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Accelerating Progress: Moving Toward a Cure for Melanoma 2008-2011 Report

I n March, an

immunotherapy called

Yervoy (ipilimumab)

became the first drug

approved by the U.S. Food

and Drug Administration

(FDA) in 13 years for

metastatic melanoma. Then the molecularly

targeted agent Zelboraf (vemurafenib), an

inhibitor of a mutated protein found in about

half of melanomas, was approved by the FDA

in August. These breakthroughs underscore

remarkable progress that has been unfolding in

the four years since the founding of the Melanoma

Research Alliance (MRA), a unique organization

whose mission is to find and fund the most

promising research worldwide. MRA is working

to better prevent, detect early and treat deadly

skin cancer, toward a day when no one suffers

or dies from melanoma.

No one person or organization can win the

battle against melanoma alone. We are thankful

to all who have engaged with us in this fight—

researchers, supporters, allies, partners, and more.

This report offers a

retrospective of the four

years since the founding

of MRA, including the

progress we have made,

a highlight of scientific

findings, and a look to

the future of melanoma research. MRA funding has

been vital to unlocking the best treatments and

continues to offer hope for better therapies and

ultimately a cure.

As the pace of progress quickens, MRA is

re-doubling its efforts to accelerate research.

With more than $30 million awarded for promising

research, MRA has catalyzed strategic and collabo-

rative research efforts to discover new biomarkers

of treatment response, demystify drug resistance

mechanisms, and improve effectiveness through

combination therapies. MRA is working with all

who care about defeating melanoma, moving from

“the year of melanoma” to “the year of the

melanoma cure” as quickly as possible.

DEBRA BLACK , co-founder and chairWENDY K.D. SELIG , president and CEO

Our First Four YearsA period of landmark advances in melanoma research and treatment.

LETTER FROM THE CHAIR AND PRESIDENT

“This is clearly the year of melanoma…”George W. Sledge, Jr., M.D., American Society of

Clinical Oncology Past President - June 2011

MRA is a public charity formed in 2007 under

the auspices of the Milken Institute, with generous

founding support from Debra and Leon Black. The

mission of MRA is to accelerate the pace of scientific

discovery and its translation in order to eliminate

suffering and death due to melanoma. Thanks to the

ongoing support of its founders, MRA applies 100

percent of public donations to its research program.

In its first four years, MRA has committed $30.4 million

to 73 research programs with the potential to make

significant, near-term clinical impact in prevention,

diagnosis, staging, and treatment. MRA is the largest pri-

vate funder of melanoma research in the United States.

MRA 2008-2011About MRA 03

About MRAStrategic

$30.4MRA has committed $30.4 millionto 73 research programs. It is thelargest private funder of melanomaresearch in the United States.

MRA research awards provide an important and unique

source of funding that addresses the gap in translational

science, which is a critical stage in moving scientific

discoveries into tools and treatments for patients.

Hallmarks of the MRA research portfolio include:

• Focus on collaborative team science: Team

Science Awards, which fund multidisciplinary research

groups, account for almost three-quarters of research

funding to date, followed by a variety of individual

awards: Established Investigator, Young Investigator,

Pilot/Development Awards, and Partnership Awards.

MILLION

THE FIRST FOUR YEARS

$35

$30

$25

$20

$15

$10

$5

0

GROWTH OF MRA’S RESEARCH PROGRAM

Fun

din

g (

Mill

ion

s)

2011

2010

2009

2008

TOTAL COMMITMENTS BY AWARD TYPE

Partnership ($0.3M)

Established($4.2M)

Pilot/Dev($0.7M)Young

($2.8M)

Team ($22.4M)

Thanks to the ongoing generous support ofits founders, MRA applies 100 percent ofpublic donations to its research program.

114PRINCIPAL INVESTIGATORS

04 www.curemelanoma.orgStrategic


.

• Investment in developing new treatments

for metastatic disease: Nearly 80 percent of

research funding is directed at melanoma treatment,

followed by diagnosis/staging and prevention. In

particular, MRA is investing significant resources to

improve upon the two new treatment approaches—

immune checkpoint targeting and selective BRAF

inhibition.

• Advances in key scientific and clinical

areas: MRA-funded research has made progress in

all of the key areas of opportunity identified at its

inaugural Call to Action meeting in 2007. Because of

NEARLY 80 PERCENT

About MRAStrategic

Nearly 80 percent of researchfunding is directed at melanomatreatment, followed by diagnosis/staging and prevention.

the rapid progress that has been made, MRA has

updated and revised this agenda for the next four years.

• Support for a diverse group of melanoma

researchers worldwide: MRA is funding 114

Principal Investigators at 56 institutions in 10

countries (Australia, Belgium, Canada, Germany,

Israel, Netherlands, New Zealand, Sweden,

Switzerland, and the United States).

TOTAL COMMITMENTS BY SCIENCE AREA

Diagnosis/Staging($3.7M)Treatment

($24.0M)

Prevention($2.8)

VFCDG8

LB

I

s

UnitedStates

Canada

Europe

Israel

9

NewZealand

MRA 2008-2011Strategic 05

AustraliaWestmeadHerstonMelbourneSydney

BelgiumLeuven

CanadaTorontoMontreal

GermanyFrankfurtHeidelberg

IsraelJerusalemRamat Gan

NetherlandsAmsterdamGroningenLeidenMaastrichtRotterdam

New ZealandWellington

SwedenLund

SwitzerlandZurich

United StatesPhoenix, AZLa Jolla, CALos Angeles, CAOakland, CAPasadena, CASan Francisco, CASanta Monica, CAStanford, CATampa, FLChampaign, IL

Chicago, ILBoston, MACambridge, MABaltimore, MDAnn Arbor, MIDetroit, MISt. Louis, MOPrinceton, NJCold Spring Harbor, NYNew York, NYRochester, NY

Cleveland, OH Philadelphia, PAProvidence, RINashville, TNHouston, TXSalt Lake City, UTCharlottesville, VASeattle, WA

Australia

Global PresenceLocation of Principal Investigators leading MRA-funded awards, 2008-2011

06 www.curemelanoma.orgCollaborative


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About MRACollaborative

Collaboration is at MRA's core—from the

team approaches to research that it funds

to the way it finds partners who can help

realize its vision. Relationships built with

other nonprofits, corporations, and individuals

continue to increase:

• MRA and Stand Up to Cancer (SU2C) are jointly

funding a Melanoma Dream Team of at least $6

million beginning in early 2012. This ground-

breaking award represents one of the largest single

private grants for melanoma research and the first

collaboration of its kind with SU2C.

• In its first ever such offering, MRA selected two

Academic-Industry Partnership Awards in 2011

that will support projects in conjunction with matched

contributions from an industry partner whose

participation is essential to the project. MRA and the

partner company will jointly support research with a

high likelihood of clinical translation to help patients

and those at risk for melanoma.

• MRA has co-funded research projects with fellow

cancer research foundations, including the Canadian

Cancer Society, Cancer Research Institute, and

Melanoma Research Foundation.

PREVENTION

Funded by an MRA Team Science Award, Drs. NicholasHayward and Nicholas Martin (Queensland Institute ofMedical Research) and Dr. Graham Mann (WestmeadInstitute for Cancer Research and University of Sydney) discovered five new melanoma risk genes that conferredan increased risk of melanoma between 14 and 22 percent. In contrast to other known risk genes, none of

these appear to be associated withphysical characteristics such as fairskin or moles.

Relative contributionof pigmentation and other genes tomelanoma risk

Relative Contribution of Pigmentation and Other Genes to Melanoma Risk

• Through its growing Allies program, MRA has raised

awareness of melanoma and funds for melanoma

research with nearly 50 companies who believe in its

mission. For example, with the help of key Corporate

Allies, including Claire’s, SkinCeuticals, and The

Sports Authority, MRA reached as many as 19 million

people with information about melanoma while

raising nearly $350,000 for melanoma research

during Melanoma Awareness Month in May 2011.

MRA also leveraged its affiliation with the Milken

Institute by hosting an outreach event on the closing

day of the 2011 Global Conference in Beverly Hills.

The event, supported by Corporate Allies Graff,

Montage and Style Network , introduced the work

of MRA to dozens of new supporters. Expanded

collaborations with additional companies that share

the MRA mission, as well as non-profit organizations

like the Girl Scout Council of the Nation’s Capital,

are underway.

• When Danny Federici, longtime member of Bruce

Springsteen’s E Street Band, passed away from

melanoma in April 2008, his family and friends

received an outpouring of support. Together with

MRA Co-Founders Debra and Leon Black, they

brought their Danny Fund under the MRA umbrella,

working to bring melanoma to the forefront of

public awareness and to fund research around the

world. Springsteen recorded a public service

announcement to raise awareness of melanoma and

MRA, which aired during the 2009 Super Bowl and in

movie theaters across the country.

MRA 2008-2011Collaborative 07

Through its growing Allies program,MRA has raised awareness ofmelanoma and funds for melanomaresearch with nearly 50 companieswho believe in its mission. MRAreached as many as 19 million peoplewith information about melanomawhile raising nearly $350,000 formelanoma research in May 2011,during Melanoma Awareness Month.

When longtime member of Bruce Springsteen's E Street Band DannyFederici (center) was diagnosed with melanoma, the Danny Fund was launched.

In just a few years of active research, theproductivity of MRA-funded investigators has been high, as measured by tangible outcomes and promising early researchresults, advancing the understanding of the causes, origins, and progression ofmelanoma and developing new therapies for patients with advanced disease:

• 9 clinical trials supported by MRA to test promising

agents alone and in combination for the treatment of

metastatic melanoma.

• $17.2 million in additional research funding leveraged

from other sources by MRA investigators. The

majority of this funding was granted by the U.S.

National Institutes of Health through a merit-based

peer-review process.

• 13 patent applications filed or new inventions disclosed

for new technologies, which have the potential to be

developed into new tools or treatments for patients.

• More than 100 collaborations initiated and

strengthened between academic, government, and

industry scientists, which enhance sharing of informa-

tion and fast-forwarding of the research.

• More than 50 papers published in high-impact journals

describing research results supported by MRA, which

inform the scientific and clinical communities about

promising new research findings so that others may

build upon the results to help patients.

• Hundreds of presentations delivered by MRA

investigators at medical and scientific meetings

around the world, where research results are shared

and the profile of MRA is enhanced.

08 www.curemelanoma.orgTransformative

About MRATransformative


.

$50

$40

$30

$20

$10

0

INNOVATIVE MRA-FUNDED RESEARCH WINS ADDITIONAL SUPPORT (IN MILLIONS)

Mill

ion

s

leveragedmelanoma

funding

$30.4 MRA investment

$17.2

With an MRA Established Investigator

Award that was leveraged to secure an

NIH grant, Dr. Stephen Hodi (Dana-Farber

Cancer Institute) conducted a Phase I trial

investigating ipilimumab with bevacizumab

in stage IV melanoma patients and worked

to define the mechanism of action for this

synergistic drug combination. Ipilimumab

works to boost the immune system to fight

cancer and bevacizumab blocks the growth

of blood vessels to tumors. Based on this

work, Hodi secured an NIH R21 grant to

continue the trial. In this small group of

patients, some have experienced durable

clinical benefits, and side effects have

been manageable.

Combination Therapies

MRA-Funded Research

At the first gathering of the world's leading melanoma

researchers and clinicians convened by MRA in 2007,

the group identified the crucial scientific and clinical

questions that need to be addressed in order to transform

the field of melanoma prevention, detection and treat-

ment, and to begin to map out concrete ways to effect that

transformation. It has been nearly four years since that

Call to Action meeting and the launch of MRA. In this

short time, MRA has become the largest private funder

of melanoma research, with programs delving into each

of the 17 scientific and clinical questions prioritized by

that distinguished group.

In 2011, MRA released its revised and updated StrategicResearch Plan, which recognizes the enormous strides

that have been made and identifies areas of greatest need

for scientific discovery. The updated research plan will

guide the development of the MRA scientific portfolio

over the next granting cycles.

Research proposals submitted to MRA are reviewed by

MRA’s Grant Review Committee (pages 19-20), a group

of leading experts in melanoma and cancer research.

Review is based on MRA’s key criteria: innovation, scien-

tific merit, and potential for rapid progression to clinical

testing. MRA’s conflict-of-interest guidelines ensure a fair

MRA 2008-2011Accountable 09

About MRAAccountable

and unbiased process. Final funding decisions are

ratified by the MRA Board of Directors.

The MRA Scientific Advisory Panel (pages 19-20)

advises, assists, and makes recommendations to the

MRA leadership team on the scientific matters and

policies of MRA, including research needs and

opportunities that may be targeted for funding and

planning scientific symposia.

Each year, MRA hosts a Scientific Retreat that attracts

more than 150 attendees, including the world’s leading

scientists, as well as senior leaders from nonprofit

foundations, government agencies, industry, and other

key stakeholders. The retreat features focused sessions on

key topics of interest, invited speakers and presentations

from MRA-funded investigators. To summarize the

meeting’s key themes, MRA publishes a report of

highlights. Since its inception, MRA has hosted four major

meetings, with the next one planned for March 2012.

Each year, MRA leadership and staff conduct outreach

and site visits to MRA-funded institutions to learn more

about their melanoma programs, hear about progress

being made thanks to their MRA award funding, and

further develop relationships with awardees.

Above left: Drs. Adrian Senderowicz of AstraZeneca, Richard Pazdur of the U.S. Food and Drug Administration, George Demetri of the Dana-FarberCancer Institute, Jonathan Cebon of the Ludwig Institute for Cancer Research, Melbourne Clinical Sciences Center, F. Stephen Hodi of the Dana-FarberCancer Institute, and Paul Chapman of the Memorial Sloan-Kettering Cancer Center discussed regulatory approval pathways for new melanoma therapies during the 2011 scientific retreat. Above right: Tony Ressler, Debra Black, MRA co-founder and chair, and Michael Milken, MRA board member, listened to presentations at the 2010 scientific retreat.

MRA has become the largest privatefunder of melanoma research.

10 www.curemelanoma.orgStrategic Research Portfolio


.

MRA focuses on innovative translational

research that has the potential to impact melanoma

prevention, diagnosis/ staging, and treatment in the near

and intermediate future.

Prevention: Research focusing on the biological

causative factors in melanoma carcinogenesis includes

studies of skin pigmentation biology and genetic muta-

tions in melanoma driving downstream biochemical

pathways. By investigating the mechanisms involved in

the development of melanoma, this work will inform bio-

logically-based strategies for prevention and therapy.

Diagnosis and staging: There are significant chal-

lenges in diagnosing and staging for primary melanoma.

The current system based on visual clues and histology

is not adequate, particularly in defining metastatic

potential, resulting in either over-treatment or under-

treatment of some patients. New imaging technology

is also needed in the metastatic setting. MRA-funded

Strategic Research PortfolioFunding by Research Area

researchers are working to identify new molecular prog-

nostic biomarkers and to develop better imaging agents.

Treatment: In March 2011, an immunotherapy called

Yervoy (ipilimumab/anti-CTLA-4) became the first

drug approved by the FDA in 13 years for metastatic

melanoma. The molecularly targeted agent Zelboraf

(vemurafenib), an inhibitor of mutant BRAF found

in approximately 50 percent of melanomas, was

approved in August 2011. These breakthroughs

underscore remarkable progress. Yet, these will not

provide cures for most patients, and much more

remains to be done until melanoma is effectively

addressed. MRA is funding key areas to accelerate the

next generation of treatments.

In 2011, Yervoy (ipilimumab/anti-CTLA-4) became the first drug approved by the FDA in 13years for metastatic melanoma.

IMMUNOTHERAPIES

Drs. Antoni Ribas (University of California, Los Angeles) andJames Heath and David Baltimore (California Institute ofTechnology), with funding from an MRA Team Science Award,developed a new tool for improving adoptive T cell transfer thera-py (ACT). Immune cells are very complex and diverse, and currenttechnologies limit the full understanding of how these cells behaveto combat melanoma cells in patients during treatment. This new tool, called a single-cell barcode chip (SCBC) is a complexnanotechnology-based platform that enables researchers for thefirst time to look at the single T cells during ACT treatment.

The SCBC consists of microchannels that contain cellsfrom a patient’s blood sample, valves and ports that isolate the cells and control reagents, and a barcodeglass substrate for protein detection (pictured as red and green lines)

• Immunotherapies: Melanoma is one of the

most immunogenic human cancers and can induce

specific anti-tumor immune responses in patients

that are potentially capable of eliminating tumor

cells. MRA-funded projects include characterizing

immunological signatures of successful therapies,

developing better melanoma vaccine strategies,

and improving adoptive T cell transfer protocols.

MRA has invested nearly $5 million in funding to

research projects aiming to improve melanoma

checkpoint blockade such as elucidating immune

correlates of response and combining these agents

with other therapies.

MRA 2008-2011Strategic Research Portfolio 11

MRA has invested nearly $5 millionin projects to improve melanomacheckpoint blockade and $6 million in BRAF-related studies.

• Molecularly targeted therapies: Targeted

cancer therapies block the growth and spread of cancer

by interfering with specific cellular signaling pathways

involved in tumor growth and progression. MRA is

funding research on therapies directed at multiple

molecular targets in melanoma as well as identifying

drug resistance mechanisms and strategies to overcome

them. MRA is investing $6 million in research involving

BRAF, including discovering drug resistance mecha-

nisms and combining BRAF inhibitors with other agents.

• Combinatorial therapies: Cancers are dependent

on a number of altered molecular pathways and can

develop resistance to single agent therapy; thus, combi-

nation regimens may provide the best hope for durable

effects. MRA-funded studies in this area include

development of two investigation agents and combina-

tions of investigational agents with approved agents.

Drug dramatically helps melanoma patients. “This news about vemurafenib is thelatest in a positive year for melanoma research, and a significant milestone.” Dr. SuzanneTopalian, MRA’s chief science officer, quoted in The Washington Post, June 5, 2011

NUMBER OF MRA-FUNDED PROJECTS IN MELANOMA PREVENTION, DIAGNOSIS/STAGING, AND TREATMENT

biological mechanisms of carcinogenesis

developing preventionagents based on studies of ultravioletlight on fair-skinnedindividuals

Prevention

5

2

identifying new biomarkers

supporting skin screening

funding imaging ofmetastases

Diagnosis/Staging

5

2

1

immunotherapy

molecular targets for therapy

combination therapy

Treatment

22

22

14

12 www.curemelanoma.orgStrategic Research Portfolio


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MRA has funded 24 Teams, 20 Established

Investigators, 19 Young Investigators, 8 Pilot

and Development projects, and 2 Academic-

Industry Partnership Awards.

Team Science Awards fulfill one of MRA’s primary goals:

to foster a collaborative research process. Multidiscipli-

nary teams consist of Principal Investigators with

complementary expertise who may be from the same

institution, inter-institutional, and/or international

institutions. Team science projects promote transforma-

tional melanoma research advances with the potential

for rapid clinical translation.

Established Investigator Awards support senior inves-

tigators with an established record of scientific productiv-

ity and accomplishment and who are past the initial four

years of their first academic faculty appointment.

Young Investigator Awards aim to

attract early career scientists with

novel ideas into melanoma

research, thereby recruiting and

supporting the next generation of

melanoma researchers. Young

Investigators are scientists within

four years of their first academic

faculty appointment. A mentorship

commitment from a senior investigator is required. The

Young Investigator Award Program grew dramatically in

2011 through a focused MRA funding campaign, almost

doubling the cohort and bringing the total program to

$2.8 million awarded to 19 investigators since 2008.

Strategic Research PortfolioFunding by Type of Award

Pilot Awards test potentially transformative ideas that

do not have extensive preliminary data but articulate a

clear hypothesis and translational goals. Resources for

such “high-risk, high-reward” projects are important to

establish proof-of-concept, which may then leverage

additional funding through more traditional avenues.

Development Awards are a subtype in which one year of

funding is provided.

Partnership Awards are designed to facilitate interac-

tions between sectors, including industry and other non-

profit organizations.

“MRA support is critical to starting new lines of research and has a multiplier effect on funding, collaborations, and progress forpatients.” Dr. Patrick Ott, 2009 MRA Young Investigator, New York University

The Young Investigator AwardProgram grew dramaticallyin 2011 through a focusedMRA funding campaign,almost doubling the cohortand bringing the total program to $2.8 million awarded to19 investigators since 2008.

Dr. Patrick Ott, 2009 MRA Young Investigator,New York University

In 2011, MRA unveiled a new logo and

website—www.curemelanoma.org—launching

a new brand that symbolizes its vision for

accelerating the pace of scientific discovery

and translation.

The internet and social media have proven to be

effective ways to reach audiences. The MRA website

receives tens of thousands of visits from people in more

than 100 countries looking for information about MRA,

melanoma, and the latest in research. With more than

2,000 followers on Facebook and Twitter who receive

daily updates on melanoma news, MRA saw a 35 percent

increase in social media followers in 2011.JENNIFER BERRY GOODEN - MISSAMERICA 2006

MRA 2008-2011Outreach 13

Outreach


.Fast-Forwarding a Cure for Deadly Skin Cancer

JENNIFER BERRY GOODEN

MISS AMERICA 2006

I was 25 years old in March 2009when I realized I needed to go in for my yearly skin check. Therehad been a small spot on my leftankle and every time I looked at it I somehow knew the spot just didn't look right.

During my appointment, the dermatologist told mehe'd like to biopsy the spot. The biopsy showed that it was melanoma.

On the day of the surgery I arrived at the hospital withbutterflies in my stomach. I was given all the scarypotentials as I signed all my consent forms.

After the surgery, they told me that everything hadgone superbly.

www.curemelanoma.org

A week or so later, I got the call telling me the biopsy on my lymph node had come back completely clear. I was cancer free. For the first time in over a month, I could breathe.

I continued to allow my leg to heal, and around the 10-week mark, it finally felt normal again, and I couldwalk. The swelling was gone, and although I had an interesting scar, I was amazed at my body's ability to heal.

This entire experience changed my life forever. I now visit the dermatologist and my surgeon every three to six months. This will be a part of my life forever. Butthese small inconveniences are well worth the chance to live and be cancer free.

I wear sunscreen every single day, and the days of lying out in the sun are long gone. But I am embracing my skin and don't take healthy skin for granted.


.

MRA saw a 35 percent increasein social media followers in 2011.

http://www.melanomaresearchalliance.org/about_melanoma/about.taf?cat=stories

yielded $5 million, all of which is

funding transformative melanoma

research programs. The dinner

featured a live auction led by Jamie

Niven, Sotheby’s chairman of North

and South America, an exclusive

preview of Impressionist and Modern

Art, and the opportunity to meet with some of the most

forward-thinking minds in cancer research. A highlight of

the auction came when supporters pledged sponsorship

for nine Young Investigator awards, nearly doubling the

number of these awards funded by MRA to date.

Wendy Selig hosted a panel that featured (left to right) Dr. JonathanSimons, president and CEO of theProstate Cancer Foundation, Dr. JohnMendelsohn, president of The Universityof Texas MD Anderson Cancer, GregoryLucier, chairman and CEO of LifeTechnologies, Jill Kargman, author of"Sometimes I Feel Like a Nut,” and Amy Harmon, Pulitzer Prize-winningreporter for The New York Times

14 www.curemelanoma.orgOutreach


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At the Sotheby’s benefit dinner, Dr. Padmanee Sharma discusseshow her MRA Young Investigator Award advanced her researchto better treat melanoma patients..

Outreach

MRA leadership reaches out to a broad audience through

conferences and panel discussions that focus on

melanoma and lessons from this field that can be applied

to other areas of medical research. For example, the 2011

Milken Institute Global Conference included an MRA

panel, "Exploring the Frontiers of Medicine: How We Will

Save Lives from Cancer.” The session was moderated

by Wendy Selig, MRA’s president and CEO. The panel

used melanoma as a case study to discuss innovative

approaches to cancer research and treatment.

MRA also expands its reach by hosting gatherings

across the country throughout the year to engage new

people and elevate the importance of melanoma

among various audiences.

Patient advocates, philanthropists,

scientists, and Corporate Allies

convened for the first annual MRA benefit dinner at

Sotheby’s in New York City on Oct. 28, 2010. The event

MRA 2008-2011Outreach 15

YOUNG INVESTIGATORS INCREASED A highlight of the Sotheby's eventauction came when supporterspledged sponsorship for nineYoung Investigator awards, nearlydoubling the number of theseawards funded by MRA to date. 10 19

Design Center. MRA Young Investigator, Dr. Roger Lo

from UCLA, gave a brief overview of his research and how

MRA has dramatically improved the field for young

doctors and researchers. The luncheon concluded with

board member, Maria Bell, sharing her personal experi-

ences with melanoma. The event introduced over 150

women in the Los Angeles area to the MRA and raised

critical funds for research.

In December

2010, Cartier's

president and CEO, Emmanuel

Perrin, and vice president of

retail, Mercedes Abramo, co-

hosted a holiday celebration

with MRA chair and co-founder,

Debra Black, and MRA board

member, Susan Hess, honoring

MRA. The event was held at the

Cartier Mansion in New York

City and was a spectacular evening. In addition to the

many events Cartier hosts in honor of MRA, the company

also supports Dr. Remco van Doorn at Leiden University

Medical Centre, through the Cartier—MRA Young

Investigator Award.

Christie's is a long term MRA partner

and collaborator. The company has

engaged with MRA in a variety of

awareness and fundraising initiatives including support

of Dr. Christian Blank at the Netherlands Cancer

Institute through the Christie's —MRA Young

Investigator Award.

A highlight of this collaboration came as Chairman of

Christie's Americas, Marc Porter joined with Wells Fargo

to co-host the first official Los Angeles showing of the

traveling Elizabeth Taylor exhibit in April 2011. The exhibit

featured Ms. Taylor's jewelry collection and was followed

by a luncheon in honor of MRA at the MOCA Pacific

MRA co-founder Debra Black (right) and Board Member Maria Bellattend the Christie's LA luncheon in honor of MRA.

Debra Black, MRA co-founderand chair, and EmmanuelPerrin, Cartier's president andCEO, at Cartier's 2010 holidaycelebration honoring MRA.

Outreach

DIAGNOSISAND STAGING

While most earlystage melanomasare cured with surgery, some recurand metastasizeeven after removalof the primary tumor.MRA EstablishedInvestigator Dr.Lynda Chin (then at Dana-Farber CancerInstitute, now at MDAnderson CancerCenter) identified six “proinvasion oncogenes” that are signifi-cantly overexpressed in metastatic lesions compared to primarymelanomas in preclinical models. The protein expression level ofone of them, called APC5, was correlated with shorter survivalof melanoma patients and, thus, may be a prognostic biomarkerin human melanomas. This work is being clinically developed bya company to build prognostic tests based on the molecularcharacteristics of early stage melanoma.

16 www.curemelanoma.orgLooking Ahead


.

Although melanoma poses many

difficult challenges, MRA and its

funded researchers are making

significant progress toward develop-

ing better diagnostic and preventive

measures, improved treatments,

and a deeper understanding of this

complex and deadly disease.

The recent advances in the development of

new therapeutic approaches have generated

excitement and optimism for a better outlook

for melanoma patients. With the significant

infusion of research funding from MRA,

investigators are finding ways to improve upon

these results as well as create new strategies

to combat melanoma. MRA seeks to engage all

stakeholders in working toward a day when no

one suffers or dies from melanoma.

Looking Ahead

MOLECULARLY TARGETED THERAPY

Approval of vemurafenib, an inhibitor of mutant BRAF involved in about half ofmelanomas, is a major breakthrough. While this drug has benefitted some patients,most relapse due to drug resistance. Research suggests that secondary mutationsin BRAF are not a common cause of resistance to BRAF inhibitors, but that multipleother mechanisms may be playing a role. Melanoma cells have additional ways to survive and grow, and emerging research has identified mutations and alterationsin genes and proteins involved in resistance. It is likely that others remain to beidentified and confirmed in patients. An MRA Team Science Award acceleratingresearch to solve this problem is being led by Drs. David Solit and Paul Chapman(Memorial Sloan-Kettering Cancer Center), Hensin Tsao, David Fisher, and KeithFlaherty (Massachusetts General Hospital), Michael Davies (MD Anderson CancerCenter), Roger Lo (University of California, Los Angeles), Katherine Nathanson

(University of Pennsylvania),and Jeffrey Sosman(Vanderbilt University).

ACP5 protein expression is greatest in melanomametastases compared with primary melanomasand benign nevi.

BRAF mutant melanoma cells have highly altered genomicprofiles, including loss of a tumor suppressor gene calledPTEN on chromosome 10 (white=copy number neutral;blue=copy number loss; red=copy number gain)

Benign Nevus PrimaryMelanoma

Melanoma Metastase

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MRA 2008-2011Financials 17

Financials(as of Dec. 31, 2010)

Thanks to the generous ongoing

support of its founders, 100 percent

of public donations to MRA directly

support melanoma research.

The independently audited financial

statements of Melanoma Research Alliance

Foundation form the basis for the following

information.

Assets 12/31/2010 12/31/2009 12/31/2008

Cash and Cash Equivalents $6,661,152 $2,228,522 $110,296 Investments - - 2,878,106 Accounts Receivable 55,025 - - Contributions Receivable 9,468,362 - - Prepaid Expenses and Other Assets 517,846 10,000 - Property and Equipment (Net) 5,395 5,116 -

Total Assets $16,707,780 $2,243,638 $2,988,402

Liabilities and Net Assets

Liabilities: Accounts Payable and Accrued

Liabilities 59,716 54,478 67,921 Due to Affiliate 10,281 291 286,735 Deferred Revenue 25,000 - - Deferred Rent 9,680 10,057 -

Total Liabilities 104,677 64,826 354,656

Net Assets:Unrestricted 6,249,741 2,178,812 2,633,746 Temporarily Restricted 10,353,362 - -

Net Assets 16,603,103 2,178,812 2,633,746

Total Liabilities and Net Assets $16,707,780 $2,243,638 $2,988,402

2010 FUNCTIONAL EXPENSES

Non-Grants ScientificProgram

5.7%

Managementand General

3.6%

Fundraising2.1%

Grants and Awards Program

88.6%

18 www.curemelanoma.orgFinancials


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Financials(as of Dec. 31, 2010)

Statement of Activities

Revenues, Public Support 2010 2009 2008 and Other Income

Contributions (including Founders’ Gift) $18,563,318 $6,533,950 6,001,150 Special Events 5,025,867 - - Investment Income (Loss) (Net) 25,642 8,718 106,106

Total Revenues, Public Support 23,614,827 6,542,669 6,107,256 and Other Income

Functional Expenses:

Program Services: Grants 8,668,371 6,828,183 3,423,577

Fundraising 194,989 - - Management and General 327,176 169,420 49,933

Total Functional Expenses 9,190,536 6,997,603 3,473,510

Change in Net Assets 14,424,291 (454,934) 2,633,746

Net Assets - Beginning of Year 2,178,812 2,633,746

NET ASSETS - END OF YEAR 16,603,103 2,178,812 2,633,746

Board of Directors

• Debra Black, Chair, MRA Co-Founder, Broadway Producer, Melanoma Survivor

• Leon Black, MRA Co-Founder, Board Member Faster Cures, CEO Apollo Management LP

• Maria Bell, Head Writer and Co-Executive Producer/Head Writer, The Young & the Restless

• Jason Federici, Art Director, Graphic Designer, Photographer

• Jami Gertz, TV and Film Actress• Susan Hess, Vice Chairman, Whitney Museum• Michael Klowden, President and CEO,

Milken Institute• Connie Mack, Senior Policy Advisor, Liberty

Partners Group LLC; Former U.S. Senator• Nancy Marks, Artist• Michael Milken, Chairman, Milken Institute;

Founder and Chairman, Faster Cures; Founder and Chairman, Prostate Cancer Foundation

• Richard Ressler, Founder, Orchard Capital Corporation and CIM Group

• Henry Silverman, Non-executive Chairman of the Board of Directors, Realogy Corporation; Senior Advisor, Apollo Global Management; Former Chairman and CEO, Cendant Corporation

• Greg Simon, SVP Worldwide Policy, Pfizer, Inc.• Jonathan W. Simons, M.D., President and CEO,

David H. Koch Chair, Prostate Cancer Foundation; Professor, Biomedical Engineering and Material Sciences, Georgia Institute of Technology, NCI Center of Cancer Nanotechnology Excellence

• Jonathan Sokoloff, Managing Partner, Leonard Green and Partners, L.P.

• Elizabeth Stanton, President, Elizabeth and Oliver Stanton Foundation

• Wendy Selig, President and CEO, MRA• Margaret Anderson, Secretary

Executive Director, Faster Cures• Kamyab Hashemi-Nejad

Treasurer, Director of Finance, Milken Institute

Scientific Advisory Panel

• Elizabeth A. Grimm, M.D., Frances King Black Memorial Professorship of Cancer Research and Deputy Chair, Department of Experimental Therapeutics, The University of Texas MD Anderson Cancer Center

• Martin Mihm, M.D., Director, Melanoma Program in Dermatology, Brigham and Women's Hospital; Associate Director, Melanoma Program, Dana-Farber/Brigham and Women's Cancer Center

• Donald Morton, M.D., Chief, Melanoma Program; Director, Surgical Oncology Fellowship Program, John Wayne Cancer Institute

• Neal Rosen, M.D., Ph.D., Enid A. Haupt Chair in Medical Oncology, Memorial Sloan-Kettering Cancer Center

• Steven Rosenberg, M.D., Chief, Surgery Branch, National Cancer Institute (Dr. Rosenberg serves on the MRA Scientific Advisory Panel in his personal capacity)

• Wendy K.D. Selig, President and CEO, MRA• Suzanne Topalian, M.D., Professor of Surgery and

Oncology, Johns Hopkins Medicine; Director, Melanoma Program, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University; Chief Science Officer, MRA

Grant Review Committee

• Jonathan Cebon, M.B.B.S., F.R.A.C.P., Ph.D., Director of Medical Oncology, Austin Health, Head Cancer Vaccine Laboratory, Ludwig Institute for Cancer Research, Professor of Medicine, University of Melbourne

• Meenhard Herlyn, D.V.M., D.Sc., Professor of Dermatology, Pathology and Laboratory Medicine, University of Pennsylvania; Chairman, Molecular and Cellular Oncogenesis Program, Wistar Institute

MRA 2008-2011Committees 19

20 www.curemelanoma.orgCommittees


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• James Allison, Ph.D., Chair of Immunology Program; Director of Ludwig Center for Cancer Immunotherapy, Investigator, Howard Hughes Medical Center, Memorial Sloan-Kettering Cancer Center

• Boris Bastian, M.D., Clinical Professor, Department of Dermatology, University of California, San Francisco

• Marcus Bosenberg, M.D., Ph.D., Associate Professor of Dermatology and Pathology, Yale School of Medicine

• Steven Burakoff, M.D., Professor of Medicine, Hematology and Medical Oncology, Professor, Oncological Sciences, Mount Sinai School of Medicine

• Paul Chapman, M.D., Attending Physician, Melanoma/Sarcoma Service, Memorial Sloan-Kettering Cancer Center; Professor of Medicine, Weill Medical College of Cornell University

• Tanja de Gruijl, Ph.D., Associate Professor, VU University Medical Center

• Charles Drake, M.D., Ph.D., Associate Professor - Oncology, Immunology and Urology; Director - Multidisciplinary Prostate Cancer Clinic, Johns Hopkins Sidney Kimmel Comprehensive Cancer Center

• Glenn Dranoff, M.D., Director, Human Gene Transfer Laboratory Core, Dana-Farber Cancer Institute

• David Fisher, M.D., Ph.D., Chief, Department of Dermatology; Director, Melanoma Program, MGH Cancer Center; Director, Cutaneous Biology Research Center, Massachusetts General Hospital; Edward Wigglesworth Professor of Dermatology, Harvard Medical School

• Allan C. Halpern, M.D., Chief, Dermatology Service, Memorial Sloan-Kettering Cancer Center

• Thomas Hornyak, M.D., Ph.D., Chief, Dermatology, VA Maryland Healthcare System; Associate Professor of Dermatology and Biochemistry and Molecular Biology, University of Maryland School of Medicine

• Michal Lotem, M.D., Sharett Institute of Oncology, Hadassah Hebrew University Hospital, Kiryat Hadassah

• Richard Marais, Ph.D., Professor of Molecular Oncology, The Institute of Cancer Research

• Kim Margolin, M.D., Physician, University of Washington/Seattle Cancer Center

• Glenn Merlino, Ph.D., Chief, Laboratory of Cancer Biology and Genetics, National Cancer Institute,

• Stan Riddell, M.D., Member, Department of Immunology, Fred Hutchinson Cancer Research Center

• Jonathan Simons, M.D., President and CEO, David H. Koch Chair, Prostate Cancer Foundation

• David Solit, M.D., Assistant Member, Human Oncology and Pathogenesis Program, Memorial Sloan-Kettering Cancer Center; Elizabeth and Felix Rohatyn Chair for Junior Faculty

• Howard Soule, Ph.D., Executive Vice President, Discovery and Translation, Prostate Cancer Foundation

• Suzanne Topalian, M.D., MRA Chief Science Officer,Professor of Surgery and Oncology, Johns Hopkins Medicine, Director, Melanoma Program, Sidney Kimmel Comprehensive Cancer Center

• Michael Weber, Ph.D., Director, Cancer Center, Weaver Professor of Oncology, University of Virginia

Donors* Multi-year support

$500,000 and Above---Akin Gump Strauss Hauer & Feld LLP*The Jeffrey A. Altman Foundation*Anonymous*Debra and Leon Black* Gagosian Gallery Rahr Enterprises*Jami Gertz and Tony Ressler*The Ressler Family Foundation*Sokoloff Family Trust*

$250,000-$499,999---The Carson Family Charitable Trust*Credit Suisse*Ellen and Gary Davis Foundation*Caryl and Israel Englander O'Melveny & Myers LLP PricewaterhouseCoopers*Elizabeth and Oliver Stanton Foundation*US Trust/Bank of America*Wachtell, Lipton, Rosen & Katz*

$100,000-$249,999---Donna and William Acquavella Aon Risk Services*Katie and Todd Boehly*Sunny and Norman Brownstein Brownstein, Hyatt, Farber & Schreck*Cartier Christie's Deloitte*Hess Foundation, Inc. Latham & Watkins, LLP*Susan and Martin Lipton Nancy and Howard Marks Montage Hotels & Resorts Morgan Stanley*Jane and Daniel Och Family Foundation Lizanne and Barry Rosenstein The Lawrence and Carol Saper Foundation, Inc.*Sony Music Merryl and James Tisch

$50,000-$99,999---Barclays CapitalCiti Global BankingErnst & Young LLP*Larry GagosianGeneral Nutrition CorporationJudy and John HannanGail and Carl IcahnJPMorgan Chase & Co.The LeBow FamilyJane Goldman and Benjamin LewisMintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.E.*Paul, Weiss, Rifkind, Wharton & Garrison LLP*Jenny and John PaulsonSkinCeuticalsSusan and Eric SmidtSotheby'sKatherine and Jerry SpeyerThe Thompson FamilyAndrew H. & Ann R. Tisch FoundationThe Winnick Family Foundation

$25,000-$49,999---Debbie and Mark AttanasioEmily and Len BlavatnikThe Brown Foundation, Inc.Kathryn and Kenneth ChenaultCiti Private Wealth ManagementSir Ronald Cohen and Sharon Harel-CohenSusan and Peter EvensenFribourg Family FoundationGenentech, Inc.The Perry & Martin Granoff Family Foundation, Inc.The Joshua & Marjorie Harris Family FoundationKristy and Robert HarteveldtSuzette and Steven KolitchMarie Josee & Henry R. Kravis FoundationJo Carole and Ronald LauderRichard S. and Karen LeFrak Charitable Foundation, Inc.Live4Life Foundation, Inc.Janet Wallach and Robert MenschelMyriam Barenbaum and Jonathan F. MillerMorgan, Lewis & Bockius, LLPKate and Robert NiehausSandy and Paul NormanPfizer Inc.The Raiff FoundationDaryl & Steven Roth FoundationRowan Family Foundation, Inc.Lily SafraAndree and Howard ShoreTG Holdings, Ltd.UBS Financial ServicesThe Viola FundWagner Family Foundation

MRA 2008-2011Donors 21

22 www.curemelanoma.orgDonors


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Leslie and Daniel Ziff$5,000-$24,999---AnonymousLaura AnrederRenee and Richard BaraschBaron Capital FoundationDror Bar-ZivThe Bell Family Foundation, Inc.Milton BerlinskiJill and Jay BernsteinBarbara and James BlockAlison Mass and Sal BommaritoThe Daniel & Estrellita Brodsky Family FoundationChristian DiorChantal and Stephen CloobeckJoyce and Barry CohenConcerts East, Inc.Vanessa and Henry CornellJim Ehrlich Memorial Family Foundation, IncPamela and Jimmy FinkelsteinSusan and Richard FriedmanAndrew GnGolden Door SpaAudrey and Martin GrussMimi and Peter Haas FundAshley Leeds and Christopher HarlandHenry Crown and CompanyHerring Finn FoundationRonnie F. HeymanCaroline P. Hirsch FoundationHudson NewsAdam KalishRobin and Joel KassimirGeorge KleinEvelyn and Leonard LauderJames B. Lee, Jr.Lorber Charitable FundLuxembourg DayanMerkin Family FoundationKen & Julie Moelis FoundationMargo and Jimmy NederlanderThe New York Community TrustNortheast Theater GroupPhRMALeon Polsky and Cynthia Hazen PolskyPeggy and Gary ReinerThe Reiss Family FoundationJonathan ResslerJacqueline and Mortimer SacklerMary and David SolomonThe Thomas Spiegel Family FoundationAmbassador Carl Spielvogel and Dr. Barbaralee Diamonstein-SpielvogelRuth StantonAlison and Leonard SternBeatrice SternThe Sternlicht Family Foundation, Inc.Jennifer and David Stockman

Dana Hammond and Patrick StubgenBarbara and John VogelsteinGregg S. WeinsteinBrigitte and Alain WertheimerPatty Newburger and Brad WechslerChef Michael White

$1,000-$4,999---A G FoundationAnn and Steven AmesJudith and John AngeloAnonymousAnonymousAnthony AufieroSusan and William BelfioreBiotechnology Industry Organization (BIO)BRELLIMichele and Martin CohenRobert de RothschildJean DoumanianPhillip DunnTherese Wareham and Merrick ElfmanJason FedericiOlivia and Adam FlattoBruce GlassmanGoldberg/Nash Family FoundationDeborah and Allen GrubmanHard Rock Cafe Foundation, Inc.The Hedges FamilyHuberfeld Family FoundationJanklow FoundationLiz Robbins and Doug JohnsonSusan Scott and Robert J. KaufmanMitchell KlineLA Smooch LLCJoel Lawson, IVNinah and Michael LynneMadison CoxRoxanne and Andrew MajkaMichael MargitichMizel International Cultural FundCarol MorganThe Daniel M. Neidich and Brooke Garber FoundationThe New York Community TrustJill and Mark Rachesky Charitable FoundationDenise RadeskyRedburn Partners USA LPEllen and Bruce ResslerFiona and Eric RudinJane and Ned SadakaDenise and Andrew Saul FoundationSchlosstein-Hartley Family FoundationJean and Martin ShafiroffMia and Sheldon SolowSource of Hope FoundationJane and James SpingarnPamela Sztybel and Elliott Stein

Thomas D. & Denise R. Stern Family FoundationMatthew StopnikFern and Lenard TesslerC. George van Kampen FoundationLauren and John VeronisMarla and Craig WassermanCarol & Michael Weisman Family Charitable TrustWellNestConstance and Sankey WilliamsLucy Halperin and Jerold ZaroMarcie and Howard Zelikow

SupportersAltamarea GroupAnreder & CompanyAxcess Luxury & LifestyleBirds Nest FoundationBloomberg LPCasa DragonesChapstickChâteau d'AussièresCIM Group Inc.Danny FundEntertainment Industry FoundationFasterCuresPatricia and Michael KlowdenLos Angeles County Museum of ArtLux Capital Management, LLCMarissa Alperin StudiosThe Milken InstituteLori and Michael MilkenMuseum of Contemporary Art, Los AngelesNGK GlobalSirius Satellite RadioBruce Springsteen & the E Street BandThe Style NetworkTribeca EntertainmentDavid Yurman

Staff• Wendy K.D. Selig, President and CEO• Suzanne Topalian, M.D., Chief Science Officer• Laura M. Brockway-Lunardi, Ph.D.

Scientific Program Director• Lauren C. Leiman, Development Director• Karen Rogers, Communications Manager• Ilyona Carter, Executive and Projects Assistant

MRA 2008-2011Support & Staff 23