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

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

3. Patient Services Research Programs Professional Education Volunteers Advocacy Community Services

4. Research funding and support • Since 1949, LLS has awarded more than $600 million to fund research

5. FamilySupport Groups First Connection Program Patient Financial Aid Patient and family support programs Co-Pay Assistance Program 877-557-2672 www.LLS.org/copay

6. Resources

7. What to expect • In this program, you will learn: • How new blood cancer treatments are developed and approved • About recent and ongoing advances in the treatment of blood cancers • To distinguish between myths and facts about clinical trials • About benefits and risks of clinical trials • To ask the right questions to help you decide if a clinical trialis right for you • How to locate clinical trials that may be right for you • The Leukemia & Lymphoma Society can help

8. What are cancer clinical trials? • Cancer clinical trials are research studies conducted by medical scientists to improve the care and treatment of cancer patients • Cancer clinical trials test new ways to: • Diagnose and treat cancer in people • Prevent or reduce disease or treatment side effects • Prevent a recurrence of cancer • Improve the comfort and quality of life of people with cancer

9. The promise of clinical trials • Progress through research has helped people with blood cancers: • Live longer • Have greatly improved quality of life

10. Drug tested in lab and animals Discovery of new drug in lab • Food and Drug Administration approves Investigational New Drug (IND) application • The drug can now be tested in humans through a clinical trial Drug discovery: The journey from lab to patient

11. Phase 1 clinical trials • What method of drug delivery and dosage is safest? • How does the drug affect the human body? Drug discovery: The journey from lab to patient • Phase 2 clinical trials • Does the drug have an effect on the cancer?

12. Drug discovery: The journey from lab to patient • Phase 3 clinical trials • Is the new drug or combinationof drugs better than the best current treatment? • Researchers submit New Drug Application (NDA) to FDA • FDA decides whether to approve the drug for use in all patients • Is it more effective or does it have fewer side effects than standard treatment?

13. Phase 4 clinical trials • Ongoing research • Study the effectiveness or side effects of an FDA-approved drug over time in a large population of patients New drug available by prescription for all patients who need it FDA approves new drug for sale and marketing in the United States Drug discovery: The journey from lab to patient

14. Treatment advances for leukemia, lymphoma, myeloma and myelodysplastic syndromes (MDS):From clinical trials to FDA approval Some of the drugs that were tested in clinical trials and then approved by the FDA in the last decade to treat leukemia include: • Acute Myelogenous Leukemia: gemtuzumabozogamicin (Mylotarg®) • Acute Lymphocytic Leukemia: clofarabine (Clolar®) • Chronic Lymphocytic Leukemia: alemtuzumab (Campath®)and bendamustine HCI (Treanda®) • Chronic Myelogenous Leukemia: imatinibmesylate (Gleevec®), dasatinib (Sprycel®)and nilotinib (Tasigna®)

15. Treatment advances for leukemia, lymphoma, myeloma and myelodysplastic syndromes (MDS):From clinical trials to FDA approval Some of the drugs that were tested in clinical trials and then approved by the FDA in the last decade to treat lymphoma, myeloma and MDS include: • Lymphoma: rituximab (Rituxan®), iodine131-tositumomab (Bexxar®), yttrium-90 ibritumomabtiuxetan (Zevalin®), bortezomib (Velcade®), bendamustine HCI (Treanda®) and denileukindiftitox (Ontak®) • Myeloma: thalidomide (Thalomid®), lenalidomide (Revlimid®) and Velcade® • MDS: Revlimid®, azacitidine (Vidaza®) and decitabine(Dacogen®) • Stem Cell Transplantation: plerixafor (Mozobil®)

16. Door is opened to discovery of other promising new targeted therapies now being tested Finding better therapies for blood cancers

17. The more people who take part in clinical trials, the faster we will findbetter ways to treat blood cancers Participation is crucial

18. Participation in clinical trials: What gets in the way? • People with cancer may… • Not have access to clinical trials • Fear or distrust medical research • Face practical obstacles • Worry about cost • Not want to ask their current doctor about clinical trials or not want to go against their doctor’s wishes • Be less likely to participate in clinical trials if they are an ethnic minority or an older patient, even though there may be many clinical trial options for them

19. Participation in clinical trials: What gets in the way? • Doctors might… • Be unaware of appropriate clinical trials • Believe that standard therapy is best • Be concerned about administrative burdens • Be unwilling to “lose control” of a patient’s care • Be concerned about how a patient will react tothe suggestion of a clinical trial • “The physician is the most trusted, primary source of awareness and influence in decisions concerning cancer clinical trials. Lack of physician communication and commitment may be the greatest barriers to clinical trial participation.”

20. Learning the facts about clinical trials

21. If I enter a clinical trial, there’s a good chance that I could receive a placebo True or false? FALSE

22. Clinical trial study design or protocol • Fact: Placebos are rarely used in cancer clinical trials • Participants usually receive: • The best known, most widely accepted treatment, called the standard treatment (control group) Or • The new treatment being studied (investigational or study group)

23. Clinical trial study design or protocol • Each cancer clinical trial has a written detailed study design called a protocol that includes: • Background of why the clinical trial is needed • Purpose of the clinical trial • What drug or drug(s) are being tested, along with a treatment and follow-up schedule • Safety measures throughout the clinical trial program • How outcomes will be measured • Who is eligible for the clinical trial • How the clinical trial will be organized, that is, one site or multiple sites • If the clinical trial is a multi-site trial, then all participating physicians must follow the same protocol

24. You have an equal chance of being assigned to one group or the other Control group gets standard treatment Randomization Investigational or study group gets new drug being tested Phase 3 clinical trials: Getting assigned to a group

25. Clinical trials are the treatment of last resort True or false? FALSE

26. Eligibility • Facts: Clinical trials… • Are not only for people with the most advanced disease • Can also be for people with newly diagnosed or very limited disease • Are appropriate for different types of people, depending on the purpose and phase of the trial • Take into account age, gender, race, stage of disease, other treatments used, and presence of any other illness

27. If I enter a clinical trial, I’ll be treated like a “guinea pig” True or false? FALSE

28. Safety • Fact: Each clinical trial is managed accordingto a strict set of rules to protect patients • Protocols regulate: • Quality of care • Safety of participants

29. How is a patient’s safety protected? • Sponsor asks outside experts to review merit of study • Institutional Review Board(committee made up of experts) • Are risks minimized and reasonable vs anticipated benefits? • Is informed consent process in place and documented? (no coercion or “undue” influence to participate) • Does data monitoring include patient safety data? • Is there a process to protect privacy of patients?

30. Take your time in reading and signing the informed consent form How is a patient’s safety protected? • Researchers must tell you the purpose, benefits, and risks of a clinical trial before you sign up • Must be in language you understand • Is called the process of informed consent • Ask questions if anything is unclear • Ask for a language interpreter if needed • Bring an advocate

31. Care and treatment received are free True or false? Both TRUE and FALSE

32. Costs Facts: • Some costs may be covered by the sponsor of a clinical trial • Other costs may or may not be covered by health insurance plans • It is important to learn ahead of time what will be covered

33. What’s covered, what’s not • Costs that are usually covered by the sponsor at no cost to the patient: • Research doctor and nurse time • Cost of drug being studied • Costs that may not be covered by your private or public health insurance (costs that you would have whether or not you were in a clinical trial): • Hospitalizations • Doctor visits • Laboratory tests • Drugs that are not part of the study design

34. What’s covered, what’s not • A growing number of U.S. states have passed legislation or instituted special agreements requiring health plans to paythe cost of routine medical care in clinicaltrials. For information on your state go to: http://www.cancer.gov/clinicaltrials/ctlaws-home • Not all of these laws are the same in defining what coverage is required • To get involved with advocacy efforts through LLS, contact our Office of Public Policy at 202-543-7033 or visit http://action.LLS.org or the National Cancer Institute’s Web site, www.cancer.gov/ clinicaltrials

35. What’s covered, what’s not • Discuss treatment costs with the clinical trial team and your insurance provider to find out • What costs will be covered by the clinical trial sponsor, your insurance provider or by you if you have no insurance or your health insurance will not pay all routine care costs • How to process an appeal with your clinical trials team if your claim is denied by the insurance company because the treatment is in a clinical trial • Check out The Leukemia & Lymphoma Society’s Financial Health Matters booklet, speak with your chapter patient services manager or contact the LLS Information Resource Center at 800-955-4572 to find out about sources of support

36. I’m too old to enter a clinical trial True or false? Increasingly, FALSE

37. Age • Fact: A growing number of clinical trials are seeking to include older adults

38. Older adults and clinical trials • Cancer treatment decisions, and decisions about clinical trials, should no longer be based on age alone • Age alone is not a predictor of how well a cancer treatment will work • Many clinical trials are targeted to older adults that address treatment challenges throughout the cancer journey • Previously untreated patients • Patients resistant or intolerant to current treatment • Patients who have relapsed

39. My Clinical Trials Journey

40. Clinical trials: Weighing the pros and cons • Possible risks • Compared with standard treatment, the new treatmentbeing studied may: • Not be better than, or as good as, standard treatment • Have more serious side effects than standard treatment • Not work for everyone • Costs may not be covered

41. Clinical trials: Weighing the pros and cons • Possible benefits • Receive, at minimum, the best standard treatment available • Be among the first to benefit from a new treatment • Receive high-quality care • Have access to doctors with extensive experience in your type of cancer

42. Clinical trials: Talking with your doctor • Be an active partner in your treatment • Ask your doctor about all available treatment options, including clinical trials • Consider bringing someone with you to help listen and ask questions Models are used for illustrative purposes only. • Have you discussed clinical trials as a treatment option with your oncologist? • Tell us why you are or why you are not comfortable asking about clinical trials at your next visit.

43. Ongoing clinical trials for blood cancers • Thousands of clinical trials are ongoing for leukemia, lymphoma, myeloma, myelodysplastic syndromes and other cancers • Whether you are newly diagnosed or further along in the journey of cancer, there might be a new drug or combination of drugs being tested to improve outcomes or quality of life • The Leukemia & Lymphoma Society’s ongoing national education and chapter education programs report on the most current disease-specific clinical trial programs • Contact your local LLS chapter or the LLS Information Resource Center to stay current at 800-955-4572, infocenter@LLS.org or www.LLS.org/clinicaltrials

44. Finding a clinical trialthat might be right for you • Talk with your doctor about clinical trials as a treatment option • Speak with an Information Specialist at LLS • Use the free LLS-supported TrialCheck®to find clinical trials near you through the LLS Web site • available at www.LLS.org/clinicaltrials

45. How The Leukemia & Lymphoma Society can help • Resources • Handouts in your workbook • How to narrow your search to find a clinical trial that’s right for you • Questions to Ask Your Healthcare Provider About Clinical Trials • LLS booklets • Understanding Clinical Trials for Blood Cancers • Financial Health Matters • Co-Pay Assistance Program • LLS Web-based educational programs • My Clinical Trials Journey & other Journeys at www.LLS.org/Journeys • Archived teleconferences on emerging therapies at www.LLS.org/survivorship • To order these materials, contact the IRC at 800-955-4572 or visit www.LLS.org

46. Leukemia, lymphoma and myeloma:The road to discovery is through clinical trials “The results of clinical trials have directly changed treatment regimens, the way we think about these diseases, improved the quality of life for patients, and improved survival for countless numbers of people…” David Porter, MDJune 2008 “Clinical trials can include studies of commercially available drugs, novel agents, or combinations of new and old drugs. Clinical trials sometimes also investigate ways to optimize the dose or schedule of approved drugs.  Finally, clinical trials can include stem cell transplant, non-chemotherapy treatments, management of side effects, measuring quality of life and many other aspects of treatment which are important for patients.  All patients can think about clinical trials because a clinical trial doesn't necessarily mean you're getting a completely novel drug or new treatment.”      Gail J. Roboz, MDJune 2009

47. Local clinical trial opportunities Local Clinical Trial Opportunities

48. Road to discovery: Emerging therapies Time for your questions!