Why TED Talks are ChildX’s Play

When the TED (Technology, Entertainment, Design) talks began in 1984 they were intended to be a one-off event. So much for that idea! Today they are a global event, with TED-sponsored conferences held everywhere from Scotland to Tanzania and India. They have also spawned a mini-industry of copycat events. Well, their slogan is “Ideas Worth Spreading” so in a way they only have themselves to blame for having such a great idea.

Dr. Maria Grazia Roncarolo

Dr. Maria Grazia Roncarolo

The latest place for that idea to take root is Stanford, which is holding a TED-style event focused on critical issues facing child and maternal health. The event – April 2nd and 3rd at Stanford – is called ChildX where x = medicine + technology + innovative treatment + wellbeing. ChildX will bring together some of the leading experts in the field for a series of thoughtful, powerful presentations on the biggest problems facing child and maternal health, and the most exciting research aimed at resolving those problems. One of the main tracks during the two-day event is a section on stem cell and gene therapy. It will raise a number of key questions including:

  • What advances have occurred to enable these therapies to move from science fiction less than a decade ago to the promise of next generation transformative therapeutics?
  • In coming years, how will these therapies allow children with presently incurable diseases to become children living free of disease and reaching their maximum potential?

The moderator for that discussion is Dr. Maria Grazia Roncarolo, and you can hear her talking about the most recent advances in the clinical use of stem cell and gene therapies on this podcast. Anytime you get a chance to hear some of the most compelling speakers in their field talk about exciting innovations that could shape the future, it’s worth taking the time to listen.

The best tools to be the best advocate

It’s hard to do a good job if you don’t have the right tools. And that doesn’t just apply to fixing things around the house, it applies to all aspects of life. So, in launching our new website this week we didn’t just want to provide visitors to the site with a more enjoyable and engaging experience – though we hope we have done that – we also wanted to provide a more informative and helpful experience. That’s why we have created a whole new section call the Patient Advocate Toolbox. shutterstock_150769385

The goal of the Toolbox is simple; to give patients and patient advocates help in learning the skills they need to be as effective as possible about raising awareness for their particular cause.

As an advocate for a disease or condition you may be asked to speak at public events, to be part of a panel discussion at a conference, or to do an interview with a reporter. Each of those requires a particular set of skills, in areas that many of us may have little, if any, experience in.

That’s where the Toolbox comes in. Each section deals with a different opportunity for you to share your story and raise awareness about your cause.

In the section on “Media Interviews”, for example, we walk you through the things you need to think about as you prepare to talk to a reporter; the questions to ask ahead of time, how to prepare a series of key messages, even how to dress if you are going to be on TV. The idea is to break down some of the mystique surrounding the interview, to let you know what to expect and to help you prepare as fully as possible.

If you are going to be asked questions about stem cell research there’s a section in the Toolbox called “Jargon-Free Glossary” that translates scientific terms into every-day English, so you can talk about this work in a way that anyone can understand.

There’s also a really wonderfully visual infographic on the things you need to know when thinking about taking part in a clinical trial. It lays out in simple, easy-to-follow steps the questions you should ask, the potential benefits and problems of being in a trial, including the risks of going overseas for unproven therapies.

The Toolbox is by no means an exhaustive list of all the things you will need to know to be an effective advocate, either for yourself or a friend or loved one, but it is a start.

We would love to hear from you on ways we can improve the content, on other elements that would be useful to include, on links to other sites that you think would be helpful to add. Our goal is to make this as comprehensive and useful as possible. Your support, your ideas and thoughts will help us do just that. If you have any comments please send them to info@cirm.ca.gov

Thomas Carlyle, the Scottish philosopher, once wrote: “Man is a tool-using animal. Without tools he is nothing, with tools he is all.” That’s why we want to give you the tools you need to be as effective as you can. Because the more powerful your voice, the more we all benefit.

The eyes have it: a video guide to stem cells

We are visual creatures. Our eyes are essential tools in getting information to our brain to help us learn and understand. For example, visuals are processed about 60,000 times faster in the brain than text is, and some 60 percent of us are visual learners, meaning we respond better to visual information than to plain text.

ben paylorThat’s why a series of 8 videos, just completed by Ben Paylor and Mike Long at InfoShots, are so wonderful. They are fun, simple and engaging videos that help make some of the complex science around stem cells readily understandable thanks to the use of eye-catching animation and simple, everyday English.

The videos are short, all around one minute (less time than it takes to make a cup of tea). Each video has a single theme – ‘What is a stem cell”, “What is stem cell tourism” – and they are all masterful at walking you through different aspects of stem cell research.

The last video in the series is about neural stem cells, which is highly appropriate considering this is Brain Awareness Week, a worldwide celebration of the brain.

The videos are a reminder that the most effective communication is often the most direct, cutting through the clutter and getting to the heart of the subject. The beauty of these is that Ben and Mike are keen to share them with as many people as possible and have made them available to anyone who wants to watch them or use them as a teaching tool.

Finally, credit for the videos also has to go to the Canada’s Stem Cell Network and the Canadian Stem Cell Foundation, which helped fund them.

Heartfelt award for brilliant young researcher

It can begin in so many different ways: a pain in the jaw, a sore neck, nausea, or more typically a crushing pain to the chest. But regardless of how it starts, surviving a heart attack is always devastating with the loss of as many as one billion heart cells and permanent damage to the heart muscle.

Dr. Reza Ardehali UCLA

Dr. Reza Ardehali
UCLA

But what if there were a way to reverse that damage, and regenerate the damaged tissue? A number of the researchers we fund are trying to find ways to do just that, and one, Dr. Reza Ardehali at the University of California, Los Angeles, has just been honored for his work in advancing that field.

Dr. Ardehali is being awarded the 2015 Douglas P. Zipes Distinguished Young Scientist Award at the American College of Cardiology’s 64th Annual Scientific Session and Expo on March 16. He is the first UCLA faculty member to receive this prestigious award.

Dr. Ardehali’s work focuses on trying to gain a deeper understanding of the role that stem cells play in the heart’s limited ability to regenerate after an injury. By identifying the mechanisms at work he hopes to be able to use that regenerative potential, and ultimately to amplify it, to treat people suffering from heart disease.

In a news release Dr. Ardehali says the award is a great honor and recognition for work that has the potential to help millions of people:

“The next step is to find out how we can regenerate injured hearts in adults by either transplanting stem cells or by enhancing the regenerative potential of the rare stem cells that already reside in the heart.”

We congratulate Dr. Ardehali on the award and look forward to following his work for many years to come.

Pioneer’s 25-year struggle to treat blindness

Being a pioneer is never easy. You are charting unknown territory, tackling problems that have defeated others before you. You have to overcome so many obstacles that at times the challenge can seem insurmountable. But for those who succeed in reaching their goal, the rewards can be extraordinary.

Graziella Pellegrini, Center for Regenerative Medicine, University of Modena, Italy

Graziella Pellegrini, Center for Regenerative Medicine, University of Modena, Italy

Last month Italian researcher Graziella Pellegrini saw 25 years of work pay off when a treatment she developed to cure a form of blindness was given approval for sale by the European Commission.

This is quite an achievement as this means her treatment, called Holoclar, is among the first commercial stem therapies in the world (the first was Prochymal, which has been approved in Canada and New Zealand for the treatment of pediatric GVHD. This drug was developed by Osiris, which was led by our current President & CEO, Dr. Randy Mills.)

Holoclar uses stem cells to help stimulate the regrowth of a cornea. It can only be used for certain rare conditions, but that in no way diminishes its importance for patients or significance for the regenerative medicine field as a whole.

Nature recently sat down with Dr. Pellegrini to talk about her work, her struggle, and the many obstacles she had to overcome to get market approval for her work.

The interview makes for fascinating reading, and is a timely reminder why this kind of groundbreaking research never goes quite as quickly, or smoothly, as one would hope.

CIRM currently has a number of projects focused treating different causes of blindness on limbal cells (the kind Dr. Pellegrini worked on) and other forms of blindness; including a project to treat macular degeneration that has been approved for a clinical trial, and a therapy for retinitis pigmentosa that we hope will be approved for a clinical trial later this year.

The search for a cure: how stem cells could eradicate the AIDS virus

It’s hard to overstate just how devastating the AIDS crisis was at its peak in the U.S. – and still is today in many parts of the world. In 1995 almost 51,000 Americans died from the disease, the numbers of new cases were at almost record highs, and there were few effective therapies against the virus.

HIV/AIDS medications

HIV/AIDS medications

Today that picture is very different. New medications and combination therapies have helped reduce the death rate, in some cases turning HIV into a chronic rather than fatal condition. But even now there is no cure.

That’s why the news that the Food and Drug Administration (FDA) has approved a clinical trial, that we are funding, aimed at eradicating HIV in the body, was so welcome. This could be an important step towards the Holy Grail of AIDS therapies, curing the disease.

The project is headed by Dr. John Zaia at City of Hope near Los Angeles. The team, with researchers from Keck Medicine of the University of Southern California (USC) and Sangamo BioSciences, plans on using an individual’s own stem cells to beat the virus.  They will remove some blood stem cells from HIV-infected individuals, then treat them with zinc finger nucleases (ZFNs), a kind of molecular scissors, snipping off a protein the AIDS virus needs to infect those cells.

It’s hoped the re-engineered stem cells, when returned to the body, will help create a new blood and immune system that is resistant to the virus. And if the virus can’t infect any new immune cells it could, theoretically, die off. Check out the video we produced a few years back about the project:

Studies in the lab show this approach holds a lot of promise. In a news release announcing the start of the clinical trial, Dr. Zaia said now it’s time to see if it will work in people:

“While we have a number of drugs that are effective in holding HIV at bay, we have nothing that cures it. In addition, for many patients, these medications come with significant long-term problems so there is a real need for a therapy that can help eradicate the virus from a patient completely. That is where our work is focused.”

Like all Phase 1 trials this one is focused on making sure this approach is safe for people, and identifying what, if any, side-effects there are from the treatment. The first group of patients to be treated consists of people with HIV/AIDS who have not responded well to the existing medications.

This is the second trial that CIRM is funding focused on curing HIV/AIDS. Our first, involving the company Calimmune, began its human clinical trial in July 2013. You can read more about that work here.

We know that the road to a cure will not be simple or straightforward. There have been too many false claims of cures or miracle therapies over the years for any of us to want to fall victim to hope and hype. It may even be that the most realistic goal for these approaches is what is called a “functional cure”, one that doesn’t eliminate the virus completely but does eliminate the need to take antiretroviral pills every day.

But when compared to the dark days of 1995, a functional cure is a world away from certain death.

How the human genome is unlocking some of the secrets of stem cells, hopefully leading to new treatments

A little over a year ago we set aside $40 million to study how variations in the human genome – the complete map of our genetic information – can affect our ability to use stem cells to treat a wide variety of diseases and disorders.

Human-Genome-Project_finalThat money helped set up the Stanford/Salk Center of Excellence in Stem Cell Genomics (CESCG) with a goal of using genomic analysis to better understand how stem cells change as they grow and become different kinds of cells, and then use that knowledge to develop new treatments for a wide variety of conditions.

Now the CESCG has just announced it is investing $11.6 million on seven different projects aimed at gaining a deeper understanding of deadly or disabling diseases and conditions, such as heart disease and autism.

As Stanford’s Dr. Michael Snyder, a co-Principal Investigator on the project, said in a news release, a major part of CESCG’s mission is to “establish a Collaborative Research Program (CRP) to support the genomics research needs of stem cell investigators in California,”

‘We don’t just provide funds we also partner with the individual researchers, providing them with the support, expertise and resources they need to conduct successful genomics analyses. We received 30 applications from throughout the State, and after peer review 7 projects were identified as the best new collaborations for the Center.”

So how does this advance stem cell science? Well, in the past researchers often depended on animal models for their work; but because results in animals don’t always translate when applied to people this was not always an effective way to work. At the University of California, San Francisco and the University of California, Los Angeles researchers Arnold Kriegstein and Gay Crooks are using genomics to better understand normal human cell identities in the brain (UCSF) and the blood (UCLA) and then applying that knowledge to help develop more accurate and more detailed stem cell-based models for us to study.

Jonathan Thomas, the Chair of our Board, says one of the best ways to do great science, is to create a great team:

“The goal of the Board in creating this program and bringing together this group of researchers was to accelerate our fundamental understanding of human biology and the ways that disease work. That knowledge will help point the way not just to new treatments but also, hopefully, to ways that those treatments can potentially be tailored to meet the needs of individual patients.”

Meryl Streep, Lindsay Lohan and the importance of staying above the fray in science communications

Carl Sagan: photo courtesy Brainpickings.org

Carl Sagan: photo courtesy Brainpickings.org

Carl Sagan, the astronomer and cosmologist (among many other things) once said: “We live in a society absolutely dependent on science and technology, and yet have cleverly arranged things so that almost no one understands science and technology. That’s a clear prescription for disaster.”

The goal of two panel discussions at the American Association for the Advancement of Science (AAAS) conference in San Jose last week was to find ways to change that: to get the public to both understand and care more about science and technology; and to get scientists to do a better job of explaining both to them.

The first challenge of course is finding scientists who want to be part of this public conversation. Dr. Nalini Nadkarni, an ecologist who studies rain forests, said for young scientists in particular it’s not just a matter of having the right skills, it’s also a matter of finding the time:

“One of the challenges of scientific engagement is that just being a scientist is a full-time job, and it’s hard to think about doing public engagement when you are trying to build a career.”

Dr. Anthony Dudo, who studies the intersection of science, media and society, says one thing universities can do is encourage outreach and engagement, maybe even make it a factor in a teacher getting tenure. He says there are a lot of researchers who are happy to do this kind of outreach – either through public talks or media interviews – and they do it for all sorts of reasons.

“Many do it because it’s something they enjoy, they consider it a civic duty, something that sparks public interest in science and raises awareness about their field. In addition some say it can enhance their own scientific reputation and increase visibility for funding.”

But there is a risk. Some scientists reported facing a backlash from colleagues who felt they were trying to hog the limelight. They fell victim to what is called the “Carl Sagan” effect, which holds that if someone is spending that much time and effort communicating science to the public they must not be a very good scientist to start with.

No one could accuse Stanford’s Dr. Noah Diffenbaugh of not being a good scientist – he specializes in studying climate change and you can read his extensive resume here –  but he is also a gifted communicator, something he says he feels is his duty:

“I feel it is my responsibility to answer questions from the public when asked, because my research group is publicly funded by taxpayer dollars through agencies like the NSF. And as a public citizen I feel responsible that if we are having a public dialogue about climate change that I should be part of that dialogue.”

But he says he is very careful to avoid taking sides in the debate. He tells of an interview he once heard where Oscar-winning actress Meryl Streep talked about the importance of keeping her personal life and beliefs private (as opposed to Lindsay Lohan’s very public private life). Streep says as an actress she wants people to be able to look at her on screen and focus on the character she is playing, and not be distracted by thinking about any very public shenanigans she may have been involved in. Diffenbaugh says a scientist’s credibility depends on them doing the same:

“I stick to the facts and don’t express personal opinions or offer advocacy positions. I feel strong that in public discussions about climate change that someone in the conversation needs to be focused on evidence. It’s a role that scientists are fundamentally equipped to play.”

But even the best communicators are finding it increasingly hard to get their message into the media these days. Fewer and fewer newspapers or TV stations have skilled, experienced health and science writers, which makes it difficult to reach the public.

Lisa Krieger is an award-winning science journalist at the San Jose Mercury News. She says she finds it challenging getting stories she wants to write into the paper because she is competing for shrinking space against stories that might seem more relevant to local readers:

“Basic science is hard to cover because readers want to know how it will benefit them directly and sometimes these things are years, or even decades, away from having any real impact on people. And that’s a hard sell to an editor to get those kinds of stories into the paper.”

Krieger says the key to getting the message out is making it personal, tell stories about real people, about the real impact something could have on someone.

While acknowledging the challenges, and risks, of being a public voice and face for science – particularly when there is so much political polarization around science these days – everyone agreed that we need more scientists who are willing and able to talk about their work in ways that will engage the public, help them understand what is being done and why they should care.

For Carl Sagan (yes, him again) the reason why scientists should engage with the public was simple; to share knowledge about the wonders of the world we live in.

“It is sometimes said that scientists are unromantic, that their passion to figure out robs the world of beauty and mystery. But is it not stirring to understand how the world actually works—that white light is made of colors, that color is the way we perceive the wavelengths of light, that transparent air reflects light, that in so doing it discriminates among the waves, and that the sky is blue for the same reason that the sunset is red? It does no harm to the romance of the sunset to know a little bit about it.”

Money matters: how investing in research advances stem cell science

Our goal at the stem cell agency is simple; to accelerate the development of successful therapies to patients with unmet medical needs. But on the way to doing that something interesting is happening; we’re helping advance the scientific understanding of stem cells and building a robust stem cell research community in California in the process.

You don’t have to take our word for it. A new paper in the journal Cell Stem Cell takes a look at the impact that state funding for stem cell research has had on scientific publications. The question the researchers posed was; have the states that fund stem cell research seen an increase in their share of scientific publications in the field? The answer, at least in California’s case, is absolutely yes.

Let’s back up a little. In the late 1990’s and early 2000’s the field of stem cell research was considered quite controversial, particularly when it came to human embryonic stem cells (hESCs). To help scientists get around some of the restrictions that were placed on the use of federal funds to do hESC research a number of states voted to provide their own funding for this work. This research focuses on four of the biggest supporters of this work: California, Connecticut, Maryland, and New York.

The researchers looked at the following factors:

  1. The percentage of scientific publications in the U.S.
  2. With at least one author from those four states.
  3. That focused on hESCs and induced pluripotent stem cells (iPSCs).
  4. Comparing the numbers from before the state funding kicked in to after.

Finally – stay with me here, we’re almost done – they compared those numbers to the number of publications for two other areas of non-controversial biomedical research, RNAi and cancer. For California the results were clear. The percentage of papers on RNAi and cancer from 1996 – 2013, that had at least one California author, stayed fairly consistent (between 15-18%). However, the percentage of papers on hESCs and iPSCS with a California author rose from zero in 1998 and 2006 (the year each was discovered) to a high of 45 percent in 2009. That has since dropped down a little but still remains consistently high.

Study graphic study code The article says the reason for this is really rather obvious: “that state funding programs appear to have contributed to over-performance in the field.”

“After the California Institute for Regenerative Medicine (CIRM) issued its first grants in April 2006, the share of articles acknowledging California funding increased rapidly. Between 2010 and 2013, approximately 55% of hESC-related articles published with at least one California author acknowledged state funding, suggesting that this funding program played an important role as California maintained and built upon its early leadership in the field.”

Connecticut also saw its share of publications rise, though not as dramatically as California. Maryland and New York, in contrast, saw their share of publications remain consistent. However, as the researchers point out, with California gobbling up so much more of the available space in these journals, the fact that both states kept their share consistent was an achievement in itself.

The researchers acknowledge that scientific publications are “only one measure of the impact of state science programs” and say it’s important we look at other measures as well – such as how many clinical trials arise from that research. Nonetheless they conclude by saying:

“This analysis illustrating the relative performance of states in the production of stem-cell-related research publications provides a useful starting point for policymakers and, potentially, voters considering the future of state stem cell funding efforts as well as others interested in state science and technology policy more generally.”

British Parliament votes to approve “three parent” baby law

After what is being described as “an historic debate”, the British Parliament today voted to approve the use of an IVF technique that critics say will lead to the creation of “three parent” babies.

UK Parliament

UK Parliament

Parliament voted 382 to 128 in favor of the technique known as mitochondrial donation, which will prevent certain genetic diseases being passed on from parents to children; diseases that can cause a wide range of conditions such as fatal heart problems, liver failure, brain disorders and blindness.

Mitochondrial donation involves replacing a small amount of faulty DNA from a mother’s egg with healthy DNA from a second woman. The technique involves taking two eggs, one from the mother and another from the donor. The nucleus of the donor egg is removed, leaving the rest of the egg contents, including the mitochondria. The nucleus from the mother’s egg is then placed in the donor egg. This means that the baby would have genes from the mother, the father and the female donor.

The vote makes the UK the first country in the world to endorse this process. It comes at the end of what supporters of the measure described in a letter to Parliament as “seven years of consultation and inquiry that have revealed broad scientific, ethical and public approval.”

Mitochondrial donation is a controversial process opposed by many religious and faith-based groups who say it creates “designer babies” because it involves implanting genetically modified embryos, and because it could result in genetic alterations that might be passed on to subsequent generations.

While many scientists support the technique some have raised concerns about it. Among those are Dr. Paul Knoepfler, a stem cell researcher at U.C. Davis, (CIRM is funding some of his work). In a recent blog on the process Paul wrote that while he is not opposed to the technique in theory, he thinks this move at this time is premature:

“There is no doubt that mitochondrial diseases are truly terrible and need to be addressed, but if the potential outcomes from the technology are still vague, there are safety concerns, and it raises profound ethical issues such as changing the human genome heritably as is the case here, then my view is that a careful approach is both practical and logical. We cannot at this time have a reasonable expectation that this technology would be safe and effective. That may change in coming years with new knowledge. I hope so.”

Supporters in the UK say the science is already good enough to proceed. Dame Sally Davies, Britain’s Chief Medical Officer, calls it the genetic equivalent of “changing a faulty battery in a car.”

Professor Lord Winston, a fertility expert at Imperial College, London, says:

“I think the case is self-evident and reasonable. This is about something that is unusual and will benefit a small number of patients. I know there are some people who think it is a slippery slope that the next thing will be choosing intelligence or blond hair, but I don’t think that. For 20 years, it’s been scientifically possible to have sex selection of embryos; we still don’t allow it in Britain apart from for heritable diseases.”

It’s important to point out that while the House of Commons passed the regulations they still have to be approved by the House of Lords before they become law. A vote is scheduled for the end of this month. Even then any future trial involving the technique will still require the approval of the Human Fertilisation and Embryology Authority (HFEA) before it can go ahead.

Even if the process is ultimately approved in the UK it will likely face an uphill battle to be approved here in the U.S. where the debate over the ethical, as well as the scientific and technical implications of the process, has already generated strong feelings on both sides of the divide.