Report to

the Nation on
Blood Cancer
Leading The Way To Cancer Cures

2017
Molly Gosch was just 5
years old when she was
diagnosed with acute
lymphoblastic leukemia
(ALL), the most common
form of childhood leukemia.
Today, she is 8 years old.
Table of Contents

2
S E C T IO N 1

Letter from the President & CEO

4
S E C T IO N 2

Our Impact

10
S E C T IO N 3

What Are Blood Cancers?

22
S E C T IO N 4

Major Accomplishments in Blood

Cancer Treatment and Survival:
1949 – Today

30
S E C T IO N 5

Accelerating Treatments
Through Innovative Research

36
S E C T IO N 6

Our Priorities for Blood Cancer Cures

44
S E C T IO N 7

How the Public Can Help Fight

Blood Cancer
2 S E C TIO N

Letter from the

President & CEO
It is never a good time to get cancer, but it is a phenomenal
time to be fighting it. I can make that statement confidently
because, as the president and CEO of The Leukemia &
Lymphoma Society, I see the extraordinary progress we are
making in fighting blood cancers and helping patients to
access lifesaving treatments and cures.
The Leukemia & Lymphoma Society (LLS) uniquely able to report on the many advances
developed this Report to the Nation on Blood and accomplishments that have occurred
Cancer: Leading the Way to Cancer Cures since our founding in 1949. From cutting-edge
to educate and engage the public in the research and precision medicine innovations
fight against blood cancers, which are the to legislative victories that improve access to
third leading cause of cancer deaths among therapies for cancer patients, LLS plays a lead-
Americans. LLS is the leading global organiza- ing – and often pioneering – role in the fight
tion dedicated to finding blood cancer cures against blood cancers.
and advancing access to treatment. We are
Advancing blood cancer treatments and
cures also means advancing the science
and treatment of other types of cancers and
certain chronic diseases. Since 2000, approx-
“This report is designed to imately 40 percent of all the newly U.S. Food
educate the public about blood & Drug Administration (FDA) approved cancer
drugs were for blood cancer, and some are
cancers – where we are now and now used to treat other forms of cancer and
where we hope to be – so we can non-malignant diseases. A “win” for blood
continue to advance research and cancers, therefore, is a “win” for the cancer
ensure access to treatments to community overall.

help save more lives.” As there are very limited means of preventing
blood cancers, the LLS research agenda is
focused on finding treatments and cures.
 THE LEU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLO OD CA N C ER 3

In fact, we have invested more than $1 billion

in research since our inception 68 years ago.
We have invested more than
This is an extremely exciting time in the field

$1 Billion
of blood cancer research and treatment. In
the past three years alone there has been
remarkable progress in treatments for patients
with multiple myeloma (MM) and chronic
lymphocytic leukemia (CLL). At the same time,
in research since our inception
emerging approaches in immunotherapy 68 years ago.
and precision medicine are showing great
promise.

Voluntary health agencies and patient advo-

cacy groups are recognizing now more than where we hope to be – so we can continue
ever the key role that they can play in bringing to advance research and ensure access to
together the entire healthcare “ecosystem” treatments to help save more lives. Public
around the development of new therapies for awareness of blood cancer and engagement
patients. Organizations like LLS are uniquely with research and advocacy organizations like
suited to serve as a convener of academic LLS is essential to making sure that we seize
researchers, regulatory agencies, payers, this unprecedented time in science to bring
patients, and the pharmaceutical industry in advances to blood cancer patients quickly.
a way that promotes collaboration and yields
new therapies for patients more quickly. At LLS, we have a saying: We are saving lives
not someday, but today. With the public’s
Given the extraordinary progress that the support, and continued collaboration among
blood cancer community has seen, it would be researchers, doctors, advocacy organizations,
easy to assume that we are near the goal line pharmaceutical companies, regulators and
of a world without blood cancers. patients, we are well on our way to making
someday today.
Unfortunately, this is not the case. Despite our
great successes, more than one-third of blood
cancer patients still do not survive five years
after their diagnosis. The death rate from
certain blood cancers, such as AML, remains
stubbornly high, with treatment protocols that
have not changed in decades. Clearly, much
work still needs to be done to understand
the genetic underpinnings of blood cancers
Louis J. DeGennaro, PhD
and find new ways to correct or block those
President and CEO
defects.
The Leukemia & Lymphoma Society
This report is designed to educate the public
about blood cancers – where we are now and
4 S E C TIO N

Our Impact
At LLS, our mission is to cure leukemia, lymphoma,
Hodgkin’s disease and myeloma, and improve the quality
of life of patients and their families. Compared to any other
blood cancer nonprofit, LLS is the largest funder of cutting-
edge research and cures.
Though LLS is known for funding ground-
breaking research to find better treatments
Our mission is to cure leukemia, and cures, we do so much more. We provide
free information, education and support
lymphoma, Hodgkin’s disease
services for those who have been impacted
and myeloma, and improve the by blood cancer. We fight for lifesaving policy
quality of life of patients and changes at the state and federal level to
their families. ensure access to quality, affordable, coor-
dinated care. We are committed to working
tirelessly toward our mission every single day,
until we find a cure.
 THE LEU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOOD CA N C ER 5

Death Rates Per 100,000 People

10
9
8
7
We’ve seen
6 an average
5 decline of
4 20 percent
3 in blood
2 cancer
1 death rates
0 since the
1990 1992 1995 1998 2001 2004 2007 2010 2013 1990s.

Leukemia Non-Hodgkin Lymphoma Hodgkin Lymphoma Myeloma

5-Year Relative Survival Rates (Percentage)

100
90
80 Since the
70 1960s,
60 survival rates
50 for many
40 blood cancer
30 patients have
20
doubled,
10
tripled
0
or even
1977 1980 1983 1986 1989 1992 1995 1998 2001 2005 2012
quadrupled.
Leukemia Non-Hodgkin Lymphoma Hodgkin Lymphoma Myeloma
6 S E CT I ON 2 → Our Impact

1 RESEARCH
Since 1949, LLS has supported remarkable scientists whose
work has led to breakthrough advances in blood cancer
treatments. To date, LLS has invested more than $1 billion
in cutting-edge research, funding nearly all of today’s most
promising advances, and bringing us closer to cures.

2 EDUCATION & SUPPORT

As the leading source of free blood cancer information,
education and support for patients, survivors, families and
healthcare professionals, LLS helps patients navigate their
cancer treatments and ensures they have access to quality,
affordable and coordinated care.

Nearly More than supported

more than
2,000 $323 74,600
inquiries per month
come in to LLS’s million patients since
Information distributed in inception.
Specialists from co-pay financial
people seeking assistance
support.
 THE LEU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLO OD CA N C ER 7

We have invested more than

4,000 300
$1 Billion research projects
have been supported
research projects are
being supported at any
in cancer research since 1949.
since 1949. given time.

Currently funding research at nearly

$50–$70 Million
100 medical institutions
across the globe. has been invested annually over the past decade.

More than More than

1,000 600,000
clinical trial searches educational booklets about specific
were conducted for diseases were distributed last year.
patients in 2016.
Our Information
Specialists are master’s
More than level oncology social
workers, nurses and
More than
4,000 health educators who
work one-on-one with

700 patients and caregivers over the

past year joined LLS Community,
blood cancer patients
and caregivers at no cost
to provide information
connections were and support tailored to
an online social network that their specific diagnosis
made in 2016
provides education and support. and needs. Services
between patients include clinical trial
and volunteers diag- searches, financial and
nosed with the same emotional support, and
disease through up-to-date disease and
Nearly treatment information.
LLS’s Patti Robinson family support
To reach an Information
Kaufmann First
Connection Program. 200 groups across the
United States.
Specialist, call
(800) 955-4572.
8 SECTION 2 → Our Impact

3 ADVOCACY
Through our nationwide grassroots network of 104,000
volunteers, LLS advocates for policies at the state and
federal level, and is committed to removing barriers to care
for blood cancer patients.

Advanced
laws in 43 states and Washington, D.C., to ensure patients
taking oral medications at home receive equitable
coverage to patients treated in a clinic.

Helped
pass the 21st Century Cures Act
into law, ensuring reform that will enable the FDA to speed
the review and approval of new therapies.

More than
100s 13,000
30,000 of calls were
made to House
advocates contributed
to the passage of 21st
letters were sent to
leaders. Century Cures Act.
members of Congress.

Advocated to ensure More than

19,000
proposed federal health-
care legislation provides
stable, quality, affordable
coverage to the thou- letters sent and hundreds
sands of blood cancer of calls made to members
patients impacted. of Congress.
 THE LEU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOOD CA N C ER 9

VOLUNTEER LED,
STAFF DRIVEN

R E SE A RCH LLS has more than

150
E D U CATION
& SU PPORT
15.5 Light The Night walks
will be held across the

A DVOCACY
million country this year.

volunteers across the nation.

Nearly
LLS volunteers
selflessly dedicate
time and energy to 5 signature
campaigns
104,000
volunteer advocates are
our organization. With for volunteers to join: Team affecting change by advo-
countless ways to In Training®, Light The cating for policy at the
Night®, Student Series, Man state and federal level.
engage, participate, & Woman of the Year™, and
and volunteer, from Leukemia Cup Regatta®.
individual fundraisers
Nearly
to corporate teams
to patient support
and advocacy, our
More than
8,000
volunteers are 27,000 volunteers across the
nation work with our
the drivers behind schools across the country patient services team
changing the are donating change in our to provide information
Student Series campaign. and support to patients
landscape of cancer. and caregivers in their
communities.
10 S E C TIO N

What Are Blood

Cancers?
Approximately every three minutes, a person in the United
States is diagnosed with a blood cancer. Every nine minutes,
someone dies from it. An estimated 173,000 Americans
will be diagnosed with leukemia, lymphoma or myeloma in
2017, and more than 58,000 Americans will die from these
diseases.

New cases of these blood cancers are

expected to account for more than 10
percent of the estimated 1.6 million new
Estimated New Cases (%) of cancer cases diagnosed in the U.S. in 2017.
Leukemia, Lymphoma and Myeloma Additionally, nearly 1.3 million people in the
U.S. are either living with, or are in remission
from, leukemia, lymphoma or myeloma.

17% As the name suggests, blood cancers affect

Leukemia – 36% the production and function of blood cells;
36% most of these cancers start in the bone
Lymphoma – 47%
marrow where blood cells are produced.
Myeloma – 17% In cancer, the normal blood cell develop-
ment process is interrupted by uncontrolled
47% growth of an abnormal type of blood cell.
These abnormal cells prevent the body
from performing many of its functions, such
as strengthening the immune system and
preventing serious bleeding.
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 11

There are three main Blood Cancer

types of blood cancers:
leukemia, lymphoma Types
and myeloma. In There are three main types of blood cancers:
addition, there are leukemia, lymphoma and myeloma. In addition,
other types that affect there are other types that affect the blood
the blood and bone and bone marrow, including myelodysplastic
syndromes and myeloproliferative neoplasms.
marrow, including
myelodysplastic While there are three main types of blood
syndromes and cancer, each cancer is unique and there
are many different subtypes. This is why
myeloproliferative new developments in precision medicine
neoplasms. treatments are targeting cancers at the
molecular level to ensure patients receive the
right treatment at the right time.
12 SECTION 3 → W hat are blood cancers ?

B LO O D CAN CER TYPES : LE UKE MIA

Leukemia
Leukemia begins in a cell in the bone marrow. immune system from effectively guarding
Once the marrow cell undergoes a leukemic against infection due to a lack of neutro-
change, the leukemia cells may grow and phils (a type of white cell).
survive better than normal cells. Over time,
the leukemia cells crowd out or suppress the • Thrombocytopenia occurs when there
development of normal cells. In 2017, more is a low number of platelets, which can
than 62,000 people will be diagnosed with cause bleeding and easy bruising with no
leukemia, and there are an estimated 364,000 apparent cause.
people living with, or in remission from,
• Pancytopenia occurs when there are low
leukemia in the U.S.
numbers of all three blood cell counts:
Without a normal number of healthy blood red blood cells, white blood cells and
cells, an individual can develop a variety of platelets.
serious health conditions:
The rate at which leukemia progresses and
• Anemia is characterized by a low number how the cells replace the normal blood
of red cells in the blood, which can cause and marrow cells is different with each type
fatigue and shortness of breath. of leukemia. There are four main types of
leukemia (see charts starting on next page).
• Neutropenia is characterized by a low
number of white cells, which prevents the

Left: Isabel Munson was an

18-year-old college freshman
when she was diagnosed
with chronic myeloid
leukemia (CML). Today,
she is a 22-year-old writer,
researcher, producer and DJ.

Right: In 2015, Matt

Fontanesi was 33 years
old and on his honeymoon
with his wife, Dani, when
he was diagnosed with
acute myeloid leukemia
(AML). Today, Matt is still
undergoing treatment for
transplant-related issues
but is doing exceptionally
well. The couple lives in San
Diego where they volunteer
with LLS.
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOOD CA N C ER 13

B LO OD CAN CER TYPES: LE UKE MIA

M AIN T YPE S OF LE UKE MIA

1 Acute Lymphoblastic Leukemia (ALL) 2 Acute Myeloid Leukemia (AML)

Description ALL is a cancer of the bone marrow and Description AML is a cancer of the bone marrow and
blood that affects the immune system. blood that affects cells that are not fully
developed. AML develops when the DNA of
a developing stem cell in the bone marrow
Prevalence/ 75,300 people are living with ALL. 5,970 is damaged, which is called an “acquired
New Cases/ new cases are diagnosed each year. 1,440 mutation.”
Deaths people die each year.

Prevalence/ 48,615 people are living with AML. 21,380

Typical age ALL is the most common cancer found in New Cases/ new cases of AML are diagnosed each year.
at diagnosis children and young adults under 20 years Deaths 10,590 people die from AML each year.
of age. About four in ten cases of ALL are
in adults.
Typical age AML generally affects adults aged 60 years
at diagnosis and older. At least half of patients are older
5-year 70.7% overall. 92% for children/adolescents than 65 when diagnosed.
survival rate younger than 15 years, and 94.1% for chil-
dren younger than 5 years. Less than 20%
for adults over age 60. 5-year 30.3% for patients 55-64 years old. 12.1% for
survival rate patients 65-74 years old.

Treatment Chemotherapy. Stem cell transplantation.

Tyrosine kinase inhibitors. Clinical trials. Treatment Chemotherapy. Stem cell transplantation.
Clinical trials.

Risk factors For most people who have ALL, there are
no obvious reasons why they develop Risk factors Repeated exposure to the chemical benzene
the disease. Researchers have found that has been identified as a potential risk factor.
more developed countries and higher People with certain genetic disorders, such
socioeconomic groups tend to have higher as Down syndrome, Familial Platelet Disorder,
ALL rates, but they have not reached any Fanconi anemia, Shwachman syndrome and
firm conclusions, which suggests that many Diamond-Blackfan syndrome, or who have
factors may be involved. Infants born with had past chemotherapy or radiation treat-
Down syndrome are at increased risk. ments for other cancers, appear to be more
People with certain genetic disorders, such likely to develop AML.
as neurofibromatosis, Klinefelter syndrome,
Fanconi anemia, Shwachman syndrome,
Bloom syndrome and ataxia telangiectasia,
are also at increased risk.

C ONT INUE S →
14 S E C T I ON 3 → W hat are blood cancers ?

B LO O D CAN CER TYPES : LE UKE MIA

M AIN TYPES OF LE UKE MIA (CONT INUE D)

3 Chronic Lymphocytic Leukemia (CLL) 4 Chronic Myeloid Leukemia (CML)

Description CLL begins in the bone marrow and is the Description CML develops when the DNA of a devel-
most common form of leukemia in adults. oping stem cell in the bone marrow is dam-
CLL does not completely interfere with the aged. CML does not completely interfere
development of mature red cells, white cells with the development of mature red cells,
and platelets. white cells and platelets. CML is usually
diagnosed in its chronic phase when treat-
It can progress either slowly or quickly,
ment is very effective for patients, and is
depending on the form it takes. It is generally
generally less severe than acute leukemia.
less severe than acute leukemia.
People with CML have an abnormal
chromosome called the Philadelphia (Ph)
Prevalence/ 162,374 people are living with CLL. 20,110 new chromosome, which leads to the develop-
New Cases/ cases are diagnosed each year. 4,660 people ment of a cancer-causing gene (oncogene)
Deaths die each year. called the BCR-ABL gene.

Typical age CLL is more common in people who are 70 Prevalence/ 44,386 people are living with CML. 8,950
at diagnosis years or older. New Cases/ new cases are diagnosed each year. 1,080
Deaths people die each year.

5-year
survival rate 85.1% overall Typical age Most cases of CML occur in adults, with a
at diagnosis median age of 64 years old.

Treatment Watch and wait. Single or combination drug 5-year 65.9% overall. Today, the 5-year survival
therapy. Targeted therapies. Monoclonal survival rate rate of those newly diagnosed with CML
antibody therapies. White blood cell growth who participated in clinical trials for imatinib
factors. Radiation therapy. Splenectomy. is more than 90 percent.
Clinical trials.

Treatment Tyrosine kinase inhibitors. Stem cell trans-

Risk factors There are no obvious reasons why people plantation. Clinical trials.
develop CLL. Experts have found that in a
small number of cases, first-degree relatives
(parents and siblings) of people with CLL are
Risk factors Two known risk factors are exposure to
three to four times more likely to develop
very high doses of radiation and high-dose
CLL than people who don’t have first-degree
radiation therapy (radiotherapy) used to
relatives with the disease.
treat other cancers such as lymphoma.
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 15

B LO OD CAN CER TYPES: LYMPHOMA

Lymphoma
Lymphoma is the name for a group of blood The two main types are Hodgkin lymphoma
cancers that develop in the lymphatic system, and non-Hodgkin lymphoma (see charts
part of the body’s immune system. In 2017, starting on next page).
there will be approximately 80,500 newly
diagnosed cases of lymphoma. An estimated
816,634 people are living with, or in remission
from, lymphoma in the U.S.

Steve McHugh, was 35

years old when he was
diagnosed with non-
Hodgkin lymphoma (NHL)
in 2010. Today, he is a
survivor and renowned
chef and owner of Cured,
a restaurant in San
Antonio, TX.

Erica Campbell was 27 years

old when she was diagnosed
with Hodgkin lymphoma (HL) in
2013. Today, she is a survivor,
inspirational speaker and model
living in Washington, D.C.
16 SECTION 3 → W hat are blood cancers ?

B LO O D CAN CER TYPES : LYMPHOMA

TYPE S OF LYMPHOMA

1 Hodgkin Lymphoma (HL) 2 Non-Hodgkin Lymphoma (NHL)

Description HL, formerly known as Hodgkin’s disease, is a Description NHL is a type of cancer that affects
cancer of the blood and bone marrow and one of the lymphatic system and generally
the most curable forms of cancer. Hodgkin lym- develops in the lymph nodes and lym-
phoma starts when an abnormal change to a white phatic tissues, but in some cases, NHL
cell (called a lymphocyte) causes it to become a involves bone marrow and blood. NHL
lymphoma cell. Lymphoma cells grow and form is not just a single disease – it is actu-
masses, usually in the lymph nodes, located ally a diverse group of blood cancers
throughout the body in the lymphatic system. that share a single characteristic in how
they develop.

Prevalence/ 186,607 people are living with HL. 8,260 new

New Cases/ cases are diagnosed each year. 1,070 people die Prevalence/ 630,027 people are living with NHL.
Deaths from HL each year. New Cases/ 72,240 new cases are diagnosed each
Deaths year. 20,140 people die each year.

Typical age Most common in young adults in their 20s and

at diagnosis early 30s or in adults over age 65. Typical age Most common among adults between
at diagnosis 65 and 74 years old.

5-year 88.5% overall. For patients who were diagnosed

survival rate under the age of 45, the survival rate is 94.3%. 5-year
72.6% overall
survival rate

Treatment Chemotherapy. Combined modality therapy, which Treatment Chemotherapy. Radiation therapy.
is when two or more types of treatment (e.g., radi- Monoclonal antibody. Stem cell trans-
ation, chemotherapy) are used alternately or at the plantation. Clinical trials.
same time. Immunotherapy.

Risk factors For most people who have NHL, there

Risk factors There are no obvious reasons why people are no obvious reasons why they
develop the disease. develop the disease.

Patients who have a history of a blood test con- Living or working in farming com-
firming mononucleosis have a three-fold increased munities and exposure to herbicides
risk of HL compared to the general population. and pesticides have been shown to
increase the risk of developing NHL.
People infected with human T-cell lymphocytotro-
pic virus (HTLV) or human immunodeficiency virus Exposure to bacteria and viruses, espe-
(HIV) also have an increased risk of HL. cially those that suppress the immune
system, has been shown to increase
the risk of developing NHL.
 THE L E U KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 17

B LO OD CAN CER TYPES: LYMPHOMA

M A IN TYPES OF NON-HODGKIN LYMPHOMA

1 Diffuse Large B-Cell Lymphoma (DLBCL) 2 Follicular Lymphoma (FL)

AG G RESSIV E OR FAST GROW IN G N H L I N DO L EN T O R S LOW G R OWI N G N HL

Description DLBCL is the most common NHL subtype. Description FL is the most common indolent (or slow
It grows rapidly in the lymph nodes and growing) NHL subtype.
frequently involves the spleen, liver, bone
Abnormal lymphoma cells are grouped
marrow or other organs.
together throughout the lymph node.
DLBCL development usually starts in lymph
nodes in the neck or abdomen and is
characterized by masses of large B cells
Incidence About 22 percent of NHL cases.
(lymphocytes).

Incidence About 30 percent of NHL cases. Typical age Most people with FL are age 50 or older at
at diagnosis diagnosis.

Typical age It most commonly occurs in middle-aged and Overall

at diagnosis older persons. The median age of diagnosis Survival Overall survival is more than 10 years.
is 65.

Overall Treatment Watch-and-wait approach. Radiation therapy.

Survival Five-year survival rate is 60% overall. Chemotherapy with rituximab followed by
radiation therapy.

Treatment Combination chemotherapy. Risk factors Most FL cells have a specific chromosome
abnormality (a translocation between parts
of chromosomes 14 and 18) that causes the
Risk factors People with B-cell–activating autoimmune overexpression of a gene, BCL-2, and makes
diseases, hepatitis C virus, first-degree family the cells resistant to therapy.
history of NHL, and greater body mass index
(BMI) as a young adult are at increased risk
for developing DLBCL.

CONT INUE S →
18 SECTION 3 → W hat are blood cancers ?

B LO O D CAN CER TYPES : LYMPHOMA

MA IN TYPES OF N ON - HODGKIN LYMPHOMA (C ONT INUE D)

3 Marginal Zone Lymphoma (MZL) 4 Mantle Cell Lymphoma (MCL)

I ND OLENT OR SLOW GROW IN G N H L AG G R ES S I V E O R FAST G R OWI N G N HL

Description MZL includes several subtypes, each Description Mantle cell lymphoma (MCL) is generally
categorized by the type of tissue where the considered an aggressive type of B-cell
lymphoma forms: outside of the lymph nodes non-Hodgkin lymphoma.
(extranodal or MALT), in the lymph nodes
(nodal), and the spleen (splenic).
It begins in B-lymphocytes in a part of the Incidence About 6 percent of NHL cases.
lymph tissue called the “marginal zone.”
The disease tends to remain localized.
Typical age MCL occurs more frequently in older adults—
at diagnosis the average age at diagnosis is the mid-60s.
About 12 percent of B-cell lymphoma cases
Incidence
are MZL.
Overall
Survival Median overall survival is 3-5 years.
Typical age Most people with MZL are ages 60-65 years
at diagnosis old.

Treatment Combination chemotherapy.

Overall Five-year survival rate is about 85% overall
Survival for extranodal (MALT) MZL.
Five-year survival rate is about 60-70% Risk factors MCL is more often diagnosed in males than
overall for nodal MZL. in females.

Overall survival is 5-10 years for splenic MZL.

Treatment Removal of the spleen. Single-agent che-

motherapy. Combination chemotherapy.
Immunotherapy with rituximab. Rituximab
combined with chemotherapy.

Risk factors People with systemic lupus erythematosus

and Sjögren’s syndrome, B-cell activating
immune conditions, hepatitis C, peptic ulcers,
asthma without other atopic diseases, and
a first-degree relative with a hematological
malignancy, are at increased risk for
developing MZL.
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 19

B LO OD CAN CER TYPES: MULT IPLE MYE LOMA

Multiple Myeloma
Myeloma is a cancer of plasma cells, which
are a type of white blood cells (also called Multiple Myeloma
plasma B cells). Myeloma develops when a
plasma cell is mutated. Healthy plasma cells
are part of the immune system and make Description Multiple myeloma is the most common form of the disease,
and it affects several different areas of the body.
proteins called antibodies, which help fight
infection. The most common form of the
disease is called multiple myeloma because Prevalence/ 110,345 people are living with multiple myeloma. 30,280 new
the malignant cells form tumors in multiple New Cases/ cases are diagnosed each year. 12,590 people die each year.
Deaths
areas of the body.

Multiple new treatments, including several

Typical age Most people who develop myeloma are older than 50 years.
antibody therapies, were approved in the at diagnosis Fewer cases occur in people younger than 40.
last three years and are likely to increase the
overall survival rate for patients diagnosed
5-year
with myeloma. 50.2% overall
survival rate

Treatment Single or combination drug therapy. Chemotherapy. Targeted

therapies. Stem cell transplantation. Radiation therapy.
Clinical trials.

Risk factors Black Americans are nearly twice as likely to develop

myeloma as white Americans. Men are at higher risk than
women.
People with a history of MGUS (monoclonal gammopathy of
unknown significance) have increased risk.
New research suggests that obese people have a higher
incidence of myeloma.
20 S EC T I O N 3 → W hat are blood cancers ?

B LO O D CA N CER TYPES : MYE LODYSPLAST IC SYNDROME S

Myelodysplastic Syndromes
Myelodysplastic Syndromes (MDS) are a released into the blood. This results in a
group of diseases of the blood and bone lower than normal number of circulating
marrow, with varying degrees of severity, blood cells. Approximately 30 percent of
treatment needs and life expectancy. MDS patients diagnosed with MDS are at high risk
begin with a change to a normal stem cell of their disease converting to acute myeloid
in the marrow. These developing blood leukemia (AML).
cells, called blast cells, die as they approach
maturity before they would normally be

Myelodysplastic Syndromes (MDS)

Description MDS are a group of diseases of the blood and bone marrow. MDS develops when blood
cell production in the bone marrow increases with more-than-the-normal number of
developing blood cells (called “blast cells”) filling the marrow.

New Cases An estimated 15,350 new cases of MDS were diagnosed each year from 2009–2013.

Typical age
Occurs more often in people over 65 years.
at diagnosis

Treatment Observation of blood cell counts. Transfusions and iron chelation therapy.
Erythropoiesis-stimulating agents (ESAs)/growth factors. Managing infections. Drug
therapy. Chemotherapy. Stem cell transplantation. Clinical trials.

Risk factors Primary MDS – no obvious cause in most patients; repeated exposure to chemical
benzene.
Treatment-related MDS – previous treatment of chemotherapy and radiotherapy for
other cancers.
 THE L E U KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 21

B LO OD CA N CER TYPE S: MYE LOPROLIF E RAT IVE NE OPLASMS

Myeloproliferative
Neoplasms
Myeloproliferative Neoplasms (MPNs) are types of
blood cancers in which bone marrow cells proliferate
abnormally and often genetic mutations are found.

Myeloproliferative Neoplasms (MPNs)

Types There are three types of MPNs: Essential Thrombocythemia (ET), Myelofibrosis (MF) and
Polycythemia Vera (PV).

Description ET/MF/PV are rare cancers in which the bone marrow cells function abnormally.

New Cases About 2.2 out of every 100,000 people are diagnosed with ET each year. About 1.5
out of every 100,000 people are diagnosed with MF each year. About 2.8 out of every
100,000 men and 1.3 out of every 100,000 women are diagnosed with PV each year.

Typical age MPNs are usually diagnosed in adult men and women. ET occasionally occurs in older
at diagnosis children.

Survival rate PV and ET can be managed effectively for a long time and, with proper treatment,
people can have a normal or near-normal quality of life. While the median survival for
people with MF is about 5 years, people younger than 55 with good prognostic factors
have a median survival of 11 years.

Treatment Drug therapy. Chemotherapy. Plateletpheresis. Phlebotomy. Stem cell transplantation.

Clinical trial.

Risk factors For most people, there are no obvious reasons why they develop ET. About 90% of
people with MF have a mutation in one of three genes – JAK2, CALR or MPL. Almost all
people with PV have a mutation of the JAK2 gene.
22 S E C TIO N

Major Accomplishments
in Blood Cancer
Treatment and Survival:
1949 – Today
Since the founding of LLS (originally known as
The Leukemia Society of America) in 1949, we have
made enormous strides in our understanding and
treatment of blood cancers.

LLS has supported the development of some – funding both clinical and basic science –
of the most effective and widely used ther- has led to groundbreaking clinical trials and
apies, from the early days of combination breakthrough research on effective treatments
chemotherapies and bone marrow transplants for blood cancer patients.
more than 50 years ago, to immunotherapies
and precision medicine today. Our support LLS has played a major role in bringing
groundbreaking treatments to blood cancer
patients.

Through LLS’s numerous research programs

and work to improve patient access to better
LLS has played a treatments, survival rates for many blood
major role in bringing cancer patients have doubled, tripled and
even quadrupled since 1960. LLS is espe-
groundbreaking
cially proud that some of the therapies first
treatments to blood approved for blood cancer patients are now
cancer patients. helping patients with other types of cancers
and serious diseases.
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOOD CA N C ER 23

CM L S POTLIG H T

From Fatal Disease to

Manageable Condition
Imatinib (Gleevec®) is a targeted therapy
originally approved by the FDA in 2001 for
the treatment of chronic myeloid leukemia
(CML), turning a once fatal diagnosis into
a manageable condition for most patients.
Given its remarkable success, the drug
also has been approved by the FDA
to treat other blood cancers, including
Philadelphia Positive (PH+) acute lympho-
blastic leukemia (ALL) in children, rare
stomach cancers and skin cancers.

The journey to develop imatinib took

more than a decade, led by the extraor- improvements. Today, the five-year sur- Brian Druker,
dinary efforts of Brian Druker, MD, who is vival rate of those newly diagnosed with MD, Director
now the Director of The Knight Cancer CML who participated in clinical trials for of The Knight
Cancer
Institute at Oregon Health & Science imatinib is more than 90 percent. The
Institute at
University (OHSU). Druker found a way to overall survival rate for CML patients is Oregon Health
“turn off” the enzymes that cause cancer. 63 percent. While the reasons for this & Science
Researchers around the country – and the disparity are not well understood, one University
world – were involved in different aspects factor may be that patients in clinical trials
of the research and development that receive continuous care and do not incur
led to the discovery of imatinib. Funding out-of-pocket costs, resulting in better
from LLS provided critical support at key adherence to treatment.
progress points, including the discovery
of a genetic abnormality by LLS-funded Imatinib is also being tested in studies
investigator Janet Rowley, MD, of the for patients with a type of colon cancer,
University of Chicago, as well as proof of neurofibromatosis, and diabetes. Imatinib
concept work by Druker. works by inhibiting a group of enzymes
that serve many functions, including roles
Clinical trials of the drug began in 1998 in cell growth and proliferation, as well
and the results were astonishing: 98% as the autoimmune response in diseases
of patients with CML showed dramatic such as diabetes.
24 S EC T I O N 4 → M a j or Accomplishments : 1949 – Today

A T I M E L I NE

The impact of LLS

funding over decades in
the fight against cancer

1949 1950 1955 1955

In 1949, Rudolph and George H. Hitchings, In 1955, William James Holland, MD,
Antoinette Roesler de PhD, and Gertrude Dameshek, MD, was among the first
Villiers, who lost their B. Elion, D.Sc., began became a medical LLS grant recipients,
teenage son, Robert, collaborating in 1945 and scientific advisor receiving funding in
to leukemia in 1944, and developed the to LLS, organizing its 1955; he went on to
established the first most widely used grant review process. become one of the
incarnation of what anti-leukemia drugs in In 1946, he was a first researchers to
became The Leukemia 1950-1951. Both later lead investigator of advance combination
& Lymphoma Society. served as medical studies that led to chemotherapy.
The impact was felt and scientific advisors the first anti-cancer
Photo: PBS/Cancer: The
right away and the to LLS, and earned chemotherapy. Emperor of All Maladies
1950s and 1960s saw the 1988 Nobel Prize
some major treatment in Physiology and
advances that were Medicine.
revolutionary for the
Photo: Wellcome Library,
time. London
 THE L E U KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 25

In 1964, the five-year survival rate

for children with the most commonly
diagnosed pediatric leukemia,
ALL, was 3 percent. Today, it’s
approximately 90 percent.

1956 1965 1970s 1980 1985

E. Donnall Thomas, The first combination The advent of the Riccardo Dalla- In 1985 and again
MD, conducted the chemotherapy 1970s brought an early Favera, MD, and his in 1989, Hagop
first successful bone was developed for understanding of the LLS-funded research Kantarjian, MD,
marrow transplant on childhood leukemia genetics of cancer team studied the received LLS scholar
a leukemia patient patients by Emil with the discovery of molecular pathway awards to study new
in 1956. Thomas “Tom” Frei, MD oncogenes. involved in immune approaches to treating
was an LLS advisor (background), and Emil B-cell activation and patients with chronic
in the 1960s, and J. Freireich, MD, under For his role in how those pathways myeloid leukemia
was awarded the the leadership of identifying oncogenes, became dysregulated (CML). He later played
1990 Nobel Prize Gordon Zubrod, MD, J. Michael Bishop, in B-cell cancers. A a significant role in the
in Physiology and (foreground), Director MD, an advisor to few decades later, he development of the
Medicine. of the National Cancer LLS, later received led a team of LLS- first targeted therapy
Institute’s Clinical the 1989 Nobel Prize funded researchers to treat CML patients.
Photo: Susie Fitzhugh in Physiology and
Center. investigating the
Medicine. By the genetic origins of
Photo: The Zubrod Family 1980s, researchers B-cell non-Hodgkin
advanced this lymphoma.
knowledge further.

Photo: PBS/Cancer: The

Emperor of All Maladies
26 S EC T I O N 4 → M a j or Accomplishments : 1949 – Today

“LLS is where it is today because it has groomed a

remarkable generation of scientists and physician
scientists who have led the extraordinary advances in
treatment of hematologic malignancies.”

GARY GILLILAND, MD, PHD, PRESIDENT OF FRED HUTCHISON

COMPREHENSIVE CANCER CENTER

1990 1996 1996 2001 2004

By the 1990s, the In 1996, LLS-funded Charles Sawyers, From 2001 to A multi-institution
discovery of genetic investigator Brian MD, began studies 2005, LLS-funded Phase II trial in 2004,
pathways was followed Druker, MD, tested that helped identify researchers David led by Alan List, MD,
by the first FDA a BCR-ABL-blocking the genetic causes of Maloney, MD, and clinical colleagues,
approvals for targeted drug. This resulted in resistance to imatinib, Margaret Shipp, MD, including LLS-
blood cancer drugs. the first clinical trial, leading the way to Felipe Samaniego, funded investigator
led by Druker and the development of MD, and others Richard Stone, MD,
In 1990, LLS-funded Charles Sawyers, MD, follow-on therapies for showed rituximab confirmed the efficacy
researcher Susan of STI-571, later known patients with imatinib- (Rituxan®) improved of lenalidomide
Rabinowe, MD, and as imatinib (Gleevec®) resistant CML. the effectiveness (Revlimid®) for patients
others, showed the in 1998. of the standard with a rare form of
protein CD20 is chemotherapy regimen myelodysplastic
consistently present for diffuse large B-cell syndromes (del(5q)
on B-cell chronic lymphoma patients. MDS).
lymphocytic leukemia
(CLL) cells.
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOOD CA N C ER 27

Forty years ago, the five-year survival rate for

someone diagnosed with myeloma was 10
percent. Today, it’s about 50 percent. Over
the past decade, treatment options, many
supported with LLS funding, have increased
significantly, and the survival rates are
expected to continue to increase.

2010 2012 2014 2016 Today

Stephan Grupp, LLS-funded research New advances in In 2016, FDA LLS is supporting
MD, PhD, played an continued to drive immunotherapy approved of a new emerging work to
instrumental role in breakthroughs and include an approach therapy, venetoclax understand how
the advancement advances, transforming called immune (Venclexta®), for mutations lead to
of immunotherapy, cancer’s grim past into checkpoint inhibitors, patients with a blood cancers. Today,
particularly in a promising future. that unleash the high-risk form of LLS supports the work
treating pediatric From 2010-2012, immune system chronic lymphocytic of Benjamin Ebert,
acute lymphoblastic John Byrd, MD, and by removing the leukemia. Since 2002, MD, PhD, and Irene
leukemia (ALL) colleagues published “brakes.” This LLS continuously Ghobrial, MD, who
patients. Almost two Phase II trial results approach is advanced supported research are studying how to
decades before, LLS for ibrutinib in CLL by researchers, by Jerry Adams, PhD, prevent recurrence
awarded Grupp with a and small lymphocytic including LLS-funded Andrew Roberts, after treatment as
Career Development lymphoma (SLL) investigators Margaret MBBS, PhD, and well as preventing
Special Fellow Award, patients, showing Shipp, MD, and Steve colleagues to advance precursor diseases
which he credited with safety and lasting Ansell, MD, PhD. this therapy. from progressing to
helping to launch his remissions. blood cancer.
career.
28 SECTION 4 → M a j or Accomplishments : 1949 – Today

C L L S POTLIGH T

Targeted Therapy Extends

Survival and Improves
Quality of Life
Ibrutinib (Imbruvica®) is a therapy that follicular lymphoma (FL), diffuse large
targets the Bruton’s tyrosine kinase (BTK) B-cell lymphoma (DLBCL), hairy cell leuke-
enzyme, a promoter of B-cell cancers, mia (HCL) and multiple myeloma (MM).
including chronic lymphocytic leuke-
mia (CLL), mantle cell lymphoma (MCL), John C. Byrd, MD, of The Ohio State
Waldenstrom’s macroglobulinemia (WM), University – a long-time LLS grant recip-
ient – began his research to tackle CLL
by targeting BTK with ibrutinib in 2009.
Because CLL does not have a single
genetic mutation, many experts predicted
that precision medicine would not be
an effective approach. However, clinical
trials of ibrutinib showed unprecedented
response rates.

First approved in 2013 for mantle cell

lymphoma, ibrutinib was then approved
for patients with relapsed or refractory
CLL in 2014, with additional approvals
following in 2015 and 2016. For thou-
sands of patients, this drug has not
only extended their survival, but also
improved their quality of life.

In 2016, a study by an LLS Specialized

Center of Research (SCOR) team led by
Tom Kipps, MD, UCSD, showed ibruti-
nib to be superior to standard therapy
with chemotherapy, leading the FDA to
approve it as a front-line treatment for
John C. Byrd, MD, of The Ohio State University CLL patients.
 THE L E U KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 29

Walter and Eliza Hall Institute of Medical Research in Melbourne, Australia

CLL S POTLIGH T

A New Option for Patients Not

Responding to Treatment
Venetoclax (Venclexta ®) is a targeted
therapy approved by the FDA in April
2016 to treat patients with a high-risk form
of chronic lymphocytic leukemia (CLL)
who have not responded to at least one
other therapy. The oral medication is for
patients who have a rare subset of CLL
known as 17p deletion, which means they
are missing a piece of chromosome 17.

Approximately 30 percent of CLL patients

who do not respond to therapy or who
have relapsed have the 17p deletion.
Venetoclax works by targeting the B-cell
lymphoma 2 (BCL-2) protein, which sup-
ports cancer cell growth.

Since 2002, LLS has supported more

than $15 million in research leading to the
development of this drug through its col-
laborative Specialized Center of Research
(SCOR) grant program. Jerry Adams, PhD,
of the Walter and Eliza Hall Institute of
Photo: Walter and Eliza Hall Institute of Medical Research in Melbourne, Australia
Medical Research in Melbourne, Australia,
leads the research team. Andrew Roberts,
MBBS, PhD, a member of Adams’s SCOR for patients with acute lymphoblastic Jerry Adams,
leukemia (ALL), mantle cell lymphoma PhD, of Walter
team, was one of the clinicians who led and Eliza
studies of the pivotal Phase 2 clinical trial (MCL) and acute myeloid leukemia (AML).
Hall Institute
of venetoclax, leading the FDA to approve This includes the work of Anthony Letai,
of Medical
the therapy. MD, PhD, Dana-Farber Cancer Institute, Research in
who is studying the effectiveness for Melbourne,
LLS is currently supporting research to AML patients, with encouraging results Australia
investigate venetoclax as a treatment reported to date.
30 S E C TIO N

Accelerating
Treatments Through
Innovative Research
There has been tremendous momentum and excitement
in blood cancer research over the past several years as
precision medicine and immunotherapies such as CAR-T
cell therapy have shown very promising early results in
treating patients and extending lives.

From the discovery of imatinib (Gleevec®),

which has transformed chronic myeloid
leukemia (CML) from a devastating disease
Our growing to a chronic condition, to new monoclonal
understanding of the antibody treatments for myeloma, to immune
checkpoint inhibitors, our growing understand-
genetic underpinnings
ing of the genetic underpinnings of blood
of blood cancer and cancer and the pace of drug discovery have
the pace of drug been super-charged.
discovery have been
LLS supports the full spectrum of research
super-charged. from bench to bedside – that is, from basic,
laboratory-based research to large-scale clin-
ical trials – with the singular goal of accelerat-
ing treatments and cures to the more than
1.2 million people in the United States living
with some form of blood cancer.
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOOD CA N C ER 31

LLS supports multiple research programs LLS supports the full spectrum of
through specialized grants, collaborations research from bench to bedside –
and venture philanthropy. Our Career
Development Program is designed to support
that is, from basic, laboratory-based
promising investigators in their developing research to large-scale clinical trials.
careers. Specialized Center of Research
(SCOR) grants fund multinational, multi-
disciplinary teams of researchers who are
engaged in collaborative efforts, while our diagnosis and/or treatment of blood cancers
Translational Research Program (TRP) brings and related pre-malignant conditions. Through
promising research findings from the labora- our Therapy Acceleration Program® (TAP), we
tory to clinical development. Our New Idea partner directly with academic institutions and
Awards fund innovative approaches that may biotechnology companies to help accelerate
fundamentally change the understanding, the development of promising therapies.

Our Investment
T H E A M O UN T L L S IS C UR R E NTLY CO MMITTE D TO INVE STING IN R E SE AR CH

AML 25% $69 M

ALL 14% $40.1 M
MDN/MPNs 13% $37.3 M
Myeloma 13% $37.1 M
Gen. Lymphoma 8% $22.5 M
CLL / SLL 7% $20.9 M

Aggressive NHL 6% $16.7 M

Indolent NHL 5% $15 M

Gen. Leukemia 5% $15 M

CML 2% $6.5 M

Hodgkin Lymphoma 2% $6.3 M

0 $10M $20M $30M $40M $50M $60M $70M

32 SECTION 5 → Accelerating Treatments Throu gh Innovative R esearch

OUR RESEARCH
PORTFOLIO

1 I N VE STS I N YO U N G S C I E N T I STS
Career Development Program attracts and retains
the highest quality young scientists, launching the
careers of many of the most productive clinicians and
researchers in cancer.

2 L E ADS T H E C H A R G E TO BE AT A M L
Beat AML Master Trial® is a groundbreaking, collaborative
clinical trial for acute myeloid leukemia (AML), a deadly
disease that has seen few improvements in treatments in
more than 40 years.

3 T RAN SL AT E S R E S E A R C H FR O M
B E N CH TO BE D S I D E
Translational Research Program was developed
in 1996 to provide early-stage support for
clinically translatable research in blood cancers.
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOOD CA N C ER 33

Nearly at more than

More than

3,000 $370 million 400

grants awarded medical and
since 1953 invested in grants academic institutions

T HIS INC LUDE S:

3 More
than 15 9 10
Nobel members of the directors of department
Laureates National Academy comprehensive chairs/section
of Science cancer centers directors

11 7
cancer centers will or more treatment study arms
participate by summer 2017 will open by summer 2017

60 is the age that newly

diagnosed patients can
enroll in trial
Patient’s genetic
analysis completed
within
7 days

700
LLS has awarded investing

$313
approximately

grants through
this program
million
34 S E C T I ON 5 → Accelerating Treatments Through Innovative R esearch

OUR RESEARCH
PORTFOLIO

4 FAST-T RAC K S T R E AT M E N T TO PAT I E N TS

Therapy Acceleration Program® expedites getting
treatments to patients by supporting promising projects
and clinical trials through collaborations with biotechnology
companies.

5 F OST E RS C O L L A BO R AT I O N AC R O S S
DI SC I P L I N E S & I N ST I T U T I O N S
Specialized Center of Research program brings together
established investigators across different disciplines from
one or several institutions to develop a research program
over five years. These synergistic collaborations greatly
advance research progress and clinical applications.

6 E N C OU RAG E S “O U T O F T H E BOX ”
T H I N KI N G
New Idea Award supports innovative “out of the box”
approaches that may fundamentally change the understand-
ing, diagnosis and/or treatment of blood cancers, but may not
be candidates for conventional government funding.
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOOD CA N C ER 35

More than invested in More than invested by

$100 50 $700 8
million projects since
2007. million companies to
push TAP closer
to the finish line.

Approximately
17 projects
$10 million invested
per year currently in the pipeline

Since the program has been

started in 2000, awarded across and

$300 50 30
million awards recipients

25
Since the program investing
started in 2013,
LLS has awarded
approximately grants $2.25
million
36 S E C TIO N

Our Priorities for

Blood Cancer Cures
The following are some of our priority areas we’ve set for
our agenda in the coming years:

1. Using Precision
In the past five years alone, LLS Medicine to Beat AML
has invested about $100 million in In 2013, LLS launched an unprecedented attack
AML research, with a focus on under- against acute myeloid leukemia (AML), a deadly
standing the underlying causes of the disease which has seen little to no improve-
ment in treatments in more than 40 years.
disease to develop better therapies
and save more lives. AML is among the most lethal of the blood
cancers, responsible for more than 10,500
deaths each year. The standard of treatment
for AML – a combination of toxic chemother-
apies – has remained the same for more than
four decades. Overall prognosis remains poor,
with a five-year survival rate below 20 percent
for patients over age 60.
 THE L E U KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOOD CA N C ER 37

LLS teamed up with Brian Druker, MD, and his LLS launched the Beat AML Master Trial®
research team at The Knight Cancer Institute in October 2016. it is the first collaborative
at Oregon Health & Science University (OHSU) precision medicine trial in a blood cancer. This
to lead the first phase of Beat AML. This initial groundbreaking clinical trial is using advanced
phase deployed advanced genomic technol- genomic technology to identify patients’
ogy to create a profile of genetic defects in genetic mutations and test several investiga-
AML cells. tional, targeted drugs to treat those patients.
This trial involves multiple medical institutions,
The goal was to collect 900 samples from drug companies and the FDA, all of whom
AML patients and screen more than 37 propri- have committed to working collaboratively
etary novel drugs and combinations. To date, to drive this master clinical trial forward. LLS
more than 700 samples have been collected. hopes it will serve as a model for other cancer
This ongoing work is helping lay the ground- research and discovery programs.
work for the second phase of Beat AML, a
Master Trial that is testing multiple drugs at With support and guidance from the FDA, and
multiple sites to find the most effective treat- LLS as the sponsor, the ambitious Beat AML
ments based on individual patients’ specific Master Trial® seeks to change the paradigm
genetic profiles. for how this deadly cancer is treated. LLS is

BeatBeat
AMLAMLMaster Trial®
Master Trial®

Clinical
Sites

Pharmaceutical
Companies

Clinical Research
Organization

Regulatory Agency

Clinical Trial
Knowledge Platform
Genomics Provider
Web-based Digital
Prototocol Solution
38 SECTION 6 → Accelerating Treatments Through Innovative R esearch

uniquely qualified to lead this unprecedented high-risk secondary AML. In April 2017, Jazz
clinical trial collaboration, which is a rare role Pharmaceuticals submitted its application to
for a nonprofit organization and a first for LLS. begin the process of requesting FDA approval
of this therapy.
Three world-renowned blood cancer sci-
entists lead the clinical trial: Brian Druker, With our ability to convene the landmark Beat
MD, The Knight Cancer Institute at Oregon AML Master Trial®, combined with a significant
Health & Science University (OHSU); investment in scientific research, as well as
John Byrd, MD, The Ohio State University patient support and education, we are leading
Comprehensive Cancer Center; and Ross the way to accelerate new treatments and
Levine, MD, Memorial Sloan Kettering Cancer cures for this deadly disease.
Center. Many other prominent researchers
lead each arm of the trial at their institutions.
LLS expects up to 500 patients to enroll in
the trial. The trial is on target to surpass 40
2. Connecting Patients
patients enrolled by June 2017. to Clinical Trials
Beat AML demonstrates LLS’s ability to One of LLS’s most important roles in accel-
convene the medical and research commu- erating the development of new therapies
nities to think and act boldly in the quest for is connecting cancer patients with clinical
new and better treatments for blood cancer trials, which are the essential foundation for
patients, and supports our goal to accelerate the advancement of scientific research and
the rate at which precisely targeted break- life-saving treatments. Despite the public’s
through therapies reach the patients who recognition of their value, many clinical trials
urgently need them. are unable to achieve their goals because
they cannot enroll enough patients. In fact,
Because of the urgent unmet medical need,
less than 5 percent of cancer patients actu-
LLS is taking a multi-pronged approach to find
ally enroll in clinical trials focused on finding
cures for AML. Currently, about one-fourth of
cancer treatments.
our research budget is dedicated to AML. In
the past five years alone, LLS has invested There are many reasons for the low clini-
about $100 million in AML research, with a cal trial enrollment rate, including patients’
focus on understanding the underlying causes misperceptions about clinical trials, and finan-
of the disease to develop better therapies and cial and logistical concerns. Provider attitudes
save more lives. and beliefs about trials are also a factor;
many providers do not feel comfortable
Another promising approach for the treatment
discussing trials with their patients and view
of AML is a result of a partnership through
clinical trials only as an option of last resort.
LLS’s Therapy Acceleration Program® (TAP). In
2009, LLS started to collaborate with Celator LLS has increased its efforts to help patients
Pharmaceuticals, which was acquired by enroll in clinical trials by expanding our
Jazz Pharmaceuticals in May 2016. The goal personalized clinical trial navigation services,
was to advance a therapy called CPX-351 aiming to help more than 1,000 patients
(Vyxeos®), which has performed better than enroll in clinical trials over the next five years.
standard therapy in clinical trials for patients Our clinical trial support service matches a
with a subtype of AML. In fact, the latest patient’s unique clinical, social and financial
Phase 3 data showed it reduced the risk of situation to available clinical trials.
death by 31 percent for older patients with
Dr. Larry Saltzman was diagnosed with chronic lymphocytic leukemia (CLL)
and small cell lymphocytic lymphoma (SLL) in January 2010. Because of
LLS’s clinical trial navigation service, he is currently enrolled in a clinical trial.
Photo: Sharon Saltzman

There are many reasons for the low clinical trial enrollment rate, including
patients’ misperceptions about clinical trials, and financial and logistical con-
cerns. Provider attitudes and beliefs about trials are also a factor; many provid-
ers do not feel comfortable discussing trials with their patients and view clinical
trials only as an option of last resort.

Medical experts in LLS’s Clinical Trial Support with blood cancer patients and caregivers at
Center (CTSC) help patients to find and enroll no cost to provide support and information.
in clinical trials based on highly detailed,
individualized assessments, along with the LLS also collaborates with Dana-Farber
provision of in-depth guidance, support and Cancer Institute on the Blood Cancer Research
resources. In fact, LLS’s medical experts Program (BCRP), which is designed to provide
engage in many interactions with each patient access to clinical trials in communities where
and often call clinical trial sites to help with there are no major medical centers. Through
trial enrollment. The result is that the majority its network of twelve sites across the nation,
of patients working with LLS’s CTSC have BCRP aims to accelerate the advancement
enrolled in one or more clinical trials. and expansion of access to well designed
and innovative clinical trials for blood cancer
Our clinical trial specialists work tirelessly to patients treated at community sites.
help patients find and enroll in clinical trials to
access the most cutting-edge treatments for By increasing access to and enrollment in
their diagnosis. These specialists are part of clinical trials, our goal is to provide patients
LLS’s team of Information Specialists, who are the opportunity to receive the newest
master’s level oncology social workers, nurses treatments and best care, while also helping
and health educators. Through a toll-free call to accelerate life-saving treatments to help
center (800-955-4572), they work one-on-one patients in the future.
40 SECTION 6 → Accelerating Treatments Through Innovative R esearch

In March 2017, LLS launched a program with the National Black Church
Initiative (NBCI), a faith-based coalition of churches, to address the
striking health disparities among African Americans with myeloma.

3. Increasing Our Over the next five years, through the mul-
tifaceted program called Myeloma Link:
Investment in Myeloma Connecting African American Communities to
Information, Expert Care, and Support, LLS will
Cures provide education about myeloma and clinical
trials to African Americans in major cities
In the U.S., myeloma is the second most
throughout the country.
common blood cancer. While there have been
many new therapies approved over the last The goal of the initiative is to improve access
decade and researchers are studying promis- to novel therapies and quality of life among
ing treatments, the disease remains incurable. African Americans with myeloma by providing
Only 49.6 percent of patients diagnosed with tools and resources to navigate the treatment
myeloma will survive five years after diagnosis. landscape more effectively and cope with the
disease.
To address this urgent unmet medical need,
LLS is taking a multi-pronged approach over On the research front, LLS plans to signifi-
the next five years to improve outcomes for cantly increase its current annual investment in
patients, by investing in scientific research myeloma research over the next three to five
as well as education and outreach efforts to years, with a focus on resistance to therapy,
improve patient access to the most promising, targeted therapies, immunotherapies and
cutting-edge treatments. preventing progression of the disease.

In March 2017, LLS launched a program with A major focus of this research investment is
the National Black Church Initiative (NBCI), a to develop targeted therapies that can be
faith-based coalition of churches, to address used alone or in combination with other newly
the striking health disparities among African approved drugs, as well as immunotherapies
Americans with myeloma. In fact, black that will harness the immune system to fight
Americans have twice the incidence of myeloma.
myeloma as white Americans, and recent stud-
ies show they are significantly less likely to Through a renewed investment in cut-
receive the newest treatments or combination ting-edge research and patient education over
therapies, and are more likely to experience the next five years, our goal is to find cures
treatment delays, including transplant delays. and significantly improve the lives of those
living with myeloma.
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 41

4. Driving
Immunotherapy
Forward
Since its beginnings, LLS has invested in
the promising field of immunotherapy, which
harnesses a patient’s own immune system to
kill cancer cells. It remains a major focus of our
research commitment today.

In fact, an LLS advisor, E. Donnall Thomas,

MD, conducted the first successful bone
marrow transplant on a leukemia patient in
1956. Bone marrow transplants provided the
first example of how the immune system can
help fight cancer. Doug Olson was diagnosed with
chronic lymphocytic leukemia
Today, immunotherapy is one of the most (CLL) at age 49. Today, he is a six-
promising treatment approaches for cancer. year survivor who benefited from
CAR-T immunotherapy.
One groundbreaking approach is called
chimeric antigen receptor (CAR) T-cell therapy,
which LLS has been investing in since the
mid-1990s.

This therapy has had remarkable early results

in the treatment of blood cancer, proving to
be miraculously effective in some patients In this therapy, immune T-cells are removed
with certain types of leukemia, lymphoma and from the patient’s body, and then are
most recently, myeloma. Dozens of adults genetically engineered to produce a protein
and children who were once near death are
on the surface of the T-cells that can bind and
now in remission, and some remain healthy
up to five years after treatment. recognize the cancer cells.

In this therapy, immune T-cells are removed

from the patient’s body, and then are genet-
ically engineered to produce a protein on
the surface of the T-cells that can bind and LLS has been funding CAR-T since the
recognize the cancer cells. These engi- beginning, through support of a team led by
neered T-cells are multiplied in the lab and Carl June, MD, University of Pennsylvania,
eventually given back to the patient through and his colleagues at Children’s Hospital of
an intravenous infusion. Once the T-cells Philadelphia, who are all credited with being
“home in” on the cancer cells, this triggers among the pioneers of this therapy. LLS has
the T-cells to multiply further and kill the invested in the work of Dr. June and his col-
cancer-ridden cells. leagues since 1998, investing more than
$21 million to advance this treatment.
42 S EC T I O N 6 → Accelerating Treatments Throu gh Innovative R esearch

The University of Pennsylvania’s program

was later licensed to Novartis. In March 2017,
5. Advocating for
Novartis filed for FDA approval of this therapy Quality, Affordable
and included clinical trial data showing the
therapy resulted in remission of 82 percent of Care
patients after three months.
Even with our focus on drug discovery, LLS
Last year, LLS announced an additional $11 mil- recognizes that finding cures is not enough;
lion investment in immunotherapy. At Memorial we need to ensure that patients have access
Sloan Kettering Cancer Center, LLS’s SCOR to the treatments they need to live longer, bet-
program is funding a research project aimed ter, healthier lives. We are dedicated to remov-
to advance a new generation of CAR-T cells ing barriers to care, and our network of nearly
to both eradicate cancer cells and shut down 104,000 advocates has a powerful voice in
cancer’s immune defense mechanisms. driving policies that accelerate the develop-
ment and approval of innovative treatments
and ensuring that patients have sustainable
access to quality, affordable, coordinated care.

As the voice for all blood cancer patients,

Last year, LLS announced an LLS’s Office of Public Policy has achieved
additional $11 million investment groundbreaking results for patients at both
in immunotherapy. the state and federal level. For example, LLS
helped to drive the 21st Century Cures Act for-
ward and advocate for oral parity legislation,
which has been signed into law in 43 states
and Washington, D.C.
Through its Therapy Acceleration Program®, While there has been great progress, much
LLS collaborates with Kite Pharma, a biotech- work remains to ensure that federal healthcare
nology company focused on immunotherapy, rules continue to protect cancer patients. LLS
to fund this promising therapy through a clini- works to advance policies that accomplish the
cal trial. Positive data from the clinical trial was following goals:
released showing that more than one-third of
refractory aggressive non-Hodgkin lymphoma ✓ Guarantee Access – Newly diagnosed
(NHL) patients in the study showed no signs of cancer patients must continue to have the
the disease after six months. Kite filed for FDA right to purchase quality, affordable health
approval of this therapy in March 2017. insurance to help them access the care
they need.
While the FDA has not yet approved CAR-T
immunotherapy, patients are able to enroll in ✓ Ensure Quality – Policymakers must
clinical trials. LLS will continue to invest in this continue to provide minimum quality
promising therapy, while providing support standards that protect patients from being
through our clinical trial navigation service to locked out of necessary treatment due to
patients who are either currently in a CAR-T barebones coverage.
clinical trial or interested in enrolling in one.
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 43

LLS recognizes that finding cures is not enough; we need to

ensure that patients have access to the treatments they need to
live longer, better, healthier lives.

✓ Promote Affordability – Premium assis- Senate champions are drafting legislation

tance and cost-sharing limits that allow a to advance this important protection for
cancer patient to use their coverage must cancer patients.
be improved.
We also focus on accelerating new ther-
✓ Provide Stability – Policymakers must apies and cures for patients through the
provide cancer patients with the peace of following:
mind that every patient will have access
to affordable, quality coverage. ✓ Patient Engagement in Drug
Development & Approval – LLS advo-
Our team of advocates also works to cates for improvements that can speed
remove barriers and ensure patients are the development and approval of safe
able to access cancer treatments. Our goals and effective treatment options for blood
include: cancer patients. LLS is working to secure
Congressional approval of FDA user
✓ Reducing Out-of-Pocket Costs for fee legislation that will provide the FDA
Medicare Patients – LLS advocates for with the resources necessary to fulfill
protections against prohibitive patient out- its commitment to amplify the voice of
of-pocket costs for life-saving prescription patients throughout the development and
medications. LLS supports legislation to approval processes.
allow Medicare Part D beneficiaries to
pay a flat, reasonable copay for essential ✓ Expanded Access Reform – LLS sup-
medications with no less costly alter- ports necessary improvements to the
natives. LLS also supports limiting the FDA’s expanded access program, which is
total annual out-of-pocket expenses for intended to help facilitate patient access
Medicare patients with high treatment to investigational drugs when they have
costs. In the near future, legislation will be no other treatment options. LLS is working
introduced advancing these policies. to build on transparency reforms enacted
in the 21st Century Cures Act that will
✓ Oral Parity for Cancer Drugs – LLS help patients and providers navigate this
works to advance federal and state leg- complex program.
islation that would promote fairness and
innovation by preventing insurance plans Our advocacy efforts are unapologetically
from requiring patients to pay significantly patients first, and we will continue to advance
more out-of-pocket costs for cancer drugs policy at the state and federal level to ensure
based on how the drug is administered. patients have access to quality, affordable,
A bipartisan coalition of House and coordinated care.
44 S E C TIO N

How the Public Can

Help Fight Blood
Cancer
As the largest voluntary cancer research agency specifically
focused on finding cures and treatments for blood cancer
patients, LLS supports hundreds of cancer scientists and
research projects around the world. But the fight against
blood cancers cannot be won without the public’s support.

Blood cancer patients, their families, friends

and colleagues, along with individuals
who have not been touched directly by
Every voice, every action, every cancer, are powerful allies in the fight, and
contribution is needed and their voices and actions carry tremendous
valued. LLS encourages the influence with researchers, physicians, pol-
public to join the fight in one or icymakers, regulators and industry. Yet, we
often hear from the public that they are not
all of these ways. sure how to get involved and whether their
individual involvement “counts.” Our answer
is an unequivocal “yes.” Every voice, every
action, every contribution is needed and
valued. LLS encourages the public to join the
fight in one or all of these ways:
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 45

Learn About and Help LLS Advocate for

1
Policies that Accelerate Treatments and Cures

LLS advocates for public policy positions that our policy goals, LLS encourages the public
accelerate progress toward cures for blood to contact their elected representatives – in
cancers and improve the quality of life of Congress and in state legislatures – to share
patients, along with their friends and families. with them the impact that blood cancers have
on millions of Americans each year, and to
Policy advocacy is essential because critical urge responsible policies that will address the
challenges remain in moving new therapies serious burdens of these diseases.
through the regulatory review process, and
many blood cancer patients still face signif- More information about LLS’s policy advo-
icant barriers to access the care they need. cacy efforts, overseen by our Office of
Regulatory agencies and legislative bodies at Public Policy in Washington, D.C., can be
both the state and federal level play a pivotal found at www.lls.org/advocate.
role in addressing these problems. To advance

Encourage Family and Friends with

2
Cancer to Participate in Clinical Trials

Advances in treatment for blood cancers their doctor about their clinical trial eligibility.
depend on clinical trials of new therapies LLS’s clinical trial navigation service can help
or new combinations of therapies. Today, patients find and enroll in an appropriate
virtually all of the established treatments for clinical trial. Patients and caregivers can
cancer are available because of clinical trials. access this service free of charge by calling
(800) 955-4572 or going to www.lls.org/
Cancer patients, especially the newly diag- informationspecialists.
nosed, should be encouraged to talk with
46 SECTION 7 → H ow the P ublic Can H elp Fight Blood Cancer

3 Volunteer and Campaign with LLS

LLS is the world’s largest voluntary health events to patient services and family support
agency dedicated to blood cancer, and volun- groups to education programs to community
teers are the heart and soul of the organiza- outreach. To learn how to get involved, visit
tion. Volunteers help in so many ways – sup- www.lls.org/volunteer.
porting patients, advocating for better access
to treatments, making fundraising efforts both LLS has been a pioneer in creating theme-
fun and effective, and always setting the stan- driven campaigns that raise funds for blood
dard for compassion and kindness. cancer research and support while engaging
the public with our mission. Our signature
With 56 chapters across the country, there are campaigns include:
many ways to volunteer – from fundraising

Team In Training is the original sports train-

ing program for charity, providing “team-
mates” a unique opportunity to experience
team camaraderie and experienced coach-
ing, personal discovery and mastery, while
supporting groundbreaking discoveries in
research. Team In Training raised $32 million
in fiscal year 2016, and has raised more
than $1.5 billion over the past 29 years,
training more than 650,000 teammates. As
it approaches its 30th year, Team In Training
is once again leading the pack with new
offerings to excite the next generation. No
longer just marathons, Team In Training’s
portfolio includes high-caliber events in
During a sports physical in 1991, when she was 15 years old, Jennifer cycling, hiking and climbing; most recently,
Swanton was diagnosed with leukemia. In 2001, she joined Team In Team In Training’s first “Climb 2 Cure” teams
Training and has completed a marathon, triathlon and century ride. summited Mount Kilimanjaro.
THE L E U KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 47

Light The Night is a powerful campaign bringing light to the dark-

ness of cancer. One million friends, families and co-workers gather
together, carrying illuminated lanterns in 150 inspirational evening
walks, to celebrate, honor or remember those touched by cancer.
Light The Night had a record year in 2016, raising $68.5 million for
blood cancer research; the campaign has raised more than $625
million since 1999. In 2016, Light The Night introduced Random Acts
of Light, engaging celebrities and local heroes to surprise people
touched by blood cancers with special meetings, to help brighten
their lives during a dark time.

Student Series is a service learning, character education and

philanthropy program where students gain the unique experience
of helping thousands of people in their fight against blood cancers.
Programs are tailored to each school level so students can grow
with Student Series and have an even greater impact on the lives of
patients each year. More than 13 million students and 850,000 edu-
cators in 27,000 schools across the U.S. participate annually. Since it
began, students have raised more than $315 million to fund break-
through therapies and patient services, with more than $26 million in
2015-2016 alone.

Leukemia Cup Regatta is a thrilling series of sailing events that

combines the joy of boating with the important task of raising money
to cure cancer. At events held at yacht clubs across North America,
skippers register their boats and recruit friends and colleagues to help
crew and to raise funds. Crew members seek donations from friends,
family, co-workers and employers to sponsor their boat. More than
$58 million has been raised through the Leukemia Cup Regatta series
for lifesaving research and patient services since its start 30 years
ago. The fiscal year 2016 Leukemia Cup Regatta campaign grossed
nearly $3.9 million.
48 S EC T I O N 7 → H ow the P ublic Can H elp Fight Blood Cancer

Man & Woman of the Year is a truly unique fundraising campaign. In

an annual competition in communities across the country, candidates
compete in honor of children who are local blood cancer survivors,
the Boy & Girl of the Year, by raising funds for LLS. By engaging
influential community leaders, Man & Woman of the Year taps into
the spirit of innovation and entrepreneurship that has allowed LLS
to achieve great success in funding groundbreaking research to
advance cancer cures. The winners receive LLS’s “Man & Woman of
the Year” titles. Every dollar raised counts as one vote and the titles
are awarded to the man and woman with the most votes at the end
of 10 weeks. Top local fundraisers become eligible to win national
titles. In 2016, candidates raised $38 million.

4 Support Blood Cancer Cures

LLS funds research based on the most urgent ✓ Improving access to affordable, quality
medical needs, provides education and and coordinated care. As the voice for
support to patients, and advocates for poli- all blood cancer patients, LLS achieves
cies that ensure affordable, coordinated care. groundbreaking results for patients by
Every dollar invested is used in a number of advocating for legislation at the state
ways in the fight against blood cancers: and federal level.

✓ Helping patients and their families. LLS ✓ Encouraging young scientists to pur-
provides information and support to guide sue blood cancer research. Grants to
patients from diagnosis through survivor- young scientists help grow research
ship, and helps ensure access to current talent even as federal research funding
treatments and clinical trials. becomes increasingly limited.

✓ Investing in immunotherapies and

precision medicine. Developing the
most cutting-edge immunotherapy and
precision medicine treatments in order
to find cures.
DE SI GN: 3 RD E D GE

donate.lls.org
 THE L EU KEMIA & LYMP H OMA S OCIETY R EPORT TO THE NATION ON BLOO D CA N C ER 49

In 2013, Christopher
Abeleda was 19 years old
when he was diagnosed with
acute lymphocytic leukemia
(ALL). Today, he is a survivor
and college graduate.
OU R MI S S I ON

Cure leukemia, lymphoma, Hodgkin’s disease

and myeloma, and improve the quality of life
of patients and their families.

3 International Drive, Suite 200, Rye Brook, NY 10573 914.949.5213 www.lls.org

LLS707 3M 4/17