clinical trial

12
Jan 2015
0 Comments

Diabetes trial cleared by Health Canada

Posted by

Recently, we blogged about the first U.S. patient successfully implanted with a device to treat type 1 diabetes.

Good news is that the VC-01 device will soon be tested on Canadian patients.…

Recently, we blogged about the first U.S. patient successfully implanted with a device to treat type 1 diabetes.

Good news is that the VC-01 device will soon be tested on Canadian patients. After the U.S. Food and Drug Administration approval of the testing in August 2014, Health Canada has now cleared the beginning of the Phase 1/2 clinical trial of the VC-01 in Canada.

The device, developed by the American company ViaCyte, is about about half the size of a credit card and can be implanted under the skin from where its progenitor cells secrete insulin whenever blood sugar levels get too high, restoring glycemic harmony. It is being tested at the University of California, San Diego Health System for its safety, tolerability and efficacy in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function.

“The first cohort of patients in this two-cohort dose escalation study of the VC-01 product candidate is currently being assessed at a single site in the United States. Health Canada’s approval represents further validation of the trial and allows us to expand internationally to one or more sites in Canada.” Dr. Paul Laikind, President and CEO of ViaCyte said in the press release.

The location and enrollment start date of the Canadian trial site are yet to be released, but there is already a strong connection between Canada and ViaCyte. Edmonton’s Dr. James Shapiro, Director of the Clinical Islet Transplant Program at the University of Alberta, is a scientific advisor with ViaCyte. In 1990s, Dr. Shapiro co-developed the Edmonton Protocol, a procedure for implementing pancreatic islets for the treatment of type 1 diabetes.

Although the VC-01 clinical trial is in its early stages and it is unsure whether it will become a safe and efficient treatment option for diabetes, it represents an important step towards a cure for the disease.

Learn here how up to 10 treatments for chronic diseases will be developed over the next 10 years thanks to the Canadian Stem Cell Strategy & Action Plan.

Click to read more Close
11
Sep 2014
0 Comments

UK researchers post ‘encouraging’ results in stroke study

Posted by

The sample size is far too small to prove much yet, but doctors in Britain have seen “very encouraging” results from a new therapy that delivers stem cells extracted from patients’ bone marrow to their brains within days of having suffered a stroke.…

The sample size is far too small to prove much yet, but doctors in Britain have seen “very encouraging” results from a new therapy that delivers stem cells extracted from patients’ bone marrow to their brains within days of having suffered a stroke.

According to a report published in August in Stem Cells Translational Medicine,  all five patients who took part in the pilot study showed improvements over a six-month follow-up period.

This is significant because all but one of the five had the most severe type of stroke from which only four per cent of patients usually recover and regain independence. A story carried in the Daily Mail reported that  all four of these severe-stroke patients were alive and three were independent after half a year.

In the trial, believed to be the of its kind, the patients received purified CD34+ cells,  which are stem cells found in the bone marrow.  The patients got the these cells within a week of their attacks (in previous studies stem cells were infused months afterwards) to release  chemicals to spur  growth of new tissue and blood vessels in the parts of the brain damaged by stroke.

Dr Soma Banerjee, a lead author and Consultant in Stroke Medicine at London’s Imperial College Healthcare NHS Trust, urged caution:  “This study showed that the treatment appears to be safe and that it’s feasible to treat patients early when they might be more likely to benefit. The improvements we saw in these patients are very encouraging, but it’s too early to draw definitive conclusions … We need to do more tests to work out the best dose and timescale for treatment before starting larger trials.”

Should the therapy prove effective in larger scale clinical trials, the implications are enormous. Stroke is a major killer and disabler.  According to the Heart and Stroke Foundation, there are 50,000 strokes in Canada each year — a rate of one every 10 minutes.

The University of Toronto’s Dr. Cindi Morshead, whose research explores using stem cell s in regenerative medicine, called the study “quite comprehensive.”  She pointed out that the researchers screened more than 80 potential candidates for the study before selecting the five who got the treatment. “It was a safety trial so they really had to be careful in their selection.  But five out of 80 people able to benefit from this, that’s still pretty good. ”

As someone who works in the field, she’s optimist about the results. “My takeaway is that it’s exciting. Two of the people in the study were quite young: 45 and 47. It’s hugely significant — they’ve only lived half their lives.”

For a comprehensive look at using stem cells to treat stroke, click here.

 

 

 

 

Click to read more Close
27
Jan 2014
0 Comments
Fehlings Screen Captre

Spinal cord study: ‘This is no flash in the pan’

Posted by

Recently we blogged about a media report on a first-of-its kind North American study to test using neural stem cells to treat spinal cord injury (See Hope not hype).

Recently we blogged about a media report on a first-of-its kind North American study to test using neural stem cells to treat spinal cord injury (See Hope not hype). The small, Phase 1 clinical trial, sponsored by StemCells, Inc.  of California, is underway in Calgary — where the first patient has received his transplant — and Toronto. NewsDesk subsequently asked Dr. Michael Fehlings, head of the spinal program at Toronto Western Hospital and the lead investigator for the trial at the University of Toronto, to share his thoughts on the experimental treatment. In this edited transcript of the conversation, he describes the excitement at seeing the trial take place here in Canada, addresses ethical concerns and stresses the importance of managing patients’ expectations.  

Q: Is this the first time that this kind of thing has been done in North America?

A: Not exactly. This is the first the first trial with adult neural stem cells in spinal cord injury in North America. But Geron, which is another California-based company, got approval to use stem cells derived from embryonic stem cells and they injected four patients with traumatic spinal cord injury in the last couple of years. Then the company stopped the trial for financial reasons.

There has been a trial with neural stem cells in patients with Amyotrophic Lateral Sclerosis (ALS). That is a different company and they have completed the Phase 1 trial. So there is some precedent in the ALS field.

StemCells, Inc. has ventured into early phase human clinical trials in pediatric neurodevelopmental conditions. They did a small Phase 1 study in children with neurodevelopmental condition called neuronal ceroid lipofuscinosis and a Phase 1 clinical trial in children with a demyelinating condition called Pelizaeus-Merzbacher disease.

But this is the first trial of its kind using adult neural stem cells. We have found very convincing evidence for regeneration of the injured spinal cord injury and, particularly, we found that the neural stem cells are effective in remyelinating residual neurons in the host spinal cord.

This trial represents an important breakthrough in Canada as well, because it is the first of its kind (here). It does reflect an acceptance by Health Canada and will provide impetus for accelerating research at the pre-clinical level. Because there are many questions that will emerge out of the clinical arena that cannot be answered in a clinic. These will be addressed in pre-clinical laboratories and we will hopefully continue to move forward into the clinical pathways.

Q: I was impressed how CTV handled the report. They didn’t create the impression that people were going to jump out their wheelchairs with this.

A: I think it is a fair statement. I think that what this type of trial does is calibrate the discussion. We have moved on from the types of dramatic reports we heard with the so called “medical tourism” — dramatic claims that raise hope but are not supported by evidence. Here, the level of rigour to get a trial like this approved by Health Canada is extremely high.  I think for us it is really important and it is very exciting for the stem cell community because it shows that Canada is on the map. I think it is very noteworthy that this American company has chosen Canada as their entry to the North American platform.

Q: As you said, it is reassuring to Canadians that the work is being done here, that they don’t have to go everywhere and pay big dollars for suspect treatments.

A: Exactly. We are treating our own citizens and patients are not being charged anything. This is not being done for profit and we are making no claims, nothing but the fact that this is an exciting experimental treatment. It’s exciting, it’s hopeful, but we also have to be realistic. Ultimately, we are trying to determine what the effects of these cells are. We don’t know if the cells will work. We hope they will show an effect.

Q: These are stem cells derived from fetal tissue. Any concern about that? *

A: Anything in the arena of stem cells can potentially generate ethical questions for individuals. Whether we deal with embryonic stem cells, stem cells from fetal tissue, induced pluripotent stem cells. My response would be that the discussion is undertaken with the individuals with spinal cord issues and with their families, so they are aware what the source of the tissue is and if they have ethical concerns, their concerns are respected. On the other hand, it needs to be respected that the people have the right to have choice.

In terms of the source, I view this as a kind of the first step. Ultimately my hope would be that we could use our patient’s own cells. I would say that while I respect that there might be a minority of individuals who might express concerns, the cells come from tissue that otherwise would have been discarded. And because they are adult cells, the risk for forming a cancer or teratoma is extremely remote.

Q: Are you concerned that you might be swamped by people with traumatic spinal cord injury who want to take part? 

A: We are getting a lot of emails and a lot of requests. Most of the individuals who contact us are not candidates. It is a challenge to manage that. We anticipated that. We are trying to manage people’s expectations. We have a long-term commitment to this. This is no short term flash in the pan. It is a long-term strategy and commitment. We want to do things right, in a very responsible manner. We are trying to help people with spinal cord injury and to do the right thing.

* Editor’s Note: Stem cell research in Canada is conducted in accordance with the Tri-Council Policy Statement: Ethical Conduct for Research Involving Humans, the Canadian Institutes of Health Research Updated Guidelines for Human Pluripotent Stem Cell Research and the Assisted Human Reproduction Act. For a more detailed explanation, click here.

Click to read more Close
14
Jan 2014
5 Comments
CTV Fehlings Capture

Dr. Michael Fehlings of Toronto Western Hospital

Hope not hype for spinal cord injury

Posted by

CTV News is to be commended for its excellent, even-handed reporting late last week of an exciting but early-

stage clinical trial to test using stem cell injections to treat spinal cord injury.…

CTV News is to be commended for its excellent, even-handed reporting late last week of an exciting but early-

stage clinical trial to test using stem cell injections to treat spinal cord injury.

The Friday report, originating in Calgary where the first North American patient has received the treatment, goes to great lengths to temper hope with the hard reality that a cure for spinal cord injury is many years away.

Reporter Karen Owen cautions that  “everyone’s expectations have to be realistic” and features University of Calgary neurosurgeon Dr. John Hurlbert talking about potential small improvements in patients’ quality of life, such as being able to hold a fork or button a shirt.

In other words, no one is raising false hope by suggesting patients might be rising from their wheel chairs and running down hospital halls any time soon.

The CTV website’s also offers an extended interview with Dr. Michael Fehlings, head of the spinal program at Toronto Western Hospital and the lead investigator for the trial at the University of Toronto, who articulately explains why it’s important for the public to hear about this kind of study — even though it’s so early in the game.

“It represents an advance in regenerative medicine technologies from the laboratory into the clinical realm,” Dr. Fehlings says. “This is  now a situation where the science has advanced to the stage where as rigorous a regulatory authority as Health Canada now feels the science is at a level where it can be ethically and scientifically studied in man. So this is a big deal in terms of the advance of the science.”

The North American trial builds on work already done in Europe where nine patients have undergone the treatment with no apparent adverse effects and some small gains observed. In essence, the researchers inject neural stem cells into the spine where there may be some intact nerve fibres to stimulate regrowth insulating layers called myelin. The goal is to restore electrical conduction along the spinal cord to restore muscle strength and sensation.

This is good news. It gives hope to the more than 85,000 Canadians who live with spinal cord injury. But it doesn’t set them up for disappointment by giving them hype.

Click to read more Close
29
Nov 2013
0 Comments
japan-flag

A small clinical trial says big things about Japan

Posted by

Japan is moving forward with its plans to fast-track the use of induced pluripotent stem (iPS) cells to treat diseases — and revitalize its economy.…

Japan is moving forward with its plans to fast-track the use of induced pluripotent stem (iPS) cells to treat diseases — and revitalize its economy.

According to the Japan Times, the country last week officially passed a law “to promote safe and swift treatment using induced pluripotent stem (iPS) cells and other stem cells.” The legislation had previously received lower-house parliamentary approval.

As touched upon in an earlier post, Japanese scientists are now conducting world’s first human tests using iPS cells to treat age-related macular degeneration – the leading cause of vision loss in people over 50.

It’s no surprise they are first. Japan’s Dr. Shinya Yamanaka demonstrated how to create human iPs cells six years ago, inducing  adult skin cells to become pluripotent (capable of differentiating into any cell the body needs). These cells function much like human embryonic stem cells but come without any controversy over destroying embryos to create them. The downside to iPS cells is a safety concern that the reprogrammed cells could potentially cause tumours to form. Researchers in Canada and around the world have been working on solutions to that.

Dr. Yamanaka  won the 2012 Nobel Prize in Physiology or Medicine for his efforts. Throwing their support behind his work, Japan’s government recently announced it would invest more than $1 billion over the next 10 years in researching and developing iPS cells.

There is much more than national pride at stake, however.  According to Bloomberg News, Prime Minister Shinzo Abe sees cellular regeneration as a key element of economic regeneration. His government, the report says, “estimates that stem cells’ potential to rejuvenate worn-out body parts or reverse degenerative diseases such as Alzheimer’s may yield $380 billion in sales by 2050.”

The macular degeneration trial involves just six patients using iPS cells generated from their own skin. It is a  tiny study by any measure. But it represents a big step in the country’s efforts to develop a stake in that potential mega-billion-dollar market.

Click to read more Close
26
Sep 2013
4 Comments
StewartGarrows400EN

Will enhanced stem cells mend broken hearts?

Posted by

The unveiling of a clinical trial to test the use of genetically enhanced stem cells to rebuild badly damaged hearts captured major media attention in early September.…

The unveiling of a clinical trial to test the use of genetically enhanced stem cells to rebuild badly damaged hearts captured major media attention in early September.

CBC News, the Sun and Postmedia newspapers all called the trial “groundbreaking” and gave it prominent play. CTV  called it a “world-first clinical trial.” The Globe and Mail  coverage was somewhat more restrained but went into fine detail to explain how the patient’s own blood stem cells are enhanced with a gene called endothelial nitric oxide synthase (eNOS) that is then infused into the heart at the site of the damage.

It didn’t hurt that the Ottawa Hospital Research Institute, where Principal Investigator Dr. Duncan Stewart is CEO and Scientific Director, was able to put a gentle human face on complicated science by presenting Patient No. 1. Sixty-eight-year-old Harriet Garrow of Cornwall, Ontario suffered a major, heart-stopping myocardial infarction in July.  Holding hands with her husband Peter Garrow, she was amiable, articulate and authentic.  Reporting the news Sept. 10, the Aboriginal Peoples Television Network  highlighted her heritage, headlining its story “Mohawk woman in centre of ground breaking medical treatment.”

This is a double-blind study – meaning neither the investigators nor the 100 participants in Ottawa, Montreal and Toronto will know who received the souped-up stem cells or the comparative controls of ordinary stem cells or placebos until after the results are in. That doesn’t matter to Mrs. Garrow, who, at the very least, is getting the best of current cardiac care. “I am thrilled to play a part in this research that could help people like me in the future and, who knows, perhaps even my children and grandchildren,” she said in the OHRI’s media release.

A decade of work

Investigators will start analyzing the results after Patient No. 100 has been enrolled and treated – more than two years from now. By that time, Dr. Stewart will have put 10 years into the project he originally started at St. Michael’s Hospital in Toronto.

But things could happen quickly after that.

“Most major medical centres in Canada and the United States have the capacity to do this kind of cell manufacturing, “ says Dr. Stewart. “So, if we allow ourselves to dream a bit, that this really is a very positive trial with major improvements, I think it could be adopted quite quickly.”

The implications – in terms of thousands of lives that could be saved and millions of dollars in health care costs avoided – are immense. About 70,000 Canadians have heart attacks every year. Dr. Stewart estimates that about one-third of those, around 23,000, suffer damage severe enough to require this level of intervention.

“This segment of the infarct population is one that’s going to cost an awful lot of money because before they die they are going to develop heart failure. They’re going to be having multiple prolonged hospital admissions and require implantation of defibrillators and are going to have all kinds of other treatments that cost the system a lot of money.”

Other trials using ordinary stem cells have shown some positive effect on repairing heart tissue, but nothing that would spark widespread change in how heart attack patients are treated. The difference here is these stem cells have rejuvenated with eNOS to do a more effective repair job.

“It’s always been our view that getting the most robust therapy requires some manipulation of the cells, particularly when you’re using the patient’s own cells,” says Dr. Stewart. “The cells are the same age as the patient, which is usually 60 or 70 years old, and they have been exposed to the same factors that produced heart disease in the first place. We know they don’t work well. But if we can recover the activity of these cells we’re going to get more benefit.”

It all depends, of course, on The Big If: If it works.

Given the rigorous controls and criteria included in the clinical trial,  the answer should be obvious within three years.

Click to read more Close
Back to Top