The BBC recently reported that an experiment using genetically-modified rabbits successfully treated a rare, but fatal, disease. The disease is called Pompe’s disease and is a muscular disorder caused by the lack of an enzyme. Children who are born with the disease usually die before their first birthday, as they have difficulties breathing and the heart and other muscles begin to give out.
Researchers at Sophia Children’s Hospital in Rotterdam, The Netherlands, first genetically modified rabbits by giving them a human gene that caused the rabbits to express the missing enzyme in their milk. The milk was then fed to four babies suffering from Pompe’s. The results were amazing. Most Pompe’s disease infants have trouble doing such basic things as rolling over, but one infant in the study was able to stand up by the time he was 1 year old. Two other babies who had almost completely lost muscle functioning and had to be placed on respirators halfway through the study had improved enough to leave the hospital on an outpatient basis.
The researchers noted that larger studies would need to be done to corroborate the initial findings and see if there are any other long-term impacts (aside from keeping infants alive past 1 year and restoring muscle function).
But as researcher Ans van der Ploeg told the BBC, “For the first time, there is a treatment for Pompe’s disease with clearly beneficial effects.”
This is an exciting time to be alive, unless you’re one of the activists bound and determined to make sure that exploration of these sorts of treatments gets stopped before they can save lives.
Source:
Rabbit milks saves babies. The BBC, July 28, 2000.