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Researchers Collaborate to Search for Answers to Children's Health Problems

  1. Top of page
  2. Researchers Collaborate to Search for Answers to Children's Health Problems
  3. National Agencies Shed Light on Global Problem of Substandard Drugs
  4. Health Survey Shows African Americans Most Willing to Participate in Medical Research
  5. St. Jude Researchers Develop Way to Mine Data to Better Understand Cancer

Virginia Polytechnic Institute and State University, Children's National Medical Center, and George Washington University have launched a joint effort to support 7 translational research projects that seek answers to questions surrounding children's health issues.

The $375,000 effort provides seed money for the projects, with the goal of eventually obtaining extramural funding through agencies such as the National Institutes of Health. It is being coordinated through the Clinical and Translational Science Institute, a partnership between Children's National Medical Center and George Washington University, both based in -Washington, D.C.

“The goal of the program is to do research in a way that none of us could do by ourselves,” says Michael Friedlander, PhD, associate provost for health sciences at Virginia Polytechnic Institute and State University, a.k.a. Virginia Tech, in Blacksburg, Virginia. “We hope these projects that are in their infancy will start to get exciting results by the end of the year.”

Researchers will focus their efforts on epilepsy; neuroblastoma, a pediatric cancer that develops from immature nerve cells found in several areas of the body; and medulloblasoma, the most common -pediatric brain tumor.

Each project will include a Virginia Tech co-investigator and a co-investigator from either Children's National or George Washington University. The institutions have an outstanding biomedical research program, while Children's National has “a wonderful sample cohort of patient materials and records,” Dr. Friedlander says. Although best known for engineering, Virginia Tech is also strong in bioinformatics, particularly data analytics and visualization, genomics, and human and brain imaging, he adds.

For one of the projects, Harry Dorn, PhD, a chemistry professor at Virginia Tech, has partnered with Anthony Sandler, MD, chief of surgery at Children's National, to target both diagnostics and therapeutics for neuroblastoma. Dorn, an internationally recognized nanochemist, developed a novel version of buckyballs, a soccer ball-shaped carbon molecule that can help show the specific borders of a tumor during an imaging procedure and deliver radiation to the tumor through brachytherapy.

Likewise, researchers Gregorio Valdez, PhD, an assistant professor at Virginia Tech, and Judy Liu, MD, PhD, an assistant professor at Children's National, will examine a new set of molecular tools to study abnormalities in the brain that can lead to difficult-to-treat seizures and characterize genetic and molecular -changes related to these abnormalities as potential targets for treatment.

A third project focuses on enabling physicians to visualize long-dwelling, peripherally inserted central catheters without having to repeatedly expose pediatric patients to ionizing radiation. Lissett Bickford, PhD, an assistant professor of mechanical engineering at Virginia Tech, along with Raj Shekhar, PhD, a principal investigator in the Bioengineering Initiative at the Sheikh Zayed Institute for Pediatric Surgical Innovation and attending neonatologist An Massaro, MD, both at Children's National, have developed novel polymers and composite materials to place on catheter tips to allow visualization using infrared light, which is safer, Dr. Friedlander notes.

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Results from studies focused on epilepsy, and pediatric cancer and brain tumors could come by year's end.

National Agencies Shed Light on Global Problem of Substandard Drugs

  1. Top of page
  2. Researchers Collaborate to Search for Answers to Children's Health Problems
  3. National Agencies Shed Light on Global Problem of Substandard Drugs
  4. Health Survey Shows African Americans Most Willing to Participate in Medical Research
  5. St. Jude Researchers Develop Way to Mine Data to Better Understand Cancer

The problem of falsified and substandard drugs is a global public health issue that requires an international effort to address, according to a report issued by the nonprofit group, Institute of Medicine (IOM).

Titled “Countering the Problem of Falsified and Substandard Drugs,” the report was commissioned by the U.S. Food and Drug Administration (FDA) and released by the IOM in February 2013 to jumpstart public discourse on the topic.

Substandard medicines are drugs that are made improperly and may not dissolve, or may be made from incorrect or impure ingredients, according to authors of the report. These drugs pose threats around the world, but cause the most significant problems in developing countries with minimal or nonexistent regulatory oversight, according to the study.

“We tried to identify the causes of the problem,” says Gillian Buckley, MPH, PhD, study director of the IOM's board on global health. “A lot of times it's a failure of manufacturing practices; they don't have access to the best equipment, quality control staff, proper cleaning protocols, or water filtration systems.”

While multinational companies operate on a large enough scale to generate profits to maintain good manufacturing processes, small companies often do not. The companies in these countries need hard currency loans to improve their manufacturing, but they have few mechanisms by which to raise capital, Dr. Buckley notes.

The IOM committee also found that crime and corruption—which are rampant in some regions of developing countries—drive the business of falsified medicines. Because the sale of medicines is not well regulated in these regions, many suppliers can, and do, charge excessively high prices for the drugs. A month's supply of the lowest-priced generic ulcer medicine costs more than 3 days’ wages for the average government worker in much of Africa, Eastern Europe, and the Middle East. But, improving the drug regulatory system in these countries is challenging, as often only a half dozen people in these regions are dedicated to maintaining drug safety, Dr. Buckley adds.

“The scope of the needs can be pretty overwhelming,” Dr. Buckley says, noting that IOM recommended overseas drug safety regulators develop a strategic plan to improve their agencies. Meanwhile, FDA officials say that the agency is collaborating with foreign counterparts to create regulatory strategies to implement and control good manufacturing practices.

While there's no lack of programs to combat malaria and infectious diseases in developing countries, it is equally important to tackle substandard and falsified drugs, says Dr. Buckley. “All of the advances we've made against infectious diseases in these countries can be undermined by drug quality,” she adds.

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Falsified and substandard drugs pose a global threat, particularly in regions of the -developing world where safety regulations are minimal.

Health Survey Shows African Americans Most Willing to Participate in Medical Research

  1. Top of page
  2. Researchers Collaborate to Search for Answers to Children's Health Problems
  3. National Agencies Shed Light on Global Problem of Substandard Drugs
  4. Health Survey Shows African Americans Most Willing to Participate in Medical Research
  5. St. Jude Researchers Develop Way to Mine Data to Better Understand Cancer

A study of nearly 6,000 people in the U.S. shows that African Americans are more likely than members of other racial and ethnic groups to be willing to take part in medical research.

“We were flabbergasted at how many people want to participate in research, and overwhelmed by the number of African Americans who were interested in participating,” says lead investigator Linda Cottler, MPH, PhD, chair of epidemiology at the University of Florida's College of Public Health and Health Professions, and the College of Medicine, both in Gainesville, Florida. Dr. Cottler is also founder of HealthStreet, a community health engagement program for underserved populations in Gainesville and in St. Louis, Missouri.

The findings by Cottler et al, published in the American Journal of Public Health in February 2013, showed that 91% of African Americans expressed an interest in participating in medical research compared with 85.5% of whites, 84.5% of Hispanics, and 79% of Asians. These responders also said they would provide blood or tissue samples, grant access to medical records, or stay overnight in the hospital if asked.

The high level of interest among African Americans and other minorities is not only encouraging, but also an indication of the positive influence the National Institute of Health's Clinical and Translational Science Awards (CTSA) program has had in engaging communities in clinical research efforts, Cottler adds. As part of the study, community health workers spoke with people in barbershops, parks, bus stops, churches, and grocery stores. Interviews were conducted by 5 universities that are CTSA recipients: Washington University in St. Louis, Missouri; the University of California at Davis; the University of Michigan at Ann Arbor; Albert Einstein College of Medicine of Yeshiva University in New York, New York, and the University of Rochester in New York.

“The change has been transformative,” Dr. Cottler says. “Our data show that when you do a study in the community, people are likely to participate, but they haven't been given opportunities to do so.” The domino effect of sending out community health workers to talk to people has helped improve education about the value of medical research, she adds.

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Linda Cottler, MPH, PhD, says community outreach efforts have helped build awareness among racial and ethnic minorities to the importance of clinical research.

St. Jude Researchers Develop Way to Mine Data to Better Understand Cancer

  1. Top of page
  2. Researchers Collaborate to Search for Answers to Children's Health Problems
  3. National Agencies Shed Light on Global Problem of Substandard Drugs
  4. Health Survey Shows African Americans Most Willing to Participate in Medical Research
  5. St. Jude Researchers Develop Way to Mine Data to Better Understand Cancer

Using genomic sequencing data previously known as “junk DNA,” scientists have developed a method to mine repetitive segments of DNA at the end of chromosomes—known as telomeres—to better understand cancer.

Their research is part of the Pediatric Genome Project, a collaboration between St. Jude Children's Research Hospital and the Pediatric Cancer Genome Project at Washington University in St. Louis, Missouri. Their findings were published in the December 2012 issue of Genome Biology. Researchers sequenced the complete normal and cancer genomes of more than 600 children and adolescents with some of the most aggressive and least understood cancers with the goal of developing new clinical tools to treat the disease.

Telomeres have been ignored in previous next-generation sequencing studies because scientists had been unable to map them to a position on the human genome due to their repetitive nature. As a result, they were labeled as “junk.”

But the researchers—led by Matthew Parker, PhD, a postdoctoral fellow in St. Jude's computational biology department—developed a method to count the telomeric DNA and look for differences between patients’ normal cells and cancer cells. This technique improves the value of whole-genome sequencing, he says.

Dr. Parker and his colleagues were able to link changes in DNA to mutations in the ATRX gene and to longer telomeres in patients with a subtype of the cancer neuroblastoma. Longer telomeres can lead to unlimited cell division, a characteristic of cancer. Understanding this connection could lead to better prognosis and treatment of neuroblastoma, which in adolescents and young adults has a long survival, yet high mortality rate, according to the study's corresponding author, Jinghui Zhang, PhD, an associate member of St. Jude's department of computational biology.

“This is the first paper to demonstrate the power of this approach; a new way of utilizing next-generation sequencing,” Dr. Zhang says, adding that the team will collaborate with researchers specializing in telomere length analysis to better understand connections to a variety of cancers and other diseases.

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Next-generation genomic sequencing could help scientists better understand genetic connections to diseases.