Gene Therapy: Curing Sickle Cell

By Shlomo Maital      

      New York Times science reporter Gina Kolata has just published a moving account (Sept. 16) about one of the first children to be treated with a new gene therapy that will free him “from the sickle cell disease that has stolen his childhood”.

      The child, 11 year old Kendric Cromer, has had periods of escruciating pain.  Sickle cell eroded his hip bones, kept him from riding a bike or playing soccer or even going outside when the temperature was below 55 F. – because the cold brought intense pain.

       Sickle cell is a hemoglobin-related blood disorder, genetic in nature,  caused by an abnormality in the oxygen-carrying protein haemoglobin found in red blood cells. 

         Red blood cells are normally round.  Deformed red blood cells are shaped like a new moon.   The deformed sickle cells cannot carry oxygen properly to all parts of the body, resulting in great pain.

          Gene therapy repairs the disease, by giving the patient’s blood-forming cells a new, normal hemoglobin gene.    Some 100,000 Americans, most of them African-Americans, have this awful disease.  Gene therapy could cure 20,000 of the most severely afflicted.  But the treatment is hugely expensive, difficult (Kevin underwent four days of intense chemotherapy to clear his bone marrow and make way for the new cells).  The treatment costs hospitals $2.2 million per patient (from the Boston biotech company Vertex) or $3.1 million from another firm, Bluebird.  And hospitals pay upfront, then are reimbursed.

           Bluebird took cells from Kendric, treated them, modified the offending gene, and then, reinjected the healthy genes into Kendric’s body.   For a month, “Kendric will be very ill, with ulcerating sores in his mouth, throat and esophagus…”

                Kendric’s goal?  “I really just want to be a kid,”  he says. 

                Go for it, Kendric.   Every kid deserves to be..just a kid.