Many people may remember the film The Curious Case of Benjamin Button, which starred Brad Pitt as the titular character who suffered from a rare aging disease.
What people may not know is that the disease in which Pitt's fictional character suffered from is known as progeria, which is an extremely rare genetic disorder that causes a person to age rapidly. Progeria reportedly affects 1 in every 20 million people and has no known cure.
A drug that was originally created for cancer patients, however, could help children who are diagnosed with progeria to live longer.
The Curious Case Of Progeria
Progeria was first discovered in 1886 and described as a very "rare," as those who have the disease are unable to pass it on due to the short lives they live. The genetic disorder is caused by an overabundance of the protein called progerin.
The drug lonafarnib, which has been used to treat cancer patients, was tested on 27 children who suffer from progeria. The participants were put on a daily two doses of lonafarnib pills, and this was conducted for over two years. Within that time, one patient died, and nine out of the 27 patients who were not treated with the drug also died.
A new study has since been conducted on 63 new patients. Out of the 63 patients, only four have died so far, which researchers believe to be an improvement. The researchers behind the newest study have also stressed that lonafarnib is not a cure for progeria but more of a treatment that could expand the lives of children who suffer from the disease.
Could Lonafarnib Give Kids More Time?
Carly Kudzia, who is an Ohio native, was diagnosed with progeria at just ten months. Carly is one few patients who have used lonafarnib and is seeing positive results from it.
Her mother, Heather Unsinger, stated that Carly is now just like "any other seven-year-old" and that since Carly has used lonafarnib, she has "more time with [her] daughter." Heather added that though the future is uncertain for her daughter, she is "thankful" for the minutes added onto her daughter's life.
Dr. Fuki Marie Hisama, who is a professor of medical genetics University of Washington in Seattle, also shared the same sentiments as Heather.
"These results are the first of their kind for this rare disease, and represent a significant breakthrough. This study gives hope to children with [progeria] and their families for better outcomes in the future," Hisama stated.
