\The natural history study of solid tumors is a
longitudinal observational study coordinated by the National
Cancer Institute's my pediatric and adult rare tumor network
known as my part. Anyone with a rare solid tumor is encouraged
to enroll, especially children, teens and young adults. This
study follows people with rare tumors over time to learn about
how rare tumors grow and develop with the aims of determining why
rare tumors form and finding new treatments. Study participants
are asked to provide a saliva sample and allow access to
medical records, imaging scans, and any tumor tissue that may be
available from previous biopsies for surgery. Participants are
also asked to complete questionnaires and surveys about
their lifestyle and their family's medical history. Since
the natural history study of our solid tumors began enrollment in
January 2019, over 400 people have joined the study. The my
partner network has also grown to include 19 rare tumor
advocacy organization partners, that my part network is a group
of doctors, researchers, patients, families and advocates
with a patient centered approach to finding treatments and
working together to improve the lives of people with rare
tumors. As the natural history study of rare solid tumors
approaches, it's third anniversary of opening
enrollment, we're looking back at all that we've accomplished
and looking forward to what we hope to achieve in the future.
rare tumors across the world are lacking in knowledge, resources,
and community. Over the last three years, since opening
enrollment on the natural history study, we have started
to scratch the surface on increasing the knowledge
resources, and very importantly, helping to develop community.
For those with rare solid tumors. Our hope for the future
is to continue to provide as much as we are able to, for
patients with rare tumors, we want to continue to increase
collaborations across the whole world, as progress cannot be
made alone. Together, we want to increase the collective data to
better understand the tumor biology and natural history,
which will then lead to the ultimate goal of new clinical
trials for rare solid tumors. Finally, we continue to strive
to be a source for those with rare tumors, to guide them to a
community that understands the struggles they are undergoing.
You know, I think in the last three years, we've been able to
begin to amass enough data to start to think about some of
these were solid tumors. So for instance, Chordoma is a very
rare solid tumor that we really didn't know a tremendous amount
before. And I think the first step in in trying to develop new
ways of treating people and better ways of treating people
with some of these rare tumors is just to understand very basic
information about them who gets these kinds of tumors, how do
they do over a over a span of time when they have these
tumors? What things that people try that have been successful.
In taking care of people with rare tumors, I think that's been
been pretty successful. The things that make the NIH
Clinical Center unique are the things that make this study
unique. We have the the ability to reach out to patients across
the United States and even across the world, and to be able
to collect information from those patients to be able to
have the time to talk with those patients to be able to
incorporate things like patient reported outcomes, how the
patients are really doing in the in their real life into a study
like this. I think that that makes it
unique. For each individual patient, we will analyze the
genes that are in your tumor, and possibly within your full
body to tell us if there are any targetable changes, meaning any
medicines that we can use, that can be specific for the genes
altered in your tumor. We also provide second opinions, and
treatment recommendations for patients. All of the services
provided to patients, whether participating at home or at the
NIH, are at minimal to no cost to the patient. For the research
community, all the data collected on this study will
become publicly available without our patient and advocacy
partners. We would not be as far along as we are and we are very
grateful for this partnership and look forward to expanding
them.
There are a lot of important outcomes that this study could
help enable. It's a great opportunity for patients to to
make an impact in the understanding of this disease
and to help advance research it's a great it's a great
opportunity for researchers to learn from the experience. of
every patient. And it's frankly a great it's a great example of
the research community coming together, researchers and
physicians across multiple institutions coming together to
answer a question that that none of them are in a position to
answer themselves. And so we're just thrilled to be able to
partner with the National Cancer Institute and with all of the
researchers and investigators across the country and across
the world, for that matter who are helping to make this study
possible.
The progress of the my partner network, and the natural history
study of rare solid tumors would not be possible without our
patients and their families.
So hi, my name is John Hill, I am 19 years old, I'll be 20. In
January, I have a SPS which is our soapbox Akuma. I've had it
for 10 years now I was first diagnosed when I was nine years
old. I live in Trinidad and Tobago, it's a very cute little
island, and the West Indies. And I was treated here for a while,
I sent my research papers and all these files and records from
the doctors to NIH. And then I started a trial with them. And
I've been with NIH for the past 10 years, doing different file
drugs. And it's a very lonely condition, especially to have
because a lot of people don't have ESPs, especially young
people, people my age is almost unheard of, especially in
Trinidad, yep, spice Island, being able to share that and
give somebody else I don't know how much yesterday now,
hopefully they see this video, that they could do it as well.
It's great. It's amazing, I'm happy. I decided to take part in
the study because I wanted to help you find that get more
information about this very rare and strange Duma. And it could
maybe help somebody in 10 years. Um, I just thought about all the
other kids who will be put in a situation like mine, where it
felt as though nobody knew anything. And then somebody
reached out and said, I actually know something, maybe I can help
you. If my tumor was the little sample that you got from me
could do that for somebody else to give them that hope and
change. Um, I was I jumped at it, I was happy to be able to be
a part of that.
So I'm, I'm Caitlin Lee. I'm 18 and at the moment, I'm going to
University of Louisiana at Lafayette. I'm studying visual
arts, and I'm a freshman. I was diagnosed with cancer at the age
of 13. In 2016. For years I had noticed a lymph node that had
been growing on the side of my neck. Along with that there had
been numerous times where I had gotten sick. I was referred to
an EMT and was prompted to have a biopsy of the lymph node.
After that biopsy, I received a diagnosis of the rare form of
cancer called me into B and was urged to meet with a with a
surgeon at the Children's Hospital in New Orleans.
Initially, my surgeon had suppose that I had a prognosis
of five years. Fortunately, in my case, I had an effective
treatment of a 12 hour operation to remove the tumor. Surgery
also involved the reconstruction of my trachea, which also cause
me to have a tracheostomy, and a total thyroidectomy and
parathyroidectomy. After having the surgery, I started long
recovery in the hospital. The hardest part for me was having
to not be able to go to go to school. It has now been five
years from 2016. And I'm thankful for the research that
has helped to save my life. Well, I decided to participate
in the study because I knew that I would have gained knowledge of
Me and to be I also wanted a way that would have made it easier
for me to explain the type of cancer that I have to my family
and friends. the adverse effects of immune to be or me and that
affect me can provide a more clear outlook on rare tumors for
doctors researching those tumors and for patients who are hoping
to understand their their diseases in a more substantial
way. Ever since I got involved in cancer research. I stopped
presenting the fact that I have cancer because along with the
others dealing with the same circumstances as me, I know that
I will never not have a major role in the ever changing
discoveries that make breakthroughs in cancer
research. All in all, the opportunity of having a part in
cancer research has given me hope towards a healthy future in
which I find a way to pursue my ambitions without letting cancer
overcome me.
What does our research progress look like? In the past three
years since the study opened, we have over 400 people enrolled in
the study representing over 50 Different rare tumor
histologies. We exchange information and resources with
19 rare tumor advocacy organizations who are my
partners, we processed over 1800 biospecimen Alec watts, created
16 new data entry forums, developed 12 New rare tumor
patient derived xenograft or PDX models, launched five new
interventional trials as part of the collaboration with the
National Cancer Institute's developmental therapeutics
clinic opened three new tumor specific sub protocols of the
natural history study, and established two new rare tumor
clinics at the NIH Clinical Center.
As opposed Bachelorette, for my part, and the daughter of a
metastatic cancer patient, I am passionate about using my
personal caregiving experience to help navigate patients and
their loved ones every step of the way on their rare cancer
journey.
To everyone involved, all the people have worked on it. All
the patients who have put up with our questionnaires and our
phone calls is that you're really making a big difference.
And hopefully, all the work that people have put in and all the
time and all of the the altruistic input from our
patients and the patient advocacy groups will really help
make a difference over time.
We truly appreciate all those that are currently participating
and those that will join in the future. We are so sorry for all
of your struggles with your rare tumor. And our team strives to
help in any way that we can. We thank you for your willingness
to partner with us to make a difference in the lives of those
with rare tumors including their own.
We're looking forward to the future of the natural history
study of very solid tumors and continued collaboration with the
rare tumor research community to accelerate advancements in rare
solid tumor treatments and interventions. Most importantly,
we look forward to continuing to work closely with our patients
without whom advancements and clinical research would not be
possible. Thank you to our patients and families. To learn
more about my part, visit our website@cancer.gov slash my part
to stay up to date about my part and the natural history study of
rare solid tumors. Subscribe to our monthly newsletter, the my
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