TARGETED THERAPY RESEARCH
Discovering the molecular abnormalities that cause particular types of blood cancer has been useful
in diagnosis and risk stratification, and in new “targeted drug” development. LLS-funded
investigators have helped advance molecularly targeted treatments that can selectively kill blood
cancer cells versus normal cells. Many of these new treatments benefit not only blood cancer
patients, but also patients with other diseases. For example:
Gleevec® is FDA-approved for patients of all ages with chronic myeloid leukemia (CML), and
is also approved for patients with one form of acute lymphoid leukemia (ALL), myelodysplastic
syndromes (MDS), myeloproliferative disorders and rare forms of stomach and skin cancers.
Related drugs, Sprycel® and Tasigna®, are approved for patients who do not benefit from
Gleevec. One or more of these drugs are also showing promise for patients with various
lymphomas, acute myeloid leukemia (AML), chronic lymphocytic leukemia (CLL), and other
cancers, including brain, breast, head-and-neck, lung, pancreatic, and prostate cancers, and
patients with other diseases including Alzheimer’s, asthma and pulmonary hypertension.
Rituxan® was the first FDA-approved, anti-cancer antibody drug, developed for patients with
forms of B-cell non-Hodgkin lymphoma (NHL). It is now also approved for CLL patients and as
a “maintenance” therapy for follicular lymphoma patients, and showing promise for patients with
ALL and after stem cell transplantation. In addition, it is approved for treating patients with
severe rheumatoid arthritis and two other types of autoimmune diseases. A related antibody drug,
Arzerra®, is approved for CLL patients and showing wider promise.
Velcade®, Thalidomid® and Revlimid® are FDA-approved for patients with myeloma and are
also helping some patients with Hodgkin lymphoma and NHL.
Krypolis® was recently
approved for myeloma patients for whom at least two prior therapies were insufficient. One or
more of these drugs are now being tested for patients with T-cell and B-cell forms of lymphoma,
acute leukemias, as well as AIDS-related Kaposi sarcoma and brain, breast, colorectal, head-andneck,
kidney, liver, lung, ovarian and prostate cancers, and Alzheimer’s disease.
Istodax®, Zolinza®, Dacogen® and Vidaza® target small chemical, “epigenetic” changes. The
first two drugs are approved for patients with peripheral T-cell lymphomas; the latter drugs are
approved for MDS patients. One or more of these drugs are being tested for patients with ALL,
AML, CML, CLL, myeloma and forms of NHL, after stem cell transplantation, and for patients
with breast, brain, kidney, colorectal, head-and-neck, lung, stomach, prostate and ovarian cancers, melanomas well as sickle cell disease and persistent HIV infections.
