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INTERVIEW

Is Special FDA Regulation Of Nanomedicine Needed? A Conversation With Norris E. Alderson

Abstract

 
Cutting-edge research in nanomedicine dominates studies in drug delivery, medical imaging, and the development of new devices. Materials and devices the size of molecules, and even individual atoms, make it possible to see a tumor when it is no more than a few atoms in size. By using material in this size range, drugs can go directly to tumors or inflamed arteries, bypassing healthy tissue. In this interview Norris Alderson of the Food and Drug Administration discusses the present and future state of nanomedicine as it applies to health care, taking into consideration benefits, risks, and how much is still unknown.

Norris Alderson: Drugs and devices using a variety of types of nanoparticles are now in the pipeline. For example, everybody is very enthused about using nano materials as a delivery platform for cancer therapy. The properties of these small materials make it possible to do things we have not been able to do before, such as send a drug, packaged in a nanoparticle, directly to a tumor. That’s the only place it goes. It goes directly to that tumor. That has been a longtime goal that is finally being realized.

How Nanoparticles Target Disease

Top How Nanoparticles Target Disease FDA Regulation Of Nanomedicine Cost Consequences Ethical Questions

 
Culliton: How does this work? Is it the nano material that gets it to where it needs to go, or is it the drug itself that is so precisely targeted?

Alderson: A combination of the properties of the nano materials such as electrostatic charge, chemical, biological, and physical characteristics come into play as a result of the increased surface area of the nanoparticle. By calibrating those properties and the properties of the drug, you’re able to design a product that has unique and highly specific abilities.

Culliton: How do you get a drug—let’s say, a small molecule—into a nanoparticle delivery system? Do the properties of the chemotherapeutic drug have to be changed?

Alderson: The drug is attached to the nanoparticle; the mechanism really depends on the properties of the nanoparticle. We’re not talking about changing the property or size of the drug.

Culliton: Has the FDA approved any nano chemotherapeutics already?

Alderson: Yes, there are several. Abraxane is a good example. Abraxane was approved in 2005 for treatment of late-stage breast cancer in women who have failed other therapy.

Culliton: Abraxane uses a protein-bound nanoparticle system to deliver a familiar breast cancer agent, Taxol. Other nano systems are also in use for the delivery of anticancer agents. As you noted earlier, each is intended to deliver the drug to the tumor with high specificity. What about other diseases or conditions?

Alderson: Nanoparticles are in use these days in a number of areas. Some are designed to help repair bone; others are used as tooth fillers; there is even a bandage impregnated with nanosize silver. In addition, nanoparticles may very well play an important role in new imaging technologies that would allow the detection of an incipient tumor of just a few cells.

Culliton: How do you know that a product contains nanoparticles?

Alderson: Manufacturers tell us if material in the product is in the nano range. Our interest is whether it’s safe and effective.

FDA Regulation Of Nanomedicine

Top How Nanoparticles Target Disease FDA Regulation Of Nanomedicine Cost Consequences Ethical Questions

 
Culliton: Your comment implies that nanomedicine does not present the FDA with any new regulatory challenges. Are drugs or devices with nanoparticles evaluated under the same regulatory framework as other products?

Alderson: Very good question, and one our current commissioner asked us. Back in 2006, Commissioner [Andrew] von Eschenbach asked senior staff members to assess our readiness to deal with nano-engineered materials in the products we regulate. So you’re scanning all the way from drugs, biologics, devices, food, food additives, nutritional supplements, cosmetics—the whole gamut has the potential for nano-engineered material to be incorporated into it. The commissioner wanted to be certain there is a predictable pathway for evaluating these products before they get to the marketplace and are used by the American consumer. So, he appointed a task force, which I cochaired.

We reached a number of conclusions. Perhaps the most important is that, for now, this technology is really no different than any other new technology that would be incorporated into FDA products. So with that in mind, we feel comfortable, if you will, using our present regulatory framework. However, we felt there is need for guidance to help this industry as it moves forward. We recognized a need for additional information in various areas, such as biosafety. FDA and other agencies are working together on that. But for now, we just do not see the need for regulations written specifically for nano-engineered materials in the products FDA regulates.

Culliton: What about the fact that nanoparticles are fundamentally different from larger-scale molecules? Some researchers worry that there may be hazards as well as benefits.

Alderson: That was an important consideration for the task force. While on the one hand saying that we do not see the need for a new regulation, we also recognize that because of these nanoparticles’ unique biological and physical properties, there is a lot we do not and cannot yet know. So until we know more or have better information about this, we are going to continue to use the same approach as we’ve used for other products to assess safety and efficacy. If science later tells us something different, we will respond. We’re ready. We’re paying close attention to this. For instance, if you nanosize a drug, you’re probably making that drug more available in terms of its biological interaction. So you might have to use less of it. That is something we would want to know.

Culliton: Is there any particular postmarketing program or anticipated program to follow patients who receive nano drugs or devices?

Alderson: No different than the regular products. The National Nanotechnology Initiative issued a report in February laying out the environmental, health, and safety concerns for nano-engineered materials and citing priority areas for research. One of these is human health. Another one is environmental health. Measurement is a big issue for nano-engineered materials because you’re talking about measuring materials that we’ve never had the need to measure before. That brings in a whole new technology in terms of instrumentation and measurement procedures

We have to be vigilant and patient. There are amazing things that we hear about every day regarding potential uses for nano-engineered materials. It’s unbelievable some of the things we hear about, not only in the medical community but for other consumer products as well.

Culliton: How are you collaborating with other federal agencies?

Alderson: Let me give you one very active example. We have established collaborations with National Cancer Institute [NCI] that will benefit FDA as a regulatory agency, and the firms, large and small, that wish to bring nanomedicine to the public. The NCI set up the Nanotechnology Characterization Laboratory in Frederick [Maryland]. That laboratory is evaluating—at no cost to the sponsor, I might add—nano materials that could be used in nanomedicine. FDA staff serve as advisers to that laboratory on the type of data that FDA would need to see for products that would be coming to FDA for cancer therapy, for instance. There is close interaction. It’s a novel approach. We’re bringing our expertise in terms of our data needs to show that a product is safe and effective. The NCI is providing the dollars and scientists to evaluate some of these basic nano materials to determine the biological, chemical, and physical properties that I talked about earlier.

Cost Consequences

Top How Nanoparticles Target Disease FDA Regulation Of Nanomedicine Cost Consequences Ethical Questions

 
Culliton: Given the specificity and potential range of nanomedicine, do you anticipate appreciable effects on the cost of medical care using these products?

Alderson: I think we’re all hopeful that nano materials will reduce costs of health care because of the products that it will bring that we have not seen before. But I think it’s just too early to really tell. Until we see some of these products make it to the marketplace, it’s all conjecture.

Ethical Questions

Top How Nanoparticles Target Disease FDA Regulation Of Nanomedicine Cost Consequences Ethical Questions

 
Culliton: Are you looking at any particular ethical questions that have to do with nanomedicine?

Alderson: To date, we have not been presented with any ethical issues. We did receive a citizens’ petition in 2004, and it presented a concern from their perspective, which was labeling of products that are made of nano-engineered materials. Our task force looked at labeling. At this point, we do not see the need to label these products in some special way if they don’t present any particular issues as a result of their nano design. If these products presented issues of safety and efficacy as a result of the nano material, it might be something we need to consider, but not yet.

The bottom line is that this is a very exciting area of medicine that may well bring about major changes in the treatment of disease. We want to be sure it is safe, and, at the same time, we do not want to create restrictive barriers to progress for no sound reason.

Editor's Notes

 
Lance Liotta (lliotta{at}gmu.edu) and Emanual Petricoin (epetricoin{at}gmu.edu) are codirectors of the Center for Applied Proteomics and Molecular Medicine and professors of life sciences in the Department of Molecular and Microbiology at George Mason University in Fairfax, Virginia. They are also founders of Theranostics Health, a biotechnology company in Rockville, Maryland, which is commercializing the protein array technology they developed. Barbara Culliton (bjculliton{at}verizon.net) is a contributing editor at Health Affairs.

The preparation and publication of this interview were supported by a grant to Health Affairs from the Pew Charitable Trusts.

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