075 | Partnerships, potential cures and pandemics

075 | Partnerships, potential cures and pandemics      Welcome to another episode of Biotechnology Focus radio. This week has brought new partnerships and novel research to light from across Canada.   An international partnership sets its sight on developing a cure for Alzheimer’s disease  A recent study on Huntington’s could aid cognitive decline  Research suggests link between mortality rates of influenza pandemics   And implanting a potential cure for type 1 diabetes   Welcome to another episode of Biotechnology Focus radio. I am your host, Michelle Currie, here to give you a run-down of the top stories of Canada’s biotech scene.    +++++    A deal has been signed between the Krembil Research Institute and French multinational pharmaceutical company, Servier, for the development of a potential disease-modifying drug for Alzheimer’s disease.  This strategic research partnership agreement with Toronto-based company Treventis Corp., a company founded by the director of Krembil, Dr. Donald Weaver, is to co-develop a lasting treatment. Studies are already underway the University Health Network (UHN).  Dr. Donald Weaver stated that “this was a very bid deal. Drug discovery is a tremendously competitive field and this partnership demonstrates the ability of Krembil and UHN to achieve a level of excellence on the world stage. It also helps cement our place as one of the leading neuroscience research facilities in Canada.”  As part of the collaborative agreement, researchers from both countries will be targeting two key proteins that have been identified with memory loss.  Weaver’s team has been working on a therapeutic strategy for near two decades to slow or stop this degenerative disease. Currently, there are no disease-modifying treatments on the market to prevent or stop the progression altogether. Alzheimer’s disease affects over 564,000 people in Canada and is expected to rise to a distressing 937,000 by 2031[1].  In 2013, Treventis was awarded $4.7-million in funding from the prestigious Wellcome Trust to investigate compounds, with the hopes of designing a drug that can safely and effectively treat people with chronic neurological dementias, such as Alzheimer’s. Funding from the Wellcome Trust, a British-based independent charity, is extremely competitive, difficult to obtain, and is traditionally awarded to researchers in the U.K.  Some of the donors and contributors to the advancement of this research are the Brain Campaign, Alzheimer Society of Canada, Canadian Institutes of Health Research, The W. Garfield Weston Foundation, Bright Focus Foundation, and the Krembil Foundation.  As part of the agreement, Servier will fund all research costs and maintain worldwide rights to develop and commercialize drugs advanced during the partnership, while the Toronto researchers set to discover a candidate for a phase I clinical trial.    +++++    A York University study might have found a new treatment option for Huntington’s disease. Christine Till, a psychology professor at the university wanted to know if working memory – a mental workspace that stores information while carrying out a task – training would help early-stage patients with the disease.  In collaboration with the funder, North York General Hospital (NYGH), she led the study where nine patients received working memory training. The results were published in the journal PLoS ONE and detailed how seven of the nine participants completed the training and perceived benefits.  Huntington disease, is a genetic brain disorder characterized by cognitive decline. It is often accompanied by mood changes such as anger, depression, and anxiety. Symptoms usually start when the patient is between 30 and 50 years of age.  The Huntington Study Group estimates that there are between 4-7 individuals per 100,000 diagnosed with Huntington’s in Canada. This number is up for debate largely due to people fearing to take the test because of possible genetic discrimination from health insurers.  This was a pilot, small study aimed at figuring out feasibility, time and costs. It allows researchers to tweak it before running a comprehensive study.  Previous studies of early-to-moderate stage Huntington disease had focused on improving motor functions like gait and balance. Till became encouraged by studies that demonstrated how mice, who were characterized as having genetic mutations mimicking Huntington disease, had benefited from environmental enrichment. These mouse models showed that an enriched environment could enhance motor activity and reduce brain loss.  Inspired by this research, Till began to construct home-based computerized training programs to enhance mental activity in participants with Huntington’s disease.  Participants underwent neuropsychological testing at North York General Hospital or York University twice – once, at the start of the study, and once one week after the completion of the training. This testing was comprised of 90 minutes of questionnaires and neurocognitive assessments.  The training itself involved 25 sessions that were run through the internet and each lasting 40 to 50 minutes. Training was typically completed over a five-week period, five days a week, using the patients’ home computers.  It consisted of exercises that targeted visual-spatial working memory and verbal working memory. Task difficulty was taken into account and adjusted automatically so that the exercises were consistently engaging.  A training coach called the participants weekly to inquire about their experience with the program and to provide motivational support and note any changes in health. Additionally, the training program provided feedback to the participant after each trial was completed. It calculated this by showing comparisons of previous scores and high scores, and providing auditory comments such as “Way to go!” when the participant’s response was correct. Daily performance on the training program was tracked by the computer.  Exit interviews provided powerful insights into the progress of the program and its effectiveness.    +++++    New research out of McMaster University and the Université de Montréal suggests that people who were born during a time of an influenza pandemic are at a higher risk of mortality amidst another pandemic arising. While typically, one develops an immunity from exposure to similar, or sometimes different, influenza A viruses, researchers are expressing that there might need to be exceptions to this rule.  The new data that was collected proposes that people who were born the time of the 1957 H2N2 or Asian Flu pandemic were at a higher risk of dying during the 2009 H1N1 Swine Flu pandemic, as well as the resurgent outbreak in 2013-2014.  The influenza virus has posed a great threat to humanity throughout the years. Its ability to cause widespread pandemics, leaves researchers and scientists scrambling to find a cure. Over the last 100 years, at least five notable pandemics have occurred. This includes the 1918 H1N1 Spanish Flu, the 1957 H2N2 Asian Flu, the 1968 H3N2 “Hong Kong Flu”, the 1977 Russian Flu, and the 2009 H1N1 Swine Flu.  The results align with at least two previous influenza A virus pandemics, in 1918 and 1968, when there were higher death rates among those born during previous pandemic years in 1890 and 1918, respectively.  Matthew Miller, senior author of the paper, and assistant professor of biochemistry and biomedical sciences at McMaster University suggests that this phenomenon is not unique. He believes that exposure to pandemic influenza early in life is a risk factor for mortality during subsequent cross-strain pandemics.  Further research is required to develop appropriate vaccines.     +++++    The University of British Columbia and Vancouver Coastal Health are testing a radical way to cure diabetes. The researchers are implanting pancreatic cells grown in a lab from embryonic stem cells to replace ones previously damaged. The hope is that the implanted cells would mature and multiply to eradicate the reliance on insulin and everyday monitoring of blood sugar via finger pricking.    Dr. David Thompson, a principal investigator in the clinical trial, says that, “If these replacement cells restore a person’s ability to produce their own insulin when needed, it would prevent dangerous episodes of low blood sugar and lessen the complications resulting from high blood sugar, such as blindness, heart attacks and kidney failure.”  The trial could involve about 10 or more people in Vancouver with a severe form of type 1 diabetes, in which a person’s immune system attacks the pancreas, degrading or eliminating its ability to produce insulin.  The team received a grant from the Stem Cell Network of Canada for $500,000 to implement these treatments. Currently, they have only implanted one person, but intend to implant more in the coming weeks. Participants will be followed for two years to see if the implanted cells mature into insulin-producing beta cells and other cells capable of controlling a person’s blood sugar, and whether there are significant side effects.  The implants are part of a larger clinical study by ViaCyte that plans to test the cell-replacement therapy on approximately 40 patients between the US and Canada. ViaCyte has developed a technique for coaxing the embryonic cells along a path to become mature pancreatic cells. The company has also developed for the clinical trial, a protective packet – smaller than a VISA card – that will be implanted just beneath the skin. The packet’s membrane will allow blood vessels to permeate inside so that oxygen and other nutrients will stimulate them to differentiate further. The researchers expect some of the cells will become beta cells, which sense blood sugar levels and release insulin when needed.  As well as these packets, other smaller “sentinel” packets will be implanted and taken out at an earlier stage to evaluate the condition and the development of the cells inside.  With the intention to prevent the participants’’ bodies from rejecting the units, they will be taking immunosuppressants. This, however, makes the patients more susceptible to other infections and is therefore only being clinically tested on people who have a particularly dangerous form of type 1 diabetes.  The procedure for implanting the cells, performed by a team led by Dr. Garth Warnock, a UBC surgery professor, is similar to transplanting clusters of beta cells, known as “islets,” from deceased donors – a treatment pioneered at the University of Alberta. If this clinical study is successful, it could be a promising lead to a cure for type 1 diabetes.    ++++++    Well that wraps up another episode of Biotechnology Focus. For the full stories, please visit the website biotechnologyfocus.ca. From my desk to yours – this is Michelle Currie.