What's Next in Cell and Gene Therapies: Anticipating the Next Advanced Therapy Breakthroughs
Insights from the "Bridging the Gap" Webinar, February 2024
Gene therapy has emerged as a promising frontier in modern medicine, offering revolutionary treatments for a range of diseases previously considered incurable. In a recent webinar as part of the Bridging the Gap series, guest speaker Dr. Bruce Levine, a cancer gene therapy professor and founding director of the Clinical Cell and Vaccine Production Facility (CVPF) at the University of Pennsylvania, along with four other co-hosts and panelists, shared key insights about the current state and future prospects of gene therapy.
Co-inventor of the First FDA-Approved Gene Therapy, Kymriah
Dr. Levine is a prominent figure in gene therapy, spearheading groundbreaking research and trials. He's been instrumental in advancing CAR T-cell therapy for cancer and driving progress in gene editing, notably with CRISPR. His expertise drives innovative projects and trials, aiming to enhance patient outcomes and transform healthcare.
During the webinar, Dr. Levine reflected on his early experiences working with patients and how that shaped his dedication to translational research. He discussed breakthroughs in cell and gene therapy, including recent approvals for sickle cell treatments and advancements in gene editing.
Webinar Highlights
In addition to insights about the development of current therapies, Dr. Levine and the panel of speakers explored the future of therapy delivery, including potential off-the-shelf treatments and the importance of specialized centers for administering these therapies.
Let's delve into the highlights from this enlightening discussion.
Viral Vector Advancements: One of the pivotal aspects of gene therapy lies in the delivery of therapeutic genes to target cells. Dr. Levine highlighted significant advancements in viral vector production that are making the process more efficient and cost-effective. Moreover, the evolution of gene editing technologies like CRISPR presents new opportunities for precise genetic modifications. "Viral vector production is getting more efficient. It's getting less expensive,” said Dr. Levine.
Accelerated Release Testing: A major challenge in gene therapy development is the lengthy process of release testing, which delays the availability of treatments to patients. The webinar emphasized the need for accelerated release testing methods to streamline the process and reduce turnaround time, ultimately expediting patient access to life-saving therapies. "We have some newer technologies in development... that can significantly accelerate the release,” said Dr. Levine.
Exciting Clinical Trials: Several groundbreaking projects in gene therapy clinical trials were discussed, offering hope for patients with various diseases. These include shortened manufacturing processes for CAR T-cell therapy, innovative brain cancer trials targeting specific antigens, and novel approaches using gene editing to tackle challenging leukemias.
Panelist Al Ribickas, Assistant Director of Cell Therapy Facility Operations at the Moffitt Cancer Center in Tampa, Florida pointed out an initiative undertaken by CAR-T families aimed at gathering comprehensive information about their children's treatment outcomes.
“With over 550 families in a private Facebook group, they are seeking approval to share relevant data with scientists and researchers,” he said. “This collaborative effort aims to provide valuable insights into the efficacy and long-term effects of CAR-T therapy, ultimately contributing to the improvement of future treatments for patients undergoing similar experiences.”
Concerns About Unproven Therapies: Despite the promising advancements in gene therapy, there are concerns about the proliferation of unproven cellular therapies marketed directly to consumers. These therapies lack regulatory oversight and may pose risks to patients, which highlights the importance of informed decision-making and adherence to established clinical trial protocols.
“Unfortunately, the FDA doesn't have the staff to close these down, and neither does the Federal Trade Commission,” said Dr. Levine. “They're operating out in the open with impunity, preying on the hope of patients who have no options." It’s important for patients to educate themselves about how to find legitimate treatments, panelists said.
In addition, Tom Whitehead emphasized the value of the Emily Whitehead Foundation in keeping track of patients, gathering valuable information for scientists, and helps patients find legitimate programs. He emphasized the need for qualified clinical sites to ensure patient safety and efficacy.
Personal Milestones and Reflections: Dr. Levine shared personal milestones in his career, including witnessing the transformative impact of gene therapy on patients' lives. From the success of CAR T-cell therapy to groundbreaking advancements in treating previously incurable diseases, these experiences underscore the profound potential of gene therapy to revolutionize healthcare.
"Things happen slowly, sometimes,” said Dr. Levine. “But sometimes there are ah ha moments. Like with our first CAR-T patients – with the assays that came in the early days after treating Emily and you see leukemia go away, and you have to just step back and say look at where are we now, and where can we go from here?”
Conclusion
The webinar provided valuable insights into the current landscape and future directions of gene therapy. While significant progress has been made, there are still challenges to overcome, particularly in ensuring the safety and efficacy of emerging therapies. By leveraging the latest technologies and maintaining a commitment to rigorous scientific standards, gene therapy holds immense promise for addressing unmet medical needs and improving patient outcomes.
Bridging the Gap Addresses Important Topics
The “Bridging the Gap Series” webinar provided a comprehensive overview of the current landscape of cellular therapies, accreditation challenges, and FACT's efforts to enhance patient care and access to innovative treatments. The commitment of organizations like the Emily Whitehead Foundation and the collaborative initiatives led by FACT underscored the collective determination to advance cellular therapies and improve outcomes for patients worldwide.
As we reflect on the insights shared during this insightful event, it is evident that a collaborative and forward-thinking approach is crucial to addressing the evolving needs and challenges in the field of cellular therapies.
Want to know more? Register for upcoming webinars in the series (held monthly on the first Friday).
About Dr. Levine
![Dr. Levine](/sites/default/files/web-media-library/blog/whats-next-cell-and-gene-therapies/Levin.png)
Dr. Bruce Levine is the Barbara and Edward Netter Professor in Cancer Gene Therapy at the University of Pennsylvania and is a distinguished figure in the field of immunology and cancer research. Dr. Levine's contributions include co-inventing the first FDA-approved gene therapy, Kymriah, and a remarkable 31 issued US patents with an extensive publication record. Dr. Levine continues to drive innovation as a Co-Founder of Tmunity Therapeutics and Capstan Therapeutics, both born out of the University of Pennsylvania. His accolades, such as the William Osler Patient Oriented Research Award and the Wallace H. Coulter Award for Healthcare Innovation, underscore his significant impact in advancing cell and gene therapy.