A trial by Seattle-based Immusoft, a biotech company, said it is the first to genetically engineer B cells and test them in a patient, Wired reported Dec. 15.
The patient has mucopolysaccharidosis type I, a genetic disorder in which the body does not produce an essential enzyme to break down long-chain sugars inside cells. The disease can be terminal, and patients currently are treated with weekly infusions of the enzyme they lack.
The new treatment aims to override a genetic mutation causing the disease by prompting the person's B cells to make the enzyme. Scientists added genetic instructions to the cells in a lab before delivering them to the patient via IV infusion in an outpatient procedure.
The initial patient underwent an infusion of his modified B cells in mid-November and is reportedly doing well. Researchers are measuring the enzyme levels in the blood and studying any changes in the patient's symptoms.
One of the concerns the FDA has with engineered B cells is the possibility for them to turn cancerous. Immunosoft used a transposon system to insert the genetic material in a random fashion but said there is a theoretical risk that it could do so near a cancer-causing gene, the report said.