When Suneel Raam was just 3 years old, doctors told his parents he likely wouldn’t live past his 20s.

Suneel was diagnosed with Duchenne muscular dystrophy (DMD) — a rare and serious disease. In kids with DMD, they’re missing a gene that makes a protein called dystrophin, which supports muscle form and function. 

For most, the journey with DMD means losing the ability to walk in their early teens and relying on breathing machines and wheelchairs by adulthood. 

But Suneel, now 28 years old, isn’t one to give up easily.

Despite the challenges, Suneel has always had a big spirit. Growing up in Buffalo, New York, he faced broken bones, weak muscles and days filled with physical pain. Tasks that most take for granted come harder for him. But nothing ever stopped his determination. Inside, he had a powerful dream — to rise above his disease and live a life full of purpose.

And that’s exactly what he’s doing.

Innovative Treatments Are Worth Traveling For

In 2020, Suneel joined a groundbreaking clinical trial called Deramiocel (CAP 1002), or HOPE-2, a name that fits perfectly with what it offers — hope.

This study tests a treatment for DMD that uses cells taken from donated heart tissue. These cells are called cardiosphere derived cells. Patients with DMD often experience heart damage or failure. These cells work to calm inflammation, repair muscle tissue and help protect the heart. 

“This treatment acts to reduce inflammation of the muscle that can help support cardiac and skeletal muscle function,” said Nancy Bass, MD, Pediatric Neurologist at Children’s Wisconsin “This treatment can prevent the progressive injury of the of the muscle, which is called fibrosis.”

Suneel travels every three months for treatment to the Children’s Wisconsin Milwaukee Hospital. At first, he made the long 10-hour drive, occasionally stopping overnight in Chicago. Now, he flies to make the journey easier and less painful.

“Our days get a little brighter when Suneel comes in,” one nurse said. “The care team has gotten to know him well — laughing, learning and working together to make each visit as smooth as possible.”

The HOPE-2 trial is run by the Pediatric Translational Research Unit (P-TRU), which is a shared service funded by the Children’s Research Institute (CRI). As a children’s hospital in an academic medical center, research is interwoven into every aspect of care. Physician-scientists, nurse-scientists, researchers and technicians at CRI seek answers to the toughest questions in pediatric medicine. Their research is focused on improving diagnostics, discovering and testing new therapies, and improving the way we prevent and manage some of the most complex medical conditions. They take discoveries from the laboratory and convert them to new therapies, diagnostics and other innovations at patients’ bedsides.

Since its inception in 2003, CRI has grown to include more than 150 researchers spanning all diagnostic, medical and surgical specialties. Together they form the largest concentration of 100% pediatric research in Wisconsin. Currently, CRI has more than 1,000 active clinical studies — including the HOPE-2 trial — as well as approximately 200 interventional clinical trials. 

A Day in the Life of Suneel at Children’s Wisconsin

On the day of his treatment, Suneel begins with premedication. Then, physical therapists test his strength and range of motion and the nurses take his vitals. Once his condition is stable, nurses start an IV to deliver the medicine while carefully following the trial’s detailed protocol. The infusion takes about an hour. It’s all part of gathering important data that may change future DMD treatment.

“The study coordinators and nurses are excellent and the care team does everything they can to make us comfortable,” said Suneel’s mom, Neera Gulati, MD. “Everyone goes far out of their way to make sure the arrangements are as easy as possible. It's really a wonderful experience.”

Suneel is the only patient in the HOPE-2 trial at Children’s Wisconsin, but he’s far from alone. He’s part of something bigger — a worldwide study that could change the course of the disease. 

“This particular treatment would open up the door for treating older patients,” said Dr. Bass, who is also a Professor of Neurology at the Medical College of Wisconsin, the academic partner of Children’s Wisconsin.

“Although DMD is a devastating diagnosis, the world has changed from even 20 years ago. There are many treatments currently being worked on for this disease.”

Bettering Outcomes, One Trial at a Time

The Hope-2 Deramiocel trial shows promise. It not only improves upper mobility but also protects the heart. These improvements may allow people living with DMD to live longer and better lives. Currently, the trial sponsor is seeking FDA approval while the trial continues as an open-label extension. This phase of the study follows an earlier randomized clinical trial primarily to gather more information about the treatment’s effectiveness, safety, and tolerability. 

The Children’s Wisconsin Neurosciences Center and P-TRU teams are proud to play a part in bettering outcomes through research. 

“Suneel’s story is inspiring and emblematic of why research is done in the first place,” said Joe Zack, MSN, RN, Clinical Research Nurse. “We do the work in the background, but it’s people like Suneel who are the heart of the study.”

Research is a pillar of the Children’s Wisconsin mission — and the Medical College of Wisconsin shares a similar commitment. This dedication to education, research and innovation allows Children’s Wisconsin doctors to constantly be pushing the boundaries of what is possible. And it’s all done in the name of improving health and well-being of Wisconsin’s children and families.

Suneel will continue traveling to Children’s Wisconsin until his 60-month trial ends. He and his family are appreciative of Children’s Wisconsin, as well as the trial itself, knowing participating in the Hope-2 trial and his advocacy work goes beyond himself.  Suneel is hoping the trial ever ends and continues until the medication is available commercially.  

“If this trial ends before this medication is available for all of us that would be the worst thing I could think of,” he said. “This medicine has really helped! Hope is never supposed to end. It’s supposed to be there for everyone.”

Suneel and his mom started a nonprofit called Suneel’s Light Foundation to raise awareness, fund DMD research and help other families who can’t afford care. The foundation offers grants to people in need and speaks to lawmakers, urging them to support policies that help people with rare diseases.

Suneel doesn’t stop at advocacy. He’s a singer in a band and authored a book called “Soaring: A Story of Courage.” His book shares how he overcame loneliness, rejection and physical pain to become a voice for others living with disabilities. 

Suneel’s courage, strength and outlook — and the clinical research efforts at Children’s Wisconsin — are changing the future for people living with chronic diseases. 

Suneel’s story reminds us that real heroes don’t always wear capes. Sometimes, they wear a smile and bring hope wherever they go. And as the trial moves forward, Suneel keeps soaring — one infusion, one song and one dream at a time.