It began with the proteins. Before Watson and Crick unraveled DNA’s double helix in the 1950s, biochemists snipped, ground and pulverized animal tissues to extract and study proteins, the workhorses of the body.
Then, in 1990, the Human Genome Project launched. It promised to uncover the underpinnings of all human biology and the keys to treating disease. Funding for DNA and RNA tools and studies skyrocketed. Meanwhile, protein science fell behind.
While genomics unveiled a wealth of information, including the identity of genes that lead to disease when mutated, researchers still do not fully understand what all the genes really do and how mutations change their function and cause disease.
Now proteins are promising to provide the missing link. …
Since 2009, Boston Children’s Hospital has committed $6.2 million to support 58 hospital innovations ranging from therapeutics, diagnostics, medical devices and vaccines to regenerative medicine and healthcare IT projects. What a difference six years makes.
The Technology Development Fund (TDF) was proposed to Boston Children’s senior leadership in 2008 after months of research. As a catalyst fund, the TDF is designed to transform seed-stage academic technologies at the hospital into independently validated, later-stage, high-impact opportunities sought by licensees and investors. In addition to funds, investigators get access to mentors, product development experts and technical support through a network of contract research organizations and development partners. TDF also provides assistance with strategic planning, intellectual property protection, regulatory requirements and business models.
Seeking some “metrics of success” beyond licensing numbers and royalties (which can come a decade or so after a license), I asked recipients of past TDF awards to report back any successes that owed at least in part to data generated with TDF funds. While we expected some of the results, we would have never anticipated such a large impact. …
Today we bring more good news: Following a successful Phase III trial, rFIXFc recently received the green light for marketing from the FDA and from Health Canada.
Developed by Biogen Idec under the trade name Alprolix™, rFIXFc—a modified version of clotting factor IX—is the fruition of a technology first envisioned by three researchers—gastroenterologists Wayne Lencer, MD, of Boston Children’s Hospital, and Richard Blumberg, MD, of Brigham and Women’s Hospital, and immunologist Neil Simister, DPhil, of Brandeis University—for large protein drugs. Their idea: to extend the drugs’ half-lives by protecting them from being ground up by cells. …
Getting drugs to stay in the bloodstream longer is a big deal when it comes to treating chronic diseases. You see, a drug’s half-life—the time it takes for half of a given dose to be cleared from the body—determines how long its effect(s) last.
If a drug’s half-life is short—meaning it’s cleared quickly—patients will have to take the drug frequently. Given that someone with a chronic condition could be on the medication for many years—say, patients with severe hemophilia, who endure frequent infusions of clotting factors—a short half-life can translate into high cost. Depending on side effects and how the drug is administered, quality of life may also suffer.
Your immune system’s B cells can produce antibodies against an amazing number of pathogens—viruses, bacteria, etc.—without ever having encountered them. That’s because, as they develop, your B cells reshuffle their antibody-producing genes into an amazing number of possible combinations—more than 100 million—to produce what’s called your primary pre-immune B cell repertoire.
It’s long been thought that in people and in mice this reshuffling process—called V(D)J recombination, after the B cells’ antibody-coding V, D and J gene segments—takes place in two places: the bone marrow and the spleen. But new research from a team led by Frederick Alt, PhD, and Duane Wesemann, MD, PhD, suggests that there may be one more place B cells go to undergo recombination: the gut. What’s more, that reshuffling in the gut may be influenced by the microbes that live there.
Imagine for a moment, that you are your immune system. On any given day, you’re faced with host of threats: a virus here, a bacterium there, a new fungus. And don’t forget those wayward cells lurking around the corner, the ones that might become a tumor.
Now, you have to respond to these challenges, but how you do it? Each looks different, meaning that you have to produce a new T or B cell (your two main tools) that can find, mark and guide the attack against each new threat.
Luckily, your T and B cells can turn to three sets of gene segments that, together, contain the genetic raw material for the variety you need. Called the variable (V), diversity (D) and joining (J) segments, they are constantly cut up, shuffled and rejoined by the genome to make new genes – a process called V(D)J recombination – for new receptors (on T cells) or antibodies (from B cells), giving your immune system the most diverse arsenal possible. …
Every year, the flu tries to outwit humanity. By shifting parts of its outer coat, the virus renders the flu vaccine from the previous year obsolete, bringing another season of misery. And every year, we fight back with a new vaccine, finding a new chink in the virus’s armor and giving ourselves another brief window of protection.
But if Stephen Harrison, chief of Children’s Division of Molecular Medicine, is right, we might be able to train our immune systems to look past the flu virus’s annual trickery and build up resistance that spans multiple seasons. That could reduce the need to develop, produce, and distribute a new flu vaccine nearly every year, a process of selection, growth, packaging, and distribution that can take upwards of seven months. …