Dear Press On supporters, we look forward to updating you periodically about progress being made as a result of Press On grants.  We are very grateful for your belief in our cause and your trust that we will use your money wisely. This edition will focus on exciting developments at St. Jude Children’s Research Hospital in the fight against neuroblastoma.  One of Press On’s primary goals is taking the laboratory discoveries enabled by Press On support and applying those discoveries in the clinic through clinical trials.  Remarkably, Dr. Wing Leung and his talented support staff have opened three clinical trials as a result of Press On funding! If that weren’t exciting enough, Dr. Leung has also published an article in the peer-reviewed journal Blood that specifically credits Press On! Vong, Q. (2014) TOX2 regulates human natural killer cell development by controlling T-BET expression. Blood, DOI: 10.1182/blood-2014-06-582965.

Relapsed and/or refractory neuroblastoma is difficult to treat and cure. St. Jude has opened a clinical trial, GD2NK, which is a safety and feasibility study, to hopefully give children with neuroblastoma a better chance at survival. This study includes adding two investigational treatments to standard chemotherapy. The first experimental treatment involves the use of a new antibody called hu14.18K322A. The second experimental treatment uses donor (usually a parent or other close relative) natural killer (NK) cells together with chemotherapy. NK cells are special cells in our immune system that specifically target cancer cells and cells that are infected by viruses.  The expectation is that giving the antibody together with NK cell infusions from a donor to children and adolescents with neuroblastoma in combination with chemotherapy will enhance the chances for a cure.


NB 2012 is another exciting clinical trial open at St. Jude because of Press On funding. Treatment for children with high-risk neuroblastoma is usually done in three phases:


Induction therapy (first line treatment with chemotherapy and surgery to kill and then remove as much tumor as possible)

Consolidation therapy (Radiation therapy and high-dose chemotherapy with stem cell infusion or autologous transplant that is given after induction to kill any remaining cancer cells)

Minimal Residual Disease (MRD) therapy (designed to kill small number of cancer cells that may be left).

The main goal of this research study is to find out if adding hu14.18K322A to the first two cycles of induction chemotherapy will result in better tumor responses, compared to treatment given to children in past studies that did not include the antibody.  In addition, the researchers at St. Jude want to find out if giving this antibody with further cycles of induction, consolidation and MRD therapy will be beneficial. Finally, the researchers will study whether NK cells, starting two days after stem cell infusion, during consolidation therapy, will benefit children with high risk neuroblastoma.


RADIANT is a phase I study designed to determine the possibility of a new treatment using stem cell transplantation from a parental donor. This is called “haploidentical” or a half-match wherein the ‘non-matched’ portion of the parent’s immune system serves as a powerful immunotherapy tool to attack the cancer cells. Typically, stem cell transplantation in children with neuroblastoma and other solid tumors involves harvesting the patient’s own stem cells (after receiving several rounds of chemotherapy), storing them, and then re-implanting them after ablating the bone marrow. The RADIANT trial is similarly a two-step process that starts with giving chemotherapy medications to ablate the patient’s bone marrow. The patient then gets stem cells from bone marrow of a related donor. The goal is for the stem cells to engraft and re-build the child’s immune system that will recognize the cancer cells as foreign and attack them.


We are excited that these new clinical trials are open at St. Jude because of Press On’s support and St. Jude’s dedicated work. We believe that donor NK cells in combination with monoclonal antibodies and haploidentical transplantation can give the most aggressive forms of neuroblastoma and other solid tumors a fight our kids will win!