But what really excites Zon, director of the Stem Cell research program at Boston Children’s Hospital, is the power of the chemical screening platform he and his colleagues used. Described last week in the journal Cell, it found a cocktail of three compounds that induced human muscle cells to grow—in just a matter of weeks. Zon believes it could fast-track drug discovery for multiple disorders. Full story »
From the category archives:
Researchers have struggled to find the right approach to regeneration. Cell transplants have been tried, but the cells don’t engraft well long term and haven’t shown efficacy. Gene therapy to spur regeneration has been tested in animals, but dosage is hard to control and there’s a risk of genes going where they shouldn’t, causing tumors and other problems. Protein drugs have been tried, but they have short half-lives, being degraded or eliminated by the body before they can do much good. They are also hard to target to the heart.
A more recent approach to cardiac regeneration is to stimulate the body itself—and, specifically, progenitor cells— to repair the heart from within. Full story »
At last month’s BioPharm America conference, what I originally thought would be a run-of-the-mill panel wound up being a frank discussion about regulatory and pricing challenges that pharma and biotech companies are facing today. I hadn’t realized these two challenges are intertwined so closely.
The regulatory and pricing paths for new drugs in the United States have become increasingly difficult to navigate. Due to outside policy pressures, the FDA is scrutinizing drugs more than in the past, requiring much more data. Even when a drug is approved, there is no guarantee that payers will cover its full cost, as they are starting to consider the drug’s overall value—improving quality of life and decreasing costs—along with its effectiveness.
Meanwhile, in many European single-payer countries, pharmaceutical companies are being told how to price their drugs before they are considered for approval by the regulatory agencies. The likely effect is less return on investment on new drugs, which could in turn decrease the pace of innovation.
There are many HSCs in the bone marrow, but getting them out in sufficient numbers is laborious—and for the donor, can be a painful process. Small numbers of HSCs circulate within the blood stream, but not nearly enough. And while umbilical cord blood from newborn babies may present a relatively rare but promising source for HSCs, a single cord generally contains fewer cells than are necessary.
And here’s the rub: The demand for HSCs is only going to increase. Once a last resort treatment for aggressive blood cancers, HSCTs are being used for a growing list of conditions, including some solid tumor cancers, non-malignant blood disorders and even a number of metabolic disorders.
So how do we get more blood stem cells? Several laboratories at Boston Children’s Hospital and Dana-Farber/Boston Children’s Cancer and Blood Disorders Center are approaching that question from different directions. But all are converging on the same end result: making more HSCs available for patients needing HSCTs. Full story »
Translational neuroscience research has seen a disappointing streak of failed clinical drug trials. While the need for therapeutics that target the nervous system is growing, recent results in diseases like Alzheimer’s and autism have disappointed, and many companies have begun to downsize their R&D investments. Prospects are glum for patients who need new therapies to help manage their disorders.
The frustration is that drug candidates that have shown promise in animal models have not demonstrated efficacy in humans. Mouse models are not proving to be sufficient surrogates for human neurologic disease. Human brains and brain cells are built and function differently, and many neurodevelopmental disorders—hard enough to diagnose in human children—don’t have identifiable behavioral counterparts in mice. As I hear over and over from scientists, there is no such thing as a mouse with autism.
A study, published in Cell Stem Cell this June and conducted by Clifford Woolf, MD, PhD, et al, is among the first to demonstrate the power of an alternative technique: modeling disease in neurons derived from induced pluripotent stem cells (iPS cells). Full story »
In the last decade, Velcade has been tested against a long list of other cancers, including melanomas, lymphomas, as well as prostate, lung and breast cancers. The results have been mixed, particularly for breast cancer.
But in the case of breast cancer, the uncertain outcomes may in part be because past trials looked in the wrong place. New research by Fabio Petrocca, MD, and Judy Lieberman, MD, PhD, in Boston Children’s Hospital’s Program in Cellular and Molecular Medicine, suggests that proteasome blockers like Velcade may indeed have a place in the breast oncologist’s armamentarium, but just for a particular aggressive kind of breast cancer called basal-like, triple-negative breast cancer (TNBC). Full story »
When James Mandell, MD, outgoing CEO of Boston Children’s Hospital, introduced keynote speaker Robert Langer, PhD, at the National Pediatric Innovation Summit + Awards, he shared one of Langer’s favorite quotes. “When scientific literature says something isn’t possible, you just have to create possibilities that don’t exist.”
Langer, the David H. Koch Institute Professor at the Massachusetts Institute of Technology (MIT) and the most cited engineer in history, walked the audience through the trials and tribulations he encountered in his four-decade career as an innovator.
When he finished his ScD in chemical engineering in 1974, Langer was heavily courted by the oil and gas industries, which aimed to leverage the knowledge of young chemical engineers to address the oil crisis. But that work didn’t appeal to him.
Instead, Langer was taken with the idea of teaching chemistry to underserved youth. Unfortunately, he could not secure a position with any of the 40-odd programs to which he applied.
Eventually, a colleague suggested to him that Judah Folkman, MD, a pioneering cancer researcher at Boston Children’s, sometimes hired “interesting people.” Langer took the bait and joined Folkman’s lab in the mid-1970s.
“I may have been the only engineer in the place. I learned so much because everyone’s backgrounds were so different,” he recalled. Full story »
The Human Genome Project’s push to completely sequence the human genome ran a tab of roughly $2.7 billion and required the efforts of 20 research centers around the world using rooms full of equipment.
But that was using technology from the 1990s to early-2000s. As by a panel of genomics experts from industry and academia pointed out at last week’s National Pediatric Innovation Summit + Awards, a scientist in a single laboratory today can sequence a genome for as little as $1,000, making sequencing almost a medical commodity.
Now what? How do we go about making clinical genomics an everyday thing? The discussion left the answer to that question—and the other questions it raises—unclear. While the panelists expressed excitement about what’s possible, they cited great uncertainty among doctors, scientists, patients, payers, companies and regulators about how to make clinical genomics work. Full story »
In Part 1 last week, Vector took a look at digital health apps, telemedicine, genomics, phenomics and new behavioral diagnostics as transformative trends in pediatrics. This week, we complete our list. These posts will also appear as an article in the fall issue of Children’s Hospitals Today magazine.
6. New pharma research and development (R&D) models
Academic medical centers have always worked with the pharmaceutical industry but never so closely as now. In the old model, industry drove therapeutic development. A company might fund an academic project or supply reagents, but the relationship generally ended with the project (and publication of a paper).
Now, with drug pipelines drying up and R&D costs rising, Big Pharma is under pressure to change. New industry-academia collaborations are forging creative partnerships, altering how both parties do business. The new models are allowing hospital researchers to do what they’ve never done before: take the lead in R&D. Full story »