4 things to know about stem cell clinical trials [Video]

Every day, we receive phone calls and emails from people who are desperately seeking our help. Sometimes they reach out on their own behalf, though often it’s for a family member or close friend. In every case, someone is suffering or dying from a disorder that has no available cure or effective treatment and they look to stem cell treatments as their only hope.

It’s heartbreaking to hear these personal stories that are unfolding in real time. Though they contact us from a wide range of places about a wide range of disorders, their initial set of questions are often similar and go something like this:

  • “Where can I find stem cell clinical trial for my condition?”
  • “What are my chances of being cured?”
  • “How much does it cost to be in a clinical trial?”
  • “How can I be sure it’s safe?”

We think anyone thinking about taking part in a clinical trial should consider these important questions. So, in addition to providing answers as we receive them through phone calls and emails, we wanted to find a way to reach out to as many people as possible. The result? The four-minute animation video you can watch below:

As mentioned in the video, the answers to these questions are only the tip of the iceberg for finding out if a particular clinical trial is right for you. The website, A Closer Look at Stem Cells, produced by the International Society for Stem Cell Research (ISSCR), is an excellent source for more advice on what things you should know before participating in a stem cell clinical trial or any experimental stem cell treatment.

And visit the Patient Resources section of our website for even more practical information including our growing list of CIRM-funded clinical trials as well as trials supported by our Alpha Stem Cell Clinic Network.

A call to put the ‘public’ back in publication, and make stem cell research findings available to everyone

Opening the door

Opening the door to scientific knowledge

Thomas Gray probably wasn’t thinking about stem cell research when, in 1750 in his poem “Elegy in a Country Churchyard”, he wrote: “Full many a flower is born to blush unseen”. But a new study says that’s precisely what seems to happen to the findings of many stem cell clinical trials. They take place, but no details of their findings are ever made public. They blush, if they blush at all, unseen.

The study, in the journal Stem Cell Reports, says that only around 45 percent of stem cell clinical trials ever have their results published in peer-reviewed journals. Which means the results of around 55 percent of stem cell clinical trials are never shared with either the public or the scientific community.

Now, this finding apparently is not confined to stem cell research. Previous studies have shown a similar lack of publication of the results of more conventional therapies. Nonetheless, it’s a little disappointing – to say the least – to find out that so much knowledge and potentially valuable data is being lost due to lack of publication.

Definitely not full disclosure

Researchers at the University of Alberta in Canada used the US National Institute of Health’s (NIH) clinicaltrials.gov website as their starting point. They identified 1,052 stem cell clinical trials on the site. Only 393 trials were completed and of these, just 179 (45.4 percent) published their findings in a peer-reviewed journal.

In an interview in The Scientist, Tania Bubela, the lead researcher, says they chose to focus on stem cell clinical trials because of extensive media interest and the high public expectations for the field:

“When you have a field that is accused of over promising in some areas, it is beholden of the researchers in that field to publish the results of their trials so that the public and policy makers can realistically estimate the potential benefits.”

Now, it could be argued that publishing in a peer-reviewed journal is a rather high bar, that many researchers may have submitted articles but were rejected. However, there are other avenues for researchers to publish their findings, such as posting results on the clinicaltrials.gov database. Only 37 teams (3.5 percent) did that.

Why do it?

In the same article in The Scientist, Leigh Turner, a bioethicist at the University of Minnesota, raises the obvious question:

“The study shows a gap between studies that have taken place and actual publication of the data, so a substantial number of trials testing cell-based interventions are not entering the public domain. The underlying question is, what is the ethical and scientific basis to exposing human research subjects to risk if there is not going to be any meaningful contribution to knowledge at the end of the process?”

In short, why do it if you are not going to let anyone know what you did and what you found?

It’s a particularly relevant question when you consider that much of this research was supported with taxpayer dollars from the NIH and other institutions. So, if the public is paying for this research, doesn’t the public have a right to know what was learned?

Right to know

At CIRM we certainly think so. We expect and encourage all the researchers we fund to publish their findings. There are numerous ways and places to do that. For example, we expect each grantee to post a lay summary of their progress which we publish on our website. Stanford’s Dr. Joseph Wu’s progress reports for his work on heart disease shows you what those look like.

We also require researchers conducting clinical trials that we are funding to submit and post their trial results on the clinicaltrials.gov website.

The International Society for Stem Cell Research (ISSCR), agrees and recently updated its Guidelines for Stem Cell Research and Clinical Translation calling on researchers to publish, as fully as possible, their clinical trial results.

That is true regardless of whether or not the clinical trial showed it was both safe and effective, or whether it showed it was unsafe and ineffective. We can learn as much from failure as we can from success. But to do that we need to know what the results are.

Publishing only positive findings skews the scientific literature, and public perception of this work. Ignoring the negative could mean that other scientists waste a lot of time and money trying to do something that has already demonstrated it won’t work.

Publication should be a requirement for all research, particularly publicly funded research. It’s time to put the word “public” back in publication.

 

 

Your Guide to Awesome Stem Cell Conferences in 2017

Welcome to 2017, a year that will likely be full of change and new surprises. I’m hoping that some of these surprises will be in regenerative medicine with new stem cell therapies showing promise or effectiveness in clinical trials.

A great way to stay on top of new advances in stem cell research is to attend scientific conferences and meetings. Some of them are well known and highly attended like the International Society for Stem Cell Research (ISSCR) conference, which this year will be in Boston in June. There are also a few smaller, more intimate conferences focusing on specific topics from discovery research to clinical therapies.

There are loads of stem cell meetings this year, but a few that I would like to highlight. Here’s my abbreviated stem cell research conference and meeting guide for 2017. Some are heavy duty research-focused events and probably not suitable for someone without a science background; they’re also expensive to sign up for. I’ve marked those with an * asterix.


January 8-12th, Keystone Symposium (Fee to register)*

Keystone will be hosting two concurrent stem cell meetings in Tahoe next week, which are geared for researchers in the field. One will be on neurogenesis during development and in the adult brain and the other will be on transcriptional and epigenetic control in stem cells. CIRM is one of the co-funders of this meeting and will be hosting a panel focused on translating basic research into clinical trials. Keystone symposiums are small, intimate meetings rich with scientific content and great for networking. Be on the look out for blog coverage about this meeting in the coming weeks.


February 3rd, Stanford Center for Definitive and Curative Medicine Symposium (Free to the public)

This free symposium at Stanford University in Palo Alto, CA will present first-in-human cell and gene therapies for a number of disorders including bone marrow, skin, cardiac, neural, uterine, pancreatic and neoplastic disorders. Speakers include scientists, translational biologists and clinicians. Irv Weissman, a Stanford professor and CIRM grantee focused on translational cancer research, will be the keynote speaker. Space is limited so sign up ASAP!


March 23rd, CIRM Alpha Stem Cell Clinics Symposium (Free to the public)

This free one-day meeting will bring together scientists, clinicians, patient advocates, and other partners to describe how the CIRM Alpha Stem Cell Clinics Network is making stem cell therapies a reality for patients. The City of Hope Alpha Clinic is part of a statewide effort funded by CIRM to develop a network of “Alpha Clinics” that has one unifying goal: to accelerate the development and delivery of stem cell treatments to patients.

City of Hope Medical Center and Alpha Stem Cell Clinic

City of Hope Medical Center and Alpha Stem Cell Clinic


June 14-17th, International Society for Stem Cell Research (Fee to register)*

The Annual ISSCR stem cell research conference will be hosted in Boston this year. This is an international conference focusing on new developments in stem cell science and technology. CIRM was one of the funders of the conference last year when ISSCR was in San Francisco. It’s one of my favorite research events to attend full of interesting scientific presentations and great for meeting future collaborators.


For a more comprehensive 2017 stem cell conference and meeting guide, check out Paul Knoepfler’s Niche blog.

Patients beware: warnings about shady clinics and suspect treatments

stem-cells therapy?

Every day we get a call from someone seeking help. Some are battling a life-threatening or life-changing disease. Others call on behalf of a friend or loved one. All are looking for the same thing; a treatment, better still a cure, to ease their suffering.

Almost every day we have to tell them the same thing; that the science is advancing but it’s not there yet. You can almost feel the disappointment, the sense of despair, on the other end of the line.

If it’s hard for us to share that news, imagine how much harder it is for them to hear it. Usually by the time they call us they have exhausted all the conventional therapies. In some cases they are not just running out of options, they are also running out of time.

Chasing hope

Sometimes people mention that they went to the website of a clinic that was offering treatments for their condition, claiming they had successfully treated people with that disease or disorder. This week I had three people mention the same clinic, here in the US, that was offering them “treatments” for multiple sclerosis, traumatic brain injury and chronic obstructive pulmonary disease (COPD). Three very different problems, but the same approach was used for each one.

It’s easy to see why people would be persuaded that clinics like this could help them. Their websites are slick and well produced. They promise to take excellent care of patients, often helping take care of travel plans and accommodation.

There’s just one problem. They never offer any scientific evidence on their website that the treatments they offer work. They have testimonials, quotes from happy, satisfied patients, but no clinical studies, no results from FDA-approved clinical trials. In fact, if you explore their sites you’ll usually find an FAQ section that says something to the effect of they are “not offering stem cell therapy as a cure for any condition, disease, or injury. No statements or implied treatments on this website have been evaluated or approved by the FDA. This website contains no medical advice.”

What a damning but revealing phrase that is.

Now, it may be that the therapies they are offering won’t physically endanger patients – though without a clinical trial it’s impossible to know that – but they can harm in other ways. Financially it can make a huge dent in someone’s wallet with many treatments costing $10,000 or more. And there is also the emotional impact of giving someone false hope, knowing that there was little, if any, chance the treatment would work.

Shining a light in shady areas

U.C. Davis stem cell researcher, CIRM grantee, and avid blogger Paul Knoepfler, highlighted this in a recent post for his blog “The Niche” when he wrote:

Paul Knoepfler

Paul Knoepfler

“Patients are increasingly being used as guinea pigs in the stem cell for-profit clinic world via what I call stem cell shot-in-the-dark procedures. The clinics have no logical basis for claiming that these treatments work and are safe.

As the number of stem cell clinics continues to grow in the US and more physicians add on unproven stem cell injections into their practices as a la carte options, far more patients are being subjected to risky, even reckless physician conduct.”

As if to prove how real the problem is, within hours of posting that blog Paul posted another one, this time highlighting how the FDA had sent a Warning Letter to the Irvine Stem Cell Treatment Center saying it had serious concerns about the way it operates and the treatments it offers.

Paul has written about these practices many times in the past, sometimes incurring the wrath of the clinic owners (and very pointed letters from their lawyers). It’s to his credit that he refuses to be intimidated and keeps highlighting the potential risks that unapproved therapies pose to patients.

Making progress

As stem cell science advances we are now able to tell some patients that yes, there are promising therapies, based on good scientific research, that are being tested in clinical trials.

There are not as many as we would like and none have yet been approved by the FDA for wider use. But those will come in time.

For now we have to continue to work hard to raise awareness about the need for solid scientific evidence before more people risk undergoing an unproven stem cell therapy.

And we have to continue taking calls from people desperate for help, and tell them they have to be patient, just a little longer.

***

If you are considering a stem cell treatment, the International Society for Stem Cell Research had a terrific online resource, A Closer Look at Stem Cells. In particular, check out the Nine Things to Know about Stem Cell Treatments page.

 

Happy Stem Cell Awareness Day!

SCAD_Logo_2015I woke up today extra early this morning feeling like a kid at Christmas time because it’s Stem Cell Awareness day!

This exciting day brings together organizations and people around the world working to ensure that we realize the benefits of one of the most promising fields of science in our time. The day is a unique global opportunity to foster greater understanding about stem cell research and the range of potential applications for disease and injury.

For the millions of people around the world who suffer from incurable diseases and injury, Stem Cell Awareness Day is a day to celebrate the scientific advances made to-date and be hopeful of what is yet to come.

Institutions and scientists around the world will be participating in talks and activities that celebrate and also educate the community about stem cell research. For a list of events, check out our Stem Cell Awareness Day webpage. You can also follow other events on twitter by following the hashtags #stemcellday and #astemcellscientistbecause.

In celebration of this exciting day, the Stem Cellar team would like to highlight a few videos and webpages dedicated to stem cell awareness. Enjoy!

Videos:

“A Stem Cell Story” from our friends at EuroStemCell

#AStemCellScientistBecause videos via Cell Stem Cell on twitter

 

Stem Cell Awareness Webpages:

Policy Matters: Stem Cells and the Public Interest

Guest Author Geoff Lomax is CIRM’s Senior Officer for Medical and Ethical Standards.

In the spirit of Stem Cell Awareness Day, Cell Stem Cell has compiled a “Public Interest” collection of articles covering ethical, legal, and social implications of stem cell research and made it freely available. The collection may be found here.

shutterstock_169882310

The collection covers issues ranging from research involving human embryos to the use of stem cell therapies in patients. For those of you interested in a good primer on the history of stem cell controversies, Herbert Gottweis provides a detailed review of the federal policy debate in the United States. This debate has resulted in inconsistent policy and disrupted research. Gottweis uses this history to support his message that a “comprehensive, and proactive policy approach in this field beyond the quick legal fix” is needed for patients to ultimately benefit from the science.

What I found most interesting about this collection was the focus on stem cell treatments and “tourism.” A majority of the articles address the use of stem cells in patients. This focus is an indicator of how far the field has progressed. Stem cells clinical trials are now a reality and this results in two separated but related considerations. First, is how to make sure prospective patients are well informed should they participate in a clinical trial. Second, how to avoid stem cell “snake oil” where someone is pitching an unproven procedure. These issues are related by their solution that involves empowerment and education of patients and their support networks.

For example, in Stem Cell Tourism and Public Education: The Missing Elements, Master writes:

“It is important for the scientific, medical, ethics, and policy communities to continue to promote accurate patient and public information on stem cell research and tourism and to ensure that it is effectively disseminated to patients by working alongside patient advocacy groups.”

Master’s team found that groups committed to the advancement of good science, including patient advocates and researchers, often lacked basic information about clinical trials and other options for patients. This lack of information may contribute to patients being wooed by those pitching unproven procedures. Thus, the research community should continue to work with patients and advocacy organizations to identity options for treatment.

Another aspect of patient empowerment is what Insoo Huyn refers to as “therapeutic hope” in his piece: Therapeutic Hope, Spiritual Distress, and the Problem of Stem Cell Tourism. Huyn suggests that a supportive system for delivering cell therapies should includes nurturing hope. He writes, “patients might understand when an intervention’s chances of success are extremely remote at best, but may still want to ‘‘give it a shot’’ as long as a beneficial outcome cannot be ruled out as categorically impossible.” Huyn recognizes that well developed early-stage clinical trials are not expected to provide a benefit to patients (they are designed to evaluate safety), but the nature of the therapeutic (often cells) means there may be some real effect.

A third piece by the ISSCR Ethics Taskforce titled Patients Beware: Commercialized Stem Cell Treatments on the Web presents a guide to evaluating therapies. They present five principles that patients, researchers and advocates can rally around to identify credible interventions. The taskforce states:

The guiding principles for the development of the recommended process were that (1) the standards for identifying and reviewing clinics and suppliers should be objective and clear; (2) the inquiry and review process should be publicly transparent and relatively straight- forward for any clinic or practitioner to comply with; (3) conflicts of interest, if any, of the declarant ought to be disclosed to the ISSCR; (4) there should be no actual or apparent conflicts of interest of staff or others involved in the inquiry or review process for any particular matter; and (5) any findings that a clinic fails to meet standards should be communicated in a specific factual way, rather than with broad conclusions of fraudulent practices.

While the Cell Stem Cell Public Interest series covers a range of issues related to stem cells and society, the emphasis on treatments and patients is a reminder of how far the field has come. There is broad consensus that patients, researchers and advocates have roles to play in advancing safe and effective cell therapies.

Geoff Lomax

International Stem Cell Conference Opens with Public Quest to Sort the Real from the Bogus

While the scientific sessions of the International Society for Stem Cell Research don’t begin until this morning, the meeting started last night with one of its most important events: a panel discussion for patient advocates and other members of the public. After presentations by the five speakers it became clear that the dichotomy between real stem cell therapies and bogus ones is breaking down. There is an increasingly large middle zone of potential therapies in sanctioned clinical trials that are not yet proven safe or effective, but usually have sound science behind them.

Kelly McNagny of the University of British Columbia led off talking about the various types of stem cells and noted that most of the current clinical trials involve adult, tissue specific stem cells. He detailed the many reasons why it has taken researchers so many years to bring these cells to the verge of routine clinical use. You have to learn how to efficiently grow them outside the body and get them to behave in a very specific way when they are transplanted into a patient. They have to know how to talk to and respond to the cells around them.

Doing all this, McNagny said, takes a village; a multidisciplinary team of researchers and clinicians. He also said it needed another component, vocal patient advocates to maintain financial support for the work, and to act as a liaison between the patient community and the research community. This call for an active patient community working closely with researchers became a theme for the evening.

Dr. Tim Caufield at the podium

Dr. Tim Caufield at the podium

Tim Caulfield of the University of Alberta, who leads a team conducting some of the most extensive investigation of unregulated stem cell clinics around the world, told the crowd of over 200 that patient groups will have to help educate those who are desperate for hope about the danger of some of these operations. He said he has tracked more than 700 clinics around the world offering to treat many different diseases while offering little scientific rational and no data on results other than anecdotal patient testimonials.

He said these clinics use the legitimate excitement about the potential for stem cell therapies to sell bogus treatments. He dubbed this “scienceploitation.” Their ads, he added, make the public take for granted the effectiveness of the supposed treatments.

The Q&A at the end of the session drew out some advice to help patients sort out claims they find on the internet. Much of what was said echoed advice found on the ISSCR “A Closer Look” web site and on our “Stem Cell Tourism” web page.

Don Gibbons

Stem Cell Researchers from around the Globe Converging in Vancouver with More Talk of Therapies than Ever before

The International Society for Stem Cell Research (ISSCR) officially opens its annual meeting tonight in Vancouver with a public session called “The Real Deal on Stem Cell Therapies.” These preview sessions for the public have become a tradition for the association and generally focus on helping the lay public sort out the legitimate science from the many questionable claims in the field.

The four-day scientific sessions are the largest gathering of stem cell researchers each year with more than 3,000 expected in Vancouver. It provides critical opportunities for scientists to share information and gather informally over coffee or drinks to bounce around ideas. These chats often result in collaborations that accelerate the science.

My colleague Kevin McCormack and I will be roaming the meeting looking for trends and breakthroughs in the science and writing them up quickly for this blog. The first post will be Wednesday morning reporting on tonight’s public event. Other posts are likely to cover progress toward clinical trails in Parkinson’s Disease, diabetes and spinal cord injury, among others.

Our coverage of last year’s public session is here, and one trend we spotted last year was a recognition of the vital importance of the environment where stem cells are grown if you want them to mature into the desired tissue.

Looking forward to spotting new trends over the next few days.

Don Gibbons

Milton Berle and the Art of Writing an Award-Winning Stem Cell Essay

It’s not often that you hear famous comedian Milton Berle quoted in an essay about stem cells, but then U.C. Davis researcher and avid blogger Paul Knoepfler wasn’t looking for an ordinary essay as the winner for his recent contest.

Knoepfler launched the contest as a way to give some lucky individual a free registration to the International Society for Stem Cell Research (ISSCR) annual conference in Vancouver, Canada later this month.

Essay winner Mohamed Gatie

Essay winner Mohamed Gatie

The winning entry came from a student Mohamed Gatie. Here’s his winning essay.

The second place essay came from Sherry Hikita. We met Sherry at the World Stem Cell Summit in San Diego last year where she signed up as one of our Stem Cell Champions.

Second-place winner Sherry Hikita

Second-place winner Sherry Hikita

Congratulations to both Mohamed and Sherry, and of course kudos to Paul for coming up with the idea and helping a fine student get a chance to go to one of the biggest events of the year for a stem cell scientist.

kevin mccormack

Write your Ticket to the Stem Cell Science Event of the Year

For many stem cell scientists one of the highlights of the year is going to the annual International Society for Stem Cell Research (ISSCR) conference. It’s like Disneyland for researchers. This year the event is being held in Vancouver, Canada. But many younger scientists won’t be going because they can’t afford it. So UC Davis researcher and avid blogger Paul Knoepfler has teamed up with ISSCR to help one lucky scientist go. Here’s Paul’s guest blog explaining how they can apply:

What’s better than a four-day stem cell meeting held in beautiful Vancouver, B.C. that includes all of the newest and most cutting edge research?

If, like me, you are a lover of all things stem cell your first impulse may be to shout out “nothing!” and I am betting you will already know that I am talking about the ISSCR Annual Meeting being held this year in that fantastic locale.

Beautiful Vancouver, British Columbia home to this year’s ISSCR Annual Meeting

Beautiful Vancouver, British Columbia home to this year’s ISSCR Annual Meeting

However, the answer I had in mind is that the only thing better than going to ISSCR is getting to go to that meeting for free.

How do you get to that stem cell happy place?

I have teamed up with ISSCR to run a contest with the prize being free registration to the ISSCR meeting that runs from June 18-21.

All you have to do is write a short essay addressing this topic: why do I want to go to ISSCR 2014?

The winner gets free registration and their essay gets published on my blog to be read by the stem cell community.

For a few additional rules and details, check out the announcement of the contest here.

The deadline for entries is Friday May 30th. The winner will be announced on Monday June 2nd.

What am I looking for in a winning essay? As a stem cell researcher and writer myself, I am hoping to get entries that are clever, creative essays that convey genuine, interesting reasons for wanting to go to this great meeting. You will have to be concise too because the limit on length is a strict 300 words.

There may be additional entries published on my blog if more than one really wows me, and other perks like stem cell swag for entrants could come into play depending on the number of entrants and the quality of their writings.

So start brainstorming and writing and then send your entries to me at knoepfler@ucdavis.edu.

I hope to see you in Vancouver.

Paul Knoepfler