Stories about: oncology

Putting patients first in the translational research pipeline

During a follow-up visit, pediatric hematologist/oncologist Sung-Yun Pai, MD, hugs a patient who received gene therapy for X-linked severe combined immunodeficiency.
During a follow-up visit at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, pediatric hematologist/oncologist Sung-Yun Pai, MD, hugs a patient who received gene therapy for X-linked severe combined immunodeficiency.

This is part II of a two-part blog series recapping the 2018 BIO International Convention. Read part I: Forecasting the convergence of artificial intelligence and precision medicine.

The hope to improve people’s lives is what drives many members of industry and academia to bring new products and therapies to market. At the BIO International Convention last week in Boston, there was lots of discussion about how translational science intersects with patients’ needs and why the best therapeutic developmental pipelines are consistently putting patients first.

As a case in point, Mustafa Sahin, MD, PhD, of Boston Children’s discussed his work to improve testing and translation of new therapies for autism spectrum disorder (ASD). As a member of PACT (Preclinical Autism Consortium for Therapeutics) and director of Boston Children’s Translational Neuroscience Program, Sahin aims to bridge the gap between drug discovery and clinical translation.

“Our mission is to de-risk entry of new therapies in the ASD drug discovery and development space,” said Sahin, who is also a professor of neurology at Harvard Medical School.

One big challenge, says Sahin, is knowing how well — or how poorly — autism therapies are actually affecting people with ASD. Externally, ASD is recognized by its core symptoms of repetitive behaviors and social deficits.

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Meet Huggable, the robotic teddy bear designed for sick kids

Nobody likes being confined to a hospital bed. Children especially can feel lonely, bored or scared in these situations. Hours feel like days, and they may not be able to fully understand or describe why they are there.

Child life specialists have long understood that tapping into playtime can bring up information about a child’s social and emotional needs that might not be revealed in more structured clinical assessments. But what if you cannot physically be in the room?

Deirdre Logan, PhD, Director of Psychology Services in Pain Medicine, and Peter Weinstock, MD, PhD, Director of the Boston Children’s Simulator Program (SIMPeds), may have found the answer. Along with a dedicated, multidisciplinary research team from Boston Children’s, MIT’s Media Lab, and Northeastern University, they have designed a robotic teddy bear that may be able to supplement care team interactions on inpatient units.

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David Nathan, MD, 85, receives Boston Children’s Lifetime Impact Award

David Nathan Hematology Oncology Lifetime ImpactPeers describe David G. Nathan, MD, president emeritus of Dana-Farber Cancer Institute and physician-in-chief emeritus of Boston Children’s Hospital, as a “a once-in-a-generation leader,” a “giant” and a “proverbial triple threat” combining clinical care, research and teaching leadership.

Nathan, whose commitment to pediatric medicine spans nearly six decades, received a standing ovation when presented with Boston Children’s inaugural Lifetime Impact Award Friday afternoon at the hospital’s Global Pediatric Innovation Summit + Awards.

The Lifetime Impact Award recognizes a clinician and/or researcher who has devoted his or her career to accelerating innovation in pediatric medicine and who has made extraordinary and sustained leadership contributions.

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Treatment abandonment in childhood cancer: Are we willing to face this challenge?

(Photo: The Advocacy Project/Flickr)

Though the diagnosis is overwhelming for patients and families to receive, many childhood cancers have become “curable diseases.” At major U.S. centers like mine, the Dana Farber/Children’s Hospital Cancer Center, research efforts now largely focus on survivorship, refining risk stratification, minimizing treatment toxicity and developing more effective salvage therapies upon relapse.

But the situation globally is quite different. The technologic and resource gap between our centers and centers in the developing world is widening. Only a fraction of the children diagnosed with cancer around the world have access to therapy, either curative or palliative.

Treatment abandonment is another significant barrier to cancer care in the developing world. The reasons aren’t only economic, but are complex and multifactorial, including limited education, fatalism surrounding a cancer diagnosis, magical thinking, mistrust of the health care system,

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Smart cancer drugs need smart clinical trials

Smart drug drawingPediatricians are accustomed to caring for patients with rare diseases. But as all physicians know, common diseases can also behave in rare ways, either presenting in unusual forms or responding only to particular treatments. Recent advances in molecular medicine have confirmed this intuition, particularly in cancer, whose varieties can be virtually as unique as we are ourselves.

Oncologists have formed cooperative groups to meet this challenge long ago. But they remain outmatched by cancer itself: its biology is complex, with different tumors growing as a result of hundreds to thousands of aberrations in cancer genomes and cells. And, until recently, they’ve been limited by the process of traditional clinical trials.

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The latest twists in angiogenesis research

Medulloblastoma cells (green) are growing around this cerebellar blood vessel and inducing growth of new vessels nearby (red). Courtesy Matija Snuderl & Rakesh Jain.

An update last week on angiogenesis research revealed surprising twists in the story of fighting cancer by cutting off the tumor’s blood supply. The latest findings, reported by top researchers at an international pediatric oncology meeting in Boston, show that the story is much more nuanced. If big questions seem to go begging in the emerging story, you’re not alone in thinking so!

Anti-angiogenic drugs can kill some tumors by cutting off their blood supply. But surprisingly, in two animal studies using very high doses, the drugs turned some tumors more malignant and metastatic, said Rakesh Jain, PhD, by increasing the hypoxic zone; low oxygen conditions somehow promote tumor progression in these models.

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