New funding initiative will speed disease research

A colony of iPS cells, courtesy of Kathrin Plath at the University of California, Los Angeles.

Yesterday was an exciting day at CIRM headquarters, especially for some of our science officers who have been hard at work putting together a new three-part funding initiative. We posted those requests for applications yesterday and expect to start receiving applications in September. (You can read those requests here.)

Admittedly, the initiative doesn’t have an exciting sounding goal: we’re planning to fund groups to create, store and distribute stem cell lines with disease characteristics.

I know, storerooms full of frozen cells doesn’t have the cache of a novel disease research. But wait: those frozen cells are more exciting than they might sound.

We’ve blogged about so-called disease-in-a-dish research, most recently in Parkinson’s disease and Huntington’s disease. The idea is that in order to develop therapies for complex diseases we need to know more about what goes awry in people who get the disease. Since it’s often difficult to directly study the brain cells of someone with, say, Alzheimer’s disease or autism, scientists have turned instead to reprogrammed stem cells.

The scientists take skin cells from people with the disease they want to understand, reprogram the cells to an embryonic-like state, then mature those cells into the ones they want to study. In the case of both Parkinson’s disease and Huntington’s disease, the resulting neurons show signs of the disease. Scientists can then study those cells to understand what goes wrong, and also expose those cells to different drugs in an attempt to find a drug that eliminates symptoms.

It’s important work and is already pointing to potential drugs for several diseases. CIRM’s goal is to help speed that critical work along by funding people to collect tissue from patients, create stem cell lines and store the cell lines. That way, if a scientist wants to study a particular disease, he or she can turn to CIRM’s repository of frozen cells and get to work.

We wrote about this initiative in our annual report. You can read that story here.

A.A.

New approach to healing hearts gets approved for clinical trial

One of the goals of the stem cell agency is to bring promising therapies into clinical trials, to see if what works in the laboratory can work in people. It’s exciting but challenging work and, like all good science, it takes time. That’s why the news this week from Dr. Eduardo Marban at Cedars-Sinai Heart Institute was so encouraging.

Dr. Marban and his company, Capricor, have been given approval by the Food and Drug Administration for an Investigational New Drug (IND). CIRM provided Cedars-Sinai with a $5.5 million Disease Team Award that contributed to this research and helped move it from the lab to clinical trials.

The therapy uses Cardiosphere Derived Cells (CDCs) to reduce scarring and repair the damage caused to heart muscle by a heart attack. The cells are found in heart tissue and have the potential to change into a variety of different heart cell types.

As Ellen Feigal, MD, CIRM’s Senior Vice President for Research and Development, said in a news release that we issued, “This is the first time that research by a CIRM-funded Disease Team has resulted in an Investigational New Drug (IND) approval from the FDA, a critical step in testing promising therapies in patients.”

This may be the first time one of the Disease Teams we have funded has reached this point. But we’re confident that others will be following along soon.

K.M.

On stem cell clinics, clinical trials, and the pace of progress

We recently blogged about a Houston Chronicle story revealing a troublesome Food and Drug Administration review of the Texas company Celltex Therapeutics. This is the company that treated Texas Gov. Rick Perry last year. And in previous posts we’ve written about concerns surrounding stem cell tourism – in which unregulated companies overseas promote unproven stem cell therapies. One of these companies in Korea was recently sued for fraud (you can read details of that case here).

These concerns are balanced by the very real hope on the part of patients that stem cell therapies will one day work for them and frustration over the pace of the field.

Harold DeMonaco recently wrote an interesting column in Heath News Review about regulation of stem cell therapies, the pace of progress and his advice to people who are seeking treatment. He writes:

For the moment, if a member of my family asked about seeking this treatment at a local clinic, I would advise them to either enter into a clinical trial or explore other options. I would want to make certain that the cells were handled properly, that I received my own stem cells and not someone else’s and that the effects of the treatment were being reviewed, so that a body of knowledge could be developed as to whether or not the treatment did more good than harm. Call me cautious. I think that the phrase, “Caveat Emptor,” is appropriate here.

This advice is similar to that of the International Society for Stem Cell Research, which advocates that people research any clinic advertising stem cell therapies before signing up. Their website has a list of what to look for when evaluating possible therapies.

CIRM and ISSCR co-hosted a public seminar about stem cell tourism. You can watch that symposium here.

A.A.

What is the Value Of Advertising On Facebook?

Starting a business extremely needs plenty of cash, particularly if you wish to plug your product. this can be why you'll raise, “What is that the value of advertising on Facebook?” you are doing not want an oversized quantity of capital. However, a large proportion are subdivided into completely different functions. one among these is employed to plug your product.

Profit is that the objective of each business. a way of profiting is to advertise your product fine. In advertising though, you may want plenty of cash yet as time and energy. you may conjointly want nice minds to try to to this that suggests that you've got to allocate a budget for selling.

Because of such responsibilities, some folks interact in advertising their businesses in Facebook before bobbing up with a good advertisement for your product. however you would possibly raise whether or not advertising on Facebook is free or not. Yes, that's right. Facebook provides you with an excellent selling strategy because it is that the most wanted social networking web site nowadays.

Facebook is understood to own over sixty five million members at the instant and it's still increasing. this might be an excellent platform to let your business be known to everyone. you are doing not got to pay even one penny for this. it'll conjointly prevent large amount of your time and energy.

You will solely have to be compelled to produce a business fan page with all of your business data in it and then create innumerable friends. it'll cause many of us changing into conversant in your advertisement. The procedure is extremely straightforward. Below are the steps to try to to it:
How to create a Facebook business page:

1.Visit this site: http://www.Facebook.com/pages/create.php.

2. choose initial a class that's acceptable for your business and then enter your business name then click the “Create Page” icon.

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The fact could be a heap of individuals can make the most of this and can tend to create false advertisements. once you are the one creating the page, it's necessary that each one the knowledge you entered into the page are true or else your name can get adversely affected.

Even though you may not face serious charges if caught, you may possibly lose your purchasers. Reading the mandatory rules and rules of Facebook will assist you plenty in creating business pages. this can keep you from facing potential charges.

Parkinson's neurons susceptible to toxins, preventible with some drugs stem cell studies show

A colony of iPS cells, courtesy of Kathrin Plath at the University of California, Los Angeles.

There’s a lot of talk these days about studying diseases and finding new drugs using stem cells. The idea is so compelling that CIRM has entered into a partnership with the NIH to create the kinds of cell banks that would be needed to carry out this work, specifically in Parkinson’s disease, Huntington’s disease, and ALS (Lou Gehrig’s disease).

Now, results of that work are starting to be published. The most recent paper in the July 4 issue of Science Translational Medicine and shows that of the many different mutations that can lead to Parkinson’s disease some also cause neurons to be more susceptible to certain kinds of toxins. Drugs that normally protect cells from damage effectively protected the neurons created from people who had one Parkinson’s disease mutation, but not another.

What this means is that people who get Parkinson’s disease for different genetic reasons might also respond differently to drugs. In a press release, the NIH suggests that these findings could be used to make sure people get the right drugs for their form of the disease.

The idea behind this and other studies hinges on the fact that there is no way to directly study the neurons of people with neurological diseases like Parkinson’s, Alzheimer’s or Huntington’s disease and many others. So instead, scientists take skin cells from people with the disease. Those skin cells contain the same mutations that led to the disease in the people’s neurons. They then convert those skin cells into embryonic-like iPS cells, and mature the iPS cells into neurons. Those neurons mimic the neurons of people who have the disease, and in many cases behave very differently in a lab dish compared to neurons generated from the skin of healthy people.

With these diseased neurons to study, scientists are for the first time starting to understand the origin of many previously mysterious neurological diseases. They can also start testing drugs to see which ones eliminate symptoms of the disease in the lab.

This study came out of a collaboration between the NIH’s National Institute of Neurological Disorders and Stroke and several other funding agencies including CIRM. A similar consortium of funders sponsored related work in Huntington’s disease that we wrote about last week.

CIRM considers this work so important that we’ll soon be releasing a new round of funding to support creating and banking iPS cells from people with a wide variety of diseases beyond the three in our NIH agreement. This CIRM press release describes that program. You can also read about our banking initiative in our annual report.

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Cooper O, Seo H, Andrabi S, Guardia-Laguarta C, Graziotto J, Sundberg M, McLean JR, Carrillo-Reid L, Xie Z, Osborn T, Hargus G, Deleidi M, Lawson T, Bogetofte H, Perez-Torres E, Clark L, Moskowitz C, Mazzulli J, Chen L, Volpicelli-Daley L, Romero N, Jiang H, Uitti RJ, Huang Z, Opala G, Scarffe LA, Dawson VL, Klein C, Feng J, Ross OA, Trojanowski JQ, Lee VM, Marder K, Surmeier DJ, Wszolek ZK, Przedborski S, Krainc D, Dawson TM, & Isacson O (2012). Pharmacological Rescue of Mitochondrial Deficits in iPSC-Derived Neural Cells from Patients with Familial Parkinson's Disease. Science translational medicine, 4 (141) PMID: 22764206

Alan Trounson discusses nanomedicine and progress toward stem cell therapies

CIRM president Alan Trouson has been in Sydney, Australia at the International Nanomedicine Conference learning about how nanotechnology might benefit stem cell researchers trying to develop new disease therapies.

Nanotechnology is, essentially, the study of really small materials. In nanomedicine, those really small materials are put to use to treat patients. Tiny particles can ferry drugs or proteins to the place in the body where they are needed, for example.

Trounson was interviewed by ABC in Australia about the intersection of nanomedicine and stem cell biology. He said scientists could attach proteins to the tiny nanoparticles, and then use those to direct stem cells to mature into the type of cell that’s needed to treat the disease. He said:

“You take these very, very tiny particles. You can label them with proteins. You can actually direct cells with these nanoparticles. You can get them to move around and change their very nature."

You can listen to the whole interview here.

Trounson also discussed his role in developing in vitro fertilization technology, which is now responsible for more than five million births.

Australia is one of the 20 countries, states and foundations that have signed collaborative funding agreements with CIRM. You can read more about our collaborative funding agreement program and see a list of CIRM grants that have leveraged partner funding on our website's collaborative funding page.

A.A.

Stem cell essays pose questions about self and point to new uses for stem cells

CIRM grantee Paul Knoepfler of UC Davis has been blogging about stem cell science. As part of his interest in drumming up public engagement in science he recently held a stem cell essay contest and has posted the two winners to his blog.

In the 18 and under category he received an interesting and thoughtful piece from 14 year old Claire August of Winchester, MA. You can read her full essay here. She poses some questions about stem cell research, including this interesting take on what it means to have cells replacing those we’ve lost to disease or injury:

If someone were to acquire a body part that would change their abilities or the way the world saw them, say becoming more athletic, is that person really the same?

Are we the sum of our organs or would changing things about ourselves also change how we treat the world or how the world treats us?

In the end she concludes that the questions are worth tackling as they arise:

I believe that we should tackle these issues as they come and advance forward into exploring regenerative properties of stem cells. We deserve the choices that they someday might offer.

The older than 19 winner was Agnes Oshiro of Davis, CA, who wrote about the potential role of reprogrammed iPS cells in the field of artificial intelligence. Her full essay is here. In her piece Oshiro suggests that divisions between university departments stand in the way of innovative collaborations, such as those between stem cell scientists and artificial intelligence. She writes:

However, the desire for innovation that runs in the scientific community as a whole will undoubtedly bridge any and all differences, in the search for a better, smarter, more powerful artificial intelligence.

A.A.