The following people and organizations are on our list of 2015 Game Changers. They did extraordinary things last year, reshaping the way we live and work.
PRACTICE MAKES (CLOSE TO) PERFECT
Using a 3-D printer to create models of their patients’ body parts lets surgeons do a test run.
By Priyanka Dayal McCluskey
Surgery is delicate work. Anything doctors can do to practice their cuts before heading to the operating room may improve a patient’s chances of surviving and thriving. That’s why so many Boston surgeons are turning to an invention of the tech world — the 3-D printer — to help them prepare for operations. Doctors and engineers program the sophisticated machines to replicate body parts of specific patients. If a patient has a brain tumor, for example, that abnormality will be reproduced exactly in the printed model of the patient’s brain. Surgeons use these 3-D models to practice surgery, to touch and feel and cut, just as they will during the actual surgery. It gives them, and their patients, a bit more confidence before the day of surgery.
It’s not so different from the way athletes practice their skills — but, of course, the stakes are higher. “You play tennis, you play golf, the first thing you do is you take practice swings,” says Dr. Peter Weinstock, head of the simulation program at Boston Children’s Hospital . “You want to rehearse, you want to give your body a sense of what it will feel like and remove all surprises as best as you can.”
Children’s Hospital operates one of the leading in-house 3-D printing programs in the country. One printer, sitting in a small room in the basement, has produced more than 200 models since 2013. And two smaller printers recently arrived.
Surgeons generally request 3-D models for their most complex surgeries, but Weinstock is seeing his colleagues use models all the time. “Why would you not take a look?” he says. “Why would you go in blind?”
That sentiment is shared by some of the surgeons at Brigham and Women’s Hospital. They have used models to practice one of the most difficult surgeries of all — the face transplant. Brigham doctors have also turned to models to prepare for treating tumors in vital body parts like the head, neck, and spine. The 3-D models are developed using digital data from scans such as MRIs and CTs. “Seeing the exact 3-D nature of the tumor can help them better plan,” says Dr. Thomas C. Lee, assistant section head of neuroradiology at Brigham.
When surgeons plan better, they can also finish procedures more quickly, cutting the amount of time a sedated patient is lying on an operating table. “By reducing OR times, it could reduce the costs associated with it,” says Dr. Andrew R. Scott, an ear, nose, and throat surgeon at the Floating Hospital for Children at Tufts Medical Center.
Doctors agree 3-D printing holds great potential for medicine, not just in modeling but in making prosthetic body parts. Tufts, for example, is using 3-D printers to make prosthetic ears for some children born without them.
As a tool for surgery, it’s still an expensive technology that requires many hours and thousands of dollars to produce the most complex models. How to pay for that remains an open question. Still, 3-D printing technology appears to be more than a passing fad among Boston surgeons. “To an increasing degree,” Lee says, “3-D printing and 3-D modeling is going to revolutionize how we treat patients.”
New drugs and medical devices offered hope to those with heart disease, multiple sclerosis, and rare genetic disorders.
By Robert Weisman
The labs and entrepreneurs that make the Massachusetts life-sciences engine hum have become reliable sources of discoveries in everything from medicine to diagnostic tools. Last year was no exception, a time when new drugs and medical devices to treat multiple sclerosis, heart disease, and rare genetic disorders took important steps forward. “Whether it’s research and development in the underlying science, or the translation into products, we’re in an enviable place that the rest of the world seeks to emulate,” says Jonathan J. Fleming, president of the Cambridge-based Network for Excellence in Health Innovation.
Five local breakthroughs stood out last year for their potential to advance the state of care for a wide range of patients: a new heart pump, a powerful MS drug, an advance in cystic fibrosis treatment, the first new hemophilia drugs in nearly two decades, and support for a promising gene-silencing technology.
Perhaps the biggest development in the fight against coronary artery disease, the leading cause of death for men and women in the United States, was the bid by Abiomed of Danvers for full approval of its Impella 2.5 heart pump. The device has been used since 2008 under a Food and Drug Administration clearance that gave access to only a narrow set of patients. Ultimately, the FDA bestowed its coveted “pre-market approval” this March for the Abiomed device, which helps patients maintain stable heart function during high-risk cardiac procedures. The new FDA designation, based on Abiomed’s clinical data, certifies that the minimally invasive device — replacing older technology involving inflatable balloon pumps — is safe and effective. It opens up a larger market for patients during elective and urgent procedures.
“That’s the gold standard,” Abiomed chief executive Michael R. Minogue says of the FDA approval. “It’s a license to become the standard of care. It’s the world’s smallest heart pump, and it can go in and start pumping blood in a matter of minutes.”
Patients suffering from the neurological disease multiple sclerosis also got some good news late last year when the FDA reversed an earlier decision and allowed the US sale of Lemtrada, an MS drug that is key to the future of Cambridge biotech Genzyme .
Regulators acted after MS patients across the country called for more treatment options and Genzyme submitted a new analysis of clinical data to address concerns about the design of trials to test the drug’s safety and effectiveness. Lemtrada is already on the market in Europe and elsewhere, but its use in the United States is reserved for patients who have tried at least two other MS drugs yet still suffer relapses.
People using the injectable drug in clinical trials suffered fewer flare-ups, and about 70 percent were able to stop taking the medication after two treatment courses. “The [patient] community made a statement by saying, ‘We need a drug like Lemtrada to be available,’ ” says Michael Panzara, therapeutic area head for Genzyme’s MS and neurological research program. “This is a product that’s doing something to the [disease] that is really durable. It’s having significant effects on disability, relapses, and inflammation in the brain.”
Meanwhile, clinical tests conducted last year by Vertex Pharmaceuticals showed a combination of two of its drugs helped people with cystic fibrosis breathe more easily. That raised hopes the two-drug combination treatment could reach the market as soon as mid-2015 and help many more people suffering from the disease. The Boston company’s study involved an experimental regimen that can treat nearly half of those afflicted with the life-threatening genetic disease — a big advance from the 4 percent able to take Kalydeco, the Vertex drug already on the market. In the clinical trials, Kalydeco was paired with an experimental drug.
Biogen of Cambridge scored a double coup in 2014 when the FDA OK’d a pair of drugs as the first significant new treatments for hemophilia in 17 years. The long-acting drugs — Eloctate for hemophilia A, Alprolix for hemophilia B — treat different forms of the inherited disorder that causes recurrent bleeding, pain, and joint damage. The two new drugs can reduce the frequency of injections for hemophilia patients. For Biogen, the largest Massachusetts-based biotech, they represent the start of a diversification beyond its portfolio of treatments for MS. Biogen is also working on an Alzheimer’s therapy, which slowed the progression of the disease in an early-stage clinical trial last year.
Alnylam Pharmaceuticals gained important momentum last year for its promising technology known as RNA interference, which fights diseases by silencing genes that cause the unwanted overproduction of proteins in cells. Genzyme, its neighbor in Cambridge’s Kendall Square, paid $700 million to buy a chunk of Alnylam and tap its pipeline of rare-disease drug candidates. It also got broad marketing rights to Alnylam’s portfolio of experimental drugs deploying RNAi against diseases such as hemophilia. Alnylam still does not have any approved drugs on the market. But the Genzyme investment was a vote of confidence that the company’s technology can lead to treatments for rare genetic diseases.
IMAGINING A NEW KIND OF CANCER FIGHTER
Among the projects brewing at Google’s hush-hush GoogleX skunkworks division: a magnetic nanoparticle that researchers hope will be able to roam the bloodstream hunting for early signs of cancer. Entrega Bio of Boston is collaborating on the project revealed last October, trying to figure out how to get those nanoparticles into the body.
Entrega is developing a patch that can be packed into a pill. The patch then sticks to the lining of the patient’s small intestine for a set period of time, allowing a substance like Google’s nanoparticles to be absorbed. The Google project is “going really well,” says Entrega cofounder Robert Langer, a professor at MIT.
Entrega signed on David Lucchino as its chief executive last year.
A BIKE RIDE A DAY KEEPS THE DOCTOR AWAY
At Boston Medical Center, doctors write prescriptions for medications, groceries, and, now, bicycles. The hospital and the City of Boston launched a “Prescribe a Bike” program last spring, offering low-income patients a $5 a year pass to access the Hubway bike-sharing system. BMC says doctors can prescribe inexpensive exercise to patients with diabetes, high cholesterol, heart disease, and other chronic conditions. Patients are allowed an unlimited number of rides of up to 60 minutes.
“This is a very innovative and cost-effective way to help our patients get exercise,” says BMC’s Dr. Alan Meyers, who spearheaded the program.
BMC has pioneered the idea of unorthodox prescriptions. Similarly, doctors also give prescriptions for fruits, vegetables, and other healthy foods that patients pick up at a food pantry twice a month.
MAKING DENTIST VISITS EASIER
If you’ve been avoiding the dentist out of fear of the drill, you might be out of excuses. Convergent Dental’s Solea laser offers an alternative to drilling in basic procedures, like filling cavities, shaving teeth to be fitted for crowns, and slicing polyps off gums.
The Natick company’s laser, which went on the market last year, works by vaporizing the mineral that makes up 90 percent of tooth enamel. Its rapid light pulses have a numbing effect that’s usually strong enough to replace Novocain. “The plan is to get rid of the drill, the scalpel, the needle, the noise, and the vibration,” says Convergent chief executive Mike Cataldo.
Convergent’s business got off to good start. Its $100,000 Solea system was purchased by more than 100 dental practices nationwide last year.
PERSONALIZED CANCER VACCINE
Dr. David Avigan of Beth Israel Deaconess Medical Center has spent 15 years working on a personalized vaccine to treat cancer patients. Now he is preparing to lead a unique “open source” clinical trial of the treatment to be conducted at a dozen cancer centers around the country.
The therapy, which uses patients’ immune systems to fight cancer, was developed in collaboration with the Dana-Farber Cancer Institute. Last year, a group funded by the National Institutes of Health agreed to sponsor the upcoming trial to treat nearly 200 patients with the blood cancer myeloma.
Beth Israel recently hosted researchers from around the country, teaching them how to make the personalized vaccine. Avigan chose to pursue the open source method to help speed the final phases of clinical testing for the therapy that showed promise in preliminary trials with patients suffering from myeloma and leukemia.
Capsules written by Steven Syre, Scott Kirsner, Taryn Luna, and Callum Borchers. Send comments to firstname.lastname@example.org.