Amy Ritter was Scientific Editor, BioPharm International.

Amy Ritter, PhD

On Apr. 29, 2011, a federal appeals court has lifted a ban on federal funding for embryonic stem cell (ESC) research. The move was celebrated by researchers hoping to use such cells for research, but it is likely that the decision will be appealed. Embryonic stem cells are pluripotent cells derived from embryonic tissue in a process that usually results in the destruction of the embryo. The ban on funding did not apply to stem cells derived from adult tissue.

The case began when a lawsuit was brought against the National Institutes of Health (NIH) by Dr. James Sherley and Dr. Theresa Deisher, scientists opposed to embryonic stem cell research.  The scientists cited a 1996 federal law called the Dickey-Wicker Amendment that prohibits funding for "research in which a human embryo or embryos are destroyed." The scientists also claimed that funding embryonic stem cell research hurt their ability to receive federal funding for their own research using adult stem cells. In August 2010, a district court agreed with the plaintiffs that embryonic stem cell research violated federal law and blocked funding, but a three-judge appeals court temporarily lifted the ban in September. The issue is whether that law's restrictions apply to research conducted on stem cell lines that had been developed without federal funding. NIH has interpreted the law narrowly, and argued that creating embryonic stem cells is not the same as using lines created without federal funding for research purposes.  In a 2-1 

 the appeals court sided with the NIH, finding the Dickey-Wicker Amendment to be “ambiguous”, and the NIH’s interpretation reasonable.

Francis Collins III, director of the NIH, issued this statement: “I am delighted and relieved to learn of the decision of the Court of Appeals. This is a momentous day—not only for science, but for the hopes of thousands of patients and their families who are relying on NIH-funded scientists to pursue life-saving discoveries and therapies that could come from stem cell research.”

Articles

A federal appeals court has lifted a ban on federal funding for embryonic stem cell research.

Federal Appeals Court Lifts Ban on Funding for Embryonic Stem Cell Research

The IMS institute has released its report on the use of medicines in the United States during 2010. The report looks at consumer spending on prescription drugs, and presents data on how many prescriptions have been filled, in which therapeutic areas, and compares use of generics with branded drugs.

Total spending on drugs was around $307 billion, an increase of 2.3% over 2009, but continuing a trend in declining growth since 2007 and representing only a 0.6% increase on a per capita basis. While branded drugs captured the greatest number of those dollars, $229 billion, this represented a small decrease of 0.7% from the previous year. In contrast, spending on generic drugs grew by 21.7%, and spending on branded generics grew by 4.5%. According to the report, “total branded and unbranded generic market share has risen each of the past five years to now account for 78% of all prescriptions dispensed.”

In today’s cost-conscious environment, generics have grown to be extremely efficient at capturing market share. In 2006, when branded products lost patent protection they still managed to keep 45% of the market share a year later. For 2010, the report indicates that, “within six months of patent loss, patients received the generic form of the molecule 80% of the time.” 

The report finds that since 2009, patient office visits have declined by 4.2%, and that the total number of patients beginning a new, chronic therapy has declined by 3.4 million patients. A larger number of prescriptions in 2010 were being filled on Medicare Part D and Medicaid than in the previous year, together accounting for 30.2% of all prescriptions in 2010. Taken together, these findings seem to represent an impact of the poor economy on health care habits and spending.

When broken down by therapy area, the top class of drug in 2010 based on spending was oncologics  at $22.3 billion. This spending represents a modest increase of 3.5% over last year, and the lowest level of growth recorded over the past five years. Respiratory agents were second at $19.3 billion, and lipid regulators were third at $18.7 billion, growing by only 0.9% with much of the class available as generics. Antidiabetics were one of the few classes that saw robust growth in spending, up 12.5% to $16.9 billion, with more than half of the growth attributable to increases in spending on human insulin and its synthetic analogs.

Overall, the report finds that while prescription drug use continues to rise, the increases are low compared to years past, and increases in revenues are eaten away by competition from generics. New and innovative medicines will continue to generate large revenues, but the pressure is in on manufacturers to keep a steady supply of premium drugs coming on to the market.

The IMS institute has released its report on the use of medicines in the United States during 2010.

IMS: Growth in Drugs Stalls, Generic Use Up

The US patent laws are undergoing their first large revision since the Patent Act of 1952. The 

 introduced by Senator Leahy (D-VT) on Jan. 25, 2011, was overwhelmingly passed by the US Senate on Mar. 8, 2011. It was then introduced in House of Representatives on Mar. 30, 2011, where it is still awaiting a vote.

The act would introduce reforms that bring US patent laws more in line with those of the EU. In particular, it would revise policy so that patents would be granted on a first-to-file basis, rather than on a first-to-invent basis. Under the current system, a division of the US Patent and Trademark Office settles disputes about who was the first to invent a product, in an expensive and uncertain process that puts cash-strapped academics at a disadvantage.

The first-to-file system is a less ambiguous measure of who has first claim to an invention. This system carries risk to academics, particularly if they begin the process of commercialization before filing. However, the act contains provisions intended to protect inventors, such as the ability to request a postgrant review during the first nine months after issue, and a provision allowing third parties to submit prior art during patent examination. First-to-file status is intended to remove much of the ambiguity around patent filings, and enable a more streamlined, less expensive patent-application process than the current procedure.

The US patent laws are undergoing a major revision, the first large revision since the Patent Act of 1952.

Congress Overhauling US Patent Laws

Nearly 900 therapies for the treatment, diagnosis, or prevention of cancer are currently in development, according to a 

released by the Pharmaceutical Research and Manufacturers of America (PhRMA). This is more than double the number of therapies that were in development six years ago.  

Underlying the increase is a greater understanding at the genetic level of the causes of cancer, and advances in research on the cellular mechanisms underlying tumor growth and metastasis. This has paved the way for targeted therapy: therapies directed toward specific features of tumor growth and survival. For example, therapies are being developed that interfere with vascularization of tumors, and others are being developed that  deprive tumors of energy derived from glucose. The hope is that these specific treatments will be effective but have less general toxicity than current treatments.  

The list of development candidates includes a variety of potential therapeutics other than small-molecule therapeutics. Biologics, including vaccines and antibody treatments (monoclonals, polyclonals and antibody fusion constructs), are well represented.  Gene therapy, antisense treatment, and a therapy using encapsulated cells are also in development. The inclusion of biologics increases the arsenal available to pharmaceutical developers, according to the PhRMA report.

According to the report, cancer occurs in many forms, and one medication does not fit all. Despite gains made in treatment, particularly of breast and prostate cancer, cancer remains the second leading cause of death in the United States. Pharmaceutical manufacturers have certainly recognized the unmet medical need, and are responding.

Nearly 900 therapies for the treatment, diagnosis or prevention of cancer are currently in development, according to a report released by PhRMA.

A Record Number of Therapies for Cancer Currently in Development

The distribution of counterfeit drugs is a serious problem in Nigeria, as in many developing nations, driven by the desire of an impoverished population for drugs at the lowest possible price. A recent

 from CNN highlights Nigeria’s response to this problem, which includes vigilant police work, and a novel strategy for easily identifying legitimate products adopted by some Nigerian manufacturers:  

The strategy is the innovation of the Boston-based company Sproxil, and relies on a code contained on a scratch card within the drug packaging. Consumers can enter the code by phone and receive instant confirmation that the product is legitimate.  Manufacturers pay for the service and have been able to recapture market share from the counterfeit market. In CNN’s video, Dr. Ashifi Gogo, CEO of Sproxil states: “In a sense, the counterfeiters are paying for this service, which puts a smile on everyone’s face, except the counterfeiters.” 

In addition to local solutions, Nigeria is reaching out to its trade partners to help combat this problem. On Mar. 16, 2011, a Memorandum of Understanding in the field of pharmaceuticals was 

 between Nigeria’s Department of Pharmaceuticals and India’s National Agency for Food and Drug Administration and Control. Among other things, the document outlines policies that will help promote mutual trade in good quality drugs, drug testing and analysis, and detection and prevention of the supply of adulterated, fake, drugs. The agreement stipulates a 

 ¾ life imprisonment ¾ for Indians caught producing or distributing counterfeit pharmaceuticals in Nigeria, as well as confiscation of property by the government.

Nigeria Looks to Simple Packaging Controls and International Cooperation to Curb Counterfeit Drugs

	ePT-the Electronic Newsletter of Pharmaceutical Technology
	The National Institutes of Health’s (NIH) definition of embryonic stem (ES) cells poses new challenges for investigators who seek federal research funding. Current NIH guidelines narrowly define ES cells, as “cells that are derived from the inner cell mass of blastocyst stage human embryos, are capable of dividing without differentiating for a prolonged period in culture, and are known to develop into cells and tissues of the three primary germ layers" (1).
	
	More than 75 such cell lines have been approved to receive federal funding for research. A smaller subset of ES cells, however, has been excluded from funding because it does nott meet the narrow definition. ES cells can now be derived from a single blastomere taken from an eight-cell embryo, in a procedure similar to that used during genetic testing on embryos created by in vitro fertilization. After the blastomere is removed, the embryo remains viable and is refrozen. Although this procedure does not destroy human embryos, cell lines derived in this fashion are currently awaiting further review by NIH.  
	
	FDA has given Advanced Cell Technology clearance to start clinical trials for macular degeneration this year using cells produced from a blastomere-derived ES cell line. The company was forced to find alternative sources of funding for their trial since their cell line is not eligible to receive federal funds.
	
	In Europe, regulations regarding embryonic stem-cell research differ from country to country. However, a ruling on Mar. 10, 2011, by the European Court of Justice of the European Communities denied patents on ES cells to Oliver Brüstle of the University of Bonn on ethical grounds. The court found that even if cell lines could be established without the destruction of embryos, the commercialization of human embryos was unacceptable, and contrary to public policy. This ruling may push European countries to adopt restrictive policies regarding embryonic stem cells and inhibit the development of ES-based therapies.
	
	


	
	1. National Institutes of Health, “

Guidelines for Research Using Human Stem Cells

The National Institutes of Health?s (NIH) definition of embryonic stem (ES) cells poses new challenges for investigators who seek federal research funding.