Bio Business July/August 2018

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CANADA NEWS

Investigating proteins as predictive biomarkers 6

NEWSMAKER

Alli Murugesan: bringing new ideas to market faster 15

MOMENTS IN TIME

Samuel Weiss opens our understanding of the brain’s potential 18

JULY/AUGUST 2018

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STEM ECONOMICS 101 A closer look at companies that supply stem cell research

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THE STEM CELL ASSEMBLY LINE A closer look at the companies providing the raw material for stem cell research

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Discovering neural stem cells changed the landscape for understanding how the brain regenerates

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Investigating proteins as predictive biomarkers 6

NEWSMAKER

Alli Murugesan: bringing new ideas to market faster 15

MOMENTS IN TIME

Samuel Weiss opens our understanding of the brain’s potential 18

JULY/AUGUST 2018

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STEM ECONOMICS 101 A closer look at companies that supply stem cell research

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David Suzuki Energy Efficiency & Technology squeeze the carbon bubble

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EDITOR’S NOTE 5 CANADIAN NEWS 6 WORLDWIDE NEWS 7 MOMENTS IN TIME 18

SEEING THE

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PUBLISHER & CEO Christopher J. Forbes cforbes@jesmar.com EXECUTIVE EDITOR Theresa Rogers trogers@jesmar.com ASSISTANT EDITOR Hermione Wilson hwilson@jesmar.com EDITORIAL INTERN

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THE FUTURE IS NOW IN STEM CELL SCIENCE BY HERMIONE WILSON

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Whenever I sit down to write an editorial for Bio Business magazine, I often like to speculate about the future possibilities of the field of research the issue is focusing on. I suppose that is the science fiction fan in me. This time, our focus is on stem cells, but there is nothing fictional or farfetched about the progress being made in this field. When I was a young journalism student in university, I interviewed a man in the advanced stages of multiple sclerosis. He was in his early 50s and living in a nursing home as he was tetraplegic and confined to a wheelchair. Against all odds, though, he was clinging to the hope that stem cell research would one day produce a treatment that would allow him to walk again. His family thought he was crazy, but he was convinced that one day he would be able to leave his wheelchair behind for good. That was in 2009. Almost 10 years later Australian researchers at Monash University have documented the amazing ability of stem cells in zebra fish to repairing severe spinal cord damage. The pictures of the mononeurons regenerating in real time are amazing, but even more amazing is the fact that the damage was repaired in just two days. The study, whose results were recently presented at the International Society for Stem Cell Research conference in Melbourne, offers new insights into the process of cell regeneration that could lead to us learning how to trigger the same rapid regeneration in human stem cells. Perhaps I am jumping the gun a bit here, to draw a line between zebrafish and the gentleman I interviewed, Hermione Wilson waiting for his miracle cure. Or maybe ASSISTANT EDITOR not.

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SECRETARY/TREASURER Susan A. Browne

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CANADIAN NEWS

BREAKTHROUGH DISCOVERY WILL CHANGE TREATMENT FOR COPD PATIENTS

Researchers at the University of British Columbia’s faculty of medicine have shown that permanent lung damage caused by chronic obstructive pulmonary disease (COPD) starts much earlier than previously thought, even before patients are showing symptoms. Dr. Tillie-Louise Hackett and her team found that patients with even mild COPD had a significant loss of their small airways – an average of more than 40 per cent. Lung samples from 34 patients were analyzed by an ultra-high resolution microCT scanner, one of only three in Canada which provide extreme detail by imaging samples that are embedded in paraffin. The findings may lead to new drugs in treating COPD at the earliest stages of the disease.

RESEARCHERS INVESTIGATE PROTEIN AS PREDICTIVE BIOMARKER FOR BREAST CANCER

APPLYING AI IN DEVELOPING NEW THERAPEUTIC ANTIBODIES

Canada’s Centre for Drug Research and Development (CDRD) has partnered with Schrödinger Inc. to more effectively develop highquality humanized antibodies with ideal biophysical characteristics using artificial intelligence and predictive modelling software. Because of the tremendous resources required and high failure rates in drug development, technologies like AI help process data faster and more accurately. Schrödinger will provide its biologics modeling technology and support antibody humanization via structure based computational approaches. The CDRD will provide antibodies for a particular target, as well as key molecular and physicochemical lab measurements. The British Columbia Cancer Agency will provide critical biological data for the initial project involving an immune checkpoint target on cancer stem cells.

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NEW CANNABIS COMPANY COMMITTED TO RESEARCH

The acquisition of MedReleaf Corp. by Edmontonbased Aurora Cannabis Inc. has created a potent new player in the growing domestic and international cannabis markets. The new company will have a funded capacity of more than 570,000 kg of high-quality cannabis per year, to be delivered via nine facilities in Canada and two in Europe. Their combined science and R&D teams, including approximately 40 PhDs and MScs will be active in research projects worldwide. In addition to studies at Aurora, MedReleaf's initiatives include a Pharmacokinetics trial, a Phase II cancer pain trial, a Phase III epilepsy trial, an observational chronic pain study, and a study assessing the correlation between genetic signatures and cannabis efficacy.

Dean Reddick, a graduate student at the University of Winnipeg, is working on a certain protein that can identify drug resistance to breast cancer treatment.

Doctors typically prescribe Tamoxifen at the start of some breast cancer treatments, but the drug is only effective in approximately half of all patients who receive it. Identifying these patients before treatment begins can save valuable time. Now, researchers at the University of Winnipeg are looking at a certain protein in cancer cells which might be able to do just that. “Tamoxifen is a fantastic drug that delivers targeted therapy to ER+ breast cancer, however not all ER+ breast cancer patients respond to it,” said UWinnipeg biology professor Dr. Anuraag Shrivastav, whose lab has been studying the topic for several years. The research has implications in the treatment of a type of breast cancer known as estrogen receptor positive (ER+), which accounts for approximately 70 per cent of all breast cancer cases, and is characterized by estrogen binding to an abnormal number of receptors. Key to solving the issue is a protein within cancer cells known as N-Myristoyltransferase (NMT), which the lab has already discovered activates with increased estrogen receptor activity. “We want to see if these different places where [NMT] is found can actually be acting as a predictive biomarker,” says researcher Dean Reddick, a graduate student in the university’s Master of Science in Bioscience, Technology and Public Policy program. “For instance, if you find all this protein inside the nucleus, are those cells going to be resistant to Tamoxifen or responsive to it?” Since last September, Reddick has produced 14 different variants of breast cancer cells, each with different localizations of the protein. The plan now is to treat each one and monitor their responses. Although the lab still has further testing to do, Reddick believes the protein is an indicator of responsive cells. “We think that [NMT] may be influencing genes that are related to cancer development and actually stopping them from being read and turned into protein. If that’s the case, then that’s going to be good for the patient, because that’s going to slow down cancer growth, period. It could also be slowing down genes that are related to the development of the resistance.” Once the indicators are identified, a simple test could improve treatment odds. Reddick says within three to four years a biopsy could be used to determine a patient’s resistance level and prescribe appropriate treatment.


WORLDWIDE NEWS

AXOL BIOSCIENCE LAUNCHES FIRST HUMAN IPSC-DERIVED ATRIAL CARDIOMYOCYTES

LIFECARE GOES PUBLIC TO FUND NEXT PHASE OF TREATMENT FOR DIABETES EPIDEMIC

Norwegian life-science company Lifecare AS has gone public on the stock exchange in Oslo to continue its advanced development of solutions to the diabetes epidemic. Lifecare researches and develops miniature sensors for convenient glucose monitoring for a global market and expects to gain additional momentum in the fight against diabetes through its public offering. Its Sencell is a long-term implantable glucose sensor that is injected under the skin and can detect changing glucose levels continuously which are communicated wirelessly to a patient’s nearby watch or phone. Clinical trials will be finalized and the company will apply for European approval and develop a working prototype within the next three years.

BIOSCIENCES PUBLISHES POSITIVE DATA FOR DUAL MECHANISM INHIBITOR AGAINST CHRONIC LYMPHOCYTIC LEUKEMIA

BOOSTING STEM CELLS WITH GROWTH FACTOR MAY HELP TREAT CRITICAL LIMB ISCHEMIA

A new stem cell treatment shows promise for patients undergoing hemodialysis who suffer from critical limb ischemia (CLI) according to a study out of Japan’s Shonan Kamakura General Hospital. Results of a phase II clinical trial show that five out of six patients were CLI-free one year after treatment. CLI is caused by severe blockage in leg arteries. It can be treated with bypass surgery, but the outcome is generally poor in patients undergoing hemodialysis or with diabetes. Since stem cells have the potential for vascular regeneration, the researchers applied granulocyte colony stimulating factor (G-CSF), a protein that stimulates bone marrow to produce more stem cells and deliver them to the blood stream.

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Human cell culture specialists at Axol Bioscience, in the UK, recently launched their human stem cell derived atrial cardiomyocytes for the discovery and development of novel treatments of cardiovascular disease. “They are the first and only iPSC-derived atrial cardiomyocyte product currently available to the research market,” says Yichen Shi, Axol’s Chief Executive Officer. "These cells will provide researchers with a quick and easy-to-use platform for studying human atrial muscle cells, creating models of atrial-specific diseases, and later developing assays for identifying disease modifying treatments." Generated from the same donor as Axol’s human iPSC-derived ventricular cardiomyocytes, this physiologically relevant human iPSC-derived cell line is ready in just seven days from thaw for electrophysiology, cardiac contractility study, screening and the investigation of cardiomyopathies. Validated through an industry recognised third party and validated against the Comprehensive in vitro Pro-arrhythmia Assay guidelines, Axol’s human iPSC-derived atrial cardiomyocytes express typical targets, ion channels and currents essential for the screening of cross target effects, the development of atrial specific target drugs, and the investigation of atrial fibrillation and inherited cardiac conditions. The overall potential and application of the human iPSC-derived atrial cardiomyocytes can be enriched when cultured with Axol’s serum-free cardiomyocyte maintenance medium, reducing variability and enhancing consistency in culture. Axol Bioscience provides highly validated primary cells and iPSC-derived cells together with optimised media and growth supplements. Through the supply of differentiated cells derived from healthy donors and patients of specific disease backgrounds, Axol aims to develop the best human cell biology research tools to advance medical research and drug discovery.

Preclinical research by Inflection Biosciences showing that the company’s dual mechanism PIM/PI3 kinase inhibitor IBL-202 has promise as a treatment for chronic lymphocytic leukemia (CLL) has been published in the most recent issue of the peer-reviewed British Journal of Haematology. These published results show that IBL-202 is cytotoxic against CLL cells under in vitro conditions that mimic the hypoxic tumour microenvironment. The publication also demonstrates the significant effects of IBL-202 on CD49d and CXCR4 gene expression and on the migration, cycling and proliferation of CLL cells, suggesting the drug may significantly impair the migratory and proliferative capacity of the leukemic cells.

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The Stem Cell Assembly Line A closer look at the companies providing the raw material needed for stem cell research BY HERMIONE WILSON

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here is an exhibit at MaRS Discovery District in Toronto which commemorates Frederick Banting and Charles Best’s discovery of insulin in 1921 at the University of Toronto. An inscription reads “Insulin: Toronto’s Gift to the World.” “And you know, it was a gift, because we gave it away,” says Andrew Booth, Chief Commercial Officer at STEMCELL Technologies. “Novo Nordisk, which is the largest supplier of insulin and insulin-related products, which is headquartered in Denmark, licensed the technology from U of T for nearly nothing and now does $18 billion in insulin and insulin-related sales around the world annually and employs 45,000 people, and they just took the European distribution rights and Eli Lilly took the North American distribution rights. Just imagine if that pharma company had been in Canada. It would have completely changed the economics of Canada.”

In 1961, James Till and Ernest McCulloch published a study that established a quantitative method to study individual stem cells in adult bone marrow.


New medicines and new therapies have to be proven in humans and in animals. The most similar model to the clinical model is the use of human hepatocytes in research. - Estephan Arredondo, Technical Director

Founded in 1993, STEMCELL Technologies has become one of Canada’s leading providers of cell culture media for research purposes, which includes reagents, recombinant cytokines and other tools for the maintenance of cell cultures, as well as a number of tissue and stem cell types. The company has more than 2,000 products in its catalogue. As in the case of insulin, Canada is also the birthplace of the field of stem cell research. In 1961, James Till and Ernest McCulloch published a study that established a quantitative method to study individual stem cells in adult bone marrow. The researchers had stumbled upon transplantable stem cells while studying radiation sensitivity in mice and that discovery had a far-reaching impact on cancer treatment, and laid the groundwork for regenerative medicine.

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We don’t want to be telling a similar story years from now about how Canada let opportunities in stem cell therapy and regenerative medicine innovation pass us by, Booth says. If you use STEMCELL’s sales as a bellwether for research activity in this area, he says, 60 per cent of the company’s sales are in the U.S. and three per cent are in Canada. “The U.S. is maybe 10 times the size of Canada in terms of GDP, but [it represents] 20 times the activity we see in Canada,” Booth says. “I’d say we’re equally penetrated in terms of our sales channel and our access to market between those two.” The difference, he says, is that the U.S. has outpaced Canada in investment in this area, in academia, as well as in the biotechnology and pharmaceuticals industry. Researchers everywhere are in hot pursuit of stem cell therapies that can treat a number of diseases and in order to do that work, they need providers of human cell and tissue products like STEMCELL Technologies, and their Spanish counterparts at Cytes Biotechnologies. Cytes is a spin-off company created at the University of Barcelona in 2015. The company specializes in organ and tissue procurement, tissue processing and isolation, cryopreservation of human primary cells – with a particular focus on liver cells, or hepatocytes – and the distribution of those cells to researchers, says Technical Director Estephan Arredondo. “If you are able to isolate human hepatocytes, you can develop drug interaction tests,” Arredondo says. “New medicines and new therapies have to be proven in humans and in animals. The most similar model to the clinical model is the use of human hepatocytes in research.” Cytes isolates hepatocytes from liver tissue, freezes the cells and characterizes them, then distributes them to researchers involved in studies about the metabolism and toxicity, among other things, he says. The company is also developing inhouse research in order to increase the viability and quality of the cells it is producing. “The isolation of hepatocytes is a very complex program that is dependent not only on the technology, but on the quality of the tissue and also is dependent on the characteristics of the donor,” Arredondo says. “We are trying to analyze which factors are the donor, which factors are the processes and which factors are the techniques may increase the viability and the characteristics of the cells we want to send to the researchers.” In the future, the Spanish company hopes to be able to provide its customers with cells for clinical use as well as for research purposes. Cytes would eventually like to be able to provide researchers with ready-to-use hepatocytes specific to the disease model that researcher is studying. At present, researchers take healthy cells and infect them with diseases in the lab in order to study disease models. “The idea of our company is, first of all, to produce

Researchers everywhere are in hot pursuit of stem cell therapies that can treat a number of diseases and in order to do that work, they need providers of human cell and tissue products like STEMCELL Technologies.

The isolation of hepatocytes is a very complex program that is dependent not only on the technology, but on the quality of the tissue and also is dependent on the characteristics of the donor. - Estephan Arredondo, Technical Director


high-quality hepatocytes, but then we want to provide to researchers with human hepatocytes, but from patients who are disease associated,” Arredondo explains. “First, we need to understand how to isolate human cells from healthy donors and then, when we have an excellent procedure [in place] we will move to isolate hepatocytes from patients with pathologies such as hepatitis A, hepatitis B, cirrhosis in order to provide researchers specific models for investigations.” Twenty-five years ago, STEMCELL Technologies started by providing a single product for growing hematopoietic stem cells, usually derived from bone marrow. “Since then, research has moved from just needing tools for hematopoietic stem cells to all different types of tissues, whether they are epithelial tissues, or cardiac tissues, and of course the pluripotent stem cells,” says Booth. Breakthroughs in the field, such as James Thomson’s description of the derivation and cultivation of human embryonic stem cells in 1998, and Shinya Yamanaka’s identification of stem cell growth factors in 2006, have pushed the needle on work being done in regenerative and personalized medicines, Booth says. That has had a profound effect on STEMCELL’s customers. “The promise of regenerative medicine has really attracted a lot of funding for our customers and that’s been a real boon, both in biotech and pharma,” he says. Advances in immunotherapy and the use of organoids in clinical research have also influenced the work STEMCELL does, Booth adds. “Every month we’re coming out with a new product that supports a specific tissue growth in organoids culture,” he says. Booth says STEMCELL Technologies is doing its part to make sure Canada is not written out of the future of stem cell technology on a global scale and to build on the momentum Till and McCulloch started more than 50 years ago. He would like to see Canada invest more in promising Canadian startups and Canadian-controlled private corporations in this area, helping them scale up their operations, rather than focusing on attracting multinationals to invest in Canada. “We have a reputation of being able to manufacture media for some of the most difficult to grow cell types,” Booth says. “It’s our hope that we can provide those products to researchers and get carried along in the progress that those researchers make toward the clinic.” STEMCELL would love it, he says, if more of those customers were Canadian. BB

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NEWSMAKER

great idea BRINGING THE NEXT

TO MARKET – AND TO SOCIETY

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BY JANA MANOLAKOS

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NEWSMAKER

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he’s a trail blazer – a passionate inventor and founder of a company with a mission to clear the way for critical drugs to move to market faster. Dr. Alli Murugesan applied a game-changing commercialization strategy to bypass traditional academic barriers and won the Mitacs Global Impact Entrepreneur Award this past June. Together with medical oncologist and University of New Brunswick professor Dr. Tony Reiman (Canadian Cancer Society Chair) and Université de Moncton associate professor Dr. Mohamed Touaibia, she co-invented a patent-filed, nature-inspired chemical compound that targets and kills blood cancer cells, including those that have grown resistant to chemotherapy. Her company, BioHuntress Therapeutics Inc., is now seeking investment and industrial funding to begin animal testing and phase one clinical trials in humans. We spoke to the Mitacs postdoctoral fellow and senior scientist at the University of New Brunswick’s Reiman Cancer Research Laboratory about her work.

You founded BioHuntress Therapeutics Inc. in 2016 and you continue to be heavily engaged in biochemical research. What made you decide to start BioHuntress?

In my academic research career, I have invented some interesting new molecules and have observed new behaviours among known ones. I always wondered what it would take for ideas born in the laboratory to become useful applications for the benefit of society. It was this deep contemplation that compelled me to become a nexus between academia and industry.

What is your company’s vision? BIO BUSINESS J U LY/A U G U S T 2 0 1 8

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The vision of my company is to develop novel, welltolerated drugs for blood cancers and resorptive bone diseases. The long-term vision of BioHuntress is to further establish itself as a sustainable incubator within academia and to aid academic researchers in articulating their value propositions, connecting with key partners and identifying channels to realize a drug. As a fledgling incubator, BioHuntress helps researchers identify ideas that can be commercialized, and helps them cross that last mile by articulating value propositions, bringing key partners and identifying commercialization channels – and in the process reducing the time to market significantly.

You were presented the Mitacs Global Impact Entrepreneur Award in Montreal in June. What did that mean to you?

As a researcher, especially somebody who dreams to invent new things, to even be associated with the words

“global impact entrepreneur” is a big honour and I found that award to be a great inspiration and a humbling moment. Entrepreneurship is not often recognized, so Mitacs is wonderful in showcasing such entrepreneurial researchers. I am very privileged to receive this award.

Why do you suppose entrepreneurship sometimes takes a back seat in academia?

If you look at classical investigative approaches, our goal as researchers is to ask questions; we put our time and energy into research and publishing the findings. Commercialization involves a lot of time and different types of resources and skills. My strategy involves deferring traditional academic publishing in favour of pursuing intellectual property and product development channels first.

Is this a new paradigm in research thinking?

As researchers, let’s ask ourselves before starting a new project, could we approach this differently? Publishing and patenting or commercializing is not mutually exclusive. It is possible to devise a plan for publishing as part of the research findings, and at the same time protect the findings by filing a patent. This allows you to take the commercialization path first, followed by publication of those findings. Both of these could be included in step one of the research project.

The new compound you and two other researchers - Dr. Reiman and Dr. Touaibia invented is referred to as “nature-inspired”. Can you explain what you mean by that?

As scientists we observe, learn and derive inspiration from nature. Natural products from medicinal plants and other natural sources are valuable in identifying new treatments for disease. Systematic biological activity-guided isolation of active principles and obtaining the desired quantity of bioactive components are some of the challenges we face in utilizing these natural products. So what are some other approaches? Can we learn and identify the key scaffolds, the structural backbone from those natural products and synthesize them in the laboratory, as compounds with potent and better effects?

Tell me about the moment when you realized the potency of your invention.

In the preclinical studies we took human blood cancer cells and treated them with these nature-inspired chemical compounds. At different stages we began to see the effect the compounds were having so those are all moments when you get excited. My passion has always been to find a cure – a novel treatment for myeloma bone cancer. It’s a very complicated disease that when you think you have treated it, comes back. To see the ability of our compound to kill the existing chemoresistant cells in these preclinical studies is very exciting for


NEWSMAKER

Time for people is of the essence when faced with devastating diseases. How long before the compound enters the market?

In North America it takes an average of 12 years for an experimental drug to travel from the laboratory to market. For example, only five in 5,000 drugs that enter the preclinical testing stage make it to human testing. Out of those five, maybe one will be approved. So its miles to go before I sleep, but I am very positive and taking an approach where this discovery, through BioHuntress, can drive collaboration between industry and academia to advance the preclinical studies further in animal models which we will complete in two years and then proceed to phase one clinical trials. There is so much energy and drive in advancing this and we really need investors to help us take it further.

You arrived in Canada in 2006. Tell us a bit about how you started in this field.

I came from Pondicherry, India. If you have read the Life of Pi, Pi hales from Pondicherry. It’s a little French colony. I’m a curious adventurer and always wanted to travel everywhere. My parents were headmasters and it was their openmindedness and liberal thinking that allowed me to travel and be myself. I owe everything to them. I did my PhD in

biotechnology and am a chemist by training and later became a biotechnologist. Everytime you acquire new information, go into a new area of research or work on a new disease model, you apply your interdisciplinary knowledge and expertise. For example, I began working on anti-diabetic models – that’s what my PhD was in, and looked for medicinal plants as a cure or treatment for diabetes. When I moved to Canada, I took that knowledge and applied it while exploring natural products and novel treatments. Now, I hope to add value to research teams who are focused on publishing, by offering insights into commercializing. BB

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me. I would be extremely delighted to see similar results in animal models. That’s the next step.

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Discovery of Adult Neural Stem Cells I

n 1992, a Canadian scientist, Dr. Samuel Weiss, from the University of Calgary, discovered neural stem cells in the adult brain, which have the potential to produce new brain cells. Stem cells are undifferentiated cells that divide and specialize in order to build or repair tissues. Adult humans don’t have pluripotent stem cells, which are cells that can differentiate into most cell types, but rather have tissue-specific stem cells that can produce, for example, new blood cells or new skin cells. Weiss’s discovery suggests that the brain may be able to repair itself, a breakthrough in our understanding of the brain’s potential, which has led to further research into brain cell replacement and repair. Neural stem cells may be used to treat degenerative brain disorders, such as multiple sclerosis (MS) or Parkinson’s disease and are a subject of growing interest. BB

BIO BUSINESS

Reference: 1. www.thecanadianencyclopedia.ca/en/article/stem-cell-research/ 2. www.thecanadianencyclopedia.ca/en/article/canada-is-in-the-vanguard-of-stem-cell-research/ 3. http://www.cihr-irsc.gc.ca/e/50677.html 4. https://weisslaboratory.weebly.com/dr-samuel-weiss.html 5. https://science.howstuffworks.com/life/cellular-microscopic/stem-cell3.htm

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