Treatments |
Improving
Drug Development and Assessment | The Essential Role of the NCI
 Survival
statistics tell us that the treatments we have are not good enough.
We continue to lose those we love. We continue to suffer
through painful and toxic therapies. And our society suffers
as patients with lymphoma become less productive and less able to
earn a living and make contributions to their communities.
Concerning current treatments for lymphoma, we
believe that the gap between what is and what's possible has never
been wider. So we dedicate this page to listing the abundant
opportunities for success, and to remind all of the urgent need to
seize them.
Treatments:
There is an urgent need to develop and clinically test new
treatments that:
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have greater specificity, more
potency, and less toxicity; |
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can overcome bulky disease; |
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can selectively kill indolent lymphoma cells,
that are not in cell cycle; |
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can induce
and enhance active immunity - the
way we naturally fight disease; |
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combine
targeted therapies that may complement each other in order to
minimize toxicity and prevent tumor escape; |
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are rationally selected
to match the differences in the disease by using genetic
and metabolic profiling;
(please review information about the National Biospecimen
Network an innovative blueprint for progress) |
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are safer,
thus allowing patients with
indolent lymphomas to safely receive
treatment early and more regularly; |
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that can
be safely combined with existing treatments in order to cure indolent
lymphomas, and more patients with aggressive disease. |
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that
address the role of the microenvironment:
(1) Altering the host environment in ways that silence or
inhibit pro-survival signals concurrent with standard therapies.
(2) Focusing, and expanding immune surveillance against tumors.
(3) Turning
off signals and inhibiting cells that contribute to drug resistance,
depressed immunity and escape from immunity. (4) Targeting tumors
in ways that minimize damage to immunity, or enhance it.
(5) Sequencing therapies in ways that restore positive elements of the
microenvironment in order to consolidate the response to treatments.
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Improving Drug
Development and Assessment:
| Ensure that there are sufficient incentives for pharmaceutical
companies to take the financial risks to
develop and test therapies for cancer.
 | Consider reducing incentives to
develop "me-too" drugs for conditions that
are not life-threatening.
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| Ensure that the FDA considers factors
such as lower toxicity and improvements in the quality of life benefits in the
assessment of new therapies.
| Encourage drug sponsors to
properly account for and measure toxicities and quality of
life in
clinical trials.
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| Increase patient participation in clinical
trials - an essential ingredient:
| Encourage drug sponsor to
consult with informed patients and treating
physicians
to help design clinical studies that are in harmony with patients survival goals.
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| Educate the communities' doctors and patients
about what studies are
available, and which show promise for specific treatment
settings.
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| Encourage treating physicians to
discuss all appropriate therapies, including investigational therapies,
during treatment consults.
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| Ensure that tests associated with clinical trials
are covered by government and / or insurance.
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| Provide, when possible,
predictive tests to determine who is more likely to benefit
from an
investigational therapy and/or a standard therapy used as a
pretreatment in studies.
 | For example, include tests for polymorphisms in studies that utilize
Rituxan.
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| Encourage drug sponsors to test
investigational bio- and immune-based therapies earlier in the course
of the disease, when they are more likely to
provide benefit -
especially for indolent lymphomas.
| Ensure that watch & wait should not be
routine. Periods of stable
disease provide an important
opportunity to test immune-based therapies and other more targeted therapies with
safer toxicity profiles.
We must encourage drug sponsors, patients, and treating physicians
to use this opportunity:
| Better immune competence |
| Less tumor burden |
| Less exposure to toxic treatments |
| Potential to improve quality of life |
| Potential to learn without precluding the use of standard treatments |
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The Essential Role of the National
Cancer Institute (NCI):
| Ensure that funding of cancer research becomes a
national priority.
| Encourage all cancer groups to work
cooperatively in this effort.
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| Encourage the NCI to carry out its promise to
conduct translational research in order to validate
and clinically test promising new therapeutics.
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| Ensure that gene-profiling technologies are fully
funded and applied as quickly as possible.
We support
the following rationale:
| Enables scientist to more rapidly identify the
gene expressions that distinguish normal from
malignant cells and
assist in the "development of molecularly targeted therapies
that have specificity
and potency for defined cancer types."
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| Enables scientists to link clinical behavior (how
aggressive or indolent the cancer is likely to be)
to gene expression, and thus better advise patients about treatment
selection and timing.
(Currently, the variable clinical course of
patients given the same diagnosis stems, in part, from
the
underlying molecular diversity among their tumors.)
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| Enables identification of viral factors that may be present and may contribute to causing,
promoting, or maintaining malignant behavior of lymphoma cells.
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| Enables scientists to discover how genes function
in immune cells and apply that knowledge to
the treatment of numerous diseases.
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| Enables retrospective testing to genetic
expression with individual responses to treatments.
The potential benefits
will enable researchers and/or doctors to:
| select treatments that are far more likely to
benefit the patients |
| spare patients from the side effects of
ineffective treatments |
| identify patients in need of experimental
approaches to treatment |
| reduce health costs by enabling rational
treatment selection, instead of by wasteful trial and error |
| identify genes in tumors that lead to
treatment resistance |
| select patients most likely to benefit from
investigational drugs in clinical
trials
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“Presently we are overlooking effective treatments for some people, while
administering ineffective
and toxic treatments to others based on
studies conducted with ill-defined aggregates of patients.”
– DF.
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| Enable testing of multiple samples from the same patient,
in order to:
| identify changes to gene
expression and other tumor characteristics in response to
treatments and over time. |
| identify changes to gene
expression and other tumor characteristics that correlate to refractory
disease, or transformation to aggressive disease, or the lack thereof.
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| Enable identification of tumor-associated antigens that can form the basis for therapeutic
cancer
vaccines.
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| Enable discovery of cells in the lymphoid microenvironment
that may be promoting or inhibiting
lymphomas.
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| Enable production of patient-specific therapeutics, such as cancer
vaccines or therapeutic antibodies.
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| Consider providing financial incentives to managers that bring new
therapeutics to cancer patients,
and to scientist who make key discoveries to make this possible. |
| Encourage clinical studies of the
many promising therapeutics and adjuvants that are not of
commercial interest, such as:
| Combinations and sequences of agents that may
complement one another.
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| Immune-based strategies that have low
toxicity on patients with indolent lymphomas.
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| Natural compounds that have
potential in combination with chemotherapies, such as fish oil
and melatonin.
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| Last, but NOT least: Design compassionate use protocols in advance
- anticipating the
needs of patients who fail standard therapies, and are ineligible for
clinical trials - so we can give patients every reasonable chance
to survive, and also test new concepts
in a timely manner.
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