Named after 6-year-old North Jersey resident Cameron Hyman, whose rare disease still has no cure

Gottheimer honors Rare Disease Week this week

Above: Gottheimer announces bipartisan legislation to help find more cures for rare diseases.

HACKENSACK, NJ — Today, U.S. Congressman Josh Gottheimer announced bipartisan legislation — Cameron’s Law — to fully restore the Orphan Disease Research Tax Credit, following the 2017 Tax Hike Bill cutting the Tax Credit in half and making treatment for rare diseases even rarer. With Cameron’s Law, New Jersey’s life sciences leaders will be able to invest more in research and development for cures for orphan diseases, which currently have no FDA-approved treatments, and grow the talent pipeline for life sciences jobs in New Jersey through training partnerships and apprenticeships.

This bipartisan legislation is named after 6-year-old Cameron Hyman from Wyckoff, NJ, who is diagnosed with the rare disease Sanfilippo Syndrome, which affects the central nervous system. Right now, there are no FDA-approved treatments for Sanfilippo Syndrome. Cameron’s Law is being cosponsored by U.S. Congressman Fred Upton (MI-6). 

“We’re here this morning to announce new bipartisan legislation, called Cameron’s Law, in the fight against rare diseases that affect far too many in our great state and country — and to announce new tools to help find a cure. As we always have, New Jersey, and our great life sciences sector — and our R&D teams — can and will lead the way. Here in New Jersey, we have some of the best hospitals and medical facilities in the country — like right here at Hackensack Meridian — the best doctors, medical school graduates, nurses, and life sciences leaders,” said Congressman Josh Gottheimer (NJ-5). “With Cameron’s Law, we will invest and restore the Orphan Disease Research Tax Credit to its original levels and give our best and brightest — many of them right here in Jersey — 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.”

In 1983, the Orphan Drug Act passed with broad bipartisan support and was signed into law by then-President Ronald Reagan. Prior to the 1983 bill, only 38 orphan drugs for rare diseases had been approved for use in the United States. By 2014, through the help of the Orphan Drug Research Tax Credit and research incentives laid out in the Orphan Drug Act, 373 drugs for rare diseases had been approved.

Through the Orphan Drug Research Tax Credit, those with mesothelioma, multiple sclerosis, cystic fibrosis, and bone marrow disorders have benefitted from discoveries in labs across New Jersey and the U.S.

New Jersey is one of the leading centers in the world for life sciences and innovation, with 13 of the world’s top 20 research-based biopharmaceutical companies.

More than 372,000 New Jersey jobs are supported by the life sciences sector with $96 billion in total economic output annually. New Jersey has been ranked the number one state in the nation for biotech growth potential and the number two state for biotech strength.

Gottheimer was joined today at Hackensack University Medical Center by Bergen County Commissioner Ramon Hache, Hackensack University Medical Center Chief Medical Officer Dr. Lisa Tank, Hackensack University Medical Center Chair of Neurology Dr. Florian Thomas, Wyckoff residents Rob and Christina Hyman of the CAM Foundation, patient advocates Stephen and Annette Leo with their two boys, Nick and Matthew, patient advocate Pat Geurds from Handful of Hope, Eagle US EVP of Oncology & Acute Care Marketing John Kimmet, SK Life Science Vice President of Medical Affairs Lou Ferrari, and SK Biopharmaceuticals.

Below: Gottheimer joined by local officials, life sciences leaders, doctors, Rob and Christina Hyman of the CAM Foundation, and patient advocates Stephen and Annette Leo with their two boys, Nick and Matthew, among others.

Watch today’s announcement event HERE.

Gottheimer’s remarks as prepared for delivery are below.

I’m very grateful to Hackensack University Medical Center for hosting us today, and for their tireless work to fight against rare diseases.

We’re here this morning to announce new bipartisan legislation, called Cameron’s Law, in the fight against rare diseases that affect far too many in our great state and country — and to announce new tools to help find a cure. As we always have, New Jersey, and our great life sciences sector — and our R&D teams — can and will lead the way.

It’s been a difficult year for so many. We’ve lost more than 500,000 Americans, including nearly 5,000 in the Fifth District, countless jobs, and have been confined to our homes. 

But there is light at the end of the tunnel: with more and more vaccines being distributed every day. The Administration has said that every adult should be able to get a vaccine by the end of May. 

This pandemic has shown us what breakthroughs in medical treatments and innovation are possible — when our country, our pharmaceutical and life sciences leaders, and our health care community prioritize and put resources behind finding cures. 

And you never know which magic bullet will lead to a cure for another disease — that may have been intended for something else.

Our COVID-19 vaccines were developed by companies like Moderna, Pfizer, and Johnson & Johnson using a new and potentially revolutionary technology called Messenger RNA or mRNA. 

Researchers have been studying and working with mRNA vaccines for decades, including for many rare diseases, but it took this pandemic to create the urgency to fully develop and utilize this capability. Pfizer and Moderna have publicly stated that they developed the design of their vaccines in just a few days. 

I’ll repeat that again because it’s so astounding: they created the rough design of their vaccine in just a few days. 

What’s also exciting is that researchers are adopting the mRNA technology for other deadly diseases, like malaria. 

Here in New Jersey, we have some of the best hospitals and medical facilities in the country — like right here at Hackensack Meridian — the best doctors, medical school graduates, nurses, and life sciences leaders. 

Just like we are coming together to combat COVID and keep our communities safe through new technology like mRNA vaccines, we need to continue to work  together to cure and treat rare diseases, many known as orphan diseases — those that often don’t get the attention and investment they need and deserve. I’m so glad that we are here today at the John Theurer Cancer Center, which has been recognized as the best cancer center in New Jersey. 

Federal investment has helped expand cancer research right here, through The National Cancer Institute, which has included this Center as a member of the Georgetown Lombardi Comprehensive Cancer Center Consortium. This Fifth District facility is now a member of one of just sixteen cancer collaboratives nationwide working every day to find cures.

Unfortunately, a few years ago, some in Congress took a giant step in the wrong direction and slashed the incentive for companies to invest in cures for orphan diseases. They cut the Orphan Tax Credit in half, in a shrill, partisan move — that actually hurt us on many fronts.

But with the important legislation I’m announcing today, with Fred Upton, a Republican from Michigan, a member of the Problem Solvers Caucus, and former Chair of the Energy and Commerce Committee, I believe the Fifth District has a tremendous opportunity to take advantage of the importance New Jersey places on public health, and to find treatments for some of the most harmful rare diseases. 

I’ve discussed this many times, because it’s so near and dear to my heart, but an orphan disease is a disease with no FDA-approved treatment. No cure. My mom died from Sarcoidosis — a rare disease without a cure.

Every day, the bio, life sciences, and pharmaceutical community makes progress toward finding more cures, but we have a long way to go. In total, the FDA approved 32 orphan indications last year — brand-new treatments for rare diseases and new discoveries from existing medications that can be used for other conditions. Researchers are taking drugs that are already approved for use and seeing if they can find a breakthrough for those with an orphan disease. 

Just last year, the Office of Orphan Products Development at the FDA received 753 new requests for orphan drug designation, a 41% increase from 2019. Even more promising, and so critically important, the FDA saw a 330% increase in pediatric cancer orphan drug designation applications. We know the science is there and the researchers want to do the work, so we must put our money where our mouths are and fund these important cures.  

In 1983, the Orphan Drug Act passed with broad bipartisan support and was signed into law by President Ronald Reagan. 

Before that, just thirty-eight 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. 

As I said, the 2017 Tax Hike Bill that also gutted our State and Local Tax deduction — and sent us in another wrong direction — cut the Orphan Drug Research Tax Credit in half, making treatment for rare diseases even rarer. 

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

But with this credit cut in half back in 2017, we’re not where we should be.

Thirteen of the world’s top 20 research-based biopharmaceutical companies here in our state. We not only have cutting-edge hospitals, but our pharmaceutical and medical device companies have led the way on some of the biggest medical breakthroughs. 

But they need help — R&D is enormously expensive and these cures often, initially, only help a limited number of people — so these life sciences companies need more help for these investments.

New Jersey now leads the nation with 139 FDA-registered biopharmaceutical manufacturing facilities — that’s more than both California and Pennsylvania. New Jersey ranks second in cancer treatment drug development, second for heart and stroke drugs and development, and has been ranked the number one state for biotech growth potential.   

We’re lucky to have so many of these cutting-edge research operations here in my District and across New Jersey. In our District, we are proud to be home to BD, Glenmark Pharmaceuticals, Zimmer Biomet, SK Bio, Eagle Pharmaceuticals, and many others. Not to mention the life-saving work happening every day all across our great state. 

More than 372,000 New Jersey jobs are supported by the life sciences sector, with $96 billion in total economic output annually. There is nowhere to go but up. 

Even though we have some of the best facilities here in Jersey, they’re facing obstacles. Since the 1990s, the cost of doing business here has increased more than most of our peers. It’s more than 10% higher than the U.S. average, primarily because of the state’s high labor costs and our high taxes. 

Now, I helped lead the charge to fully eliminate the federal Medical Device Tax last Congress, which helps. However, the cost of doing business in New Jersey has continued to rise.

Compounding these difficulties, during the past decade, New Jersey’s biopharma industry has experienced increased competition from other states that have also been trying to raise their quotient of biopharma start-ups.

California and Massachusetts have, respectively, created ten and four times the number of companies compared with New Jersey. That’s 885 biopharma companies founded in California between 2012 and 2015, and 430 in Massachusetts — with only 89 in New Jersey. Medical device companies are also being outpaced by increased employment in states like Massachusetts, Indiana, and Minnesota.

But New Jersey remains “the medicine chest of the world.” I couldn’t be more proud — look at how this sector just saved the world with the COVID-19 vaccine.

I want us to double down so that we expand in the years ahead — and continue leading the way in medicine, developing the next cures for orphan diseases, cancers, and other diseases, and developing innovative medical devices that allow people to walk again and live normal, healthy lives.

After all, we have more than half of the twenty best global pharmaceutical companies operating here. This represents a significant opportunity to attract more companies, grow revenues, attract more jobs here, and continue leading in improving the health of people all around our nation.

It also presents an opportunity to cure rare diseases that affect our residents, including many families and children I’ve had the honor to meet and work with. Two years ago, I stood with Sean O’Malley at Hackensack Meridian Health’s children’s hospital, who beat the odds and is now in remission from pediatric cancer and living a normal healthy life.

Today, I’m very lucky to be joined by Rob Hyman, whose six-year-old daughter, Cameron Hyman, has a rare disease called Sanfilippo Syndrome, which affects the central nervous system — specifically the brain and spinal cord. Right now, there are no FDA- approved treatments for Sanfilippo Syndrome. It’s one of the reasons I’m here today, because we need to change that.

The Hyman family, from Wyckoff, launched the CAM Foundation, to raise funds to help invest in trials and to find a cure for this degenerative disease. Cameron is far from alone in the fight for cures for rare diseases. There are nearly 30 million people in our country who have been diagnosed with a rare disease, meaning none of us are very far from these diseases — or very far from seeing all the work that we still need to do to make sure more cures are found. 

I heard this first-hand this week, during Rare Disease Week, when I joined many of my constituents who face debilitating and life-threatening rare diseases. We must do more to provide hope and support to the families who struggle every day managing a rare disease, and also provide opportunities for our cutting-edge research institutions to finally get us to the desperately needed cures. I know we can do this.

That’s why, I will be formally introducing bipartisan legislation — Cameron’s Law — to fully restore the previous slashing of the Orphan Disease Research Tax Credit. 

We will invest and restore the credit to its original levels and give our best and brightest — many of them right here in Jersey — 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. These drugs are often then used to cure other diseases too.

This tax credit will give families like Cameron’s the resources and hope for curing these rare diseases. It will give everyone here more research opportunities so that we can all come together and continue to ensure New Jersey is a leader in this fight. 

Here in the greatest country in the world, we can come together to solve problems and give everyone a fighting chance. Once again, thank you for joining me here today.

Thank you for everything you all do, God bless you, and may God continue to bless and watch over brave families like the Hymans and our great country.

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