Clinical trials

03
Nov 2015
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(Lisa Willemse photo for OIRM) Stem cell co-discover Jim Till and award winner Tim Keiffer

On the verge of new treatments that will change lives

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We are at a crucial moment in time for stem cell research and development in Canada.

For almost two decades the field of stem cells and regenerative medicine “has been long on promise, short on product,” according to a feature article in the current issue Biotechnology Focus magazine.…

We are at a crucial moment in time for stem cell research and development in Canada.

For almost two decades the field of stem cells and regenerative medicine “has been long on promise, short on product,” according to a feature article in the current issue Biotechnology Focus magazine.

But that is changing quickly, the article points out.  In clinical trials underway across Canada, researchers are using stem cells to treat diabetes, heart attacks, osteoarthritis and spinal cord injury to name just a few. There is a growing feeling that the field is on the verge of delivering new treatments that will change the lives of patients suffering from chronic, debilitating diseases.

That sentiment was also on display at last week’s Till & McCulloch Meetings in Toronto. The Canadian Stem Cell Foundation is a partner  with the retiring Stem Cell Network, the Canadian Centre for Regenerative Medicine and the Ontario Institute for Regenerative Medicine, who co-host the premier stem cell event in Canada. It brings together some 400 scientists, clinicians and industry leaders to share insights into how to move the field forward.

Chosen as the 2015 Till & McCulloch Award Winner, Dr. Timothy Kieffer of the University of British Columbia gave the keynote lecture at the closing session and shared his optimism at the progress his lab and others are making towards defeating diabetes.

Drawing from a paper published in Nature Biotechnology last year, he described how his team reversed diabetes in mice using insulin-producing cells derived from human stem cells.  Looking forward to moving his work into clinical trials, Dr. Kieffer says it’s just a matter of time before stem cells provide the needed source of cells to replace insulin injections, sparing millions of diabetics of the need for needles and rigorously monitoring their blood sugar levels several times a day.  He predicts this will happen within 10 years.

In short, good things are happening. Obstacles are being overcome. Cures are on their way. The horizon is getting closer and we can see the possibilities more clearly now.

 

 

 

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27
Jul 2015
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Dr. Duncan Stewart (Ottawa Citizen)

‘It would be a shame for us to slide back’

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Canada is losing ground in the field it founded, says Dr. Duncan Stewart, one of the world’s leading stem cell scientists.…

Canada is losing ground in the field it founded, says Dr. Duncan Stewart, one of the world’s leading stem cell scientists.

“Generally, the pace (of funding) is slower and I think we are losing ground compared to other jurisdictions,” Dr. Stewart told the Ottawa Citizen’s Elizabeth Payne. “Canada has been a leader in this area. It would be a shame if we were to slide back.”

Dr. Stewart, Executive Vice-President of Research at The Ottawa Hospital,  recently published results of the world’s first clinical trial of a genetically enhanced stem cell treatment for pulmonary arterial hypertension, a deadly disease for which there is no cure. The  results are promising, but a larger study is needed to see if the new therapy can produce long-term results. The money to do it, however, just isn’t there.

As the Citizen article points out: “Just as the promise of potential new stem cell therapies is blossoming, research funding is more uncertain than ever.”

The reality is Canadian researchers are poised to deliver new cures for devastating conditions like diabetes, heart disease, cancer, autoimmune disorders like MS and Crohn’s, and neurological conditions like Parkinson’s, stroke and spinal cord injury. But it will take a coordinated effort to make that happen.

Our Foundation leads the coalition of researchers, medical professionals, health charities, industry leaders and philanthropists behind the Canadian Stem Cell Strategy & Action Plan. A bold private/public partnership, it would see Canada lead the way in delivering up to 10 new curative therapies within 10 years.

 

 

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14
May 2015
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Vision loss trial cleared in California

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Millions of people in North America live with varying degrees of irreversible vision loss. Some good news is that a novel stem cell therapy for retinitis pigmentosa (RP), an inherited condition that slowly damages the retina and can result in blindness, has been cleared by the U.S.…

Millions of people in North America live with varying degrees of irreversible vision loss. Some good news is that a novel stem cell therapy for retinitis pigmentosa (RP), an inherited condition that slowly damages the retina and can result in blindness, has been cleared by the U.S. Food and Drug Administration (FDA) to start a Phase I clinical trial.

The therapy for RP, which affects 1 in 3500 Canadians, was developed by Dr. Henry Klassen, Associate Professor at the Gavin Herbert Eye Institute, UC Irvine School of Medicine, and will consist of injecting patients with stem cells to help replace the cells destroyed by the disease and to save those cells that are not damaged yet.

The goal of the trial, which will involve up to 16 patients, is to evaluate the safety and identify side effects of the treatment. Although it is early to speak about a cure for RP, the researchers are hopeful.

“This milestone is a very important one for our project. It signals a turning point, marking the beginning of the clinical phase of development, and we are all very excited about this project.” said Dr. Klassen in the CIRM press release.

The trial has received almost $20 million in funds from the California Institute for Regenerative Medicine (CIRM).

“One of the goals of the agency is to provide the support that promising therapies need to progress and ultimately to get into clinical trials in patients. RP affects about 1.5 million people worldwide and is the leading cause of inherited blindness in the developed world. Having an effective treatment for it would transform people’s lives in extraordinary ways.” Jonathan Thomas, Ph.D., J.D., Chair of the Governing Board of the CIRM, said in the CIRM press release.

Click here to discover more about how stem cells are being used to understand and treat eye diseases on our Toward Treatments page.

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13
May 2015
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The state of regenerative medicine industry: ARM report

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The Alliance for Regenerative Medicine (ARM), a global advocacy organization active since 2009, has recently published the ARM Quarterly Data Report on the state of regenerative medicine industry.…

The Alliance for Regenerative Medicine (ARM), a global advocacy organization active since 2009, has recently published the ARM Quarterly Data Report on the state of regenerative medicine industry.

The report includes analysis of trends and metrics provided by 580 therapeutic companies worldwide. More than 300 of these companies are located in North America.

“This sector has come off a strong year in 2014 and 2015 is off to an even stronger start. As 2015 continues to unfold, we anticipate more high-value deals, and increased investor and public interest in what advanced therapies can offer.” Patricia Reilly and Nancy Dvorin from Informa Business Intelligence, Pharma and Healthcare, ARM’S data partner, wrote in the report.

Higher investments in advanced therapies are in fact among the major trends of the first quarter of 2015. Currently, 486 clinical trials (phase I, II or III) are underway. More than 1/3 of current clinical trials are in oncology, and more than 1/10 are in the cardiovascular area.

Source: Alliance for Regenerative Medicine

Current Clinical Trials by Therapeutic Category. Source: ARM Quarterly Data Report

Additional ARM reports, released at the end of each quarter of the year, will include updates to the current information. To view and download the Q1 Report click here.

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21
Jan 2015
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Toronto MDs ‘put skin in the game’ to try to cure arthritis

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Physicians at Toronto’s University Health Network have tapped their own savings to start the first North American stem cell trial for osteoarthritis.…

Physicians at Toronto’s University Health Network have tapped their own savings to start the first North American stem cell trial for osteoarthritis.

The trial, part of the UHN’s Arthritis Program, will involve 12 patients between the ages of 40 and 65 with moderate to severe osteoarthritis in their knees. Bone marrow extracted from the back of their hips will be used as a source of mesenchymal stromal cells, which UHN scientists will grow in an incubator for four to six weeks and inject into the patients’ knees.

The ultimate goal is to cure arthritis, which affects about 4.6 million Canadians and has a huge impact on the Canadian economy in terms of health care costs. Osteoarthritis is one of the most common forms of arthritis, which can affect people of any age and ethnicity. Visit our Toward Treatments page to learn more about the disease and the stem cell research now underway to combat it.

To kick-start the network’s trial, 10 orthopedic surgeons at UHN’s Toronto Western site donated a total of $1.25-million of their own money over five years.

“We felt very strongly that we had to have our own commitment beyond just the time and effort we would all have to put in,” Dr. Nizar Mahomed, director of the Arthritis Program and one of the surgeons who donated $125,000 over five, years told Kelly Grant of the Globe and Mail. “We needed to make a commitment of actual dollars and put skin in the game.”

The doctors’ decision to start the funding campaign, which has now raised around $38 million thanks to other private gifts, was driven by the fact that landing Canadian Institutes of Health Research (CIHR) public grant funding has become an increasingly competitive process. As reported in the Globe and Mail, the success rate for applications to CIHR fell to 18% for 2014-2015, down from 33% less than a decade earlier.

But the philanthropic initiative is just the initial step. “The goal here is not to rely purely on philanthropic funding for the research program, but to get pilot data to then be able to prove to CIHR that what we’re proposing is going to be effective,” Dr. Mahomed told the Globe and Mail.

The UHN initiative illustrates a creative solution to a serious problem. While Canada is a world leader in stem cells, Canadian researchers often face challenges translating their discoveries into new therapies and treatments that can be tested through clinical trials. To pave the way for that to happen, the Canadian Stem Cell Strategy & Action Plan, developed by a coalition of researchers, medical professionals, health charities and business leaders, calls for a $1.5-billion private/public investment in stem cell research and development over 10 years. Its goal is for Canada to lead the way in delivering five to 10 safe and effective treatments for chronic diseases within 10 years.

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03
Nov 2014
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First patient gets cell-based treatment for diabetes

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In a recent blog post, we reported a prediction by the University of British Columbia’s Dr. Tim Kieffer that it is “only a matter of time” before stem cells provide the needed source of cells to replace insulin injections.…

CIRM Screen Capture

In a recent blog post, we reported a prediction by the University of British Columbia’s Dr. Tim Kieffer that it is “only a matter of time” before stem cells provide the needed source of cells to replace insulin injections. He’s confident this will occur within 10 years.

One way it could happen is now being tested in a clinical trial at the University of California, San Diego Health System, where an American company called ViaCyte has successfully implanted its first patient with a device to treat type 1 diabetes.  Not only does ViaCyte have a Canadian connection, the exciting news from California underscores the importance of Canada implementing the Canadian Stem Cell Strategy & Action Plan.

The California Institute of Regenerative Medicine (CIRM), which has supported ViaCyte in its work, blogged about the VC-01, describing it as “about the length and thickness of a credit card but only half as wide.” Implanted under the skin, the device’s progenitor cells secrete insulin whenever they detect that blood sugar is high, restoring glycemic harmony. While it can move out the insulin as needed, the device stops the immune system from moving in and destroying the insulin-producing cells.

These are, of course, early days: the primary object of the trial is to see if the device is safe, if patients can tolerate it with no adverse effects. And if they can, does it, in fact, treat their diabetes?

That said, CIRM’s leaders are understandably chuffed. The news “that this is now truly out of the lab and being tested in patients is an important step in a long road to showing that it works in patients.”  They urge cautious optimism: “The people at ViaCyte, who have been working hard on this project for many years, know that they still have a long way to go but for today at least, this step probably feels a little bit more like a skip for joy.”

What’s the Canadian connection? ViaCyte has one of the world’s top insulin experts as a scientific advisor: Dr. James Shapiro, Director of the Clinical Islet Transplant Program at the University of Alberta. In the 1990s, Dr. Shapiro co-developed the Edmonton Protocol, a procedure for implementing pancreatic islets for the treatment of type 1 diabetes.

Canada has been a leader in diabetes research since the early 1920s, when Drs. Frederick Banting and Charles Best discovered insulin. The more recent work done by Dr. Shapiro and the University of Alberta team, and stem cell experts like Dr. Kieffer and the University of Toronto’s Dr. Derek van der Kooy, who first isolated pancreatic stem cells, has kept Canada at the forefront.

But, as Dr. Alan Bernstein told the Globe and Mail last week at the launch of the Canadian Stem Cell Strategy & Action Plan, “the rest of the world is not standing still.”

California has invested $3 billion in stem cell R&D. The CIRM/ViaCyte news is proof the investment is  yielding significant dividends.

“We risk slowing down our investment while the rest of the world is speeding up, so relatively we will fall further and further behind,” Dr. Bernstein, Chair of the Canadian Stem Cell Foundation,  told the Globe. “This sort of research and the clinical trials are both long-term [prospects]. They need sustained investment and they are expensive.”

The Strategy & Action Plan plots a course for Canada to lead the way in delivering up to 10 new treatments for diseases to the clinic within 10 years that could transform the health care landscape. Find out more about the Strategy here.

 

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11
Jun 2014
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ISSCR Screen Capture

What’s the ‘real deal’ on stem cells?

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The day before 3,600 scientists, clinicians, educators and industry professionals from around the world gather in Vancouver next week for the International Society of Stem Cell Researchers’ summit, the public will get a chance to hear about the ‘real deal’ on stem cells.…

The day before 3,600 scientists, clinicians, educators and industry professionals from around the world gather in Vancouver next week for the International Society of Stem Cell Researchers’ summit, the public will get a chance to hear about the ‘real deal’ on stem cells.

Moderated by the Vancouver Sun’s Pamela Fayerman, the Tuesday, June 17th symposium focuses on why stem cells, which have been hailed for the past two decades as having the potential to fight so many diseases, have — with some notable exceptions — been slow to deliver.

The panel that includes Prof. Timothy Caulfield, author of The Cure for Everything and member of our Foundation’s Science Leadership Council, and stem cell transplant recipient Jennifer Molson will tackle the question: Why is it taking so long to make these promised therapies a reality? Industry investment expert Gregory Bonfiglio of Proteus Venture Partners and University of British Columbia stem cell researcher Dr. Kelly McNagny will also share their views.

It’s an important question. The translation of stem cell research discoveries into stem cell therapies takes a long time. It includes securing funding, getting regulatory approvals and conducting rigorous — and hugely expensive — clinical trials. In the meantime, unregulated clinics are popping up around the planet, offering “miracle” stem cell cures that have not been proven safe or effective.

The symposium, sponsored by the Stem Cell Network and the Centre for Commercialization of Regenerative Medicine, will be held in the OmniMax Theatre at Science World at TELUS World of Science. For more information, click here.

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12
May 2014
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Putting patients first in Strategy

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Making sure that Canadian patients have safe, early access to the kind of innovative treatments that will emerge from stem cell research was the focus of the Second Health Charities Forum.…

Making sure that Canadian patients have safe, early access to the kind of innovative treatments that will emerge from stem cell research was the focus of the Second Health Charities Forum.

“There clearly is strong support for moving stem cell research forward safely and effectively,” says James Price, President & CEO of the Canadian Stem Cell Foundation, which organized the Forum in partnership with the Health Charities Coalition of Canada.  “The health charities, which represent millions of patients, realize they have an absolutely vital role to play in making sure that patients benefit from the emergence of new stem cell based treatments and cures.”

Held April 29th in Toronto, the second Forum — a first was held in November — was part of an ongoing national dialogue to craft a strategy for advancing stem cell research and development.  Discussion centred on ensuring patients can safely access innovative therapies as they make the transition from clinical trials stage to clinical practice.

The health charities leaders agreed that their organizations are ready to play an important role in patient/public engagement for the Canadian Stem Cell Strategy & Action Plan and its ultimate implementation.

Along with input from the health charities, the final draft of the Strategy,  slated to be ready this fall,  will reflect more than nine months of expert stakeholder consultations with  stem cell researchers, business and community leaders, industry and other leaders.

Second Health Charities Forum Participants

• Bill Barrable (The Rick Hansen Institute)
• Alan Bernstein (Canadian Stem Cell Foundation)
• Sîan Bevan (Canadian Cancer Society)
• Sue DeLisle (Canadian Stem Cell Foundation)
• Sam Donaldson (Ontario Stem Cell Initiative)
• Debbie Fung (KPMG)
• Janet Hux (Canadian Diabetes Association)
• Lori Lyons (The Foundation Fighting Blindness- Canada)
• Robin Markowitz (Lymphoma Canada);
• Michael May (Centre for Commercialization of Regenerative Medicine)
• Rob Oliphant (The Asthma Society)
• Biljana (Billie) Potkonjak (Canadian Liver Foundation)
• James Price (Canadian Stem Cell Foundation)
• Carolyn Pullen (Heart and Stroke Foundation)
• Johnathan Riley (The Arthritis Society)
• Janet Rossant (Ontario Stem Cell Initiative)
• Lisa Salapatek (Crohn’s and Colitis Foundation of Canada)
• Marla Spiegel (Muscular Dystrophy Canada)
• Phil Welford (Stem Cell Network)

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24
Apr 2014
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JulieLessard IRIC 2-cropped

Gene that regulates leukemia will take time (and patience) to tame

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While patience is a virtue for most of us, it is an absolute prerequisite for stem cell researchers.

The recent news that scientists have identified a gene called BRG1 that appears to regulate leukemia stem cells marks an important advance in understanding the dread disease.…

While patience is a virtue for most of us, it is an absolute prerequisite for stem cell researchers.

The recent news that scientists have identified a gene called BRG1 that appears to regulate leukemia stem cells marks an important advance in understanding the dread disease. It also signifies years of work by the team led by Dr. Julie Lessard at the Institute for Research in Immunology and Cancer (IRIC) of Université de Montréal.

“About four years,” says Dr. Lessard, pictured left, one of Canada’s leading researchers in the field of hematopoiesis — the art of blood production.

Using mice as subjects, Dr. Lessard’s team found that removing the BRG1 gene left the leukemia stem cells and progenitors unable to survive, divide and make new tumors, permanently shutting down the cancer.  But while they are delighted with their findings, the researchers know they are in for many more years of work.

“We need to identify BRG1 inhibitors that will work in vitro (in test tubes and Petri dishes) and in vivo (with animals and humans),” says Dr. Lessard. “We believe that it is the ATPase activity that is the essential function we need to target for potential drug development, so that’s what we’re going after.”

In essence, that means finding small molecules that can stifle BRG1, the research equivalent to finding a needle in a haystack. Fortunately, IRIC is equipped with computer-driven high throughput screening to search their library of about 120,000 molecules for one that will do the trick. “We are hoping we can get there in the coming years,” she says.

Dr. Lessard’s findings further strengthen Canadian leadership in the field of stem cells and hematopoiesis. It was two Ontario Cancer Institute researchers — Drs. James Till and Ernest McCulloch — who first proved the existence of stem cells in the early 1960s while trying to find new treatments for leukemia. Dr. John Dick, of Toronto’s University Health Network, first identified tumour-initiating cancer stem cells in 1997.

What’s particularly intriguing about Dr. Lessard’s findings is that shutting down the BRG1 gene only appears to affect leukemia-generating stem cells. “Its function in the normal stem cell is rather modest. So you can take the gene out of leukemic cells and it will shut them down without shutting down the other stem cells you need to continue growth.”

While Dr. Lessard is excited about this project, she’s realistic about the amount of time and work involved.

“First of all, we have to have a very solid preclinical product to test in animals.  We think that a therapeutic window must exist. And this is what makes this study more interesting. It will be very exciting to explore in the coming years.”

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11
Apr 2014
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CIRM logo

Stem cell research in California: ten years later

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Ten years ago, Californians voted in favor of Proposition 71 to support stem cell research, committing $3 billion to stem cell research and creating the California Institute for Regenerative Medicine (CIRM), the state agency that allocates the funds.…

Ten years ago, Californians voted in favor of Proposition 71 to support stem cell research, committing $3 billion to stem cell research and creating the California Institute for Regenerative Medicine (CIRM), the state agency that allocates the funds.

In 2011, CIRM announced it would fund its first human clinical trial using a stem-cell derived therapy, committing $25 million to a company called Geron to test the use of neural stem cells in patients in spinal cord injury. It was an inauspicious start: Geron shut down the trial after about six months and gave back the money, citing a desire to focus on experimental cancer therapies that were further along in the development pipeline.

However, CIRM has carried on. And according to the Sacramento Business Journal nine of the first 14 stem cell trials the agency has funded have either started enrolling patients or will begin doing so by the end of the year. Two trials are already underway: one testing therapies for HIV/AIDS, the other for congestive heart failure. Other trials set to begin include therapies for leukemia and solid tumor cancers, degenerative eye disease, Type I diabetes, sickle cell disease and a serious blood disorder.

“It’s the largest funding in the world for this particular science and it turned California into the epicenter for stem cell research,” said Dr. Jonathan Thomas, board chairman for the CIRM.

The process of transition from studies in mice to human trials is daunting and complex. (Read our recent blog entry on the state of stem cell trials about the state of stem cell clinical trials around the world.) But bringing more potential therapies to clinical trials is absolutely crucial.

“The world is full of cured mice,” says Dr. Jonathan Thomas. “You never know what you get in humans but this is how you find out. You have to get to clinical trials.”

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