PROJECT STELLA MILESTONE:

First CAR T Patient Enrolled and Expanded Mission

We are thrilled to share that the CAR T cell therapy developed in the Project Stella lab has officially enrolled its first patient! This marks a significant milestone in our mission to provide a potentially curative therapy for children battling AML RAM/CBFGLIS—a diagnosis that was once considered incurable. Please join us in sending positive thoughts, prayers, and hope to the little boy in Seattle who received his CAR T infusion just last week.

Building on our success in identifying unique therapeutic targets and developing novel treatments, we are expanding Project Stella’s scope to accelerate the discovery of additional therapies. While our primary focus remains on infants and young children, we are broadening our research to include targets across other subtypes of pediatric leukemia.

This expansion is critical, as several aggressive forms of AML are unique to young children and predominantly affect those under five years old. These include leukemias with alterations in NUP98, ETV6, and KMT2A, which, along with CBF/GLIS (RAM) translocations, account for nearly half of all AML cases in children under five.

New Targets & Therapies in Development

•    CLEC2a – The most advanced target in clinical development, this promising therapy is designed for KMT2A-rearranged AML, a highly aggressive leukemia prevalent in infants and young children. A CAR T-cell therapy targeting CLEC2a is currently being developed in Rome, with the first pediatric patient expected to receive treatment by the end of this year. A similar effort is underway in the U.S.

•    PRAME – Expressed in AML RAM/CBFGLIS, as well as other subtypes of AML, PRAME is a highly promising target. We are finalizing laboratory studies to support its transition into clinical development.

•    WT1 – One of the most compelling targets in pediatric AML, WT1 is highly expressed in infants, including those with CBF/GLIS-RAM AML and other high-risk AML subtypes. Our team is actively developing immunotherapies targeting WT1, currently in the preclinical stage.

Addressing Challenges & Strengthening Collaborations

While the recent news of Sutro’s decision to de-prioritize STRO-002 and limit access to this agent is disappointing, we are actively engaging with Sutro to discuss opportunities to maintain access to STRO-002 for our patients.  As stated in our previous newsletters, STRO-002 was developed by Sutro Biopharma as an antibody-drug conjugate targeting the folate receptor alpha (FRα).  It's originally designed for the treatment of ovarian, endometrial, and non-small cell lung cancers. Given its success as a drug, it was selected as a treatment for AML-RAM/CBF-GLIS. The drug has been successfully administered to more than 30 patients, significantly increasing survival rates for certain AML subtypes, with some patients reaching survival rates as high as 90%. However, despite its effectiveness, some patients develop resistance over time, highlighting the need for additional treatment options to ensure continuity of care, particularly for children. Strategies are in place to identify patients more likely to respond to STRO-002.

In response to Sutro stopping the trials, Dr. Meshinchi has contacted Sutro’s CEO and Chief Medical Officer, who have agreed to provide STRO-002 on a compassionate use basis to patients in the U.S. and Europe until the remaining supply runs out.  Dr. Meshinchi also facilitated a connection between Sutro and the National Cancer Institute's (NCI) Cancer Therapy Evaluation Program (CTEP) to explore the possibility of transferring the drug supply to CTEP.   A follow-up call with CTEP is scheduled to discuss options for acquiring or purchasing the drug and running a trial under the NCI mechanism.

Dr. Meshinchi is also exploring alternative treatments, including the FOLR1-targeting ADC being developed by GenMab. He is requesting a Material Transfer Agreement (MTA) to test the drug as a potential future therapy for patients who were previously treated with STRO-002.  These efforts aim to ensure that patients continue to have access to viable treatments, even as the availability of STRO-002 is limited.  

Additionally, Dr. Soheil Meshinchi continues to collaborate with Dr. Franco Locatelli, Head of Pediatric Hematology and Oncology at IRCCS Bambino Gesù Children's Hospital in Rome. This partnership is accelerating the development of these therapies, ensuring faster global access for children facing high-risk leukemia diagnoses.

We need an additional $500,000 in funding to support ongoing work in advancing and supporting the upcoming clinical trial and the pursuit of two additional targets, PRAME & WT1.  Dr. Meshinchi and his team have found that these targets are present in 30 to 40% of all pediatric AML patients.

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