Reflections on Past, Present and Future Milestones in Immunotherapy

September 30, 2024 by Aldevron

From Emily Whitehead and Beyond

Emily Whitehead is a household name in the rare disease community. In 2010, she was diagnosed with acute lymphoblastic leukemia at just five years old. Her leukemia became resistant to treatment, and she relapsed twice, leaving her with limited options. Her last hope was to enroll in the highly experimental clinical CAR T-cell therapy trial at Children’s Hospital of Philadelphia (CHOP) where she bravely underwent a first-of-its-kind treatment. As the first child to receive CAR T-cell therapy, Emily and her story gained national attention, and she has been cancer free ever since!

The success of this clinical trial at CHOP set the stage for all future immunotherapies to come, serving as the most significant milestone in G&C therapy and showcasing the life-saving potential of such treatments. Emily’s incredible story provides hope for cancer patients and their families across the country. It still serves as an inspiration for us at Aldevron, as we strive to advocate for rare disease patients, like Emily, each day.

As part of our Advocacy blog series, we’re reflecting on the overall progress of the immunotherapies field over the past decade, while looking back on Aldevron’s notable contributions to these innovations.

Achievements Made Since Emily’s Treatment in 2012
In the 12 years since Emily’s clinical trial, the industry has made significant strides in the manufacturing and regulation of immunotherapies. In 2017, the FDA approved the first CAR T-cell therapy in the United States: Kymriah, a genetically modified autologous T-cell immunotherapy for cancer patients. Since then, the FDA has approved nearly 40 cell and gene therapies, showing the rapid progress and enormous potential of this field. There are currently six FDA-approved CAR-T therapies, and more than 1,000 programs in ongoing clinical trials.

Our work at Aldevron has been instrumental in this journey, playing a crucial role in developing some of the most innovative and life-saving therapies. We have supported over 1,000 clients in turning their possibilities into reality by manufacturing high-quality DNA, RNA, gene-editing nucleases and other proteins. These are essential components that have enabled such advancements in the field. Today, 15 commercial cell and gene therapy programs depend on Aldevron’s products and services.

Challenges to Address
Despite the exciting advancements, there are still complex hurdles we must confront. Since Emily’s treatment, more than 30,000 children have been treated, but that’s only a fraction of the need that exists. About 400,000 children are diagnosed with cancer each year, so it is important that we continue to develop more efficient processes and maximize the impact of cellular immunotherapies.

The most significant challenge to address is that many patients cannot afford these life-saving treatments or must travel long distances to access them. The high cost of materials and development pose barriers, especially given how many patients desperately need these treatments. The industry as a whole must uncover new strategies to decrease costs and improve access, while keeping safety the top priority.

To truly make a difference, collaboration across the healthcare ecosystem is essential. We must reduce manufacturing costs through automation and work with payers to ensure treatments are more affordable and accessible for patients. Additionally, we need to increase the number of health systems that can administer these therapies, so patients who are already struggling do not have to travel long distances to receive care. By working together, we can transform pediatric cancer treatment and continue to maximize the impact of our innovations.

Future Ahead
Reflecting on our achievements in the immunotherapy space, we take pride in our contributions and remain dedicated to helping create more impactful and accessible treatments. We are committed to the future of gene and cell therapy, which promises to treat a wider array of conditions and expand the number of patients who can benefit. For instance, over the next five to ten years, we hope to see immunotherapies that can treat solid tumors, autoimmune disease, cardiovascular disease, general pain management, and more. The possibilities for cell and gene therapy are truly limitless.

As cancer and rare diseases impact so many of our lives, we are driven by the urgency that patients and their families feel. With the launch of our advocacy program, New Frontiers in Cellular Immunotherapies, we draw continuous inspiration from patients’ stories like Emily’s. Together, we are committed to pioneering innovative solutions and forging a path toward an exciting future of immunotherapies.

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