The first 4 children treated with the experimental drug were saved from paralysis & a few have already been able to stand & walk again.
An effective experimental drug has been found in Jerusalem for a rare genetic disease of paralysis affecting Jews originating in North Africa, who previously refused to vaccinate their babies against childhood diseases because they thought that caused them to suffer from neurological damage.The first four children with the disease, which has caused not only paralysis of the muscles but also of the vocal cords (making them lose their ability to speak) have been treated with the experimental drug by doctors at Hadassah University Medical Center in Jerusalem’s Ein Kerem, with very good results.
The disease doesn’t even have a formal name except “The Curse,” which it is referred to by the susceptible population.
Prof. Orly El Peleg of Hadassah’s genetics department and Prof. Dror Mevorah, of the Center for Rheumatology Research and the internal medicine department, succeeded in identifying the gene and discovered the drug that halts progression of the disease.
The first four children who were treated were saved from paralysis, and some of them have already been able to stand and walk.
Their research findings appear in the latest issue of the journal Blood.
Peripheral neuropathy affects nerves that transmit signals from the spinal cord to the muscles of the limbs. The speedy and normal transmission of signals depends on the coating of the nerve, which is called myelin. In recent years, babies younger than a year were diagnosed with erosion of myelin and gradually became paralyzed in their limbs. All the parents were of North African Jewish origin – from Egypt, Libya and Iraq – and all the babies were completely healthy until they suffered from a simple viral infection with fever. But then they developed paralysis.
Since all the children were of North African origin and one of the families had two children with the condition, the geneticists were able to identify the defective gene using advanced technology.
The normal gene form serves as a brake against the virus, so the defective gene allows the awakened immune reaction against it to continue to attack the baby’s myelin.
The defective gene appears in 1:66 healthy people of North African origin.
Mevorah, who was in close contact with the families, chose a synthetic, experimental drug that halts the function of the immune system for another rare disease called PNH (paroxysmal nocturnal hemoglobinuria) – and it was effective on children with “The Curse.”
View original Jerusalem Post publication at: http://www.jpost.com/Health/Article.aspx?ID=296180&R=R1