Research Success Stories, for Blood Cancer Patients and More!

1. Research Success Stories, for Blood Cancer Patientsand More! Deborah E. Banker, Ph.D. Vice President, Research Communications

2. What is exciting in research ? Targeted therapies!!!

3. What are targeted therapies? • inhibit or otherwise use specific molecules (targets) to selectively kill cancer cells • Many of 1st advances in blood cancers; e.g. Gleevec, Velcade (and Rituxan – more in a minute)* • LLS funding critical in all of these success stories * see Fruits of our Funding in LLSpace

4. A favorite targeted therapy success story Gleevec – approved 2001 for CML patients • now 90%+ 5-yr survival for newly diagnosed CML • required decades of research investment - - from basic science to clinical trials - many researchers around the country / world • LLS funded research at critical points – including discovery of genetic abnormality by Dr. Rowley and proof that inhibitor drug can work by Dr. Druker

5. More success! • Gleevec also FDA-approved for children and adults with a form of ALL, and patients with myelodys-plastic syndromes and myeloproliferative disorders – related molecular abnormalities • LLS kept funding research that helped lead to even more potent drugs – Sprycel and Tasigna - work for some patients that Gleevec doesn’t help! - may not need long-term treatments

6. More success! • also approved for rare stomach and skin cancers – previously no effective therapy for these patients – related molecular abnormalities! • Gleevec showing promise for patients with brain, breast, ovarian and many other cancers – already in late-stage clinical trials – related abnormalities • and, in Alzheimer’s – again a related molecular abnormality involved – will need to be reformulated to work better in the brain, but how cool is this??

7. What else is exciting in research ? Immunotherapies!!!

8. What are Immunotherapies? Targeted therapies that • help a patient’s own immune system fight infections and cancer • include stem cell transplantation, vaccines, antibodies LLS funding critical here too.

9. What areAntibodies? • natural or engineered immune cell products • bind cancer molecules /cells specifically • focus anti-cancer immunity and/or direct cancer killing

10. A favorite antibody success story – Rituxan • LLS funding critical across ~20 years, multiple researchers, from target discovery to clinical trials in non-Hodgkin lymphoma patients, and beyond • Rituxan was the first targeted cancer medicine approved by the U.S. FDA (1997), for patients with certain forms of non-Hodgkin lymphoma (NHL).

11. More success! • Rituxan now approved to treat multiple forms of NHL, and patients with Hodgkin lymphoma or chronic lymphocytic leukemia (CLL), thanks to advances by LLS-funded investigators. • Rituxan is also used to treat patients with myeloma, Waldenstrom’s macroglobulinemia, ALL, and to prevent GVHD after stem cell transplantation, advanced by multiple LLS-funded investigators

12. And, more success! • Rituxan now proven as “maintenance” therapy, safely preserving remissions in NHL patients • Rituxan + methotrexate is approved for patients with rheumatoid arthritis and being tested for patients with other autoimmune diseases • LLS helping to develop other therapeutic antibodies * see Fruits of our Funding in LLSpace Research section

13. Multiple targeted therapies are probably needed (incl. drugs, antibodies, vaccines…)for optimal therapies as most cancers involve multiple abnormalities. We are part way there!!

14. LLS Continues Funding Success • blood cancer patients still have urgent needs • almost 400 research projects last year alone ($72M) • the best, around the world • from basic to clinical research • advances help many cancer patients

15. Far-Reaching Success • 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. • All 19 being tested for patients with other diseases • 5 (incl. Gleevec) already approvedfor more patients, beyond those for whom they were first developed

16. Learn More on LLSpace Dr. Banker's Research Nuggets- One-page summaries of important research stories LLS Research Fast Facts – The basics you need to know for your next meeting LLS Research Areas and Advances – More, including specific funding distribution Fruits of our Funding - How LLS funding advanced important treatmentsincl. for other patients LLS Research Grants In Force -Find researchers in your chapter area

17. Food for Thought How can you use this information to steward LLSsupporters or reach out to new prospects? i.e. Why should LLS supporters care????? How can you make these stories your own?