Bluebird Sickle Cell Gene Therapy Ends Painful Attacks for Some
(Bloomberg) -- A single infusion of Bluebird Bio Inc.’s gene therapy for sickle cell disease eliminated serious pain crises and the need for red blood cell transfusions in a small study of patients getting the most advanced form of the therapy.
The results from 17 patients treated with Bluebird’s LentiGlobin were presented at the American Society of Hematology’s annual conference in Orlando, Florida. Over 21 months, none needed the transfusions that are a common treatment and none of those who previously suffered from pain attacks experienced any. More than 40% of the hemoglobin in their red blood cells came in a healthier form that was produced by the gene therapy.
The findings, albeit early stage, foreshadow a potential battle with two rival drugmakers to treat patients that until a few weeks ago had been overlooked for decades. Sickle cell disease, a genetic condition that deforms the red blood cells that deliver oxygen throughout the body, affects as many as 100,000 Americans, mainly blacks. Only last month did the U.S. Food and Drug Administration approve the first drug specifically designed to treat the condition, Novartis AG’s Adakveo, quickly followed by a second one, Global Blood Therapeutics Inc.’s Oxbryta.
Adakveo, a monthly infusion, cuts pain crises by half, while Oxbryta, a daily pill, appears to slow the progression of the disease. Both are more conventional treatments than LentiGlobin, a one-time injection that Bluebird hopes will eradicate debilitating episodes known informally as painful events.
“The patients that we’re treating here, and the possibility of fundamentally changing their lives and arguably their lifespan over time, that simply doesn’t exist with the other medicines,” Bluebird Chief Executive Officer Nick Leschly said. “You’re basically turning off the lights on events so far, versus some modest reduction for some period of time.”
The Cambridge, Massachusetts-based company presented data on 26 sickle cell patients treated up to four years earlier. Among those who received LentiGlobin made using an earlier manufacturing process, seven out of nine hadn’t needed any transfusion. All continued to produce some level of the anti-sickling hemoglobin -- the healthier form -- and had total hemoglobin levels of at least 9 grams per deciliter, higher than the average for patients with the condition.
Crispr Therapeutics AG and Vertex Pharmaceuticals Inc. are also working on a novel approach to treat sickle cell disease, using gene-editing tools. The companies reported promising data last month in the first sickle cell patient to receive CTX001, a one-time treatment that allows the body to resume making a healthier form of hemoglobin normally produced by infants.
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