Will Gene-edited Stem Cells Hold the Key to Fighting HIV/AIDS?

In a research conducted at Harvard University, a new gene-editing technique was used to create what could prove to be an effective method for blocking HIV from invading and destroying patients' immune systems. The work was led by Chad Cowan and Derrick Rossi, Associate Professors in Harvard's Department of Stem Cell and Regenerative Biology (HSCRB). 

This is the first published report of the Harvard researchers using CRISPR/Cas technology to efficiently and precisely edit clinically relevant genes out of cells collected directly from people, in this case human blood-forming stem cells and T-cells, researchers said. 

In theory, such gene-edited stem cells could be introduced into HIV patients via bone marrow transplantation—the procedure used to transplant blood stem cells into leukemia patients, to give rise to HIV-resistant immune systems. 

Human Immunodeficiency Virus or Acquired Immune Deficiency Syndrome (HIV/AIDS) is one of the most catastrophic threats to human health in the world. Improved treatment options and methods of diagnosis have helped to moderate the growth of the epidemic, presenting opportunities for companies prepared to engage actively in this field. However, the management of HIV/AIDS is still, in many respects, a very significant threat, and there is an ongoing, urgent need for promising new research as well as optimal exploitation of the treatment and diagnostic options already developed.

According to the Joint United Nations Programme on HIV and AIDS (UNAIDS), there were 35 million people across the globe living with human immunodeficiency virus (HIV) in 2012. In 2012, 1.6 million people died of HIV/AIDS, including 1.2 million AIDS-related deaths in sub-Saharan Africa.

According to a BCC Research report, in 2012 the HIV therapeutics market was worth $17.5 billion. The global market is expected to peak at $20.9 billion in 2016 and will shrink back to $19.6 billion in 2018, representing an overall compound annual growth rate (CAGR) of 1.5%. The patent expiration of leading antiretroviral drugs and the subsequent introduction of generic drugs will create cost pressures that will drive overall revenues down, resulting in suppressed market revenue growth.

Though this new approach to HIV therapy might be ready for human safety trials in less than five years, the researchers are still cautious about celebrating victory. Even if this new approach works perfectly, further developments need to be carried out before they are introduced in the global market.

For more information on a BCC Research market report about HIV therapeutics and diagnostics, visit the following link:

http://www.bccresearch.com/market-research/pharmaceuticals/hiv-diagnostics-therapeutics-phm058b.html

Growing Prevalence of a Silent Killer— Diabetes

The number of people around the world suffering from diabetes has skyrocketed in the last two decades, from 30 million to 230 million, claiming millions of lives and severely taxing the ability of health care systems to deal with the epidemic, according to data released by the International Diabetes Federation. The demographics of the diabetes epidemic are also changing rapidly at the same time. While the growing problem of diabetes in the United States has been well documented, the federation’s data shows that 7 of the 10 countries with the highest number of diabetics are in the developing world.

Type I diabetes is an autoimmune disease; it appears in childhood, is lifelong, and currently must be treated with insulin. Type II diabetes typically appears in middle age. It is linked to obesity and therefore is more prevalent in developed countries with relatively affluent lifestyles, sedentary occupations, and dietary overindulgence. 

According to the American Diabetes Association's journal, Diabetes Care, Asia accounts for 60% of the world's diabetic population. In recent decades, Asia has undergone rapid economic development, urbanization, and transitions in nutritional status. China now has the largest number of diabetics over age 20, around 39 million people or about 2.7 percent of the adult population, according to the federation. The group also mentions India with the second largest number of cases with an estimated 30 million people, or about 6 percent of the adult population.

There are many factors driving the growth in diabetes worldwide, but most experts agree that changes in lifestyle and diet are the chief culprits, in addition to genetic predisposition. As developing countries rapidly industrialize, people tend to do work involving less physical activity. At the same time, the availability of food that is cheap but high in calories becomes more common.

Typically, type II diabetes occurs after a person becomes obese, when insulin resistance occurs;  the diabetes comes next. When this occurs, the cells do not respond properly to insulin; glucose does not enter the cells and blood glucose (sugar) levels rise. When fat is stored in the "wrong" places (blood vessels, heart and muscles) in the body, insulin resistance is much more likely to occur. Experts are not sure exactly how the association works.

The most common treatment for type II diabetes today involves initially placing the patient on a special diet; sometimes they may need to take pills that increase insulin secretion and also make the cells more sensitive to insulin. Occasionally they are given tablets to bring down the production of glucose. However, after a few years, for about one-third of all patients these treatments gradually lose their efficacy and insulin injections are needed. 

The most effective treatment today, however, to prevent type II diabetes onset among very obese patients is bariatric surgery.

World-renowned British specialist Dr David Cavan, Director of policy at the International Diabetes Federation, hands patients a lifeline with a simple regime that can reduce the devastating effects of type II diabetes. His plan includes adopting a healthy diet, getting support from your family, boosting exercise, assessing current diabetes drugs, keeping up to date with monitoring the condition – and, finally, being realistic about what you want to achieve.

According to Dr Cadan, people with type II diabetes will be motivated to change their lifestyle if they realize that it is possible to become free from diabetes rather than if they think that whatever they do, they will always have it. He added that reducing sugar and understanding that some starchy carbohydrates have almost the same effect as eating sugar can bring about swift changes.

According to the International Diabetes Federation and other major professional organizations, the global population of individuals with diabetes (type I and II) was about 240 million in 2010, and is expected to rise to 300 million by 2025.  The corresponding market of products used to diagnose and treat diabetes was $118.7 billion for 2012, and is expected to rise to almost $157 billion over the next five years.  The market for monitoring equipment stands at approximately $14 billion and is set to rise toward $21 billion by 2017.

Working towards introducing innovative solutions in this area, French pharmaceutical company, Servier, is planning to pioneer a tiny drug-loaded implantable pump, developed by a Boston-based start-up, Intarcia Therapeutics Inc., which is anticipated to transform the global market for patients with diabetes.

Servier has agreed to pay Intarcia Therapeutics Inc. $171 million up front, with potential additional payments that could increase the total to more than $1 billion, for rights to co-develop the device for most markets outside the U.S., the companies said. Closely held Intarcia retains full rights to the treatment for the U.S. and Japan. The pump hasn’t yet been approved for sale; the companies plan to submit it to regulators in the first half of 2016.

In Sweden, the researchers from Stockholm University say that they have uncovered a new mechanism that encourages glucose uptake in brown fat. They explain that brown fat's main function is to create heat by burning fat and sugar. By using this new knowledge, the researchers say they may be able to stimulate this signalling pathway with drugs, lowering blood sugar levels and potentially even curing type II diabetes.

The brown fat is active in adults, acting as one of the bodily tissues that can be encouraged to take up large amounts of glucose from the bloodstream to use as a fuel source to create body heat, the researchers said. As such, increasing the uptake of glucose in brown fat can quickly decrease blood sugar levels, they added.

In a person with the condition, the body's tissues are unable to respond to insulin, rendering them unable to take up sugar from the blood. Because insulin is released after eating to regulate blood sugar, when the insulin signal no longer functions properly, blood sugar levels rise. Very high blood sugar levels are dangerous to organs in the body and can lead to heart disease, kidney failure, blindness, peripheral nervous system damage, amputations and even early death.

"This is completely new and groundbreaking research," Prof. Tore Bengtsson of Stockholm University's Department of Molecular Biosciences said.

On December 20, 2006, the United Nations (UN) passed a resolution to designate November 14 as World Diabetes Day. The occasion aimed to raise awareness of diabetes, its prevention and complications and the care that people with the condition need. World Diabetes Day was first commemorated on November 14, 2007, and is observed annually.

For BCC Research market reports on diabetes, visit the following links:

http://www.bccresearch.com/market-research/healthcare/diabetes-therapeutics-diagnostics-markets-hlc029d.html

http://www.bccresearch.com/market-research/healthcare/insulin-drug-delivery-devices-hlc149a.html

http://www.bccresearch.com/market-research/healthcare/continuous-glucose-monitoring-cgm-technology-markets-hlc102a.html

http://www.bccresearch.com/market-research/biotechnology/bioengineered-protein-drugs-report-bio009f.html

http://www.bccresearch.com/market-research/pharmaceuticals/autoimmune-disease-diagnostics-report-phm160a.html

http://www.bccresearch.com/market-research/healthcare/eldercare-market-hlc062b.html

The Impact of DNA Vaccines on the Biotechnology Industry

Vaccines are considered a standard preventive treatment in many clinical situations today. They work by inducing an immune response against an inert pathogen to protect against future infection.

A new type of vaccine – DNA vaccines – provide an alternative method to produce immunity in organisms. First developed during the 1990s, DNA vaccines use genetically engineered DNA to produce an immune response. They work by causing the body to translate the injected DNA sequences into pathogenic proteins. The body then creates antibodies specific to the proteins, which creates immunity without causing infection. This is important for immune-compromised patients, including those infected by HIV; conventional vaccines can potentially trigger an actual infection in weakened immune systems. 

Though currently still in the experimental stages, DNA vaccines have several advantages over conventional vaccines. Conventional vaccines cover only a small number of diseases, but DNA vaccines are relatively easy to design for a range of difficult pathogens. DNA vaccines will target a wide range of diseases, such as cancers and allergies, as well as infectious diseases. Studies over the past decade suggest that DNA vaccines can be used for immunity against infections and diseases such as HIV/AIDS and malaria that kill millions worldwide every year.

DNA vaccines are also easier to distribute than traditional vaccines because they are more stable, avoid the risk of accidental infection by the pathogen, and require no refrigeration. Conventional vaccines can potentially become inert when stored in improper environments, while DNA vaccines are less susceptible to damage due to environmental conditions, such as extreme temperatures or humidity. They can be administered safely to people who live in areas where regular vaccines are difficult to maintain or may be compromised due to the lack of proper storage facilities.

DNA vaccines, if integrated into the body appropriately, can produce a sustained immune response, making booster vaccinations unnecessary. After receiving a single DNA vaccine, an individual can have lifelong immunity to a disease, decreasing the need (and cost) for booster shots.

In addition to the general medical benefits, DNA vaccines can provide a large economic benefit. Due to the decreased restrictions in the production and storage of DNA vaccines compared to regular vaccines, the cost of producing and maintaining DNA vaccines is much lower. This can be especially beneficial to people in developing countries. According to certain case studies, the cost of developing and manufacturing a successful and beneficial conventional vaccine can range from $500 million to $1 billion. Comparatively, the development and manufacturing of a DNA vaccine ranges between $200 and $300 million.

Currently there is limited knowledge of the effects of DNA vaccines on humans, since most tests have been conducted only on lab animals. Potential side effects could include chronic inflammation, because the vaccine continuously stimulates the immune system to produce antibodies. Other concerns include the possible integration of plasmid DNA into the body’s host genome, resulting in mutations, problems with DNA replication, triggering of autoimmune responses, and activation of cancer-causing genes.

A 2014 market research report published by BCC Research forecasts the global market for DNA vaccines will grow from $305.3 million in 2014 to $2.7 billion in 2019, yielding an impressive compound annual growth rate (CAGR) of 54.8%. While research tools and animal health applications currently comprise the commercialized market, human clinical DNA vaccines will make up the vast majority of this market by 2019.

In the age of genomics where DNA can be sequenced and created more quickly, accurately, and cheaper than ever before, and where safety and handling live pathogens is fraught with risk and difficulty, further research on DNA vaccines is surely a worthwhile pursuit when addressing modern food security, animal health and perhaps even human healthcare challenges.

For our market research report on DNA vaccines, visit the following link:

http://www.bccresearch.com/market-research/biotechnology/dna-vaccines-bio067b.html