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May 17, 2002


Atlanta Researchers to Launch Clinical Trial of Progesterone for Treatment of Brain Injury

Researchers from Emory University and Morehouse School of Medicine soon will begin enrolling patients in the world's first clinical trial of the hormone progesterone as a treatment for moderate to severe traumatic brain injury, which annually claims the lives of 50,000 Americans and disables 80,000 more, at an estimated cost of $56 billion. Currently, there is little that doctors can do for this condition beyond providing supportive care.

The three-year pilot study is called "ProTECT", which stands for Progesterone for Traumatic brain injury, Experimental Clinical Treatment. The study has been approved by the U.S. Food and Drug Administration and is funded by the National Institutes of Health (NIH). It will be based at Grady Memorial Hospital, where physicians from Emory will work side-by-side with colleagues from Morehouse to evaluate a treatment that has proved effective in animal models but has not yet been tested in humans.

Researchers, led by Emory University neurobiologist Donald Stein, PhD, have found in a series of experiments that male and female rats with brain injury develop less brain swelling and recover more completely when they are treated with progesterone shortly following injury. The hormone seems to moderate the inflammation that frequently leads to dangerous brain swelling following head injury. Progesterone also seems to slow or block a cascade of damaging chemicals known as free radicals that are unleashed by traumatic injury, leading to the wholesale death of brain cells. Progesterone occurs naturally in small amounts in the brains of male and females, both in animals and in humans.

"Progesterone has been safely used for decades to treat medical conditions in both women and men, sometimes for months or even years at a time," said Arthur Kellermann, MD, MPH, chairman of emergency medicine at Emory and principal investigator of the new study. "Furthermore, progesterone has been safely given intravenously to humans at much higher doses than that planned in our study, which will limit progesterone treatment to only three days."

When corticosteroids, which were once prescribed to control brain swelling, were found to be ineffective, emergency physicians and trauma surgeons were left with no effective therapy for blunt force trauma to the brain. Although barbiturates, mannitol, and surgery are sometimes used in an attempt to relieve pressure inside the skull that can occur when a victimÕs brain swells, they are measures of last resort that may or may not work.

"In contrast to the many options available to treat other types of injuries," Dr. Kellermann notes, "brain injury remains the final frontier. We want to take the crucial next step and determine whether giving progesterone to victims of moderate to severe traumatic brain injury will save lives and preserve mental function."

The new study will begin on May 28th and will enroll up to 100 victims of traumatic brain injury over the next two years. Four out of five patients (80%) will be administered progesterone by intravenous infusion for three days. One out of five patients (20%) will be administered a placebo, consisting of intravenous fluid without progesterone. Patients will be evaluated for mental functioning on discharge from the hospital and at one month following injury. Neither the patients, their families, the doctors or nurses treating them, nor the study team will know who received progesterone and who did not until the end of study.

When progesterone is administered to men, it temporarily suppresses the level of the male hormone testosterone in the blood. However, when progesterone wears off, testosterone levels return to normal. Although progesterone may slightly increase the risk of thromboembolism (blood clots) in the legs or lungs, researchers believe the risk is minimal compared to the potential benefits for victims of brain injury.

Most study participants will be victims of an automobile or motorcycle crash, a fall, or an assault with a blunt object such as a baseball bat. The study will not enroll victims of gunshots or other penetrating head wounds.

The study will be open to all races and both sexes. Pregnant women and individuals suffering from soybean or egg allergies or cancer of the breast or reproductive organs will not be eligible. Because the tests used to evaluate recovery have only been validated among English-speaking patients, enrollment will be limited to adult English speakers. Since brain-injured patients cannot think clearly, consent must be granted on their behalf by a family member or legal representative. To gain entry into the study, patients must arrive at Grady and a family member must be found to grant consent within 10 hours of injury -- the sooner, the better. If a family member cannot be located within that time frame, the patient cannot be enrolled in the study.

Patients will be monitored closely in the Surgical or Neuro intensive care unit at Grady or in a new NIH-funded area of Grady called the General Clinical Research Center. An independent safety committee appointed by the NIH will oversee the project. This group has the power to end the study early if it is clear that one treatment group is doing better than the other. If the results of the pilot phase study are promising, the researchers anticipate that the study will be extended to additional level I trauma centers in other US cities in future years.

Rates of traumatic brain injury are considerably higher in Georgia and in metro Atlanta than in the country as a whole. In 1999, DeKalb County reported 110 deaths from traumatic brain injury and Fulton County 193 deaths, ranking them in the 75th to 90th percentiles nationally among all US counties. In 1999, traumatic brain injury was responsible for 56 deaths at Grady, out of 429 brain injury patients presenting to the emergency department, and in 2000, 53 deaths, out of 568 brain injury patients.

"Traumatic brain injury is one of the most devastating and debilitating injuries anyone can suffer, and it inflicts its damage disproportionately in metro Atlanta," said Dr. Kellermann. "We are tremendously excited by the prospect that through this clinical trial, we may be able to offer hope to those devastated by brain injury."

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