Once again, for the 2011 funding cycle, we had some great submissions from around the world. Unfortunately, despite the high quality of the grant applications, we can only afford to fund two for this funding cycle. The two $50,000 grants for this year were awarded to Dr. Miguel-Angel Perales of Memorial Sloan-Kettering Cancer Center of New York, NY and Dr. Omar Abdel-Wahab of Memorial Sloan-Kettering Cancer Center, as well . Though both of the awards go to the same institution, the requests are individually unique. We congratulate both of these recipients for their comprehensive applications and wish them significant success with these projects that they might help us find a day when everyone survives.
Dr. Perales describes his research entitled “T-cell Precursors in Allogeneic Hematopoietic Stem Cell Transplant” in the following manner:
Approximately one-third of the nearly 4000 acute lymphoblastic leukemia (ALL) cases in the United States occur in adults. Although remission rates are nearly equivalent in children (> 90%) and adults (> 78%), the overall cure rate is much lower in adults than children (< 40% versus 80%). Patients with relapsed or refractory leukemia have a dismal prognosis with a short survival. Allogeneic hematopoietic stem cell transplant (HSCT), where stem cells are obtained from the blood or bone marrow of a donor, is a curative therapy for many patients with leukemia. However transplants are associated with delays in recovery of the immune system, which results in increased risks of infection and leukemia relapse. We are working on new strategies to enhance immune recovery after transplant and improve overall outcomes. The immune system is made up of white blood cells and includes B cells that make antibodies and T cells that are responsible for killing cells infected with viruses. T cells are also able to kill tumor cells in some cases. Recently, we have developed a novel approach to promote T cell recovery based on the transfer of T cell precursors that can be grown in the laboratory. The T cell precursors are derived from cord blood and will develop into T cells once they are transferred into the patient who is undergoing a transplant. We have developed a specialized “Notch-based” culture system to grow T cell precursors in the laboratory and are able to further enhance the anti-leukemia activity of these cells by engineering them to express receptors specific for CD19, a marker present on the surface of most ALL and non-Hodgkin lymphoma (NHL). The specialized receptor is called a chimeric antigen receptor or CAR. The goal of this proposal is to establish an FDA-approved culture system for the generation of human T cell precursors engineered to express a CAR targeting human CD19. We will then conduct a phase I clinical trial to evaluate the T cell precursors in patients undergoing an allogeneic transplant for advanced ALL or NHL. The project draws on the resources and expertise of several teams at MSKCC, including members of the Bone Marrow Transplant and Leukemia Services, and the Gene Transfer and Somatic Cell Engineering Facility. The study represents an innovative immunotherapeutic approach for patients with advanced leukemia, including relapsed or refractory ALL.
The goal of Dr. Abdel-Wahab’s study, entitled “Understanding the role of ASXL1 mutations in acute myeloid leukemias” is explained as follows:
The development of genomic technologies has uncovered a number of genetic abnormalities in patients with cancer, including acute myeloid leukemia (AML). In large-scale genomic studies we have found that the gene ASXL1 (Addition of Sex Combs-Like 1) is commonly mutated in AML patients. Moreover, we have identified that patients with ASXL1 mutations have a significantly worse survival. We have therefore performed studies to understand the function of ASXL1. In addition to furthering our knowledge of AML development we hope to identify strategies to aid in the therapy of AML patients with this genetic abnormality.
We are proud to partner with Drs. Perales and Abdel-Wahab on these initiatives and pray that the results of their research will take us all a step closer to finding a cure for leukemia. As well, we are proud to announce a special award this year, the Chairman’s Choice Award. Upon receipt of the requests this year, there was one request that didn’t fit into our typical laboratory based research, however, it was so compelling from a humanitarian standpoint, the Medical Advisory Board asked if we could find a way to fund it. Therefore, in addition to the two $50,000 research grants going to Memorial-Sloan Kettering, an additional $25,000 award will go to Sara Day, RN, PhD in the International Outreach Center at St. Jude Children’s Research Hospital. Here’s how Dr. Day will use her award.
Although 80% of children with acute lymphoblastic leukemia in high-income countries are cured, most children live in low-income countries where cure rates are much lower. In these countries, deaths due to side effects of treatment (toxicity of treatment) and missed appointments during active treatment (abandonment of treatment) are the two most common causes of treatment failure and occur even more frequently than relapse. Improved supportive care and family education can address these problems, and the role of the pediatric oncology nurse is therefore critical. Well-trained nurses prevent infection through rigorous infection control efforts and reduce mortality by early detection of symptoms and prompt intervention. They also can reduce abandonment by educating patients and families and increasing their confidence in the health system.
Unfortunately in low-income countries there is inadequate nursing education, and one nurse is often required to care for 10 to 20 patients. The purpose of this study is to assess the impact of improved nursing education and staffing on toxic death and treatment abandonment in children with acute leukemia at the National Pediatric Oncology Unit in Guatemala, where 350 children with newly-diagnosed cancer are treated annually. The effect of nursing education and better nurse staffing on patient outcomes has been extensively researched; however, almost all studies were conducted in high-income countries. This will be the first study to attempt to establish a relationship between abandonment of treatment and toxic death and interventions specifically related to nursing in a low-income country.
With the funding of these three grants totaling $125,000, the When Everyone Survives Foundation has funded $625,000 in leukemia research initiatives since inception. We know that more must be done but for a fledgling charitable foundation to fund well over half a million dollars in just five funding cycles is something for which we are excited and thankful to the many donors and supporters that made it possible. By continuing to work together in the future, it is our prayer that we may find that day when everyone survives.
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