Mohamed S. Abdelbaki, MD

Project Summary:

Dr. Mohamed Abdelbaki, a CureSearch Catapult awardee at Washington University School of Medicine in St. Louis, is conducting a phase I clinical trial using a Natural Killer (NK) cell therapy for children and young adults with recurrent brain tumors. NK cells are immune cells that can recognize and kill cancerous cells and have shown promise in treating several cancers. However, their widespread use has been limited due to production challenges and the ability of brain tumors to escape killing by secreting an immunosuppressive molecule known as transforming growth factor-beta (TGFβ). Dr. Abdelbaki and his team have established an NK cell therapy that overcomes these obstacles. They have developed a method for producing large numbers of NK cells from healthy donors and by chronically stimulating the cells with TGFβ, they have enhanced their function and made them resistant to the effects of TGFβ in the body. This ‘off-the-shelf’ cell therapy reduces the cost and eliminates barriers associated with manufacturing cells on a patient-by-patient basis, making a potentially superior treatment option more widely available to patients.

In this CureSearch-funded clinical trial, 24 children and young adults with recurrent brain tumors will receive infusions of the enhanced NK cells directly into the surgical cavity following tumor removal. This approach bypasses the blood-brain barrier and treats the tumor more effectively by concentrating the NK cells inside the tumor site. The team will also study how long the NK cells remain active in the brain and how this affects the overall response to treatment. This trial will be conducted through the Pacific Pediatric Neuro-Oncology Consortium (PNOC), making it the first multi-institutional consortium-wide study to evaluate the safety and efficacy of intra-tumoral NK cell infusions in children with recurrent brain tumors.

Research Update December 2025:

Dr. Abdelbaki and his team continue to make strong progress on their NK cell therapy trial for children and young adults with recurrent malignant brain tumors. The study is now open at 7 U.S. sites, including the University of California, San Franciso (UCSF), Washing University in St. Louis/St. Louis Children’s Hospital, Indiana University/Riley Children’s Hospital, University of Alabama Birmingham, Johns Hopkins Hospital, Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center, and Seattle Children’s Hospital. Three additional U.S. sites, including the Children’s Hospital of Philadelphia (CHOP), the Children’s Hospital of Los Angeles (CHLA), and Nationwide Children’s Hospital, are expected to open soon. Three patients have been enrolled in the trial since March 2025. The first two patients successfully completed the initial 30-day safety period without experiencing any dose-limiting toxicities (DLTs), while the third patient began treatment in early November. The team has completed next generation sequencing of tumor samples from all three patients and will continue to expand these analyses as more samples become available.

To broaden access to the trial, the team has successfully amended the study to include additional brain tumor types, and work is underway to open the study internationally in Israel, Australia, and New Zealand. Following the completion of the third patient’s safety window, the team anticipates advancing to the next dose level in mid-December. Additionally, they plan to expand enrollment to include children ages 1-8, allowing younger patients, particularly those with recurrent medulloblastoma and ATRT, to participate in this promising study.

Research Update June 2025:

One year into receiving the CureSearch Catapult Award, Dr. Abdelbaki and his team have made steady progress in launching their groundbreaking clinical trial of an NK cell therapy for children and young adults with recurrent malignant brain tumors. The study is now open for enrollment at five institutions across the country, including UCSF, Washing University in St. Louis/St. Louis Children’s Hospital, Indiana University/Riley Children’s Hospital, University of Alabama Birmingham, and Johns Hopkins Hospital. The first patient was enrolled in March 2025 and has safely completed the first cycle of treatment—receiving three NK cell infusions with no serious side effects. To expand access and accelerate enrollment, the team is actively working to open additional sites nationally and internationally, with several expected to launch in the coming months. Additionally, they have made a key change to the study protocol to allow enrolment of patients with a wider range of brain tumors, including infra- tentorial, multifocal, and metastatic disease, ensuring that more patients have the opportunity to benefit from this promising new therapy.

Research Update December 2024:

In the first 6 months of the award, Dr. Abdelbaki’s team worked diligently with PNOC to prepare for the study launch by ensuring that all necessary documents have been provided to all participating sites. The team expects that the 6 participating institutions who have successfully completed site initiation visits will open the study within the next few weeks and enroll the first patient soon after. These include UCSF, CHOP, CHLA, Washington University in St. Louis, Indiana University, and University of Alabama Birmingham. The team is continuing to work with additional PNOC sites that have expressed interest in participating to launch the trial, including international sites such as Princess Maxima (Netherlands) and IPEN Shaare Zedek (Israel).