Dr. RICHARD CHILDS (National Institutes of Health) (Examining Patient Tamir Orbach): Sores in the mouth or no sores in the mouth?
TAMIR ORBACH (Patient): Minor sores in the mouth, but nothing major.
BETTY ROLLIN: A couple of years ago when he was 30, Tamir Orbach learned that he had PNH, a potentially fatal bone marrow disease.
Mr. ORBACH: Before the trial, I was having a blood transfusion every three weeks. I had severe abdominal pain, probably two weeks out of a month. I was prone to infections. I was in the hospital probably one week every month because of my low immunity. And life was generally very miserable and I was out of work.ROLLIN: Tamir was a candidate for a bone marrow transplant, which for PNH patients can be deadly. He chose instead to enter a clinical trial conducted at the National Institutes of Health, where he would receive blood stem cell transplants taken, in his case, from his brother's bone marrow.
Dr. CHILDS: So our goal is to improve outcome with blood stem cell transplantation, to make it more effective and to improve its safety.
ROLLIN: Tamir followed the usual procedure and signed a consent form, which outlines the purpose of the trial and its risks, which are always there and sometimes unknown.
Mr. ORBACH: Dr. Childs and some of his team spent a long time explaining to me exactly what was going on, gave me every opportunity to ask questions.
ROLLIN (To Dr. Childs): Do you always feel confident that the patient understands?
Dr. CHILDS: I look at them for signs that they're understanding. I look, you know -- they should be nodding their head, how they should be asking me questions. If they are sitting there and they're not asking me questions, then I get concerned.Mr. ORBACH: I had plenty of time, and I could have backed out right until the last moment, the day before the transplant. The day of the transplant I could have said, "Look, I'm not interested."
ROLLIN: So far this trial has gone remarkably well for Tamir.
Mr. ORBACH: As of today I am cured of PNH, and I'm doing very well. I'm back at work, I'm back at [the] gym, my life is pretty much back to normal. The other thing is that this also provides hope for future for other patients who have similar conditions where they might benefit from this type of trial.
ROLLIN: Clinical trials are overseen by institutional review boards, IRBs, to ascertain that trials are scientifically worthy and that ethical guidelines are met -- which means that potential benefits for participants should outweigh risks and that participants know what the risks are. The IRBs also monitor the trial during its run and may even halt the trial if serious problems are reported. But the system to protect participants doesn't always work, especially when children are involved.A trial conducted in the 1990s at the New York State Psychiatric Institute recruited the younger brothers of young men in prison. Charisse Johnson's younger son, Isaac, then six years old, was given fenfluramine, which was supposed to measure Isaac's susceptibility to the kind of aggressive behavior shown by his brother.
Mrs. Johnson signed a consent form, which included a description of possible side effects, and was given $100. The ill effects of the drug, she claims, began shortly after the trial and continue to this day.
CHARISSE JOHNSON: He, like, hyperventilating, couldn't catch his breath, like anxiety attacks and things like that -- and started getting, like, these big, extreme headaches.ROLLIN: There is no proof that the drug has caused Isaac's illness, but Mrs. Johnson is suing the doctors. She said she didn't read the consent form carefully.
VERA SHARAV (President and Founder, Alliance for Human Research Protection): There is an element of coercion in all of this. Let's think about the position of such parents, most of whom probably were not educated, didn't know where to turn. These were from the parole officer; they were being recruited, their little ones were being recruited because the older child is a delinquent. That is a heck of a position to be in. That is not voluntary, informed consent.
Dr. MARY FAITH MARSHALL (Bioethicist, University of Minnesota): Not all the research that happens in this country is funded by the federal government; not all of it comes under federal oversight mechanisms, and we don't really have a good database. We don't know exactly how many studies are being done in this country. We're not quite even sure how many IRBs there are -- that every investigator is well-trained in research ethics.
Dr. MARSHALL: I think that there are some people who lose sight of the fact that the research subject should come first, and they get so excited about the potential benefits of the research for society as a whole that they might skimp on the protections, and that happens.
Dr. JOHN GALLIN (Director of Clinical Research, National Institutes of Health): We are at a point in history where our science is coming out with some of the most wonderful advances that we could ever imagine. The opportunities for bringing forward new medicines is just really spectacular.
ROLLIN: Imperfect as they may be and as they may always be, the future of medicine depends on clinical trials. Even critics feel they are necessary. But scientific investigators have to convince would-be participants that there is more reason to hope than to fear.
