The University of Alabama at Birmingham has been awarded a $7 million research grant to continue its leadership in pioneering clinical trials in the treatment of neurofibromatosis 1 and 2 and schwannomatosis, all rare genetic diseases. According to the Birmingham Business Journal, the circumstances of these diseases lead to non-cancerous tumors forming on the nerves and potentially causing blindness, hearing loss, learning disabilities, pain or deformity.
Al.com reports that UAB is the operations center for the clinical research group that runs the trials and hosts the trials’ data management site. The university also enlists patients for the trials along with 12 other medical centers in the United States and Australia.
UAB Hospital
Image courtesy of Birmingham Business Journal
“We are tremendously grateful to be able to continue this work,” Bruce Korf, M.D., Ph.D., chair of the Department of Genetics at UAB and principal investigator for the research group said in an article on the UAB website. “Life for these patients is at best an anxious wait for the next complication and at worst a long trial of recurrent surgery, progressive loss of function or even death.”
Until neurofibromatosis’ genetic mechanisms were understood, researchers were unable to identify new treatments for the disease. Doctors tried to surgically remove persistent tumors, manage pain and use chemotherapy in rare cases where tumors became cancerous, but they weren’t always successful in treating the disease. Today, new drugs are available that allow effective clinical trials to take place. The $7 million research grant will also allow researchers to further improve clinical trials.
Researchers who design these trials have had to contend with the how rare and complex the disorders are. No single trial site can enlist the help of enough patients to complete a clinical trial for the majority of the symptoms. The volatile nature of neurofibromatosis and the fact that no two people have the same complications also makes designing clinical trials challenging. The UAB led consortium is doing their best to propose newer, more effective treatments, however, and the trials will greatly benefit from the new research grant.
University of Alabama's Bruce Korf
Image courtesy of University of Alabama
“The hope of these patients and families rests with the development of new treatments based on modern medicine, but the search for treatments could not even begin until we had a system in place capable of asking the right questions,” Korf said. Now that neurofibromatosis’ genetic nature has been discovered and the university has received a generous research grant, UAB hopes to continue clinical trials with a greater ability to successfully treat the disease.
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