NanoPediatrics 2008 Symposium

Symposium Speakers

View the video - "Why NanoPediatrics? Because Children are Not Small Adults!"

Edward R.B. McCabe, M.D., Ph.D.

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Imagine if doctors could search out and destroy the very first cancer cells that would otherwise have caused a tumor to develop in the body. What if a broken part of a cell could be removed and replaced with a miniature biological machine? What if pumps the size of molecules could be implanted to deliver lifesaving medicines precisely when and where they would be needed?

The term “nano” is short for nanometer, a linear measure representing one billionth of a meter. To place nano in perspective, by comparative size, a nanometer is to a meter what a marble is to the Earth, and a man’s whiskers grow one nanometer longer in the time it takes him to lift the razor to his face.

The NanoPediatrics Program will focus on the development and use of nanomedicine for the care of children. Nanomedicine – healthcare on a molecular scale – uses nanomaterials and limitless applications of molecular technology, such as nanoelectronic biosensors. The promises of nanotechnology and nanomedicine are new diagnostic and therapeutic tools that will provide truly individualized medicine to improve health.

Already, nanotechnology is changing the way we diagnose illness. For example, a new diagnostic technology involving the identification of the presence or absence of small circular DNA structures generated by normal cells and developed through a partnership of subspecialists in Mattel Children’s Hospital UCLA will help newborn members of the Navajo tribe by revealing severe combined immunodeficiency (Bubble Boy disease). This disease affects 1 in 50,000–100,000 babies in the general population, but 1 in 2,000 Navajo babies. Such an early diagnosis will enable physicians to apply an existing therapy to help these infants.

Mattel Children’s Hospital UCLA established a research core that uses millions of short DNA molecules on a chip the size of a thumbnail to identify gene expression and deletion to discover new causes of and better therapies for such conditions as tumors and genetic diseases. The NanoPediatrics Program proposes to provide pilot funding for projects selected on a competitive basis to permit investigators to obtain larger National Institutes of Health (NIH) grants, thus leveraging the initial investment.

Researchers are developing novel molecular-based nanomachines that will deliver therapies to patients at the cellular level. For example, one therapeutic application under investigation involves a molecular structure made up of protein molecules that appears to “breathe” like a very small bellows, slowly releasing proteins or DNA to target cancers or genetic diseases.

Mattel Children’s Hospital UCLA is strategically located on the Westwood campus, where it is positioned to translate basic science research into improved health for children. The Hospital will partner with the California NanoSystems Institute (CNSI), a research center at UCLA established to encourage University collaboration with industry and to enable the rapid commercialization of discoveries in nanosystems.

Our innovative initiatives will affect children’s care from the community to the international levels through the use of new technologies and the I-PERT Network. An integrated plan will support and disseminate these efforts to dramatically change the way medicine is practiced here at UCLA and beyond. With the Mattel Children’s Foundation’s support, the Hospital can continue to be a leader in improving the quality of life for children by moving this technology forward and leading the way with the first-ever NanoPediatrics Program.