What It Means to Go First.
For these patients, giving up wasn’t an option.
For patients fighting disease, biotechnology offers hope. Cell and gene therapies gave Marci McCue, Emily Whitehead, and Jennelle Stephenson options that conventional treatments could not provide. Their stories demonstrate the power of biotechnology and the courage it takes to be the first.
Marci McCue
When Marci was diagnosed with multiple sclerosis (MS), she had to face the reality of a disease that would change her life. Determined to redefine her future, she enrolled in a clinical trial and, in 2024, became the first patient in a U.S. clinical trial to receive CAR-T cell therapy for MS – an approach that resets a patient’s own immune cells to fight disease from within. Today, she has no detectable oligoclonal bands, a key marker commonly associated with MS disease activity.
“I think that when you’re in a mindset of trying anything that’s available to stop your disease, you’re not afraid to be patient number one,” Marci says.
Tom and Emily Whitehead
At five years old, Emily Whitehead was diagnosed with acute lymphoblastic leukemia. Her parents, Tom and Kari, were told the disease is highly curable in children — but after Emily relapsed and standard treatments failed, the family was advised to consider hospice care. Refusing to give up hope, Emily’s parents pursued a novel treatment option that would make history.
In 2012, Emily became the first child with cancer to receive CAR-T cell therapy. Just weeks after her genetically modified cells were infused, her cancer was gone. Today, Emily remains cancer-free and is considered cured. Her treatment helped pave the way for immunotherapies that have since reached tens of thousands of patients worldwide.
“I am alive today because of research, science and a belief in what’s possible,” Emily says. “Amazing progress has been made in cell and gene therapy since my treatment, but there are far too many patients and families who lack access. My hope is a future where everyone who can benefit from advanced therapies has that life-saving chance.”
Jennelle Stephenson
Jennelle Stephenson was born with sickle cell disease. For most of her life, pain was simply her normal – she was the kid who was always sick. At 26, she decided she was ready to change her story.
After learning about an experimental gene therapy study at the National Institutes of Health (NIH), Jennelle enrolled in the clinical trial and became one of the first people to receive gene therapy for sickle cell disease. Her participation helped pave the way for FDA approval in 2023.
For Jennelle, the choice came down to hope. As she explains: “It came down to me choosing between two uncertainties. What was certain was that I was living 26 years in pain, and that had to change.”
Today, more than eight and a half years later, Jennelle hasn’t experienced a single sickle cell crisis. Jennelle’s bravery is a call to keep fighting so innovation can meet the courage of every patient who takes that leap.