Human Embryonic Stem Cells (hESC) Market Analysis By ... Yahoo Finance NEW YORK, March 29, 2017 /PRNewswire/ -- The global human embryonic stem cells (hESCs) market is anticipated to reach USD 1.06 billion by 2025, ... Global Human Embryonic Stem Cells (HESC) Market 2017- Astellas ... |
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In Brief A Japanese man has become the first recipient of donated, reprogrammed stem cells as a treatment for macular degeneration. If the treatment proves effective against the age-related eye condition, it could halt or prevent the vision loss of the 10 million people in the U.S. who have macular degeneration. A New Treatment for Macular Degeneration
Macular degeneration is the leading cause of progressive vision loss with almost 10million Americans affected by the disease. Currently, there are no known cures for the conditionalthough stem cells might change that entirely.
Macular degeneration occurs when the central portion, the macula, of the retina is deteriorated. This is where our eyes record images and send them to the brain through the optic nerve. The macula is known for focusing our vision, controlling our ability to read, recognize faces, and see objects clearly.
A Japaneseman in his sixties is the worlds first person to receive induced pluripotent stem (iPS) cells donated by a different individual. Rather than tip-toeing around the ethics of embryonic stem cells, scientists were able to remove mature cells from a donor and reprogram them into an embryonic state, which then could be developed into a specific cell-type to treat the disease. Physicians cultivated donated skin cells that were transplanted onto the mans retina to halt the progression of his age-related macular degeneration.
While the mans first surgery was a success, the doctors have said they will make no more announcements about his progress until they have completed all five of the planned procedures. While the effectiveness of this technique cannot be evaluated until the fate of the donated cells and the progression of the patientsmacular degenerationhave been fully monitored, there is increasing interest inusing iPScells for theraputic purposes.
A similar therapy was performed at the Kobe City Medical Center General Hospital in Japan in September 2014, but with a slight difference. In this case, the patient received her own skin cells reprogrammed into retinal cells. As hoped, a year after the surgery her vision had no decline, seemingly halting the macular degeneration. Four more patients in the clinical trial are expected to receive donor cells as well.
The donor-cell procedure, if successful, could help pave the way for the iPS cell bank thatShinya Yamanaka is establishing. An iPS cell bank would allow physicians find theperfect iPS donor per each patients biological signatures. Yamanaka is a Nobel-prizewinning scientist at Kyoto University who pioneered the iPS cells.
Yamanakas idea of a iPS cell bank has the potential torevolutionize modern medicine. It would provide patients with ready-made cells immediately, givinga widespread population access to more treatment options bylower all-around costs. While the risk of genetic defects or a poor donor match still remains, the new procedurecould offer enormous advantagescompared toother alternatives.
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A Japanese Man Has Become the First Recipient of Donated, Reprogrammed Stem Cells - Futurism
March 30, 2017
Researchers at Case Western Reserve University School of Medicine have successfully grown stem cells from children with a devastating neurological disease to help explain how different genetic backgrounds can cause common symptoms. The work sheds light on how certain brain disorders develop, and provides a framework for developing and testing new therapeutics. Medications that appear promising when exposed to the new cells could be precisely tailored to individual patients based on their genetic background.
In the new study, published in The American Journal of Human Genetics, researchers used stem cells in their laboratory to simultaneously model different genetic scenarios that underlie neurologic disease. They identified individual and shared defects in the cells that could inform treatment efforts.
The researchers developed programmable stem cells, called induced pluripotent stem cells, from 12 children with various forms of Pelizaeus-Merzbacher Disease, or PMD. The rare but often fatal genetic disease can be caused by one of hundreds of mutations in a gene critical to the proper production of nerve cell insulation, or myelin. Some children with PMD have missing, partial, duplicate, or even triplicate copies of this gene, while others have only a small mutation. With so many potential causes, researchers have been in desperate need of a way to accurately and efficiently model genetic diseases like PMD in human cells.
By recapitulating multiple stages of the disease in their laboratory, the researchers established a broad platform for testing new therapeutics at the molecular and cellular level. They were also able to link defects in brain cell function to patient genetics.
"Stem cell technology allowed us to grow cells that make myelin in the laboratory directly from individual PMD patients. By studying a wide spectrum of patients, we found that there are distinct patient subgroups.
This suggests that individual PMD patients may require different clinical treatment approaches," said Paul Tesar, PhD, study lead, Dr. Donald and Ruth Weber Goodman Professor of Innovative Therapeutics, and Associate Professor of Genetics and Genome Sciences at Case Western Reserve University School of Medicine.
The researchers watched in real-time as the patients' stem cells matured in the laboratory. "We leveraged the ability to access patient-specific brain cells to understand why these cells are dysfunctional. We found that a subset of patients exhibited an overt dysfunction in certain cellular stress pathways," said Zachary Nevin, first author of the study and MD/PhD student at Case Western Reserve University School of Medicine. "We used the cells to create a screening platform that can test medications for the ability to restore cell function and myelin. Encouragingly, we identified molecules that could reverse some of the deficits." The promising finding provides proof-of-concept that medications that mend a patient's cells in the laboratory could be advanced to clinical testing in the future.
The stem cell platform could also help other researchers study and classify genetic diseases with varied causes, particularly other neurologic disorders. Said Tesar, "Neurological conditions present a unique challenge, since the disease-causing cells are locked away in patients' brains and inaccessible to study. With these new patient-derived stem cells, we can now model disease symptoms in the laboratory and begin to understand ways to reverse them."
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Stem cells help explain varied genetics behind rare neurologic disease - Medical Xpress
Dr. Lauralyn McIntyre is a Senior Scientist in the Clinical Epidemiology Program and a physician in Critical Care at The Ottawa Hospital. She is also an Associate Professor in the Department of Medicine at the University of Ottawa. One main area of interest ties all of these together: septic shock.
Although septic shock is a serious medical condition that has been portrayed on countless medical dramas, it isnt widely understood. Sepsis refers to a bodys response to a serious infection and can manifest itself in many different ways. The new definition refers to a serious infection and at least one dysfunctional organ. This could be your brain, lungs, kidneys, or bone marrow and it could happen to anyone especially individuals with chronic diseases or suppressed immune systems. If symptoms include low blood pressure, its called septic shock and carries with it a high mortality rate and risk of long-term complications for those who survive.
Over time, the medical community has made some improvement in patient outcomes with better identification, resuscitation, and antibiotics. Survivors, however, are often faced with serious long-term issues including psychological and post-traumatic distress, physical weakness, and depression.
It goes on and on, describes Dr. McIntyre. It has a huge burden of illness, both from the perspective of death and even for the people who survive it.
"The type of stem cells that were studying for the treatment of septic shock is completely novel and highly experimental, and has great potential to help patients."
Dr. Lauralyn Mcintyre, The Ottawa Hospital
In Canada, approximately 50,000 patients every year are admitted to hospitals with sepsis or septic shock and 20 to 40 per cent of those patients will die. Dr. McIntyre estimates that sepsis and septic shock account for about 20 per cent of admissions to the ICU at The Ottawa Hospital.
Sepsis and septic shock represent an enormous challenge to patients, to the health care system, and to the caregivers who look after patients after they leave the hospital.
Dr. McIntyre and her colleague Dr. Duncan Stewart, saw a big challenge and lots of room for potential. And thats where stem cells come in. They have been part of a team of specialists whove spent the last five or six years working towards conducting a phase one trial to treat septic shock using stem cells.
The type of stem cells that were studying for the treatment of septic shock is completely novel and highly experimental, and has great potential to help patients, says Dr. McIntyre.
Early research in animal models showed stem cells reduce death in animals with sepsis by calmingthe immune system, restoring organ function and clearing the pathogens faster from the system.
In the phase one trial, medical staff tested stem cell treatment in a small subset of patients with septic shock. They were looking at the tolerability of cells and trying to determine the best dose to use in a subsequent randomized control trial.
That first phase was completed in June and it was a success. It was the first clinical trial of its kind in the world.
The cells were very well tolerated and that has given our team the impetus, the rationale, and the motivation, to go forward to a much larger study, which will be a randomized study, says Dr. McIntyre.
The cells at the centre of the study are stem cells that come from the bone marrow of healthy volunteers who donated their marrow for this clinical trial.
When researchers originally isolated stem cells, they thought they supported blood and the bone marrow. Scientists eventually discovered these cells have a great ability to modulate inflammation. This is a very key process central to septic shock, describes Dr. McIntyre. Its an inflammatory cascade gone awry.
We used to think the cells facilitate their actions by grafting, through implanting themselves in wounded tissue or into the organs that are not working say in sepsis, says Dr. McIntyre. As it turns out they dont make themselves at home, they just seem to cross talk to host cells including cells that are responsible for killing pathogens to regulate them and restore homeostasis. They are like the traffic cops of their microscopic world, and whats more, within a few days they are gone from the system.
This is a critical area of research, not only because of the numbers of patients affected but the trickle down effect that results, such as days lost at work and extra labour for caregivers. Septic shock also comes with a hefty price tag. The estimated cost to the Canadian health care system is $4 billion.
I think its important because theres so much potential to help, says Dr. McIntyre.
The results of the phase one trial will be published within the next few months and funding applications are already being made for the next phase of the trial. If the funding falls into place, the team hopes to begin the next phase by the middle of next year, and take one step closer to a treatment that may help thousands of people across Canada, and the world beyond.
This research was supported by the Canadian Institutes of Health Research, the Ontario Institute for Regenerative Medicine, the Stem Cell Network and The Ottawa Hospital Foundation
To find out more on how you can support stem cell research at The Ottawa Hospital visitohfoundation.ca
This is part of an ongoing series about The Ottawa Hospital. Look for the evolving archive on obj.ca.
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Real Life Medical Drama Starring the "Traffic Cop" of Cells - Ottawa Business Journal
ZURICH Novartis AG on Wednesday said the U.S. Food and Drug Administration (FDA) has agreed to accelerate its review of the Swiss drugmaker's CTL019 therapy for young patients with B-cell acute lymphoblastic leukemia.
The move would keep Novartis on track with the development of its so-called chimeric antigen receptor T cell therapy, or CAR-T, in partnership with University of Pennsylvania researchers.
ZARA FOUNDER TO SPEND $344M ON BREAST CANCER-SCREENING FOR SPANISH HOSPITALS
The therapy involves a patient's own T-cells being altered in the lab to help the immune system find and kill cancer cells before being re-infused into the patient.
Basel-based Novartis' first CAR-T therapy license application with the FDA has put the company in pole position with regulators as it pushes for approval alongside rivals including biotech Kite Pharma Inc that are developing similar therapies.
"With CTL019, Novartis is at the forefront of the science and development of immunocellular therapy as a potential new innovative approach to treating certain cancers where there are limited options," Vas Narasimhan, Novartis head of drug development, said in a statement.
CTL019 will likely cost hundreds of thousands of dollars per patient if approved, and Novartis counts it among drugs it believes will eventually exceed $1 billion in annual sales.
DEVON AND LEAH STILL CELEBRATE TWO YEARS IN REMISSION
In a Phase II study, Novartis said 82 percent of patients infused with CAR-T cells achieved complete remission or complete remission with incomplete blood count recovery at three months after treatment. In December, Novartis estimated that 60 percent of those responders were relapse-free after six months.
The company plans to submit an application for market authorization with the European Medicines Agency (EMA) later this year.
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Novartis says US regulator grants speedy review of CAR-T cell therapy - Fox News
ViaCyte, which is working on stem cell-based therapies for diabetes, has partnered with W.L. Gore to develop a delivery device for cell therapies that will protect the cells from being rejected by patients immune systems.
ViaCyte is developing a treatment that deliversstem cell-derived islet replacement therapies for patients with Type 1 diabetes and some patients with Type 2 diabetes, according to a statement.
The PEC-Encap product combines a delivery device, the Encaptra Cell Delivery System, with PEC-01 pancreatic progenitor cells. The Encaptra system delivers the PEC-01 stem cellswhich have the ability to grow into insulin-producing pancreatic cellsand shields them from the host immune response, which removes the need forimmunosuppressive drugs.
While the combo treatment has shown promise in early, preclinical evaluation, nonclinical results have shown that modifying the Encaptra device could improve PEC-Encap, ViaCyte said in the statement. The engraftment of the stem cells could also be improved.
As ViaCyte advances our next generation encapsulation technologies for cell therapies, Gores contribution to the material and design improvements of the Encaptra delivery system is expected to support the reliable and robust long-term engraftment that is required for the PEC-Encap product to be most effective, said ViaCyte CEO Paul Laikind, in the statement. With Gores help, we plan to improve on the results we have seen with PEC-Encap, which would then have the potential of benefiting all patients with insulin-requiring diabetes, both type 1 and type 2.
ViaCyte and Gore first looked into collaborating in 2016, said Edward Gunzel, technical leader for Gore PharmBIO Products, in the statement. The companies have created a joint development team. Financial details were not disclosed.
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ViaCyte taps Gore to develop delivery devices for diabetes cell ... - FierceBiotech
FINDINGS
UCLA researchers have developed a stem cell gene therapy cure for babies born with adenosine deaminase-deficient severe combined immunodeficiency, a rare and life-threatening condition that can be fatal within the first year of life if left untreated.
In a phase 2 clinical trial led by Dr. Donald Kohn of theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Researchat UCLA, all nine babies were cured. A 10th trial participant was a teenager at the time of treatment and showed no signs of immune system recovery. Kohns treatment method, a stem cell gene therapy that safely restores immune systems in babies with the immunodeficiency using the childs own cells, has cured 30 out of 30 babies during the course of several clinical trials.
Adenosine deaminase-deficient severe combined immunodeficiency, also known as ADA-SCID or bubble baby disease, is caused by a genetic mutation that results in the lack of the adenosine deaminase enzyme, which is an important component of the immune system. Without the enzyme, immune cells are not able to fight infections. Children with the disease must remain isolated in clean and germ-free environments to avoid exposure to viruses and bacteria; even a minor cold could prove fatal.
Currently, there are two commonly used treatment options for children with ADA-SCID. They can be injected twice a week with the adenosine deaminase enzyme a lifelong process that is very expensive and often does not return the immune system to optimal levels. Some children can receive a bone marrow transplant from a matched donor, such as a sibling, but bone marrow matches are rare and can result in the recipients body rejecting the transplanted cells.
The researchers used a strategy that corrects the ADA-SCID mutation by genetically modifying each patients own blood-forming stem cells, which can create all blood cell types. In the trial, blood stem cells removed from each childs bone marrow were corrected in the lab through insertion of the gene responsible for making the adenosine deaminase enzyme. Each child then received a transplant of their own corrected blood stem cells.
The clinical trial ran from 2009 to 2012 and treated 10 children with ADA-SCID and no available matched bone marrow donor. Three children were treated at the National Institutes of Health and seven were treated at UCLA. No children in the trial experienced complications from the treatment. Nine out of ten were babies and they all now have good immune system function and no longer need to be isolated. They are able to live normal lives, play outside, go to school, receive immunizations and, most importantly, heal from common sicknesses such as the cold or an ear infection. The teenager, who was not cured, continues to receive enzyme therapy.
The fact that the nine babies were cured and the teenager was not indicates that the gene therapy for ADA-SCID works best in the youngest patients, before their bodies lose the ability to restore the immune system.
The next step is to seek approval from the Food and Drug Administration for the gene therapy in the hopes that all children with ADA-SCID will be able to benefit from the treatment. Kohn, who is a professor of pediatrics and microbiology, immunology and molecular genetics at the David Geffen School of Medicine at UCLA, and colleagues have also adapted the stem cell gene therapy approach to treat sickle cell disease and X-linked chronic granulomatous disease, an immunodeficiency disorder commonly referred to as X-linked CGD. Clinical trials providing stem cell gene therapy treatments for both diseases are currently ongoing.
Kohn is a member of the UCLA Jonsson Comprehensive Cancer Centerand member of the UCLAChildrens Discovery and Innovation Institute at Mattel Childrens Hospital. The first author of the study is Kit Shaw, director of gene therapy clinical trials at UCLA.
The research was published in the Journal of Clinical Investigation.
The research was funded by grants from the U.S. Food and Drug Administrations Orphan Products Clinical Trials Grants Program (RO1 FD003005), the National Heart, Lung and Blood Institute(PO1 HL73104 and Z01 HG000122), the California Institute for Regenerative Medicine (CL1-00505-1.2 and FA1-00613-1), the UCLA Clinical and Translational Science Institute (UL1RR033176 and UL1TR000124) and the UCLA Broad Stem Cell Research Center.
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Pioneering stem cell gene therapy cures infants with bubble baby disease - UCLA Newsroom
A woman will hold a stem cell registration drive in Livingston after treatment saved her brother-in-laws life when he was diagnosed with leukaemia.
Claire Craig will host the drive for charity DKMS two years after her brother-in-law Jamie, from Livingston, received life-saving stem cell treatment thanks to the help of a donor from Germany.
Jamie was diagnosed with Acute Myeloid Leukaemia in August 2014 aged 31 and was given only an 18 per cent chance of survival unless a stem cell donor match was found.
With none of his five siblings proving to be a match, Jamie finally received good news in October 2014 that a female donor had been found in Germany, by February 2015 Jamie was in remission.
Read more: Woman celebrates her 107th birthday West Lothian
Inspired by her brother-in-laws story Claire has decided to hold a drive for the stem cell charity at the Salvation Army hall in Howden, Livingston, on June 1, with 10-minute slots available from 11am to 6 pm.
Claire, who now lives in Berwick, said: I have decided to hold this event, hopefully to raise awareness and get as many people as I can to sign up to be on the register.
To join the register all we need is a simple, painless swab of the inside of both cheeks, along with some basic contact details. This will take less than ten minutes but could give someone a second chance of life.
This is the second drive Claire has held after hosting one in Berwick last month and she hopes to encourage as many 17 to 55-year-olds as possible to sign up for the register.
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Lisa Nugent, head of donor recruitment at blood cancer charity DKMS, said: Were grateful to Claire for hosting the donor drive event in Livingston and call on anyone aged between 17 and 55 years and in general good health to register at the event or by visiting http://www.dkms.org.uk.
She added: Every 20 minutes someone in the UK is diagnosed with a blood cancer. Finding a blood stem cell match from a genetically similar person can offer the best treatment, a second chance of life. Thats why DKMS exists to register people as a blood stem cell donor and become a potential life-saver it costs just 40 to register a new donor.
Anyone who wants to register can visit http://www.facebook.com/dkmssignuplivingston or contact Claire on 07826 516504.
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Woman to hold West Lothian stem cell drive after treatment saved brother-in-law's life - Scottish Daily Record
Laijun Lai, an Associate Research Professor at the University of Connecticut is currently doing research to find treatment for cancers, autoimmune deficiencies and genetic diseases through the use of T cells and stem cells.
The first area of his research focuses on gene engineering approaches to treating cancers and autoimmune diseases.
Lais research has resulted in two patents and several grants from NIH, the American Cancer Society and the Connecticut Regenerative Medicine Fund.
We are very excited with our results. We believe that our research will eventually lead to the new approach in the treatment of cancer, autoimmune diseases or genetic diseases, such as (DGS), Lai said.
The progression of cancerous tumors is accompanied by a very significant suppression of the immune system, which interferes with the bodys ability to send an effective immune response in order to eliminate chemotherapy, Lai said.
In terms of autoimmune disease, disorders develop when the immune system targets and destroys the bodys own tissues, Lai said.
Therefore, the study investigates new approaches to enhance T cell function for use in the treatment of cancer, while also looking for new ways to inhibit T cell function to treat autoimmune disease, Lai said.
Several T cell inhibitor molecules have been identified. Were trying to find a new T cell inhibitor molecule by using the bioinformatic approach to identify several new genes that are related to T cell inhibitor molecules, Lai said.
He then used the gene engineering approach to produce recombinant proteins from these genes. And these preliminary studies have shown that in a dish, the proteins can inhibit T cell function.
By using the gene engineering approach we can enhance the immune function that can fight a lot of diseases, such as cancer and infections, Lai said.
The second area of my research is using stem cell technology to prevent and treat autoimmune disease, Lai said.
The thymus, an organ of the immune system, is the primary organ that naturally produces T cells for the body.
Thymic epithelial cells (TECs) mediate T cell selections, generating T cells that are able to react with foreign antigens, such as bacteria and viruses, Lai said.
In the prevention or treatment of autoimmune diseases, it would induce immune tolerance of certain antigens by using the mechanisms that would occur in the thymus under normal circumstances, Lai said.
However, the thymus undergoes age-dependent involution resulting in a serious compromise of T cell function in the elderly, Lai said. Many studies have shown that embryonic stem cells (ESCs) or pluripotent stem cells (iPSCs) have huge potential to treat many diseases because these cells can change into many types of cells in a dish.
Through the transplantation of ESCs can cause immune tolerance to the disease causative self-antigens and treat or even prevent autoimmune diseases such as Multiple Sclerosis, Lai wrote in a research statement.
The third area of my research is using stem cell technology to model and treat genetic diseases such as DiGeorge Syndromealso known as DGS, Lai said.
DGS is one of the most common genetic diseases in humans.
One of the characteristic features of DGS is that the patient has a profound thymic aplasia or hypoplasia that results in T cell immunodeficiency, Lai said, So we are going to determine the ability of ESC-derived TECs to prevent and treat DGS.
KAMLOOPS My curiosity was sparked when I read that a stem cell centre was opening in Kamloops (Kamloops This Week, March 21, 2017).
So I went to the location of the centre at 470 Columbia St only to find a parking lot. Thinking that the address might be wrong, I searched the directory of the medical building next door and found that no stem cell centre was listed.
The Stem Cell Centers website lists Kamloops as the only one in Canada. Dr. Richard Brownlee is named as the surgeon with more information coming soon.
Stem cell therapy, says the website, can help with orthopedic or pain management, ophthalmological conditions, cardiac or pulmonary conditions, neurological conditions, and auto-immune diseases, among many other conditions and disease that results in damaged tissue.
One of the ophthalmological conditions they treat is macular degeneration. If your vision is fading due to macular degeneration, you know its time to seek help. Our non-invasive Stem Cell Therapy treatment might be the solution for you.
I wanted get Dr. Brownlees reaction to news that an unproven stem cell treatment had resulted in blindness according to the New England Journal of Medicine as reported in the Globe and Mail, March 20, 2017.
This week, the New England Journal of Medicine (NEJM) reported on three individuals who went blind after receiving an unproven stem cell treatment at a Florida clinic. The patients paid thousands of dollars for what they thought was a clinical trial on the use of stem cells to treat macular degeneration.
The writer of the Globe and Mail article, Timothy Caulfield, Research Chair of the in Health Law and Policy at the University of Alberta, doesnt name the Florida clinic.
The Stem Cell Centers website refers optimistically to treatment for macular degeneration at a Florida clinic, although apparently not theirs since no Florida clinic appears on their list. It tells of how Doug Oliver suffered from macular degeneration before stem cells were extracted from his hip bone and injected them into his eyes. Almost immediately, Olivers eyesight started to improve. I began weeping, he said.
Caulfield encourages caution. Health science gets a lot of attention in the popular press. People love hearing about breakthroughs, paradigm shifts and emerging cures. The problem is, these stories are almost always misleading. It can also help to legitimize the marketing of unproven therapies.
Reports from the Stem Cell Centers own website are cautionary as well. It quotes an abstract from a study done by the Southern California College of Optometry on how stem cells might ultimately be used to restore the entire visual pathway.
The promise of stem cell research is phenomenal. Scientific American (Jan., 2017) reports that brains can be grown in a lab dish from stem cells taken from skin. These samples can be used to research brain disorders ranging from schizophrenia to Alzheimer's disease, and to explore why only some babies develop brain-shrinking microcephaly after exposure to the Zika virus.
However, Dr. George Daley, dean of Harvard Medical School, concludes that there are only a handful of clinical applications available and they are for skin and blood-related ailments.
Practice, it seems, has not yet matched the promise of stem cell research.
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Stem cell centre coming to Kamloops? - CFJC Today Kamloops