John F. Crowley, president and CEO of the Biotechnology Innovation Organization (BIO), delivered the keynote address at the Emily Whitehead Foundation's annual Believe Ball on September 25, 2026, at The Crystal Tea Room in Philadelphia's historic Wanamaker Building.
The event brought together over 400 guests—cancer patients, caregivers, and biotech industry leaders—to celebrate advances in cell and gene therapy and accelerate access to breakthrough treatments. Crowley's presence at the gala reflects a shared mission: advancing life-saving medical innovation for patients facing rare and childhood diseases. His keynote highlighted how strategic partnerships between patients, researchers, and biotech leaders drive the breakthroughs that transform treatment options.
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Table of Contents
- John Crowley's Path to Biotech Leadership
- Emily Whitehead's CAR-T Breakthrough
- How CAR-T Cell Therapy Works
- The Emily Whitehead Foundation's Mission and the Believe Ball
- Why This Matters to Treatment Access
- Frequently Asked Questions
John Crowley's Path to Biotech Leadership
john F. Crowley currently leads BIO, the world's largest biotechnology advocacy organization, overseeing the industry's voice on innovation and access policy.
His commitment to biotech stems from personal experience: in 1998, two of Crowley's children were diagnosed with Pompe disease, a rare genetic disorder. Rather than accept the diagnosis, he co-founded Novazyme Pharmaceuticals to develop a treatment—a story later immortalized in the 2010 film "Extraordinary Measures." Crowley went on to serve as chairman and CEO of Amicus Therapeutics, further establishing his track record of turning scientific advances into patient treatments.
Emily Whitehead's CAR-T Breakthrough
Emily Whitehead's story is at the heart of the foundation bearing her name. At age 6, she became the first child in the world to receive CAR-T cell therapy for acute lymphoblastic leukemia at Children's Hospital of Philadelphia. The treatment was nearly fatal but achieved complete remission—a result that seemed impossible at the time.
Now over 18 years old and more than 12 years cancer-free, Whitehead's case became the proof-of-concept that justified fda investment in CAR-T therapies. In 2017, the FDA approved tisagenlecleucel (Kymriah), the first CAR-T cell therapy, catalyzed directly by Whitehead's clinical outcome and subsequent evidence. CAR-T is now standard treatment for relapsed B-cell cancers and continues to expand to other malignancies.
How CAR-T Cell Therapy Works
CAR-T is an immunotherapy that reengineers a patient's own immune cells to fight cancer. Doctors extract T cells—white blood cells central to immune defense—and engineer them to recognize and attack cancer cells displaying specific surface proteins.
Once reinfused, these CAR-T cells seek out and destroy cancer throughout the body. The approach nearly killed Whitehead during treatment due to severe inflammatory side effects, but the complete remission it achieved proved the therapy's potential. Today, CAR-T options exist for multiple cancer types, though access remains limited and costs exceed $500,000 in most cases.
The Emily Whitehead Foundation's Mission and the Believe Ball
The Emily Whitehead Foundation supports patients and caregivers affected by childhood and rare diseases and advocates for scaling CAR-T cell and other advanced cell and gene therapies globally. The foundation operates on an annual budget of $6 million, funding research, patient support programs, and industry engagement.
The annual Believe Ball bridges this ecosystem by convening patients fighting rare diseases directly with industry scientists, developers, manufacturers, and healthcare providers. This in-person collaboration accelerates clinical trial recruitment, investment in underfunded disease areas, and real-world problem-solving. Crowley's keynote emphasized how stories like Whitehead's—paired with leadership from industry visionaries—inspire the next wave of breakthrough therapies.
Why This Matters to Treatment Access
For patients diagnosed with relapsed blood cancers or other rare diseases, events like the Believe Ball represent tangible pathways to treatment. Before Whitehead's breakthrough, many pediatric and adult patients had exhausted conventional options.
However, access remains uneven: insurance coverage varies widely, and eligible patients must navigate clinical trials or compassionate-use programs to receive treatment. Crowley's leadership at BIO and his keynote likely emphasized the advocacy and policy levers needed to widen this access—a message directly aligned with the foundation's work.
Frequently Asked Questions
Who is John F. Crowley?
Crowley is president and CEO of BIO, the world's largest biotechnology advocacy organization. He co-founded Novazyme Pharmaceuticals to treat Pompe disease after his own children were diagnosed in 1998; his story inspired the 2010 film "Extraordinary Measures."
What is CAR-T cell therapy?
CAR-T is an immunotherapy that extracts a patient's T cells, engineers them to recognize and attack cancer, and reinfuses them. It achieved FDA approval in 2017 and is now standard treatment for certain blood cancers.
Why is Emily Whitehead significant?
At age 6, Whitehead became the first child in the world to receive CAR-T therapy for leukemia. Her complete remission paved the way for FDA approval that now benefits thousands of cancer patients.
When and where was the 2026 Believe Ball held?
The event was held on September 25, 2026, at The Crystal Tea Room in the Wanamaker Building in Philadelphia, with over 400 guests including patients, caregivers, and biotech industry leaders.
