First-in-World Trial Explores Subcutaneous Blinatumomab in Rare Leukemia

In a conversation with CancerNetwork^®, Ashkan Emadi, MD, PhD, discussed the background of a first-of-its-kind phase 2 trial (NCT07222579) investigating subcutaneous blinatumomab (Blincyto) as a treatment for patients with CD19-positive mixed phenotype acute leukemia (MPAL). According to a recent press release from West Virgina University (WVU) Medicine, the first patient enrolled on the trial initiated treatment with blinatumomab on January 16, 2026. After a single cycle of therapy, the patient experienced complete remission with full hematologic recovery, which included transfusion independence and the absence of the Philadelphia chromosome. Additionally, treatment transitioned fully to the outpatient setting after the first week.

Emadi noted that this patient was the first in the world to receive subcutaneous blinatumomab for MPAL, a rare form of acute leukemias. He outlined the key clinical characteristics and prognosis associated with MPAL, which currently lacks an FDA-approved therapeutic option for patients. Given current survival expectations and no available treatment strategies, Emadi and colleagues aimed to assess whether blinatumomab, which has demonstrated efficacy in other populations like B-cell acute lymphoblastic leukemia (ALL), could provide a new option in this rare leukemia subtype.

Emadi is chair of the Department of Medical Oncology; Alexander Bland Osborn Endowed Chair and distinguished professor of Medical Oncology; physician-in-chief of Medical Oncology; and associate director for Clinical Research at WVU Cancer Institute.

Transcript:

The currently approved version of blinatumomab has been an extremely successful story in terms of the improvement of overall survival and quality of life of patients for both adults and children with B-cell ALL. Continuous infusion of blinatumomab is approved for relapsed/refractory B-cell ALL for minimal residual disease–positive B-cell ALL and for upfront treatment in combination with multi-agent chemotherapy for patients with B-cell ALL. Subcutaneous blinatumomab has been used in clinical trials by different institutions for treatment of patients with relapsed or refractory B-cell ALL, but it has never been used for the treatment of MPAL. This patient that we are reporting [on] is the first patient on the planet Earth, on the globe, with MPAL who has been treated with subcutaneous blinatumomab.

Let’s talk about MPAL for a second. MPAL is a very rare type of acute leukemia, [representing] 1% to 3% of acute leukemia. These cells have both myeloid markers as well as lymphoid markers. The lymphoid marker can be either B cells or T cells. The target population are patients with B/myeloid MPAL. They are about 1% to 2% of all acute leukemia, which is about 500 patients in US per year. It looks like it’s very rare, but if there are 500 patients in the US, that means that there are 10,000 patients everywhere in the world who are diagnosed with this disease. Unfortunately, as of today, there are absolutely no FDA-approved disease-specific therapies for patients with MPAL. [For] people over the age of 80, the median survival of patients with MPAL is only 2 months; only 3.5% of people [with this disease] are alive in 2 years. [For] people over the age of 70, only 5.5% are alive in 2 years. It's a clear definition of unmet need.

The idea [behind the trial] was simple. Blinatumomab is very successful for CD19-positive B-cell [ALL], and now we have a much less cumbersome [subcutaneous] formulation of blinatumomab. Why not provide it for patients with CD19-positive MPAL who have absolutely no other options?

Reference

WVU Cancer Institute first in the world to treat ultra-rare leukemia using novel subcutaneous immunotherapy. News release. WVU Medicine. February 17, 2026. Accessed February 18, 2026. https://tinyurl.com/2s4y3xmr