MESOTHELIOMA

CLINICAL TRIALS
INFORMATION FOR
PATIENTS AND FAMILIES
IDENTIFYING
AND EVALUATING
EXPERIMENTAL
TREATMENTS
mesothelioma and clinical trials
table of contents
Mesothelioma is a rare cancer diagnosed each year in approximately
3,500 people in the United States. To date, there is only one FDA-
approved treatment for patients with mesothelioma.
In 2004, the combination of pemetrexed (best known as Alimta) and
cisplatin was approved for the treatment of mesothelioma following
the completion of a randomized controlled phase III clinical trial. The
trial, which enrolled 456 participants, reported a 40% response rate,
and showed increased survival as compared to cisplatin alone. This
breakthrough advance was met with wide-spread excitement, because
prior to the introduction of this combination of agents, mesothelioma
was thought to be resistant to chemotherapy. The Alimta trial
demonstrated that patients with mesothelioma can and do respond to
treatment.
We now refer to this regimen as frst-line therapy and, in the last
decade, thousands of mesothelioma patients have benefted from this
development.
The participation of patients who volunteered for the various phases
of the Alimta clinical trial was essential in bringing this drug to market
and making it available to future generations. Although, currently there
is no FDA-approved treatment option following this frst-line therapy,
clinical trials offer patients the opportunity to try new therapies,
while helping develop potential breakthroughs in the treatment of
mesothelioma.
NOTE: A clinical trial can be entered during any of its three phases,
provided the patient meets eligibility criteria.
PHASES OF A CLINICAL TRIAL
Phase 1
To determine the maximum dose tolerated with acceptable side
effects
To test a new intervention (e.g.; surgical trial)
To determine the effect of this drug on cancer and the human body
Observing patient response to the drug
Phase 2
Builds upon what is learned during the Phase 1 trial
Focus is on response to this therapy with a particular cancer
Side effects continue to be monitored
Phase 3
To determine if the new treatment or intervention is better than the
standard of care
If successful this may result in an FDA approval
Pg. 2................ Phases of a Clinical Trial
Pg. 3................ Mesothelioma and Clinical Trials
Pg. 4 - 5........... Is a Clinical Trial the Right Choice for Me?
Pg. 6................ Weighing the Risks and Benefts
Pg. 7................ You Decided to Enroll in a Clinical Trial: Now What?
Pg. 8................ About the Mesothelioma Applied Research Foundation
2 3
While there is no question that without mesothelioma clinical trials
the development of new procedures and medicines would come to a
virtual standstill, this may not be of much concern to a newly diagnosed
mesothelioma patient facing a prognosis of a short median survival time.
It is true that clinical trials are somewhat altruistic, because they
beneft future patients, but this shouldnt be the primary motivation for
participating in trials. With only one, non-curative FDA-approved treatment
for mesothelioma in the medical kit bag, mesothelioma clinical trials
are often the most effective and, sometimes, the only way a patient can
access innovative treatment options.
When deciding whether a clinical trial is the right choice for you, it is wise
to ask questions and to fully understand the risks and expected benefts
before signing the informed consent form.
Questions to ask include:
What is the purpose of the trial?
What tests and treatments will be used?
What is the track record, if any, of the experimental treatment?
What are the benefts, risks and side-effects in contrast to other
treatment choices?
What steps will be taken to monitor safety and who will be
responsible for actions in the event of a problem?
How long will the trial take and how long will I need to
participate?
Can I opt out of the trial and under what conditions?
Who will conduct the trial and what are their credentials?
What portion of the trial is subsidized and by whom?
What drugs will be used during the clinical trial? Are these
covered by the study?
What portion of my treatment will my insurance cover and how
much will I be responsible for?
IS A CLINICAL TRIAL THE RIGHT CHOICE FOR ME?
If you are a surgical patient, some additional questions to ask might
include:
How do your stage and histology (epithelial, biphasic or
sarcomatoid) impact upon the expected results?
Is there an opportunity to try an intervention or drug under
investigation at the time of surgery that might have a positive
impact on my survival?
Is there an experimental intervention that might lessen some
of the expected side effects?
If you are not a candidate for surgery you will want to learn about:
Pemetrexed and cisplatin and what you can expect to gain
from this combination of drugs alone
Consider a trial that delivers a new drug in addition to, or
following standard treatment with pemetrexed and cisplatin.
In either instance, due to varied guidelines, clinical trial eligibility
largely depends on the patients previously administered treatments.
To maximize the availability of treatment options, it is crucial for
you to know about all treatments available prior to beginning any
treatment regimen, experimental or not.
4 5
mesothelioma clinical
trials are often the most
effective and, sometimes,
the only way a patient
can access innovative
treatment options
The primary risk of a clinical trial is that it may not work and the
patient may continue to get worse. Since one of the primary objectives
of a clinical trial is to establish the safety of the treatment, some
participants may experience side-effects that could range from being
uncomfortable to being life-threatening.
When researching and preparing for a clinical trial, some points you
may want to consider include:
How is the clinical trial treatment different than the standard
treatment?
Are there any side-effects that are specifcally associated to the
treatment?
Despite the experimental nature of clinical trials, the institutions that
offer them are committed to minimizing the above risks as much as
possible. The goal, after all, is to fnd a treatment that will help the
patients, not harm them. This process is aided by the requirement that
clinical trials be approved by two boards, one being the Institutional
Review Board (IRB), prior to opening for enrollment.
Clinical trials must adhere to the same legal and ethical standards that
apply to general medical practices and most American clinical trials
are registered with, and regulated by, the United States government to
ensure consistency and enforcement of standards.
WEIGHING THE RISKS vs. BENEFITS
YOU DECIDED TO ENROLL IN A CLINICAL TRIAL:
NOW WHAT?
Following your decision to enroll in a clinical trial, there are a few more
considerations to keep in mind:
Keep the lines of communication open between you and the
doctor who has been overseeing your care to this point. Keep them
informed of your progress, side-effects, and results.
The great news is that you will receive regular and careful medical
attention from the research team working on your clinical trial.
That will include doctors, nurses, and other health professionals.
You will be followed very closely by the research team.
You also have the support of the Mesothelioma Applied Research
Foundation. Our nurse practitioner can assist you in identifying
clinical trials that might be appropriate based upon your personal
health history, as well as guide you though all stages of your
disease. Your well-being and support of you and your loved ones
remains our top priority.
Always remember that you are not only getting the latest
in available treatments, but are also furthering the feld of
mesothelioma research for generations to come.
6 7
Clinical trials must
adhere to the same legal
and ethical standards
that apply to general
medical practices
ABOUT THE
MESOTHELIOMA APPLIED
RESEARCH FOUNDATION
The Mesothelioma Applied Research Foundation is the only
501(c)3 non-proft organization dedicated to eradicating
mesothelioma and easing the suffering it causes by:

funding the highest quality and most promising research
projects from around the world through our rigorous peer-
reviewed process;

helping patients connect with national mesothelioma experts
and obtain the most up-to-date information on treatment
options;

advocating in Washington D.C. for federal mesothelioma
research funding to stop this national tragedy.
Mesothelioma Applied Research Foundation
1317 King Street
Alexandria, VA 22314
TOLL-FREE NUMBER
(877) 363-6376
(877) EndMeso
[email protected]