Bradley Byrne: The fight against childhood cancer
As a father of four, it is difficult to even contemplate a child’s life being cut short due to cancer or another terminal illness. As Danny Thomas, the founder of St. Jude Children’s Research Hospital, once stated, “no child should die in the dawn of life.” This powerful statement rang true recently when I met the mother of William the Warrior. Just one month ago, three-year-old William Marion lost his battle after being diagnosed with a rare form of sarcoma. His mother, Jessica Marion, recently visited my office in Washington to share the story of her son’s life and their family’s tragic loss. Sadly, William is just one of thousands of American children who have been impacted by cancer. Each year, 175,000 children ages 14 and under are diagnosed with some form of pediatric cancer. Thankfully, due to medical innovation, more than 80 percent of children diagnosed in the United States become survivors. However, we must continue to fight for better treatments and for those who are not among the fortunate. Jessica brought to my attention H.R.820, the Childhood Cancer STAR Act, and asked for my support. After learning more about the bill and hearing Jessica and William’s story, I happily agreed to add my name as a cosponsor in memory of William and all other children who have lost their lives to cancer. The STAR Act enhances efforts to identify and track childhood cancer cases, seeks to improve the quality of life for childhood cancer survivors, and expands research opportunities for therapeutics used in the medical treatment of childhood cancer. It is imperative that we redouble our efforts to end childhood cancer and ensure no more children and families will suffer from this tragedy. While this legislation cannot bring William back, the STAR Act reaffirms my commitment to keep alive the quest for a cure. The STAR Act is not the first time I have supported live-saving initiatives. Throughout my time in Congress, I have strongly supported efforts to boost funding for medical research and reform the process for approving new medical treatments. Most recently, I voted in favor of increased medical research funding, which will ensure our doctors and scientists have the resources they need to perform life-saving research. Among many other important provisions, the most recent government funding bill increased funding for the National Institutes of Health by $3 billion, which will continue progress toward finding cures for cancer, Alzheimer’s disease, and many other life-threatening conditions. Additionally, the House recently passed H.R. 5247, the Right to Try Act of 2018, with my strong support. As you may recall, President Donald Trump discussed the need for “Right to Try” legislation in his State of the Union speech earlier this year. Right to Try allows terminally ill patients to take advantage of experimental medical treatments that otherwise may not be available. I also strongly supported the bipartisan 21st Century Cures Act to improve our nation’s medical research programs and spur medical innovation. It is not often that Congress passes legislation that can actually save lives, but the 21st Century Cures Act can do just that by making some critical updates to our nation’s health programs. Ultimately, there is no legislation that can take away the heartbreak that comes with losing a loved one to cancer or another terminal disease. However, Congress must continue to make medical research a top priority as we fight to end childhood cancer once and for all. • • • Bradley Byrne is a member of U.S. Congress representing Alabama’s 1st Congressional District.
Fate hazy for GOP bill helping dying patients try new drugs
A leading House Democrat announced his opposition Monday to a Republican bill making it easier for some terminally ill patients to try experimental drugs, clouding the measure’s fate. Republicans are hoping for House approval Tuesday, seven months after a similar package cleared the Senate. The “Right to Try” bill gained added momentum after President Donald Trump called for its passage in January’s State of the Union address. Under the bill, if a doctor and drug maker agree to let a patient with life-threatening ailments try an experimental treatment, the federal Food and Drug Administration would no longer need to consent. The FDA would have to be notified of the decision and of any problems that occur. New Jersey Rep. Frank Pallone, top Democrat on the House Energy and Commerce Committee, said the measure is risky, unneeded and “provides false hope to patients.” He also noted that the bill doesn’t require drug makers to provide these treatments to patients, which is often where stumbling blocks can occur. Companies sometimes prefer using what they’ve produced for clinical trials needed for final FDA approval, or worry that a problem could hurt the drug’s marketing prospects. A recent study by the Government Accountability Office, Congress’ investigative agency, found the FDA currently approves 99 percent of such requests, often within hours. The measure strikes a balance between safety and providing “hopeful news for patients desperately seeking the right way to try” experimental treatments, said Energy and Commerce Chairman Greg Walden of Oregon and Michael Burgess of Texas, another top panel Republican. Pallone’s stance is important because GOP leaders want to rush the legislation through the House using a process, normally reserved for uncontroversial measures, that could require a two-thirds majority. If all Republicans vote yes, they’d need the backing of at least 50 Democrats. House Minority Leader Nancy Pelosi, D-Calif., hadn’t taken a public position on the bill by late Monday afternoon. Republicans released the latest version of their proposal Saturday. More than 40 patients’ groups have expressed opposition to the measure, saying it would “likely do more harm than good.” PhRMA, the trade group representing major drug companies, has taken no public stance. Republished with the permission of the Associated Press.
Mo Brooks fights for patients’ ‘Right to Try’
On Thursday, Alabama 5th District U.S. Congressman Mo Brooks delivered a house floor speech requesting a vote on S. 204: the Right to Try Act, which gives terminally ill patients the option to try experimental treatments that have not yet been approved by the Food and Drug Administration (FDA). Often the FDA’s approval of new treatments is a lengthy and complex processes that can takes decades to fully be completed. This bill would allow terminally ill patients to try potentially life-saving experimental treatments and gives patients the right to fight their diseases without having to fight federal bureaucracy. “Patients shouldn’t have to give up their liberty, their freedom, their fight against terminal illness merely because the FDA says so” said Brooks. Brooks was inspired by the story of Steve Mayfield, a beloved football coach at Central High School in Lauderdale County Alabama. Mayfield died in March of 2017 after a long and arduous battle with both Lou Gehrig’s disease (ALS) and the FDA. Like most terminally ill Americans, Mayfield did not have access to investigative treatments due to the FDA’s current policies. “While the Food and Drug Administration grants compassionate use waivers, meant to allow terminal patients access to experimental drugs, only about 1,500 waivers were granted in 2016” said Brooks. “What are other terminally ill Americans to do? Nothing? Just waste away and die without a fight?” Brooks believes the Right to Try Act is the best solution to this problem as it only grants access to treatments that have successfully completed the FDA’s Phase 1 approval requirement. This keeps patients safe from potentially harmful treatment processes while allowing them access to try potentially life changing medical care. “Given the stark contrasts between life and death, between freedom and federal dictates, between hope and hopelessness, the House should take up and pass the Right to Try Act, thereby giving a chance for life to terminally ill patients and their families.” The Senate passed Right to Try legislation last year, and it currently awaits a vote in the House.
Angi Stalnaker: A few good things happened on the way to Sine Die
Pick up any newspaper or turn on any local television broadcast and you would think that the Alabama Legislature adjourned without doing anything productive. That is not exactly true. Several high-quality pieces of legislation became law this session courtesy of the Legislature and Gov. Robert Bentley‘s signature: The Right to Try Act Anyone who spent any time at the Alabama State House this session was sure to notice a very energetic red-headed little boy seated in the gallery or skipping through the hallways. That little boy is Gabe Griffin and he suffers from Duchene Muscular Dystrophy, which will leave him wheelchair bound in a few years if a treatment is not found. Thanks to a bill sponsored by Rep. April Weaver, Gabe and other Alabamians that suffer from terminal illnesses, will now have the opportunity to receive drugs that have not completed the burdensome FDA approval processes but have shown signs of being able to help. Closing loopholes in the Open Meetings Law Alabama’s open meetings law had gaps large enough to fly an Airbus through but Sen. Cam Ward helped pass a bill that restores one of the country’s oldest public meeting statutes to its original intent, and the taxpayer is better off because of his efforts. Shining a little more sunlight on government is always a good thing. Fostering Hope When foster children “age out” of the system after high school, they are often left with few resources and even fewer choices about their futures. Things will change for the state’s foster care graduates now as a result of Sen. Dick Brewbaker’s legislation. Foster kids will now be able to receive tuition, books and fees for their college education as long as they maintain the necessary academic requirements. Thie bill gives a future to hundreds of kids who deserve it. Angi Stalnaker is Alabama native and political consultant who has worked on numerous statewide, legislative and constitutional amendment races for conservative causes and candidates. She is the founder of Virtus Solutions, a full service government relations and communications firm.
Family behind new Alabama “Right to Try” law looks beyond the bill
Gabe Griffin lobbied his first bill at the age of 9. It became law when he turned 10. He’ll be in a wheelchair when he turns 12. Without the right drugs, his parents say he’ll be dead in a decade. Gabe doesn’t know he’s dying, though. In fact, he doesn’t even know what a law does. He knows it could help him, but when you ask him how, he pulls his black baseball cap all the way over his red hair, over his freckled face and asks his mom and dad when they’ll stop peppering him with questions so he can go eat cheese dip. Gabe has Duchenne Muscular Dystrophy, a rare muscular degenerative disease that gradually and then rapidly leads to incapacitation before ultimately to an early death. Right now, he can whack a baseball with a bat like any boy, but tires before first base. He dreams of being an “armian” — his way of pronouncing Army man — but has to be carried upstairs. The 10-year-old became a familiar face in the Alabama Legislature during the 2015 Legislative Session, as his family visited time after time from their Birmingham-area home to ask lawmakers to pass a bill that might someday get him drugs that don’t yet exist. The Gabe Griffin Right to Try Act, which Gov. Robert Bentley signed into law Wednesday, seeks to allow access to drugs that have completed a first clinical trial but that haven’t been approved for general use by the U.S. Food and Drug Administration. Gabe’s mother and father, Traci and Scott Griffin, have been searching for treatment since he was diagnosed seven years ago. The Griffins, and others facing a number of terminal diseases, think the FDA approval process is too tedious, too selective and too time-consuming to provide any hope. “It’s one of those things that you can see and you can smell but you can’t touch,” Scott Griffin said. Two drugs in clinical trial for Duchenne patients — one by Sarepta Therapeutics Inc. and the other by BioMarin Pharmaceutical Inc. — have promise. Neither is yet suitable for Gabe’s case, but when they are, the Griffins want to take the risk. Alabama is one of several states that passed a “Right to Try” law during their 2015 session, a legislative trend that advocates say provides patients with new options and that others say creates new risks. “We talk about side effects often in safety, and I get it, I get it,” Scott Griffin said. “But my son is going to have tubes coming in and out all parts of his body, with a steel rod shoved in his back, his Achilles heel chords cut, not being able to roll over, not being able to lift his head up. I don’t think it gets much worse than that.” According to the Goldwater Institute, one of the groups behind other states’ Right to Try laws, 19 states have such laws; in another three states, legislation is awaiting the governor’s signature. The FDA, tasked with the duty of ensuring drugs are safe and effective, has not taken a position on any state’s “Right to Try” legislation. “It is critical for the public to understand that FDA is not a barrier to accessing investigational drugs or medical devices,” FDA spokesman Jeff Ventura said. “The FDA is an important part of the process and helps to ensure patients are adequately protected from receiving a potentially harmful or ineffective treatment.” The agency is trying to work with terminally ill patients who fear they’re out of options. Through its Expanded Access — or “compassionate use” — program, the FDA from 2010 to 2014 allowed 99 percent of the applications it received to proceed. This year, the agency announced plans to simplify and accelerate the application process for patients who want to try unapproved drugs. The pace of drug discovery for orphan diseases — those affecting a relatively small population — has accelerated. According to Pharmaceuticals and Research Manufacturers of America, the FDA has approved about 500 drugs for orphan diseases since the passage of the Orphan Drug Act of 1983, which aimed to hasten the approval of drugs for orphan diseases. During the past decade it approved 200. Last year, 17 of the 41 drugs approved by the FDA were for orphan diseases. PhRMA has not taken an official stance on Right to Try legislation or on related bills at the federal level, but Sascha Haverfield, PhRMA’s vice president of scientific and regulatory affairs, said in a prepared statement that the organization has “serious concerns” with alternative approaches to making investigational medicines available. But the Griffins don’t know how long they’ll have Gabe. Some Duchenne patients live into their twenties. Others die of heart failure as teenagers. Meanwhile, the Griffins have started a nonprofit, Hope For Gabe, to raise awareness in the state and across the country. Last summer, Birmingham attorney Michael Staley and Indiana University student Wes Bates rode their bicycles across the U.S. to advocate for Duchenne. Staley, the former chief of staff of former Alabama U.S. Rep. Spencer Bachus, a Republican, has been helping the Griffins push for changes at the state and federal level since he first met Gabe during the Griffins’ visit to Washington, D.C. “As a dad, and as mom, you’re ready to jump in front of a train, a moving train, to save your child,” Scott Griffin said. “Duchenne is that moving train, and we are going to do everything humanly possible to try to make Gabe, and this generation of Duchenne boys, the first generation to survive this disease. I don’t know how it’s going to turn out, but man, we’re close. We’re close.” Republished with permission of The Associated Press.
Senate OKs bill to give dying patients “right to try” promising meds
Doctors will be allowed to prescribe experimental drugs to terminally ill patients under a new law passed in the Senate. Senate Bill 357 gives patients the “right to try” drugs the FDA has designated as safe for clinical trials. The legislation was inspired by 9-year old Gabe Griffin of Shelby County. Gabe suffers from an incurable disease called Duchenne Muscular Dystrophy. Duchenne causes generalized weakness and muscle wasting that increases over time and with muscle activity. Through a nonprofit, Hope for Gabe, created by his parents, the Griffin family putting focus on Duchenne to ensure that Gabe and others like him have access to drugs that could possibly save their lives. Gabe’s parents say clinical trials are his only option. His father, Scott Griffin, said, “As we get further down the road with Gabe’s terminal condition, we are running out of time. As a father, the last thing I want to do is sign my son up for a clinical trial where he may receive a placebo drug. If a drug is safe enough to be in an FDA trial where humans are already taking it, then Gabe should have the right to be guaranteed access and that is what this is about to Traci and I as his parents.” In a prepared statement, bill sponsor Sen. Cam Ward, an Alabaster Republican, said, “Terminal patients should have a right to try, and as a father and an elected official I believe we should do everything we can to help remove unnecessary bureaucratic red tape from the process so that doctors and terminally ill patients can determine the best course of action in each individual situation.” The bill will now move to the House for consideration. Read more about Gabe Griffin in an Alabama Today editorial.
