Category: Health

New Brain Study Tailored for Nonverbal Children on the Autism Spectrum

Originally published in Bench to Bedside, the CHOP Research monthly publication.

I composed this original article based on interviews with the investigators.

Excerpt:

The children who speak least — or not at all — are rarely represented in cutting-edge brain imaging research on autism spectrum disorder (ASD). Researchers at The Children’s Hospital of Philadelphia aim to change that with a new study enrolling children with ASD who are minimally verbal or nonverbal.

“This is really an underserved community who have not been given the opportunity to participate in research,” said Timothy Roberts, PhD, vice chair of research in the Department of Radiology at CHOP and a professor of Radiology at the Perelman School of Medicine at the University of Pennsylvania. “More importantly, the results of the research are not directly applicable to them, so even if we developed a drug for ASD impairments led by our biomarkers, we wouldn’t know if this treatment really was good for individuals with ASD with more limited verbal and cognitive ability.”

Dr. Roberts and colleagues from CHOP’s Center for Autism Research want to know whether certain aspects of neural rhythms and timing of neural firing that they found to be characteristic of ASD in previous studies, using noninvasive brain imaging called magnetoencephalography (MEG), are indeed common across the spectrum.

“When neural activity is happening, it produces electrical and magnetic fields,” said J. Christopher Edgar, PhD, a co-leader of the study and a clinical neuropsychologist and brain imaging researcher in the Department of Radiology at CHOP. “We use this machine to measure the magnetic field. We do that to look at brain function.”

The neural biomarkers that Dr. Edgar and Dr. Roberts have found correlate with the level of clinical impairment in the children they have studied to date, particularly in the realm of language ability.

“There is a scientific question: Does this correlation extend into this nonverbal and minimally verbal population in a continuous way, or is it a separate disorder?” Dr. Roberts said. “I suppose a fundamental question is: Is the autism spectrum continuous or discrete in terms of these brain markers?”

Researchers Test Magnetic Re-Seeding to Speed Blood Vessel Recovery

Originally published in Bench to Bedside, the CHOP Research monthly publication.

I composed this original article based on an interview with the investigator.

Excerpt:

Reopening blocked blood vessels can mean the difference between life and death for many patients, including children with pulmonary hypertension and adults with coronary artery disease. But the procedure used for restoring blood flow can cause extensive vascular injury, wiping away an essential protective layer of cells lining the walls of arteries and veins: the endothelium.

It may take the body weeks or months to heal that damage. Some long stretches of damaged vessels may never fully regrow the endothelium, which naturally propagates inward from the healthy edge toward the center of the wiped-out area.

“Our idea is that if we could put some seeds of endothelium regrowth within the boundaries of this denuded arterial segment, so that the endothelium can grow from those foci, we could speed up the process dramatically,” said Michael Chorny, PhD, a researcher at The Children’s Hospital of Philadelphia and research assistant professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania, who specializes in developing delivery systems for therapies. “By changing the pattern of endothelium recovery, we may in practice restore it on scale of days.”

Researchers Ask How Children With Autism Communicate Pain

Originally published in Bench to Bedside, the CHOP Research monthly publication.

I composed this original article based on an interview with the investigator.

Excerpt:

When children with autism spectrum disorder (ASD) have acute medical needs, such as a broken bone or appendicitis, they are as likely to experience pain as any other child. But social and communication differences associated with ASD may cause clinical caregivers to miss cues showing that pain. This may compromise timely and accurate assessment and treatment. Recognizing this challenge, researchers at The Children’s Hospital of Philadelphia conducted a qualitative study that suggests clinicians should be prepared to ask children on the spectrum about pain in different ways.

The interdisciplinary study team set out to learn more about how these youth express and describe the experience of pain. They enrolled participants with an ASD diagnosis who came to CHOP for surgery and whose verbal language proficiency was sufficient to complete interviews about their pain during their hospital stay after their operation. The results were published in January in the Journal of Developmental and Behavioral Pediatrics.

Early Smell Exposure May Be Critical for Sensory Development

Originally published in Bench to Bedside, the CHOP Research monthly publication.

I composed this original article based on interviews with the investigators.

Excerpt:

Many parents try to offer their healthy babies a rich sensory environment, full of new sights, sounds, smells, and tastes, to stimulate the developing brain to appreciate life’s delicious complexities to the fullest. And for vision and hearing, there is a solid foundation of research showing that there is a critical period early in life when adequate stimulation of those senses is essential for their healthy development.

Now, a pilot study conducted at The Children’s Hospital of Philadelphia and published in the journal JAMA Otolaryngology provides some of the first-ever evidence for a critical period in developing the sense of smell, or olfaction, too. The findings have particular implications for rehabilitating young patients, including severely premature infants, who receive lifesaving medical interventions that temporarily prevent airflow through the nasal passages during this potentially critical period.

Four Projects, One Goal: Curing Childhood Cancer

Originally published in Bench to Bedside, the CHOP Research monthly publication.

I prepared this article based on the project descriptions and email correspondence with the investigators.

Excerpt:

Many cancer treatments have harmful side effects when they act on healthy tissues in addition to cancer cells. A team led by Garrett M. Brodeur, MD, director of the Cancer Predisposition Program at CHOP, and funded by a CURE grant, is seeking ways to increase drug delivery to the tumors to improve drugs’ effectiveness while reducing their toxicity.

Their method uses tiny nanoparticles as delivery vehicles. Nanoparticles are a promising way to get drugs into tumors because tumor blood vessels are leaky, and the nanoparticles can enter the tumor much more easily than normal tissues.

“By increasing drug delivery to tumors by one or two orders of magnitude, we can achieve dramatically better anti-tumor effects while simultaneously decreasing total drug exposure to patients,” Dr. Brodeur said.

CHOP Big Data Center Visits White House for Precision Medicine Summit

Originally published on Cornerstone, the CHOP Research Blog.

I composed this original article based on an interview with the investigator.

Excerpt:

Today at the White House, President Obama welcomed guests, including Adam Resnick, PhD, representing The Children’s Hospital of Philadelphia, for a morning of remarks and discussions about what the Precision Medicine Initiative (PMI) has achieved to date, and how it can take the next steps into the new era of medicine that delivers the right treatment at the right time to the right person. One way CHOP will be an integral part of that effort is through its commitment to data-driven discovery in pediatrics.

The PMI, which President Obama first announced in his 2015 State of the Union speech, launched last year with a White House event attended by CHOP leukemia patient Emily Whitehead and by the hospital’s then-CEO, Steven Altschuler, MD. The pair was invited in recognition of the progress at CHOP and the University of Pennsylvania in developing an investigational precision-medicine T-cell therapy for cancer patients like Emily.

But that type of discovery is only one part of the precision medicine equation, according to Dr. Resnick, co-director and co-founder of CHOP’s new Center for Data-Driven Discovery in Biomedicine (D3B).

“Even to make a new T-cell therapy, you have to begin with data about what to target,” Dr. Resnick said. “The other side of the coin in harnessing the potential of precision medicine is empowering the pediatric community to share and use data transparently and collaboratively through initiatives that connect patients, clinicians, and researchers to that data.”

Proton Therapy Neuropsychology Study Receives Dissertation Award

Originally published on Cornerstone, the CHOP Research Blog.

I composed this original article based on interviews with the investigators.

Excerpt:

Babies being treated for brain cancer have not received traditional radiation therapy since the 1980s. At that time, doctors realized that the side effects of radiation hitting healthy developing brain tissues in very young children was simply too severe. But within the past decade, proton therapy has become available to some of even these youngest patients. This newer radiation therapy method has a more targeted radiation beam that better concentrates its effect on the tumor while hitting fewer healthy tissues — but the nature of its effects on the developing brain are still being studied.

One such study, a doctoral dissertation project by a researcher at The Children’s Hospital of Philadelphia, was recently recognized for its quality of design and potential impact with the John E. Gordon Dissertation Award from the Philadelphia Neuropsychology Society (PNS) and Clinical Neuropsychology Associates.

Discovery Unites Three Families with Rare Syndrome

Originally published in the 2015 CHOP Research Annual Report.

I wrote this article based on a new interview with a parent and a past press release and blog post.

Excerpt:

When they met as a group for the first time, Leta, Liam, and Nadira seemed to bond instantly. All three are under 4 feet tall, have similar mannerisms, wide-set eyes, and bubbly personalities. They live with chronic respiratory problems and other medical challenges, in addition to cognitive and physical developmental delays.

“They look more like siblings than their actual blood siblings,” said Melissa Ashton-Grant, mother of Nadira. “To see how they interacted with each other, it felt right.”

The children’s similarity was no coincidence — and confirming and understanding their connection was no small matter. The three families gathered in March to celebrate because researchers at The Children’s Hospital of Philadelphia had discovered the rare genetic mutation their children share, and had given their cryptic constellation of symptoms a name: CHOPS Syndrome. CHOPS is an acronym that stands for Cognitive impairment and coarse facial features, Heart defects, Obesity, Pulmonary involvement, Short stature and skeletal dysplasia (abnormal bone development).

Beyond Imagining: Pediatric HIV Research Faces the Future

Originally published in the 2015 CHOP Research Annual Report.

I composed this original article based on multiple interviews and background literature research.

Excerpt:

The HIV epidemic in 2015 and beyond is a dramatically different one than ever seen or imagined during the height of the AIDS crisis in the 1980s and ‘90s.

“In the early days, up to a quarter of all infants born to women with HIV became infected. Now it’s less than one percent,” said Richard Rutstein, MD, an HIV clinical research leader and medical director of the Special Immunology Service at The Children’s Hospital of Philadelphia since its inception in 1989. “For those infected, HIV has changed from a rapidly fatal disease to a chronic illness.” Dr. Rutstein is also a professor of Pediatrics at the Perelman School of Medicine at the University of Pennsylvania.

At the front line of this evolution, CHOP researchers are helping infected pregnant women, infants, children, and youth around the world live full, productive lives.

Scientists Discover a Better ALK Inhibitor to Treat Neuroblastoma

Originally published on Cornerstone, the CHOP Research Blog.

I edited this article based on a CHOP press release and an additional interview with the investigator.

Excerpt:

Pediatric cancer researchers at The Children’s Hospital of Philadelphia believe they have succeeded in their search for a powerful next-generation drug for neuroblastoma tumors with mutations in the anaplastic lymphoma kinase (ALK) gene associated with the cancer. Based on their preclinical findings, they are fast-tracking the launch of a clinical trial this year.

Usually appearing as a solid tumor in the chest or abdomen, neuroblastoma accounts for a disproportionate share of cancer deaths in children, despite many recent improvements in therapy.

The search for better ALK inhibitors originated when, in 2008, CHOP pediatric oncologist Yael Mossé, MD, and colleagues identified ALK mutations as a driver of most cases of rare, inherited neuroblastoma. Subsequent research showed that abnormal ALK changes drive approximately 14 percent of high-risk forms of neuroblastoma.

Based on this knowledge, scientists including Dr. Mossé in the multicenter Children’s Oncology Group were able to repurpose crizotinib, an ALK inhibitor already approved to treat adults with lung cancer, in clinical trials of children with neuroblastoma. But they found that different mutations within the ALK gene in neuroblastoma responded differently to crizotinib, and a mutation labelled F1174L was resistant to the drug.