Even at a place like Boston Children’s Hospital, where doctors regularly see children with rare diseases from all over the world, there are big challenges when it comes to drug discovery and treatment.
“Roughly 70 percent of drugs to treat children are used off-label,” says David Williams, Boston Children’s chief scientific officer. “That’s because these drugs were initially developed for adults and have not been tested formally in children.”
In order to cure rare diseases in children and adults, scientists must bridge the gap between research and industry. On May 25, Boston Children’s Technology and Innovation Development Office (TIDO) and MassBio held a candid panel discussion about what it will take to advance the development of rare disease therapies. Here are three of the biggest takeaways …
When Boston Children’s Hospital decided to hire its first chief scientific officer (CSO) in eight years, the institution sought an individual who could spotlight the hospital’s robust scientific enterprise and effectively connect it to clinical medicine and industry. David Williams, MD, president of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and director of clinical and translational research at Boston Children’s, was the ideal choice.
An award-winning researcher, Williams trained in the clinic but also pursued basic science, developing techniques for introducing genes into mouse and human blood cells. He focused on blood stem cell biology, leukemia and gene therapy to correct genetic blood disorders, becoming a 16-year Howard Hughes Medical Institute Investigator, a Member of the National Academy of Medicine and a Fellow of the American Association for the Advancement of Science. He has secured multiple patents for techniques still in use today.
Williams spoke about his vision as CSO to align basic research and clinical care at Boston Children’s and the challenges ahead. …
The twists and turns of Stephen Friend’s career are both dizzying and thrilling. In the early days, Stephen Friend, MD, PhD, CEO and co-founder of Sage Bionetworks, spent many a late night as a resident in the emergency room at Children’s Hospital of Philadelphia with Gary Fleischer, MD, current pediatrician-in-chief at Boston Children’s Hospital.
Friend later wound up at Boston Children’s as well, where he did his pediatric hematology-oncology fellowship and later, as part of the faculty, helped co-lead the team that identified the first tumor suppressor at Boston Children’s. A few years later, Friend left academia to pursue his passion in a startup and later engineered a landing at Sage Bionetworks, a nonprofit focused on patient engagement and open science in the research process. The Resilience Project, one of Sage’s research initiatives, analyzes DNA from healthy volunteers to discover rare mutations that protect resilient people from serious childhood illnesses.
Academic researchers and physician innovators are great at making research discoveries and developing inventions at an early stage. But if you were to fund them to turn their research findings into a product, would they have the expertise and experience needed to be successful? Most would not.
The investment community talks about the innovation funding gap, a.k.a. the “valley of death.” But there is also a knowledge gap on the part of academic researchers when it comes to transforming their technologies into therapeutics. Most want their findings to lead to new treatments for patients, but they lack the experience and expertise that companies have to advance early-stage research to a clinical stage. That includes expertise in designing pre-clinical experiments and navigating regulatory pathways for commercial development.
Academics often enter agreements with pharmaceutical companies, many of which are early-stage research grants. Often, these industry-sponsored research projects end with a scientific publication and are unsuccessful in generating new therapeutics—a subpar outcome for the company investor. …
Successful therapeutic development requires multiple stakeholders along the path from discovery to translation to clinical trials to FDA approval to market availability. At various points along this path, academia, industry, government, hospitals, nonprofits and philanthropists may work together. Would bringing these stakeholders together from start to finish lead to greater success?
A growing number of private-public consortia are launching in defined “pre-competitive” spaces where potential rivals collaborate to generate tools and data to accelerate biomedical research. In 1995, consortia were rare in health care: Only one was created. In 2012, 51 new consortia were launched, according to the organization Faster Cures.
Why? you may ask. Banding together in consortia can reduce costs, minimize failures and shorten the timeline to approval for new drugs. …
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. …
Genomic sequencing and molecular diagnostics are becoming a global business. At the recent American Society of Human Genetics meeting, dazzling technologies for reading genetic code were on display—promising faster, cheaper, sleeker.
Nevertheless, it’s become clear that the ability to determine someone’s DNA or RNA sequence doesn’t automatically translate into useful diagnostics or even actionable information. In fact, the findings are often confusing and hard to interpret, even by physicians.
That’s where academic-industry partnerships can flourish—tapping the deep expertise of medical research centers to bring clinical meaning to sequencing findings. Yesterday, Boston Children’s Hospital and Life Technologies Corp. announced a new venture with a great list of ingredients: fast, accurate, scalable sequencing technology—Life’s Ion Proton® Sequencer—but also research and clinical experience in rare and genetic diseases, bioinformatics expertise to handle the big data, and the medical and counseling expertise to create meaning from the results. …
In recent years, creative new partnerships have demonstrated big pharma’s recognition that academic medical centers hold many important cards in clinical research: scientific expertise, animal models of disease, patient samples and phenotypic data.
Increasingly, these partnerships involve academic and company researchers developing joint grant proposals in targeted areas, selected (by joint agreement) for company sponsorship. Some, like the Immune Disease Institute’s $25M arrangement with GlaxoSmithKline, are specific to one academic institution; others, like Pfizer’s Centers for Therapeutic Innovation (CTI) program, provide the same resources under the same deal structure to multiple institutions. Each new deal advances the interaction and understanding between academia and pharma around the common goal of finding new compounds and bringing them to clinic.
Now, in an exciting twist on its track record of partnerships with academic institutions, Roche has brought together three Harvard-affiliated organizations to screen and identify new drugs for the treatment of autism spectrum disorders (ASDs). …