A New Investigational Drug for Brain Injured Patients
Joanmarie Pellegrini, MD
Tuesday, March 27, 2012
Brain injury is very difficult to treat because there are no effective medications. When someone suffers a serious brain injury the only options for the medical team is to support them through the healing process. Sometimes a neurosurgeon may offer surgery to drain blood or remove part of the brain, however, even after surgery there is only supportive care. Over the past few decades there have been several studies looking at various medications that may improve outcomes from brain injury. Unfortunately the results have not been promising.
Physicians who care for these patients noticed many years ago that women tended to have better outcomes. The first study on this involved rats. Male and female rats were subjected to a traumatic brain injury and the female rats had better outcomes (they measured outcome using mazes and testing the rats’ ability to navigate the maze). It was hypothesized that it might be the female hormones that were helping the brain heal. So, the next study looked at “pseudo-pregnant” rats and these rats did even better in functional outcome. More studies were done that led to the hypothesis that it was progesterone that was acting as a “neuro-steroid” or as a neuro-protective hormone enabling better healing and functional outcomes.
BHR Pharma is a Belgian company that specializes in producing medications that contain progesterone. There have been phase 1 and 2 studies done that give progesterone to brain injured patients. These initial studies have had promising results. In fact, it seems that not only do less patients die but the ones that survive have better functional outcomes. The results are promising enough that the FDA gave BHR permission to conduct a large phase 3 trial. This trial is a randomized, placebo-controlled, double-blinded study that is looking to enroll over 1100 patients internationally. This type of study is considered to be the most rigorous for testing a hypothesis. EMMC is one of the study sites.
This is how the study works: when a patient suffers a serious head injury they are almost always transferred to the nearest trauma center (which is EMMC for this part of the state). The emergency personnel and trauma physicians will give all of the standard care that the patient needs. Part of the evaluation always includes a CT scan of the head. If this confirms the presence of a serious head injury and the patient is in a coma, then the patient may be a candidate for the study and the family may be approached and asked for their consent.
The study requires that each patient be randomly assigned to either the “placebo” group or the “test drug” group. The drug is a high dose five day infusion of progesterone. All of the other standard treatments and supportive care are still given to the patient. The patient then comes back to EMMC at 3 months and 6 months for a lengthy questionnaire about how well they are doing and whether there is anything they cannot do that they could do before their injury.
There are a few things that make it difficult to do this study. First, in order to have the most impact on brain injury, any treatment must be given as soon as possible. For this particular study, the study drug must be given to the patient no later than 8 hours after injury. Also, the next of kin must be present at the hospital in order to give consent to be enrolled in the study.
This study is still enrolling patients and will not be closed for another year or so. Therefore, it will be at least another two years before the data is published. It is my hope and the hope of the medical community that this drug turns out to be effective. If so, this would be the first promising new treatment for brain injury.