A teenager suffering from Sickle Cell Disease underwent a new procedure and has been symptom free
for 15 months
Orange County, CA - March 3rd 2017 - Sickle cell disease is a hereditary disease that affects a group of red blood cells. The normally round and healthy red blood cells that carry oxygen through the body wither to become hard and sticky C-shaped cells. These cells have a shorter life cycle than normal cells and cause a constant shortage of red blood cells. When traveling through narrow blood vessels these cells can fasten together and block blood flow, potentially causing serious pain and if persistent organ failure. The disease has a range of severity differing from patient to patient with different treatment options for each person, but a new revolutionary procedure could reverse the disease.
Necker Children's Hospital in Paris performed a unique procedure on a French teenager suffering from the disease. The disease had progressed to such an extent that the patient was forced to receive monthly blood transfusions to dilute the defective blood. Even with these transfusions there was so much internal damage to the patient’s body his spleen was removed and his hips were replaced. At 13 years old, his doctors decided to try a different treatment that requires removal of bone marrow for genetic alteration before returning the marrow to the patient. The modifications to the marrow are done by laboratories with a virus which aids in correcting the defective agents in the blood.
Results from the procedure show the patient to be producing normal blood cells for the past 15 months, with no signs of relapse. Though still wary of using the word cure, Dr. Philippe Leboulch, a professor of medicine at the University of Paris, has said the patient, the first in clinical trials for the treatment, hasn’t shown any signs of the disease returning, has had no pain, and no longer requires monthly blood transfusions.
Dr. Deborah Gill, from the gene medicine research group at the University of Oxford, views the work as very significant. “I've worked in gene therapy for a long time and we make small steps and know there's years more work, but here you have someone who has received gene therapy and has complete clinical remission - that's a huge step forward."
Though the treatment has the opportunity of significantly reducing the number of people suffering from the disease, it can only be carried out in modernized facilities. Even more troubling, the majority of sickle cell patients reside in Sub-Sahara Africa. For now, the team is going to continue trials in hopes of helping the millions of people suffering from the disease.
To read their study click here.
