WVU Cancer Institute World's First to Treat Ultra-Rare Leukemia Using Novel Subcutaneous Immunotherapy
By Connect-Bridgeport Staff on February 18, 2026
A WVU Cancer Institute team has achieved a global medical first in a clinical trial examining the efficacy of subcutaneous blinatumomab in treating one of the rarest and most aggressive forms of leukemia.
The investigator-initiated Phase II clinical trial, led by Ashkan Emadi, M.D., Ph.D., principal investigator of the study and Alexander Bland Osborn Distinguished Professor of Medical Oncology in the WVU School of Medicine, marks the first-ever use of a subcutaneous formulation delivery for blinatumomab in CD19-positive mixed phenotype acute leukemia (MPAL). 
While MPAL only accounts for one-to-three percent of acute leukemias diagnosed, adults diagnosed with MPAL face a median survival rate often measured in months. There is no consensus among medical experts on treatment, and no FDA-approved, disease-specific therapies are currently available to address a critical, unmet need.
“Patients with MPAL have faced an extraordinary therapeutic void,” Dr. Emadi said. “This first-in-world treatment offers real hope that we can improve both outcomes and quality of life for a patient population that has been historically overlooked.”
The trial’s first patient received the milestone blinatumomab dose on Jan. 16.
“After only a single cycle of subcutaneous blinatumomab, the patient achieved complete remission with full hematologic recovery, including transfusion independence and the absence of the Philadelphia chromosome, an abnormal chromosome associated with certain types of leukemia, which is an outcome that is both unprecedented and highly encouraging,” Emadi said.
“The therapy was exceptionally well tolerated and transitioned entirely to the outpatient setting after the first week. The patient is currently at home with outstanding performance status and preserved organ function as we prepare to initiate cycle two.”
An innovation in immunotherapy
Blinatumomab (Blincyto®), a bispecific T-cell engager targeting CD19 has increased survival in patients in B-cell acute lymphoblastic leukemia. Treatment has traditionally required continuous intravenous (IV) infusion via pumps over a period of 28 days, prolonging hospitalization or home infusion.
The WVU Cancer Institute-led trial is evaluating a subcutaneous formulation designed to deliver comparable therapeutic exposure without the burden of continuous IV administration. Investigators believe this approach may significantly reduce treatment complexity, lower hospitalization requirements, and improve patient experience.
“Access to clinical trials is critical in treating patients with cancer, including those who have rare types of cancer like MPAL,” Hannah Hazard-Jenkins, M.D., executive chair and director of the WVU Cancer Institute, said. “Clinical trials offer the highest level of care for patients, even in our most rural communities. These trials represent our continued focus on doing what is best for our patients by providing innovative treatments and compassionate care to improve outcomes.”
This Phase II study will enroll up to 75 patients across three clinical cohorts, including:
- Newly diagnosed MPAL patients who are older than 75 years of age or unable to undergo intensive chemotherapy
- Patients in morphologic complete remission with measurable residual disease (MRD) positivity
- Patients with relapsed or refractory MPAL
As enrollment expands, this trial, if successful, could redefine the standard of care for CD19-positive MPAL and establish a new paradigm for delivering complex immunotherapies with a safer, more patient-centered approach to patients nationwide and internationally.
“This breakthrough reflects the core of our mission at West Virginia University – advancing research that improves health and changes lives,” Clay Marsh, M.D., chancellor and executive dean for WVU Health Sciences, said.
“By leading a first?in?world therapy for such a rare and aggressive leukemia, our team is showing how innovation and scientific rigor at the WVU Cancer Institute can create real impact for patients who have long lacked options. This work strengthens our commitment to transforming care and improving the well?being of the communities we serve.”
The WVU Cancer Institute serves as the coordinating center for this multicenter trial, which is expected to open at approximately 15 sites across North America. Due to the extreme rarity of MPAL, enrollment is projected at one-to-three patients per site, per year. The WVU Cancer Institute is currently the only active enrollment site and is accepting trial participant referrals from across the globe.
“This achievement underscores that transformative cancer innovation is not confined to large institutions,” Emadi said. “It’s happening here in West Virginia, and it’s reaching patients who need it most.”
To learn more about the WVU Cancer Institute MPAL clinical trial or refer a patient, email MPALStudyWVUCI@hsc.wvu.edu or call 304-293-5949.
Editor's Note: Shown is Ashkan Emadi, M.D., Ph.D., principal investigator of the study.
