The patient must remain in the hospital during this period, protected from everyday pathogens to which he or she has no resistance. Eight patients have been followed for three months or more. In a second study, Gewirtz and Luger are delivering the antisense agent by direct infusion, avoiding bone marrow transplantation altogether. In a Phase I clinical trial of this approach, twenty patients with untreatable or refractory leukemias were enrolled. Treatment by infusion is simple from the patient's point of view.
Patients receive the drug through a central catheter by continuous infusion for one week.
Then they go home for three weeks. As long as the disease remains stable or improves, the patients can return for further treatments. This can go on indefinitely and requires large amounts of the drug. In one especially dramatic case, a woman lived for almost a year and half, coming to Penn one week out of each month for the infusion. Average life expectancy in the blast phase of CML is typically no more than a few months.