In this issue...
Advancing Advocacy in the Genetic
Disorder Community
RAN Co-Founder receives Art of Advocacy Award
The
Genetic
Alliance – an international coalition
comprised of more than 600 advocacy, research, and healthcare organizations
representing millions of individuals with genetic conditions – recently
convened its annual conference in
North
Bethesda,
Maryland. Held
from July 29th – 31st, the 2005 “Leadership in
Alliance” conference brought together
advocates, researchers, healthcare providers, and patients for three dynamic
days to focus on capacity-building within this community serving the patients
and families living with genetic disorders and rare diseases.
Many genetic conditions
have very small patient populations, and as a result, the resources required to
advance research are frequently limited or nonexistent. To combat this
obstacle, the Genetic Alliance has created an effective model for equipping
advocates with the skills they need to drive the research agenda for these
diseases: leveraging the communities’ varied expertise and voices to effect
change for patients.
Workshop sessions, presentations, and speakers at the
conference all echoed this theme of bringing the community together to learn
from each other, share best practices, and support one another in advocacy
efforts that can at times feel overwhelming given the limited amount of
resources or public attention currently focused on genetic disorders and rare
diseases. Specifically, sessions were offered on working with the FDA
throughout the drug development process; effective lobbying and understanding
public policy; driving research from the laboratory to patients; creating and
leading successful advocacy organizations; and contributing effectively to
research and advisory committees. These sessions all focused on providing
concrete information and tools to the advocates at the conference, in a
supportive atmosphere that fostered active discussion, idea-sharing, and
learning among the participants. For more information about the Genetic
Alliance or to download presentations from the conference go to: http://www.geneticalliance.org.
The Genetic Alliance’s
focus on capacity-building and resource-sharing among its network of members
has given these organizations an opportunity to drive the research agenda for
their specific diseases and conditions, achieving success through collaboration
that could seem unreachable if these groups worked individually.
This model also embraces collaboration and learning through
the recognition of significant contributions made by individual advocates. At
this year’s conference, the Genetic Alliance celebrated the achievements of
Elda Railey by honoring her with the “Art of Advocacy” award. Previous
recipients of the prestigious “Art of Advocacy” award have included Nancy
Wexler and Patricia Barr, underscoring the Genetic Alliance’s dedication to
acknowledging the advocate community’s current momentum and accomplishments,
while paving the way for future advances.
Issues in the
Ongoing Biospecimens Debate
NCI’s Biospecimen Coordinating Committee convened two
workshops this summer to gather input on key issues surrounding efforts to
systematize and regulate the collection of patients’ tissue.
The first workshop, entitled “Biospecimen Access and Ethical,
Legal and Policy Issues” was held in North
Bethesda, Maryland
on June 23rd and 24th, 2005. This workshop was chaired by Dr. Arthur Caplan
of the University
of Pennsylvania, and
its focus was on providing an overview of the ethical, legal and policy
issues associated with the collection and use of human biospecimen samples.
By
providing solid background information on these considerations, this workshop
primed participants for the follow-up workshop in July, when they could then
make specific recommendations on the management of patients’ tissue. Mary Lou
Smith and Judy Perotti, co-founders of Research Advocacy Network participated
in this workshop.
Held on July 18th – 20th, 2005 in Washington, D.C.,
the follow-up workshop on “Best Practices and Recommendations for Establishing
and Maintaining Biorepositories that Support Cancer Research” was chaired by
Dr. Mark A. Rubin of Dana-Farber Cancer Institute. During this workshop,
participants including Mary Lou Smith of Research Advocacy Network had the
opportunity to discuss specific infrastructural, operational and procedural
concerns surrounding biospecimens.
At both workshops, several key themes emerged that need the
attention and input of advocates, to ensure that the patient perspective is
clearly represented as NCI, researchers, and industry continue to grapple
with the most ethical – yet efficient – ways to manage biospecimens in order
to streamline research and hasten results.
Ownership
The question of who owns the tissue once it has been donated –
as well as who owns any commercial products that may result from research
conducted with that tissue sample – continues to create debate. This also
raises questions about whether patients have the right to ask for their
tissue back or request that some be saved for them in case they need it in
the future, and if so, how it has been tracked throughout the research
process and whether it can be located. This can be problematic if the tissue
has been transferred several times from patient to research institution to
pathologist, or if it has been destroyed after the sample was used for
research. And even if a sample is still intact to return to a patient, it is
not useful to them or their physician if there isn’t any accompanying data on
the research outcomes from their sample.
Confidentiality
Confidentiality continues to be a major concern of patients,
who worry about insurers or the government storing private genetic
information when they only intended to donate their tissue to support
research efforts. Some advocates recommend that genetic information related
to a tissue sample be handled differently than any other information derived
from that sample. This solution, however, is somewhat complex since more and
more often there is already genetic information attached to a given sample
when it is donated. However, if there is a process in place to ensure that
samples are consistently de-identified at the time of donation, patient
confidentiality could still be protected.
Findings
Patients considering the possibility of donating their tissue
often ask how it will be used and whether they will be informed of the
research’s outcomes. These questions support the concept of developing
well-documented, transparent tracking systems for all biorepositories to use.
Although it may not be appropriate to provide patients with a detailed
itinerary of how their tissue was utilized, the research community has an
opportunity to build trust with patients and advocates by providing
information on the overall research findings, such as whether new tumor
markers were discovered.
Willingness to Donate
Frequently, researchers believe that patients won’t consider
donating their tissue for research because they may not understand the
concept, or they are not willing to undergo the overwhelming process of
deciphering and signing consent forms. Advocates, however, don’t believe this
is true. Their experiences with patients indicate that if the information is
provided in an easy-to-understand manner and if their questions are answered
thoroughly, that they are much more likely to consider donating tissue.
These issues and concerns underscore the need to establish
better systems for tracking tissue, protecting confidentiality, and
communicating with patients, in order to harness the potential of biospecimens
research and to translate those learnings into improved patient care and
outcomes.
At the Forefront of Cancer’s Future
The Emerging Field
of Nanotechnology
The National Cancer Institute (NCI) is embarking upon a new
area of research, in an effort to fight cancer at its very core by exploring
the emerging field of nanotechnology.
But what is nanotechnology? According to the National
Nanotechnology Initiative – a federally-funded R&D program to coordinate
the multi-disciplinary and multi-agency efforts in this field – nanotechnology
can be defined as the understanding and control of matter at dimensions of
roughly 1 to 100 nanometers, where unique phenomena enable novel
applications.
To put this in perspective, a single nanometer is just
one-billionth of a meter, and a sheet of paper is about 100,000 nanometers
thick. Nanotechnology encompasses nanoscale science, engineering, and
technology, and involves the imaging, measuring, modeling, and manipulating
of matter at this infinitesimal scale.
At this level, the physical, chemical, and biological
properties of materials differ in fundamental and valuable ways from the
properties of individual atoms, molecules, or bulk matter. Nanotechnology
R&D is directed toward understanding and creating improved materials,
devices, and systems that exploit these new properties.
One way in which cancer research is already reaping the
benefits of nanotechnology is through the development of Abraxane, the only
taxane without solvents, which has been approved for the treatment of breast
cancer after failure of combination chemotherapy for metastatic disease or
relapse within six months of adjuvant chemotherapy. Scientists developed a
method of making nanoparticles (think of a
tiny hollow ball) from the protein albumin and loading paclitaxel into these
nanoparticles. This allows patients to safely receive 50 percent more
paclitaxel per dose. With the advent of
this new area of research, it is our hope that Abraxane will be just the
first of many nanotechnology-based advances for those affected by cancer.
For its part in driving this research forward, NCI has created
the Alliance
for Nanotechnology in Cancer, an initiative that will use this new technology
to change the ways in which cancer is diagnosed, treated, and even prevented.
NCI’s research will focus on the development of extremely sensitive devices
constructed of nanoscale components – such as nanocantilevers, nanowires, and
nanochannels – that have the potential to detect rare molecular signals
associated with malignancy, find gene mutations and genome instability in
their earliest stages, or even monitor patients “real-time” to track their
exposure to environmental triggers and life-style risk factors for cancer.
Specifically, the Alliance
has secured $144 million in federal funding to explore the role of
nanotechnology in several aspects of cancer research, including:
- Researching tools to identify new biological targets
- Developing agents to monitor predictive molecular
changes and prevent pre-cancerous cells from becoming malignant
- Creating imaging agents and diagnostics to detect
cancer in the earliest, most easily treatable, pre-symptomatic stage
- Developing multi-functional targeted devices to deliver
multiple therapeutic agents directly to cancer cells
- Coming up with systems to provide real-time assessments
of therapeutic and surgical efficacy
- Discovering novel
methods to manage symptoms that affect quality of life
Currently, the Alliance is
reviewing grant proposals from researchers across the United States and will
begin to
award grants that it believes will further our understanding of
nanotechnology and its ability to impact cancer care. Those grants will be
awarded on a five- or six-year basis, with the possibility of ensuing
clinical trials based on findings from the initial research.
It is the Alliance’s
hope that this focus on nanotechnology will help achieve NCI’s overarching
goal of eliminating death and suffering due to cancer by 2015. According to
Wayland Eppard, co-chair of the North Central Cancer Treatment Group and
consumer advocate to the Alliance, “As nanotechnology becomes even more
sophisticated and as scientists understand it better, it will be truly
powerful because it will be able to diagnose cancer at the cellular level and
kill just that malignant cell – this technology has incredible potential.”
Although the Alliance is just at the very beginning of
this significant research, it is still important for advocates to follow this
science as it develops. One way to do that is by subscribing to the weekly
and monthly nanotechnology articles that are available online at the Alliance’s
News
Center at http://nano.cancer.gov.
Stay tuned for further updates from the Research Advocacy
Network on this exciting area of cancer research.
New Fact Sheets Available
Two new fact sheets have been added to the
"What it Means for Me" Fact Sheet Series. The new topics are on the
results of the Avastin and Herceptin studies in breast cancer. These are
available for download on the Research Advocacy Network Publications area
of the website.
Research Advocacy Network Website
www.researchadvocacy.org
Check out the first version of our new website at
www.researchadvocy.org. New areas have been added and more will come soon. Be
sure to join Research Advocacy Network with the "Join" link. There
is no charge for Network membership and this will assure that you receive all
notices and have access to Network programs
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