Gene Therapy Helps Weak Mice Grow Strong

A virus that shuttles a therapeutic gene into cells has strengthened the muscles, improved the motor skills, and lengthened the lifespan of mice afflicted with two neuromuscular diseases. The approach could one day help people with a range of similar disorders, from muscular dystrophy to amyotrophic lateral sclerosis, or ALS. Many of these diseases involve defective neuromuscular junctions—the interface between neurons and muscle cells where brain signals tell muscles to contract. In one such disease, a form of familial limb-girdle myasthenia, people carry two defective copies of the gene called DOK7, which codes for a protein that’s needed to form such junctions. Their hip and shoulder muscles atrophy over many years, and some eventually have trouble breathing or end up in a wheelchair. Mice similarly missing a properly working Dok7 gene are severely underweight and die within a few weeks.
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Ebola, Marburg DNA Vaccines Prove Safe in Phase 1 Trial

A phase 1 clinical trial showed that 2 experimental vaccines, 1 against Ebola and 1 against the related Marburg virus, are well-tolerated and produce immunity after a course of 3 shots and a later booster. The results were published online September 14 in the Journal of Infectious Diseases. Both viruses are in the Filoviridae family, both cause hemorrhagic fever, and both rely on a glycoprotein on their surface for entry into human cells. Previous studies in nonhuman primates showed that vaccines based on this glycoprotein in its natural, wild-type form are safe and provide better protection than vaccines in which the glycoprotein had been altered.

The current trial tested each vaccine in 10 healthy adults aged 18 to 60 years who were recruited in 2008 and 2009. The vaccines were made with a strand of DNA encoding the virus's wild-type glycoprotein. The DNA does not replicate in human cells, but the patient's body makes glycoproteins from the DNA instructions, triggering an immune response. The Marburg virus vaccine was based on the glycoprotein from the Angola strain, and the Ebola vaccine was based on glycoproteins from the Zaire and Sudan species. (The current Ebola outbreak in western Africa involves the Zaire species.)
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New Immunotherapy Vaccine Developed For HER2 Positive Breast Cancer

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Researchers at the University of Texas MD Anderson Cancer Center have developed a new and promising immunotherapy breast cancer vaccine candidate, GP2, which prevents disease recurrence in high-risk patients, especially in combination with a powerful immunotherapy drug. The GP2 vaccine was tested in a phase II randomized trial with 190 patients having varying levels of HER2, an oncoprotein promoting tumor growth which is expressed in 75 to 80 percent of breast cancers. The study used the GP2 vaccine, which is designed to stimulate CD8+ cells, in combination with an immune stimulant (GM-CSF). The patients were monitored for nearly three years for disease progression and recurrence.
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Gene-Silencing Drugs Finally Show Promise

In 1998, researchers at the Carnegie Institution and the University of Massachusetts made a surprising discovery about how cells regulate which proteins they produce. They found that certain kinds of RNA—which is what DNA makes to create proteins—can turn off specific genes. The finding, called RNA interference (RNAi), was exciting because it suggested a way to shut down the production of any protein in the body, including those connected with diseases that couldn’t be touched with ordinary drugs. It was so promising that its discoverers won the Nobel Prize just eight years later. Initially researchers ran into several problems making the use of RNAi problematic, but in recent years breakthroughs have given rise to hope for this type of treatment in the future.

Treating cancer is one area where RNAi’s particular advantages are expected to shine. Conventional chemotherapy affects more than just the target cancer cells—it also hurts healthy tissue, which is why it makes people feel miserable. But RNAi can be extremely precise, potentially shutting down only proteins found in cancer cells. And Dahlman’s latest delivery system makes it possible to simultaneously target up to 10 proteins at once, which could make cancer treatments far more effective. Lab work like this is far from fruition, but if it maintains its momentum, the drugs currently in clinical trials could represent just a small portion of the benefits of the discovery of RNAi.
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Ebola, Fast-tracking treatments

The death toll from the Ebola virus is continuing to grow alarmingly. On September 9th the World Health Organisation (WHO) said it had recorded 4,293 cases in five west African countries, of which at least 2,296 people had died. But even the WHO’s experts believe that is an underestimate as many people are suspected to be dying at home. By some estimates 12,000 people have been infected with Ebola so far. The scale of the present outbreak, together with the fear and suffering it is causing, has resulted in a burst of scientific activity to find new treatments and vaccines. Some of these medicines look promising. But to contain the spread of Ebola, scientists and health officials will have to bypass many of the existing rules that govern the delivery of new drugs, and develop potential remedies with unprecedented speed.
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Cancer Research UK, CRT Partner with Asterias to Trail Novel Immunotherapy Treatment for Lung Cancer

CANCER RESEARCH UK and Cancer Research Technology (CRT), the charity's development and commercialisation arm, have reached an agreement with Asterias Biotherapeutics, Inc. (OTCBB: ASTY), a biotechnology company in the emerging field of regenerative medicine, to take forward Asterias' novel immunotherapy treatment AST-VAC2 into clinical trials in subjects with non-small cell lung cancer. AST-VAC2 represents the tenth treatment to enter Cancer Research UK's Clinical Development Partnerships (CDP) scheme, with six having progressed into the clinic to date. CDP is a joint initiative between Cancer Research UK's Drug Development Office (DDO) and Cancer Research Technology, to develop promising anti-cancer agents which pharmaceutical companies do not have the resources to progress through early phase clinical trials. AST-VAC2 is a non-patient specific (allogeneic) cancer vaccine designed to stimulate patients' immune systems to attack telomerase, a protein that is expressed in over 95 percent of cancers but is rarely expressed in normal adult cells.
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61 Percent Fall in Female Genital Warts Due to Free HPV Vaccine

GPs in Australia are managing 61 per cent less cases of genital warts among young women since the introduction of the national human papillomavirus (HPV) vaccination program, a new study from the University of Sydney reveals. The study, which reviewed more than a million patient encounters between 2000 and 2012, showed a significant year-on-year reduction in the management rate of genital warts in women aged 15-27 years since the vaccination program started. The findings are published in PLOS One journal. The HPV vaccination program was introduced in 2007, and the rate of genital wart presentation fell dramatically from 4.33 per 1,000 encounters pre-program (2002-2006) to 1.67 per 1,000 encounters in the post-program period (2008-2012).
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F.D.A. Allows First Use of a Novel Cancer Drug

The Food and Drug Administration on Thursday approved the first of an eagerly awaited new class of cancer drugs that unleashes the body’s immune system to fight tumors. The drug, which Merck will sell under the name Keytruda, was approved for patients with advanced melanoma who have exhausted other therapies. Cancer researchers have been almost giddy in the last couple of years about the potential of drugs like Keytruda, which seem to solve a century-old mystery of how cancerous cells manage to evade the body’s immune system. The answer is that tumors activate brakes on the immune system, preventing it from attacking them. Keytruda is the first drug approved that inhibits the action of one of those brakes, a protein known as PD-1, or programmed death receptor 1.
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What are the 10 Possible Cures and Vaccines for Ebola?

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Doctors from around the world are gathering in Geneva to discuss the possible usage of ten vaccines and cures for Ebola, as the virus continues to spread with ever-increasing speed. The World Health Organisation (WHO), which is hosting the conference, said earlier this week that 1,900 people are now known to have died of Ebola – mostly in Guinea, Liberia and Sierra Leone. Seven people have died in Nigeria, which has counted a total of 22 cases, while one case has been confirmed in Senegal. And at least 30 more people have died in a separate outbreak in the Democratic Republic of Congo.
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Ascend Biopharmaceuticals Provides Update on Clinical Trial Program for Lead Immunotherapy Products

Ascend Biopharmaceuticals, a Melbourne-based immunotherapy company, has released an update on the clinical trial pipeline for its lead immunotherapy products treating basal cell carcinoma (BCC) and breast cancer. The Company plans to begin a Phase 2 clinical trial on ASN-002, an injectable immunotherapy for basal cell carcinoma (BCC), by H1 2015. Interim results from the trial are expected in mid-late 2015. Ascend has also announced plans to begin a Phase 1b study on a therapeutic cancer vaccine for breast cancer, ASN-004, in the second half of 2015. Both trials are dependent on the completion of fundraising.
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