The Emily Whitehead Foundation, established by Tom and Kari Whitehead, the parents of Emily Whitehead, the first pediatric patient to receive life-saving CAR T-cell therapy, is significantly expanding its mission. Previously focused on pediatric cancer and exclusively CAR T-cell therapy, the Foundation is now extending its policy advocacy and patient support to benefit all cancer and rare disease patients, and to ensure access to all types of advanced therapies.
The Emily Whitehead Foundation Helps Accelerate and Increase Access to Life-saving Advanced Therapies.
This pivotal evolution reflects the Foundation’s enduring commitment to Activate the Cure for more individuals facing life-threatening conditions. Emily Whitehead ignited a global movement to advance CAR T-cell therapies and drive continued innovation of other advanced therapies by overcoming acute lymphoblastic leukemia (ALL) at the age of five.
Now, over a decade after her groundbreaking CAR-T treatment, with Emily thriving and considered cured, the Foundation is uplifting the broader potential of advanced therapies.
Access to the revolutionary CAR T-cell therapy
“When we started the Emily Whitehead Foundation, our sole focus was on ensuring other children with pediatric cancer had access to the revolutionary CAR T-cell therapy that saved Emily’s life,” said Tom Whitehead, co-founder of the Emily Whitehead Foundation.
“As the field of cell and gene therapies has rapidly advanced, we’ve seen the incredible potential of these innovative treatments to transform lives across a wider spectrum of diseases. It’s time to extend our reach and fight for every patient who can benefit from these life-changing breakthroughs,” Whitehead said.
The Emily Whitehead Foundation’s expanded mission will encompass:
- Broader Disease Focus: Advocating for patients with all types of cancer and a range of rare diseases for which advanced therapies offer promising avenues for treatment.
- Comprehensive Therapy Scope: Supporting awareness, access, and research across the full spectrum of innovative treatments, moving beyond CAR T-cells to include gene editing, stem cell therapies, and other cutting-edge treatment approaches.
- Policy and Public Advocacy: Advancing policies that support scientific innovation, accelerate the development of groundbreaking therapies, and ensure equitable access for all patients.
- Enhanced Patient and Family Support: Continuing to provide vital resources, connection to experts, and emotional support to patients and their families navigating complex treatment journeys, now for a wider patient population.
Amplify the voices of more patients
“By broadening our mission, we will further amplify the voices of more patients, connect more families with critical resources, and contribute to a future where advanced therapies are a viable option for everyone who needs them,” noted Emily Whitehead Foundation Executive Director George Eastwood.
“By working closely with policymakers, building strategic coalitions, and championing reforms that remove barriers to care, we are committed to shaping a policy environment that enables faster cures and broader impact for individuals and families everywhere,” Eastwood added.
New identity
Symbolizing the Foundation’s broader mission, the organization has unveiled an updated brand identity. A new logo retains visual similarities to the Foundation’s original logo to capitalize on its existing recognition, but now features a sun icon to represent a new era of hope. The sun icon is completed with “Emily Whitehead Foundation,” reinforcing that hope is found through the Foundation’s work and support.
Additionally, the Foundation has launched a new website featuring information on advanced therapies, patient support resources, patient and family stories, signature events, grassroots initiatives, and ways for organizations and individuals to contribute to the cause.
An Extraordinary History
At five years old, Emily Whitehead was diagnosed with acute lymphoblastic leukemia (ALL), the most common childhood cancer, which constitutes approximately 25 % of cancer diagnoses among children under 15 years of age. Although the disease typically has an 85%-90% chance of being cured in children as a result of progressive improvement in the efficacy of risk-adapted multiagent chemotherapy regimens, relapsed/refractory childhood ALL is associated with poor outcomes. As a result, ALL still represents the leading causes of cancer-related deaths.[1][2][3]
However, Emily’s cancer resisted for over 16 months of the standard chemotherapy treatment protocol. After relapsing twice following unsuccessful chemotherapy and a disease that had progressed so rapidly that she was ineligible for a bone marrow transplant to treat it, her medical team told her parents, Tom and Kari Whitehead, that Emily would not survive. They recommended hospice care, allowing her to die peacefully, surrounded by loved ones.
Holding on to the belief that Emily could be cured, Emily’s parents discovered a novel CAR T-cell therapy available as a clinical trial at the Children’s Hospital of Philadelphia (CHOP). They enrolled her in an experimental clinical trial for CAR T-cell therapy, hoping that the team at CHOP, led by Stephan Grupp, MD, Ph.D, a pioneer in the field of cellular immunotherapy,* could help.
At the time, Grupp and his research team, working in collaboration with Carl H. June, MD, Bruce L. Levine, Ph.D., and David L. Porter, MD, at the University of Pennsylvania Medicine’s Abramson Cancer Center, were about to enroll patients in the first phase 1 trial for CAR T-cell therapy in pediatric patients diagnosed with ALL. Their decision was based on the early success Penn scientists had seen in three adult patients with chronic lymphocytic leukemia.[4] What happened next was a real medical miracle.
Previously untried on a pediatric patient, CAR T-cell therapy reprograms a patient’s own immune cells to fight cancer. As the first pediatric patient treated with CAR-T, Emily became the international face of the advanced-therapy revolution, inspiring a global shift toward curative treatments.
A novel CAR T-cell therapy was Emily’s last chance for survival. In March 2012, she became the first child with cancer to receive CAR T-cell therapy when her T-cells were harvested and reengineered to become chimeric antigen receptor (CAR) T-cells to target CD19, a protein found on the surface of ALL cancer cells. Nearly a month later, Emily received the first pediatric CAR T-cell therapy.
Emily’s treatment triggered a reaction now known as cytokine release syndrome (CRS), an on-target and the most common type of toxicity of CAR T-cell therapy, which represents a systemic inflammatory response resulting from the overproduction of inflammatory cytokines characterized by systemic immune activation resulting in fever, hypotension, respiratory insufficiency, and capillary leak. [5] With a worsening condition, her parents were (again) told to prepare for the inevitable. However, noticing that interleukin-6 (IL-6), a pro-inflammatory cytokine, was particularly elevated, Emily’s medical team treated her with tocilizumab (Actemra®; Genentech/Roche), at the time, a newly approved drug for the treatment of arthritis that blocks, bringing the adverse event under control.** [6][7][8]
Notwithstanding the initial side effects, the novel engineered CAR T-cell treatment worked, *** and Emily’s cancer went into complete remission. In June 2012, at age 7, Emily was discharged from the hospital, and today she is thriving and considered cured.[9][10]
Foundation
In 2015, three years after Emily completed her treatment, the Whitehead family established the Emily Whitehead Foundation to increase awareness of advanced therapies and the need for wider access to treatment..
Since its inception, the Foundation has connected countless families with experts and clinical trials, helped pave the way for over 45,000 individuals worldwide to access CAR T-cell therapy, and raised over $4.5 million for revolutionary research to help more patients live long, whole lives.
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Note: * Stephan A. Grupp, MD, PhD, is Section Chief of the Cellular Therapy and Transplant Section, Inaugural Director of the Susan S and Stephen P Kelly Center for Cancer Immunotherapy, and Medical Director of the Cell and Gene Therapy Laboratory and Children’s Hospital of Philadelphia. He holds the Yetta Deitch Novotny Endowed Chair in Pediatric Oncology.
** Today, tocilizumab is a standard part of the CAR T-cell treatment protocol for patients who present with cytokine release syndrome (CRS).
*** On August 30, 2017, five years after Emily was treated, the U.S. Food and Drug Administration (FDA) approved tisagenlecleucel (Kymriah®; Novartis), the CAR T-cell therapy that was used to treat her.
Highlights of Prescribing Information
Tocilizumab (Actemra®; Genentech/Roche) [Prescribing Information]
Tisagenlecleucel (Kymriah®; Novartis)[Prescribing Information]
Reference
[1] Tran TH, Hunger SP. The genomic landscape of pediatric acute lymphoblastic leukemia and precision medicine opportunities. Semin Cancer Biol. 2022 Sep;84:144-152. doi: 10.1016/j.semcancer.2020.10.013. Epub 2020 Nov 13. PMID: 33197607.
[2] Pui CH, Yang JJ, Hunger SP, Pieters R, Schrappe M, Biondi A, Vora A, Baruchel A, Silverman LB, Schmiegelow K, Escherich G, Horibe K, Benoit YC, Izraeli S, Yeoh AE, Liang DC, Downing JR, Evans WE, Relling MV, Mullighan CG. Childhood Acute Lymphoblastic Leukemia: Progress Through Collaboration. J Clin Oncol. 2015 Sep 20;33(27):2938-48. doi: 10.1200/JCO.2014.59.1636. Epub 2015 Aug 24. PMID: 26304874; PMCID: PMC4567699.
[3] Rheingold SR, Ji L, Xu X, Devidas M, Brown PA, Gore L, Winick NJ, Carroll WL, Hunger S, Raetz EA, Loh ML. Prognostic factors for survival after relapsed acute lymphoblastic leukemia (ALL): A Children’s Oncology Group (COG) study. J. Clin. Oncol., 37 (2019), p. 10008
[4] Kalos M, Levine BL, Porter DL, Katz S, Grupp SA, Bagg A, June CH. T cells with chimeric antigen receptors have potent antitumor effects and can establish memory in patients with advanced leukemia. Sci Transl Med. 2011 Aug 10;3(95):95ra73. doi: 10.1126/scitranslmed.3002842. PMID: 21832238; PMCID: PMC3393096.
[5] Brudno JN, Kochenderfer JN. Toxicities of chimeric antigen receptor T cells: recognition and management. Blood. 2016 Jun 30;127(26):3321-30. doi: 10.1182/blood-2016-04-703751. Epub 2016 May 20. PMID: 27207799; PMCID: PMC4929924.
[6] Acharya UH, Dhawale T, Yun S, Jacobson CA, Chavez JC, Ramos JD, Appelbaum J, Maloney DG. Management of cytokine release syndrome and neurotoxicity in chimeric antigen receptor (CAR) T cell therapy. Expert Rev Hematol. 2019 Mar;12(3):195-205. doi: 10.1080/17474086.2019.1585238. Epub 2019 Mar 18. PMID: 30793644.
[7] Stitt GA, Hall MW, Diliberto MA, Zuppa AF. Real-World Tocilizumab Use in Pediatric Inpatients. J Pediatr Pharmacol Ther. 2025 Oct;30(5):638-644. doi: 10.5863/JPPT-24-00130. Epub 2025 Oct 17. PMID: 41112348; PMCID: PMC12533711.
[8] Barrett D. IL-6 Blockade in Cytokine Storm Syndromes. Adv Exp Med Biol. 2024;1448:565-572. doi: 10.1007/978-3-031-59815-9_37. PMID: 39117839.
[9] Awasthi R, Maier HJ, Zhang J, Lim S. Kymriah® (tisagenlecleucel) – An overview of the clinical development journey of the first approved CAR-T therapy. Hum Vaccin Immunother. 2023 Dec 31;19(1):2210046. doi: 10.1080/21645515.2023.2210046. Epub 2023 May 15. PMID: 37185251; PMCID: PMC10294746.
[10] Bouzianas D, Bouziana S. First pediatric B-acute lymphoblastic leukemia patient treated with anti-CD19 chimeric antigen receptor T-cell therapy: Long-term remission or early cure? Hum Vaccin Immunother. 2024 Dec 31;20(1):2321678. doi: 10.1080/21645515.2024.2321678. Epub 2024 Feb 25. PMID: 38402637; PMCID: PMC10898498.
Featured image: Emily Whitehead and Stephen A. Grupp, MD, Ph.D. Image courtesy © 2025 The Children’s Hospital of Philadelphia. Used with permission.
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