More About the Mission Research Updates to Share With Your Donor Network As you continue in your training and fundraising efforts and begin to look forward to event weekend, you and your donors may want to know more about the progress we’re making toward curing blood cancers and ensuring higher quality of life for patients and families. Here are a few research updates to share as you continue in your journey toward the real finish line – cures for all blood cancers! We hope these short snapshots of progress in research will motivate and inspire! Targeted Therapies Targeted therapies inhibit or otherwise use specific molecules to selectively kill cancer cells. Compared to other treatments such as chemotherapy or radiation that harm healthy cells in an effort to eradicate the cancer, targeted therapies “hone in for the kill” and the patient faces fewer side effects because cancer cells are specifically targeted. This results in higher quality of life during treatment and after. Many of the first advances in use of targeted therapies were the direct result of research into blood cancers and LLS funding has been pivotal in moving this research forward. Targeted therapies first developed to treat blood cancers are showing promise in the treatment of breast cancer, Ovarian cancer and even Alzheimer’s. Gleevec – More on a Classic Success Story Gleevec, a targeted therapy approved in 2001 for the treatment of CML, required decades of research from basic science all the way through to clinical trials. Researchers around the country and in fact the world were involved in different aspects of investigation that led to Gleevec and LLS funding was critical at several key points, including discovery of genetic abnormality by Dr. Rowley and proof that inhibitor drug can work by Dr. Druker. The end result? Five year survival rate for newly diagnosed CML is now over 90% - roughly double what patients and families faced prior to the approval of Gleevec. Gleevec is also now FDA-approved for children and adults with a form of ALL, and patients with myelodys-plastic syndromes and myeloproliferative disorders. We didn’t stop with Gleevec but kept pushing forward, funding research that helped lead to even more potent drugs – Sprycel and Tasigna. These two drugs work for some patients that Gleevec doesn’t help and some patients may not need long-term treatments. Gleevec is now approved for rare stomach and skin cancers – previously there were no effective therapies for these patients. Gleevec is also showing promise for patients with brain, breast, ovarian and many other cancers. Several investigations are already in latestage clinical trials. For patients with Alzheimer’s, Gleevec will need to be reformulated to work better in the brain, but how amazing would it be for one drug to do so much for so many? How is it possible? All of these diseases carry related genetic abnormalities that have allowed one targeted therapy to be applied to improve outcomes and quality of life for so many. It took decades of research, but it is decades of research extremely well invested. Immunotherapies What are immunotherapies? Targeted therapies that help a patient’s own immune system fight infections and cancer. These include stem cell transplantation, vaccines, antibodies. Antibodies are natural or engineered immune cell products. In cancer treatment, they bind to cancer molecules and cells specifically to focus anti-cancer immunity and to directly kill cancer. Again, LLS funding has been critical across 2 decades and many researchers from target discovery to clinical trials and beyond! Rituxan – Another Important Success Story Rituxan is an antibody and was the first targeted cancer medicine approved by the U.S. FDA (1997). It’s used for patients with certain forms of non-Hodgkin lymphoma (NHL). Rituxan is now approved to treat multiple forms of NHL as well as Hodgkin lymphoma and CLL thanks to advances by LLS-funded investigators. It’s also used to treat patients with myeloma, Waldenstrom’s macroglobulinemia and ALL. It can be used to prevent GVHD after stem cell transplantation. All of this progress impacting both outcomes and quality of life was advanced by LLS-funded investigators. Rituxan has now been proven as a “maintenance” therapy, safely preserving remission in NHL patients. In combination with methotrexate, Rituxan is approved for patients with rheumatoid arthritis and is being tested for patients with other autoimmune diseases. And while this great progress is continuing, LLS is helping to develop other therapeutic antibodies. More Research is Needed It’s likely that multiple targeted therapies are needed for optimal treatment, including drugs, antibodies and vaccines. Most cancers involve multiple abnormalities and combined or multiple approaches may yield the best results for patients. We are part way there! Funding Success Success has been phenomenal, but blood cancer patients still have urgent needs and the advances we make in blood cancer treatments will help many other cancer patients. LLS funded almost 400 research projects last year alone, amounting to an investment of $72 million. We invest in the best around the world from basic science to clinical research and will continue our commitment to funding cures and improving quality of life during and after treatment. Of 39 new cancer drugs approved by the F.D.A since 2000, almost half (19) were approved as treatments for blood cancer patients! Many of these were advanced with LLS support and all 19 are being tested for patients with other diseases. Five have been approved for more patients beyond those for whom they were first developed.