Expanded Access Programs: A Time of Amazement in Melanoma

Immunotherapy Science Mag CoverFor those of you keeping a close eye on the field of melanoma treatments, you know that we find ourselves living in a simply amazing period of opportunity and innovation—even though there is still a long way to go.

The creation of new cancer drugs is pharmaceutical alchemy, transmuting a base idea into a life-prolonging new treatment that is both safe and effective.  While not magic, drug development is an expensive, incredibly complex and time-consuming process.  And when it works well—when there are exceptionally promising treatments near at hand—the wait for full availability to those agents can be frustrating beyond measure.

Such is the case right now with certain immunotherapy drugs that stimulate the patient’s immune system to recognize and attack cancer.  The data with these immunotherapies targeting the PD-1/PD-L1 axis (anti-PD-1 drugs) is so encouraging that three have earned U.S. Food and Drug Administration (FDA) Breakthrough Therapy designation* which aims to speed FDA approval of the most promising drugs.

This is fantastic, but how are promising drugs made available to patients in the pre-approval period?

To accelerate the accessibility of new drugs to patients, drug developers may open so-called Expanded Access Programs (EAP).  EAPs are compassionate use programs whereby drugs are made available to patients before they are fully approved for sale by the FDA.  EAPs consume resources from treatment centers and drug companies alike, but EAPs endeavor to do the right thing to help patients before drug approval and sale. MRA is proud to have been part of the conversation with companies, clinicians and the FDA that encouraged and facilitated the opening of the anti-PD-1 drug EAPs.

So, why talk about EAPs and anti-PD-1 drugs right now?

Because melanoma is once again a case study for the cutting edge. Presently, three EAPs for melanoma are open with similarly targeted agents (the anti-PD-1 antibodies). Merck’s pembrolizumab was the first EAP to open for melanoma in March 2014, followed by BMS’ nivolumab EAP in May.  Most recently, in July, BMS launched an EAP for the combination of nivolumab with its anti-CTLA4 drug, ipilimumab.  This may not sound like much, but to have three EAPs at the same time for the same cancer is unprecedented.  To have this happen with the same type of drug is nothing short of amazing!

While anti-PD-1 treatments don’t work for everyone and do have certain side-effects, MRA and others are funding research to overcome these limitations.  Nevertheless, the demonstrated progress against melanoma fuels the hope that we’ll look back to 2014 and say, “That was the year everything changed.”

About the Author

Louise Perkins HeadshotLouise M. Perkins, Ph.D., joined the Melanoma Research Alliance (MRA) as Chief Science Officer in 2013 where she is responsible for the development and implementation of MRA’s scientific strategy.  Her interests center on translational research with specific concentration on genomics, drug discovery and the advancement of novel therapeutic approaches. Prior to joining MRA, she was Chief Scientific Officer at the Multiple Myeloma Research Foundation (MMRF) for five years following a research career of 16 years at two major pharmaceutical companies.

*Note: Merck’s anti-PD-1 pembrolizumab received Breakthrough Therapy designation for melanoma in May 2013; BMS’ anti-PD-1 nivolumab received Breakthrough Therapy designation for Hodgkin Lymphoma in May 2014; Genentech/Roche’s anti-PD-L1 MPDL-3280A received Breakthrough Therapy designation for bladder cancer in June 2014.

Immunotherapy & Awareness: Fighting Melanoma on Two Fronts

MRA is saddened by the news that Mark Carter lost his battle with melanoma this week.  We send our condolences to his family and friends.   He had hoped that sharing his story would educate and raise awareness of this deadly disease.    

I first went to see my doctor in 1999 after my wife’s prompting.  She had noticed a mole on my lower right abdomen that had changed shape and wanted me to get it checked.  My doctor told me it was nothing to be concerned about and sent me home.  The mole continued to grow and again my wife told me to go see the doctor.  I did and this time he agreed to remove it.

A week later, my doctor rang me at work and told me that it was malignant melanoma.  Both my wife and I were totally devastated by this news.

Things were a bit of a blur after this, but I had a wide-level excision and regular check-ups for 10 years after surgery.  Finally, I was discharged from the hospital feeling incredibly relieved that the disease had not recurred.

In April 2012, I woke up one morning with a painful lump under my left armpit and the alarm bells started ringing in my head.  I saw my doctor who said he was 80% sure it was an infected lymph node but could not rule out melanoma due to my past history.  The lump never improved, so it was removed by surgery and I was told that the melanoma had returned.  I was now stage IIIC.

The first CT scan taken within three months of my surgery showed that my disease had not been contained, so I was put on the BRAF inhibitor treatment, vemurafenib.  I was now a stage IV terminal patient.  Three months later, the next scan revealed that I had shown a complete response to this amazing drug.  Unfortunately, 13 months later the disease came back.  Vemurafenib is only a life prolonger, not a cure, and resistance usually occurs.

Finally, I was put on the immunotherapy drug, ipilimumab, and a few weeks ago I finished my last infusion.  My next scan will show if I am a responder or not, but even if I do fail to respond I am lucky enough to have been offered the chance to take the new anti-PD-1 drug, nivolumab, under the expanded access scheme in the UK.

Although I had 13 years clear of the disease, I now count my lucky stars that there are treatments available that have kept me alive for 18 months.  I truly believe there has been a huge paradigm shift in melanoma treatments, particularly in the field of immunotherapy.  In maybe five years’ time, melanoma will no longer be a terminal disease but more of a chronic illness.

I now devote most of my time to raising awareness of the risk factors and causes of melanoma.  Still far too many people are ignorant of the dangers of excessive UV exposure.  I believe that education is the key to increasing awareness and ultimately reducing the recent explosion in melanoma incidence.

 

Mark and Liz Carter edit

About the Author

Mark Carter, 56, lived in the United Kingdom with his lovely wife Liz.  He was a UK-based melanoma awareness advocate and anti-sunbed campaigner who worked to raise awareness of this deadly disease.  You can learn more about Mark’s journey and his advocacy work on his blog: http://www.melanomaman.co.uk/

 

 

An Outdoor Enthusiast’s Guide to Playing It Sun Safe

divingoffboatsmaller

Johnie Gall || DirtbagDarling.com

Over the past few years, “fear” has become something of a challenge in my vocabulary. I went from someone who was scared to try surfing to someone who lives out of a revamped Dodge Sprinter van traveling the country in search of adventure. I’ve been fortunate enough to surf in Hawaii, to hike the highest peaks in Colorado, to snorkel with sharks in the Florida Keys, and to free rappel 200 feet from an arch in the middle of the Utah desert.

That’s not to say I’m fearless—there are many things that still frighten me about spending so much time in the outdoors. Bears. Falling. Broken limbs. Getting lost. Melanoma.

Yes, melanoma is a very real consideration of everything I do—though you might not believe me judging my criss-cross lattice of tan lines and premature wrinkles. Tan happens, especially when you spend the majority of your day outdoors (all the sunscreen in the world won’t change that), but so does melanoma, and I’ve chosen not to be so bold as to think it won’t happen to me. That’s why protecting my skin has become as much a part of my adventure prep as loading up my backpack and buying spare fuel.

Don’t get me wrong—I wasn’t always so cautious about skin cancer. Flashback to high school and you’d find me in a tanning booth prepping for prom and roasting at the beach with my friends. I thought hiking was synonymous with sports bras and fishing meant donning nothing more than a bikini. I actually shake my head thinking of the damage I did, but like they always say, hindsight is 20/20.

That lifestyle came to screeching halt when I took my first trip to the dermatologist in my late teens—I had a mole that looked suspicious, and my doctor wanted it off. After the biopsy, he told me it was benign. The danger was over, but the shock that something I’d always (foolishly) thought could never happen to me was actually happening was still there. It was a huge wake-up call, but I was lucky.

After my initial scare, I know that skin cancer prevention begins long before the threat becomes deadly and these days, when being outside is part of my job, I know that shielding my skin doesn’t have to mean sacrificing my active lifestyle—it just means getting creative. Here’s what I do to stay protected:

Sunscreen: Because I spend a lot of time in the water, I need a screen that won’t harm the coral reefs or marine animals when it washes off. I never leave the house without at least coating my hands, feet and face with SPF 30, and follow up with a water resistant one all over my body as soon as we start any activity.

UPF Clothing: How genius is sun protective clothing? It’s one of the first things I look for in my outdoor clothing—the good companies always make their sweat-wicking shirts and pants with UPF 15 or more. When in doubt, I slather on a layer of sunscreen under my clothing, too.

In the water: I rarely go swimming in the ocean without a rash guard—but long gone are the days when donning a quick-drying shirt meant a men’s style tee or neon monstrosity. I’m lucky enough to have a few friends who are at the helm of swimwear companies aimed at protecting skin, so surf leggings and rash guards are always in my bag or stashed in the trunk of my car.

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Giant. Hats: Here’s the great thing about wearing hats—you never have to worry about what your hair looks like. I can go without a shower for a week (something I often have to do living out of a van) and no one is any the wiser. I stock up on lifeguard-style straw hats at the flea market for summer and keep a collection of wool beanies, baseball caps and floppy felt hats in my closet for the colder months.

And if there’s one thing everyone should buy, it’s a white fishing shirt (yes, even if you hate fishing). They are light, airy, and dry like lightening. Dunk them in the water to cool off on boat rides, or wear them over your hiking clothes on hot days.

Most importantly, I’ve learned to find ways to stay out of the sun. My philosophy is this: being outside is part of my life. It always has been. It always will be. Tan will happen, but as long as I’m making every effort I can to stay safe, then I won’t have any regrets (and hopefully a healthy and happy skin suit!).

 

About the Author: Johnie Gall is the founder of DirtbagDarling.com, an online magazine for women that aims at inspiring and educating women of all skill levels on how to make the most of their outdoor experience. She’s a writer and a creative consultant who calls Pennsylvania home base (but you’re more likely to find her traveling the country in her Dodge Sprinter turned RV).

 

Partners in Prevention: MRA and The Surgeon General’s Call to Action to Prevent Skin Cancer

Two years ago, the Melanoma Research Alliance (MRA) met with Dr. Howard Koh, the Assistant Secretary for Health at the Department of Health and Human Services (HHS), and asked the question that launched a milestone project to elevate melanoma and skin cancer on the agenda for public health: “How can MRA do more in partnership with government health agencies in the fight against skin cancer and melanoma?”

SG Call to Action - Lushniak Koh Selig

Dr. Howard Koh, Wendy Selig and Dr. Boris Lushniak

With his medical background in oncology and dermatology, Dr. Koh needed no convincing about the importance of this cause given the dangers of skin cancer and the public health imperative for a coordinated national prevention strategy. To catalyze development of a national health agenda in the fight against skin cancer and melanoma, Dr. Koh and MRA convened a meeting of the leaders of all the relevant agencies within the U.S. Public Health Service, including the Office of the Surgeon General, the National Institutes for Health (NIH), the Centers for Disease Control (CDC), the U.S. Food and Drug Administration (FDA) and the Agency for Healthcare Research and Quality.

Government officials, medical experts, researchers, and thought leaders joined together 22 months ago for this important gathering and an incredible effort of collaboration began.

Fast forward to July 2014: yesterday with MRA’s President & CEO Wendy Selig in the front row, Dr. Koh and Acting Surgeon General Dr. Boris Lushniak, himself a dermatologist, unveiled The Surgeon General’s Call to Action to Prevent Skin Cancer. The Call to Action seeks to engage all levels of government as well as individuals, private sector institutions and organizations in a coordinated, multifaceted effort to prevent skin cancer.

The Call to Action is the most recent and final achievement in Dr. Howard Koh’s five years as a public servant before he returns to the faculty at the Harvard School of Public Health. MRA thanks Dr. Koh for his tireless work, leadership and advocacy of disease prevention and is honored to be recognized by him as one of the leading “partners in prevention” to instigate a national public health agenda to fight skin cancer and melanoma.

The Surgeon General has sounded the call for the nation to join together to fight this terrible but preventable cancer. The time for action is now.  Join us in the fight against skin cancer and melanoma!

Melanoma Awareness From a High Schooler’s Perspective: Where We Are and Where We Need To Be

Not many teenagers know that melanoma is the number one new cancer diagnosed in young adults ages 25-29. It is also difficult to believe that as with many mistakes made at a young age, neglecting sun safety during childhood and adolescence can harm you later in life.

Most of my peers would never dream of lighting a cigarette, yet few would think twice about laying out unprotected at the pool or beach.  Luckily, people are beginning to understand the dangers of indoor tanning, appropriately likening tanning beds to coffins. In previous years, girls would go to tanning beds before school dances. They now settle for a safer (but orange) alternative: spray-tans. Skin cancer awareness has increased, sadly due to the rise in its prevalence, but there is still a ways to go. Most people I know have yet to realize that unprotected sun exposure is just as deadly as a tanning bed.

They're called artificial sun for a reason

They’re called artificial sun for a reason

My paler friends are the most vigilant about sunscreen use. Not one of them wants to get sunburned, so they’re sure to use (and re-apply!) broad-spectrum, high-SPF sunscreen to protect themselves. They do this to escape short-term effects of the sun, but rarely think about the sun’s capability for long-term damage. They are correct in practice, but lack part of the motive for their protective actions. Maybe this is why the occasional girl will still come home from spring break bright red.  They don’t know the danger and how one blistering sunburn in childhood or adolescence doubles their risk for melanoma.

Many of my friends have olive-toned skin, like I do, that tans easily and rarely burns. It is hard for people with this skin type to think twice about going to the beach and getting a dark tan.  Ironically, we are quick to criticize our fair-skinned friends when they get badly sunburned, but the truth is everyone is at risk for melanoma, the most dangerous form of skin cancer.

Tanning is a sign of our body’s response to damage caused by harmful UV rays. Without sunscreen, UV rays from the sun penetrate the skin and damage DNA. Cells called melanocytes can begin to grow uncontrollably because of change in their genetic makeup, and melanoma (skin cancer of melanocytes) develops. Many people do not see skin cancer as a big deal. It is often thought of as a spot that can be removed and forgotten. However, this is not the case with melanoma, which makes up only 4% of skin cancer cases but 80% of skin cancer deaths. Melanoma spreads extremely rapidly and if not caught and treated early on, will spread to the lymph nodes and vital organs. Stage 0 or 1 melanoma has a 90% cure rate, while Stage 4 melanoma patients have a median life expectancy of less than a year. If more people knew these facts, the melanoma death rate could decrease significantly.

What does the future hold for melanoma awareness? Teenagers have already progressed to the point where most of us avoid tanning beds. The next step is to be better at protecting ourselves from the sun, even though it means giving up tanning in favor of sunscreen, healthy skin, and a melanoma-free life. If we can be truly and completely aware of the sun’s hazards, we can motivate ourselves and others to eradicate this aggressive but completely preventable disease.

About the Author

abouttheauthorI’m Isabella Todaro, a rising junior at Georgetown Visitation in Washington, D.C. I have spent this past week as a volunteer at MRA and learned a lot in the process. My cousin has been working here for two years, so I was already interested in the organization before I decided to volunteer. The experience has been great, as I have learned information that otherwise might have remained unfamiliar to me. Like many people, I used to think that getting sunburned, but not getting a tan, puts people at risk for melanoma. Now I know that both of these dangerous behaviors are risky. After learning this and so much more at MRA, I intend to practice sun safety and let others know why they should, too.

Melanoma News Round-Up, July 12

Hello everyone! We’re back to bring you the news highlights for all things melanoma and MRA.

JCrew 1

In celebration of UV Safety Month this July, J.Crew will donate $1 to MRA for every Instagram upload showing how you cover up and have fun in the sun.  Just tag it #JCrewSmartSun and help win donations for MRA.  Thanks to the generosity of our founders, 100% of funds raised will go directly toward melanoma research.  Stay sunsafe and help fund cutting-edge research – it’s a win-win!

This week marked game-changing progress for melanoma treatment as regulators in Japan granted the world’s 1st approval of the anti-PD-1 drug, nivolumab.  News also broke that Bristol-Myers Squibb will be seeking FDA approval of nivolumab, making this breakthrough immunotherapy available in the United States months ahead of schedule.

MRA also hailed the launch of an Expanded Access Program for advanced melanoma patients to receive a combination therapy of ipilimumab and nivolumab.  This method of combination therapy has shown promising results for late-stage melanoma patients.

On the prevention front, MRA is delighted to report Hawaii became the 10th state to ban indoor tanning for minors under 18 years of age.  There is much more work to be done to reduce everyone’s risk for skin cancer and melanoma, but the passage of this bill is a step in the right direction.

You don’t want to miss this great interview on the dangers of sun exposure and skin cancer with dermatologist, Dr. Mitchell Kline, by legendary PIX11 broadcaster, Marvin Scott.

GENlogoCMYK_092313Finally, we’re proud to share that MRA was ranked the #1 Grant-Giving Disease Foundation by Genetic Engineering & Biotechnology News (GEN).  As always, we are dedicated to finding a cure for melanoma and working toward a day when no one will suffer or die from this disease.

 

Melanoma News Round-Up, June 21

Hello everyone!  We hope you had a wonderful week and you’ve been staying sun safe.  Here are a few highlights to share with you:

High school students created melanoma awareness videos to warn their peers about the dangers of melanoma as part of the Jeff Dulude Melanoma Foundation & Edgemaker’s Defeat Melanoma PSA contest.  We think you’ll agree that the results are powerful and moving.

 

Top 30 Grant-Giving Disease Foundations via GEN (Spoiler: MRA is #1)

More Arrows in the Arsenal: dramatic advacenments in Immunotherapy offer news options for patients with melanoma via John Hopkins Medicine

What’s the Best Sunscreen For You via U.S. World News & Report

Balloons raise money for melanoma sufferer via The Examiner

Sunscreen alone not enough to protect against melanoma via Medical News Today

Checkpoint agents are shifting paradigms toward immunotherapy via OncLive

Cancer survivor continues to fight via Livingston Daily

Tales from ASCO: The future of cancer research via Philly.com

Best not to fool around with the sun gods via Dallas Morning News