You can make a difference. Children’s Stem Cell Program has an ambitious goal: bring its first stem cell-based therapy to the clinic within five years to usher in a new era of healing. We have a detailed strategic plan outlining every milestone and investment necessary to achieve this goal.
Your gift, in any amount, no matter how small, will help get us there. Already, generous donors like you have helped our scientists:
- Launch the first clinical trial of a drug discovered through stem cell research.
- Turn a patient’s skin cells into stem cells that can be made into treatments, promising a future in which we each become our own medicine chest.
- Make breakthrough after breakthrough as they push to bring new, stem cell-based treatments to patients with disorders as different as leukemia, diabetes and ALS.
To reach the clinic in the shortest possible time, Children’s Stem Cell Program must raise $36 million over the next four years.
Every gift counts as we race the clock to:
- Create customized treatments. Cell therapy’s great potential lies in creating healthy cells the patient’s body won’t reject. Children’s stem cell researchers lead in developing the techniques that will make this possible.
- Reverse genetic disorders. Stem cells made from a patient’s own tissues and genetically corrected promise life-saving treatment for diseases, like sickle cell anemia, caused by a single faulty gene. Our researchers were first to show, in animals, that this approach could work. Now, they are perfecting it for humans.
- Detect and defeat cancer stem cells. Rare “cancer stem cells” give rise to and maintain many tumors. Our researchers are revealing how these cells form, how they are sustained, and, most important, how to kill them.
- Model disease. Children’s researchers have created the world’s largest repository stem cell lines for specific diseases — 17 and counting. They and colleagues throughout the world are using these cell lines to unlock the secrets of disorders including diabetes, muscular dystrophy Huntington’s and immune deficiencies.
- Discover new drugs. As they seek chemicals that turn immature stem cells into the cells needed for treatment, as they study cancer stem cells and model disease, our scientists are discovering new drug targets. Their first discovery, already in clinical trials, promises more successful bone marrow transplant for cancer patients.