Medical Device Daily National Editor

Nanotechnology and personalized medicine – two relatively new concepts that would seem to go arm in arm.

But exactly how early in the individual's life can personalized medicine be used – and how soon should that relatively uncharted territory of nanotechnology be used?

"That's one of the things we want to investigate – when is it appropriate?" says Edward McCabe, physician-in-chief at Mattel Children's Hospital at the University of California Los Angeles (UCLA).

He hopes that this and related questions will be answered by a new effort at the hospital, the Mattel UCLA NanoPediatrics Program, to explore personalized medicine's applications for children, including both the opportunities and the risks of applying nanomedicine for this group.

The hospital says that the program is one of the world's first dedicated solely to the use of nanomedicine for pediatric patients.

As founding director of the program, McCabe rhetorically asks another question: "Why develop a nanopediatrics program?"

"Because," he told Medical Device Daily in answer, "one of our mantras in pediatrics is that children are not small adults."

He cites the evidence that drugs work differently in children – "they metabolize, excrete and may even utilize, developmentally, specific receptors differently than adults." And he emphasizes that an adult-only approach as a priority for research in this area is a "flawed strategy" that could put children at risk as nanotechnology reaches maturity.

McCabe says that while the potential dangers of nanotechnology have been overblown "with no evidence," it is clearly necessary "to look at the risk side, not bury our heads."

He adds: "One of our founding members is Dr. Andre Nel. He has done a lot of work with nanotoxicology to look at this — not only at the risks to adults, but also to animal models, [this research] fitting into the pediatric period."

Nel is founder and chief of the Division of NanoMedicine at UCLA's Department of Medicine, which defines its mission as educating "physicians and scientists in the principles and application of nanoscience and nanomaterial applications and safety in medicine."

The Mattel NanoPediatrics Program was officially formed in May of this year but rolled out at a special symposium this past Friday for the purpose of announcing a $1.8 million grant from the Mattel Children's Foundation to fund its research work.

Using the personalized medicine/nanomedicine approach early in an individual's life is an obvious basic target for this field, McCabe says.

As one example of nanomedicine's potential benefits, he cites premature infants "that are exposed to antibiotics associated with hearing loss in individuals who have a mutation in their mitochondrial DNA. Wouldn't it be great to take DNA from that neonate and in 15 minutes ... check to see if this is a baby at risk?"

He cites also the development of a pilot program in Wisconsin, through a partnership of sub-specialists at Mattel Children's, to identify the presence or absence of small, circular DNA structures generated by normal cells, by revealing severe combined immunodeficiency (known also as "Bubble Boy disease"), much more common than average among Navajo neonates. He notes that this disease afflicts one in 50,000 to 100,000 babies in the general population, but one in 2,000 Navajo babies.

He says this represents the use of microarrays at the nanoscale level to apply an existing therapy to help these infants.

Friday's symposium to announce program funding also featured presentations on nanomedicine, a majority of them focusing on pediatric oncology. And McCabe told MDD that cancer is likely to be a primary focus of the initial research work by the NanoPediatrics Program.

Mattel Children's says that it already is working to develop nanodevice procedures for the treatment of children with genetic diseases and cancer, and investigate the use of nanoparticles for diagnostic imaging both during pregnancy and after birth. McCabe says that the NanoPediatrics Program will partner with the California NanoSystems Institute at UCLA, an integrated research center established in 2000 to encourage university collaboration with industry and enable the rapid commercialization of discoveries in nanosystems.

The $1.8 million funding for the program is essentially start-up funding, seed financing that will offer a platform for developing research targets and applications that will be used to leverage grant support, several-fold, McCabe says, from the National Institutes of Health. One possibility he predicts is an application for funding a program to train clinicians in the use of nanomedicine for pediatric applications.

Kevin Farr, CFO of Mattel Children's and chairman of the Mattel Children's Foundation, said that the foundation "is excited to support this groundbreaking program in nanopediatrics, which can potentially revolutionize the research and treatment of illnesses that affect young patients."

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