Here in Texas we have a highly respected magazine named Texas Monthly. It is a true read for all Texans that live in the state or around the world.
Much to my surprise last night, I was reading the current edition that I had just received when I saw the title of an article..."Bad Blood...Meningococcemia can kill its victims with stunning speed. But a new medicine tested by a Dallas doctor offers hope---if the FDA will approve"
The article starts out by telling the story of a young four-year-old girl named Brianna and her contracting menino. Sadly it goes into the gory details of what this disease does to the human body and more sadly the untimely death of this young child.
The doctor treating this young patient was one Dr. Brett Giroir and as the result of this case he began his quest to find a better treatment for this disease. It was at a Santa Fe, New Mexico conference that he heard of a "small" biotech company named XOMA. At the time XOMA was working with BPI for use in adult infections of various kinds and they apparently expressed to Dr. Giroir that they were not interested in pursuing trials with young children.
Apparently Dr. Giroir was a dedicated doctor, because as we all know, XOMA got FDA approval to start testing in the spring of 1995 a new BPI product, called Neuprex.
Now to the good part of the article and I quote..."Results were positive, immediate, and dramatic. Of the 26 patients, all children, who were treated with Neuprex at Children's(Dallas) and five other medical centers, only ONE died...a remarkable outcome, considering that the expected mortality rate had been 30%."
The article goes on to tell the story of a young 18 year-old from East Texas and his survival from the disease, based on his treatment with Neuprex.
Now for the conclusion of the article and the frustrating part.
I quote...."Given that kind of dramatic success one might think that BPI would have long since been okayed by CBER, especially considering that it has no known side effects. But even after three more years of testing on a total of 350 children, approval is not a sure thing. Our initial findings were good, says Girior. But CBER must see not only a dramatic reduction in mortality, they must see it prove out over the course of enough cases that there's no doubt that the reduction was due to use of the new medication. That's fairly easy to do with new medication for something that's widespread..say, hypertension."
"I just hope that CBER can see that this is an exceptional situation," he says, "a rare disease where mortality is so high that any new treatment that has shown it can reduce mortality by even a modest percentage is better than having half these kids die from this".
It concludes....For now, Girior can only wait and hope. The last phase of the BPI study will end sometime this spring; he expects to hear from the FDA later this year. And if the agency does demand more study, he'll continue his mission...because, he says, "What happened to Brianna was simply unacceptable." |
