Advances in Treatment and Promising New Therapies

Highlights from the Past Twenty Years

Theraputic Advances
Myeloma
The introduction of autologous (patient’s own) stem cell transplants, and the use of targeted drugs such as
Revlimid® and Velcade®, have improved median survival from less than three years to between five and ten
years.

Non-Hodgkin Lymphoma
The antibody drug, Rituxan®, has become standard therapy for patients with B-cell lymphomas. Rituxan taken
along with standard combination chemotherapy led to the first survival improvement for patients with diffuse
large B-cell lymphoma in more than two decades.

Chronic Lymphocytic Leukemia

Rituxan added to various combination chemotherapies to extend survival for patients with CLL, marks the first
significant advance in CLL treatment since the 1970s; five-year survival improved to nearly 80%.

Chronic Myelogenous Leukemia

Before the targeted drug Gleevec®, five-year survival was less than 40% for all CML patients; with Gleevec five-
year survival is 95%, with high quality of life, for early stage CML patients.

Promising Therapies on the Horizon

Vaccines and other immunotherapies:

Myeloma
Researchers are developing a new immunotherapy that can rebuild a patient’s immune system quickly after
stem cell transplantation, teaching the patient’s own immune cells to prevent life-threatening infections and to
attack tumor cells. The therapy is being tested for myeloma patients but is likely to also be applicable to patients
with other types of cancer.

Non-Hodgkin Lymphoma
Researchers are developing customized vaccines – made with proteins from a patient’s own tumor – that are
already showing promise in reducing relapse for patients with follicular non-Hodgkin lymphoma.

Leukemia
Learning how leukemia cells evade a patient’s immune system is helping researchers learn what causes
resistance to today’s therapies and how to bolster anti-cancer immunity tomorrow.

Making Therapies Safer

Nonmyeloablative (mini) stem cell transplants
Researchers have discovered that using radioactive antibodies to target cancer cells prior to a transplant
eliminates the need for high-dose chemotherapy, or whole body radiation, reducing the toxicity and serious side-
effects of the transplant procedure for many patients.
 Cord blood stem cell transplants
Stem cell transplants using umbilical cord blood cells are proving to be just as effective as transplants from
other sources, with a marked reduction in the risk of dangerous immune responses – known as Graft vs. Host
Disease. It seems that cord blood stem cells are less mature and do not need to be as well-matched as cells
from other sources.

Safer treatment of pediatric acute lymphocytic leukemia

Survival rates have reached nearly 90% for children with ALL but many children have suffered serious late
effects. Elimination of high-dose radiation treatments for most children has significantly reduced cognitive
deficits and other once common side-effects. Biological understanding has also led to treatments that reduce
the risk of heart damage from a common chemotherapy.

New Strategic Directions for LLS

Setting the agenda
The Leukemia & Lymphoma Society (LLS) is taking a more proactive approach to funding research, issuing
requests for applications from researchers to solve very specific problems. Examples:

• Following up on the successes noted above, LLS is actively seeking grant applications for studies of the
biological mechanisms that cause late effects from cancer therapy and impair quality of life.

• LLS has partnered with the International Waldenstrom’s Macroglobulinemia Foundation to seek research
proposals that will develop immortal cell lines that can be used to test potential new treatments for patients
with this disease.

• LLS has partnered with the Multiple Myeloma Research Foundation to support research focused on multiple
myeloma cancer stem cells needed to advance the understanding of drug resistance and relapse in patients
with the blood cancer.

Therapy Acceleration Program

LLS launched the Therapy Acceleration Program (TAP), a bold initiative intended to advance therapies likely
in the near-term to benefit patients suffering from blood cancers. Through TAP, LLS is taking a more results-
oriented approach, partnering directly with biotechnology companies with promising therapies to move them
more quickly through the FDA drug approval pipeline. The Academic Concierge Division of TAP provides support
to move LLS-funded academic research projects to the product development phase. Examples:

• LLS is supporting a clinical trial to test a promising immunotherapy treatment for chronic lymphocytic
leukemia patients who did not benefit from standard chemotherapy or have a “high risk” chromosomal
abnormality. The first two patients in the trial had no detectable leukemia cells after one cycle of treatment
and more patients are currently being treated.

• The LLS research team is helping an LLS-funded academic researcher advance a project testing a generic
FDA-approved antifungal medication as a potential treatment for patients with drug-resistant acute
myelogenous leukemia into a Phase I clinical trial scheduled to open in September 2009.

www.LLS.org • 888.HELP.LLS

Our Mission: Cure leukemia, lymphoma, Hodgkin’s disease and myeloma, and improve the quality of life of patients and their families.