Monthly Archives: February 2010

Off-Label Chemotherapy Use

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Off-label use

An FDA approval implies that the drug can now be used to treat a specific condition when it is administered in the manner described on the drug’s label. Further clinical research may be required to study other potential uses of the drug, for example, to treat a different form of cancer. An off-label use of the drug is said to have occurred when an FDA-approved drug is prescribed by a doctor to treat conditions that are not mentioned on the label or in a manner that is not described on the label. There are many reasons as to why it is a common practice to use off-label drugs to treat cancer. Firstly, drugs are usually approved for the treatment of a specific form or stage of cancer. Once approved, a drug that effectively treats a specific form of cancer is often identified as an effective treatment for other types of cancer as well. Secondly, there are certain cancers that necessitate the use of multiple drugs. In that case, one or more drugs are often used off label. Treatment regimens involving multiple drugs are also evolving constantly, as doctors evaluate new drug combinations to improve the quality of patient care.

Getting FDA approval for new labeling information

In order to get FDA approval for new labeling information meant for an existing drug (FDA approved), the drug sponsor should file a supplemental marketing application with the FDA. This is done to ensure the drug’s safety and effectiveness, as relevant to the new labeling information. In certain cases, the sponsor may find out that the information required to file a supplemental application is not as extensive as may be necessary for initial approval. The FDA has launched a variety of initiatives to encourage supplemental applications involving products used in the treatment of cancer. For instance, the FDA constantly interacts with academic, private and professional groups engaged in cancer-related research and treatment. After obtaining their views about off-label drug, the FDA then contacts drug sponsors to assist in the supplemental application submission process.

Drug Development and Approval: Part II

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Orphan drugs

Drugs that are used to treat rare diseases affecting less than 200,000 Americans are referred to as orphan drugs. Since only a small number of patients need these drugs, it may not be possible for the drug’s sponsor to recover the drug development costs. Under the Orphan Drug Act, sponsors are given incentives to develop new drugs for the treatment of rare conditions, including several different types of cancer.

Making the drugs available to patients

Drugs that are being studied and have yet to receive FDA approval are called investigational drugs. Access to these drugs is available only to patients who have enrolled for clinical trials. The drug may receive FDA approval after the end of the clinical trial if there is evidence to prove that benefits of the drug outweigh its potential risks. Based on the awareness that patients, after having tried all available treatment options, may be willing to try new treatments with higher risks, the FDA has initiated the following three programs to ensure that patients with serious illnesses such as cancer can have access to investigational drugs.

Special exception/compassionate exemption: Although enrolling in a clinical trial is the best way to access investigational drugs, these trials often have eligibility criteria (strict rules) to determine if they will be appropriate for a patient. In case the trial is considered inappropriate for a patient, a special exception/compassionate exemption can be used to provide that patient with access to the investigational drug. An exception request should be submitted to the FDA jointly by the patient’s doctor and the sponsor of the drug. The FDA reviews every application on a standalone basis.

Treatment INDs: The treatment Investigational New Drug (treatment IND) program run by FDA allows drug sponsors to request that access to an investigational drug be provided to patients who are not considered appropriate for clinical trials or those for whom no other suitable treatment option may be available. There should be sufficient evidence to prove the drug’s effectiveness and also that it does not have unreasonable risks.

Group C drugs: An agreement between the National Cancer Institute (NCI) and the FDA, the Group C program seeks to provide oncologists with investigational drugs that normally are available via participation in an NCI protocol. Usually these drugs are in their phase III trials and there is evidence to prove their effectiveness in relation to a specific type of tumor. Using this route and after obtaining informed consent, patients who may not have participated in clinical trials can still have access to and benefit from a potentially effective treatment.

Patient involvement in the approval of new drugs

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Patient can contribute to the drug development and approval process by enrolling themselves in clinical trials. Participating in clinical trials not only contributes to improvements in cancer research and treatment but also allows enrolled patients access to closely monitored care and regular reviews on the status of their disease. The FDA has launched two programs to seek patients’ involvement in the drug development and approval process.

FDA’s Cancer Drug Development Patient Consultant Program seeks to include the views of patient advocates, as relevant to the drug development process. In meetings (via phone), selected patient advocates interact with the FDA and drug companies and carry out discussions on the different areas of the drug development process.

The Patient Representative Program provides cancer patients with the option to participate as a patient representative in the advisory committee meetings. A patient representative is expected to provide insight on problems, issues and /or questions, as considered important and relevant to the patient and their family members. A patient representative is required to meet several different qualifications, and those designated as voting members should be assigned the status of special government employees.

Some FDA initiatives for improving the process of drug development and approval in the United States.

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1) Accelerated approval

The accelerated approval program run by FDA allows sponsors to request approval on the basis of alternate findings. Also called surrogate endpoints, these are indirect assessments of disease status, for example, a change in lab results or improvements in symptoms related to the disease. These endpoints can also be used to assess the effectiveness of the treatment or therapy. With accelerated approval programs, the approval process for drugs meant for life-threatening diseases can be expedited in order to provide for an unmet medical need.

2) Engaging drug sponsors early in the drug development process

By interacting with drug sponsors in the early stages of the drug development process, the FDA is trying to reduce the time spent on the development of new drugs. With this effort, the FDA seeks to speed up the development, review and marketing of new treatment options. The FDA conducts meetings with the drug sponsor on a regular basis in order to ensure that an agreement is reached on the ideal design of clinical research and the resulting data is reviewed in a timely manner.

Cancer Cell Metabolism Could Be Key To New Cancer Therapies

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Way back in the 1920s, a German scientist by the name of Otto Warburg reported to the world that cancer cells metabolize sugars in different ways than normal healthy cells. While such a discovery may not seem particularly momentous, this near century-old discovery is continuing to prove its importance by opening the door to a wide number of potentially effective new cancer therapies.

Warburg’s discovery has already resulted in dramatic cancer treatment breakthroughs – modern-day PET-CT scans help pinpoint tumors by highlighting locations in the body that are high in metabolic activity. Now, scientists are once again revisiting the topic of cancer cell metabolism as they search for new ways to not only diagnose the disease, but fight it as well.

As a recent Harvard Medical School study suggests, a cancer cell’s ability to maximize sugar metabolism is likely a critical factor in the disease’s ability to rapidly divide and spread throughout the body. Indeed, the Harvard study concluded that cancer cells use less chemical energy during cell division, and still manage to over-produce resources necessary for new cell growth.

Similar studies have suggested that augmentation to a healthy cell’s metabolism function is critical for cell mutations.

As a result of cancer’s strong ties to sugar metabolism, researchers are hopeful that starving cancer cells of important enzymes that are necessary for processing sugar can shut off cancer growth.

While the line of research is still in its infancy, a groundswell of support is currently in the works. Recently, Agios Pharmaceuticals raised $33 million for the specific purpose of studying metabolic functions. Additionally, the American Association for Cancer Research recently held a 4-day meeting devoted entirely to the topic.

Cancer metabolism is seen as a promising path due to its wide-sweeping occurrence. As it turns out, there are very few commonalities shared between different types of cancers that can be effectively targeted for broad treatment purposes. However, it appears that sugar metabolism is a key component of cancer growth among the majority of cancer types.

While it is still too soon, and perhaps overly optimistic, to suggest that disrupting cancer metabolism may be the breakthrough necessary for curing cancer, the field remains promising. Agios has already identified an enzyme that is necessary for cancer metabolic functions. Presently, they are working to develop a drug that blocks this enzyme without harming normal cell production.

Resource:

http://www.boston.com/business/healthcare/articles/2009/12/21/old_discovery_could_bring_new_cancer_therapies/

FDA fast track program

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With its fast track drug development programs, the FDA seeks to facilitate the development of new drugs and speed up their approval process. This is especially true in case of drugs that can provide for unmet medical needs pertaining to debilitating or life-threatening diseases such as cancer. An unmet medical need is said to exist when no standard treatment is available for a specific disease or condition or when the existing treatment is not effective. Based on these factors, drug sponsors may request the FDA to assign the fast track designation to the new drug. If approved, the drug may come under the purview of a fast track program, which can speed up the approval process.

Improving the Approval Process for Foreign Drugs

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The drug regulatory agency in every country has its own specific requirements for drug approval. A drug that has received approval in Japan or Europe cannot be sold in the U.S. market until it gets approved by the Food and Drug Administration. The creation of the International Conference on Harmonization (ICH) was part of the cooperative effort between the drug regulatory agencies of U.S., Japan, and Europe. The primary objective of ICH is to ensure that information required for making decision related to drug approval are similar between different countries. With ICH, drug sponsors who may have received approval for a drug in Japan or Europe can file the same research papers with the FDA. They may not have to conduct additional research to get approval for the general use of the drug in the United States.

More Asbestos Found on Florida Highway

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Road workers in Florida are still finding masses of dangerous asbestos at a highway construction site near Fort Myers. Work crews employed by Lee County to look into the site near Summerlin Road came across three pieces of the dangerous substance on the site’s east side. They also exposed asbestos-laced concrete chunks that had been hidden under the surface of the road, in trenches excavated up to eight feet deep along the side of the highway.

These most recent findings, combined with more than thirty pieces of affected concrete retrieved on the west side of the crossing of Summerlin Road and College Parkway last month, are part of the mounting evidence uncovered during a probe of the contracting firm involved in road improvements at the intersection. The ongoing investigation has held up the project that was expected to widen Summerlin Road to two more lanes. Work on the project, with an estimated cost of $25.2 million, has been at a standstill for almost two months.

Lee County’s director of transportation, Scott Gilbertson, has said that investigators with his office want to be sure that the amount of information they are able to gather on the potential contamination in the area is complete before they allow workers to restart on the project. As a result, he is not able to offer a schedule as to when work on the project can resume. He also said that his workers will “have to dig through” the site for potential additional asbestos findings before he can clear the site as a safe working environment.

Officials with the Florida Department of Environmental Protection, along with Lee County investigators and workers with Posen Construction, continue to work towards determining the level and extent of the asbestos contamination at the proposed site. If the amount of asbestos fiber found exceeds two hundred sixty linear feet, executives with Posen could be charged with violations of worker safety and environmental laws, punishable by jail time or fines of up to ten thousand dollars per day.

Although workers with the company are cooperating in the investigation, attorneys with the firm have not claimed any responsibility for the contamination. Officials with the company have also refused to answer questions from the press detailing their involvement in using the contaminated materials. However, Posen has hired out another contracting firm that specializes in asbestos removal and remediation to report on the site and to help with other facets of the probe.

Lee County Commissioner Brian Bigelow voiced his hope that the data compiled by the investigators would reveal the level of potential health hazard that the asbestos-laced concrete could pose to both workers and to drivers using the newly expanded road. Although asbestos is considered “safe” as long as it stays bonded to the concrete material, the fibers can become highly dangerous when allowed to become loose and airborne, according to agency staff at the Department of Environmental Protection. DEP workers carried out air and soil quality tests at the site and have yet to find any loose fibers, but the investigation will continue until all parties can resolve the contamination issue.

Sources:

http://www.news-press.com/print/article/20100108/NEWS01/1080407/Roadside-asbestos-keeps-on-coming

http://www.abc-7.com/Global/story.asp?S=11792227

California Slated to Receive Asbestos Cleanup Funds

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In December, President Obama signed a defense-funding bill that would allot $3.2 million to clean up and reclaim land around California’s Mount Umunhum near San Francisco. The area previously hosted Almaden Air Force Station from 1957 to 1980, a site that was home to many operations that left toxic wastes in the water and soil, including asbestos from abandoned buildings and polychlorinated biphenyls (PCBs).

The federal funds are believed to be only a small fraction of the estimated $11 million required to fully reclaim the land. Rudy Jurgensen, a spokesman for the current owners of the site, the Midpeninsula Regional Open Space District, said that his office would coordinate fundraising efforts to bring in the remainder of the funds needed to start the project.He stated that the efforts would also include approaching local, state and federal agencies for grants and low-interest loans.

Congressman Mike Honda, a Democrat who represents the district and led the charge for the funding on Capitol Hill, said that the cleanup project would start as soon as officers from the Army Corps of Engineers begin to develop a plan to remove the asbestos, lead paint and other toxic materials from the site. The Corps had previously surveyed the site to determine the levels of the various harmful substances present in both the still-standing structures and the surrounding soil and groundwater supplies.

According to the congressman, plans are already underway to tear down more than eighty structures on the site, including former officer housing, enlisted men’s barracks and a Cold War-era radar station known as “the Cube”. Congressman Honda also said that he would pursue another $800,000 in federal funding to cover the costs of the demolition. However, no mention was made on how the project would cover the costs of asbestos remediation and removal, nor were any timelines for the completion of the asbestos abatement efforts.

The radar station, erected in 1957, was used primarily as part of the North American Air Defense Command (NORAD) to oversee and intercept potentially hostile air traffic in northern California. During the base’s construction, workers used many materials that are now considered too dangerous or toxic. Many of the structures, including some of those used to house base personnel, used asbestos as insulation and fireproofing material.

The Air Force closed the station in 1980 and sold the land to the Midpeninsula Regional Open Space District in 1986. The district was created in 1972 to create and preserve open spaces on public land in the San Francisco Bay area. Until recently, the land around Mount Umunhum contained such high concentrations of asbestos, waste oil and other toxic chemicals that area had to be closed to the public.

With the start of the cleanup efforts slated for early 2010, area officials hope to create a public park from the scenic site. Park planners have submitted ideas for jogging and hiking trails that will allow visitors to enjoy the view from the top of Mount Umunhum, which spreads from the downtown San Francisco skyline to the waters of Monterey Bay, within the next two years.

Sources:

http://www.mercurynews.com/breaking-news/ci_14044562

http://kliv.com/Mount-Umunhum-in-line-for-federal-cleanup-funds/5952353