CHOP forms first startup firm to develop treatments for peripheral artery disease

Building on its extensive laboratory research using magnetically guided nanoparticles to deliver drugs to diseased blood vessels, The Children's Hospital of Philadelphia has just spun off its first startup company, Vascular Magnetics, Inc. (VMI).

By licensing its technology to VMI, a new company formed to develop the lab findings into a commercially viable therapy, the Hospital aims to create a novel, greatly needed treatment for peripheral artery disease (PAD). Characterized by blocked arteries, primarily in the legs, PAD affects more than 27 million older adults in North America and Europe, with diabetes patients and smokers at particularly high risk. Current treatments for PAD, including drug-eluting stents, are ineffective, with re-blockage of the arteries occurring at a high rate.

In addition to being the Hospital's first startup company, the program targets patients outside the Hospital's usual pediatric age group. "While our first target group is adult patients, the technique represents a new platform technology, potentially adaptable to delivering a variety of therapies to children as well as adults," says the technology's inventor, Robert J. Levy, M.D., the William J. Rashkind Endowed Chair in Pediatric Cardiology at The Children's Hospital of Philadelphia.

Levy co-founded VMI with Richard S. Woodward, Ph.D., the company's Chief Executive Officer. The two first joined forces through the QED Proof-of-Concept Program sponsored by the University City Science Center in West Philadelphia. The goal of the QED Program, the nation's first multi-institutional proof-of-concept program for life sciences technologies, is to accelerate research from academic laboratories into the marketplace. A unique feature of this program is that academic scientists such as Levy are partnered with experienced business advisors such as Woodward, who has a background that includes developing nanoparticles and polymeric coatings.

"The Science Center is proud to have played a role in the launch of Vascular Magnetics," said Stephen S. Tang, Ph.D., MBA, president and CEO of the University City Science Center. "VMI's launch is helping to prove our concept that the early addition of business advice is a key element of the tech transfer puzzle."

In a series of animal studies over the past decade, Levy and his team at Children's Hospital have investigated his new approach to stent-based therapy. They have developed nanoparticles, extremely tiny spheres made of a biodegradable polymer impregnated with iron oxide. Under a low-power, uniform magnetic field, much lower than that produced by existing MRI machines, magnetic forces drive the nanoparticles into metal stents and the surrounding artery. The nanoparticles carry a therapeutic payload of the drug paclitaxel, which is released into the surrounding blood vessel tissue in order to slow arterial re-blockage. In 2008, Forbes magazine named Levy's work a promising "disruptive technology," one that might eventually supplant conventional technology, in this case, drug-eluting stents.

As they advance the technique to human trials, Levy and Woodward envision a future therapy called Vascular Magnetic InterventionTM which would serve as an adjunct to artery stenting. A physician would open and stent the blocked artery and then insert a catheter tipped with an expandable magnetic targeting device. The targeting device would be expanded against the walls of the artery.

A magnetic field is then applied to the leg, and paclitaxel-containing magnetic nanoparticles would be administered through the catheter. The targeting device develops strong magnetic gradients that force the nanoparticles into the wall of the artery. After treatment and removal of the catheter, the wall of the artery is uniformly coated with the nanoparticles, which slowly biodegrade and release the drug. The uniform coating provides a higher dose of drug than is achievable with a drug-eluting stent. In addition, the technique could be used to re-treat arteries where the stents have become re-blocked.

The technology, said Levy, is highly adaptable. Instead of paclitaxel, it could deliver other therapeutic compounds, DNA for site-specific gene therapy, therapeutic cells, or other treatments. In addition to treating PAD, the technique might carry paclitaxel to narrowed coronary arteries, chemotherapy drugs to a tumor, or other medications to a bile duct or a urinary tract. Eventually, said Levy, the technology could be applied to types of pediatric heart disease, such as primary pulmonary hypertension or heart defects.

As its lab research continues, Vascular Magnetics is moving ahead to attract venture capital. "Our plan is to prove the efficacy of this therapy in humans by late 2015," said Woodward. "The revenue projections for the company suggest sales of over a billion dollars per year within about four years after commercial launch."

"Besides the great potential medical benefit in PAD, commercial success for this technology could enhance our mission of improving children's health-by setting the stage for developing and investing in innovative therapies for children," added Ellen Purpus, Ph.D., director, Office of Technology Transfer at The Children's Hospital of Philadelphia.

Source: Children's Hospital of Philadelphia