macular degeneration

16
Mar 2017
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Unproven stem cell treatment leaves three women blind

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Three women who believed they were participating in a clinical trial either lost all or most of their vision after being injected with stem cells derived from their own fat in a Florida clinic. …

Three women who believed they were participating in a clinical trial either lost all or most of their vision after being injected with stem cells derived from their own fat in a Florida clinic.  The case points to the dangers of unproven treatments offered by private clinics.

Within days of treatment the women, who all suffered from macular degeneration, began to experience severe side effects including bleeding in the eye, detached retinas and vision loss as reported in a study released week in The New England Journal of Medicine.   Expert ophthalmologists tried to reverse the damage but were unable.

“There’s a lot of hope for stem cells, and these types of clinics appeal to patients desperate for care who hope that stem cells are going to be the answer, but in this case these women participated in a clinical enterprise that was off-the-charts dangerous”  said Dr. Thomas Alibini, a lead author of the study, in a press release.

This isn’t the first time there have been adverse reactions to unproven treatments offered at clinics, many using stem cells drawn from the patient’s own fat, and we’ve written about it several times on this blog warning patients to be cautious of claims that appear too good to be true.

So how can patients protect themselves?  The California Institute of Regenerative Medicine offers this advice:

  • There is almost no evidence that the fat/blood stem cell combination the clinic used could help repair the photoreceptor cells in the eye that are attacked in macular degeneration.
  • The clinic charged the women $5,000 for the procedure. Usually in Food and Drug Administration (FDA)-approved trials the clinical trial sponsor will cover the cost of the therapy being tested.
  • Both eyes were injected at the same time.  Most clinical trials would only treat one eye at a time and allow up to 30 days between patients to ensure the approach was safe.
  • Even though the treatment was listed on the clinicaltrials.gov website, there is no evidence that this was part of a clinical trial, and certainly not one approved by the FDA.

Most importantly, patients should check with their doctor before considering any medical treatment or participating in any clinical trial.  You can also find great information for patients considering stem cell treatments here that has been produced by the International Society for Stem Cell Research (ISSCR).

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09
Aug 2016
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Dr. Molly Shoichet

Breakthroughs can produce a lot of hope, but also frustration

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You read about a stem cell breakthrough that could lead to a whole new way of treating — maybe even curing — a disease. …

You read about a stem cell breakthrough that could lead to a whole new way of treating — maybe even curing — a disease. Then … nothing happens.

The gap in time between a medical science discovery and actual clinical application can seem like a millennium, especially if you are living with the disease in question.

This week in the Toronto Star, Dr. Molly Shoichet articulately — and compassionately — explains why it takes so long to move from the “Eureka!” moment in the research lab to a “We can treat this” scenario at the hospital bed.

She knows first-hand, having worked with a University of Toronto team including Drs. Derek van der Kooy,  Cindi Morshead, Brian Ballios and Michael Cooke that created a hydrogel to help transplanted stem cells thrive in the brain and eye. By injecting photoreceptor cells, the team was able to restore vision by approximately 15% in blind mice.

Their 2015 discovery garnered plenty of media attention, sparking hope that people with macular degeneration — a currently incurable condition — could have a new treatment. More than a year later, no such treatment is available or even on the horizon.

“For many people with macular degeneration, or any degenerative disease for that matter, studies like mine often produce a lot of hope, but also frustration that the ‘product’ is not available to them,” she writes in the Star.

As Dr. Shoichet explains, it can easily take 17 years or more to move a discovery from the lab to the patient.  It takes lots of testing with animals before a new treatment can safely move to the three stages of human clinical trials, with all the attendant regulatory controls and approvals.  It involves plenty of hard, painstaking work. And it costs lots of money.

But Dr. Shoichet remains optimistic. The opportunity to improve the lives of people with deadly diseases is what drives scientists to get up in the morning and get to work.  As she writes, “I am optimistic we will deliver on the promise of regenerative medicine — but it won’t happen right away.”

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21
Jul 2016
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Dr. Valerie Wallace (UHN Photo)

Cure for macular degeneration may be years away but scientist sees exciting advances on the horizon

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Dr. Valerie Wallace is realistic about finding cell-based cures for blindness.

“It’s a long road,” says the Dr. Wallace, Chair of the Vision Science Research Program at University Health Network’s Krembil Research Institute in Toronto.…

Dr. Valerie Wallace is realistic about finding cell-based cures for blindness.

“It’s a long road,” says the Dr. Wallace, Chair of the Vision Science Research Program at University Health Network’s Krembil Research Institute in Toronto. “Cell transplantation is tremendously complicated, difficult, and painstaking.”

But she is also optimistic about what the future holds for gaining a better understanding of the underlying causes of macular degeneration, which is triggered by deterioration of the cone photoreceptors (cones) in the centre of the eye that mediate reading and seeing fine detail. (In contrast, rod photoreceptors are concentrated along the edges of the retina to facilitate peripheral vision.)

“We are learning a lot about the biology that underpins photo cones, says Dr. Wallace. “They are a rare cell type and understanding their development has been difficult. Now we have tools to do that. That’s what I’m excited about.”

Her lab has developed a new mouse strain with fluorescently tagged cones to allow the researchers to track the progress of the cells to see if they can rescue visual function when transplanted into blind mice.

“We’re looking directly at this. Can cone cells transplant? Can they rescue vision? And if they can, what stage of cone development is the best for transplantation?”

The work she is doing feeds into a larger team effort dedicated to optimizing how to make photo receptors from human stem cells. What she learns from trying to engraft mouse cells will be applied to people. It also has implications for finding therapies for other neural degenerative diseases, such as Parkinson’s disease, Alzheimer’s and stroke.

“The eye has always been thought of as very different from the brain,” she says, “but it is part of the brain.  Our work on identifying novel approaches to promote the survival of these cells could extend to  promote neuron survival in other parts of the central nervous system.”

And the eye, aside from being the window to the soul, can also be a window into a person’s health. Says Dr. Wallace: “People are now using non-invasive imaging of the eye to look at markers of disease. There is a large study in Alzheimer’s that is imaging the eye to identify people at early stages of the disease. That’s happening more and more. People are appreciating that some aspects of eye health might inform the progression or even the diagnosis of other diseases.”

Dr. Wallace, who calls herself “a developmental biologist at heart,” doesn’t forget who she’s working for: people struggling with vision loss. Her past work with the Foundation Fighting Blindness, which helps fund her research, guarantees that. “I hear a lot about what’s important to patients.”

While progress is taking time, the science is steadily moving forward. “A lot of the things we’re doing now were not even being talked about 10 years ago.”

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