Happy Thanksgiving from CIRM

We have a lot to be thankful for this year at CIRM, including clinical trials that have started recently testing therapies for HIV/AIDS and heart disease. We are also thankful for the passionate patient advocate community that has been so supportive of our work.

We’re taking an e-break for the holiday, returning with more stem cell news December 2. We’ll be slower than usual posting and responding to comments during that time.

Happy Thanksgiving from all of us at CIRM!

Forbes takes a look at stem cell therapies for ALS

Forbes has a good story today about a stem cell trial for ALS (Lou Gehrig’s disease) being carried out by Neuralstem. We don’t fund that company, but we do fund a disease team award to Clive Svendson at Cedars-Sinai that is a sort of son-of-Neuralstem.

The Neuralstem trial is starting with neural progenitor cells–cells that are already well on their way to becoming neurons–and injecting them into the spine of people with ALS. There, the idea is that the cells will repair the damage that is causing the disease. The company is now in the second phase of testing their approach in clinical trials.

The Forbes article makes a point of how complicated the surgery is:

This is a complicated operation (recent video), one requiring special training for the surgeon, as it involves exposing the patient’s spine and using a custom delivery system that has to counteract the subtle movements of the patient, even under anesthesia, to avoid damaging spinal nerves. According to CEO I. Richard Garr, these are the world’s first intraspinal injections, directly into the gray matter, not the spinal cavity or spinal fluid.

Svendson’s work starts the same place, but he and his team are modifying the cells so that they produce a protein called GDNF, which can protect neurons. His hope is that the cells combined with the GDNF will be even more effective than the cells alone.

This tends to be the way therapies move forward. One group tries something and has some success. Another group has an idea that can improve on the original success. And so on until you get to the most effective therapies.

Svendson talked about the Neuralstem trial and his own work recently in a Google hangout on ALS. You can watch his description of the work at about 13 minutes into the hangout video:

Amy Adams

High School students asking to learn about stem cells

You know a new technology has permeated the consciousness of the population when high school students around the country are telling their teachers they want to learn about it. Today’s students are asking to learn about stem cells, and we at CIRM have just the solution for teachers looking for tools: our high school stem cell curriculum.

At the National Association of Biology Teachers conference in Atlanta where I am presenting our curriculum I have had a constant stream of teachers from many different states come by and say things like “fantastic” and “so cool.” I don’t think I have been thanked so many times in 24 hours in my life.

They consistently voice relief and gratitude when they hear our online stem cell curriculum has an extensive backgrounder and glossary built into each unit just for the teachers. Stem cells were not taught when the vast majority of these teachers were in college. Several said they were a little fearful of the topic but felt they had to find some help to get past their trepidation and were thrilled that we were providing it.

Another consistent theme has been an excitement for the fact that they can combine our curriculum with the other content on our web site to create inquiry-based projects for their students that are the heart of the new Next Generation Science Standards.

A teacher from Alaska came up and said he had to drop by our booth because while he was here this week he assigned his student to do independent study projects based on the content on the CIRM web site. “My kids want to know about this and CIRM is the only place I can send them.”

One bit of hyperbole could make my mother proud. One teacher came up to the CIRM booth yesterday evening and said, “my friend attended the workshop you gave this afternoon and she said you changed her life, so I had to come by and meet you and get your materials.” While I doubt that any workshop is life-changing, if even one of her students goes on to become a stem cell scientist, he or she could indeed produce life-changing breakthroughs.

Don Gibbons

Best of the blog: Progress in blindness research

Vision loss at the center of the field of vision caused by macular degeneration

Macular degeneration is an irreversible form of blindness in which a person loses vision at the center of their field of view. It’s caused by progressive damage to the central retina, or macula. It’s the leading cause of blindness in Americans over 60 years old and is the second leading cause of irreversible blindness in the world (only outstripped by cataracts).

On this blog, we’ve written about how our grantees are progressing toward treatment for macular degeneration, but we wanted to pull the best of those posts into one place. Here are some of the most interesting posts if you want to learn more about macular degeneration and research by CIRM grantees.

1. Early phase stem cell trials show promise in stroke and blindness

2. Finding a therapy for macular degeneration

3. Japan to begin first human tests with induced pluripotent stem cells 

4. Stem cell therapy for vision loss: experts discuss research progress 

For a complete list of macular degeneration research we’ve funded, see the macular degeneration fact sheet on our website.

Amy Adams

CIRM grantees find new genes associated with Parkinson’s disease

Our grantees at the Parkinson’s Institute have found new genes associated with Parkinson’s disease. These will help the scientists understand the origins of the disease, develop drugs and also screen for people who are at risk.

Until now, scientists knew only a small number of gene mutations that play a role in Parkinson’s disease. These are ones that you can get screened for through organizations like 23 and Me, which look for known mutations that can put you at higher risk of some diseases.

But many people who don’t have any of those known mutations still get the disease, and some who have the mutations never show symptoms. Clearly, there’s more going on in the disease than those few known genes would suggest. Scientists have thought there are probably additional genes waiting to be discovered, as well as environmental risk factors like exposure to certain chemicals.

The Parkinson’s Institute scientists worked with the Michael J. Fox Foundation and Population Diagnosics, Inc to find the additional genes. A press release on the Parkinson’s Institute website has more about the collaboration.

It’s true that finding a gene is a big leap from finding a cure, but it’s a step in that direction. One immediate benefit is that doctors might be able to do a better job of identifying people who might be at risk of developing the disease and help them get early treatment. That doesn’t stop the disease, but can certainly  help people start managing their symptoms.

The genes will also help those scientists working to develop therapies, which is the stem cell connection in this research. We’ve funded the Parkinson’s Institute to collect skin samples from people with a genetic risk of developing Parkinson’s disease, then convert those skin cells into reprogrammed iPS stem cells and mature those into neurons.

The neurons they’ve already developed from people with Parkinson’s disease behave very differently than normal nerve cells. By studying those cells, the scientists can learn more about what causes the disease and also look for drugs that return those cells to normal function.

Now that they know about the additional genes, they can develop neurons in a lab dish from people with those mutations and compare them with other Parkinson’s neurons. Also, they might be able to learn more about whether some drugs work better in people with different mutations.

We have more about CIRM funding for Parkinson’s disease on our Parkinson’s disease fact sheet.

Amy Adams

CIRM grantees at Salk create kidney buds from human stem cells

Kidney bud created from human stem cells (in blue and green). Cells in red are mouse kidney cells. Courtesy of the Salk Institute for Biological Studies

Our grantees at the Salk Institute for Biological Studies have used stem cells to create three dimensional kidney buds in a lab dish. These miniature structures aren’t intended as replacement parts, but as little laboratories for testing drugs to treat kidney disease.

Over the years scientists have been stymied in their attempts to coax human stem cells — either embryonic or reprogrammed iPS cells — into cells of the kidney. The goal had been to generate human kidney cells in a lab dish that could then be used to screen drugs to treat kidney disease. But they cells just didn’t want to cooperate.

The team at Salk, led by Juan Carlos Izpisua Belmonte, started out by getting the human stem cells part way down the path to becoming kidney cells. Then, to get the rest of the way, placed the cells in a dish with embryonic mouse kidney cells. Those mouse cells gave the human pre-kidney cells signals they needed to fully develop into the types of three dimensional buds that you’d find in a developing kidney. This work was published in the November 17 Nature Cell Biology.

They took this approach one step further, and created iPS cells from people with a form of kidney disease called PKD. Using their technique, the group coaxed those cells to form kidney buds in a lab dish. These cells provide the first access scientists have had to directly study diseased kidney tissue in a lab dish.

A press release from Salk quotes lead study author Ignacio Sancho-Martinez, a research associate in Izpisua Belmonte’s laboratory.

“Our differentiation strategies represent the cornerstone of disease modeling and drug discovery studies. Our observations will help guide future studies on the precise cellular implications that PKD might play in the context of kidney development.”

This is one of several studies CIRM funds in which scientists are, for the first time, able to study functional human tissue in a lab dish. Now, the scientists can test drugs to improve how the cells function rather than testing the drugs in animals, which in many cases respond to those drugs very differently than humans.

CIRM funding:  Yun Xia and Keiichiro Suzuki (TG2-01158)

Amy Adams

ResearchBlogging.orgXia Y, Nivet E, Sancho-Martinez I, Gallegos T, Suzuki K, Okamura D, Wu MZ, Dubova I, Esteban CR, Montserrat N, Campistol JM, & Belmonte JC (2013). Directed differentiation of human pluripotent cells to ureteric bud kidney progenitor-like cells. Nature cell biology PMID: 24240476

Breakthrough discoveries: how iPS cells are changing stem cell science

iPS cells

When the Japan-based Alliance Forum Foundation brought its meeting to San Francisco last Friday it had two goals. The non-profit wanted to deepen the long tradition of collaboration between Japan and the U.S. and to honor Shinya Yamanaka, who won the Nobel Prize last year and who bridges the two countries with labs at Kyoto University and the Gladstone Institutes in San Francisco.

Throughout the day Yamanaka’s prize-winning idea for reprogramming adult cells into embryonic-like stem cells was referred to as game changing for all of biology. Those induced Pluripotent Stem cells, or iPS cells, were lauded as valuable research tools today and revolutionary therapies in the not too distant future.

Mahendra Rao, director of the NIH Center for Regenerative Medicine, likened iPS technology to PCR, polymerase chain reaction, a chemical trick invented 30 years ago that lets scientist amplify any desired set of genes for study. This empowered countless opportunities to ask questions about the function of genes. He said biology is viewed as pre-PCR and post-PCR, and he added that iPS is causing a similar paradigm shift. In particular he noted the opportunity to create personalized cells that can have their genes edited or corrected, if they have an inherited error.

Sandy Williams, the president of the Gladstone Institutes, ran through a scenario for how iPS cells can save society billions of dollars. He suggested that the ability to make iPS cells — and in turn nerve cells — from patients with Alzheimer’s Disease will provide such a valuable platform for drug screening that finding a drug to treat the dementia was inevitable. If such a drug was just 50 percent effective, he said it would save the U.S. $540 billion a year in costs associated with the disease by 2050.

Shortly after Sandy spoke, Nancy Stagliano, CEO of biotech company iPierian, provided strong support for his premise. She said that within 12 months the company expects to begin a clinical trail with an antibody therapy identified with an Alzheimer’s patient’s iPS cells.

All of CIRM’s grants can be sorted by the type of cell used and you can read about CIRM grants using iPS cells here.

Leonore and Leonard Herzenberg: courtesy Stanford Medical School

One of the earlier speakers made a fitting tribute to another game-changing technology and the couple who made it happen, Leonard and Leonore Herzenberg, always known on the Stanford campus when I was there as just Len and Lee. Kenichi Arai said that the cell sorting machines they invented and continued to perfect over the years brought drug development to the cellular level and made much of stem cell science possible. Len died the week before the conference, but Lee was able to attend. Stanford’s obituary for Len has more about their discovery.

Don Gibbons

Stem Cells and Society, charting a course for the future

Anytime you get two Nobel Prize winners and several world-class experts on stem cell research on the same panel, particularly when the topic of conversation is “Stem Cells and our Society”, you know you are going to hear some interesting thoughts and observations.

That was certainly the case at the World Alliance Forum conference in San Francisco where Nobel Laureates Shinya Yamanaka and Paul Berg were joined by Hiro Nakauchi (who has just joined Stanford – you can read about that in our news release and blog), Hideyuki Okano from Keio University in Japan, Deepak Srivastava of the Gladstone Institutes in San Francisco, and Irv Weissman from Stanford.

Asked whether they thought we were asking too much of a still quite young science to say that that stem cells will be able to cure currently intractable diseases, Paul Berg said “It’s a mistake to ask if we are asking too much; it’s the nature of science to ask for the moon and then figure out how to get there.”

Berg was full of praise for Proposition 71, the voter initiative that created the stem cell agency, calling it an ‘extraordinary development.” He said that helped drive the research and marveled that:

“It’s only about 8 years into its existence and to imagine we would be hearing about fixing organs and fixing hearts in the body, those are remarkable achievements. I think CIRM has been one of the most extraordinary social developments, comparable to the Manhattan project.”

But the praise was also tempered with a strong dose or realism. Onaka, Weissman and Srivastava said there is still so much we don’t understand about stem cells and how they work in the body, and we will need to figure this out if we are to be able to turn this potential into actual therapies and deliver them to the public. They said we will also have to expect to overcome some ethical questions, such as is it right to breed animals that have human organs for use in transplantation.

The scientists say we have to be open and honest with the public about the work we do and the reasons why we do it. Only by being fully transparent about the science can we expect the public to support it.

Srivastava said new approaches, new knowledge and new technologies are helping push the field of stem cell research along at a rapid pace:

“We now know a lot more about the gene networks that control cell fate in our body, and I’m confident this is going to move along, and we need to push it hard to get where we need to be.”

He said all new avenues of research face problems and obstacles but it is the nature of science to push past those in search of truth, and in search of hope. He said his commitment springs from being a pediatrician who has to talk to parents, often under very difficult circumstances:

“When your child is faced with death, it’s not an abstract policy discussion, it’s a life and death discussion, and that changes everything. People want life and will act accordingly.”

Yamanaka said that when we talk about “society” we cannot limit ourselves to thinking just about the community or country we live in, we have to think about the global society. He said we need to keep in mind that the diseases we are attempting to treat are global in nature and so the therapies we devise have to be able to be delivered on a global scale. To do anything less would be to create two worlds, a healthy developed world and a sick developing one, and that, ultimately, would not be good for any of us.

Kevin McCormack

World renown stem cell scientist moves research to Stanford University with CIRM award

Dr. Hiromitsu Nakauchi recieved a CIRM award to move his world renown stem cell research lab to Stanford University.

When sports teams lure a star player away from a rival team they don’t shy away from boasting about their new signing and what a coup it was to get him or her. The world of academic stem cell research is a tad more subdued than sports, but the players can still get quite worked up, in their own way, about a new star coming to campus.

Stanford is celebrating, and rightly so, their recruitment of Dr. Hiromitsu Nakauchi to their stem cell program. Dr. Nakauchi is considered one of the leading scientists in stem cell biology in the world, so luring him away from his post at the University of Tokyo is no small achievement. An achievement, we should mention, that was done thanks to a $6.15 million Research Leadership Award from the stem cell agency.

A news release from Stanford University quotes Irv Weissman, the director of their stem cell institute, talking about the recruitment:

“We are very excited to be bringing Dr. Nakauchi to Stanford. He is one of the world’s leading stem cell scientists. His recent discoveries that tissues and organs can be developed from pluripotent stem cells of one species in the body of an animal of another species promise an important path to using stem cell biology to advance human regenerative medicine.”

CIRM President Alan Trounson, PhD, is equally lavish in his praise of Dr. Nakauchi, calling him “an extraordinary talent,” and saying:

“I think he is one of the most accomplished scientists that I have ever met. It is going to considerably enhance our capacity to deliver the remarkable potential of stem cell science to have him working together with the Californian stem cell research community. I look forward to the outcomes of his research, as will the patients who are awaiting the delivery of these new therapies.”

As we point out in our news release on Dr. Nakauchi’s move to Stanford, this is the fifth world-class stem cell researcher we have helped recruit to California through our Research Leadership Awards. Having people of this caliber move here to carry out their research is one of the reasons why California has become a world leader in stem cell research.

Kevin McCormack

Estudiante Latina de Secundaria Fija su Interés en Células Madre / Latina High School Student Sets Her Sights on Stem Cells


(note: if the English subtitles don’t appear, press the “CC” button below the video window)

De acuerdo, mentí. Hace unas semanas escribí lo que supuestamente iba a ser el ultimo blog sobre los Premios a la Creatividad del 2013, un programa de verano financiado por CIRM que dió la oportunidad, a 70 estudiantes de 40 escuelas secundarias en California, de realizar estadas internas en laboratorios de celulas madre de nueve universidades Californianas. Después de un total de más de 300 blogs, fotos de Instagram y videos de YouTube presentados por los estudiantes, no parecía que había mucho mas que decir acerca de la continuidad del éxito del programa.

Pero se me ocurrió que a pesar de que hablamos con frecuencia sobre la maravillosa diversidad de procedencias entre los estudiantes internos , contamos la historia en un solo idioma: el Inglés . El propósito del blog de hoy es el de introducir un corto video en lengua Española que filmamos con Jazmin Penado, una estudiante senior (2014) de Balboa High School en San Francisco que se hizo su estancia como estudiante interna en la UCSF. Me hace mucha ilusión poder decir que este es nuestro primer video de CIRM en un idioma diferente al Inglés.

Nos lo pasamos realmente bien durante la sesión de vídeo con Jazmin. Su entusiasmo genuino por la investigación con células madre fue una inspiración. Y me quedé impresionado con su opinion acerca de la oportunidad de formación que CIRM ha creado con los Premios a la Creatividad. El hecho que hay una prevalencia muy alta de las enfermedades del corazón en las poblaciones hispanas y latinas, lo traduce Jasmin en la oportunidad que la investigación con células madre podría ayudar directamente a su familia, que es originaria de El Salvador:

Tengo un abuelo que tiene problemas del corazón y creo que estas células que poseen propiedades regenerativa pueden ayudar …. a mi abuelo …. con sus problemas cardíacos. 

Y extrapolando, Jazmin reconoce cómo ella podría contribuir a hacer una diferencia en su propia comunidad :

 … con este programa, he aprendido que puedo convertirme en alguien que otros aspiran a ser o que ven como un modelo a seguir porque no hay tantas mujeres latinas que quieren hacer este tipo de trabajo.

¡Buena suerte Jazmin!

=======

Ok, so I lied. A few weeks ago I submitted what was supposedly the final blog about the 2013 Creativity Awards, a CIRM-funded summer program that gave 70 students from 40 high schools throughout California the opportunity to do stem cell research internships at nine universities. After a total of 300+ blogs, Instagram photos, and YouTube videos submitted by the students, it didn’t seem like there was much left to say about the continued success of the program.

But it occurred to me that although we spoke frequently about the wonderfully diverse backgrounds of the student interns, we told that story in only one language: English. So the purpose of this blog is to introduce a short Spanish language video we filmed with Jazmin Penado, a 2014 senior at Balboa High School in San Francisco who interned at UCSF. I’m excited to say this is our first ever non-English CIRM video.

I really enjoyed our video shoot with Jazmin. Her genuine excitement about stem cell research was inspiring. And I was impressed with her perspectives about this training opportunity. With a high prevalence of heart disease in the Hispanic and Latino populations, she understands how stem cell research might directly impact her family who is originally from El Salvador:

I have a grandfather who has heart problems and I think these cells being regenerative they can help….my grandfather….with his heart problems.

And in the bigger picture, Jazmin recognizes how she can make a difference in her community:

…with this program, I have learned that I can be someone who others aspire or see as a role model because there are not so many Latina women who want to do this type of work

 Good Luck Jazmin!

Todd Dubnicoff