Holding on to the future of medicine

New repository saves precious resources for disease-fighting discoveries

Every year hope is put on hold as promising medical research gets delayed and scientists struggle to obtain rare resources vital to their work.

The resources are patient tissue and blood samples containing cells, plasma, serum, DNA, RNA and other biological components that help form the building blocks of life. These samples are vital to research and help provide clues to finding new causes and cures for disease.

At Cincinnati Children’s, large quantities of these samples are collected as part of routine clinical care from more than 300,000 children who visit the medical center annually for treatment. Portions of the samples are used for each child’s diagnosis and care. The rest of the material is destroyed and lost forever, along with the biological information it contains. Meanwhile, researchers spend hundreds of thousands of dollars (or more) and months or years searching for rare patient samples, just so a study can get under way or funded.

“We have an obligation to make sure the samples collected here are not just thrown away, especially when they are so critical to most of the research we do,” explains Michael Barnes, PhD, a researcher in the Division of Rheumatology.

The biobank project

So Barnes helped spearhead and will direct a new project at Cincinnati Children’s, the Cincinnati BioBank, which will save and store remnant clinical samples. “There is a broad need for these samples and this project will be significant in helping fill the gap,” he says.

Meticulously reviewed and approved by the medical center’s Institutional Review Board (IRB), the project’s primary purpose is to assemble, organize and store leftover clinical specimens.

The specimens will form a centralized institutional collection of biological samples for Cincinnati Children’s researchers and those with whom they work. Researchers will not have access to patient identities; they will focus exclusively on biological components in the samples and how they relate to their specific area of scientific or biomedical inquiry.

One of only a few

The collection of residual clinical samples will be one of only a handful of such programs in the nation, in keeping with the institution’s role as an innovator in pediatric medicine and research, Barnes says.

The biobank could allow important studies to proceed more quickly, at less cost. It also is expected to simplify pilot stage research projects at Cincinnati Children’s and make grant submissions more competitive for research dollars from the National Institutes of Health and other funding organizations.

Ultimately, getting research started more quickly and efficiently with critical biological resources at hand could lead to faster answers for some of the complex medical questions that stymie researchers and clinicians. But Barnes, ever the scientist, is cautious on this point, saying, “We’ll have to see what happens.”

Patient protections crucial

The IRB protocol document for the biobank includes specific requirements for the protection of patients and their personal information. It also includes a number of review processes to ensure samples are used only for ethical research projects of valid scientific merit.

Clinical samples can be stored in the biobank only if a patient or his legal representative signs a form agreeing to participate. Scientists using the samples will not have any personal information about the patients because samples are “de-identified.” Researchers must agree not to attempt to identify patients whose samples they use.

As a further safeguard, samples from the biobank will be made available only to scientists whose studies are approved by the biobank’s “tissue use committee” for appropriate scientific merit. Merit is decided by whether the project takes aim at a legitimately important biomedical issue and whether the project has a reasonable chance – by virtue of its design and other factors – of succeeding.

“We evaluate each project and investigator to make sure the request makes sense,” Barnes says. “They have to explain to us in exhaustive detail everything they want to do with samples. They aren’t allowed to do anything they want. The samples must be used for legal, ethical research that has a reasonable chance of success.”

Program safeguards are so stringent that even sample amounts released to approved researchers will be tightly controlled to ensure they receive only what is needed to complete specific study experiments.

A competitive edge

In the end, having a facility like the biobank on premises will make studies conducted at Cincinnati Children’s more competitive and successful – in part because it prepares the institution for one of the harsher realities of biomedical research: funding.

Money – especially federal grant funding – is harder to win in an increasingly competitive environment, says John Harley, PhD, director of the Center for Autoimmune Genomics and Etiology at Cincinnati Children’s and the Division of Rheumatology.

A successful biomedical researcher with decades of experience, Harley sees the biobank as vital to the success of the institution and its individual scientists.

“Between 1980 and 2007, the number of faculty members in US medical schools increased from something like 19,000 to 120,000 – a sixfold increase in the number of people trying to conquer disease, all competing for the same resources,” Harley says.

He notes that even the best scientists can have a less than one-in-10 chance of winning NIH funding for a project. The result is that sometimes, good research projects go unfunded and uncompleted, and scientists leave the field to o something else.

“With a biobank centralizing collection of these materials, it provides an institutional base so that an investigator wanting to study a particular disorder would be able to retrieve samples with a minimum of effort,” says Harley. “This would put them in a much stronger position to compete for funding and to start and sustain he work.”