Ups and Downs for Melanoma. But Staying Focused.

By Louise M. Perkins, PhD

Chief Science Officer Louise Perkins Headshot

This time a week ago I was jubilant. The SU2C-MRA Melanoma Dream Team’s paper describing a precision medicine approach to treating patients had just been published. And in the middle of the week an important article published in the prestigious scientific journal Cell describing the comprehensive genomic landscape of over 300 melanomas by The Cancer Genome Atlas team – an enormous work that allows us to better understand and target melanoma.

Clearly, with all of this information at our fingertips, with new immunotherapy treatments, with targeted therapy options and new ways to precisely match treatments to patients we have never been closer to defeating this disease. So much progress! I was so excited.

This week I got a text that brought me back to the real world.

In April, I met a businessman at the Milken Institute Global Conference. He told me of his friend, J, who had advanced melanoma. He asked if I might try to help J in some way. I spoke with her and learned that fortunately, she was already connected with some of the leading docs in melanoma. She’d gotten all the new treatments. But she wasn’t doing great and felt like she needed to move back home to be closer to her mom. I had a chance to learn her story and get to know her a tiny bit and was hopeful that things might turn out well.

The text I awoke to let me know that J. died earlier this week. At 32, she’d lost her fight to melanoma.

Earlier this week, MRA met with folks from the FDA along with two leading melanoma experts (Michael Atkins and Paul Chapman) to discuss the latest in the field from the recent cancer meetings. These conversations help to identify new opportunities and help keep the focus on melanoma. The FDA colleagues are great, by the way, and have been amazing partners in bringing new treatments to patients with melanoma very quickly.

In the discussion, both Drs. Atkins and Chapman pointed out that older melanoma patients, the ones who would typically be least likely to tolerate and respond to chemotherapy, actually seem to respond to AND tolerate checkpoint therapy (anti-CTLA-4 and/or anti-PD-1) treatment better than younger folks. The reasons for this aren’t quite clear but two possible explanations spring to mind. Perhaps the tumors in these older individuals have more mutations acquired over a long life and so have more ‘hooks’ for the immune system to latch on to and recognize as foreign. Another hypothesis is that the older folks have immune systems that aren’t as robust as those in younger patients. That robust immune system might get a little overactive in young folks – harming good cells alongside the tumor ones (leading to more side effects) and also prematurely turning off the anti-tumor response.

Is it possible this is like the Spanish Flu where younger, fitter patients were more at risk than older patients? Researchers are working on understanding the differences between responders and non-responders to try to determine what might be tweaked to elicit more and more responses in patients. One of the key takeaways from the meeting with the FDA was the need to continue to focus on those patients who aren’t fully benefiting from current therapy to understand the unique features of their melanoma and work together to beat it. We talked about sub-types that form in the eye, the mucosal surfaces and on non-sun-exposed sites like soles of the feet. We talked about melanoma that has metastasized to the brain – a vexing problem for too many. All of these patients who don’t respond to treatments, old or young, need better options.

Unfortunately, it is too late to help J, or Jackie, or Tara – the young women MRA got to know in the last two years – or Claire, the 17 year old who went to my high school. The deaths of these young women put a fine point on the urgency to keep the pressure on for all melanoma patients to find a cure.

Thanks to all of you for funding the research to understand what’s different and what to do to change the outcomes for patients like these young women – and all of those with advanced melanoma. We’re committed and appreciate your commitment, too.

About the Author

Louise 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.


The First FDA Approval of Two Melanoma Therapies in Combination

By: Louise M. Perkins, PhD, MRA’s Chief Science Officer 

You may have heard this from other sources already, but we want to take a minute and explain why we’re so excited at MRA by some recent news.

This week, GlaxoSmithKline announced that it had received approval from the U.S. Food and Drug Administration (FDA) of a combination treatment for melanoma.  The agency approved the combination of two genetically-targeted drugs, dabrafenib (Tafinlar™) and trametinib (Mekinist™), for patients with metastatic or unresectable melanoma whose tumors possess either the BRAF V600E or V600K genetic mutations.

Why is this so important?  Well, the excitement is on two fronts.

On the one hand, the excitement is high because this was an accelerated approval, a type of approval which allows the FDA tApproved Image for FBo expeditiously approve treatments for serious conditions that fill an unmet medical need.  This is good news for patients because it means the treatment is available to patients sooner than if it was felt to be important to wait for the final, definitive clinical trial to be completed and analyzed.

Secondly, it’s exciting because combinations of treatments are like a one-two punch, attacking the cancer from two different angles and hopefully stalling—or stopping—tumor resistance and improving patient outcomes. These treatments were approved as single agents back in the summer of 2013, and having the data that shows how and why to combine them is important for doctors and patients.

MRA and the melanoma patient community have voiced the need for more treatment options for this disease, and this approval shows that companies and the FDA are responding to this urgency.

MRA has played a critical role in funding combination approaches to fight melanoma.  We have dedicated nearly $12 million to research into this area and anticipate that this figure will increase in March, after we announce our 2014 grant awards.

We’re glad to start off 2014 with a bang, and we hope this approval is only a foretaste of the exciting melanoma developments yet to come this year!

Melanoma News Round-Up, November 22


There’s been lots of exciting melanoma research in the news recently, especially since this week marked the 10th Annual Society For Melanoma Research (SMR) Congress meeting. Check out the stories below for all the research updates and stories you’ll want to share with your friends and family!

New Cancer Treatments Seek to Give the Immune System Superpowers via BIOtechNow

Merck Drug Boosts Survival of Advanced Melanoma Patients via Bloomberg

Amgen Drug Also Increases Melanoma Survival via Drug Discovery & Development

Roche’s Zelboraf improves survival in melanoma patients with brain metastases via ThePharmaLetter

UCLA research could enhance treatments for drug-resistant melanoma via UCLA News

Pfizer, GlaxoSmithKline forge cancer alliance in combo melanoma trial pact via FierceBiotech

60 Years of Data Reveal US Melanoma Epidemic via Medscape

Hugh Jackman: I had skin cancer removed from my nose via Fox News