Immunotherapy for aggressive T-cell cancers developed by WashU Medicine researchers moves to faster approval pathway
A cell-based immunotherapy designed to treat rare and aggressive types of blood cancer has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA). Developed by researchers at Washington University School of Medicine in St. Louis, this innovative CAR-T cell therapy is licensed to Wugen, a WashU Medicine startup biotechnology company based in St. Louis’ Cortex Innovation District.
The immunotherapy was developed by WashU Medicine physician-scientists who treat patients at Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine.
The therapy — called WU-CART-007 (soficabtagene geleucel) — targets specific blood cancers called T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LL). These are aggressive forms of blood cancer that originate in the immune system’s T cells, a type of white blood cell important for the body’s immune response. These cancers often don’t respond at all to standard care or return after several rounds of treatment, leaving patients with few treatment options and poor prognoses. Stem cell transplantation is the only curative treatment for such cancers, but these patients rarely qualify for it because they must first achieve remission following early rounds of chemotherapy, which is rare for these blood cancers.
The FDA’s Breakthrough Therapy designation aims to speed up the development and regulatory review of treatments for serious or life-threatening conditions, especially therapies that may offer substantial improvements over existing options. The Breakthrough Therapy designation for Wugen’s immunotherapy is based on preliminary clinical evidence showing early success in treating these aggressive blood cancers. Early-phase clinical studies have demonstrated that the therapy can selectively target and eliminate cancerous T cells with manageable side effects.
About 1,000 people are diagnosed with T-cell cancers each year in the U.S. If the cancer does not respond to treatment or returns after initial treatment, patients survive an average of six months, and fewer than 7% are still living at the five-year mark.
“This therapy has the potential to enable long-term survival for this patient population by controlling the disease and allowing patients — who would otherwise not be eligible — to proceed to stem cell transplantation, the only potentially curative treatment for these blood cancers,” said WashU Medicine oncologist John F. DiPersio, MD, PhD, the Virginia E. & Sam J. Golman Professor of Medicine and director of WashU Medicine’s Center for Gene and Cellular Immunotherapy, who first developed the therapy in his lab at WashU Medicine. “We remain hopeful that the ongoing Phase 2 study will be completed soon, and we’ll have positive results — but we’ll need some time to see how the patients do in both short-term and long-term follow-up.”
DiPersio treats patients at Siteman Cancer Center and founded Wugen alongside other WashU Medicine investigators, including Matthew Cooper, PhD, who then was on the WashU Medicine faculty and now serves as Wugen’s chief scientific officer. The researchers worked with WashU’s Office of Technology Management (OTM) to launch the company in 2018.
The early-phase clinical trial that led to the Breakthrough Therapy designation was conducted in multiple study centers in the U.S., Australia and Europe. The Phase 1 study included 28 adult and adolescent patients with either T-cell lymphoblastic cancer that returned after several lines of therapy or that never responded to treatment. Of 11 patients who could be evaluated after treatment, the overall response rate was 91%, meaning 10 patients either showed no signs of cancer after treatment or their cancer cell burden was reduced significantly. Eight out of 11 patients (72.7%) achieved complete remission. At the study’s data cutoff, six who underwent a transplant remained in remission, with no evidence of disease six to 12 months later, according to the study published in the journal Blood.
“This FDA Breakthrough Therapy designation for soficabtagene geleucel highlights the role of Siteman Cancer Center, a leading NCI-designated Comprehensive Cancer Center, and WashU Medicine in advancing innovative CAR-T cell therapies for aggressive T-cell leukemias and lymphomas,” said Timothy J. Eberlein, MD, director of Siteman Cancer Center and the Spencer T. and Ann W. Olin Distinguished Professor at WashU Medicine. “The dedicated work of our physician-scientists and clinicians is translating the most cutting-edge cellular immunotherapy research into the newest treatment options for patients with relapsed or refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic lymphoma.”
The phase 2 trial is currently ongoing. At the Siteman site, the clinical trials have been led by principal investigator Armin Ghobadi, MD, a professor of medicine, director of cellular therapies at Siteman, and clinical director of WashU Medicine’s Center for Gene and Cellular Immunotherapy. Siteman Kids at St. Louis Children’s Hospital and WashU Medicine is a key site for the pediatric portion of the clinical trial, co-led by Thomas Pfeiffer, MD, an assistant professor of pediatrics. Ghobadi and Pfeiffer have no financial interest in Wugen.
A major advantage of the treatment is its “off-the-shelf” availability, eliminating the need to manufacture an individualized cell product for each patient. The cell therapy can be prepared in advance from cells donated by healthy individuals and used to treat any patient with a T-cell cancer. In contrast, already-approved CAR-T cell therapies are adapted from the patient’s own immune cells, a process that typically takes three to four weeks. The accelerated treatment timeline of the Wugen immunotherapy reduces logistical and financial barriers associated with most cell-based therapies. This speed can make a meaningful difference because it is not unusual for patients with these aggressive cancers to die while waiting for the therapeutic cells to be prepared.
These particular blood cancers present a unique challenge because the therapeutic cells and the cancer cells are both T cells, so DiPersio and his colleagues came up with further innovations to prevent the therapeutic T cells from mistaking one another for the cancer and causing CAR-T cell fratricide. All other approved CAR-T cell therapies target B cell cancers, which do not have this T cell self-targeting complication.
Stewart, who is also the Gerty Cori Professor and vice chair of the Department of Cell Biology and Physiology at WashU Medicine, will be sworn in at the AACR Annual Meeting, April 17–22 in San Diego.
AACR is one of the world’s largest cancer research organizations, with more than 60,000 members in 143 countries and territories. As a board member, Stewart will work with other leaders to oversee the strategic direction, financial health and governance of the organization. Her term continues until 2029.
“I’m honored to serve on the AACR Board of Directors at such a pivotal time for cancer research. Indeed, while there are significant challenges to sustained funding and ensuring the success of the next generation of cancer researchers, our understanding of the processes that drive cancer is providing enormous opportunities for identifying new therapeutic targets. I look forward to working with this extraordinary community to accelerate discoveries that improve patients’ lives,” Stewart said.
“This national leadership role reflects Siteman’s own commitment to shaping the future of cancer care, and together we’re creating the partnerships and scientific momentum that will ultimately benefit patients everywhere.”
As a cancer biologist at Siteman Cancer Center and WashU Medicine, Stewart studies how noncancerous cells, known as stroma, in tumors promote the development of cancer. In particular, she and her lab investigate how age-related changes in the stroma modulate the immune response and affect dormant tumor cells, thus promoting disease. The goal is to identify points of intervention at which the process can be interrupted, thereby preventing or treating cancer.
As vice chair of the Department of Cell Biology and Physiology at WashU Medicine, she develops strategies and programs to support the professional and personal development of the department’s trainees, as well as to enhance faculty recruitment and professional development.
Stewart earned her bachelor of science degree in microbiology from the University of Minnesota and her PhD in microbiology and immunology from the University of California-Los Angeles (UCLA). She completed her postdoctoral fellowship in cancer biology at the Whitehead Institute at Massachusetts Institute of Technology. She joined the WashU Medicine faculty in 2003.
Led by Kelly Chase, the March 27 event brings NHL legends and special guests together to raise funds for cancer research, patient care and lifesaving advances.
Siteman Cancer Center is honored to once again be a beneficiary of the annual St. Louis Blues alumni game led by Kelly Chase, returning for its third year on March 27.
Tickets are available now for this special evening, which includes other National Hockey League (NHL) alumni and special guests — a powerful show of support for cancer research, patient care and Chase’s own battle with cancer.
A veteran for 12 seasons in the NHL, seven with the Blues, Chase has been a bruising defender, including against his diagnosis of acute myeloid leukemia (AML).
Thanks to the incredible generosity of the community, the first two events raised more than $1.2 million in support of Siteman. This year’s game promises to build on that momentum with another unforgettable night.
Proceeds from the March 27 event at Centene Community Ice Center in Maryland Heights will benefit Siteman and The V Foundation for Cancer Research, helping to advance lifesaving work and provide hope to patients and families.
Fans can purchase general admission tickets for $50 or VIP tickets for $500. VIP packages include all-inclusive food and beverage, along with exclusive access to a postgame party featuring players and celebrity guests.
Scheduled appearances include Blues greats and other notable guests such as Brett Hull, David Backes, Brendan Shanahan, Dierks Bentley and Sean Payton, among others.
The three-year award will significantly advance a unique viral-based immunotherapy for glioblastoma, a lethal brain cancer that typically results in death within two years.
Chheda’s research repurposes the Zika virus, which has been linked to neurological adverse events. Instead, Chheda and colleagues engineered and tested the virus to selectively target and destroy glioblastoma tumor cells while sparing healthy brain tissue. The virus has proven highly effective in mice, giving a powerful boost to an immunotherapy drug and training the immune system to surveil and prevent against recurrence. The Kleberg Foundation award is essential for sustaining momentum, allowing Chheda and his team to complete key work needed to transition to a first-in-human clinical trial. Chheda previously received a Kleberg Foundation grant in 2017 to support this work.
Glioblastoma is the most common and aggressive form of brain cancer. About 12,000 people are diagnosed each year in the U.S.
Chheda also is director of neuro-oncology at WashU Medicine and a physician-scientist and associate director at The Brain Tumor Center at Siteman.
Recognition follows strong early clinical results for a novel off-the-shelf CAR T therapy targeting rare and aggressive T-cell malignancies
A novel off-the-shelf CAR T-cell therapy pioneered at Siteman Cancer Center at Barnes-Jewish Hospital and WashU Medicine is gaining national recognition after delivering striking clinical results in patients with rare and aggressive blood cancers.
The FDA has granted Breakthrough Therapy Designation to WU-CART-007, an allogeneic anti-CD7 CAR T-cell therapy developed by WashU Medicine researchers. In early global trials, 73% of adults and adolescents with relapsed or refractory (R/R) T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma (T-ALL/LBL) achieved full remission following treatment — an outcome that positions the therapy as a potential gamechanger in T-cell malignancies.
“Relapsed T-cell leukemias and lymphomas represent one of the most challenging areas in hematologic oncology,” said oncologist John DiPersio, MD, PhD, director of the Center for Gene and Cellular Immunotherapy at WashU Medicine and an internationally recognized cell therapy leader at Siteman Cancer Center. “We are leading transformative advances for patients with these rare and aggressive cancers. Developing an off-the-shelf CAR T platform that can induce high remission rates in this population reflects the translational depth and cellular therapy infrastructure we’ve built at Siteman.”
DiPersio and Matthew Cooper, PhD, developed the therapy — manufactured using an off-the-shelf approach from healthy donors — to target CD7+ malignancies. The two founded the biotech company Wugen in 2018 to advance the research. Further clinical trials are underway in the U.S., Europe, Asia and Australia, including at Siteman Cancer Center and Siteman Kids at St. Louis Children’s Hospital.
In reviewing results from earlier clinical trials in children, researchers at Siteman Kids noted that WU-CART-007 (also known as soficabtagene geleucel, or sofi-cel) could be a gamechanger if the therapy continues to move almost all patients from disease-state to remission, thus enabling patients to undergo stem cell transplantation.
The rapid advance of WU-CART-007 is just the latest example of what Timothy J. Eberlein, MD, director of Siteman Cancer Center, says arises out of a robust eco-system of innovation, collaboration and excellence in cancer research at WashU Medicine.
Siteman is known internationally for its basic and translational research efforts and is one of only a few institutions to receive three prestigious Specialized Program of Research Excellence (SPORE) grants from the National Cancer Institute (NCI), for leukemia, endometrial and pancreatic cancer research.
Blood Cancer United, formerly known as the Leukemia & Lymphoma Society, also has awarded scientists at Siteman a Specialized Center of Research (SCOR) grant for lymphoma research. Such grants are specifically designed to accelerate promising translational research into patient care.
“We are committed to continually advancing treatments for cancer and broadening options for patients,” Eberlein said. “Toward that goal, we have initiated several home-grown clinical trials that have changed the course of treatment for many cancers. Our depth and breadth of oncology research is wide, and our expertise is the result of innovation, dedication and multidisciplinary cross-collaboration that occurs throughout our center.”
Other examples of comprehensive, specialized programs at Siteman include the:
Brain Tumor Center, known nationally and internationally for innovative care and neuro-oncology research, including multiple investigator-initiated clinical trials
Translational Research Highlights
In addition to the latest breakthrough in leukemia and lymphoma research and care, examples of other research efforts that have changed practice guidelines include:
New Standard of Care Established for Locally Advanced Head and Neck Cancers — In the first change in standard-of-care therapy in more than 20 years, the FDA approved the use of the immunotherapy drug pembrolizumab (Keytruda) for treatment of resectable locally advanced head and neck squamous cell carcinoma (HNSCC) in adults. The approval, announced in mid-2025, came after a clinical trial initiated at Siteman in 2013. That trial and later ones, including an international trial, demonstrated greater tumor shrinkage prior to surgery and longer survival rates when immunotherapy was added. “It’s exciting to see our ideas move toward clinical practice with such impressive and potentially life-changing results,” said Douglas Adkins, MD, co-director of the Head and Neck Tumor Center at Siteman, who co-led the clinical trials at Siteman and elsewhere.
Addition of Brentuximab Vedotin for Relapsed Diffuse Large B-Cell Lymphoma Results in Statistically Significant Survival Benefit — With approximately 40% of patients diagnosed with diffuse large B-cell lymphoma (DLBCL) having relapsed or refractory disease, researchers at Siteman, led by Nancy Bartlett, MD, found in the ECHELON-Phase Three clinical trial that the use of an antibody-drug conjugate brentuximab vedotin, when combined with either lenalidomide or rituximab, was not only safe but also demonstrated improved survival benefit in patients with R/R DLBCL.
Dostarlimab Plus Chemo for Primary Advanced or Recurrent Endometrial Cancer — Matthew Powell, MD, co-led national studies that found adding immune checkpoint inhibitors to standard therapy for endometrial cancer improves outcomes for many patients, with an average increase in overall survival of 31%.
T-cell Immunotherapy Effective in Treating Rare Soft Tissue Cancers — Siteman Cancer Center’s Sarcoma program was a major clinical trial site for this study, which found that T-cell immunotherapy, specifically the drug afamitresgene autoleucel, or afami-cel, was effective and generated long-term responses in patients with rare soft tissue cancers.
Sotorasib Approved as Targeted Therapy for Patients with Specific Type of Non-Small-Cell Lung Cancer — Following clinical trials at Siteman and globally, the FDA approved sotorasib for patients with non-small-cell lung cancer whose tumors express a G12C mutation in the KRAS gene and who have already undergone previous treatment. Ramaswamy Govindan, MD, who led the study, noted that the drug targeted the most common mutation, reduced tumor sizes, and improved overall survival rates.
Medicare Approves Whole-Genome Test for Blood Cancers — A test for acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) developed at Siteman was the first whole-genome sequencing test for cancer to be approved for reimbursement by the Centers for Medicare & Medicaid Services. Called ChromoSeq, the test is now routinely used by oncologists to guide treatment decisions for patients with blood cancers.
“At Siteman, we have built one of the world’s leading cellular immunotherapy programs focused on developing next-generation treatments for rare and refractory cancers,” Eberlein said. “Many of our most impactful cell therapy trials are investigator-initiated and originated here, reflecting a translational infrastructure designed to bring innovative therapies to patients with the most aggressive and rarest cancers.”
Recognition follows strong early clinical results for a novel off-the-shelf CAR T therapy targeting rare and aggressive T-cell malignancies
A novel off-the-shelf CAR T-cell therapy pioneered at Siteman Cancer Center at Barnes-Jewish Hospital and WashU Medicine is gaining national recognition after delivering striking clinical results in patients with rare and aggressive blood cancers.
The FDA has granted Breakthrough Therapy Designation to WU-CART-007, an allogeneic anti-CD7 CAR T-cell therapy developed by WashU Medicine researchers. In early global trials, 73% of adults and adolescents with relapsed or refractory (R/R) T cell acute lymphoblastic leukemia or T cell lymphoblastic lymphoma (T-ALL/LBL) achieved full remission following treatment — an outcome that positions the therapy as a potential gamechanger in T-cell malignancies.
“Relapsed T-cell leukemias and lymphomas represent one of the most challenging areas in hematologic oncology,” said oncologist John DiPersio, MD, PhD, director of the Center for Gene and Cellular Immunotherapy at WashU Medicine and an internationally recognized cell therapy leader at Siteman Cancer Center. “We are leading transformative advances for patients with these rare and aggressive cancers. Developing an off-the-shelf CAR T platform that can induce high remission rates in this population reflects the translational depth and cellular therapy infrastructure we’ve built at Siteman.”
DiPersio and Matthew Cooper, PhD, developed the therapy — manufactured using an off-the-shelf approach from healthy donors — to target CD7+ malignancies. The two founded the biotech company Wugen in 2018 to advance the research. Further clinical trials are underway in the U.S., Europe, Asia and Australia, including at Siteman Cancer Center and Siteman Kids at St. Louis Children’s Hospital.
In reviewing results from earlier clinical trials in children, researchers at Siteman Kids noted that WU-CART-007 (also known as soficabtagene geleucel, or sofi-cel) could be a gamechanger if the therapy continues to move almost all patients from disease-state to remission, thus enabling patients to undergo stem cell transplantation.
‘An Eco-System of Innovation and Excellence’
The rapid advance of WU-CART-007 is just the latest example of what Timothy J. Eberlein, MD, director of Siteman Cancer Center, says arises out of a robust eco-system of innovation, collaboration and excellence in cancer research at WashU Medicine.
Siteman is known internationally for its basic and translational research efforts and is one of only a few institutions to receive three prestigious Specialized Program of Research Excellence (SPORE) grants from the National Cancer Institute (NCI), for leukemia, endometrial and pancreatic cancer research.
Blood Cancer United, formerly known as the Leukemia & Lymphoma Society, also has awarded scientists at Siteman a Specialized Center of Research (SCOR) grant for lymphoma research. Such grants are specifically designed to accelerate promising translational research into patient care.
“We are committed to continually advancing treatments for cancer and broadening options for patients,” Eberlein said. “Toward that goal, we have initiated several home-grown clinical trials that have changed the course of treatment for many cancers. Our depth and breadth of oncology research is wide, and our expertise is the result of innovation, dedication and multidisciplinary cross-collaboration that occurs throughout our center.”
WashU Medicine grant recipients will focus on lung and blood cancers
Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine, is pleased to announce the next cohort of pilot projects funded by the Institutional Research Grant from the American Cancer Society. The three projects are described below.
Jason Weber, PhD
Under the leadership of Jason Weber, PhD, who has been principal investigator of the grant since 2011, these awards support independent, self-directed investigators early in their careers and enable them to conduct research in areas of special interest to the American Cancer Society.
WashU has funded early-career oncology researchers with this grant since 1958. Learn about projects initially supported in 2022, 2023, 2024 and 2025.
Project Title: Complement Modulation as a Strategy to Sensitize Tumors to Radiotherapy Radiotherapy Principal Investigator:Vaishali Kapoor, PhD
Vaishali Kapoor, PhD
Summary: Lung cancer is the leading cause of cancer-related deaths in the U.S., and non-small cell lung cancer (NSCLC) accounts for about 85% of all cases. Radiation therapy (RT) is one of the most common and effective treatments for NSCLC, used in combination with chemotherapy and immunotherapy. While many patients initially respond to RT, most eventually experience cancer recurrence. One reason for treatment failure is that tumors can change their surrounding environment to hide from the immune system and resist therapy. This research focuses on the complement system, a part of the body’s natural immune defense, which helps recognize and destroy harmful cells. Surprisingly, new evidence suggests that in cancer, activation of the complement system can have the opposite effect: Instead of helping the immune system, it may help tumors survive. Researchers have found that RT activates the complement system, releasing a molecule called C3a, which then signals through a receptor called C3aR to recruit cells that suppress the immune system. At the same time, RT generates another molecule called iC3b, which programs certain immune cells to become less effective at fighting cancer. In this project, the researchers will investigate how these processes occur and test whether blocking C3aR — using a drug that already exists — can reprogram the immune system, allowing it to work together with RT to fight cancer more effectively. The long-term goal is to develop new combination therapies that enhance the power of RT, make immunotherapies more effective and improve survival for patients with lung cancer.
Project Title: Glycan-Guided Pathomic Signatures of Immunotherapy Response in Non-Small Cell Lung Cancer
Summary: A type of treatment called immunotherapy has transformed care for some patients with advanced lung cancer by helping the immune system recognize and attack cancer cells. Unfortunately, this treatment does not work for everyone, and doctors currently lack reliable ways to know in advance which patients will benefit. As a result, many patients are exposed to treatments that may not help them while losing valuable time for other therapies. Researchers will explore whether patterns inside tumor tissue can help predict which patients are most likely to respond to immunotherapy. They will use lung tissue samples that were already collected from patients treated by WashU Medicine physicians. With the help of advanced imaging techniques, the researchers can map molecules such as sugars and proteins within the tumor. These maps will then be linked to digital images of the same tissue under the microscope. By analyzing these images with computer algorithms, scientists can detect subtle patterns that are invisible to the human eye. The goal of this work is to identify new patterns in tumor tissue that indicate whether a patient will respond well to immunotherapy. If successful, this approach could improve the way doctors select treatments for lung cancer, ensuring patients receive therapies that are most likely to help them. In the future, this research may also open the door to better tests that can guide treatment decisions for many other types of cancer.
Project Title: Germline Genome Sequencing in Patients with Myeloid Neoplasms in Paraguay Principal Investigator:Samuel Urrutia, MD, MS
Samuel Urrutia, MD, MS
Summary: This study is about understanding what causes myeloid neoplasms, a type of blood cancer, in a diverse population in Paraguay. While treatments for these cancers have improved in high-income countries, they’re often not available in places like Paraguay. A project called GEMA is already underway there, using advanced genetic testing to diagnose these cancers. This new project, an expansion of GEMA, aims to go a step further. It will look at two main things. First, it will search for inherited genetic changes (germline variants) that make people more susceptible to these cancers and see how they interact with new genetic changes (somatic alterations) that happen later in a person’s life. Second, the study will investigate how a person’s genes and their environment (gene-environment interaction) might work together to cause these cancers. To do this, researchers will analyze genetic information from saliva samples collected from patients. By combining this new information with data on their ancestry, environment and the genetic changes in their cancer cells, the study’s researchers hope to get a complete picture of why these cancers develop in this specific population. Ultimately, the goal is to identify new risk factors and better understand the unique challenges faced by patients with myeloid neoplasms in Latin America, which could lead to more effective prevention and treatment strategies in the future.
WashU researchers at Siteman Cancer Center are advancing breast imaging to help patients avoid invasive procedures and improve the screening experience
In a new discovery at Siteman Cancer Center at Barnes-Jewish Hospital and WashU Medicine, a team of researchers and physicians found that utilizing ultrasound-guided diffuse optical tomography technology can reduce unnecessary breast biopsy rates by nearly 25%.
Now for the first time in a clinical trial, Siteman Cancer Center is using these new methods first, rather than starting with a biopsy, to determine if additional diagnoses are needed to test for cancer. The research was recently published in Breast Cancer Research and supported by a nearly $2 million grant from the National Cancer Institute of the National Institutes of Health.
Every year in the United States, more than 1 million breast biopsies are performed. Yet 75-80% of those biopsies turn out to be benign, meaning patients undergo an invasive procedure, anxiety, and waiting — only to learn cancer is not present.
New Research in Breast Cancer Imaging
In a double-blind clinical trial involving 226 patients, the WashU Medicine research and physician team at Siteman found that ultrasound-guided diffuse optical tomography (DOT) — used alongside standard breast ultrasound — can reduce unnecessary benign biopsies by nearly 25%, while maintaining a false negative rate below 2%, consistent with American College of Radiology safety standards.
By combining standard ultrasound with diffuse optical tomography, radiologists gain additional information about tissue biology, not just structure.
Why This Matters for Patients Undergoing Breast Imaging
A biopsy remains the only way to definitively diagnose breast cancer. However, many suspicious findings on mammography or ultrasound are not cancer — and the uncertainty between imaging and biopsy can be one of the most stressful periods in a patient’s experience.
This research aims to improve how physicians distinguish which lesions truly require biopsy and which may be safely monitored. For patients, it means care that is not only the most advanced but thoughtfully designed to ensure that cancer is detected as early and screening is as accessible and low-cost as possible for more people.
Leadership in Breast Imaging Innovation
The study was led by:
Quing Zhu, PhD, Edwin H. Murty Professor of Engineering, WashU McKelvey School of Engineering
The study team also included Steven P. Poplack, MD, formerly of WashU Medicine and Siteman, who is now a professor of radiology (breast imaging) at Stanford University.
# # #
Zhu Q, Bennett D, Hagemann IS, Mannix J, Wiele K, Luther M, Luo J, Poplack SP. Ultrasound-guided diffuse optical tomography: An adjunct to ultrasound that can reduce unnecessary breast biopsies. Breast Cancer Research, published online Dec. 31, 2025. DOI: https://doi.org/10.1186/s13058-025-02206-3
Funding for this research was provided by the National Cancer Institute of the National Institutes of Health (R01CA228047).
Siteman Cancer Center at Barnes-Jewish Hospital and WashU Medicine is pleased to announce funding for 13 new projects, including two clinical trials and community outreach and engagement efforts.
Through this research, investigators aim to improve the understanding of tumor formation and growth, develop safer, more effective therapies, and remove barriers to precision medicine.
The funding is awarded through the Siteman Investment Program, which supports and accelerates the pace of innovation in cancer research. The money awarded comes from a variety of sources, including The Cancer Frontier Fund at The Foundation for Barnes-Jewish Hospital, which includes gifts from Pedal the Cause, the Foundation’s annual Illumination Gala, and donations throughout the year; the Cancer Center Support Grant (CCSG) from the National Cancer Institute; the Alvin J. Siteman Cancer Research Fund; Swim Across America – St. Louis; and various philanthropic gifts.
Please see below for more details about each funded project.
New Clinical Trial Category
Project Title: A Phase II, Single-Center, Open-Label Study of First-Line Ipilimumab plus Nivolumab and Nogapendekin Alfa Inbakicept (N-803) in Patients with Stage IV or Recurrent Non-Small Cell Lung Cancer (FLINN)
Goal: The study tests the hypothesis that adding nogapendekin alfa inbakicept (N-803), a drug that helps boost the immune system, to the already FDA-approved drug combination of nivolumab (anti-PD-1) and ipilimumab (anti-CTLA-4) will enhance anti-tumor responses in patients with stage IV or recurrent non-small cell lung cancer (NSCLC). Each drug activates the immune system through distinct but complementary mechanisms. Together, these agents may improve the depth and durability of clinical benefit compared with nivolumab and ipilimumab alone. In this study, patients will receive all three agents (nivolumab, ipilimumab, and N-803), and researchers will measure progression-free survival, overall clinical efficacy and the safety of this combination. They will also collect blood and tumor samples to understand how this treatment affects the immune system and the area around the tumor. The results of this study will clarify whether N-803 can further enhance the therapeutic effect of first-line immunotherapy and could establish a foundation for a future definitive trial aimed at improving outcomes for patients with advanced NSCLC.
Morgensztern
Project Summary: Lung cancer is the leading cause of cancer-related death in the U.S., and most patients are diagnosed only after the disease has already spread and can no longer be cured with radiation or surgery. At this stage, the main goals of treatment are to relieve symptoms, slow cancer progression and help patients live longer. One of the most important advancements in lung cancer care has been the development of immunotherapy, a class of medications that help the body’s immune system recognize and attack cancer cells. Two immunotherapy drugs, nivolumab and ipilimumab, are already FDA-approved for first-line treatment of advanced non-small cell lung cancer (NSCLC). These medicines work by “releasing the brakes” on the immune system, allowing immune cells to attack the cancer more effectively. Although some patients achieve a long-lasting response to the immunotherapy combination, most either do not benefit or eventually experience cancer progression, highlighting the need for more effective and durable treatment options. Our study will test whether adding a new medicine called nogapendekin alfa inbakicept (N-803) can improve how well nivolumab and ipilimumab work in controlling lung cancer. N-803 acts like a natural protein that boosts the activity of two key immune cells: natural killer (NK) cells and CD8+ T cells. Earlier research has shown that combining N-803 with nivolumab is safe and may help control the cancer. N-803 is already FDA-approved for the treatment of early-stage bladder cancer. By giving all three medicines together, we hope to strengthen the immune system’s ability to fight lung cancer and achieve a more durable response. If successful, this study could lead to a new, more effective, chemotherapy-free treatment option for patients with advanced lung cancer and pave the way for a larger national study.
Project Title: A Double-Blind, Placebo-Controlled Phase lb Study Evaluating the Safety and Toxicity of Recombinant Human IL-7 (NT-I7) in Relapsed/Refractory Multiple Myeloma Following BCMA CAR-T Therapy (Cilta-cel)
Goal: This is a two-arm, double-blind, placebo-controlled, randomized, phase Ib study testing the safety and toxicity of adding NT-I7 to BCMA CAR-T (standard of care) therapy in patients with relapsed/refractory multiple myeloma (RRMM). The hypothesis is that NT-I7 will help CAR T cells expand more and persist longer in the body, which will help get rid of multiple myeloma cells while still being safe. Patients receiving standard of care therapy will be randomized to either receive the addition of NT-I7 or a placebo. Correlative studies will evaluate CAR-T cell expansion, persistence, immune-phenotype, function and correlate with clinical outcomes.
Project Summary: Multiple myeloma is treated with medical therapy and stem cell transplant, but none of these therapies are curative. An alternate approach in myeloma therapy is to engineer patients’ own immune cells to detect and destroy myeloma cells. These chimeric antigen receptor T cell (or “CAR T cell”) therapies cause the cancer to shrink in 9 of 10 patients and can sometimes control myeloma for years, but are still not curative. Based on work in the researchers’ laboratory, the addition of a naturally occurring protein messenger called interleukin 7 (IL-7) can improve the ability of CAR T cells to get rid of blood cancer cells. In response to IL-7, CAR T cells divide more and persist longer, raising the possibility that the combination may be able to permanently eradicate myeloma cells. This study proposes the use of NT-I7, a long-acting version of IL-7, to improve myeloma-directed CAR T cell therapy. Similar drugs have been used to treat patients with severe infections and have been shown to be safe in that setting, with few side effects reported. By combining NT-I7 with standard-of-care CAR T cells, the researchers hope to enhance CAR T cell efficacy, achieve deeper remissions and achieve cures for patients with myeloma.
Pre-R01 Category
Project Title: Metabolic Control of Antigen Presentation in aCD40 Cancer Immunotherapy
Goal: To uncover how a chemical called itaconate affects the way our body recognizes and attacks harmful invaders called antigens. The researchers want to discover which antigens are most important for making our immune system respond strongly. By using advanced tools to study genes, metabolites and DNA, they will learn which immune cells are involved and how they can help the cells identify and destroy tumors more effectively.
Project Summary: This project explores how immunometabolic regulation within myeloid cells affects tumor antigen presentation and response to immunotherapy. The labs of Artyomov and Robert Schreiber, PhD, have long investigated mechanisms of tumor rejection, emphasizing the role of myeloid antigen-presenting cells. Their joint studies reveal that during effective immunotherapy, myeloid cells undergo a shift toward a pro-inflammatory and metabolically remodeled state — highlighting the metabolite itaconate as a key player in this transformation. Itaconate is produced in activated myeloid cells and has been shown to regulate immune responses. Preliminary data from the Artyomov lab show that mice deficient in itaconate (Irg1-/-) completely reject EG7 tumors when treated with αCD40 immunotherapy, while wild-type mice fail to do so. Mechanistically, itaconate inhibits GILT, a thiol reductase crucial for processing disulfide-rich neoantigens, thus impairing effective antigen presentation in wild-type settings. These findings support a novel hypothesis: Itaconate suppresses tumor antigen processing via GILT inhibition, thus limiting anti-tumor immunity. The significance and innovation of this proposal lie in several novel insights: (1) New Mechanistic Insight: It is the first to suggest that itaconate modulates cancer immunotherapy by directly impacting antigen processing through covalent modification of GILT and other endosomal proteases; (2) Complete Tumor Rejection Phenotype: Unlike previous studies focused on T cell–centered therapies like checkpoint inhibitors, αCD40 therapy in Irg1-/- mice results in complete tumor clearance, revealing the central role of myeloid metabolism in driving anti-tumor responses; and (3) Cutting-edge Tools and Expertise: The team possesses all required platforms — from in silico neoantigen prediction to in vivo validation — to dissect antigen presentation and immune responses.
Project Title: Defining and Targeting the Non-Transcriptional Functions of MYC in Acute Myeloid Leukemia
Goal: To understand the different ways the MYC protein helps leukemia grow, both inside and outside the cell’s nucleus. By figuring out how MYC moves around the cell and controls RNA, the researchers hope to uncover new weaknesses in leukemia cells. Ultimately, this work aims to create safer and more effective treatment strategies for patients with acute myeloid leukemia (AML).
Project Summary: AML is one of the most aggressive blood cancers. Many patients relapse after treatment, and older adults often cannot tolerate intensive options like chemotherapy or bone marrow transplant. Because AML is driven by many different genetic changes, it has been very difficult to develop effective, targeted and less toxic treatments. One protein, called MYC, is abnormal in almost every case of AML and is a major driver of the disease. MYC normally works inside the nucleus of the cell to turn genes on and off. However, we still do not fully understand how MYC causes leukemia, especially because traditional drugs that try to block its gene-regulating role have not worked well in patients. Our recent research shows that MYC does more than control genes. We found that certain mutations in MYC can cause leukemia by changing where MYC is located inside the cell and by altering how it interacts with RNA. These effects happen outside the nucleus and represent a completely different way MYC may drive cancer. These discoveries suggest that MYC has additional, “extranuclear” functions that help leukemia grow, and that these functions could be targeted separately from MYC’s usual gene-regulating role. By revealing these new and unexpected roles of MYC, this research aims to lay the groundwork for more effective and less toxic treatments for AML and potentially other MYC-driven cancers.
Project Title: The Role of Endogenous Memory NK Cells in Non-Small Cell Lung Cancer
Goal: To improve a type of immune cell called memory-like (ML) natural killer (NK) cells to help fight lung cancer. The researchers want to study these special immune cells in lung cancer patients to learn more about how they work. This understanding will give them the tools to develop better NK cell therapies for lung cancer. They will apply this knowledge to optimally design clinical trials that boost the patient’s own immune system to fight cancer.
Project Summary: Lung cancer is the leading cause of death from cancer. Recent therapies have been aimed at activating the patient’s immune system and have seen improved responses, but only a subset of patients have benefitted from this treatment. Therefore, new treatment options are needed. In this proposal, we are focused on natural killer (NK) cells, a type of immune cell that can kill cancer. We previously found that when we treat NK cells in the lab with three specific proteins, the NK cells develop memory, which means they are better at fighting cancer. For instance, lab-created memory NK cells have been shown to be effective at treating blood cancer. The researchers believe that memory NK cells could improve outcomes for solid tumor patients as well. They have found that memory NK cells were increased in lung cancer patients and that this increase remained even in lung cancer that has spread to the brain. However, researchers do not understand why memory NK cells, which are more effective at fighting blood cancer, are increased within lung cancer. This is the first study to identify that memory NK cells can reside in cancer without first creating them in the lab. This proposal will determine the ability of the endogenous — the body’s own — lung memory NK cells to fight off lung cancer. The researchers will measure the cells’ abundance. Also, they will measure the cells’ proximity to the tumor. Researchers will also identify how to can make the endogenous memory NK cells better at fighting cancer. These results are expected to provide critical data for longer-term funding. Working with collaborators, the researchers expect to develop a new clinical trial to activate endogenous memory NK cells in the future.
Project Title: A Novel Targeted Treatment Combination for BRAF Mutant Melanoma
Goal: To improve the effectiveness of current melanoma treatments to shrink tumors more and for a longer time. The researchers will test the combination of two FDA-approved drugs, dabrafenib and entrectinib, against human melanoma tumors previously isolated from patients and now grown in mice to mimic the natural behavior and growth environment of these tumors. This would establish a novel combination of FDA-approved drugs for melanoma that has a specific mutation (in a gene called BRAF) and no longer responds to current treatments. Success would support future clinical trials for this drug combination in humans.
Project Summary: Melanoma usually arises first on the skin and is extremely dangerous due to its tendency to spread, sometimes early, to other organs. Newly developed drugs have improved our ability to treat melanoma and prolong many patients’ lives. However, even our most quickly effective treatments, called targeted therapies, often work only for a limited time as melanoma eventually becomes resistant to these drugs and continues to grow and spread. For many patients, these targeted therapies are the last line of treatment that they can tolerate due to other medical issues or because of ongoing negative effects from prior treatments that overactivate the immune system. In this proposal, we seek to understand how melanoma becomes resistant to current targeted therapy and determine if we can overcome this resistance with other drug combinations. In our studies thus far, we have found an exciting new combination of existing and readily available drugs that may overcome this resistance in certain melanomas. This combination of two drugs works in resistant melanoma cells grown in the lab, and we now aim to test their effectiveness against human cells in mouse models. If successful, this study would provide essential support for future clinical trials for this drug combination in patients who have exhausted currently available treatments and who desperately need these additional treatment options for melanoma that would otherwise be nearly untreatable.
Project Title: Tumor-Associated Macrophage Modulated Radioimmunotherapy of Head and Neck Squamous Cell Carcinoma
Goal: Head and neck squamous cell carcinoma (HNSCC) is a serious type of cancer that is hard to treat successfully. The area around the tumor (called the tumor microenvironment, or TME) can weaken the body’s immune response, making treatments less effective. To tackle this problem, the researchers have developed a new type of treatment that uses radiation to target certain immune cells called tumor-associated macrophages (TAMs). By doing this, they hope to change the TME so other treatments, like chemotherapy or immunotherapy, work better. The goal is to create a targeted radiation therapy that attacks specific TAMs called CD163+ to improve treatment for people with HNSCC.
Project Summary: Despite advances in prevention and treatment, survival for HNSCC patients has minimally improved over the past 30 years. The substantial morbidity and mortality rates for HNSCC and the toxicity associated with the standard treatment options emphasize the need to seek alternatives. Targeted radionuclide therapy (TRT) is a kind of treatment that delivers radiation directly to the cancer cells to minimize damage to healthy cells. This method has helped improve outcomes for some types of cancer. However, TRT can still cause problems over time, like treatment resistance and cancer coming back. So, the researchers need new ways to use TRT for treating HNSCC. Tumor-associated macrophages (TAMs), especially CD163+ TAMs, are an essential component of the tumor microenvironment and maintain a critical role in orchestrating tumor progression, metastasis and resistance to therapies. The goal of this application is to develop CD163+ TAM- targeted therapy for HNSCC. The researchers believe that using a special probe labeled with radioactive copper (64Cu/67Cu) can help them see and change the tumor environment, making other treatments like immunotherapy work better.
Project Title: Targeting COPS5 to Overcome PARP Inhibitor Resistance in Ovarian Cancer
Goal: To develop and test a new therapy for the most common type of ovarian cancer by targeting a protein called COPS5. Through rigorous mechanistic, translational, and preclinical studies, this work aims to establish COPS5 as a target for a new therapy that will weaken the tumor and make it more susceptible to other known treatments, such as PARP inhibitors.
Project Summary: Most patients with ovarian cancer develop resistance to standard treatments, including platinum chemotherapy and PARP inhibitors, resulting in a low five-year survival rate. Once resistance develops, there are limited effective treatment options. This research focuses on a protein called COPS5, which helps cancer cells repair DNA and survive therapy. Early results show that blocking COPS5 makes resistant ovarian cancer cells more sensitive to PARP inhibitors and increases treatment-related DNA damage. Patients with high COPS5 levels have worse outcomes. In this project, the researchers will determine whether COPS5 is elevated in tumors that do not respond to PARP inhibitors, test whether blocking COPS5 safely strengthens PARP inhibitor effectiveness, and study how COPS5 helps cancer cells resist therapy. This work will lay the foundation for developing new treatments that overcome resistance and help more women benefit from PARP inhibitors.
Project Title: Predicting Response of HER2-low Breast Tumors to Trastuzumab-Deruxtecan Through Quantitative Imaging
Goal: The vast majority of patients with advanced breast cancer become resistant to anti-HER2 antibody-drug therapies. The use of predictive biomarkers (genes, proteins, or other molecules in the body) to guide antibody-drug response is necessary for improving survival in patients with breast cancer and for sparing them from unnecessary side effects. This proposal seeks to optimize a whole-body imaging approach that can identify how well Trastuzumab-drug conjugates will work against HER2-low breast tumors.
Project Summary: Breast cancer leads to a significant number of deaths each year. To reduce these numbers, we need effective ways to detect the disease and treat it. Antibody-drug conjugates, which are a combination of antibodies that specifically target cancer cells and a drug that kills them, have shown promise in treating breast tumors with high levels of a protein called HER2. Recently, some of these drugs have also been found to be effective in patients with tumors that have lower levels of HER2, but resistance occurs over time. In this proposal, the researchers will test approaches of whole-body imaging to identify which breast tumors will benefit most from antibody-drug therapies, reducing unnecessary side effects for those who may not respond well. In addition, the researchers will combine antibody drugs with other treatments to improve their efficacy. This approach will lead to future clinical trials that offer more effective options for diagnosing and treating breast cancer.
Project Title:Targeted Radionuclide Therapy for Cervical Cancer
Goal: To evaluate a new treatment for cervical cancer that uses a radioactive peptide that sticks to a specific protein (called integrin αvβ6) on the surface of tumor cells. If successful, this method will boost the effectiveness and safety of radiation treatment.
Project Summary: Cervical cancer is among the top cancers in incidence and mortality of young women worldwide, with about 350,000 cancer-related deaths per year. Following standard-of-care treatment, many locally advanced cervical cancer patients experience recurrence and have a five-year survival rate below 10%. Therefore, more sophisticated targeted therapeutic options are urgently needed to improve clinical outcomes. To address this, the researchers propose to deliver radiation in a specific manner to a protein that is highly expressed on cervical cancer cells but not on normal tissues. This protein is ideal for delivery of highly toxic radiation that can kill the cancer cells while not being toxic. The researchers have developed a novel peptide that carries radiation and, when injected into a living subject, will seek out this protein and bind strongly to it. In this proposal, the researchers will investigate the radioactive peptide for its binding properties to cervical cancer cells in a dish, followed by its evaluation in mice that have cervical cancer tumors. At the conclusion of these studies, the researchers anticipate they will have a well-characterized radioactive peptide that is ready to be moved into the clinic for the treatment of cervical cancer. In addition, this peptide can also be used for the treatment of other cancers, such as pancreatic, lung or breast cancer, since the protein it binds to is also highly expressed in these cancers.
Project Title: Imaging, Phenotyping, and Molecular Targeting of CD38-Resistant Multiple Myeloma Cells
Goal: Multiple myeloma (MM) is an incurable blood cancer that nearly always relapses, and patients who fail CD38-targeted immunotherapies have poor survival measured in months. This project directly addresses an urgent clinical need by investigating mechanisms of resistance to CD38-targeted therapies. The findings of this proposal will ultimately guide development of new interventions to improve survival in patients with relapsed and refractory MM. The overall goal is to address the unmet clinical need to identify these aggressive and highly metastatic MM cells early on and identify effective treatments that will improve patient outcomes.
Project Summary: MM is the second most common blood cancer. It arises from abnormal plasma cells in the bone marrow that multiply uncontrollably and produce harmful levels of antibodies. This disease damages the bones, weakens the immune system, and can lead to kidney failure. Current treatments often work well at first, but nearly all patients eventually relapse with more aggressive disease, and no existing therapy can cure MM. A new class of drugs has targeted a protein called CD38, which is found in high amounts on most myeloma cells. Drugs such as daratumumab and isatuximab initially work well, but many patients either do not respond or become resistant, with survival dropping to less than six months once these treatments fail. Because CD38-targeted therapies are now being used earlier in treatment, resistance is expected to become even more common. Our research focuses on understanding why MM becomes more aggressive when CD38 is lost. In laboratory mouse models, myeloma cells without CD38 caused more bone damage, spread to the kidneys, and grew faster than normal myeloma cells. We also observed changes in several cellular pathways and abnormal blood chemistry, indicating that “CD38-low” myeloma is particularly dangerous. This project will study both the tumor cells themselves and the surrounding microenvironment to uncover how CD38 loss drives aggressiveness and resistance. By identifying the key pathways and markers of these high-risk cells, the researchers aim to develop strategies to detect them earlier and create more effective treatments. The long-term goal is to improve survival and quality of life for patients facing this incurable and often devastating disease.
Project Title: Identifying a DHX9 Pathway Vulnerability in Triple Negative Breast Cancer
Goal: To define why triple negative breast cancer (TNBC) depends on the RNA helicase DHX9. The work will determine how TNBC cells use DHX9 to grow and survive. The project will test the DHX9 inhibitor ATX968 in TNBC models and measure its ability to slow growth and kill tumor cells. The results aim to provide the evidence needed to support a clinical trial in TNBC patients.
Project Summary: TNBC refers to breast cancers that lack estrogen, progesterone and HER2 receptors. This aggressive subtype of breast cancer is often metastatic and is associated with lower overall survival across all stages compared to other breast cancer subtypes. TNBC poses significant challenges to patients, clinicians and researchers due to a lack of effective therapies, its high mortality rate and the absence of a well-defined molecular target. Recent work points to a new opportunity. DHX9 is a protein that plays a crucial role in several important functions within cells, including how genes are turned on and off, and how genetic material is kept stable. Many cancers produce high levels of DHX9, and this pattern is linked to poorer outcomes. TNBC cells appear to rely on DHX9 to manage complex RNA structures that would otherwise trigger stress or cell death. This makes DHX9 a promising target for therapy. The researchers’ work shows that DHX9 is highly active in breast tumors with worse prognosis. The scientists reduced DHX9 in aggressive breast cancer cells and found that this led to slower growth and more cell death. These results suggest that TNBC cells depend on DHX9 to survive. Blocking the activity of the DHX9 protein may also help the immune system recognize and attack these tumors. This project will build on these findings using tissue samples from patients and mouse models of TNBC. The researchers will use a new drug called ATX968 that inhibits DHX9 activity and has already been cleared for initial testing in humans.
COE Supplement
Project Title: Leveraging Community Input for AI-based Applications for Cancer Patients and Caregivers
Goal: To define the unique factors that impact molecular cancer testing and outcomes in patients throughout the Siteman catchment area. The researchers aim to deliver community-informed, AI based methods to compare access to precision medicine and its impact on cancer outcomes. They further expect to develop a prototype AI-based application for interactive use by community patients and caregivers to address barriers to molecular testing and precision medicine therapies.
Gomez
Project Summary: In this pilot study, the researchers will leverage large language models (LLM), a type of artificial intelligence designed to standardize and accelerate the review of bulk data, to collate cancer molecular testing data, social determinants of health (SDOH) and cancer-specific outcomes from notes extracted from medical records. LLM-extracted data will be compared to manually abstracted data performed by an oncology clinical coordinator from the same patient charts.
In parallel, the researchers will seek input and feedback from their community partner, the Cancer Support Community of Greater St. Louis. Through that network, the researchers will engage participants to understand their knowledge of precision medicine, the barriers they have faced and their thoughts on artificial intelligence.
Linnenbringer
The researchers will incorporate their input into a prototype AI-based application and elicit additional feedback via testing of the improved prototype.
Access to the novel immunotherapy — which “has the potential for cure” — is the latest offering by the national leader in pediatric hematology, oncology, and cell therapies.
Siteman Kids at St. Louis Children’s Hospital, along with WashU Medicine, is the first site to test a novel immunotherapy drug for the treatment of autoimmune diseases such as systemic lupus erythematosus, juvenile myasthenia gravis, juvenile dermatomyositis, and ANCA-associated vasculitis in children and young adults.
The clinical trial, called HELIOS Descartes-08 (202510129), evaluates dosing levels of an autologous chimeric antigen receptor T-cell (CAR-T) therapy that targets the B-cell maturation antigen (BCMA). Significantly, no neoadjuvant chemotherapy is needed prior to the start of Descartes-08 therapy.
“We are the first site in a multicenter clinical trial that will offer qualified patients an infusion of mRNA-modified CAR-T cells that target plasma cells,” said WashU Medicine hematologist/oncologist Melissa Mavers, MD, PhD, site principal investigator and a leader on the childhood cancers specialist team at Siteman Cancer Center. “This trial is exciting because this is the first available therapy targeting plasma cells in autoimmune disease and has the potential for cure for these patients.”
Earlier results of the Descartes-08 therapy, developed by Cartesian Therapeutics, have been promising. The FDA has granted it Orphan Drug Designation and Regenerative Medicine Advanced Therapy Designation.
To be eligible for the clinical trial, a patient must:
Be age 12 or older
Diagnosed with systemic lupus erythematous, juvenile myasthenia gravis, juvenile dermatomyositis, or ANCA-associated vasculitis
Have undergone systemic treatment
The trial will be administered through the Pediatric Hematopoietic Cell Transplant and Cellular Therapy Program at Siteman Kids at St. Louis Children’s Hospital.
Other Major Advancements
The clinical trial launch follows these recent advances at Siteman Kids:
The FDA granted Breakthrough Therapy Designation for WU-CART-007, an allogeneic CAR-T therapy for T-cell malignancies developed by WashU Medicine researchers at Siteman Cancer Center. WU-CART-007 is an off-the-shelf, anti-CD7 CAR T-cell therapy for children and adults diagnosed with relapsed or refractory T-cell lymphoblastic leukemia or T-cell lymphoblastic lymphoma (R/R T-ALL/LBL). The FDA designation, announced in January, came after global clinical trials found that WU-CART-007 (also known as soficabtagene geleucel, or sofi-cel), resulted in 73% of adults and adolescents in the trials achieving full remission after receiving a full dose of the cellular therapy. WashU Medicine pediatric hematologist/oncologist Thomas Pfeiffer, MD, co-led the pediatric component of the clinical trial at Siteman Kids.
Global consultations and referrals continue to expand the reach and impact of an International Neuro-Oncology Tumor Board that originated at Siteman Kids. WashU Medicine neuro-oncologist Mohamed Shebl Abdelbaki, MD, director of the Pediatric Neuro-Oncology Program at Siteman Kids, began the monthly tumor board meetings in early 2021. To date, more than 2,500 health care specialists from 71 institutions in 41 countries have participated in the meetings, which have resulted in several advancements in best care practices for the management of complex brain tumors in infants, children and young adults.
Also available at Siteman Kids is participation in the CONNECT1906: PEP-CMV vaccine phase II clinical trial that is testing a CMV-directed peptide vaccine in children with recurrent medulloblastoma or newly diagnosed high-grade gliomas or diffuse intrinsic pontine gliomas. The multicenter study advances investigations into whether CMV-targeted vaccines can serve as a novel immunotherapeutic approach and boost responses against these cancers in pediatric patients. Eligible patients will receive a single, 5-day course of oral chemotherapy followed by PEP-CMV injections. Siteman Kids’ site principal investigator is Eric Thompson, MD.
For patients with malignant brain tumors, Siteman Kids also is involved in a multicenter phase I trial investigating the safety and dosing of intra-tumoral injections of NK cells in patients with recurrent or progressive tumors located in the upper part of the brain. Siteman Kids is one of only seven institutions offering this trial through the Pediatric Neuro-Oncology Consortium. WashU researchers are known internationally for their breakthroughs in the development and use of NK cell immunotherapies. Site principal investigators are WashU Medicine neuro-oncologists Mohamed Shebl Abdelbaki, MD, and Michael Angelo Huang, MD, MS.
“These are just a few of the leading-edge research efforts and collaborations we have underway at Siteman Kids,” said Jorge Di Paola, MD, chief of the Division of Pediatric Hematology and Oncology and the Elizabeth Finney McDonnell Endowed Chair in Pediatric Hematology Oncology at Siteman Kids at St. Louis Children’s Hospital and WashU Medicine. “We have 12 basic and translational research labs solely dedicated to advancing understanding and treatments for a wide range of pediatric cancers and more than 280 clinical trials available.”
For more information regarding clinical trials at Siteman Kids, email [email protected] or call 800-600-3606 to make a referral.
