On Sunday afternoon, I received an e-mail from Jeff Allen, the Executive Director of Friends of Cancer Research, the organization that hosted the Breakthrough Therapies Designation Briefing held in the Senate today, asking if Chip or I would be interested in serving on the panel to discuss our personal experience on LDK 378, now known as Zykadia, since this drug was designated as a breakthrough therapy drug by the FDA last week. I was happy to do it if being on tap for two days in a row was going to be too much for him, but Chip didn’t hesitate for a second. He happily agreed to serve on the panel. As a panelist, Chip testified about his participation in the trial and what it meant to him and our family to have had a solid 7 1/2 month stretch of “health” and normal living.
Prior to today’s briefing, the panelists and moderator had an opportunity to meet with Senator Bennet (D-CO) who, along with Senator Hatch (R-UT) and Senator Burr (R-NC), introduced the “Advancing Breakthrough Therapies for Patients Act” in the Senate last spring.
What does Breakthrough Therapy Designation mean? In total laymen’s terms, after animal testing is done, drugs go through three different phases:
- Phase 1–A small group of qualifying candidates (the criteria and protocol for each clinical trial is unique) receive the drug to determine proper dosage levels, how long it takes your body to metabolize the drug and any side effects are recorded. The number of subjects typically ranges from 20 to 80.
- Phase 2–Barring unacceptable toxicity levels and once the proper dosage is determined, the drug then moves into the expansion phase. Chip entered the LDK378 trial in Phase 2. The pharmaceutical company wants to get the drug to as many qualifying candidates as possible to determine if the drug is indeed effective in treating the targeted disease, and any short-term side effects are further documented. Only a couple of hospitals or cancer centers will execute a particular clinical trial at any given time, hence patients quite often having to travel for treatment, because there are thousands of clinical trials for all diseases being conducted across the nation at once, so not all hospitals can conduct all trials. Typically, the number of subjects in Phase 2 studies ranges from a few dozen to about 300. If there is substantial evidence the drug is having an effect on the disease, and more specifically over other comparable or existing treatments, the drug moves ahead to Phase 3.
- Phase 3–More information is gathered regarding the safety and effectiveness of the drug, they study different populations and dosages and study the drug in combination with other drugs. The number of subjects usually ranges from several hundred to about 3,000 people.
Breakthrough Therapies allows a drug, if designated by the FDA, to skip Phase 3 and move directly to the market, so now every ALK+ patient across the U.S. has access to this drug. Both of us being from small, rural towns, it has never been lost on either Chip or me how fortunate we are to now live where we do and to have access to such great medical centers like Johns Hopkins in Baltimore and Fox Chase Cancer Center in Philadelphia that are conducting these clinical trials.
View the Breakthrough Facts Handout for more information.
The FDA approved LDK378 to “break through” to the market by skipping Phase 3, because unfortunately, those with non-small cell lung cancer who are ALK+, are being treated for longevity and quality of life, so they just need this drug. They aren’t worried about potential long-term side effects of the drug or how it interacts with Lipitor, for example. Or in some instances, it may be a drug treating an older population or children that can’t withstand the side effects of traditional chemo, aren’t of child-bearing age, etc., so there is no need for a drug that has proven to be effective in treating their type of disease, to go through all of this additional testing. To think that a potential ALK+ NSCLC patient from Marks, MS, or Conway, NH, could now receive this drug in part to the clinical trial Chip was one of 164 to participate in is really amazing.
It truly meant much to both of us to be able to say thank you to so many in the Senate, the FDA, healthcare providers, pharmaceutical companies and various cancer organizations today for all of the work they have been doing that we are directly benefiting from; likewise, I think it was equally as rewarding for them to attach a name and face to a real person who is a husband and father just trying to live a quality-filled life. We repeatedly heard the message today that they were doing it all for us. For real people like US. Wow.
Tomorrow’s hearing in the Senate Special Committee on Aging regarding The Fight Against Cancer: Challenges, Progress, and Promise in which Chip is testifying, begins at 2:15 PM EST and will be streamed live from the Committee’s website. Riveting television, I know, but many of you have asked how to watch, so if you are indeed interested, click on http://www.aging.senate.gov/hearings/the-fight-against-cancer-challenges-progress-and-promise for your viewing pleasure. The Committee typically leaves a recording of their hearings up on their website, too, so you can watch it later, as well. It is recommended you use Chrome or Firefox as your browser instead of Internet Explorer.
Wish us luck!