are carefully governed medical processes based on rules and guidelines known as a protocol. The protocol describes what types of patients may participate in the clinical trial, schedules of tests and procedures, drugs and dosages, and the length of the study. The protocol describes exactly how the trial will be carried out. Participants in a clinical trial must agree to be treated by the terms of the protocol. This is part of the informed consent of the clinical trial, which is a tool developed to protect the participant within the clinical trial. By law, the researchers must provide you with a full written description of the clinical trial to read and sign before you agree to participate in the clinical trial. It is important to review all the information given to you and understand exactly what you are agreeing to within the clinical trial. Who Is Eligible for Participation? The protocol defines the rules for participation in a clinical trial and each trial is unique in the requirements for participation. Some trials are specifically designed with one gender in mind, others sometimes require your CD4 or viral load count to be at a certain level, and still others are geared towards people who are currently not on any drug therapies. There are many different opportunities available and if you are interested in participating in a clinical trial, talk with your physician to find out what is available for you. What Are the Benefits of Participating? Access to new treatments before they are available to the public Access to medical care which monitors your health very carefully The possibility of receiving some or all of your medications free of charge Helping the community by contributing information about new treatments Are There Risks to Being Involved in Clinical Trials? The experimental drugs you have access to may not be effective. During clinical trials, new treatments are often compared to already existing treatments or non-existent treatments, also known as a placebo. Participants do not have the option to choose which part of the clinical trials they participate in nor do they know whether they are receiving the new treatment or the placebo. Therefore, access to the new treatments is not guaranteed by the researcher. You may be required to stop taking your already existing drug therapies that may be currently effective for you. The new treatments may have unanticipated side effects. The clinical trial you are participating in may require a lot of your time and frequent visits to the study site. Should I Participate in a Clinical Trial? Clinical trials definitely have the potential to not only benefit you, but also provide information to help the greater community. However, it is important to recognize potential risks associated with clinical trials. If you are considering participation in a clinical trial, you and your physician should discuss whether this is the best decision for your health. Here is a list of questions to consider when making your decision: What is the purpose of the study? What other treatment options are available to me? Who will pay for the costs of the study? How often will I have to go to the clinic? What are the drug side effects? What was learned in previous studies of this particular treatment? Will I need to stop any drugs or other treatments I am in the process of using? Can I be reimbursed for travel? Where is the location of the clinic? Is childcare available? Will I be hospitalized for the trial? Will I be able to stay on the study treatment after the trial is over? How is my confidentiality protected? Am I committed to staying with the clinical trial until it is complete? How to Find Information on Clinical Trials? The following resources are available to you: National AIDSinfo: www.AIDSinfo.nih.gov, 1-800-448-0440 AIDS Treatment Information Service: 1-800-874-2572 Metro Atlanta AIDS Survival Project: (404) 874-7926 AIDS Research Consortium of Atlanta (ARCA): (404) 876-2317 Emory Clinical AIDS Research: (404) 616-6333 Participating in a Clinical Trial August 7, 2006 What Is an AIDS Clinical Trial? Who Can Participate in a Clinical Trial? What Are the Benefits of Participating? What Are the Risks? How Are Participants Protected? Should I Participate? To Find Out More About Clinical Trials

What Is an AIDS Clinical Trial? Before new drugs can be sold to treat HIV disease, they must be proved to be safe and effective. The Food and Drug Administration (FDA) approves new drugs and other treatments based on the results of laboratory tests, animal tests, and tests in humans (clinical trials). New treatments are tested in humans only if there were good results from laboratory tests and animal studies. In the first clinical trials, the treatment is tested for safety in a small group of people. Later trials with many more participants test how well the treatment works. AIDS InfoNet Fact Sheet 105, How HIV Drugs Get Approved, has more information on the phases of clinical trials. A clinical trial is a carefully planned medical experiment. The guidelines for a clinical trial are called a protocol. The protocol is a document that describes exactly how the trial will be carried out.

Who Can Participate in a Clinical Trial? The protocol explains the rules for participation in a clinical trial. Each trial is different. For example, some trials require certain viral loads or CD4 cell counts. You normally cannot participate in a clinical trial if you have any opportunistic infections, or are using any treatments that might make it difficult to measure how well the test treatment is working. You also cannot participate if the study treatment might harm you. For example, women sometimes cannot participate in trials during the first three months of pregnancy, because of the risk of birth defects for their newborn child. Trials are carried out at different hospitals and clinics throughout the world. Some hospitals participate in many clinical trials. Others may not offer any. Some trials will reimburse your travel costs to a study center.

What Are the Benefits of Participating? You could get a new treatment before it is available by prescription. Your health will be watched very carefully. You might get some or all of your medications paid for. You might also get some lab tests or other care for free. You will be helping others by contributing information about new treatments.

What Are the Risks? In trials, new treatments are compared to the best available medication or to a dummy medication (a "placebo"). You might not get the new treatment. Patients and health care providers in these trials are not told who is getting the new treatment. You might have to stop taking other medications during the trial. Study treatments might not work. Study treatments might have serious side effects. Participating in a study might take a lot of time. It could require special record-keeping or many trips to the study location.

How Are Participants Protected? There are strict laws on research using human participants. The main tool to protect you is called "Informed Consent." You will be given a full, written description of the clinical trial to read and sign before you agree to participate. Take your time to review the Informed Consent before you sign it. If you need an interpreter to help you understand it, ask for one. If you have questions, be sure you get the answers before you sign. There are also local and national boards that review and monitor each clinical trial before it starts and while it is in progress. Trials can be stopped early if they are harming participants. You can decide to drop out of a clinical trial at any time, for any reason.

Should I Participate? You and your health care provider should discuss the possible benefits and risks of taking part in a clinical trial. Here are some of the questions you should consider: What is the purpose of the study? How long will it last? Where is it being conducted? How will I take the medication (pills, shots, intravenous infusion, other)? What else do I have to do (records to keep, office visits, etc.)? What will I have to pay for? Can I be reimbursed for travel expenses? Is childcare available? Will I be able to stay on the study treatment after the trial is over? Who will pay for it? What was learned in previous studies of this treatment? Will I have to stop any drugs or other treatments I am now using? Will taking part in this study exclude me from other clinical trials?

To Find Out More About Clinical Trials For information about participating in clinical trials or trials availability throughout the U.S., call the AIDSinfo Service at 1-800-448-0440, or visit their Internet web site at http://aidsinfo.nih.gov. The FDA web site has information on the drug development process at www.fda.gov/cder/handbook/dev_rev.htm. ________________________________________ U.S. NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES What Is a Clinical Trial? Choosing to participate in a clinical trial is an important personal decision. The following frequently asked questions will provide you with detailed information about clinical trials. In addition, it is often helpful to talk to your health care provider, family members, or friends about deciding to join a trial. After you have identified some trial options, the next step is to contact the study research staff and ask questions about specific trials. What Is a Clinical Trial? What Is a Protocol? What Are Clinical Trial Phases? What Protections Are There for People Who Participate in Clinical Trials? What Is Informed Consent? Who Can Participate in a Clinical Trial? Who Sponsors Clinical Trials? What Happens During a Clinical Trial? What Is a Placebo? What Is a Control or Control Group? What Is a Blinded or Masked Study? What Is a Double-Blind or Double-Masked Study? What Are Side Effects and Adverse Reactions? What Are the Benefits and Risks Associated with Clinical Trials? What Should I Know Before I Join a Clinical Trial? How Should I Prepare for the Meeting with the Research Coordinator or Doctor? What Questions Should I Ask? Should I Continue Working with My Primary Health Care Provider if I Participate in a Trial? Can I Leave a Clinical Trial After it Has Begun? Will I Be Paid for Participating in a Clinical Trial?

What Is a Clinical Trial? A clinical trial is a research study to answer specific questions about vaccines or new therapies or new ways of using known treatments. Clinical trials (also called medical research and research studies) are used to determine whether new drugs or treatments are both safe and effective. Carefully conducted clinical trials are the fastest and safest way to find treatments that work. Ideas for clinical trials usually come from researchers. Once researchers test new therapies or procedures in the laboratory and get promising results, they begin planning Phase I clinical trials. New therapies are tested on people only after laboratory and animal studies show promising results.

What Is a Protocol? All clinical trials are based on a set of rules called a protocol. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

What Are Clinical Trial Phases? Clinical trials of experimental drugs proceed through four phases: In Phase I clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. In Phase II clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety. In Phase III studies, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely. Phase IV studies are done after the drug or treatment has been marketed. These studies continue testing the study drug or treatment to collect information about their effect in various populations and any side effects associated with long-term use.

What Protections Are There for People Who Participate in Clinical Trials? The government has strict guidelines and safeguards to protect people who choose to participate in clinical trials. Every clinical trial in the U.S. must be approved and monitored by an Institutional Review Board (IRB) to make sure the risks are as low as possible and are worth any potential benefits. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensures that a clinical trial is ethical and the rights of study participants are protected. All institutions that conduct or support biomedical research involving people must, by federal regulation, have an IRB that initially approves and periodically reviews the research.

What Is Informed Consent? Informed consent is the process of learning the key facts about a clinical trial before you decide whether or not to participate. These facts include: Why the research is being done. What the researchers want to accomplish. What will be done during the trial and for how long. What risks are involved in the trial. What benefits can be expected from the trial. What other treatments are available. The fact that you have the right to leave the trial at any time. If you are considering joining a clinical trial, the research staff will give you informed consent documents that include the details about the study. If English is not your native language, you can ask for the consent documents in languages other than English. Since joining a clinical trial is an important decision, you should ask the research team any questions you may have about the study and the consent forms before you make a decision. It is also a good idea to take the consent documents home and discuss them with family members or friends. Talking about your options can help you to feel comfortable with your decision. If you decide to join the clinical trial, be sure to ask for a copy of the informed consent documents so you can review them at any time. Remember informed consent is more than signing a form. It is a process that continues through the study. You should feel free to ask the research team questions before, during, and after the study. Informed consent continues as long as you are in the study.

Who Can Participate in a Clinical Trial? All clinical trials have guidelines about who can get into the program. Guidelines are based on such factors as age, type of disease, medical history, and current medical condition. Before you join a clinical trial, you must qualify for the study. Some research studies seek volunteers with illnesses or conditions to be studied in the clinical trial, while others need healthy volunteers. Healthy volunteers participate in Phase I trials, vaccine studies, and trials on research on preventive care for children or adults. The factors that allow you to participate in a clinical trial are called inclusion criteria and the factors that keep you from participating are called exclusion criteria. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

Who Sponsors Clinical Trials? Clinical trials are sponsored by government agencies such as the National Institutes of Health (NIH); pharmaceutical companies; individual physician-investigators; health care institutions such as health maintenance organizations (HMOs); and organizations that develop medical devices or equipment. Trials can take place in a variety of locations, such as hospitals, universities, doctors' offices, or community clinics.

What Happens During a Clinical Trial? The clinical trial process depends on the kind of trial you participate in. The team will include doctors and nurses as well as social workers and other health care professionals. They will check your health at the beginning of the trial, give you specific instructions for participating in the trial, monitor you carefully during the trial, and stay in touch with you after the study. Some clinical trials involve more tests and doctor visits than you would normally have for your illness or condition. For all types of trials, you will work with a research team. Your participation will be most successful if you follow the protocol carefully and stay in contact with the research staff. Some terms that will help you understand what happens in a trial are defined below.

What Is a Placebo? A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment's effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or treatment.

What Is a Control or Control Group? A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.

What Is a Blinded or Masked Study? A blinded or masked study is one in which participants do not know whether they are in the experimental or control group in a research study. Those in the experimental group get the medications or treatments being tested, while those in the control group get a standard treatment or no treatment.

What Is a Double-Blind or Double-Masked Study? A double-blind or double-masked study is one in which neither the participants nor the study staff know which participants are receiving the experimental treatment and which ones are getting either a standard treatment or a placebo. These studies are performed so neither the patients' nor the doctors' expectations about the experimental drug can influence the outcome.

What Are Side Effects and Adverse Reactions? Side effects are any undesired actions or effects of drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

What Are the Benefits and Risks Associated with Clinical Trials? There are both benefits and risks associated with clinical trials. By participating in a clinical trial, you can: Take an active role in your own health care. Gain access to new treatments that are not available to the public. Obtain expert medical care at leading health care facilities during the trial. Help others by contributing to medical research. Clinical trials have risks: There may be side effects or adverse reactions to medications or treatments. The treatment may not be effective for you. The protocol may require a lot of your time for trips to the study site, treatments, hospital stays, or complex dosage requirements.

What Should I Know Before I Join a Clinical Trial? You should know as much as possible about the research study. It is important for you to feel very comfortable asking questions and the staff should answer them in a way you can understand. A list of sample questions appears below.

How Should I Prepare for the Meeting with the Research Coordinator or Doctor? Plan ahead and write down the questions you want to ask. Ask a friend or relative to come with you for support and to hear the responses to your questions. Bring a tape recorder so you can replay the discussion after you get home.

What Questions Should I Ask? Some questions you might ask about the research include: Why is this research being done? What is the purpose of the study? Who is sponsoring the study? Who has reviewed and approved this study? Why does the research team think the treatment, drug, or medical device will work? Some questions about your participation in the study include: Where is the study site? What kinds of therapies, procedures, and/or tests will I have during the trial? Will they hurt? If so, for how long? How will the tests in the study compare to tests I would have outside the study? How long will the study last? How often will I have to go to the study site? Who will provide my medical care after the study ends? Will I be able to take my regular medications during the trial? What medications, procedures or treatments must I avoid while in the study? What are my responsibilities during the study? Will I have to be in the hospital during the study? Will the study researchers work with my doctor while I am in the study? Can anyone find out that I am participating in a study? Can I talk to other people in the study? Will I be able to find out the results of the trial? Questions about risks and benefits include: How do the possible risks and benefits of the study compare with approved treatments for me? What are the possible immediate and long-term side effects? Other questions include: What other treatment options do I have? Will I have to pay anything to participate in the study? What are the charges likely to be? Is my insurance likely to cover those expenses?

Should I Continue Working with My Primary Health Care Provider if I Participate in a Trial? Yes. Most clinical trials provide short-term treatments related to a designated illness or condition, but not extended or complete primary health care. In addition, by having your health care provider work with the research team, you can ensure that your other medications or treatments will not conflict with the clinical trial protocol.

Can I Leave a Clinical Trial After it Has Begun? Yes. You can leave a clinical trial at any time. If you plan to stop participating, let the research team know why you are leaving the study.

Will I Be Paid for Participating in a Clinical Trial? Some clinical trials will pay you for joining the trial, while others will not. In some programs, researchers will reimburse you for expenses associated with participating in the research. Such expenses may include transportation costs, child care, meals, and accommodations.

Gene Therapy Apr 18, 2006 Hi Dr, Bob What do you think about gene therapy ? The San Francisco Chronicle reported that Gene therapy has promissing result I just like to hear from an expert like you thanks Doc tony

Response from Dr. Frascino Hi, I assume you are referring to the gene therapy clinical trial currently underway in San Francisco, Los Angeles and Sydney. For our readers unfamiliar with this study, I'll briefly explain. The 74 clinical trial participants first spend 16 hours hooked up to a machine that filters out stem cells and returns the rest of the blood to the body. This is called aphoresis. Next, the harvested stem cells are infected in the laboratory with a harmless mouse virus specifically engineered to carry the ribozyme gene. These genes are genetic blueprints that tell the cell how to make the enzyme ribozyme. Ribozyme is a custom-built chemical "scissors" that cuts up one of HIV's nine genes just as the virus tries to replicate itself. Three days after the gene is transferred into the stem cells, these genetically altered stem cells are returned to the patient's bloodstream. In subsequent weeks, these gene-modified stem cells produce a variety of infection-fighting white blood cells, each containing the ribozyme that we hope will be able to ward off HIV. If all goes according to plan, when HIV tries to infect these fortified blood cells, the virus will be destroyed. Ultimately we may even be able to rebuild or reconstitute the HIV-damaged immune system. As you can see, this is very high-tech stuff. One of my good friends, Dr. Ron Mitsuyasu, director of the UCLA Center for Clinical AIDS Research and primary investigator on this clinical trial in Los Angeles, collaborated with the Australian team on the first pilot study of the new therapy, showing it was safe. None of us, including Dr. Mitsuyasu, believes this complex therapy will replace effective antiretroviral drugs. However, studies like these are opening new avenues of research that allow us to explore ways of using the immune system, gene therapy and stem-cell therapy in combination to work towards our ultimate goal a cure for HIV. Stay tuned to this site, Tony. We'll keep you updated as this and other immune-based-therapy stories evolve. Dr. Bob

gene therapy Nov 12, 2006 I just read in the newspaper that phase 1 testing of a new gene therapy has concluded with wonderful results. The study involved people who were drug resistant and were running out of options. Basically they introduced some other vaccine into the person's body and over the nine months of the trial run, immune systems were strenghtened and hiv count decreased! This study only involved 5 people but i believe they all had good results, and now they are going to start more testing with people on meds. This sounds wonderful, doesn't it? Do you think the virus will be able to mutate to counter this gene therapy? The article said the big advantage was that it only required one dose instead of daily medications. So tell me your thoughts im interested to hear what you think about this new study. Hey we finnally got the house and senate back now we can focus on 08. We need to find someone good, that americans will back, i Like obama. hooray, the democrats finnally got the majority, now we just need to take the rest of the those gay bashing republicans and some of the democrats out. Hope to hear back from you on this

Response from Dr. Frascino

Hi, I addressed the results of this very preliminary study involving gene therapy "antisense" techniques recently. See below. Regarding the elections, the voters finally realized the hypocrisy of phony swaggering, blustering and bellicosity without competency and accountability! If they had been reading this forum they would have realized all this six years ago. Better late than never. Stay well. Dr. Bob Fighting HIV with HIV? Nov 11, 2006 Hello Sexy, Is this true? http://today.reuters.com/news/articlenews.aspx?type=healthNews&storyid=2006-11-06T220335Z_01_N06300058_RTRUKOC_0_US-AIDS-VIRUS.xml&src=rss&rpc=22 For the lazy bums: WASHINGTON (Reuters) - An AIDS virus genetically engineered to fight other AIDS viruses worked better than expected, suppressing the virus and renewing the immune systems of a few patients, researchers reported on Monday. The study involved just five people, and such an approach needs years more study, they cautioned -- but the surprising results offer new hope both for the field of gene therapy and for treating the fatal and incurable AIDS virus. Thanks! Response from Dr. Frascino Hello, Yes, this very intriguing paper was published in a well respected peer-reviewed medical journal this week (Proceedings of the National Academy of Sciences). It was a "safety and feasibility" study, not an efficacy study, and consequently involved only five patients. It involved a gene therapy procedure called "antisense," which involves genetically altering HIV to make it unharmful ("nonpathogenic"). CD4 cells were removed from the five test subjects. Those cells were then infected with the genetically altered virus and reinfused back into the patients. Four of the five test subjects showed some degree of immune restoration. It is important to note this is a very preliminary report and even if it does work, it will take a number of years to develop and will require monitoring the patients for short- and long-term complications. Is antisense gene therapy anywhere near ready for prime time? Nope! Is it cause for optimism? Absofrickinlutely! Stay tuned to The Body. We'll keep you updated as this potential new therapy evolves. Dr. Bob



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