Why informed consent is important

Steve Simon

2003-04-30

To understand why researchers today must obtain informed consent from their patients before they subject them to experiments, you need to understand some of the previous abuses of medical research.

Eva and Miriam Mozes

My thoughts were interrupted by the sound of the cattle car door as it opened. “Schnell, schnell.” The SS soldiers were ordering everybody out. As soon as we stepped out onto the cement platform, my mother grabbed my sister and me by the hand, hoping somehow to protect us. Everything was moving very fast. I suddenly realized that my father and my two older sisters, Edit and Aliz, were gone. I never saw them again. I think the whole thing took 10 minutes; they were lost in the crowd as Miriam and I clutched my mother’s hand. The SS soldiers walked by, shouting louder. Suddenly, they stopped my mother and looked at my twin sister and me, because we dressed alike and looked very much alike. “Are they twins?” one soldier asked my mother. My poor mother was bewildered. What was this place? she must have thought. What was happening here? What were the rules? What was a good answer and what was bad? She asked the SS soldier if being a twin was good. The guard nodded his head. My mother said very hesitantly, “Yes, they are.” Without further explanation, the officer grabbed Miriam and me, and another SS soldier grabbed my mother and pulled her in the opposite direction. We screamed and pleaded as we were separated. I remember looking back and seeing my mother’s arm stretched in despair as she was being pulled away. I never even said goodbye to her. I did not know that was the last time we would see our mother. – Eva Mozes-Kor

It was the Spring of 1944. Eva and her twin sister were at Birkenau. Dr. Josef Mengele was conducting medical research, and twins were highly prized for this research. No one told Eva or Miriam what was going on, what was being done to them or what their ultimate fate would be.

Eva was infected with a germ-she doesn’t know which one-and developed a fever. She has put in a hospital, but did not receive any care at all. Dr. Mengele and a team of doctors reviewed Eva’s fever chart but did not otherwise intervene. They gave her no medicine, no food. She had to crawl by herself to a water faucet to drink.

Eva realized that she would never get out until she convinced these doctors that she no longer had a fever. She manipulated the thermometers so that her fever gradually disappeared. After three weeks, she was re-united with her sister.

In very cold and cruel terms, Miriam would have been a matched control subject. Her healthy organs at autopsy would be compared to Eva’s disease ravaged organs.

Eva and Miriam had to endure a second experiment where Miriam was deliberately infected. They survived this and endured until the concentration camps were liberated. They emigrated to Israel in 1950.

Other medical experiments in the concentration camps

The medical experiments in the Nazi concentration camps were incredibly cruel and barbaric.

Many prisoners died during these experiments and more suffered permanent debilitating injury. The doctors' own scientific reports described the great agony that these patients suffered.

The victims who did not die in the course of such experiments surely wished they had. A long report written in July 1942 … describes an experiment on a former delicatessen clerk, who was given an oxygen mask and raised in the chamber to an atmospheric elevation of over 47,000 feet, at which point the mask was removed and a parachute descent was simulated. The report describes the victim’s reactions

The abuses in the Nazi concentration camps led to the development of the Nuremberg Code, the first document that provided guidelines on research ethics. The Nuremberg Code emphasizes that voluntary consent is a requirement for ethical research.

Examples of unethical research in the United States

While nothing can compare to the cruel and barbaric experiments at the concentration camps it would be a mistake for us to become too smug.

In each of these examples, the research subjects did not know they were part of an experiment and were not given a chance to refuse participation. Sadly, in the study of hepatitis, the parents of the mentally retarded children did sign a consent form on behalf of their children.

Jesse Gelsinger

Jesse Gelsinger had a rare disorder, ornithine transcarbamylase deficiency, that prevented his liver from removing the buildup of ammonia in his blood. This is a condition that often kills, and when Jesse was young, he did indeed almost die. A rigid diet and a daily cocktail of 32 pills eventually brought this condition under control.

Jesse’s condition made him an ideal test subject for a gene therapy experiment. This experiment would insert human gene fragments into a virus and infect patients with that virus. The hope was that this virus would insert these gene fragments into liver cells and return them to their normal genetic function.

The problem with gene therapy is that introducing viruses into a human body can make you very sick. Part of the trick in this research is to identify the right virus and to modify it so that it causes fewer side effects. Jim Wilson was head of the Institute for Human Gene Therapy at the University of Pennsylvania. He developed the largest program of gene therapy research in the nation and was looking for a candidate disease to show that gene therapy worked.

Ornithine transcarbamylase deficiency seemed like an ideal condition to try first. The initial experiments were solely to assess toxicity levels and Jesse Gelsinger, the eighteenth and last patient to be recruited for this study, understood that he himself would not benefit from this research. He wanted to do this for the children, he told his father. About half of all infants born with this deficiency die within a month.

On September 13, 1999, on Jesse Gelsinger’s eighteenth birthday, he arrived at the University of Pennsylvania to be injected with genetically modified adenoviruses. He was told to expect a minor reaction, such as a fever or flu-like symptoms.

Jesse’s parents recall their phone conversation that night.

PAUL GELSINGER: I received a call late in the evening from Jesse. He wasn’t feeling well, he was starting to have the side-effects from this treatment.

MICKIE GELSINGER: And I said are you feeling nauseous and he goes a little bit, I’m feeling out of sorts and I said you know it’s going to get a little bit worse, be prepared. He says yeah I know, I just don’t like it.

– BBC - Horizon - Trial and Error. www.bbc.co.uk/science/horizon/2003/trialerrortrans.shtml

The next morning, Jesse was disoriented and jaundiced. By that evening, he had slipped into a coma. The situation grew worse and no one understood why.

MICKIE GELSINGER: …this wasn’t anything they had expected and it was unbelievable. This boy, this young boy who at one time was such a little boy. His, he had, he had swollen, his whole body had swollen, he was unrecognisable. It, it just was heartbreaking.

PAUL GELSINGER: I couldn’t recognise Jesse. He was so swollen. It had, he was so swollen it had swollen his eyes shut.

MICKIE GELSINGER: And you were just standing there and thinking can’t believe this, this is your son and what have they done to him.

PAUL GELSINGER: It was shocking, it was not something I ever want to witness again.

– BBC - Horizon - Trial and Error. www.bbc.co.uk/science/horizon/2003/trialerrortrans.shtml

On September 17, Jesse Gelsinger died after the doctors removed life support. Jesse’s parents found out later that there were serious problems with the conduct of this research study.

When Jesse Gelsinger signed the informed consent form for the study, no one told him that

This problem was confounded by this particular patient. Jesse Gelsinger had a far more extensive liver deficiency than any of the previously tested patients and was ineligible for participation in the study.

Although Jesse Gelsinger had signed a sheet of paper, he had not given informed consent. He did not know about the risks identified in previous animal research. He did not know about risks identified in previous human volunteers. He did not know that his liver deficiency was worse than any of the previous seventeen research subjects. He did not know that dose that he was receiving was larger than that given to any previous subject.

Research guidelines in place today require the you seek out informed consent from your research subjects whenever you directly intervene or interact with a patient that would not otherwise occur or would occur differently except for this research. You also need informed consent when you review confidential patient records and analyze them for unusual trends, patterns, or associations.

Informed consent must state clearly that

There are some exceptions to informed consent.

The rules on informed consent are quite tricky, and you are not allowed to determine on your own whether your research does not require informed consent. You should discuss any proposed research with your IRB to get guidance on how best to proceed.

Disclaimer

I am not an expert on medical ethics or informed consent. I hope that this web page gives you greater awareness about why informed consent is so important, but I do not intend for this page to replace the detailed guidance available from your IRB or from the many resources on the web. See the reference list below for a few good examples.

Conclusion

Abuses of medical research in the Nazi concentration camps has led us to understand that research subjects must give free consent before we can undertake research on them.

The scientists of the world must remember that the research is being done for the sake of mankind and not for the sake of science.; scientists must never detach themselves from the humans they serve. I hope with all my heart that our sad stories will in some special way impel the international community to devise laws and rules to govern human experimentation. – Eva Mozes-Kor

References

Informed consent in medical research: Journals should not publish research to which patients have not given fully informed consent–with three exceptions. Doyal L. BMJ 1997: 314(7087); 1107. Available in html format

The Mengele Twins and Human Experimentation: A Personal Account. Mozes-Kor E. In: Annas GJ and Grodin MA ed. The Nazi Doctors and the Nuremberg Code. 1992; Vol. New York NY: Oxford University Press; 53-59.

Waiver or alteration of informed consent. Centers for Disease Control and Prevention. Available in pdf format. Link is broken.

Guidelines for Writing Informed Consent Documents. Office of Human Subjects Research. Accessed on 2003-05-02. “The ethical principle of respect for persons requires that subjects be given the opportunity to choose what shall and shall not happen to them. Valid informed consent requires: (1) Disclosure of relevant information to prospective subjects about the research; (2) their comprehension of the information, and (3) their voluntary agreement, free of coercion and undue influence, to research participation.” ohsr.od.nih.gov/info/finfo_6.php3

The Experiments. Tyson P. Accessed on 2003-04-30. “During World War II, Nazi doctors conducted as many as 30 different types of experiments on concentration-camp inmates. They performed these studies without the consent of the victims, who suffered indescribable pain, mutilation, permanent disability, or in many cases death as a result. At the Nuremberg “doctor’s trial,” which brought 23 German doctors to trial immediately after the war, prosecutors found 15 defendants guilty of war crimes and crimes against humanity; seven were hung.” www.pbs.org/wgbh/nova/holocaust/experiside.html

Bioethics: Gene therapy business: the tragic case of Jesse Gelsinger. News Weekly. Accessed on 2003-04-30. “A year ago Christmas, Jesse Gelsinger had a scary brush with death. He suffered from a disorder called partial ornithine transcarbamylase (OTC) deficiency, which kept him perpetually in danger of hoarding toxic levels of ammonia in his blood. Half of all infants born with the condition die within a month. But Jesse was lucky. Five days of intensive care and a change of medication revived him.” www.newsweekly.com.au/articles/2000aug12_bio.html

Trial and Error - programme summary. BBC Horizon. Accessed on 2003-04-30. “Jesse Gelsinger was born with a liver disorder, a rare condition called ornithine transcarbamylase (OTC) deficiency that stops the liver metabolising ammonia. People with the disease can suffer from brain damage or coma. At its most extreme the illness is fatal. Jesse was lucky, able to lead a fairly normal life although he had a daily cocktail of drugs to control his condition. Jesse wanted to help others. When he was offered a chance to take part in a medical trial to test the safety of using gene therapy for OTC deficiency, he was keen to participate. He knew this was not a cure for his condition but that, by volunteering he might be able to help others in the future.” www.bbc.co.uk/science/horizon/2003/trialerror.shtml

The Tuskegee Syphilis Study. Idea Works, Inc. Accessed on 2003-04-30. “600 low-income African-American males, 400 infected with syphilis are monitored for 40 years. Even though a proven cure (penicillin) became available in the 1950s, the study continues until 1972 with participants denied treatment. Perhaps as many as 100 died of syphilis during the study (Allen, 1978).” showme.missouri.edu/~socbrent/tuskegee.htm

You can find an earlier version of this page on my original website.