The Institutional Review Board’s Role in Research

CHOC_IRB_ResearchBy Mellisa Henry, director of marketing, CHOC Children’s

As the marketing director at CHOC Children’s, I have a lot of different opportunities to participate in committees that may seem out of the normal marketing realm. One of the most interesting is having a seat on our Institutional Review Board (IRB) for clinical research.

Clinical research is used to determine the safety, dosing and effectiveness of medications, products and treatments used in prevention, diagnosis or treatment of various illnesses or medical conditions. It can range from immunizations to cancer to allergies and anywhere in between.

Any organization that is conducting clinical research is required by law to have its trial approved by an IRB. Organizations have the choice to hire an external IRB, or as CHOC chooses to do, structure an internal IRB. Committees must consist of members with both scientific and non-scientific expertise, and they have the duty to decide whether the proposed research is ethical, informed consent is sufficient, and that appropriate safeguards have been put in place for anyone participating in these trials.

And our responsibility weighs even heavier on my mind because we are acting on behalf of children.

As a non-clinician, I feel my responsibility is to look at the information with the eye of a mom. Do I understand what the research is trying to accomplish? Is the consent in language that allows me to understand? Do I understand what it means to the care of my child? What would my responsibility be in the process? And can I fulfill the responsibilities?

These may all seem like pretty basic questions, but the amount of information presented to a parent when their child is receiving care can be daunting. I want to ensure they can understand the information they receive without too much difficulty and make the choice that is right for their child and family.

Information gathered through clinical trials, and the advances in care that result, are incredible. I am forever in awe of the scientific minds that make these advances possible. But as a parent, it assures me to know that checks and balances are established through an IRB.

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Infectious Disease Clinical Trials at CHOC

Infectious_Disease_Research_CHOCClinical trials involving real patients help doctors and researchers learn the effectiveness of a new drug or medical device, and lead to advancements with potential to improve thousands of lives.

At any time, CHOC Children’s staff doctors, nurses and others are involved in about 300 clinical trials in many specialties, says Dr. Antonio Arrieta, director of infectious diseases and the director of infectious disease clinical research at CHOC.

These trials examine the safety and effectiveness of new medications, vaccines and medical devices as CHOC research physicians and their staffs seek to answer medical questions and develop new or improved drugs that can help children worldwide.

“CHOC is one of the best hospitals in the country and in the world when it comes to providing clinical care,” says Dr. Arrieta. “We also are seeking to make CHOC equally a leader in the discovery and development of new medications and medical devices for children.”

Dr. Arrieta and his team in are assessing the impact of a new and improved vaccine that would reduce invasive Pneumococcal disease, which can cause serious illnesses like pneumonia, meningitis and others.

Dr. Arrieta recently presented data from this research to the international community at a conference in Dublin hosted by the European Society for Pediatric Infectious Diseases and attended by more than 3,000 clinicians, researchers, residents and students.

“We want to share this with the international community,” Dr. Arrieta said. “This infection is of greater severity and more common in third-world countries that don’t have this vaccine.”

Orange County has seen a decline in cases of children with invasive Pneumococcal disease since the first vaccine was released in 2010. It is given to babies at the ages of 2, 4, 6 and 18 months.

Another area of research Dr. Arrieta’s team is working on involves an effort to treat a severe form of fungal infection in infants called candida, which can be fatal for premature babies.

“The current standard of care of is quite toxic so we are involved in an international trial involving a new agent that we think is going to improve the overall outcome of these babies,” he says.

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Autism and Stem Cell Research: Past and Future

training course 1Scientists and researchers at the CHOC Children’s Research Institute continue to study Autism Spectrum Disorders (ASDs) in an effort to find the causes and develop effective treatments for these perplexing and mysterious disorders.

In one of  CHOC’s research labs, Dr. Philip Schwartz and his team, including Drs. David Brick and Omar Khalid, use stem cells made from the skin of patients with autism or ASD and turn them into brain cells to better understand the conditions.

“Autism is a brain disease, so to unravel autism, you have to study the brain. While this seems obvious, it is extremely difficult,” says Dr. Schwartz. “The fact that we now can grow a brain cell from a skin cell is a huge advance.”

Their research began in 2009, when the National Institutes of Health awarded Dr. Schwartz a $3 million grant to hunt for autism’s genetic fingerprint. Colleagues at UC Davis provided skin biopsies from 100 carefully screened autistic boys to establish a cell bank at CHOC for research use by Dr. Schwartz and his team as well as other scientists around the world.

“The goal is to come up with therapies for autism that are based on science and also to develop diagnostic tools so we can diagnose autism or ASD in kids easier and earlier,” says Dr. Schwartz. “As we do this, we can start treatment sooner.”

This research allows scientists to have a culture of a live patient’s brain cells for study in the lab without touching the brain of the actual patient. The only other way to obtain brain cells for this purpose would be to wait for a child with autism or ASD to die, and then seek a brain sample.

The technique of growing brain cells from skin-derived stem cells was developed in 2006 by Shinya Yamanaka, a Japanese scientist who won a Nobel prize in 2012 for his work with stem cells.

Scientists can study autism patients of different backgrounds to make new discoveries, says Dr. Schwartz. He noted that autism is a spectrum of diseases, not just one, and that different patients will likely react to different drugs or may require several drugs.

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  • Research Leads to More Gratitude
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CHOC Using Stem Cells To Study Impact Of Autism

Last year, CHOC Children’s Research Institute received a National Institutes of Health (NIH) Research Grant to generate, investigate, and store neural stem cells derived from skin cells, donated by children with autism. The program is designed to provide an important new tool for studying the impact of autism on the developing brain.

Check out what Philip H. Schwartz, Ph.D., principal investigator on the NIH grant and founding director, National Human Neural Stem Cell Resource at CHOC, has to say about this exciting research:

Q: How unique is this study, and what do we hope to learn from it?
A:  By using easily obtainable skin cells, we can now generate patient-specific brain cells in the laboratory. This allows us to study what is going wrong in the brain of a patient with a genetic disease such as autism without ever having to touch their brain, a huge leap forward if there ever was one!

Only a very few laboratories are doing this and, in fact, the National Institutes of Health is convening a special meeting of scientists, including me, this October to discuss the best ways to move this new and exciting research forward.

Q: What are neural stem cells and how are they obtained?
A:  Neural stem cells are immature brain cells that can divide many, many times and can mature into all the types of brain cells that make up our brains; all the brain cells that make up our entire brain are derived from neural stem cells. We can obtain these cells from the brain itself during surgery or after death or we can derive these cells, using modern technology, from skin rather than the brain.

Q: How will this study benefit patients and families?
A:  Because we can now make brain cells from skin, we can now study brain cells from many patients simultaneously. This will allow us to directly probe what is wrong with these cells and, as a result, come up with new ways to diagnose and treat these very prevalent brain diseases.

Importantly, autism seems to be a class of diseases rather than a single disease and because we can now make patient-specific brain cells from the patient’s own skin, we may be able to tailor therapy to the patient.