Hoping to create more effective cancer medications with fewer side effects, two Cambridge start-up companies are beginning to test therapies that rely on particles about the size of viruses to ferry potent drugs directly to tumor cells.
Cerulean Pharma Inc. has launched clinical trials of tiny Trojan horse-like particles aimed at lung, ovarian, and kidney tumors, and BIND Biosciences Inc. is evaluating targeted, drug-laden nanoparticles in an early-stage study of patients with various advanced cancers.
The efforts are part of a broader movement in medicine, where drug development has been merging with engineering at the smallest scale to enable “nanomedicine” that can better target and deliver medications, or detect disease. The techniques hold particular promise for cancer, where therapies are sorely needed that can target cancer cells and spare normal ones. The National Cancer Institute funds about $130 million in nanotechnology research each year.
“There is growing interest in delivering drugs that are too toxic and failed in clinical trials, and packaging them in a nanoparticle,” said Piotr Grodzinski, director of the National Cancer Institute office of cancer nanotechnology research. “You can . . . benefit from their potency, but enable safe delivery.”
The two nanoparticle drugs being developed locally are given intravenously like conventional chemotherapy, but they are believed to be less toxic to normal cells and more effective against cancer because they are designed to aggregate and be released at tumor cells.
Cerulean, which uses a nanoparticle attached to an extremely toxic chemotherapy agent called camptothecin, announced last week it had enrolled 150 patients in a randomized study of its drug in patients with nonsmall cell lung cancer, and expects data to be ready for analysis at the end of the year. The company has also recently treated its first kidney cancer patient with a combination of its experimental treatment and the drug Avastin. It expects this month to administer the drug to an ovarian cancer patient in a trial at Massachusetts General Hospital.
Cerulean says it has found in early trials that its method allows the delivery of a highly toxic drug without most of the side effects associated with standard treatment. That opens up the possibility of not only using a drug with severe side effects, but also allowing researchers to combine it with other drugs.
“I think where this technology excites us the most is in terms of these combinations, which heretofore might not have been thought possible because of the cumulative toxicity,” said Dr. Edward Garmey, chief medical officer of Cerulean. He said reducing the harmful side effects of medications would be essential if cancer care is to evolve — as many expect it will — into “cocktails,” or combinations of drugs, intended to keep a chronic disease in check.
He said that the company, which has 32 employees and raised slightly more than $70 million during the last five years, is planning further trials in gastric cancer and small cell lung cancer. It is also doing preclinical research on a technique for delivering short segments of RNA directly to tumor cells, a therapy that can be used to turn off particular errant genes.
Meanwhile, in April, BIND Biosciences reported results in the journal Science Translational Medicine from an early trial in patients with advanced cancer. The company, using technology developed in the laboratory of bioengineer Robert Langer at the Massachusetts Institute of Technology, crafted a nanoparticle carrying the drug docetaxel, capable of homing in on cancer cells by identifying a landmark on the surface of a number of cancer cells and the blood vessels that feed tumors.
Such techniques are similar in their general concept to a class of drug that garnered widespread attention at the meeting of the American Society of Clinical Oncology last month. That type of drug, called an antibody-drug conjugate, consists of an antibody that binds to a cancer cell, linked to molecules of a specific drug.
But Scott Minick, chief executive of BIND Biosciences, said his company’s approach will allow flexibility to deliver a wide array of drugs because the therapeutic nanoparticle can be engineered to carry thousands of molecules of a drug.
The company, which has 35 employees and has raised about $90 million, plans to launch a larger cancer trial later this year, although it has not disclosed a specific tumor type. But it also sees other potential applications for the technology, in treating inflammatory disease and pain.