The findings of the researchers at the Johns Hopkins University may help save the lives of over 30,000 Americans suffering from Huntington's disease. Moreover, the drug may help prevent another 200,000 people from of inheriting the ailment.
Foremost, proteins and salts (components of a brain cell) need to move in and out of the nucleus in order to function properly. Now, the HTT gene that triggers Huntington disease secretes proteins that clump together in the nucleus to block the passage. If the proteins fail to enter, the cell will shut down and die eventually.
The study may also aid scientists in understanding other fatal brain diseases like amyotrophic lateral sclerosis and dementia. John Grima of Johns Hopkins University conducted the research published in the journal "Neuron". He is a graduate student from the Department of Neuroscience in the School of Medicine.
According to Grima, his team has found a "traffic jam" in cells. If they could fix it, a new avenue for treatment of neurodegenerative diseases may become available soon. Nonetheless, he is not yet sure whether clearing these "traffic jams" could stop cells from dying and could hinder the progression of Huntington's disease. In any case, Grima assured that his group aims to develop a drug that could do both.
As of now, Morningside Maryland reported, there are only two treatments available for Huntington's. The latest was approved by the FDA just over a week ago. Both drugs improve mobility and gait problems associated with Huntington's patients. Unfortunately, there is no available medication that could slow or stop the progression of the disease. That is what Grima's team is working on right now.
For the record, the onset of the symptoms begins between ages 30 and 50. Alarmingly, half of these patients will transfer the ailment to their offspring. If not treated within 10 to 25 years after initial diagnosis, the affected person would lose the ability to speak, walk, and reason out.
Per Medical Express, the researchers are working in the lab of Dr. Jeffrey Rothstein, the director of Hopkin's Brain Science Institute. Other schools like the University of Florida and the University of California (Irvine) took part in the demonstration of how Huntington's disease disrupts normal cell activity.
