Category Archives: Transhuman News

Gene Therapies

Takara Bio advances clinical development of the following gene therapies:

Takara Bio acquired the HF10 business from M's Science Corporation in November 2010 and is currently conducting a Phase I clinical trial in the United States for the treatment of head and neck cancer and other solid tumors, which is sched-uled for completion in fiscal 2014. HF10 is a spontaneously occurring attenuated mutant of Herpes Simplex virus Type 1 that displays strong antitumor activity (oncolytic activity) when locally injected into tumors. Moreover, preclinical data suggests that HF10 also contributed to the acquisition of immunity against the tumors.

In Japan, three clinical studies were conducted in patients with breast cancer, head and neck cancer, and pancreatic cancer at Nagoya University School of Medicine. The results of these studies showed oncolytic activity and tolerability of HF10.

In December 2011, Mie University Hospital began a clinical study on solid tumors. Further, in April 2013, Nagoya University School of Medicine commenced a clinical study with patients suffering from non-resectable pancreatic cancer for the treatment in combination of HF10 with existing anticancer drugs.

MolMed S.p.A, of Italy, which has in-licensed the RetroNectin method from Takara Bio, is now conducting a Phase III clinical trial of HSV-TK gene therapy for high-risk, acute hematological malignancies in Europe. Takara Bio has exclusive rights to this treatment technology in most Asian countries.

In 2008, Takara Bio began conducting a Phase I clinical trial of HSV-TK gene therapy (project code: TBI-0301) at the National Cancer Center Hospital for treatment of patients with relapsed leukemia after hematopoietic stem cell transplantation. However, the number of existing patients with this condition remained low. In order to accelerate this gene therapy project, the TBI-0301 trial was discontinued and a new joint clinical trial in Japan and South Korea (phase I / II clinical trial, project code: TBI-1101) is in preparation to commence in fiscal 2016. The new trial will focus on patients with hematological malignancy after having undergone HLA mismatched hematopoietic stem cell transplantation.

Takara Bio, in a joint effort with both the University of Pennsylvania and Drexel University, commenced an endoribonuclease MazF based gene therapy Phase I clinical trial in the United States for patients that have been infected the human immunodeficiency virus (HIV, otherwise known as the AIDS virus). This clinical trial is scheduled for completion in fiscal 2016.

In the mechanism of AIDS, replication of the virus in infected HIV immune cells causes However, the MazF-modified T-cells (a type of immune cells) are expected to remain functional even if infected by HIV, by preventing replication of the virus. MazF genes are transduced into patient-derived T-cells ex vivo using retroviral vectors that express MazF conditionally upon HIV infection. The MazF-modified T-cells that are infused back into the patients will cleave the RNA strand of HIV and thereby block the replication of the virus when it infects the transduced T-cells. As a result, this method has the potential to become a gene therapy treatment for HIV infections.

Takara Bio, in collaboration with Mie University Hospital, is preparing a Phase I clinical trial in fiscal 2014 on MAGE-A4 antigen-specific T-cell receptor (TCR) gene therapy utilizing next-generation retroviral vectors developed by the Company. The main mechanism of TCR gene therapy is that the gene-transduced lymphocytes, which acquire the capability to specifically recognize cancer cells, attack and eliminate the cancer. TCR genes that are capable of recognizing cancer antigens are tranceduced into the patient's own lymphocytes, which are then re-infused into the patient.

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Gene Medicine | Business Outline | About Us | TAKARA BIO INC.

Welcome to The Journal of Gene Medicine Clinical Trial site, the most comprehensive source of information on worldwide gene therapy clinical trials available on the internet.

From this page you may access the following:

Charts and Tables

Charts and tables showing the number of approved, ongoing or completed clinical trials worldwide. Data is available for:

Interactive Database

A database with detailed information on individual trials. Data search is available for:

The data were compiled and are regularly updated from official agency sources (RAC, GTAC etc..), the published literature, presentations at conferences and from information kindly provided by investigators or trial sponsors themselves.

Information on the trials performed in the United States is derived directly from the OBA/RAC website. Elsewhere, the information is not so readily available. and in some countries regulatory agencies simply do not disclose any information.

Consequently, information on some trials is incomplete. We have adopted as standard procedure the inclusion of all trials where the country and disease addressed is known, even if no other details are available. While it is not entirely satisfactory to have information missing on the type of gene used and the vector etc..., it does have the advantage of providing a more accurate overview of the real number of trials and where they are being performed.

Your input is valuable in assisting us to provide a comprehensive, accurate and up-to-date information service to the gene therapy community. If you are sponsoring or conducting a trial, or if the information on your trial is incomplete, please contact us at

Excerpt from:
Gene Therapy Clinical Trials Worldwide

A gene is the basic physical and functional unit of heredity. Genes, which are made up of DNA, act as instructions to make molecules called proteins. In humans, genes vary in size from a few hundred DNA bases to more than 2 million bases. The Human Genome Project has estimated that humans have between 20,000 and 25,000 genes.

Every person has two copies of each gene, one inherited from each parent. Most genes are the same in all people, but a small number of genes (less than 1 percent of the total) are slightly different between people. Alleles are forms of the same gene with small differences in their sequence of DNA bases. These small differences contribute to each persons unique physical features.

Genes are made up of DNA. Each chromosome contains many genes.

Genetics Home Reference provides consumer-friendly gene summaries that include an explanation of each genes normal function and how mutations in the gene cause particular genetic conditions.

The Centre for Genetics Education offers a fact sheet that introduces genes and chromosomes.

Next: What is a chromosome?

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What is a gene? - Genetics Home Reference

PUBLIC RELEASE DATE:

20-Dec-2013

Contact: Jessica Studeny jessica.studeny@case.edu 216-368-4692 Case Western Reserve University

Many rare disorders are caused by gene mutation, like sickle cell anemia. Yet until now the underlying genetic cause of more common conditions for example, rheumatoid arthritis has evaded scientists for years.

New research from Case Western Reserve University School of Medicine to appear in the journal Genome Research finds that six common diseases arise from DNA changes located outside genes. The study from the laboratory of Peter Scacheri, PhD, shows that multiple DNA changes, or variants, work in concert to affect genes, leading to autoimmune diseases including rheumatoid arthritis, Crohn's disease, celiac disease, multiple sclerosis, lupus and colitis. Further, for each disease, multiple different genes are manipulated by several small differences in DNA.

"We've known that rare diseases are due to one change within one gene with major effects. The key take away is that common diseases are due to many changes with small effects on a handful of genes," said Scacheri, associate professor of genetics and genome sciences.

The research is in advanced online publication and can be found at http://tinyurl.com/okml3ag.

The human genome includes 3 billion letters of DNA. Only 1 to 2 percent of the letters are used as the blueprint for proteins, the body's building blocks. Scacheri's team is part of group of scientists investigating where and why DNA goes awry in the remaining 98 percent the regions between genes. These regions contain thousands of genetic switches that control the levels of genes. This new finding shows that in common diseases, the fine-tuning of those switches is not quite right, leading to incorrect expression of some key genes previously unidentified.

"This is a paradigm shift for the field with respect to pinpointing the genetic causes of common disease susceptibility," Scacheri said.

"The Scacheri lab's study provides a new model for understanding how genetic variants explain variation in common, complex diseases such as rheumatoid arthritis and colitis. That is, the effect of an individual variant may be very small, but when coupled with other nearby variants, the manifestations are much greater, said Anthony Wynshaw-Boris, MD, PhD, chair of the Department of Genetics and Genome Sciences at Case Western Reserve University School of Medicine and University Hospitals Case Medical Center and the James H. Jewell MD '34 Professor of Genetics at the School of Medicine. "This model may also help to explain why genetic studies of these and other common diseases have so far fallen short of providing a satisfactory explanation of the genetic pathways important for the development of these disorders."

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Common disorders: It's not the genes themselves, but how they are controlled

Dec 18, 2013 5:00pm

By Steven C. Moyo, M.D.

A gene mutation can increase your risk of heart attack and death as much as smoking does, new research suggests.

Duke University researchers reported today finding a link between a gene mutation known to increase the bodys response to stress and heart health. They found in a study of more than 6,000 patients with heart disease, carriers of this genetic mutation had a 38 percent increased risk of heart attack or death.

Genetic mutations are not just the stuff of movies and comic books. They are changes in our DNA code that affect the color of our eyes, our risks for cancer and, as this study shows, even our response to stress.

When we are stressed, it sets off a chain reaction of chemical signals in our bodies. The first of these is the release of a chemical called serotonin in the brain something that happens as soon as we get yelled at by our boss, for example, or get cut off in traffic. This release of serotonin lights the fuse for the explosive cascade of chemicals that follows, eventually leading to increased levels of cortisol in our system.

What this gene mutation does is produce a slightly different serotonin receptor in the brain one that causes an even greater than normal release of cortisol in response to stress.

This is bad news for our hearts. Specifically, increased cortisol has been shown to be associated with higher levels of calcium deposits in the hearts blood vessels, and blockage of these vessels is linked to increased risk of heart attack and death.

Men with this gene mutation have been shown to have a two- to three-times larger cortisol response to stress, said study investigator, Dr. Redford Williams, professor of medicine at Duke University. This higher-than-normal cortisol response from this gene, he said, boosts the risk of heart attack or death. Williams added that more than one in eight men and up to 2 percent of women in the general population are thought to carry this gene mutation, and that the increased risk associated with this gene is comparable to the increased risk associated with a smoking habit.

Williams said he hopes the findings will shed further light on the role of genes in stress-related heart ills a hope shared by heart disease experts not involved with the study.

Read more:
'Stress Gene' Ups Heart Attack, Death Risk

By Charles E. Buban Philippine Daily Inquirer

A Genetic test is a medical procedure that looks at a persons genetic material and may be used to help a person know if he or she has genetic diseases or is at increased risk for some medical problems.

While this concept of gene testing is often met with fear and apprehension, a number of experts believe it should be welcomed with the kind of fervor and enthusiasm from anybody who wants to understand his/her healths predisposition.

Why wait for a health issue to strike when you can prevent it so you can enjoy life better? Your gene test should be able to guide you on how to live your life so those health issues that you are likely to have will not manifest and [so you can] preempt them with proper health maintenance courtesy of a specifically tailored health program that suits your body, said Dr. Ben Valdecaas, medical director of LifeScience Center for Wellness and Preventive Medicine.

A pioneer in preventive care and customized health management in the country, LifeScience Center for Wellness and Preventive Medicine believes that gene testing is one of the steps that one can take to become fully equipped with information about ones self.

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Tags: genetic test , health and wellness , health management , preventive care

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Why not gift yourself with gene test this Christmas?

Heart disease and stroke killed more than 787,000 people in 2010 -- about one in three Americans. A new study suggests a stress gene may be to blame for some of these deaths.

While obesity, high blood pressure, smoking, physical inactivity and eating a high-sodium diet devoid of fruits and vegetables all can raise risk for cardiovascular problems, the study authors say theyve found a biological explanation for why some people are predisposed to develop heart disease or die early from a stroke or heart attack.

"The exciting part to me this is that this genetic trait occurs in a significant proportion of people with heart disease," Dr. Beverly Brummett, an associate professor of psychiatry and behavioral sciences at Duke University School of Medicine in Durham, N.C., said in a statement.

The study builds on work scientists previously did to identify a genetic variant called a single nucleotide polymorphism, or SNP, in a DNA chain that makes a receptor for the neurotransmitter serotonin. Serotonin is a chemical messenger in the brain that is tied to emotion regulation. Essentially, an SNP is when one letter in a DNA sequence is swapped with another letter, which leads to a change in function. I this case, the function of the gene is changed to cause a hyperactive reaction to stress.

The researchers point out that a study last year found men with these gene variants had twice as much cortisol, a hormone related to stress, in their blood while under stress compared to men without the variant.

"It has been shown that high cortisol levels are predictive of increased heart disease risk. So we wanted to examine this more closely," said Brummett.

They conducted a genetic analysis of more than 6,100 people who underwent cardiac catheterization procedures, two-thirds of whom were male. In that procedure, a long, flexible tube is threaded through a blood vessel in the arm so doctors can perform tests or treatments that directly target the bloodstream.

They found the overactive stress gene in 13 percent of the patients. After tracking them for six years, the researchers discovered the genetic trait raised risk for heart attack and early death by 40 percent. This was the case even after ruling out other factors like age, obesity, smoking history and severity of their heart disease.

The study didnt look specifically at what is going on in the body to cause this increased risk, but the researchers have a theory that rising cortisol levels cause elevations in an ezyme called MMP9. This enzyme breaks down plaques in blood vessels, making them more likely to form cloths that could cause a blockage that leads to a heart attak.

"If we can replicate this and build on it, we may be able to find ways to reduce the cortisol reaction to stress -- either through behavior modification or drug therapies -- and reduce deaths from heart attack," said Brummet.

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"Stress gene" may raise heart attack risk in healthy people

A stress gene has been linked to having a higher risk of dying from a heart attack or heart disease.

Heart patients with the genetic change had a 38 per cent increased risk of heart attack or death, say US researchers.

Personalised medicine may lead to better targeting of psychological or drug treatment to those most at risk, they report in PLOS ONE.

The study adds to evidence stress may directly increase heart disease risk, says the British Heart Foundation.

A team at Duke University School of Medicine studied a single DNA letter change in the human genome, which has been linked to being more vulnerable to the effects of stress.

They found heart patients with the genetic change had a 38 per cent increased risk of heart attack or death from heart disease after seven years of follow up compared with those without, even after taking into account factors like age, obesity and smoking.

This suggests that stress management techniques and drug therapies could reduce deaths and disability from heart attacks, they say.

director of the Behavioural Medicine Research Center at Duke University School of Medicine, Dr Redford Williams, said the work is the first step towards finding genetic variants that identify people at higher risk of cardiovascular disease.

This is one step towards the day when we will be able to identify people on the basis of this genotype who are at higher risk of developing heart disease in the first place, he told BBC News.

Thats a step in the direction of personalised medicine for cardiovascular disease.

Originally posted here:
Stress gene linked to heart attack – Study