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.
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.
disease doesn’t even have a formal name except “The Curse,” which it is referred
to by the susceptible population.
Prof. Orly ElPeleg 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.
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.
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