Featuring the Cancer Progress Report

MEDICINES IN
OPMENT |
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DEVELOPMENT
15
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P O RT
CA N C E R
FOR CANCER
ED

A REPORT ON CANCER
M

More Than 800 Medicines and Vaccines in Clinical

JUST THE FACTS
Testing for Cancer Offer New Hope to Patients
In recent decades, tremendous for more targeted treatments.
– almost previously unthinkable – Researchers are exploring new high-
Cancer death rates
progress has been made in the fight tech methods to fight the disease
have declined

22%
against cancer. Advances in molecular as well as new ways to maximize
and genomic research have revealed the use of existing medicines,
underlying complexities and pro- either alone or in combination with
since the early 1990s 1 vided insights into cancer, which we other therapies, to treat various
now know is actually more than 200 forms of the disease. In fact,
unique diseases. Continued research biopharmaceutical companies are

83%
has expanded our knowledge of how developing 836 medicines and
the disease develops and how to vaccines5 for cancer, all of which
target medicines for specific cancer are in clinical trials or awaiting
of survival gains in types – resulting in more effective review by the U.S. Food and Drug
cancer are attributable therapies for patients. Administration (FDA). The medicines
to new treatments, in development include:
Evidence of progress can be found in
including medicines 2
the number of cancer survivors living 123 for lung cancer, the leading
in the United States – a number that cause of cancer death in the United
has increased from 3 million in 1971 to States, with more than 158,000
Approximately
14.5 million in 2014.1 Currently, about deaths expected in 2015.1

80%
of pipeline cancer
4 percent of Americans are cancer
survivors.1 Although great progress
has and continues to be made in the
106 for several types of leukemia,
which account for more than 3 percent
of all new cases of cancer.1
drugs are potentially fight against cancer, this complicated
first-in-class medicines 3

 disease remains what author 92 for lymphoma, including non-
Hodgkin lymphoma which accounts
Siddhartha Mukherjee termed “the
for more than 4 percent of all new
emperor of all maladies” – it is the
cancer diagnoses.1

73%
second leading cause of death in the
United States, accounting for nearly 82 for breast cancer, the leading
1 of every 4 deaths. It is estimated cancer diagnosed in women in the
that in 2015, more than 1.6 million new United States with 231,840 new cases
of cancer medicines
in the pipeline have cancer cases will be diagnosed, and expected in 2015.1
the potential to be nearly 600,000 Americans will die 58 for brain tumors, including
personalized medicines 4 from cancer, a rate of approximately gliomas, which represent 80 percent
1,600 people per day.1 of all malignant brain tumors.6

America’s biopharmaceutical 53 for skin cancer, including

companies are responding to the melanoma, which accounts for 2
needs of cancer patients, working percent of all skin cancer cases, but
to develop innovative approaches most of the skin cancer deaths.1

MEDICINES IN DEVELOPMENT | 2015 REPORT

(cont from page 1)
Chronicling Progress:
Other medicines in development
target colorectal, ovarian, prostate and AACR Cancer Progress Report
childhood cancers as well as solid Cancer research is advancing The fifth edition of the report, to
tumors, among others. rapidly and new medicines be released Sept. 16, 2015, not
The progress made in the fight against in development take many only chronicles advances made
cancer has resulted in better outcomes innovative approaches to against cancer between Aug.
for patients, helping them to lead longer, fighting the disease. 1, 2014, and July 31, 2015, but
healthier lives. The more than 800 also contains a special feature
The American Association
medicines being developed represent our that documents the remarkable
for Cancer Research (AACR)
best hope for continuing that progress pace of progress against the
– the oldest and largest
and lessening the burden of cancer for collection of diseases we call
professional organization
patients around the world. cancer in the five years of
in the world dedicated to
Some medicines are listed in more than publishing the report. The
advancing research to prevent
one category. For a complete list of the report delineates how advances
and cure cancer – produces
836 medicines in development, please in precision medicine are
an annual “Cancer Progress
visit http://phrma.org/sites/default/files/ transforming lives, like those of
Report” that encapsulates
pdf/2015_cancer_drug_list.pdf the 13 courageous individuals
this progress and outlines the
who shared their experiences
resources needed to continue
to make further progress.

Although extraordinary advances

Medicines and Vaccines are being made against cancer,

the disease remains a major
in Development to health care challenge and a

Treat Cancer huge financial burden, both

nationally and internationally.
Many of the medicines in the pipeline Moreover, because most cancer
today are using novel approaches diagnoses occur in those who
to attack cancer at the molecular are age 65 and older – a segment
level. Among the 836 medicines and of the U.S. population that is
vaccines in development are potential expected to double by 2060 – it
treatments for: is predicted that the number of
cancer diagnoses will increase with cancer in the publication.
COLORECTAL CANCER These personal stories remind
dramatically in the future. The
A humanized monoclonal antibody in rising economic and personal us that each advance, no matter
development for metastatic colorectal burden of cancer highlights the how small, can be meaningful.
cancer targets the cell surface protein crucial need for more research
Learn more at
“endosialin,” which is expressed on cells to develop new prevention and
www.cancerprogressreport.org
that are part of the tumor blood vessel treatment approaches.
structure. In preclinical studies, blocking
the function of endosialin inhibited
tumor growth and metastases. factor-beta (TGF-beta) signaling. The overexpression of TGF-beta in
cells may enhance tumor growth and intensify metastases.
LIVER CANCER
SOFT TISSUE SARCOMA
A medicine in development for
hepatocellular carcinoma (the most In malignant solid tumors, oxygen levels are often low (called tumor
common form of liver cancer) is a small hypoxia) relative to oxygen levels in healthy tissue. Tumor hypoxia
molecule kinase inhibitor designed to is associated with tumor progression, metastases, and resistance to
selectively block transforming growth chemotherapy and radiation treatment. A medicine in development is
 “RESEARCH IS THE
FOUNDATION OF
NEW AND BETTER
APPROACHES TO
CANCER PREVENTION,
DETECTION,
DIAGNOSIS, AND
TREATMENT, WHICH
ARE DRIVING DOWN
OVERALL U.S. CANCER
DEATH RATES AND
INCREASING THE
NUMBER OF PEOPLE
WHO ARE LIVING
LONGER, HIGHER-
QUALITY LIVES AFTER
A CANCER DIAGNOSIS.”

— American
Association for
Cancer Research

activated when it reaches the hypoxic region of the tumor environment where
it eventually kills tumor cells in its vicinity, essentially attacking the tumor from
the inside. If approved, it would be the first new medicine for soft tissue sarcoma
patients in over 20 years.

STOMACH CANCER

A monoclonal antibody in testing for stomach cancer with high MET (mesenchymal-
epithelial transition factor) expression is designed to inhibit hepatocyte growth factor
(HGF)/scatter factor from binding to the c-MET receptor. The HGF/c-Met signaling
pathway is thought to play a role in tumor growth and metastases in many cancers,
including stomach cancer. An exploratory biomarker analysis found that the addition of
the medicine to chemotherapy improved median overall survival in patients with tumors
that expressed high levels of MET protein.

MEDICINES IN DEVELOPMENT | 2015 REPORT

The 2015 PhRMA Research & Hope Awards
The PhRMA Research & Hope Awards estimated 10,380 new cases of cancer are
recognize advances made across a critical expected to occur among children less than
disease or health condition by individuals 14 years of age, representing less than 1
or organizations in the biopharmaceutical percent of all new cancer diagnoses. About
sector, academia, government, and 1,250 children are expected to die from
provider and patient organizations. The cancer this year. Survival rates for childhood
awards illustrate how biopharmaceutical cancers have increased 43 percent since
researchers and others in the innovation the mid-1970s. The most common types
ecosystem work together to not only bring of childhood cancer are leukemia (30
new medical advances to patients, but percent of all childhood cancers), and
thwart deadly diseases through increased brain and central nervous system tumors
awareness, public health efforts, and (26 percent). Other pediatric cancers
increased collaboration. include: neuroblastoma, Wilms tumor, non-
Hodgkin lymphoma, Hodgkin lymphoma,
This year’s program celebrates the rhabdomyosarcoma, osteosarcoma,
progress and promise of oncology retinoblastoma and Ewing sarcoma.
research and care. Awards will be
presented in the following categories: SPOTLIGHT: MELANOMA 1
Melanoma accounts for less than 2
• Academic Research: J. Silvio Gutkind, percent of all skin cancer cases, but the
Ph.D., National Institutes of Health vast majority of skin cancer deaths. An
estimated 73,870 new cases of melanoma
• Biopharmaceutical Industry Research:
will be diagnosed in 2015, and 9,940
Merck & Co., Inc.
deaths from melanoma will occur this year.
• Excellence in Advocacy & Activism: The treatment of advanced melanoma
Vicki Kennedy has changed in recent years with the FDA
approval of targeted drugs and cancer
• Community Champion: Rick Dunetz,
immunotherapy. There are 53 medicines
Side-Out Foundation
being tested as potential treatments for
Footnotes:
• Visibility & Progress: Liz and Jay Scott, skin cancer.
Alex’s Lemonade Stand 1. American Cancer Society
SPOTLIGHT: GLIOMA 6
SPOTLIGHT: BREAST CANCER 1 2. E. Sun, et al., “The Determinants
There are over 120 types of brain tumors. of Recent Gains in Cancer Survival:
An estimated 234,190 new cases of breast Gliomas, a broad term which includes all An Analysis of the Surveillance,
cancer are expected to be diagnosed in Epidemiology, and End Results (SEER)
tumors arising from the supportive tissue of
Database,” Journal of Clinical Oncology,
2015, with 99 percent of those in women. the brain, represent 30 percent of all brain May 2008 Supplement (Abstract 6616)
While more than 40,000 Americans are tumors and 80 percent of all malignant
expected to die from breast cancer in 3. Analysis Group
brain tumors. There are three types of glial
2015, death rates have steadily decreased cells that can produce tumors – astrocyte 4. Tufts Center for the Study of Drug
in women since 1989. These decreases Development
cells (which produce astrocytomas and
represent improvements in both early glioblastomas), oligodendrocyte cells 5. Number of medicines obtained
detection and treatment. There are 82 through public, government and
and ependymal cells. Mixed gliomas
industry sources, and the Adis “R&D
medicines being tested as potential show a mixture of these different cells. Insight” database; current as of
treatments for breast cancer. Astrocytomas and glioblastomas August 21, 2015

combined represent 76 percent of all 6. American Brain Tumor Association

SPOTLIGHT: CHILDHOOD CANCER 1
gliomas. There are 58 medicines being
Although uncommon, cancer is the second
tested as potential treatments for brain
leading cause of death in children ages 1-14,
tumors, including gliomas.
exceeded only by accidents. In 2015, an

MEDICINES IN DEVELOPMENT | 2015 REPORT