Article Topic: Blood Cancers

Vij Named Inaugural Jeffrey S. and Prue H. Gershman Distinguished Professor

Renowned physician-scientist is national leader in research and treatment of blood cancers

Ravi Vij, MD, MBA, who has dedicated his career to advancing treatments for blood cancers, has been installed as the inaugural Jeffrey S. and Prue H. Gershman Distinguished Professor in the John T. Milliken Department of Medicine at WashU Medicine.

Vij, a professor of medicine in the department’s Division of Oncology, treats patients at Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine. He was installed by Chancellor Andrew D. Martin and David H. Perlmutter, MD, executive vice chancellor for medical affairs, the Spencer T. and Ann W. Olin Distinguished Professor and the George and Carol Bauer Dean of WashU Medicine. The professorship was funded by St. Louisans Jeffrey and Prue Gershman, who are dedicated philanthropists and volunteers supporting local education, health and arts organizations.

“Jeffrey and Prue are deeply committed to improving the lives of the people of St. Louis, and it is a true honor that they have chosen WashU to be partners in that goal,” Martin said. “Through this professorship, their generosity will accelerate progress against blood cancers by supporting Dr. Vij’s work to bring new, more effective treatments to patients. His leadership has helped grow WashU Medicine’s reputation as a national force in stem cell transplantation and immunotherapy, and with the Gershmans’ support, that momentum will continue.”

As the principal investigator of the Multiple Myeloma Tissue Banking initiative at Siteman, Vij leads a collaborative research team studying the genetic underpinnings and cellular microenvironment of multiple myeloma, a cancer of the plasma cells in bone marrow. He has led several clinical trials of investigative therapies for blood cancers, including immunotherapy agents and novel stem cell transplant strategies, that went on to become standard treatments. He has authored over 300 scientific publications in the arena of blood cancers.

“Dr. Vij has consistently pushed the field forward, pursuing multiple promising avenues to improve outcomes for patients with blood cancers, particularly multiple myeloma,” Perlmutter said. “His work spans discovery science, clinical trials and national collaboration — advancing new therapies while building the partnerships that move the field as a whole. His ability to translate scientific insight into real-world advances continues to shape the future of care in this field.”

Vij has served on the American Society of Clinical Oncology education and scientific committees and on the myeloma committees of the Clinical Trials Network and Alliance for Clinical Trials in Oncology. He currently serves as senior editor of the journal Clinical Lymphoma, Myeloma and Leukemia and is a past chair of the American Society of Hematology scientific committee on plasma cell dyscrasias, a type of cell disorder linked to blood cancers. Vij has received the Multiple Myeloma Research Foundation Innovator Award, the Center of Excellence Award and the Leukemia & Lymphoma Society Visionary of the Year Award.

A respected and effective educator, Vij has mentored 25 early-career researchers over his career and in 2007 received the Teacher of the Year Award from the Hematology and Oncology Fellowship Program at WashU Medicine.

“Dr. Vij is an expert in myeloma whose warmth and support give his patients confidence that they are in the best possible hands and getting the best treatment,” said Victoria J. Fraser, MD, the Adolphus Busch Professor of Medicine and head of the Department of Medicine. “He is widely recognized as a leader in the field for his research, his thoughtfulness as a physician and his creativity as a clinical investigator and mentor. The tremendous energy he brings to resources such as the Multiple Myeloma Tissue Banking initiative will benefit the field for years and decades to come.”

Vij completed his medical education at Maulana Azad Medical College in New Delhi, India, followed by postgraduate training at Halifax General Hospital and Royal Infirmary in the U.K. He completed an internal medicine residency at Rush University in Chicago and fellowships in medical oncology and hematology and in bone marrow transplantation at WashU Medicine. He joined the WashU Medicine faculty in 2000.

Jeffrey S. and Prue H. Gershman

Jeffrey S. and Prue H. Gershman, of Clayton, Mo., have supported numerous programs and organizations in and beyond St. Louis through philanthropy and through volunteer service for the arts, education, healthcare and community organizations.

Jeffrey is an attorney who has practiced business, real estate and tax law in the St. Louis area since 1981. He is active in the St. Louis business community as a director on the boards of Central Bank of St. Louis and Gershman Investment Corp. Prue has worked for 40 years as an educator and social worker at several institutions, most recently as the director of counseling and wellness at John Burroughs School in Ladue.

Gene-Edited Stem Cell Transplant Shows Promise for Aggressive Blood Cancers

Strategy seeks to expand immunotherapies for leukemia, other malignancies

For highly aggressive types of blood cancer, stem cell transplantation is often the only potentially curative therapy, yet even after a transplant, these cancers often return.

Now, a clinical trial, led by researchers at Washington University School of Medicine in St. Louis, shows that a stem cell transplant in which the donor cells have been genetically engineered to remove a particular protein helps prevent toxic side effects and potentially improves the effectiveness of therapies given after a transplant to help prevent cancer recurrence.

The study was conducted at Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine, and 14 other sites in the U.S. and Canada. The findings are published May 12 in the journal Nature Medicine.

According to the study’s corresponding author, John F. DiPersio, MD, PhD, the Virginia E. & Sam J. Golman Professor of Medicine at WashU Medicine, this gene-editing technology could help address a longstanding frustration in the field: CAR-T cell therapy — an immunotherapy that effectively treats some aggressive blood cancers — has not worked against all blood cancers, including acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

According to DiPersio, who treats patients at Siteman and is a research member there, myeloid cancers like AML and MDS are tricky to treat with CAR-T cells because the same proteins on cancer cells that the immunotherapy homes in on for destruction are also present on healthy myeloid cells, including therapeutic donor stem cells. As such, the anti-cancer therapy carries a high risk of toxicity because it also destroys healthy blood stem cells, which can trigger a dangerous inflammatory cascade. This effect also could dilute the effect of the anti-cancer therapy because so many of the CAR-T cells are attacking the wrong targets, leaving many cancer cells untouched.

This basic concept was first described by Miriam Y. Kim, MD, now an assistant professor of medicine at WashU Medicine. She began this research as a postdoctoral researcher at the University of Pennsylvania and continued the work in the DiPersio lab before becoming an independent investigator in the WashU Medicine Division of Oncology. She treats patients at Siteman and is also a research member there.

For this clinical trial, patients with AML and MDS received donor stem cells that had a target protein, called CD33, removed, in hopes that immunotherapy targeted against CD33 would kill the cancer and ignore the healthy cells.

“We are encouraged by the results of this study showing that a CD33-deleted stem cell transplant looks very similar to the outcomes of standard stem cell transplantation,” said DiPersio, who also directs WashU Medicine’s Center for Gene and Cellular Immunotherapy. “In the future, we are hopeful we will be able to combine this with CD33-targeted immunotherapies, such as CAR-T cells, and improve treatment options for patients with these very aggressive blood cancers.”

To that end, DiPersio and his collaborators have also published a single case study of a patient with high-risk AML who received a CD33-deleted stem cell transplant and later, upon relapse after the transplant, received a CD33-targeted CAR-T cell therapy, which used T cells from the same donor who provided the stem cell transplant. The patient — who had one of the most aggressive types of AML — achieved complete remission and remains cancer free over one year after receiving the CAR-T cell therapy. The patient also had normal blood cell production return with all blood cells lacking CD33, providing evidence that the genetically engineered donor cells had established themselves in the bone marrow. DiPersio is the senior author of this study, published in October 2025 in JCO Precision Oncology.

Shielding Healthy Cells

CD33 is an appealing protein to delete from donor stem cells because it is only present on blood-forming cells and not in other tissues, and because there is evidence it is not required for the proper function of blood stem cells, given that individuals born without CD33 have no apparent health problems. After a patient has successfully received this type of stem cell transplant, any remaining cells in the body with CD33 on the surface should, in theory, only be the cancer. Then, CAR-T cells or another immunotherapy designed to target CD33 would kill only the cancer cells and leave healthy donor stem cells untouched.

In this phase 1/2 multicenter clinical trial, 30 adult patients with AML or MDS at high risk of relapse received a stem cell transplant in which CD33 had been removed from the donor cells using CRISPR gene editing technology before the transplant procedure. The CD33-deleted stem cell product is called tremtelectogene empogeditemcel (trem-cel) and was made by Vor Biopharma, which funded the study.

As proof of concept, the patients also received a maintenance therapy that targets CD33, after they underwent the stem cell transplant. While not a CD33-targeted CAR-T cell, the maintenance therapy, called gemtuzumab ozogamicin, is a type of engineered antibody that targets CD33 and carries an anti-cancer drug. Gemtuzumab ozogamicin is approved by the Food and Drug Administration to treat CD33-positive AML and is in clinical trials for CD33-positive MDS. While it helps prevent relapse, the drug’s use is limited because it can cause liver toxicity and damage to blood cells, including dangerously low counts of white blood cells, red blood cells and platelets.

All patients achieved engraftment of their transplanted stem cells by day 28, meaning the cells had gathered in the bone marrow and started working. Some patients met this goal sooner, and platelet production returned by day 16, on average. These timeframes are comparable to those of standard transplanted stem cells.

Average survival was just over 14 months. Nineteen patients received at least one cycle of the antibody maintenance therapy as part of a dose-escalation protocol, and the researchers were able to establish the recommended dose. The researchers found that patients maintained blood cell counts across all doses, suggesting that the gene-edited stem cell transplant protected patients from the dangerously low blood cell counts typically seen during this maintenance therapy following a standard stem cell transplant.

Side effects during the treatment were similar to those of standard transplants, including anemia, low platelets, fever, infections and graft-versus-host disease, in which the donor cells attack the patient’s healthy tissues. Seven patients died during the study, with four due to the cancer progressing and three due to transplant-related causes, including kidney failure, liver toxicity and sepsis.

DiPersio said the results of the study lay the groundwork for developing paired CD33-deleted stem cell transplant and CD33-targeted immunotherapy interventions that avoid destruction of healthy donor cells in the course of cancer treatment.

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DiPersio JF, Koehne G, Shah NN, Bernard L, Suh HC, Koura D, Tamari R, Mushtaq MU, Maakaron J, Rimando J, Kennedy VE, Patel SS, Hudson C, Loken M, Stanizzi DA, Lee-Sundlov MM, Thosar S, Mundelboim G, Guo G, Ge HG, Li BE, Xavier-Ferrucio J, Hyzy SL, Lin MI, Raffel GD, Cooper BW. Phase 1/2 trial of trem-cel CD33-deleted allogeneic hematopoietic cell transplantation with gemtuzumab ozogamicin maintenance in adult high-risk AML. Nature Medicine. May 12, 2026. DOI: 10.1038/s41591-026-04362-1.

This work was supported by Vor Biopharma. Several co-authors were employees of the company when the work was conducted.

Donor provides gift for blood cancer research

Anita Palmer Corbin learned to meet challenges head-on when she was diagnosed with Type 1 diabetes at age 10 in 1964. Her mother first noticed the symptoms, and after blood tests confirmed a problem, she was admitted to Cardinal Glennon Children’s Hospital in St. Louis, located one hour north of her home in Ste. Genevieve, Missouri. No visitors were allowed except immediate family members, and since both of her parents worked, she was left with few visitors for nearly two weeks while doctors brought her diabetes under control.

From the time of her diagnosis, Anita, who died in 2023, was told she had a disability that would prevent her from living a full life. On the contrary, she never allowed the disease to stand in her way. She enjoyed a more than 20-year career at St. Louis-based Ralston Purina Co., which became Nestlé Purina PetCare after merging with Nestlé in 2011, and became the company’s youngest and first female officer. In her retirement, Anita traveled the world, riding elephants in Thailand, walking with lions in South Africa, and snorkeling the Great Barrier Reef in Australia.

Anita wanted to create a permanent legacy to honor her medical journey and the experiences of family members who faced serious illnesses. Now, an $11 million gift from her trust, which is expected to grow in the coming years, is helping advance critical work at Washington University School of Medicine. The gift established three endowed funds to drive research and develop new treatments for Type 1 diabetes, leukemia, and lymphoma. The funds augment three corresponding spendable funds created by Corbin in 2022.

Anita hoped to inspire all individuals battling a disease to live boldly, says her husband, Daniel Corbin. “She refused to let her diagnosis define her life and didn’t accept it as a limitation to what she could accomplish,” he says. “Every time she talked with other people facing health challenges, she would tell them, ‘You can do anything you want.’ She encouraged them to take ownership of their disease so it didn’t own them. She left a lasting impression on countless lives.”

Innovative treatments for blood cancers

Anita’s gift is also fueling research at the Alvin J. Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine. The gift established two endowed funds at the cancer center — $7 million for leukemia research and $1.25 million for lymphoma research.

The Trevor Stuart Palmer Memorial Leukemia Endowed Fund pays tribute to Anita’s nephew, who died from the disease shortly after his diagnosis at age 26. She believed if Trevor had received care at a world-class cancer center like Siteman, he would have been accurately diagnosed earlier and obtained lifesaving treatment.

Payout from the fund will further efforts that are part of the School of Medicine’s Specialized Program of Research Excellence (SPORE) in leukemia, a prestigious grant from the National Cancer Institute. The medical school’s SPORE in leukemia is one of only two such programs in the nation.

The leukemia fund currently supports work with patients who have acute myeloid leukemia and myelodysplastic syndrome with mutations in an important gene called TP53. Outcomes for patients with these types of blood cancers are typically poor, with survival rates of less than a year. Researchers in the lab of Dan Link, MD, professor of medicine and principal investigator for WashU Medicine’s leukemia SPORE, have identified a promising new drug combination that selectively kills leukemia cells with TP53 mutations. A clinical trial based on their observations is in development.

The Daniel E. Corbin Lymphoma Research Endowed Fund created by Anita’s gift recognizes the many years of outstanding care Daniel received at Siteman. “When I initially visited another facility, the providers treated many different types of cancer in one room,” he says. “Siteman was a godsend because my entire care team specialized in lymphoma, and I felt reassured that each person was an expert who could solve any problem that might come up.”

This year, the fund’s resources are bolstering efforts led by John DiPersio, MD, PhD, professor of medicine, to improve an immunotherapy technique called CAR-T cell therapy for individuals with a fast-growing non-Hodgkin lymphoma. Investigators are testing different combinations of cytokines, proteins that help activate the immune system, to enhance the ability of CAR-T cells to kill lymphoma cells.



“Siteman is at the forefront of advancing groundbreaking discoveries that revolutionize the way we understand and treat blood cancers,” says cancer center director Timothy Eberlein, MD, who also serves as the Spencer T. and Ann W. Olin Distinguished Professor and senior associate dean for cancer programs at WashU Medicine and BJC HealthCare. “It is through partnerships with forward-thinking philanthropists like Anita that visionary ideas come to life. Investments in our research have the power to save lives and set new standards of care worldwide.”

A remarkable life

a portrait of a wife and husband over a bridge with a river behind them
Anita Palmer Corbin shared an adventurous spirit with her husband, Dan Corbin. The couple’s many travels included a visit to southern England. (Courtesy photo)

Anita grew up the youngest of four children and the only daughter. As a child, she ran home from school to finish her homework and chores so she had time to play outside. Daniel says her motto was always work first, play second.

Though her family thought it would be difficult for her to go to college, Anita earned her undergraduate degree in accounting from the University of Missouri-St. Louis and received a master’s degree in business administration with a focus on finance from Saint Louis University.

Anita began her career with Ernst and Young LLP and then joined Ralston Purina in 1979 as a senior accountant. She held several positions of increasing responsibility and was named corporate vice president and controller of the company in 1994.

She met Daniel at Ralston Purina, though they did not begin dating until years later after their first marriages had ended. Together, the couple enjoyed logging miles on their motorcycles. Anita was also an avid bicyclist, covering ground from the Blue Ridge Mountains to Florida beaches. Still, she experienced lifelong complications from diabetes, which contributed to her death.

Today, Daniel wears a silver necklace engraved with Anita’s index fingerprint and the inscription “The love of my life, I am a better man and person because of you.” The engraving shows dozens of scars from where she pricked her finger to test her blood.  “Diabetes is a terrible disease,” he says. “If Anita’s gift to WashU Medicine spares even one person from experiencing what she went through, her struggles will have been worthwhile.”

Grants Bolster Research on Myelodysplastic Syndromes

Foundation awards $1.5 million total in grants to WashU Medicine researchers at Siteman Cancer Center to advance precision diagnosis and treatment for patients with myelodysplastic syndromes

The Edward P. Evans Foundation has awarded separate three-year Discovery Research Grants to Jeremy Baeten, PhD, an assistant professor of medicine, and Matthew Walter, MD, the Edward P. Evans Endowed Professor of Myelodysplastic Syndromes, both in the Division of Oncology at WashU Medicine. Walter treats patients at Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine. He and Baeten are research members at Siteman.

The awards, which total $1.5 million, represent an investment in research that is advancing the field of myelodysplastic syndromes (MDS), a group of blood cancers characterized by low blood cell counts.



Baeten aims to improve treatments for patients who have MDS with mutations in an important gene called TP53, which causes resistance to current therapies. Baeten will study how a new drug combination kills these cancer cells better than available drugs and how blocking another gene might make these treatments even more effective.



Walter will explore using whole-genome sequencing to monitor mutated blood cells in patients with MDS or clonal cytopenias of undetermined significance, a disease that predisposes patients to developing MDS, including tracking whether changes in mutations are linked to disease progression. The funding also will help advance his work in collaboration with Kelly Bolton, MD, PhD, an assistant professor of medicine in oncology and a Siteman research member, to examine how mutated cells respond to targeted treatments, which could help guide more personalized treatment choices.

The Edward P. Evans Foundation previously provided grant funding to WashU Medicine, including support for Baeten and Walter. In 2019, the foundation established the Edward P. Evans Center for Myelodysplastic Syndromes at WashU Medicine, one of four MDS-supported centers nationally. The foundation also funded the endowed professorship that Walter holds.

Innovative CAR-T cell therapy receives FDA breakthrough therapy designation

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.

FDA Breakthrough Therapy Designation Reflects Practice-Changing Trends Only 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.

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‘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.”

Other examples of comprehensive, specialized programs at Siteman include the:

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.”

FDA grants breakthrough therapy designation to treatment for rare blood 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.”

Learn more: U.S. FDA Grants to Wugen’s WU-CART-007 Breakthrough Therapy Designation

Siteman Cancer Center announces 2026 American Cancer Society-funded pilot projects

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
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
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

Principal Investigator: José Marcio Luna, MS, PhD

José Marcio Luna, MS, PhD
José Marcio Luna, MS, PhD

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
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.

Inflammatory immune cells predict survival, relapse in multiple myeloma

New ‘atlas’ of immune cells in bone marrow cancer lays foundation for more reliable prognosis

A new study maps the immune cell landscape of bone marrow in patients with multiple myeloma, a rare cancer that develops in the plasma cells of the bone marrow and has no cure. This large immune cell atlas, which includes robust patient outcome data, provides unparalleled new insights into how the immune system interacts with cancerous plasma cells and can be used to determine how aggressive a patient’s multiple myeloma is likely to be. The knowledge may improve survival predictions, guide treatment decisions and help in the development of new immune-based therapies for patients with multiple myeloma.

The study — co-led by WashU Medicine researchers at Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine, in collaboration with the Multiple Myeloma Research Foundation (MMRF) and other leading institutions across the country — appears Jan. 9 in the journal Nature Cancer.

“It is time for a better understanding of the immune system in multiple myeloma,” said WashU Medicine co-senior author Li Ding, PhD, the David English Smith Professor of Medicine and a research member of Siteman. “In addition to targeting the cancerous plasma cells directly, we also want new and better ways to activate the immune system to attack the malignant cells. This large-scale immune cell atlas will serve as a critical resource to investigators studying multiple myeloma and working to develop better therapies.”

While considered a rare cancer, multiple myeloma is the second most common blood cancer after leukemia, accounting for about 15%-20% of new blood cancer diagnoses in the U.S. annually. Plasma cells are white blood cells in the bone marrow. When they grow out of control, they crowd out healthy blood cells. About 60% of patients are still living five years after diagnosis.

Many new treatment options have emerged for multiple myeloma in recent years that can extend survival for many patients, sometimes for more than a decade. Even so, the disease almost always returns after periods of remission, emphasizing the need for new and better options.

Several of the newest therapies for multiple myeloma are immune system-based, including CAR-T cells and what are known as bispecific antibodies. But researchers suspect there may yet be untapped opportunities for immune-based treatments for multiple myeloma, and the immune cell atlas is a new tool to harness in pursuit of such therapies.

“This immune atlas provides a roadmap for the next generation of myeloma care,” said coauthor and WashU Medicine oncologist Ravi Vij, MD, the Jeffrey S. and Prue H. Gershman Distinguished Professor of Medicine and a research member of Siteman. “As immunotherapies like CAR-T cells and bispecific antibodies become central to treatment, understanding the immune context in which they operate is essential. Clinically, this work lays the foundation for immune-informed risk stratification and rational development of new therapies that not only target the tumor but also restore effective anti-myeloma immunity.”

Vij treats patients with multiple myeloma and other blood cancers at The Blood Cancer Center at Siteman.

A cell-by-cell catalog of multiple myeloma

The research team performed a rigorous and cutting-edge genetic analysis called single-cell RNA sequencing of almost 1.4 million individual plasma and immune cells in bone marrow sampled from 337 newly diagnosed multiple myeloma patients. This type of analysis can reveal how individual immune cells may function — or become dysfunctional — in the context of multiple myeloma.

The data describe patients enrolled in MMRF’s CoMMpass Study, which is the first large-scale, long-running study of patients with multiple myeloma focused on analyzing disease progression and treatment response based on the genomic and molecular profiles of the patients. WashU Medicine is one of multiple sites participating in the CoMMpass Study.

The investigators found that patients with certain types of immune cells in their bone marrow at diagnosis were more likely than others to relapse quickly, meaning their cancer returned soon after a first round of treatment. The researchers identified signaling patterns between the cancer cells and immune cells that drive inflammation, which might be boosting the cancer’s growth in patients with aggressive disease.

The team also identified a type of T cell that had stopped working as expected and, rather than attacking the tumor as it should, acted to suppress immune activity against the cancer. Together, these findings could help make prognosis more accurate and aid in selecting the best therapies.

“This immune atlas represents the power of collaborative science,” said co-senior author and MMRF Chief Scientific Officer George Mulligan, PhD. “The MMRF’s CoMMpass Study, combined with the expertise from investigators at WashU Medicine and leading institutions across the country, has created an unprecedented resource that will accelerate discovery and improve outcomes for myeloma patients.”

Importantly, the researchers showed that knowledge of the immune environment in a patient’s bone marrow could improve upon current methods for predicting which patients are most likely to experience an aggressive course of the disease and have shortened survival. Such predictions can help guide treatment decisions in terms of matching the intensity of the treatment with the aggressiveness of the cancer.

Current methods for determining whether a patient has high-risk multiple myeloma versus standard risk rely on knowing the genetic features of the cancer cells combined with clinical aspects of the patient’s health. The new study found that adding an immune component to this analysis would likely improve the accuracy of the categorization.

“More work is needed to develop specific immune-based blood tests, for example, that clinicians could order to better identify the aggressiveness of a particular case of multiple myeloma and help them select the best treatments for that patient,” Ding said. “This immune cell atlas fills a gap in knowledge that is needed to develop these types of new clinical tools.”

The MMRF designed and funded this research, which involved collaboration among the Georgia Institute of Technology, Emory University, Harvard Medical School and the Icahn School of Medicine at Mount Sinai.

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Pilcher WC, Yao L, Gonzalez-Kozlova E, Pita-Juarez Y, Karagkouni D, Acharya CR, Michaud ME, Hamilton M, Nanda S, Song Y, Sato K, Wang JT, Satpathy S, Ma Y, Schulman J, D’Souza D, Jayasinghe RG, Ohlstrom D, Ferguson KE, Cheloni G, Bakhtiari M, Pabustan N, Nie K, Foltz JA, Saldarriaga I, Alaaeldin R, Lepisto E, Chen R, Fiala MA, Thomas BE, Cook A, Vieira Dos Santos J, Chiang I, Figueiredo I, Fortier J, Slade M, Oh ST, Rettig MP, Anderson E, Li Y, Dasari S, Strausbauch MA, Simon VA, Immune Atlas Consortium, Radkevich E, Rahman AH, Chen Z, Lagana A, DiPersio JF, Rosenblatt J, Kim-Schulze S, Lonial S, Kumar S, Bhasin SS, Kourelis T, Dhodapkar MV, Vij R, Avigan D, Cho HJ, Mulligan G, Ding L, Gnjatic S, Vlachos IS, Bhasin M. A single-cell atlas characterizes dysregulation of the bone marrow immune microenvironment associated with outcomes in multiple myeloma. Nature Cancer. Jan. 9, 2026. DOI: 10.1038/s43018-025-01072-4.

This work was designed and supported by the Multiple Myeloma Research Foundation; the Myeloma Solutions Fund; the Paula C. and Rodger O. Riney Blood Cancer Research Fund; and the National Institutes of Health (NIH), grant numbers U24CA224319, U01DK124165, P30CA196521, R50CA211466, R35CA210084, 5K12CA090628, U24CA211006, U2CCA233303, PJ000021702, R01CA258776 and UL1TR004419. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.

Genetic study suggests ways to catch blood cancer earlier

New understanding of how mutations interact could pave way for early detection, prevention strategies

As we age, our cells replicate, and the DNA in these cells can acquire mistakes — or mutations — every time the sequence is copied. Most newly acquired mutations are harmless, but some can tip the balance toward cancer development later in life.

Now, a new study led by researchers at Washington University School of Medicine in St. Louis shows that such newly acquired mutations interact with our inherited mutations — those passed down by our parents — in important ways that influence a person’s lifetime cancer risk. Understanding such interactions could guide development of new methods for early detection and prevention of cancer.

The research, published in Nature Genetics, focused specifically on the risk of blood cancers such as acute myeloid leukemia (AML), although interactions between inherited and acquired mutations likely have roles in other types of cancer.

Inherited mutations are carried in the egg and sperm and are therefore present in every cell starting at birth, whereas acquired mutations accumulate gradually with age in different cells. Led by Kelly Bolton, MD, PhD, an assistant professor of medicine in the Division of Oncology at WashU Medicine and the study’s senior author, the research team set out to understand how interactions between these two types of mutations influence a person’s risk of developing blood cancer.

In particular, they focused on a blood condition called clonal hematopoiesis that is known to increase a person’s risk of developing blood cancer. Clonal hematopoiesis is caused by a mutation in blood stem cells — cells that give rise to all the different cell types in the blood — that gives those cells a slight survival advantage over the normal stem cells. Such stem cell clones multiply more and are at risk of transforming to blood cancer.

“Most people with clonal hematopoiesis never develop blood cancer,” said Bolton, who treats patients at Siteman Cancer Center, based at Barnes-Jewish Hospital and WashU Medicine. “To a certain extent, it’s a normal aging process. However, we think that many if not all individuals who develop blood cancer pass through a phase of clonal hematopoiesis at some point. We are still in the early stages of trying to figure out which individuals with clonal hematopoiesis will go on to develop blood cancer and which will not.”

Studying genomic data of more than 730,000 people, including from blood samples, the researchers found that clonal hematopoiesis was more common among those with inherited mutations in certain genes already known to increase the risk of cancer. They also found that such inherited mutations had an impact on patterns of newly acquired mutations that cause clonal hematopoiesis. If stem cell clones go on to acquire just a handful more harmful mutations, the clonal hematopoiesis can transform into a blood cancer, such as AML, in which the cells stop doing their jobs and multiply until they crowd out healthy cells.

With the goal of finding ways to detect and eliminate pre-cancerous cells in people at high risk of blood cancer, Bolton and her colleagues found that among individuals with clonal hematopoiesis, those who had inherited mutations that predispose to clonal hematopoiesis had a higher risk of developing blood cancer than those without inherited mutations.

“Our study is a first look at the inherited genetic background that is providing the soil, so to speak, and we’re seeing what undesirable seeds that are acquired later in life are more or less likely to grow from that soil,” Bolton said. “The goal is to stamp out the weeds early, before they can take root and become full-blown cancer.”

Though clonal hematopoiesis is part of normal aging, certain factors such as smoking or prior exposure to radiation or chemotherapy can speed up the process and increase the risk of it transforming into cancer. Still, some people progress to cancer without major environmental risk factors, and the new study suggests that the interaction of their inherited genome with newly acquired mutations plays an important role in this cancer progression.

The study’s first author Jie Liu, a graduate student in Bolton’s lab, noted: “It’s exciting to see how combining large-scale genomic data can reveal how inherited and acquired mutations work together to influence cancer risk. These insights move us closer to identifying high-risk individuals before cancer develops. Our work shows that it’s not just the mutations you’re born with or those you acquire later in life, it’s the interaction between them, and we can now measure that.”

Earlier intervention

Bolton said being able to detect and measure both inherited cancer risk and clonal hematopoiesis would likely be a powerful way to identify individuals who would benefit most from early prevention strategies, such as targeted therapies for the most damaging mutations. At present, clonal hematopoiesis is difficult to identify without specialized blood tests that are not given as part of routine care. Even though such individuals already have clones taking up a greater proportion of their blood stem cells, they can still show normal blood cell counts as part of blood tests typically given at an annual well visit, for example.

In theory, if scientists know what gene mutations to look for, they could develop new blood tests to identify such individuals before any evidence of a problem could be detected with routine blood screening tests. The new study singles out many genes of interest that could be key in the future development of such a blood test.

“Because leukemia is so hard to treat, we hope to find ways to intervene early — when it’s still pre-cancerous — so we can stop clonal hematopoiesis from transforming into leukemia,” Bolton said. “We would want to start with preventive clinical trials for people who have certain inherited mutations and who already have evidence of clonal hematopoiesis, such as one or two clones expanding in their blood.”

Researchers at Siteman are now conducting clinical trials investigating whether specific drugs called IDH1 and IDH2 inhibitors can stop the expansion of certain types of blood stem cell clones before they become cancer. For now, such trials only include people who could be identified as having clonal hematopoiesis because they already had progressed to having abnormal blood cell counts, placing them on the cusp of full-blown leukemia.

“We are hopeful about the prospects of these preventive treatments, but we would like to have tools to identify these individuals even earlier, before their blood cell counts become abnormal,” Bolton said. “There are a lot of targeted therapies that are being developed right now and new approaches researchers are looking at for this purpose.”

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Liu J, Tran D, Xue L, Wiley BJ, Vlasschaert C, Watson CJ, MacGregor HAJ, Zong X, Chan ICC, Das I, Uddin MM, Niroula A, Griffin G, Ebert BL, Mack T, Pershad Y, Sharber B, Berger M, Sehir A, Ptashkin R, Levine RL, Papaemmanuil E, Joseph V, Gao T, Kemel Y, Mandelker D, Stopsack KH, Pharoah PDP, Mukherjee S, Ding L, Cao Y, Walter MJ, Blundell JR, Chatterjee N, Offit K, Godley LA, Link DC, Stadler ZK, Bick AG, Natarajan P, Bolton KL. Germline genetic variation impacts clonal hematopoiesis landscape and progression to malignancy. Nature Genetics. July 15, 2025. DOI: 10.1038/s41588-025-02250-x.

This work was supported by the National Institutes of Health (NIH), grant numbers R01HL148050, R01HL168894, DP5 OD029586, R01AG088657 and R01AG083736; the MDS Foundation; the Children’s Discovery Institute; a Prostate Cancer Foundation Challenge Award; the Edward P. Evans Foundation; the SciLifeLab & Wallenberg Data Driven Life Science Program, grant number KAW 2020.0239; the Swedish Cancer Foundation, grant numbers 22.0577JIA and 22.2362Pj; the Swedish Research Council, grant number 2023-03131; a Burroughs Wellcome Fund Career Award for Medical Scientists; a Pew Charitable Trusts and Alexander and Margaret Steward Trush Pew-Stewart Scholar for Cancer Research Award; and a Hevolution/AFAR New Investigator Award in Aging Biology and Geroscience Research. The study was conducted using the U.K. Biobank Resource and data provided by patients and collected by the National Health Service. It was also conducted using data from the All of Us Research Program of the National Institutes of Health. The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.