Year 3

During the past 12 months, our disease team has made further progress in

the development of stem cell targeted treatment for chronic lymphocytic

leukemias and other leukemias. Stem cells express some molecules on the

surface that are different from the corresponding molecules on adult

cells. The ROR1 molecule is highly expressed by malignant cells from

patients with chronic lymphocytic leukemia, as well as by progenitor cells

from other forms of leukemia and lymphoma. It is not expressed by normal

adult cells. With the support of the CIRM Disease Team grant, the

cooperating investigators have prepared a humanized monoclonal antibody against the

ROR1 molecule, that is potent and specific. In animal models, the

antibodies can retard leukemia growth and spread. Unlike other anti-cancer

drugs, the new antibodies are not toxic for normal bone marrow cells.

Thus, they can potentiate the action of other agents used for the

treatment of leukemia.

The disease team is now focused on the pre-clinical development, safety

testing, and scale-up manufacturing of our new, promising agents, in

preparation for their introduction into the clinic.