They need cures now

By Earl L. “Buddy” Carter

 

Guest Columnist

Maybe your grandmother was diagnosed with cancer, your friend lives with Crohn’s disease, or your colleague’s child battles Duchenne muscular dystrophy. In one way or another, we’ve all been touched by disease, and we all have a different story to tell.

As a lifelong health care professional, I saw the heartbreaking devastation of disease and the toll it takes on patients and their families every day.

A patient from Augusta University in Augusta knows the struggles of living with a disease firsthand. This patient lives with sickle cell disease, a genetic mutation of hemoglobin, the red blood cell component that delivers oxygen to cells throughout the body, causing the cells to take a crescent shape.

Describing living with sickle cell disease, the patient said, “Sometimes it’s just a dull, nagging ache. Other times it feels like getting run over by a Mack truck and then thrown into a meat grinder.”

No one should have to live a life like this where pain becomes so bad “it’s like saying, ‘I’m gonna beat the crap out of you.’” Tragically, this is reality for many Americans across the country.

Fortunately, health care innovation is better than ever before, and medical breakthroughs are happening all around us, even here at home in Georgia. This patient and others with sickle cell disease are able to access a better quality of life thanks to visits and clinical trials at the AU Comprehensive Sickle Cell Center.

The prominent center is responsible for groundbreaking findings, including the Stroke Prevention Trial in Sickle Cell Anemia by former AU faculty Dr. Robert Adams. Dr. Adams and his team discovered a way to reduce pediatric sickle cell patient’s stroke risk by 90 percent with regular blood transfusions.

Impressed? This is just one example of the cutting-edge research happening at the center.

AU researchers are laying the groundwork for new anti-sickling therapies and collaborating with researchers from institutions such as Harvard and Stanford to discover what molecular events could eliminate fetal hemoglobin. AU researchers are even working to identify how cells signal each other during drug-induced production of fetal hemoglobin.

Today, there are more than 10,000 known diseases but only 500 have treatments. Thanks to tremendous research and findings like those at AU, this gap soon will hopefully narrow. However, even though medical breakthroughs are happening at lightening speed, laws and agencies can’t keep up preventing them from reaching the patients.

That is why the 21st Century Cures Act, which recently passed in the United States House of Representatives, the United States Senate, and has now been signed into law, is so important. This critical law will work to break down barriers and get the government out of the way, allowing the development and delivery of new treatments and cures to patients.

Cures removes barriers to increased research collaboration, incorporates the patients prospective into the drug development and regulatory review process, modernizes clinical trials, provides new incentives for the development of drugs for rare diseases, and helps the entire biomedical ecosystem coordinate more efficiently to find faster cures.

Patients at AU and across the country deserve the best treatments available and the government and bureaucracy need to get out of the way. Twenty-first Century Cures is now law and will ensure this happens by paving the way for the next generation of cures and treatments. Whether a patient at AU, a patient I saw in my pharmacy, or any other patient from across the nation, they need cures now.

(The writer has represented Georgia’s District 1 in the U.S. House of Representatives since 2015.)

 

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