Join us for our next installment of “Ask The Experts” on July 31st.

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Join us for our second installment of “Ask the Expert” at 12pm, PST on July 31st! This live interactive event will feature a conversation between Drs. Clive Svendsen, Robert Baloh from Cedars-Sinai, Dr. Ralph Kern, the Chief Operating Officer and Chief Medical Officer of Brainstorm Therapeutics, and CIRM Senior Science Officer Dr. Lila Collins.

In addition to the two clinical trials that CIRM is currently funding – one with Dr. Svendsen’s team and one with Brainstorm Therapeutics – they’ll also explore some of the biggest problems facing the field and where the research is heading. Dr. Ralph Kern, whois currently running a Phase 3 clinical trial for ALS we are funding, will also discuss his perspective on some of these problems as well.

This event is open to everyone and it can be accessed by simply logging onto our Facebook page at 12pm PST. We extend a special invitation to patients and the patient advocate community. Your voices and thoughts are important to us. You will be able to post comments and ask questions throughout the one-hour event, and we will do our best to get to as many of those as possible.

Like us on Facebook to get updates on this event, and others in the future at https://www.facebook.com/CaliforniaInstituteForRegenerativeMedicine/

 

 

How the Ice Bucket Challenge changed the fight against ALS

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200 people in Boston take the Ice Bucket Challenge: Photo courtesy Forbes

A couple of years ago millions of people did something they probably never thought they would: they dumped a bucket of ice cold water on their head to raise awareness about a disease most of them had probably never heard of, and almost certainly knew very little about.

The disease was ALS, also known as Lou Gehrig’s disease, and the Ice Bucket Challenge was something that went from a fun idea by a supporter of the ALS Association, to a blockbuster $220 million fundraiser. Like any good idea it sparked a backlash with critics accusing it of being a lazy way for people to feel good without actually doing anything, of diverting money from other charities, and even of just wasting water at a time of drought (at least here in California.)

But two years later we can now look back and see if those critics were correct, and if the money raised did make a difference. And the answer, I’m happy to say, is no and yes. In that order.

An article in the New Yorker magazine, by James Surowiecki, takes a look at what has happened since the Ice Bucket Challenge exploded on the scene and it has some good news:

  • Contributions to the ALS Association remain higher than before the Challenge
  • The average age of donors dropped from 50+ to 35
  • The Challenge may have helped spur an increase in overall donations to charity

All this is, of course, excellent news. But there’s an even more important point, which is that the money raised by the Challenge has helped advance ALS research further and faster than ever before.

Barbara Newhouse, the CEO of the ALS Association told Surowiecki:

“The research environment is dramatically different from what it was. We’re seeing research that’s really moving the needle not just on the causes of the disease but also on treatments and therapies.”

As an example Newhouse cites a study, published in Science  last summer, by researchers at Johns Hopkins that helped explain protein clumps found in the brains of people with ALS. Philip Wong, one of the lead authors of the study, says money raised by the Challenge helped make their work possible;

“Without it, we wouldn’t have been able to come out with the studies as quickly as we did. The funding from the ice bucket is just a component of the whole—in part, it facilitated our effort.”

And just this week the ALS Association said funding from the Challenge helped identify a gene connected to the disease.

Having been one of those who took a dunk for science – and we did ours early on, when the Challenge had only raised $4m – it’s nice to know something as silly and simple can have such a profound impact on developing treatments for a deadly disorder.

 

 

Throwback Thursday: Progress to a Cure for ALS

Welcome to our new “Throwback Thursday” (TBT) series. CIRM’s Stem Cellar blog has a rich archive of stem cell content that is too valuable to let dust bunnies take over.  So we decided to brush off some of our older, juicy stories and see what advancements in stem cell research science have been made since!

ALS is also called Lou Gehrig's disease, named after the famous American baseball player.

ALS is also called Lou Gehrig’s disease, named after the famous American baseball player.

This week, we’ll discuss an aggressive neurodegenerative disease called Amyotrophic Lateral Sclerosis or ALS. You’re probably more familiar with its other name, Lou Gehrig’s disease. Gehrig was a famous American Major League baseball player who took the New York Yankees to six world championships. He had a gloriously successful career that was sadly cut short by ALS. Post diagnosis, Gehrig’s physical performance quickly deteriorated, and he had to retire from a sport for which he was considered an American hero. He passed away only a year later, at the young age of 37, after he succumbed to complications caused by ALS.

A year ago, we published an interesting blog on this topic. Let’s turn back the clock and take a look at what happened in ALS research in 2014.

TBT: Disease in a Dish – Using Human Stem Cells to Find ALS Treatments

This blog featured the first of our scintillating “Stem Cells in Your face” video series called “Treating ALS with a Disease in a Dish.” Here is an excerpt:

Our latest video Disease in a Dish: That’s a Mouthful takes a lighthearted approach to help clear up any head scratching over this phrase. Although it’s injected with humor, the video focuses on a dreadful disease: amyotrophic lateral sclerosis (ALS). Also known as Lou Gehrig’s disease, it’s a disorder in which nerve cells that control muscle movement die. There are no effective treatments and it’s always fatal, usually within 3 to 5 years after diagnosis.

To explain disease in a dish, the video summarizes a Science Translation Medicine publication of CIRM-funded research reported by the laboratory of Robert Baloh, M.D., Ph.D., director of Cedars-Sinai’s multidisciplinary ALS Program. In the study, skin cells from patients with an inherited form of ALS were used to create nerve cells in a petri dish that exhibit the same genetic defects found in the neurons of ALS patients. With this disease in a dish, the team identified a possible cause of the disease: the cells overproduce molecules causing a toxic buildup that affects neuron function. The researchers devised a way to block the toxic buildup, which may point to a new therapeutic strategy.

New Stem Cell Discoveries in ALS Make Progress to Finding a Cure

So what’s happened in the field of ALS research in the past year? I’m happy to report that a lot has been accomplished to better understand this disease and to develop potential cures! Here are a few highlights that we felt were worth mentioning:

  • The Ice Bucket Challenge launched by the ALS Association is raising awareness and funds for ALS research.

    The Ice Bucket Challenge launched by the ALS Association is raising awareness and funds for ALS research.

    Ice Bucket Challenge. The ALS Association launched the “world’s largest global social media phenomenon” by encouraging brave individuals to dump ice-cold water on their heads to raise awareness and funds for research into treatments and cures for ALS. This August, the ALS Association re-launched the Ice Bucket Challenge campaign in efforts to raise additional funds and to make this an annual event.

  • ALS Gene Mapping. In a story released yesterday, the global biotech company Biogen is partnering with Columbia University Medical Center to map ALS disease genes. An article from Bloomberg Business describes how using Ice Bucket Money to create “a genetic map of the disease may help reveal the secrets of a disorder that’s not well understood, including how much a person’s genes contribute to the likelihood of developing ALS.” Biogen is also launching a clinical trial for a new ALS drug candidate by the end of the year.
  • New Drug target for ALS. Our next door neighbors at the Gladstone Institutes here in San Francisco published an exciting new finding in the journal PNAS in June. In collaboration with scientists at the University of Michigan, they discovered a new therapeutic target for ALS. They found that a protein called hUPF1 was able to protect brain cells from ALS-induced death by preventing the accumulation of toxic proteins in these cells. In a Gladstone press release, senior author Steve Finkbeiner said, “This is the first time we’ve been able to link this natural monitoring system to neurodegenerative disease. Leveraging this system could be a strategic therapeutic target for diseases like ALS and frontotemporal dementia.”
  • Stem cells, ALS, and clinical trials. Clive Svendsen at Cedars-Sinai is using gene therapy and stem cells to develop a cure for ALS. His team is currently working in mice to determine the safety and effectiveness of the treatment, but they hope to move into clinical trials with humans by the end of the year. For more details, check out our blog Genes + Cells: Stem Cells deliver genes as drugs and hope for ALS.

These are only a few of the exciting and promising stories that have come out in the past year. It’s encouraging and comforting to see, however, that progress towards a cure for ALS is definitely moving forward.

A Cool New Way of Raising Funds and Awareness

Raising money to help fight a disease is tough. Trying to raise awareness about the disease can be just as tough. Doing both together is positively masochistic; an experience that is often as rewarding as dumping a bucket of ice cold water over your head.

Have you taken the ALS Ice Bucket Challenge?

Have you taken the ALS Ice Bucket Challenge?

And that’s precisely what a growing number of people around the country are doing to raise awareness about—and money for research into—Amyotrophic Lateral Sclerosis (ALS) also known as Lou Gehrig’s disease. They are dumping buckets of ice-cold water on their head.

It’s called, not surprisingly, the Ice Bucket Challenge. The idea behind it is simple. You dare someone you know to dump a bucket of ice cold water over their head within the next 24 hours or make a donation to help fight ALS. Once the person you have challenged either completes the challenge or makes a donation they then challenge other people—usually three other people—to do the same. And of course there’s nothing stopping you both dumping the water on yourself and making a donation.

The idea started out with people who had ALS and their friends and family but it has quickly spread. Celebrities such as Facebook’s Mark Zuckerberg, singer/actor Justin Timberlake, TV newsman Matt Lauer and even New Jersey Governor Chris Christie have all taken the Challenge. In fact the campaign has gone viral with videos and pictures of people taking the Challenge popping up on social media – Facebook and Instagram in particular – at a bewildering rate.

It’s more than just an opportunity to laugh at a potential Presidential candidate taking a self-inflicted cold shower it’s also raising a ton of money. The ALS Association says it raised $4 million in donations between the end of July and August 12th. That’s more than three and a half times more than it raised during the same period last year. They have also added more than 70,000 new donors to their cause.

That money goes to research into finding new treatments for ALS because right now there is no effective therapy at all. It also goes to help people living with this nasty, debilitating and ultimately deadly disease.

In a blog on the ALS website Barbara Newhouse, the President and CEO of the ALS Association said:

“We have never seen anything like this in the history of the disease. We couldn’t be more thrilled with the level of compassion, generosity and sense of humor that people are exhibiting as they take part in this impactful viral initiative.”

What I love about this is not just that it is raising awareness and funds for a truly worthwhile cause but that it also shows how a little bit of creativity can create so much more interest in a disease, and the people suffering from it, than any amount of well-meaning, more traditional attempts at education.

At the Stem Cell Agency we have worked closely with our friends in the ALS Association for many years and they do terrific work (you can read about our funding on our ALS Fact Sheet). But it’s a relatively rare condition – only affecting some 30,000 people in the U.S. at any one time – so it always struggles to get people’s attention compared to bigger diseases such as Alzheimer’s or stroke. But with this campaign they have changed that. They have taken a simple idea, a simple challenge, and used it to open people’s eyes to what they can do to help fight back against a deadly disease.

I find that really refreshing. As refreshing as a bucket of water over my own head.

Kevin McCormack