Q: What kind of side effects have you seen in the study?
The most common toxicities that we found were minor nausea and vomiting. We also had some toxicity related to what we call the hematological parameters, which is a little bit of anemia, and decrease of white blood cells, which is no surprise in patients with leukemia at the start.
Q: How safe is this drug in children?
Although we did escalate the drug by different levels, and we increased the drug at a fairly high amount of total dose, we did not encounter significant toxicity of one organ or one function of the body that will prevent using the drug in children at the set dose. So, actually the drug was well tolerated for most of the kids.
Q: And when will the trial be completed?
The trial is still underway. We're looking for more patients so that we can test the drug at two levels, to find out if the preliminary information we got is consistent.
Q: This drug has been tested for safety in children, but what about efficacy?
We have no data yet that I can comment on. But we hope at least from our preclinical model that it will work, because the Philadelphia chromosome, which is the defining feature in these types of leukemia, is not different in the pediatric patients than it is in the adults. But we have to figure that out. We just don't know yet.
Q: Do you foresee any future implications for the use of Gleevec in children with leukemia?
There will be many challenges, because these particular types of leukemia are different in children than they are in adults. We'll ask medical oncologists to figure out what the best timing and schedule would be for giving the drug to children. We'll have to look at how we can incorporate the drug with chemotherapy trials, to see if we can build better results by using combination therapies.
Q: Do you believe that this drug could be a cure for leukemia in children as well as adults?
I hope for some kids it will be the solution. But you have to keep in mind that these types of leukemia are rare in children. They represent only about 2 to 3 percent of the most common type of leukemia, which is the acute lymphoblastic leukemia. So the major proportion of patients will have to rely still on other therapeutics to achieve a cure.
