Yale School of Medicine

Yale School of Medicine

Grants and Contracts Administration47 College Street, Suite 203

New Haven, CT 06510-3209

United States

Development of Nanoparticle-Encapsulated Chemo/Radio-Sensitizers for Intrathecal Delivery

Background
Pediatric brain tumors often are treated with high doses of chemotherapy and radiation therapy. These treatments are effective but lead to significant late effects on long-term survivors.

Elucidating the Role of Human FAN1 Nuclease in DNA Crosslink Repair

Leukemia, Chronic Myeloid Leukemia (CML), Acute Myeloid Leukemia (AML)

Background

Fanconi anemia is a childhood disease associated with impaired bone marrow function, developmental retardation, premature aging and early onset of cancer limiting the lifespan of patients to 35 years. Fanconi anemia results from impairment of one or more steps in the preventive pathway that removes aberrant DNA structures, which are caused by environmental stresses like pollutants, toxins in diet, etc.

Creating Primary Glioma Growth Delay Assays

Brain Tumors, Glioma

Background

Diffuse Intrinsic Pontine Glioma (DIPG) is a rare but lethal childhood tumor with no known effective treatment. Given its location in the brainstem, surgical resection is not feasible, and the only available treatment is radiation therapy, which is palliative in nature. As such, better therapies are needed which can be combined with radiation therapy for durable disease control. Remarkably, there are limited platforms suitable for use in DIPG screening studies to identify such agents.

Creation and Validation of Novel Cell Line Models for Alveolar Rhabdomyosarcoma

Rhabdomyosarcoma

“My POST program experience was the first opportunity I had to work completely independently in the lab….. both my successes and my disappointments were my responsibility, and I became more invested in my research as a result.” –Kaoru Takasaki

Identifying Therapeutic Targets for MiT Associated Pediatric Cancers

General Pediatric Cancer

The long-term goal of this research is to identify therapies for the aggressive translocation renal cell carcinomas that affect children and young adults. A majority of these tumors present at advanced stage and half of the patients with translocation renal cell carcinoma will parish from the cancer. Thus, there is an urgent need to define therapeutic strategies to treat these cancers. To accomplish this goal, we propose to study the microphthalmia (MiT) family genes TFE3, TFEB, and MITF that are known to drive these cancers.

Treating Brain Tumor by Changing Cell Division Pattern

Brain Tumors

A major challenge in treating malignant brain tumors is why killing the vast majority of cancer cells does not lead to a cure. The apparent similarity between the ability of cancer cells to proliferate uncontrollably and that of the stem cells to continuously replenish themselves (self-renew) has led to the notion that cancers are diseases of stem cells and that a few cancer stem cells are sufficient to form new tumors to cause relapse and prevent cure.

Elucidating the Initiation Mechanism of the Fanconi Anemia Pathway of DNA Damage Repair

General Pediatric Cancer

Fanconi anemia (FA) is a childhood disease characterized by multiple devastating symptoms, which include bone marrow failure leading to aplastic anemia, congenital abnormalities, and a high incidence of cancer. Although recent medical advances have increased the life expectancy for FA patients, they often succumb to tumor malignancies before reaching adulthood. Therefore, research into the cellular and molecular basis of FA-associated cancer is urgently needed.

Alex's Lemonade Stand Foundation for Childhood Cancer