Our Mission:
Cure leukemia, lymphoma, Hodgkin’s disease and myeloma,
and improve the quality of life of patients and their families.
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• Since 1949, LLS has awarded more than
$600 million to fund research
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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 trial
is right for you
– How to locate clinical trials that may be right for you
– The Leukemia & Lymphoma Society can help
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
The promise of clinical trials
• Progress through research
has helped people with
blood cancers:
– Live longer
– Have greatly improved
quality of life
Drug discovery:
The journey from lab to patient
Discovery of
new drug in lab
Drug tested in
lab and animals
• 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
Phase 1 clinical trials
– What method of drug delivery
and dosage is safest?
– How does the drug affect
the human body?
Phase 2 clinical trials
– Does the drug have an
effect on the cancer?
Drug discovery:
The journey from lab to patient
Phase 3 clinical trials
– Is the new drug or combination
of 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?
Drug discovery:
The journey from lab to patient
FDA approves
new drug for sale
and marketing in
the United States
New drug available by
prescription for all
patients who need it
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
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: gemtuzumab ozogamicin
(Mylotarg®)
• Acute Lymphocytic Leukemia: clofarabine (Clolar®)
• Chronic Lymphocytic Leukemia: alemtuzumab (Campath®)
and bendamustine HCI (Treanda®)
• Chronic Myelogenous Leukemia: imatinib mesylate
(Gleevec®), dasatinib (Sprycel®) and nilotinib (Tasigna®)
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 ibritumomab tiuxetan (Zevalin®),
bortezomib (Velcade®), bendamustine HCI (Treanda®) and
denileukin diftitox (Ontak®)
• Myeloma: thalidomide (Thalomid®), lenalidomide (Revlimid®)
and Velcade®
• MDS: Revlimid®, azacitidine (Vidaza®) and decitabine
(Dacogen®)
• Stem Cell Transplantation: plerixafor (Mozobil®)
Finding better therapies for blood cancers
Door is opened to discovery of other promising
new targeted therapies now being tested
Participation is crucial
The more people who take part in
clinical trials, the faster we will find
better ways to treat blood cancers
Participation in clinical trials:
What gets in the way?
• People with cancer may…
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–
–
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
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 to
the 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.”
Learning the facts about clinical trials
True or false?
If I enter a clinical trial, there’s a good chance
that I could receive a placebo
FALSE
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)
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
Phase 3 clinical trials:
Getting assigned to a group
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
True or false?
Clinical trials are the treatment of last resort
FALSE
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
True or false?
If I enter a clinical trial,
I’ll be treated like a “guinea pig”
FALSE
Safety
• Fact: Each clinical trial is managed according
to a strict set of rules to protect patients
– Protocols regulate:
• Quality of care
• Safety of participants
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?
How is a patient’s safety protected?
• Researchers must tell you the purpose, benefits,
and risks of a clinical trial before you sign up
–
–
–
–
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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
Take your time in reading and signing
the informed consent form
True or false?
Care and treatment received are free
Both
TRUE and FALSE
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
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):
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–
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Hospitalizations
Doctor visits
Laboratory tests
Drugs that are not part of the study design
What’s covered, what’s not
• A growing number of U.S. states have
passed legislation or instituted special
agreements requiring health plans to pay
the cost of routine medical care in clinical
trials. 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
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
True or false?
I’m too old to enter a clinical trial
Increasingly, FALSE
Age
• Fact: A growing number of clinical trials
are seeking to include older adults
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
My Clinical Trials Journey
Clinical trials: Weighing the pros and cons
• Possible risks
– Compared with standard treatment, the new treatment
being 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
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
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.
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
Finding a clinical trial
that 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
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
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, MD
June 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, MD
June 2009
Local clinical trial opportunities
Local Clinical Trial Opportunities
Road to discovery: Emerging therapies
Time for your questions!