FEATURE
Cancer Moonshot
our patients. We can learn from our solid
tumor colleagues and they, in turn, can
learn from us.”
Dr. Singer said that determining the
best way to share data is a challenging problem in cancer today, particularly due to the
large datasets clinical trials are generating.
“To truly learn from these data, they need
to be complemented by patient annotation.”
“That’s why the Moonshot Initiative
is so important,” Dr. Anderson added.
“Hopefully, the Vice President is setting an
example that will inspire people to work
in partnership on a scale that we simply
haven’t seen previously.”
The Promise of Immunotherapy
The Moonshot Initiative seems to be
marked by its macro- and micro-level views
of cancer. “On the one hand, the Moonshot
Initiative is exploring big data initiatives to
define the heterogeneity of disease, but, just
as importantly, it is also exploring personalized immunotherapies – the most promising area in cancer management today,” Dr.
Anderson said. “It’s a very exciting time.”
The Moonshot Initiative will take a
deep dive into immune system–based
therapies, which have shown success in
melanoma, leukemia, and lymphoma.
According to the White House, “this approach is ripe for further exploration in a
wider range of cancers.”
“The immune system is selective, potent, and very adaptable,” Dr. Anderson
explained. “It may be able to overcome
the evolution and changes in cancer cells
that ultimately lead to resistance and
relapse of disease.”
As part of the Moonshot Initiative,
researchers are also developing vaccines that
could prevent cancer from ever developing
or that induce an immune response earlier
in the disease course – based on the success
of vaccines for cervical cancer and other
cancers caused by human papillomavirus.
According to the White House, funding
will go toward “speeding the development,
evaluation, and optimization of safe cancer
vaccines targeting unique features of
individual cancers.”
“The concept is th at these approaches
vary and have unique mechanisms of action, but the future promise lies in combining these therapies,” Dr. Anderson said. If
researchers are able to achieve a memory
immune response against a patient’s own
cancer through combination therapies,
someday it may be possible that a patient
never develops active cancer, he explained.
Targeting the Genome
Although the Moonshot Initiative is still taking shape, the National Institutes of Health
(NIH) and the White House have already
announced progress in increasing our understanding of the genetic changes that occur
within the cancer cell. The NIH is leading
the Precision Medicine Initiative (PMI), a
research effort to use genomics and big data
analytics to develop personalized treatments
for many diseases, including cancers. The
largest component of this Initiative is the PMI
Cohort Program, a long-term health study
that will examine genetics, lifestyle factors,
and health in an effort to propel precision
medicine and identify cancer cures.
In February, the program began accepting volunteers. The NIH wants to enroll
the first cohort of 79,000 participants into
the precision medicine database by the end
of 2016, with a projected total enrollment
of 1 million participants by 2019. To spur
enrollment, the NIH provided funding to
Vanderbilt University in Nashville, Tennessee, to perform a pilot study of direct
recruitment initiatives, including a participant website and phone line.8
The NIH also pledged to work with
federally funded community health centers
to encourage cohort volunteers from
underserved populations. “For most of
history, medicine has been based on trying to identify what works for the average
person,” NIH Director Dr. Collins said in a
press conference discussing the program.
“We’re all different. This one-size-fits-all
approach is far from optimal.” The goal of
the PMI Cohort Program and recruitment
initiatives is to “empower any person, anywhere in the United States, to raise their
hand and volunteer to participate.”
“The PMI focuses on implementing
what we have already learned about cancer
genomics to clinical trials,” Dr. Singer
added. “The PMI and the Blue Ribbon
Panel share complementary agendas and,
hopefully, both efforts would be moved
under a common umbrella down the line.”
Looking to the Future
The White House has set forth a bold
goal – eliminating an indiscriminant
disease that is estimated to kill 600,000
Americans this year.1 So, with what we
know about the Initiative so far, will the
moonshot lead to more hits or misses?
“What the NCI has learned in the
last decade is that cancer is diverse and
is driven by mutations that continue to
accumulate as cancer grows,” Dr. Singer
said. Some of the mutations are common
to many cancers, but others are unique. In
other words, one size does not fit all.
While strategies that target the immune system may be more universally
applicable and certain approaches target
commonalities found within multiple
forms of cancers, individual treatment
strategies will still be necessary to effectively treat each type of cancer, she added.
“We are going to have a lot of different approaches, but the more we learn, the more
we can develop those targeted therapies.”
“The solutions are not going to be
simple,” Dr. Anderson said. “We will likely
look to targeted therapies in combination
with immunotherapeutic approaches admin-
istered early in the disease course – when the
potential for the treatment’s effect is greatest.”
There are still many questions about
the future of the Moonshot Initiative –
which priorities will be identified and
adopted, for one – but those involved in
the process say the hematology/oncology
community won’t have to wait long before
more specifics are known.
A clearer picture will start to emerge
by this fall, when the Blue Ribbon Panel
will present its top priorities and recommendations to the Task Force and National Cancer Advisory Board. The Task
Force is expected to deliver its full report,
including the top research priorities, to
President Obama in December.
“This is moving fast,” Dr. Mayer said.
“I think we have a very strong panel
bringing different aspects of cancer and
cancer care to the table, but we all recognize our charge is really to look at the
whole picture.”—By Jill Sederstrom ●
REFERENCES
1. The White House. Fact Sheet: Investing in the National Cancer
Moonshot. Accessed May 4, 2016 from https://www.whitehouse.
gov/the-press-office/2016/02/01/fact-sheet-investing-nationalcancer-moonshot.
2. The White House. Memorandum: White House Cancer Moonshot Task
Force. Accessed May 4, 2016 from https://www.whitehouse.gov/
the-press-office/2016/01/28/memorandum-white-house-cancermoonshot-task-force.
3. National Cancer Institute. Blue Ribbon Panel announced to help guide
Vice President Biden’s National Cancer Moonshot Initiative. Accessed
May 4, 2016 from http://www.cancer.gov/news-events/pressreleases/2016/blue-ribbon-panel-announced.
4. Friends of Cancer Research. “Pink Sheet - FDA Oncology Center
of Excellence Coming, Moonshot or Not.” Accessed May 5, 2016
from http://www.focr.org/news/pink-sheet-fda-oncology-centerexcellence-coming-moonshot-or-not.
5. Carter AJR, Nguyen CN. A comparison of cancer burden and research
spending reveals discrepancies in the distribution of research funding.
BMC Public Health. 2012;12:526.
6. “Sustaining the New ‘Golden Age’ of Medical Research.” Panel discussion at the Milken Institute Global Conference, May 2, 2016; Los
Angeles, California.
7. “Highlights 2016: Vision for the Future.” Plenary session at the
American Association of Cancer Research Annual Meeting. April 20,
2016; New Orleans, Louisiana.
8. National Institutes of Health. “Preparing to Launch the Precision Medicine Initiative Cohort Program.” Accessed May 6, 2016 from https://
www.nih.gov/about-nih/who-we-are/nih-director/statements/
preparing-launch-precision-medicine-initiative-cohort-program.
ASH Offers Expertise to Help With
Initiative
Pictured L to R: Suzanne Leous, ASH staff; Kenneth Anderson, MD, president-elect of ASH; Greg Simon, executive director of
the Cancer Moonshot Initiative; Martin Tallman, MD, and Tracy Roades, ASH staff.
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ASH Clinical News
Leaders of the Cancer Moonshot Initiative are asking for
public comments, and ASH has made sure hematology’s
priorities have been heard. In April, ASH representatives
met with Executive Director Greg Simon at the White House
to share ASH’s scientific recommendations for the Initiative.
“Hematologists are pioneers in several fields that are
revolutionizing cancer research, including immunotherapy,
cell therapy, and genome sequencing – areas of study that
are now showing great promise in treating solid tumors
in addition to blood cancers,” ASH President Charles S.
Abrams, MD, said in a statement supporting the Initiative.
Commenting on the meeting, Kenneth Anderson, MD,
said that ASH was well received. “Mr. Simon could not have
been nicer and more receptive and spent several hours with
us and appreciated the value that ASH can bring to the Moonshot effort.”
Prior to the meeting, an ASH task force on precision
medicine was charged with creating a series of recommended
action items for the Moonshot Initiative.
For a full list of ASH’s recommendations, visit
www.hematology.org/Research/Recommendations/
Moonshot-Initiative.
June 2016