Above: Gottheimer and Annette Leo speak about the importance of funding rare disease research, flanked by her two sons, Matt and Nick

HACKENSACK, NJ – On Tuesday, February 5, 2019, U.S. Congressman Josh Gottheimer (NJ-5) introduced Annette Leo as his guest to the State of the Union Address tonight in Washington, D.C. He also announced his plan to introduce bipartisan legislation to restore the Orphan Disease Tax Credit in full and ensure that diseases like Sarcoidosis remain counted as rare diseases, so that they will be eligible for the same research opportunities.

“Increasing the Orphan Disease Tax Credit is key to development of treatments for orphan diseases because it allows the researchers to maintain the capital necessary to complete their research and bring a treatment to market. Our legislation will restore the Orphan Disease Tax Credit to 50 percent of clinical testing expenses to give these rare disease treatments an even better shot at reaching the patients who so desperately need them,” said Congressman Josh Gottheimer (NJ-5). “We must keeping investing in the best scientists in the world, that are right here in America. It’s time to invest in innovation in medicine and take a chance on big ideas. It’s time to invest in caretakers and support networks that are not heroes by virtue of their birth, but by virtue of their remarkable love and tireless work for the ones they love. It’s time to invest in hope.”

Leo, of Closter, is the mother of two who have been diagnosed with Ataxia Telangiectasia (A-T), a rare, progressive neurological disorder that affects about 600 people in the United States.

The new legislation discussed by Gottheimer would restore the Orphan Disease Tax Credit that was cut in half by the federal tax bill in 2017. The legislation will be cosponsored by Congressman Fred Upton (MI-6).

Video of the event can be found HERE.

Below: Gottheimer speaks about his plans to restore funding for rare disease research

 

 

Gottheimer’s full remarks as prepared for delivery are below

Thank you, Deputy Mayor Sims, great to see you again. Bob, it’s always great to be back at Hackensack University Medical Center. And thank you to the Leo family for being here today.  We’re blessed here in North Jersey to live within miles to the top medical facilities in the country – and I’m extremely lucky to represent them. Hackensack consistently ranks in the top ten percent of hospitals in everything from cancer treatment to neurology to cardiology, so thank you for everything you do here for our community. Thank you also to Dr. Thomas for being here talk about rare diseases.

With 12 of the world’s top 20 research-based biopharmaceutical companies here in our state, New Jersey is one of the leading centers in the world for life sciences and innovation.  We not only have cutting-edge hospitals, like this one, but our pharmaceutical companies have led the way on some of the biggest medical breakthroughs. New Jersey ranks second in cancer treatment drug development, second for heart and stroke drugs and development.  In 2017, 50 percent of all of new FDA approvals came from companies with a New Jersey footprint.

As lucky has we are to lead the way, it’s no secret that we could always being doing more to improve our health care system. That’s why I have fought so hard to strengthen the Affordable Care Act, and help stabilize the individual marketplace, so that we can help get health care costs down and choices of coverage up, especially for those with preexisting conditions.  Families like the Leos. They should never have to worry about health care coverage. Even with that, and with all of our remarkable progress, we still have a serious gap. 

Today, we are here to talk about an issue that I know is close to the Leos hearts, to mine, and to so many families: How we can do more to help cure and treat rare diseases, known as orphan diseases – those that often don’t the attention and investment they need and deserve.  

We put a man on the moon when they said we couldn’t. We cured polio when the doctors said it would never happen.  We only did it when we were willing to invest and put American grit and tenacity behind it. It’s long time we did that with cures and treatments for more orphan diseases. It’s time we increased and encouraged more investment—more moon shots – and that’s I’m going to announce here today with the bipartisan expansion of the Orphan Disease Tax Credit, and what I’ll reinforce tonight with my guest at the State of the Union – Annette Leo.

Now, this isn’t a Republican or Democratic issue: it’s a mom and dad issue. I have two kids, and I know that any parent would move heaven and earth to make sure their child or their loved one had the medical care he or she needed. 

The more I learn about the Leo family, the more I understand what that looks like. Almost twenty years ago, right here at Hackensack University Medical Center, Nick and Matt Leo were diagnosed with Ataxia-Telangiectasia, or A-T, a rare, progressive, genetic disease that affects a wide variety of systems in the body. The first signs of the disease usually appear in early childhood, when children begin to walk. 

Nick and Matt, who I’m lucky to have with us here today, are tremendous examples of perseverance and grit. They have overcome incredible circumstances, defying the predictions of doctors and constantly amazing those around them. This is, by the way, in no small part because the love and support of their parents. There are many difficult moments which require patience and sacrifice, but Annette and Steve are unconditionally there for their sons. I am so deeply honored to represent this brave family in Congress, and I know that their story will inspire others, as well.

Nick and Matt are just two of about 600 people in the United States with A-T, but the Leo’s are far from alone. There are nearly 30 million people in our country that have been diagnosed with a rare disease, meaning none of us are very far from rare diseases. In Demarest, there is a 12-year old named Gabe Patnoi who was recently diagnosed with Ewing Sarcoma, a vicious disease that causes tumors to grow inside bones. Gabe is a remarkable young man, and our community has really rallied around him, and we are all praying for him. 

My interest in and commitment to addressing rare diseases is also deeply personal. In the fall, I lost my mother to Sarcoidosis, a disease that causes the inflammation of formation tissues in multiple organs in the body, centering on the lungs and the lymph nodes. There are rare forms of sarcoidosis, like the one that my mom had, which largely don’t respond to existing treatments. 

My mom lived for her children and her grandchildren, the loves of her life. She felt tremendous joy for all of us – and a true passion for life — and yet, we watched her struggle with every day  functions – ones that we often take for granted, while living with a disease for which there is no cure.

When we learned of my mother’s illness, naturally, we started doing our research to see if there was anything we could possibly do. It can be a lonely, helpless feeling when a loved one is diagnosed with a disease that you’ve never even heard of, let alone knowing how to tackle it.

What I learned about rare diseases was staggering. In 1983, the Orphan Drug Act passed with broad bipartisan support and was signed into law by President Ronald Reagan. Before that, just 38 orphan drugs for rare diseases had been approved for use in the United States. By 2014, partly as a result of the research incentives laid out in the Orphan Drug Act, 373 drugs for rare diseases had been approved. This progress has been critical, but we still have a long way to go.  Only four percent of rare diseases have at least one approved treatment. 

Here’s the disturbing news.  Believe it or not, last year’s new tax law sent us in the wrong direction – cutting the Orphan Drug Research Tax Credit in half — making treatment for rare diseases even rarer. To me, this seems like giving up, basically saying to doctors, researchers, life sciences companies, patients and families: “these problems are too hard, why try?”

Last time I checked, in the greatest country in the world, we don’t throw our hands up and just quit. We rise to the challenge and, as President Kennedy so famously said, choose to do hard things because they are hard, “because that challenge is one that we are willing to accept, one we are unwilling to postpone, and one we intend to win.” 

Thanks to the Orphan Tax Credit, and our desire to win, individuals with mesothelioma, multiple sclerosis, cystic fibrosis, and bone marrow disorders have benefitted from discoveries in labs across New Jersey and our great country.

We must keeping investing in the best scientists in the world, that are right here in America. It’s time to invest in innovation in medicine and take a chance on big ideas. It’s time to invest in caretakers and support networks that are not heroes by virtue of their birth, but by virtue of their remarkable love and tireless work for the ones they love. It’s time to invest in hope.

That’s why I will be introducing a bipartisan bill with Congressman Fred Upton from Michigan that does just that. My legislation will reverse the previous slashing of the Orphan Disease Tax Credit.  We will invest and restore it to its original levels and give our best and brightest a fighting shot to find effective treatments for rare diseases and, eventually, find a cure to the most ruthless diseases that plague our families, loved ones, and neighbors. 

Drug research for rare diseases is much riskier than for other diseases because the treatments are often much harder, and more costly, to clinically develop. Increasing the Orphan Disease Tax Credit is key to development of treatments for orphan diseases because it allows the researchers to maintain the capital necessary to complete their research and bring a treatment to market. Our legislation will restore the Orphan Disease Tax Credit to 50 percent of clinical testing expenses to give these rare disease treatments an even better shot at reaching the patients who so desperately need them.

This legislation will also ensure that diseases like Sarcoidosis — for which it is already qualified — remain counted as rare diseases, so that they will be eligible for the same opportunities as other rare diseases. 

More than 200 advocacy groups, from grassroots activists to experts to patients, have called on Congress to do right by them. Today, we are taking a critical step, showing the world that we don’t turn our backs on those in need. 

We meet that challenge head on because we know that these victories could literally save a life. It could allow a grandmother to be there for a college graduation. It could mean a bigger family picture at a wedding. It could mean one more trip to the Jersey shore to make a memory that will last a lifetime. 

That’s what we are trying to do here. And I could think of no better partner in this mission than the Leo family, who have already done so much advocacy for A-T research. It will be my honor to have Annette Leo as my guest at the State of the Union Address later tonight in Washington, D.C. to continue to shine a light on our families that live with rare diseases every day.   

Thank you, God bless you, and may God continue to bless and watch over our great country and families like the Leos.