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We are continuing to make significant strides at the Children’s Research Institute at UT Southwestern (CRI). CRI is now home to 44 researchers who continue to focus on biomedical research that has the potential to change scientific fields and yield new strategies for treating diseases that would impact countless children in Texas and beyond.
We’re focusing our research efforts at the interface of regenerative medicine, cancer biology and metabolism, because those three areas are intricately involved in the way healthy and diseased cells function in the body, offering significant opportunities to increase our knowledge and make progress toward the treatments of tomorrow.
As I’ve said many times, ultimately our goal is to cure somebody who would not have been cured otherwise. We still have a great deal of work to do, but here’s an update on a few of the exciting projects we’re working on and why they’re important.
In my lab, we have developed an innovative model for predicting the progression of skin cancer in patients by observing the rate at which human cancer cells spread in mice. By transplanting Stage III human melanoma cells from patients into mice, my team was able to analyze the rate at which the cancer spread in the mice, which correlated with clinical outcomes in the patients.
Simply put, the melanomas that metastasized (spread) most efficiently in the mice also progressed to advanced Stage IV disease in the patients, spreading to other organs such as the brain, liver or lungs; those that did not metastasize as efficiently in the mice did not form distant metastases in the patients either and tended to be cured by surgery.
We’re now working to identify new prognostic markers that will allow us to predict when patients with melanoma are in trouble and new therapeutic strategies to treat the disease.
Our goal is to focus on the most effective medication or treatments and, just as we are now able to know ahead of time from mice which patients with melanoma are at the greatest risk, we hope we eventually will be able to know ahead of time from mice which treatments will have the greatest chance for success.
We’ve also been working on identifying the environment in which blood-forming stem cells thrive within the body, which is an important step toward improving the safety and effectiveness of bone marrow transplantation.
Bone marrow transplants are the best example of stem cell therapy and save thousands of lives each year. As a result of this research, we hope to one day make it possible to take a small number of stem cells out of a patient’s own bone marrow, grow them into a larger number of stem cells in the lab, and then transplant them back into the patient. This would improve the safety of bone marrow transplants and make it available to more patients, including children who receive bone marrow transplants following treatment for leukemia.
We also believe these efforts may be effective for treating other illnesses associated with loss of infection-fighting cells, including certain immunodeficiency syndromes in children such as Severe Combined Immune Deficiency.
These projects highlight what we are trying to achieve at CRI: take a basic scientific approach to solve a fundamental problem that has the potential to change the way we treat disease in the future, which will ultimately help Children’s fulfill its mission to make life better for children.