Treating Children with Cancer

September is National Childhood Cancer Awareness Month. Watch the below video in which two FDA experts discuss existing and new efforts to encourage the development of medicines for kids with cancer.

The conversation is between Robert “Skip” Nelson, M.D., Ph.D., deputy director and senior pediatric ethicist in FDA’s Office of Pediatric Therapeutics, and Gregory Reaman, M.D., associate director of the Office of Hematology and Oncology Products.

It begins with a discussion of FDA’s role in evaluating medications used to treat children with cancer and what measures are underway to encourage further development of these important drugs.

For More Information

Pediatrics

New Pediatric Labeling Information Database

Cancer Liaison Program

Office of Hematology and Oncology Products

Friends of Cancer Research Honors Our Dr. Richard Pazdur

By: Margaret Hamburg, M.D.

One of the privileges of being the head of the Food and Drug Administration is being able to report on the accomplishments of the dedicated and highly skilled employees who are advancing the public health mission of our agency. Last night, I had the pleasure of participating in an event honoring one of our scientists for work that makes us particularly proud: his outstanding contribution to the fight against cancer.

Margaret Hamburg, M.D. and Richard Pazdur, M.D.Dr. Richard Pazdur, the director of our Office of Hematology and Oncology Products, joined Senator John McCain and Neera Tanden, president of the Center for American Progress, as a recipient of the Cancer Leadership Award from the Friends of Cancer Research, an important organization that helps advance medical progress.

Senator McCain and Ms. Tandem have been tireless advocates for public investment in cancer research, getting cancer drugs to market more quickly, and developing prevention strategies that are known to reduce the risk of cancer. Dr. Pazdur, who was honored for his leadership in cancer research, makes sure that the drugs used to treat cancer patients are not only available for patients who need them as fast as science will allow, but above all, that they are safe and effective.

Achieving these three goals requires highly sophisticated and frequently very difficult decisions. Thanks to Dr. Pazdur, who arrived at FDA more than a decade ago as a highly regarded physician and researcher, our agency is up to the task. He has helped make FDA a more transparent, innovative and compassionate organization grounded in solid science.

At FDA, we appreciate both his professional and personal qualities. As an FDA official and scientist, Dr. Pazdur explores hard data with industry, academia, and patient advocates in search of innovative approaches to expediting the approval of new drugs. When talking to cancer patients and their advocates, Rick Pazdur, the physician, responds with deeply-felt empathy. But even this great compassion does not weaken his resolve to perform his foremost duty, which is to ensure that the benefits of cancer drugs are not outweighed by their risks.

Dr. Pazdur’s leadership is a hallmark of FDA’s pursuit of science in the service of public health. I am proud to congratulate him on his richly deserved award on behalf of our entire agency.

Margaret Hamburg, M.D., is Commissioner of the U. S. Food and Drug Administration

FDA Voice interviews Helene D. Clayton-Jeter, O.D., on the Cardiovascular and Endocrine Liaison Program

FDA Voice: Thank you for blogging with FDA Voice today. Can you tell us about the Cardiovascular and Endocrine Liaison Program in FDA’s Office of Special Health Issues?

Helene Clayton-Jeter, O.D. Helene Clayton-Jeter: Millions of Americans have cardiovascular disease (CVD) and diabetes, and their ranks are expected to swell as the population ages and the obesity epidemic continues.

To help fight the spread of these conditions, Janelle Derbis, PharmD, and I established the Cardiovascular and Endocrine Liaison Program (CELP) in 2011. We facilitate interactions among patients, health professionals, and the FDA Centers that regulate the medical products that are used to treat diabetes, hypertension and heart disease.

I was an optometrist for 21 years before joining FDA. Treating diabetes and hypertension is near and dear to my heart because many of my patients were affected by these conditions. And Janelle brings her background as a pharmacist to this work.

Patients with these conditions and the health professionals who treat them want an effective, consistent way to interact with FDA and to become part of FDA’s decision-making processes.

FDA Voice: What is the public impact of these two medical conditions?

Clayton-Jeter: Heart disease and stroke are the first and third leading causes of death, respectively, for both men and women in the United States, accounting for more than one-third of all deaths, according to the Centers for Disease Control and Prevention. An estimated 23.6 million people in the U.S.—approximately 7.8% of the population—have diabetes. This chronic illness is the number one cause of blindness in the U.S and one of the leading causes of disability. Complications associated with these conditions can result in significant costs to patients and to the health care system.

FDA Voice: Can you describe some of the CELP tools and activities?

Clayton-Jeter: We recently launched two new web pages, one for diabetes and one for CVD. Both include information on product safety, new drug and device approvals and clearances, prevention and wellness information, and opportunities for public comment.
We also launched two e-mail lists: FDA DiabetesMonitor and FDA CardioBeat. These e-mail lists deliver FDA updates on safety and regulatory issues related to diabetes and CVD, including product approvals, safety warnings, notices of upcoming public meetings, and notices of draft regulatory guidances.

CELP takes advantage of our office’s existing communication tools to facilitate and moderate stakeholder calls on new product approvals. These calls help patient and health professional groups better explain the issues to their constituents because their representatives get the information first-hand.

We are working with Medscape—part of WebMD’s health professional network— to provide information about new topics within FDA. Working with Medscape, we have developed two articles on drug-eluting stents — a medical device and a drug used to treat blocked arteries – and risks posed when taking high dose simvastatin – a cholesterol-lowering drug.

FDA Voice: What do you have coming up on the horizon for CELP?

Clayton-Jeter: We plan to introduce more web-based training programs on CVD and diabetes issues. We will also be speaking at patient advocacy and health professional conferences, such as the Second Annual Health Professional Organization Conference scheduled for October 4, 2012 at FDA’s White Oak Campus.

We encourage you to visit the web pages and sign up for the e-mail lists to stay current on FDA announcements in the areas of CVD and diabetes.

Helene Clayton-Jeter, O.D., co-manages the Cardiovascular and Endocrine Liaison Program (CELP), at FDA’s Office of Special Health Issues.

FDA Voice interviews Paul C. Howard, Ph.D, on Nanotechnology

FDA Voice: Thank you for taking time to discuss the exciting field of Nanotechnology with us.  We’ve heard so much about Nanotechnology – what is it exactly and why has it been tagged as the second industrial revolution?

Paul C. Howard, Ph.D., Dr. Howard:  Thank you for the opportunity. It’s been tagged as the “second industrial revolution,” because of its ability and promise to create new materials with new properties.  Decreasing larger-size materials down to sizes in the nano domain may change the properties of these materials. This could have enormous benefits in many arenas. That’s why there is considerable worldwide investment and research for product performance improvement, usefulness, and marketability using nanotechnology.

Nanotechnology is the science of manipulating materials on a scale so small that they can’t be seen with a regular microscope. The technology could have a broad range of applications, such as increasing the effectiveness of a particular drug, improving the packaging of food, or altering the look and feel of a cosmetic.

Nanotechnology could also be used in medicines designed for the detection, treatment, and prevention of disease; food production and preservation; water decontamination and purification; environmental remediation; lighter and stronger materials for construction and transportation; and energy resources such as solar cells and fuel-efficiency additives, just to name a few.

FDA Voice: Is Nanotechnology considered a new field?

Dr. Howard:  Nanomaterials are not new. They have been around as a result of natural and man-made processes for a long time; however, the ability to see and the ability to manipulate matter at the nanoscale are relatively new.

Nanotechnology draws from several science fields like physics, material science, supra-molecular and polymer chemistry, interface and colloidal science, as well as from the chemical, mechanical, biological, and electrical engineering fields.

FDA Voice: If nanotechnology-sized particles are so tiny, how can you actually see them?

Dr. Howard: The best light microscopes you can look through with the human eye get close to seeing materials on the nano scale.  However, to best view nanomaterials, electron microscopes are needed.

Typically, “transmission” electron microscopes – either like an X-ray, where the electrons go through the material – or “scanning” electron microscopes – which bounce electrons off the surface of the object to give stunning three-dimensional images, are used.

Another type of microscope is the atomic force microscope. Fundamentally, it is a bit like an old phonograph, with an arm with a tiny tip on the end (stylus) and it “feels” along the surface.  When it encounters a nanomaterial, or larger, it deflects, giving a measure of its height.

At the National Center for Toxicological Research (NCTR) and the adjacent FDA Office of Regulatory Affairs laboratory, we use all three types of microscopes to image the smallest nanomaterials.

FDA Voice: How does  nanotechnology impact FDA research?

Dr. Howard: Nanotechnology can be used in FDA-regulated products and we are aware that materials that exist in the nanoscale may have unique properties, and thus may warrant examination.

FDA participates in research programs with collaborating federal agencies to understand the behavior of nanomaterials in biological systems, especially any risk of short- or long-term toxicity.  FDA also collaborates with the National Nanotechnology Initiative (NNI), which serves as the central point of communication, cooperation, and collaboration for all Federal agencies engaged in nanotechnology research.

FDA Voice: Finally, please tell us about the Nanotechnology Core Facility at FDA’s National Center for Toxicological Research.

Dr. Howard: FDA is investing in an FDA-wide nanotechnology regulatory science program that further enhances FDA’s scientific capabilities, including Laboratory Core Facilities, to support regulatory science.

FDA’s Nanocore facilities are located in Maryland and at FDA’s Jefferson Laboratories just south of Little Rock, Arkansas. The Arkansas Nanocore facility is a joint effort between FDA’s NCTR and Office of Regulatory Affairs’ Arkansas Regional Laboratory. Both facilities are co-located on the Jefferson Laboratories campus.

Nanocore has been designed to support research scientists by providing the equipment and knowledge to characterize nanomaterials. Nanocore also anticipates the needs of scientists by being involved in developing methods to detect nanomaterials in biological samples following the use of nanomaterials in biology experiments.

Characterization is very important in any science endeavor, especially research where you are trying to understand the relationship between an object and its good or bad properties when it is in a biological system.

For example, characterization for nanomaterials includes the average size, the aggregation (where they stick together to form a larger mass), shape, chemical composition and purity, surface area, chemistry properties on the surface, chemical/electrical charge on the surface, and stability.  A good description of these is at Characterization Matters,  Nanocore experts work with scientists who need to understand nanomaterial behavior or toxicity and support the work of other scientists who investigate the safety of nanomaterials that may be used in FDA-regulated products. We also characterize the nanomaterials before, during, and after biology or toxicology experiments. Since the equipment and expertise are centralized into this core facility, FDA Nanocore operations also serve as a training resource for FDA regulatory scientists in nanomaterial characterization and detection methods.

Anyone interested in more on nanotechnology can find a wealth of information on FDA’s website, the U.S. National Nanotechnology Initiative (NNI), and the National Science Foundation.

Paul C. Howard, Ph.D., is the Director of the Office of Scientific Coordination and Director of the Nanotechnology Core Facility at FDA’s National Center for Toxicological Research near Jefferson, Arkansas.