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.

Advertisements

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.