Page 16

THE SCIENCE

Yongjian Liu, PhD; Hong Chen, PhD; Joshua Rubin, MD, PhD; Yuan-Chuan Tai, PhD

Rizwan Romee, MD; Todd Fehniger, MD, PhD; Jeffrey Bednarski, MD, PhD

IMAGE-GUIDED DRUG DELIVERY FOR IMPROVED TREATMENT OF DIFFUSE INTRINSIC PONTINE GLIOMA

MEMORY-LIKE NATURAL KILLER CELL IMMUNOTHERAPY FOR PEDIATRIC AND YOUNG ADULT LEUKEMIA PATIENTS

Washington University School of Medicine bioengineers Yongjian Liu, PhD, and Hong Chen, PhD, are using CDI funding to develop an innovative strategy to treat the single greatest cause of brain tumor–related deaths in children — diffuse intrinsic pontine glioma (DIPG).

Jeffrey Bednarski, MD, PhD, pediatrics; Todd Fehniger, MD, PhD, medicine; and Rizwan Romee, MD, medicine, have joined forces to create hope for children with acute myeloid leukemia (AML). This type of cancer has remained a challenge to treat, requiring many to undergo a bone marrow transplant. For patients who relapse after transplant, no viable options for treatment exist.

Traditionally, the location and scattered nature of DIPG prohibits surgery. Moreover, the disease does not respond to radiation and chemotherapy. Working to provide clinicians with more options, this research team will test the effectiveness and safety of using focused ultrasound to noninvasively, locally and temporally open the blood-brain barrier, which prevents most drugs from reaching the brain tissue. That would allow for ultra-small nanoclusters loaded with an imaging agent and chemotherapy drugs to reach the tumor. Drs. Chen and Liu are testing this novel approach in collaboration with Children’s pediatric neuro-oncologist Joshua Rubin, MD, PhD, using brain tumor models based in mice.

Working to provide clinicians with more options, this research team will test the effectiveness and safety of using focused ultrasound to noninvasively, locally and temporally open the blood-brain barrier.

Their small, phase 1, “first-in-human” clinical trial provided evidence that the immune system’s “natural killer” (NK) cells can be dialed up in the laboratory, trained to recall that activation and then unleashed to destroy cancer cells in some patients. This research team wants to develop an option by trying to mirror the success Drs. Fehniger and Romee had in showing promise for immunotherapy in treating adults with AML. Their small, phase 1, “first-in-human” clinical trial provided evidence that the immune system’s “natural killer” (NK) cells can be dialed up in the laboratory, trained to recall that activation and then unleashed to destroy cancer cells in some patients. Responses to the treatment were observed in five of the nine adult patients that could be evaluated.

To ensure they are hitting their targets, the researchers track the nanoclusters using PET imaging and modeling technologies developed by Dr. Liu’s colleague, Yuan-Chuan Tai, PhD, at Washington University’s Mallinckrodt Institute of Radiology.

14 AS LONG AS THEY FIGHT, WE FIGHT Pediatric Cancer Research

2017 Report to CDI Investors  
2017 Report to CDI Investors