If it looks like a drug and acts like a drug, then it's a drug, right?
Not necessarily. As medical technology reaches the nano-scale, inventors developing innovative treatments, and especially the investors backing them, are very carefully splitting hairs to define a new class of therapies that steer clear of protracted regulatory approval processes to deliver novel products to market faster.
Nanoparticles injected into the human body for the treatment of tumors depend on an electro-mechanical activation to deliver their therapeutic effect, and therefore can be classified as medical devices and not medications.
The patient will not see any difference.
A doctor injects a fluid through a hypodermic needle and then asks the patient to lie still as he or she is passed through an X-ray, an electromagnetic field, or in another case, is given a pinpoint laser blast deliver through an endoscope.
But for venture capitalists impatient with the 10-year development cycle for bio-pharma products, the fracture line between pharma and med-tech regulation suddenly is shining as brightly as a vein of gold leading to a mother lode of fast returns.
"The beauty of medical technology is the low cost to get to first-in-man [FIM]," said Thom Rasche with Earlybird Ventures (Hamburg, Germany), who added the investment required is "a couple million" rather than 10 times that amount for a biopharma project.
Christophe Douat of Matignon Investissement (Paris) said his firm dedicates a third of its funds to medical devices because of the sector's dynamics that promise sustained opportunities.
"Let's just say 'Baby Boomers' and not talk about an aging population," he said, adding that helps people understand better that markets for cancer therapies are going to be at least the double of what they have been in the past.
He also said there is a "wealth of technology that is emerging with nano-particles and nano-biotechnologies, with information systems, and with new materials such as polymers" that is leading a lot of innovation in the field.
Three companies with nano-mechanical tumor-killers — MagForce Nanotechnologies (Berlin), NanoBioTix (Paris) and Photo Dynamic Therapy (PDT; Vienna) — presented their progress and the financial backing needed to bring to market their respective products to a roomful of venture capitalists during the Investing in Innovation-Europe conference held in Paris last week.
The nano-particles from MagForce and PDT require activation by a special apparatus that is sold as part of a package to a hospital or clinic.
Meanwhile, the nano-particle developed by NanoBioTix requires a common X-ray for activation and consequently will "revolutionize radiotherapy," according to CEO Laurent Levy, as an estimated 50% to 60% of all cancer patients are treated at some point with a stream of high-energy gamma rays.
The particle is a non-drug agent with at its core an inert and inactive substance called Nbtxr3 coated with a material taken up by cancerous cells, thereby attracting a concentration of particles on the diseased cells while leaving healthy cells untouched.
Introducing a blast of gamma rays activates Nbtxr3 locally, setting off tumor necrosis by inducing a chain reaction where a reactive oxygen intermediate blocks the formation of free radicals and arrests the cytotoxic response of tumor cells.
The non-toxic composition of the non-drug agent and its localized reaction are expected to "dramatically reduce" adverse effects associated with radiotherapy.
Current studies of the effects of Nbtxr3 are conducted at one-third the dosage of conventional X-ray therapy levels, though Nanobiotix is experimenting with higher gamma dosages to demonstrate superiority to conventional radiotherpay results.
The discovery and early development that led to Nbtxr3 was carried out by Levy at the State University of New York at Buffalo.
Nanobiotix has set a FIM milestone for 1Q09, with a clinical proof-of-concept trial anticipated in 2010.
To accentuate the speed possible for agents not subject to pharmaceutical approvals, Levy said he projects approval for 2012 and an immediate market launch thereafter.
The five indications for Nbtxr — for prostate, breast, colon, lung and pancreatic cancers — cover the majority of the rapidly expanding radiotherapy market that Levy estimated at 5 million patients per year worldwide.
Levy is seeking €15 million ($23 million) for a financing round to be completed in 1Q09.
MagForce Nanotechnologies also attacks tumors from the inside out, by dusting them with rust and then zapping them.
Iron oxide coated with a non-toxic layer is directly injected into a tumor and the patient is positioned under the Magforce MFH-300, a magnetic field applicator with variable power settings and a software suite for planning and implementing the therapy.
Cranking up magnetic forces to cause the iron oxide particles to vibrate, generating heat that can be controlled between 41 to 70 degrees Celsius (105 to 158 degrees Fahrenheit).
On the lower end of the scale, cancerous cells are subjected to hyoperthermic effects, and as the temperature tops out, thermoablation occurs.
Now in Phase II clinical studies, treatment with 100 patients has shown that a single injection is good for six sessions, each of which lasts an hour under the magnetic field.
Emerging from discovery and development at the Humboldt University Hospital Charité (Berlin), MagForce is traded on the Frankfurt exchange and holds a 1150 million ($230 million) market capitalization.
CEO Dr. Uwe Maschek is conducting efficacy trials, though the CE-mark approval only requires a demonstration of product safety, and is opening 10% of the company's capital to fund those trials in parallel with setting up distribution and marketing partnerships.
He said the company intends to have direct sales in France and Germany, but will license the technology in the rest of the world.
Maschek said he expects a CE mark and the start of active marketing in 2Q10.
PDT proposes a "photosensitizer" is administered to a patient ahead of a photonic therapy session where the agents that accumulate in the tumor are then subjected to a programmed exposure of a laser, killing the abnormal cells activity.
Dependent on a locally administered laser exposure, photon therapy can be used to treat lung, colon and pancreatic cancers, with evidence-based applications from PDT for cancers of the esophagus and bile duct.
PDT's business model calls not only for specific equipment, but for PDT to serve as a direct provider offering consultation to clinicians through clinical advisers, as well as providing administrative support for both marketing and reimbursement.
PDT is privately held, with discovery and development supported in part by the Austrian government and the city of Vienna.
COO G nther Hofmann, who owns the company entirely with his wife, told investors the company is cash-positive and comfortable with its original €5 million ($7.7 million) funding.
Hofmann said he projects €828 million ($1.3 billion) in revenues by 2011, and he is actively seeking strategic partners to build the market mechanisms and upscale operations for worldwide roll-out.