Wednesday, December 15, 2010

Patrick the Optimist: Exciting Times

If you don't know why Patrick is an incurable optimist, read more about his story here.

Patrick, an artist and MND sufferer, is on a quest to create 100 portraits of fellow patients before he loses the ability to paint forever. Here is a brief glimpse into his life, an entry posted on his blog that describes his participation in a study to identify a blood-based biomarker of his disease.


Research into MND is important. Very important. Clearly this is a horrible disease that wrecks lives. It kills people before their time, slowly, cruelly, relentlessly. So on an abstract level, the importance of research is obvious. If we could understand how MND works, then we could devise a treatment or even a cure. This would save thousands of lives every year. But this importance is abstract, you can understand it on an intellectual level, but can you feel it coursing through your veins, in your soul? There is one group of people for whom the importance of research is visceral, a basic, urgent, desperate need – like the need to breathe.

These are the MND sufferers and their families, the people that live with MND on a daily basis. But why is it so important to them? Even if a discovery was made today, that was to lead to a cure, these things take years, and MND moves very fast – any discovery made today will probably be too late for them, and too late for me. So why do many sufferers spend so much of the last few months and years they have left, raising money to power this research? It is like the landowner who plants a tree, knowing he will not live long enough to walk beneath it’s boughs. We need to know that MND will be stopped, that our children’s generation will not lose five thousand people a year to the dread disease that took their father, brother, or friend.

But these are exciting times. We have some good people on our side. Some of them, like Dr Emily Goodall have lost loved ones to MND, and this drives their research, fired by love, loss and the determination that comes with grief. But most of them do not have a personal connection to MND, yet they are united by a desire, a zeal that goes beyond doing their job well. If you look into their eyes, as I have done, you will see passion in them – passion for the prize of stopping MND and passion for the chase. This is not a stagnant field, MND research is going at a breakneck pace. In the UK we have some of the best MND scientists in the world, people like Dr Martin Turner and Dr Ammar Al-Chalabi, both of whom have recently made important discoveries, which have excited me and inspired me to write this blog. Ammar, working with an international team, discovered a gene in chromosome 9 that is involved in sporadic MND. This is big news, a milestone, and may well turn out to be a big leap forward on the road to that glittering prize. And Martin’s discovery? Well, I shall tell you about that in a minute, because I was involved with that one.

It is entirely normal for patients with MND to speculate about their disease, about what caused it and why – and I am no different. In my opinion this is part of the process of taking control of the disease – not letting it control you, and is perfectly healthy. Historically the only contact a patient would have had with a neurological specialist would have been fifteen minutes with his doctor every three months. Many patients felt alienated and resentful towards the scientists involved in MND research. There was not much understanding of what they are doing or feeling of any common ground. The MND association realised this and started making moves to get patients involved in research, to help bridge the patient/scientist gap. By the time I was diagnosed, this process was in full swing, and I answered a little advert in my local branch newsletter asking for volunteers for a project in Oxford looking for a MND biomarker. The person running the project was called Dr Martin Turner.

There is currently no test for MND. Any diagnosis is made clinically, meaning that a neurologist will observe your symptoms as they progress and then test for and exclude any other disease that could produce a similar effect. A biomarker is a test for MND, it could be a blood test or a brain scan, or a number of other things. A test would speed up the diagnosis process and make it more certain, but it would also have another, very important use. Because there is no test for MND, there is no easy way of testing new MND drugs quickly and cheaply. The only way at present is to give the drug to 100 patients, give a placebo to another 100, wait 18 months and then count how many are dead in each group. This process is slow, and as you can imagine, very expensive, and limits how many new treatments can be tested. If we had a biomarker then new drugs could be tested on small groups of people in a fraction of the time, and at a fraction of the cost. This would speed up the pace of research enormously. An MND biomarker is a prize of incredible value – priceless really.

I have been going to Oxford every six months now for two years. I spend a day there each time, having cognitive tests, MRI scans, blood tests and a lumber puncture. And I get to talk to Martin Turner. Martin is kind, and generous with his time, a lovely man. I am very lucky, through talking to Martin, to have been given a window into the world of MND research, and I like what I see there. I have been inspired by Martin to read more and more about the state of MND research and the people involved in it. I now want other people to share the confidence and excitement I have in what they do.

You can imagine my excitement then, when Martin told me he had actually found a biomarker for MND, by looking inside (amongst others) my brain! It was like entering a prize draw to win a car, and then actually winning it. But then what happened next was, I had to sit on the knowledge for months while Martin feverishly prepared his paper for publication. So I was very pleased when it was finally published last week and I could tell you about it. This is amazing news, brilliant news – all the more so for me because I bore a part in its making.

The MND Association should not be forgotten here either. They got me to Oxford and they helped fund Martin’s research. But while we certainly have a lot of good people on our side, it is not an army by any means. MND research is underfunded and we need more money, a lot more money – I can not be bitter however, these are exciting times, exciting times indeed.


You can follow Patrick's blog at

Another blogger we follow, Anne Marie Schlekeway of, passed away last week. Anne's unflinching honesty about the disease and her persistent sense of humor served as an inspiration for ALS patients the world over. Our thoughts are with her family... she will truly be missed. You can read an article about Anne's story here.

Tuesday, November 23, 2010

A Brief Interview with The CEO of Brainstorm Cell Therapeutics Re: Upcoming ALS Clinical Trial

On October 11th, Brainstorm Cell Therapeutics announced that the Israeli Ministry of Health had granted clearance for a Phase I/II clinical trial using the company’s autologous NurOwn™ stem cell therapy in patients with ALS (pending completion of validation studies).

To find out more about what this means to the field, and why Brainstorm has decided to focus on ALS research, I spoke with the Director and CEO of the company, Mr. Rami Efrati. Mr. Efrati has worked with Brainstorm for three years, having previously served for seven years as Vice President of Sales, Business Development, and Marketing at NICE Systems.

In a nutshell, Brainstorm’s therapy involves extracting stem cells from a patient’s bone marrow, inducing these cells to differentiate into supportive neurological cells, and injecting these cells back into the same patient at the site of damage. Because the stem cells are taken from a patient’s own marrow, there is a reduced risk of the body rejecting them once they are re-transplanted. The adult nature of the stem cells also allows Brainstorm to sidestep any ethical or moral controversy associated with embryonic stem cell research. While the therapy has potentially far-reaching applications to a variety of neurological diseases, Brainstorm’s focus remains, for the time being, on ALS.

“Brainstorm originally started working on Parkinson’s disease. Two years ago, our company made the decision to focus our efforts on diseases which would bring us as quickly as possible to clinical trials in order to help people,” said Mr. Efrati. As ALS was among the diseases that were candidates for this targeted approach, “…we wanted to invite ALS patients to come to discuss their experience with the disease. I kept hearing the name Avichai Kremer [CEO and co-founder of Prize4Life,] and I said, ‘who is he?’ We invited him in, and we became good friends. I came to believe in the mission of Prize4Life, and believe in Avi’s fight against ALS.”

Avi’s enthusiasm that ALS research was at a tipping point was apparently quite infectious. Choosing to focus on a disease that was ripe for a breakthrough was not just a marketing decision; it was an attempt to get a promising therapy to suffering patients quickly. As Chaim Lebovits, President of Brainstorm, said shortly after the announcing of the trial, “…the greatest satisfaction comes from saving a human life, not to mention thousands of lives; G-d willing, I am hopeful we can achieve that.”

But the decision was not received well by those wary of the return on investment. ALS is considered an orphan disease, meaning that because it is so rare (a disease with orphan designation must affect fewer than 200,000 individuals according to US criteria,) there is little financial incentive for companies to invest in the research. “People asked me, ‘Are you crazy? This is not logical,’” Mr. Efrati recalled. But the figures seemed more encouraging to Brainstorm. “I explained to our skeptical investors ‘There are at least 100,000 people with ALS in the Western World [author’s note: and 600,000 worldwide]. This means an estimated $6 billion market. And our therapy has the potential to be applicable to many disorders of the central nervous system, such as Multiple Sclerosis and Parkinson’s disease’. I think they found this persuasive and worth the many risks.” All told, Brainstorm estimates the potential global market for these diseases at $214 billion.

The trial will begin after the screening of patients. “The clinical trial will be headed by medical doctors at Hadassah Medical Center, under the direction of Prof. Dimitrios Karussis,” explained Mr. Efrati. “Because this is the first time we are treating a human being, the first phase of our trial will be focused entirely on safety.”

But researchers are hopeful that after the safety of the procedure is assured, they will be able to track the efficacy of the treatment. “Imagine that soon, if a doctor tells you that you have ALS, he or she can also say that there is a treatment that will slow or stop the progression of this disease. This will be an exciting moment.”

Certainly there are thousands of eyes on this trial, eager to share in that excitement. The trial will recruit 24 patients and take place in Jerusalem. Potential candidates will be screened by a panel of doctors affiliated with the Hadassah Medical Center.

For more information on the trial, visit the National Institute of Health’s database of clinical trials here.

To learn more about Brainstorm Cell Therapeutics, you can visit their website at

For more information about the Hadassah University Medical Center, go to

Thursday, November 11, 2010

The Two Fronts

As we take time to honor the veterans who have served our country, it is important to remember that for many, the battle continues to rage long after they return home.

Men with any history of military service are at nearly a 60% greater risk of being diagnosed with ALS than men who did not serve in the military. The study by researchers at Harvard University's School of Public Health showed that military personnel have an increased risk regardless of when or where they served, and regardless of whether they experienced combat.

The United States and Canada offer benefits to veterans suffering from ALS. The US Department of Defense funds ALS research. A study by researchers at the Veterans Affairs Medical Center in Bedford, MA pointed to the link between head trauma and ALS as one possible explanation as to why ALS has been diagnosed in military veterans at higher rates. Others have posited increased exertion or exposure to chemicals as potential causes.

The connection between ALS and military service is undeniable, but the reasons behind that connection remain, for the moment, unclear. As with so many of the other mysteries associated with ALS, we are left to wonder simply, why?

Now, there is a tool that might help us answer that question. Recently, the Centers for Disease Control launched the National ALS Registry, an online platform that collects, manages, and analyzes data about people with ALS. It is hoped that the registry will help answer some of the most fundamental questions about the disease, not only telling us why certain groups are more prone to develop ALS, but also providing researchers and scientists with critical information that will improve diagnosis and care, and speed up the development of treatments.

If you or a loved one have ALS, please join the registry today. Together, we can honor the bravery of veterans and ALS patients the world over by taking another step towards a cure.

The National Amyotrophic Lateral Sclerosis (ALS) Registry: It's Here! — (800) 232-4636

If you want to learn more about the connection between ALS and the military, read this excellent paper written by the ALS Association.

Friday, October 29, 2010

Another Amazing 5K4Life!

If you weren't in Kendall Square last Sunday, you missed out on a great race! If you were, thanks for participating and/or sorry for making you sit in your car while the horde of runners zoomed by!

The second annual 5K4Life was a great success. We had 429 runners register, and we raised over $30,000 to support ALS research!

You can see a full listing of the results from the race here.

And feel free to peruse some photos from the 5K in our gallery!

All of us at Prize4Life want to thank the hundreds of people who made this event possible. In addition to all of those who ran and raised money, we had 37 extremely dedicated volunteers who helped us with everything from bag stuffing and water distribution to manning the registration tables and serving up pasta and sandwiches.

We also want to give a big shout-out to two teams in particular that really made the 5K4Life shine, Team Hammy and Team Ashley.

You've gotta love the level of enthusiasm Team Hammy brought! They were our top team fundraiser, contributing $5,510 to support our mission of accelerating the discovery of treatments and a cure to ALS.

Team Ashley
sent us scrambling for more registration cards, mobilizing 83 runners to participate in the race! The eponymous Ashley Agri had registered for the 5K4Life weeks in advance, but she was involved in a car accident that left her in critical condition and unable to compete. To show their support, Ashley's friends and family took up her mantle and ran for her. While Team Ashley was in high spirits for the race, the incident is a stark reminder of how fragile life can be and how so many of us take the ability to run or even walk for granted.

To all of those who ran or helped others run in support of the thousands of people currently afflicted with ALS who could not, we give our deepest and most heart felt thanks. And our best wishes and hopes for a speedy recovery to Ashley!

And if you are feeling bummed that you missed out, there's always next year!

Monday, October 18, 2010

ALS-TDI Hosts 2010 Leadership Summit

On October 3rd and 4th, the ALS Therapy Development Institute, our colleagues in Kendall Square, opened their doors for their sixth annual conference and open house, and Prize4Life was in attendance.

One of the great things about ALS-TDI’s Summit is the lengths they go to ensure the event is open and comprehensible to patient and families, not just researchers and scientists. As open and comprehensible as an incredibly complex and evolving scientific field can be, of course…

The research symposium portion of the Summit featured speakers such as Dr. Steve Perrin, CEO and Chief Scientific Officer of ALS-TDI, Dr. Fernando Vieira, Director of In Vivo Validation at ALS-TDI, Dr. Merit Cudkowicz, ALS Clinic Director at Mass General Hospital and Co-Founder of the Northeastern ALS Consortium, and several others. Topics ranged from drugs currently in the development pipeline and new mouse models, to the ongoing controversy over stem cells and which clinical trials show the most promise for participants.

This article in the MDA/ALS Newsmagazine covers many of the presentations and discussions in great detail. And ALS-TDI has also posted video of the conference on its website, though you do have to register with an email address to view it.

Two portions of the day were of particular interest to us at Prize4Life: Dr. Cudkowicz’s and Dr. O’Neill’s (of Biogen Idec) discussions on the need for a biomarker to advance research.

Dr. Cudkowicz acknowledged some of the frustration she has witnessed in the ALS community at the fact that the disease was discovered in the late 1800’s and yet there remains no effective treatment. But she pointed to the relatively rapid progress following the discovery of a gene associated with ALS in 1993, which brought new scientists, new minds, and new ideas into the ALS field. More recently, she discussed how the 2006 correlation of TDP-43 with sporadic ALS caused scientists studying TDP-43 in other fields, such as dementia, to begin to think about ALS and how a therapy might potentially be developed.

In order to spark a leap forward in research progress, Dr. Cudkowicz says a biomarker is needed. The vast majority of ALS treatments fail in Phase II trials. This makes the development of drugs both incredibly time-consuming and incredibly costly. A biomarker dramatically shortens the time required for drug development, thus driving down the overall cost of that development. If such a biomarker were discovered, “an explosion of [ALS] therapies would result,” according to Cudkowicz. When asked by patients what they can contribute, it is towards the discovery of a biomarker that Cudkowicz points them—encouraging them to donate blood and tissue samples which are critical tools for researchers on the biomarker quest.

Dr. Gilmore O’Neill, Vice President of Experimental Neurology at Biogen Idec, also emphasized the importance of a biomarker. He explained that any trial has three possible outcomes. A positive result is, of course, the most desirable—when a drug slows or arrests the progress of a disease. But the least desirable result is not a negative study, but rather a failed one—a study where not only does the drug not have the desired effect, but researchers cannot even be certain if the drug hit its intended target.

In essence, failed trials tell us nothing, and they do not advance research in the way that negative results can. An effective biomarker means, in Dr. O’Neill’s words, that researchers “…can throw out the garbage very quickly.” Without a biomarker, trials are forced to include more patients, must last longer, and are more likely to yield uninterpretable (failed) results.

Dr. O’Neill discussed biomarkers in the context of drug development for several other diseases—his underlying theme being the need for an ALS biomarker. At Prize4Life, we wholeheartedly agree. We believe the discovery of a biomarker for ALS is so important that we’ve put up a $1 million prize to spur on research.

And there has never been an organization more anxious for a reason to give away its money.

Tuesday, October 12, 2010

Join us on October 24th for the 5K4Life

In 16 years with the New York Yankees, Lou Gehrig played in 2,164 games. From 1925 to 1939, he played in 2,130 consecutive games. He played sick, and he played hurt. He played through slumps and stardom. Whenever Gehrig’s number was called, he stepped up to the plate. You aren’t born with a nickname like ‘The Iron Horse;’ you have to earn it. For 14 years, there was no force on heaven or earth that could keep Henry Louis Gehrig from playing baseball.

And then, on May 2nd, 1939, after months of steadily declining performance, Lou benched himself and ended his streak. In June of that year, he received the diagnosis: amyotrophic lateral sclerosis. ALS. Lou Gehrig’s disease. He would never play again.

More than 70 years later, there is still no cure for ALS. And that’s why Prize4Life was founded. Our CEO was diagnosed with the disease in 2004 at the age of 27. He knew that existing resources and research were not enough—something was needed to bring new minds and new money into the fight. Our model fills that need. Prize4Life offers large, cash prizes for targeted scientific breakthroughs that will help accelerate the search for a cure.

On Sunday, October 24th, we will host our second annual 5K4Life, a road race aimed at raising awareness and funds to help us achieve our mission and bring us closer to eradicating ALS once and for all.

The race will take place at 11AM in Cambridge Center. There will be food, refreshments, music, gifts, and prizes for the top finishers and fundraisers. For more information, or to register for the race, visit

Today, 30,000 Americans have ALS. Most will die within 3 years. The disease will strip from them all voluntary muscle movement. They will not be able to feed themselves, let alone run.

But you can. Stand in solidarity with the patients and families struggling with this disease, and join us on October 24th.

Upon his death, Gehrig’s wife Eleanor said, “It was strange because there was no particular reason to keep playing without a break, no particular compulsion–except the fascination to add one more day, one more week, whatever you lost.”

As long as there is no cure for ALS, the reason seems clear: one more day, one more week may be all some people have. But we can change that.

For more information on Prize4Life, visit us at

Monday, September 20, 2010

The Candle Problem

I have a riddle for you!

On a table, there’s a candle, a book of matches, and a box of tacks. But here’s the problem: no one knows how to fix the candle to the wall without the wax dripping onto the table below.

Representatives of the candle, match, and tack industries have poured millions of dollars into studies in order to crack the case. A $20 million effort attempted to jam tacks through the candle to attach it to the wall. A $30 million study looked into the possibility of melting the wax on one side of the candle to make it stick to the wall. Neither idea worked.

Desperate to find a solution, the Big 3 decided on a novel approach—they issued a public challenge and promised a large sum of cash to whoever could end their candle dilemma once and for all.

An out-of-the-box thinker came to the rescue. “Why not, you know, take the tacks out of the box, and use that?”


But is that how it works in real life? Dan Pink, a career analyst and former speechwriter for Vice President Al Gore, contends problem-solving is slightly more complicated than that.

In his TED speech entitled ‘The Surprising Science of Motivation,’ Pink references a study that shows how individuals that are promised a reward for solving the candle problem actually perform WORSE than their un-rewarded competitors. On the surface, the finding doesn’t really make sense—isn’t money the ultimate motivator in our market-driven society?

(By the way, if you’re not familiar with TED, it’s an incredible series of talks that allow some of the brightest minds in the world to widely share their ideas. Check it out at

There are two reasons why the candle bonuses failed. First, rewards are excellent at narrowing our focus to solve a very specific problem, but they’re not nearly as useful in helping spur broad, creative thinking. Second, a reward must be tied to some intrinsic motivation to be truly effective—unfortunately, most of the study participants didn’t feel any personal desire to keep the table wax-free, they just wanted the cash.

At Prize4Life, we’re not just throwing money at a problem. Our prizes are highly focused on specific outcomes. Find a biomarker to track the progression of the disease and/or add a promising candidate (with a powerful effect on an ALS mouse model) to the drug development pipeline, and you’ll be rewarded. We’ve taken a broad goal—curing ALS—and narrowed the focus to these key challenges that prizes can help meet. By solving these smaller problems, we are opening the floodgates, and industry will pour resources into the search for a cure.

In addition, the people competing for our prizes have a powerful intrinsic motivation. Whether they know someone with ALS or not, helping to find a cure for this horrific disease means becoming part of something important—giving a normal life to thousands upon thousands of people.

The moral of this story: money can’t solve everything. But if used right, it sure can make a difference.

For another inspiring and fascinating discussion on how and why problems should be shared with a more extensive audience of potential ‘solvers,’ watch Ted Andersen, curator of the TED conferences, in his talk entitled ‘Crowd Accelerated Innovation.’

Monday, September 13, 2010 and The French Connection

In the early 19th century, Napoleon, Emperor of the French, faced quite the dilemma. His armies were stretched across the European continent, and there was no easy way to deliver much-needed brie and escargots to the front lines.

Napoleon knew that an army marches on its stomach, and so he offered a cash prize of 12,000 francs to any inventor who could devise a cheap and effective method of preserving large quantities of food. Nicolas Appert, a French candy-maker, began experimenting with a method of preserving food by placing it in glass jars, sealing them with cork, and boiling the jars in water. In 1810, Appert presented his invention to Napoleon and was awarded the 12,000 francs.

As a general rule, the Obama administration tends to avoid any potential association with someone like Napoleon, but even an ardent Francophobe has to admit his idea of using prizes to solve big problems was a good one. And in that spirit, the White House Office of Science and Technology Policy recently announced the launch of, an online platform that solicits the ideas of ‘citizen solvers’ and applies them to a host of national problems. features over 35 challenges posed by more than 15 government agencies. Some of the challenges include:

• The Kids.Gov ‘How Do I Become President?’ Challenge:
This being one of the most frequently asked questions of, a $5,000 prize will be awarded to the creator of the best visual aid that explains the process.

• The NASA Green Flight Challenge:
In order to spark the development of an aircraft that can fly 200 miles in less than two hours using the energy equivalent of less than one gallon gasoline per occupant, NASA is offering a $1.5 million prize.

• The Progressive Automotive X Prize:
Pairing with the eponymous insurance agency, the Department of Energy is offering $10 million to any one who can build a safe, affordable, production-ready vehicle that gets 100 miles to the gallon or greater.

• The Apps4Africa Contest: Our personal favorite at Prize4Life because of its incredibly catchy title format, this challenge offers $15,000 to the designers of open digital tools that can be used to address community challenges such as healthcare, education, and governance in East Africa.

According to Tom Kalil, Deputy Director for Policy in the White House Office of Science and Technology Policy, “These challenges are just a handful of those featured on and just a taste of what’s to come. By making it simple and free to post challenges, will accelerate agency adoption of prizes as a means of spurring innovation.”

Prize4Life is eager to see this platform succeed. Just like our prizes for ALS breakthroughs can act as a tipping point in the search for a cure, so too can and similar efforts act as a tipping point for inducement prizes as a whole. The prize model has an enormous potential to leverage new investment in any number of fields and create solutions to problems that have plagued us for too long.

Nicolas Appert was not a scientist. He couldn't explain why his canning method worked; it would be 50 years before Louis Pasteur discovered why food spoiled. He was not a wealthy man. In fact, the factory that Appert bought with his prize money was destroyed by Allied soldiers when they invaded Paris, and Appert died penniless.

In so many ways, Nicolas Appert was an average man, no more than a footnote in history. But his invention has helped feed the world, saving an untold number of lives. Appert had no grand fortune, no great education, but he did have an idea that would change the course of history.

And all it took was a prize to bring it to light...

Tuesday, September 7, 2010

Cutting-edge ALS Technology

Until medicine proves otherwise, technology IS the cure…

That’s the mantra of the ALS Residence Initiative, a group spearheading the construction of a series of permanent residences specifically designed for individuals living with the fatal neurodegenerative disease, amyotrophic lateral sclerosis. The first such residence officially opened in Chelsea on August 13th, and it comes equipped with a technological payload that would make a nuclear submarine commander proud.

Each resident has a computer control panel mounted on his or her wheelchair. Controlling the computer mouse using whatever muscular movement they still possess (their head, eyes, or fingers,) the resident can issue commands that are beamed via infrared transmitters to a series of receivers scattered throughout the center. The commands are then bounced to a master computer that opens doors, turns lights on and off, draws the blinds, takes room service orders, and even operates toilets. The end result is a smart house that provides once unimaginable levels of independence to individuals suffering from neurodegenerative disease.

The center came to life through the work of Barry Berman, CEO of the Chelsea Jewish Foundation, and Steve Saling, a 41-year old former landscape architect who was diagnosed with ALS four years ago. The Chelsea Jewish Foundation operates the Leonard Florence Center for Living, a 100-bed nursing home in Chelsea. In addition to the ALS residence named after Saling, the Leonard Florence Center has also built a residence designed for multiple sclerosis patients.

Asked what he would do if he lost the ability to move his computer mouse using his eyes and head, Saling pointed to technology currently in development that would allow ALS patients to control computers with their brain waves. That technology would be the subject of the keynote speech at the 4th Annual ALS Roundtable that followed the grand opening of the residence.

Prize4Life partnered with the Massachusetts Chapter of the ALS Association to launch the Roundtables. Recognizing that there are many organizations across Massachusetts working to cure ALS, Prize4Life co-led the charge to bring these groups together so that they could share information and best practices and identify opportunities to work together towards a common cause. We are enormously proud of what the Roundtables have yielded, including the opportunity to learn from Dr. Leigh Hochberg the latest developments in the brain wave technology known as ‘BrainGate.’

BrainGate aspires to literally turn thoughts into action. A chip is placed in the brain and records signals that correspond to imagined limb movement. Decoder software and hardware then translate these signals into usable commands for an external device. In Saling’s case, the external device would be a computer mouse that would allow him to ‘think’ the cursor across the screen. But BrainGate’s imagination reaches much farther than that. Scientists hope that the technology can one day be used to control prosthetic limbs, or even coupled with electronic stimulation so that the mind can bypass damaged nerves and move once-paralyzed muscles.

But for the time being, ALS patients like Steve Saling must be content with the simpler joy of turning a light off when they are ready to sleep and merely dream of much more complex joys like walking and talking. Steve’s son, Finn, was born just one month before Steve was diagnosed. When interviewed for an article in the Boston Globe, Steve described his role as a father. “I’m right now watching my brother canoe to shore with Finn in his lap. I wish that could be me.”

While the Leonard Florence Center for Living Steve Saling Residence in Chelsea is a quantum leap forward in technology, it is, sadly, not a cure. It provides independence to be sure, but not the freedom to hold one’s child in one’s arms. To achieve that, we have no alternative but to bring together the minds, money, and breakthroughs needed to eradicate ALS.

That’s where Prize4Life comes in. Prizes have the ability to attract new ideas from new sources. And when those prizes are highly focused on results, are ours are, they can drive breakthroughs that will accelerate progress toward a cure. Awarding prizes in the biomedical field is a new, relatively unproven model. But the old ways simply haven’t gotten the job done.

Learn more about our prizes here.

For more information on the Leonard Florence Center for Living, you can visit

For more information on BrainGate, visit

Monday, August 2, 2010

Drug Development for Neurodegenerative Diseases Part 4

This next installment of blog coverage from the Drug Development for Neurodegenerative Diseases conference organized by marcus evans focuses on the much buzzed about topic of regeneration.

Neurodegenerative diseases such as Alzheimers Disease, Parkinson’s Disease, Huntington’s Disease and Amyotrophic Lateral Sclerosis (ALS) are characterized by a significant and increasing loss of neurons. In the case of ALS, loss of the neurons that control voluntary muscle movement (motor neurons) underlies the progressively debilitating symptoms experienced by ALS patients. Even before patients start to notice problems with their motor function, changes have already begun to occur. It is estimated that 30-50% of a patient’s motor neurons have already degenerated by the time an ALS diagnosis is made.

As neurons begin to die, the remaining motor neurons can partially compensate for this loss (by increasing the strength and number of connections to the target muscle), but eventually the tipping point comes when too few neurons remain to properly control the muscle and clinical symptoms begin to manifest.

What can we do about this? Ah, regenerative medicine!

Speaker Judith Kelleher-Anderson, President and Chief Scientific Officer at Neuronascent, Inc., described to the audience that much of the focus in regenerative medicine has been placed on invasive therapies involving transplantation of healthy cells into the diseased area. These studies have led to a handful of clinical trials in which preparations of either human embryonic stem cells or human neural stem cells have been surgically transplanted in patients with Parkinson’s Disease, spinal cord injury (SCI) and more recently ALS.

After transplantation, these healthy cells face the formidable challenges of both maturing into the appropriate type of cell - a dopaminergic neuron for PD, an oligodendrocyte for SCI, or a motor neuron for ALS - and then rewiring themselves back into the existing cellular network. Functional recovery for patients depends on both of these steps happening.

The invasive nature of these regenerative therapies and additional safety and efficacy concerns (ie: potential to form tumors, uncertainty whether stem cells will integrate and restore function, and tissue graft rejection) have caused some researchers to pause and consider if there is another, less traumatic way to replace damaged cells in the CNS.

Kelleher-Anderson and her colleagues at Neuronascent are among the researchers considering an alternative approach in regenerative medicine. This line of research takes advantage of the revolutionary discovery made in the 1990s that, just as adults have the ability to generate new muscle, skin, and blood cells, adult brains have the ability to generate new neurons (you have to scroll down a half a page to get to the reference). This remarkable property opens up another avenue for therapeutic intervention for treating neurodegenerative disease. Rather than surgically adding back neurons, perhaps it is possible to deliver drugs that can stimulate the brain’s natural ability to generate more neurons.

Kelleher- Anderson presented some early results from her team at Neuronascent. They have identified a candidate small molecule drug that seems to enhance the ability of human neural stem cells to generate new neurons in culture. When they tested this candidate drug in a mouse model of aging, they saw that the drug could also enhance generation of neurons in the brains of treated mice and - even better - they saw that drug treatment could reverse the learning and memory deficits normally associated with aging.

Encouraged by these findings, they tested their drug in a rat model of neurodegeneration and found similar beneficial effects – new neurons were born and rats showed improvements in motor function. One exciting interpretation of these observations is that the newly generated neurons integrated into the existing neuronal circuitry to restore motor function in animals – but this awaits further studies.

The field of regenerative medicine is growing and fostering innovative ways to address the needs of patients with neurodegenerative diseases. From transplanting stem cells to harnessing the body’s own regenerative capacity, researchers are energetically working towards this goal. The road to safe and effective regenerative therapies has thus far been studded with both success and failure. Nevertheless, the road of regenerative medicine is one that many researchers and patients alike believe is worth traveling.

---Sheila Menzies

Monday, July 26, 2010

Drug Development for Neurodegenerative Diseases Conference Part 3

After a bit of a hiatus (following the departure of Meghan Kallman, our fearless former Communications Manager), we now bring you Part III of our blog series covering the marcus evans 2nd Annual Drug Discovery For Neurodegeneration Conference (see Part I here and Part II here).

Switching tracks, the next speaker, Dr. Gregory Stewart, Director of CNS Drug Therapy R&D for Medtronic, offered a slightly different perspective on therapeutic development in his talk entitled: Targeted Drug Delivery for Neurodegenerative Disease: A New Hope.

Medtronic is the world’s largest medical technology company. One of their major markets is drug-infusion pumps. With current revenues over $1.4B annually, drug delivery is a good market to be in!

Dr. Stewart discussed many of the reasons why drug developers should be thinking carefully about drug delivery. Questions of safety, cost, speed, compliance, and intellectual property may all be factors influencing drug delivery. As one example, if a patient takes morphine orally for pain relief, they need 300 times (30,000%!!!) more drug then if the morphine were delivered locally (via a pump for example).

As another example, patients taking a drug orally can get effects (including side effects) anywhere in their bodies, as most organs of the body will be exposed to a given drug (whereas, as Stewart pointed out, using a pump limits exposure of a given drug to the organ of interest). This can be a good thing, as when you take an aspirin for a headache and then go on to stub your toe (the aspirin can affect the pain in both places) but can be less good when you take that same aspirin for your headache and it upsets your stomach.

Targeted delivery has the further benefit of bypassing the liver (which constantly breaks down and filters out substances in your body), which can lead to a reduction of drug dosage at the desired target. Dr. Stewart provided several other compelling benefits for use of targeted delivery for drugs, particularly when the brain is the target.

In Medtronic’s view, the key failure of biotechnology to date has been the problem of failing to understand the nuances of drug delivery. Now that we have a vastly improved understanding of delivery principles, the drug development community is poised to make great advances, particularly at the final frontier of crossing the blood-brain-barrier and getting drugs directly to the brain and spinal cord.

With the increasing number of big and bulky drugs like antibodies and cell-based therapeutics being developed for ALS, drug delivery issues will only continue to grow in importance. Dr. Stewart urged drug developers to think about drug delivery issues early in the process, as this will impact the ultimate efficacy of any candidate therapeutic.

Friday, July 23, 2010

Drug Discovery for Neurodegenerative Diseases Part II

After a bit of a hiatus (following the departure of Meghan Kallman, our fearless former Communications Manager), we now bring you Part II of our blog series covering the marcus evans 2nd Annual Drug Discovery For Neurodegeneration Conference (see Part I here and Part III here).

We thought that there were some points worth mentioning from a presentation entitled: Translational AD Drug Discovery and Development: The Potential Role of In Vivo Multi-modal Imaging Techniques (yes, it is a mouthful), presented by Dr. Feng Luo of Abbott Laboratories.

Dr. Luo started off by reminding the audience of the recent dismal track record of developing treatments targeting neurodegenerative diseases. Over the past several years, there has been a 90% attrition rate in drugs for neurodegeneration. Clearly, this high failure rate suggests that the drug development community has a problem. The question is, how do we fix it?

Dr. Luo started off his presentation by telling the audience that he and his colleagues at Abbott Labs believe that one of the keys to more effective drug development is finding better biomarkers and creating more relevant “translatable” preclinical studies. They are specifically interested in answering the question “How could we incorporate biomarkers into early [i.e. preclinical] phases of drug development to increase R&D efficiency?” He went on to explain that the approach they are most interested in is the development of more reliable animal imaging tools. In pursuing this approach, they are adapting the most widely-used human imaging (PET, MRI) methods for mice. As a test of this, they sought to characterize one of the most widely-used mouse models of Alzheimer’s Disease (AD), the Tg2576 mouse.

It has long been observed that the human Alzheimer’s brain shows lowered energy usage (hypometabolism). Luo’s group was interested in exploring whether this hypometabolism could serve as a preclinical biomarker; however, no one had ever looked to see whether the Tg2576 mice display this feature of AD. Using three different imaging methods (FDG-PET, fMRI, and MRS) Luo and his group analyzed brain metabolism of the Tg2576 mouse over time. They were shocked to find that 7 month old Tg2576 mice, the age in which animals normally begin to display Alzheimer’s symptoms, not only failed to show decreased brain metabolism, but were instead hypermetabolic (meaning rather than using less energy, the brains of these mice were using more energy)!

As these studies showed, the Tg2576 mouse does not model all aspects of the disease in humans, suggesting that the Tg2576 mouse model may be of limited use in the development of imaging-based disease biomarkers for AD. As Dr. Luo noted in his presentation, animal models are often poorly characterized from a translational perspective. If we are to be able to assess the true utility of any potential therapy, it is critical to show that the therapy acts at its intended target early in the discovery and development process.

This finding in the AD model calls attention to the critical importance of careful characterization of animal models used to research other neurodegenerative diseases such as ALS. While it is generally accepted in the medical research community that there is no perfect animal model for any human disease (meaning disease in an animal will never perfectly mimic disease in a human), developing a detailed description and understanding of all relevant aspects of a given animal model enables researchers to understand the limitations inherent to any particular model so that results can be properly interpreted. The availability and use of multiple animal models for pre-clinical testing increases the probability of finding a drug that will translate to humans.

Although imperfect, animal models have long served as valuable drug development tools, leading to FDA approval of multiple important drugs now widely and safely used in humans. Fortunately for those interested in breakthroughs in ALS therapeutics, extensive characterization of the SOD1 mouse model by researchers at ALS TDI and many other institutions have enabled better and more interpretable studies using this valuable drug development tool. ALS TDI recently announced that extensive characterization of the new TDP-43 mouse model of ALS is also underway (

Monday, June 28, 2010

ALS Drug Development Update

It can be difficult to keep abreast of all of the news that is relevant to research and treatment of ALS. Because of that, Prize4Life has designed tools to help keep researchers and the public up-to-date on what is happening in the research and drug development worlds (you can sign up for the free Weekly Digest and ALS Forum Newsletter if you want to get these alerts in your inbox). Below is a roundup of the most relevant ALS drug development news from the last six weeks. Check it out!

Invitrogen Contributes New Research for ALS
Invitrogen's stem cell business is participating in a new research program designed to find treatments for ALS. Life Technologies, Invitrogen's parent company, has partnered with the University of California San Diego and the Salk Institute for Biological Studies to research cell transplant therapies as a potential ALS treatment.

FoldRx Pharmaceuticals secures $29 million financing

FoldRx Pharmaceuticals has closed a $29 million financing deal to support a program in neurodegenerative disease. "With the addition of two top-tier venture firms to our already strong investor base, we believe we now have access to the resources necessary to commercialize tafamidis and build FoldRx into an independent, orphan-disease focused company," said Richard Labaudinière, Ph.D., President and CEO of FoldRx, in a FierceBiotech release. FoldRx Pharmaceuticals is focusing on disease-modifying small-molecule therapeutics to treat diseases of protein misfolding and aggregation, key pathological processes for many chronic neurodegenerative diseases, including ALS.

Aukera Therapeutics Named 2010 MIT Business Plan Contest Life Sciences Track Winner
Cambridge-based start-up biotech, Aukera Therapeutics, is developing Angiogenin, a novel protein therapy, to treat ALS. As the winner of MIT's Business Plan Contest, Aukera will receive seed funding and in-kind support from The Cambridge Innovation Center.

Stem Cell Therapeutics Wins US Patent
Stem Cell Therapeutics has received US patent rights for production of neural stem cells, a technology intensively investigated for its therapeutic use in ALS and other neurodegenerative diseases.

Amorfix Life Sciences and PREVENT Sign Licensing Agreement on ALS Vaccine
Amorfix has granted exclusive licensing rights to PREVENT for clinical development of its lead ALS vaccine for therapeutic purposes. Amorfix has retained the rights to develop the antibodies for diagnostic purposes.

Q Therapeutics’ Collaborators at Johns Hopkins Receive $1 Million to Study Glial Cells for Neurodegenerative Conditions
This infusion of funds is expected to accelerate characterization of Q Therapeutics’ proprietary glial cell-based therapeutic (Q-Cells®). This technology is being developed as a treatment for ALS and other neurodegenerative diseases.

ALS TDI: Changes in the Works
ALS TDI’s expertise using the SOD1 based model for target identification and drug discovery has made significant contributions to the ALS field. Recently, the non-profit ALS biotech has announced incorporation of a TDP-43 mouse model into its drug development program. This is a valuable effort that could bear dividends for the entire ALS research community.

Cytokinetics Announces Opening of a Phase IIa "Evidence of Effect" Clinical Trial of CK-2017357
Cytokinetics will begin a Phase II study of its fast skeletal muscle troponin activator, CK-2017357, for patients with peripheral artery disease and claudication. This announcement follows an ongoing Phase IIa trial in ALS patients. Cytokinetics believes this second study will further inform their understanding of possible therapeutic applications for this novel drug candidate for ALS and other skeletal muscle-associated diseases.

Neuralstem Updates Clinical Trial Progress
The first FDA approved stem cell clinical trial for ALS is reported to be on track. Neuralstem has completed safety evaluations of its first cohort of ALS patients and plans to move forward with the second cohort using a more extensive treatment regime.

Pfizer, Washington University announce collaboration
Pharma giant Pfizer grants Washington University unprecedented access to proprietary information regarding 500+ drug candidates. The collaboration is expected to uncover new uses for existing compounds.

Teva Reports Disappointing ALS Phase II Results for Talampanel
Teva’s talampanel is a no-go for ALS after discouraging Phase II data are reported. Despite demonstrating safety, talampanel did not reach its primary endpoint and will not be advanced as an ALS therapeutic.

New Virtual ALS-focused Company Aukera Therapeutics Wins MIT Award
Aukera, a finalist in the MIT $100K Business Plan Contest, wins the audience award for its development of a novel ALS therapeutic. The technology is based out of research from Harvard Medical School.

Repligen Files IND for Neurodegenerative Disease Treatment
Repligen has filed an investigational new drug application with the FDA for a Phase I study of its selective HDAC3 inhibitor, RG2833. While Repligen's initial drug development focus will be Friedreich's ataxia, RG2833 may have a broader therapeutic use for the treatment of ALS and other neurodegenerative diseases.

Monday, June 21, 2010

Global Day for ALS

As a member of the International Alliance of ALS/MND Associations, Prize4Life is pleased to participate in Global Day for ALS. Since 1997, the International Alliance has celebrated 21 June as the global day of recognition for ALS– a disease that affects people in every country of the globe.

June 21st is the summer solstice—a turning point—and every year the ALS community undertakes a range of activities to express its hope that this day will be another turning point in the search for cause, treatment and cure of this disease.

Members of the International Alliance, and those who have been touched by ALS worldwide, do a number of things to call attention to the plight of ALS patients. Some organize meetings of patients and caregivers; they put on social events, create working groups, hold fundraisers, or simply come together to reflect on being part of the global fight against ALS.

The following is a reprint of the annual letter from Gudjon Sigurdsson, the Chairman of the International Alliance of ALS/MND Associations:

Global day 2010

The fight continues for a world free of ALS/MND. This year’s global day is no different from others. We will do our best to spread the word about the damn thing we all fight against. Soon we will celebrate this as the day we remember as the day we won the fight and got rid of ALS/MND.

Until then we all must do our best to enjoy every moment of each day. As well as we possibly can. “A glass full of darkness and sorrow is always by our hand. A bit further is a glass filled with light and opptimism. Reach for it.” These words of an Icelandic poet who died from ALS/MND, are worth keeping in mind.

My wife and I are celebrating our 25 years anniversary of marriage this month. I am very lucky she has put up with me all this time. We have agreed that this is only possible if both do their best to make it last and be enjoyable. Friendship and connections with others does not happen by itself. No, we need to work on it. Communicate and tell people we love, that we do.

In those times of crisis we need to defend our rights, find new ways of doing things and do not forget we are here for all the PALS around the world. Making it possible for them to enjoy life, support research and inprove the law and regulations in our home country.


Gudjon Sigurdsson
Chairman, International Alliance of ALS/MND Associations

Global Day is a day of commemoration and strength-building. It, like ALS Awareness Month here in the United States, is an opportunity to assert the resilience of the human spirit over the ravages of ALS.

Prize4Life believes deeply in this resilience, and makes change our business. We know that it is a priority to assist those in need right now, and our colleagues in patient service programs are doing a spectacular job of that. Other organizations, such as the ALS Association and the MDA, fund researchers upfront, and their support has done incredible things for the progression of ALS science.

Prize4Life’s programs are intended to speed up this work by providing resources and incentives that may not otherwise be available to researchers and to the ALS community. Our prizes are intended to foster creativity and new approaches (see the range of talented teams who are currently competing here). This blog, for example, is designed to bring different stakeholders and opinions together to discuss various elements of the ALS, R&D, and philanthropy spaces. Our ALS Forum is a free news source for patients and researchers alike, a cutting-edge resource that gathers the most up-to-date ALS research information in one place.

We are proud of these contributions, and we are always on the lookout for other ways to bring innovative parties and stakeholders together.

What will you do to help win this battle? For a list of ideas, click here. Or email us!

Thursday, June 10, 2010

Prize4Life holds Art, Life, Spring Gala and Art auction to raise funds for ALS

On Thursday, May 27, 2010, Prize4Life held its inaugural A.L.S. Gala and Art Auction at the Roger Smith Hotel in New York City. Sponsored by Bloomberg LP, Vertex Pharmaceuticals, Biogen IDEC, and Bristol-Myers Squibb, the event raised over $110,000 towards Prize4Life’s innovative efforts to find a cure for this devastating neurodegenerative disease (for more on our programs, click here).

The evening included dinner and an auction, led by Sotheby’s auctioneer Jamie Niven (son of David Niven, the famed actor who passed away from ALS). Mayor Bloomberg presented the evening’s opening remarks, and attendees included Nobel laureate Dr. Torsten Wiesel, Asaf Shariv, the Consul General of Israel, and renowned sports painter Bill Lopa.

Mayor Bloomberg presented the evening's opening remarks

Sotheby's Jamie Niven led the A.L.S. Art auction

A companion cocktail party and art showing, held at Sotheby’s on May 20th, also drew a large crowd, including guest of honor Gabriella Shalev, the Israeli Ambassador to the United Nations, who graciously gave an introduction to the evening. Guests sipped champagne, watched a short video on Prize4Life's co-founder and CEO Avi Kremer, and enjoyed the art that was hung for display.

Prior to the auction, the Huffington Post ran an emotive op-ed by artist Miriam Cabessa on her work's physical qualities, and its relationship with ALS. See that piece here. Photographs from both events are available here.

Works auctioned on May 27th included Gold Landscape, by Miriam Cabessa, At the Beach by Natan Elkanovich, Hanna & Shemu'el, by Boaz Vaadia, Ruth and Gehrig by Bill Lopa, The Unveiling by MuKha, Window Box, 2002 by Tim Prentice, Untitled #100 by Leora Laor, and Boy's Head, by Itmar Jobani.

Thanks to all who attended and helped make both events such a great success!

Wednesday, May 19, 2010

Drug Development for Neurodegenerative Diseases Conference Part 1

Prize4Life is pleased to bring you exclusive coverage as the conference blogger for Drug Development for Neurodegenerative Diseases, a conference organized by marcus evans. With over 40 representatives from industry and academia, the conference is focused on new therapeutic developments, biomarkers, target identification, and issues in clinical trial design for neurodegenerative disease. Without further adieu we bring you the round-up from day one….

The first set of sessions focused on current updates in therapy development for neurodegenerative treatment.

First up was a joint presentation entitled: Protein Kinase C: A Basis for Biomarkers and Therapeutics for Alzheimer’s Disease co-presented by Drs. C. Anthony Altar and Daniel Alkon of the Blanchette Rockefeller Neuroscience Institute.

This first set of presentations focused on a signaling pathway that may serve as a new target for Alzheimer’s Disease (AD) and eventually other neurodegenerative conditions. A key deficit common across neurodegenerative disease is the loss of synapses, the contact and communication point between cells of the nervous system. Activation of the molecule Protein Kinase C-epsilon appears to mitigate and potentially even reverse synapse loss.

As the presenters explained, dramatic pathologic changes like plaques and tangles are late events in Alzheimer’s, “tombstones rather than precursors”. Early events, like the loss of synapses (a process that occurs well before formation of the famous plaques and tangles we usually hear about), correlate well with a given individual’s degree of dementia, but plaques and tangles don’t. Given that memory loss is the hallmark of AD, the presenters were interested in gaining a better understanding of the molecular changes that store memory: changes in the synapse.

Dr. Tony Altar, the former director of The Biomarkers Consortium for the Foundation for the National Institutes of Health, and the current Director of the Alzheimer’s Diagnostic Laboratory of the Blanchette Rockefeller Neurosciences Institute, spoke first.

He presented recent data indicating that defects in PKC-epsilon signaling can be sensitively detected in cultured skin cells called fibroblasts. Moreover, he showed that the PKC-epsilon pathway defects seen in cultured fibroblasts from AD patients were similar to those found in postmortem AD brain tissue. The implication is that these easily accessible fibroblast cells can be used to potentially diagnose and screen treatments for Alzheimer’s Disease (and potentially other neurodegenerative diseases). Using fibroblasts from both patients with presumed AD and control patients without the disease, Dr. Altar showed that his lab could use fibroblasts to distinguish these two populations with a high degree of sensitivity and specificity.

In future studies his group hopes to further refine the system so as to provide early diagnosis of AD and discriminate AD from other forms of dementia, thereby allowing better stratification of patients for therapeutic trials and treatments.

In a companion presentation, Dr. Daniel Alkon, Scientific Director of the Blanchette Rockefeller Neurosciences Institute, delved into how his group was using knowledge of the PKC-epsilon pathway to develop potentially novel AD treatments.

PKC-epsilon is a form of Protein Kinase C found almost exclusively in the brain. Dr. Alkon’s group has identified a class of compounds that can activate PKC-epsilon without any obvious toxicity and with good blood brain barrier penetrance, ideal qualities for a drug to treat diseases of the brain!

Their lead compound, bryostatin, is able to prevent and reverse the damage seen in the brains of a variety of different models of AD transgenic mice. Intriguingly, bryostatin has also been shown to induce synapse formation in an animal model of Fragile X, a disease in which animals are born missing many synapses, suggesting that this drug may be efficacious even when administered late in AD disease progression after significant synaptic loss has already occurred.

Dr. Alkon is now preparing for an FDA-approved phase II trial of bryostatin (which had previously been shown to be safe in a large cancer trial). Our reporter probed as to whether these researchers would consider testing bryostatin in motor neurons and/or ALS mouse models and was pleased to hear a yes! This identification of the critical role of certain members of the Protein Kinase C family in synapse formation and maintenance holds potential implications for all diseases and disorders in which neuronal cell communication is disrupted. A promising avenue of discovery Prize4Life will continue to track closely.

Wednesday, May 12, 2010

Untangling ALS: A Conversation with Dr. Richard Bedlock on Off-Label ALS Therapies

60-Minutes recently did a full length feature entitled “21st-Century Snake Oil: 60-minutes cameras expose con men who prey on dying victims”, discussing the dangers of untested off-label therapies that have been pushed by non-medical doctors.

“Today, quack medicine has never been bigger. In the 21st century, snake oil has been replaced by bogus therapies using stem cells. Stem cells may offer cures one day, but medical charlatans on the Internet are making outrageous claims that they can reverse the incurable,” reports CBS. The piece highlighted the case of Lawrence Stowe, a so-called stem cell doctor whose ‘clinic’, based in Mexico, alleged to cure patients of their ALS. Credible ALS researchers dismiss Lawrence Stowe’s claims as baseless.

Yet, every year, patients are taken in by incredible claims such as Stowe’s, often losing hundreds of thousands of dollars in the process.

ALS clinicians worry about the preponderance of these shams, and Dr. Richard Bedlack is at the forefront of efforts to ensure that patients have credible, scientifically-supported information to inform their decisions. Prize4Life was pleased to have the opportunity to speak with Dr. Bedlack, Director of the Duke University MDA/ALS clinic.

Dr. Bedlack is a member of the ALS Research Group (ALSRG), and a founder of two related ALSRG initiatives to promote patient safety and information: ALSuntangled, a Twitter feed, wherein ALS patients can ask questions and receive answers about off-label or alternative ALS Therapies (you can follow the feed at and a founder and moderator of a social community (see it as a NING site) that is dedicated to discussing the safety and efficacy of off-label ALS treatments (known as AOTs).

Dr. Bedlack spoke with Meghan Kallman, Prize4Life’s Marketing & Communications Manager.

MK: Thank you for being with us. To start off, why do you think there is so much confusion surrounding off-label ALS treatments?

RB: The internet has created an amazing opportunity for connecting sellers and buyers, whether it be for antique cars or for treatments for incurable diseases. Unfortunately, while the breadth of all this information is astonishing, there is often little depth. There are usually no “internet police” to check sellers’ facts. Add to this the desperation of patients with ALS (which may lower their skepticism some), and it is easy to see a recipe for potential disaster.

MK: What is the ALS Research Group? What do you do that differentiates you from other research collaborations?

RB: The ALS Research Group (ALSRG, is comprised of more than 100 ALS Clinician-Scientists from across North America. It was established in 2003 with the purpose of improving ALS patient care, research and education. Most research collaborations are designed around clinical trials. The ALSRG is broader in scope and more interested in infrastructure for patient care, research and education.

MK: The ALS Research Group, ALSUntangled, and the NING site are all related in that they help ALS patients make sense of competing information about alternative therapies. Why did you find alternative off-label therapies in the context of ALS concerning?

RB: ALSUntangled is just one of many current ALSRG projects. The goal of ALSUntangled is to use social networking to bring patients, clinicians and scientists together for examining alternative and off-label ALS treatments (AOTs).
Patients who want to use ALSUntangled can ask about AOTs via twitter or email. Specific AOTs that patients ask about are brought into an invitation-only part of the web called NING, where they are discussed among invited, accomplished ALS clinicians and scientists. Finally, a summary of the discussion with a consensus conclusion is published via free access in each issue of the ALS Journal (see for further details).

A major focus of several current ALSRG projects is to improve enrollment in ALS research studies. A survey of ALSRG investigators suggested that one reason patients decline enrollment in a research study is to pursue AOTs (see Amyotrophic Lateral Sclerosis 2008, Vol. 9, No. 5: Pages 257-265). Unfortunately, we have found that the evidence presented for the cost, efficacy and safety of AOTs is often either scant or inaccurate (Amyotrophic Lateral Sclerosis. 2009 Jun; 10(3):182-4.). Thus, we decided to form ALSUntangled to help patients better understand AOTs and make more informed decisions.

It is clear that not all AOTs, nor their proponents, are the same. When ALSUntangled goes into an investigation of a particular AOT or clinic, it does so with an open mind. Some of these therapies are being offered by proponents who genuinely believe they have something that works for ALS. Others are being offered by con men. Our goal is to not to discover the proponent’s motivation, but simply to clarify the AOT’s cost, rationale, evidence for safety and evidence for efficacy.

MK: What is the NING site? Why was it developed?

The NING is just one part of ALSUntangled. The NING site is only for invited ALS clinicians and scientists, and is used for us to privately discuss the AOTs that we are asked about. We then to come to a consensus for our publications. We wanted a site like this so that we could be sure about the credentials and motivation of those weighing in, and so we could be free to discuss each AOT without having to worry that any of us would be personally attacked because of our individual opinion.

Did you find that ALS experts were willing to participate in NING and ALSUntangled? Did you encounter particular enthusiasm or particular resistance?

RB: We currently have 66 experts from across 5 different countries participating in ALSUntangled. Those experts are also registered and active in the NING portion of the project. While most of my fellow members of the ALSRG are enthusiastic and excited about this program, it does appear that the social networking technology may be a barrier for some. We are thus building a new website which will have a forum section whereby patients and experts can even more easily discuss AOTs.

MK: Have ALS patients been using ALSUntangled? How has it been received?

RB: Now just one year old, we currently have 160 followers on Twitter, and many more patients reading our free online investigations. The project appears to be well received, and we look forward to growing in popularity and utilization when we launch our new website later this year.

MK: Why did you choose social media as an outlet for ALSUntangled? Have there been tools that you have found particularly useful?

RB: While social media such as twitter and NING are used most commonly for recreation, they are incredibly powerful tools for bringing people from widely different geographies and backgrounds together for a common purpose.

MK: What have been the results of some of the NING investigations, in addition to providing a forum for clinicians? Has the group published anything?

RB: We have published investigations on Lyme Disease, Iplex, the Hickey Wellness Center, Stem Cell Transplants at Hospital San Jose Tecnologico de Monterrey, and the XCell-Center. These can be found in the ALS Journal or on various websites (see, for example

MK: Why do you think that shams such as Lawrence Stowe’s ‘clinic’ in Mexico (featured on 60 Minutes) continue to be successful?

RB: If I did not have a scientific background, and my wife had just been diagnosed with ALS, the claims of sellers like Stowe would be awfully tempting. Again, this terrible disease really makes people vulnerable.

MK: What is the ALS clinical community doing to counteract these sorts of problems with ‘snake oil’ salesmen?

RB: Individual ALS clinicians have long fielded questions from patients about AOTs. For years they had to try and investigate these questions on their own. ALSUntangled tries to make this process easier and much more powerful. Once we publish an investigation, future internet searches of that AOT should yield not only the original advertisement for it, but also our detailed critique. Patients can then make a more informed decision as to whether they wish to pursue the AOT.

MK: Do you have any observations on how to help ALS patients avoid this kind of fraud? Are there good ways to know when the science is legitimate? What can ALS patients do to protect themselves?

Patients with ALS should always discuss any AOT they are considering with their doctor and/or an ALS expert. There are some definite red flags suggesting that a particular AOT probably should be avoided. These include a large up-front cost, use in multiple diseases with diverse causes (example: a treatment that claims to work in ALS, MS, PD, stroke, fibromyalgia, cerebral palsy), the use of case reports as evidence of effectiveness, and lack of any safety data. ALSUntangled is here to help!

We encourage ALS patients and caregivers to make extensive use of ALSuntangled. Thanks very much to Dr. Bedlack and his colleagues for creating such a valuable resource!

Tune in next Tuesday and Wednesday as we bring you live coverage of the marcus evans Second Annual Drug Development for Neurodegenerative Diseases Conference, from May 19-20 in Boston, MA. Prize4Life will be blogging live on the conference, which will feature speeches from industry professionals and leading pharmaceutical and biopharma companies. Presenters will discuss innovation in clinical studies, clinical trial designs, biomarker discoveries and drug discovery for neurodegenerative disease. To learn more or register, click here.

Monday, April 26, 2010

“Benefit Corporations”

It’s a well-known fact that most non-profits struggle with finding sufficient funding, and that most for-profit businesses are penalized for considering anything but their bottom line in the development of business strategy. Now Maryland, leading the way in what may become a national effort, has created a new legal structure for an entity that is not-quite-business, not-quite-charity. Signed into law by Governor O’Malley, the bill requires that the newly named benefit corporations create a positive impact on society. Decisions made by these entities must take into consideration potential impact on employees, localities, and the environment, rather than profit only. The law also offers legal protections to its Board members for considering social and environmental issues in decision-making processes (a similar bill recently passed in Vermont’s senate).

"For the first time, we have a market-based solution supporting investors and entrepreneurs who want to make money and make a difference," Andrew Kassoy says in an article from the Chronicle of Philanthropy. Kassoy’s non-profit, B Lab, administers a certification program for socially responsible businesses.

“The new law tackles a major concern of entrepreneurs who need to raise money to expand their social-purpose businesses but fear losing control of the companies' social or environmental mission,” the Chronicle of Philanthropy notes.

Russell Sullivan, staff director for the US Senate Finance Committee, thinks that new tax structures could create a “blurring of the lines” between charities and for-profit companies generally. Historically, the tax code endeavors to group all entities into either the for-profit or the non-profit bucket.

"We might see the emergence of some proposals to establish what I'll call, for the lack of a better term, a for-benefit corporation -- something that is in between a private taxable company that's not under our rules of C corporations or S corporations and partnerships but also not under our rules having to do with charities," he said in another Chronicle of Philanthropy article. Mr. Sullivan continued: "But I see even more blurring of the lines over the past decade." Green energy companies in some cases intend "actually to develop or promote a cleaner environment -- they are just doing it through a corporate structure."

This is an interesting approach, indicative of a changing national tax climate and approach to doing business in many industries. Prize4Life has blogged extensively on collaboration between non-profits and pharma/biotech recently (see posts here and here) in the context of drug discovery and development; could the creation of a benefit corporation be the next step for disease research? Would it follow the model of, for example, the ALS Therapy Development Alliance? What promise might such a national change hold?

Tuesday, April 13, 2010

ALS Awareness Day

On Wednesday, April 7, Prize4Life participated in the ALS Association’s ALS Awareness Day, working to raise awareness of ALS among legislators and to promote the funding of the ALS registry. Being a so-called ‘Orphan Disease’, ALS does not get the attention and funding that many feel it deserves, and this day was an opportunity to both push for awareness, and to focus priorities on a specific program goal. Above: Prize4Life staff Dr. A. Sheila Menzies, Dr. Melanie Leitner, Meghan Kallman, and Brooke Larimer at the Statehouse.

As noted in the ALS Association’s 2010 policy priorities, the Massachusetts ALS Registry is a state-wide ‘surveillance mechanism, enabling researchers to look at patterns of disease in the Commonwealth in their search for a cure (see a release on the project here). This registry is intended to help researchers study both the incidence and prevalence of the disease, identify trends, and develop additional research studies related to better understanding ALS. In the words of the ALS association: “The ALS Registry in Massachusetts brings us one step closer to a cure.”

The Registry was first authorized in Massachusetts by Section 26 of Chapter 140 of the Acts of 2003 (ie, the State budget). The budget line established a program for identifying all the ALS cases in Massachusetts through the Department of Public Health. The program was funded at $150,000 annually since it was initially authorized in 2003. However, the 2010 budget reflected a cut to less than half of that amount. The ALS Association believes that this project is too critical to let fall by the wayside, and rallied to lobby for funding in the original amount to maintain the ALS Registry.

The day opened with speeches by Debra Sharpe, president of the ALS Association. ALS advocate and patient Steven Saling, an architect working closely on the Leonard Florence Center for Living, gave a moving speech on his involvement in the center and his approach to life with ALS (the Center is a home designed especially to enable ALS patients to live independent, fulfilling lives—very cool. Check it out here). Suzanne Condon, Associate Commissioner of the Massachusetts Department of Public Health, updated the audience on the status of the state ALS Registry in Massachusetts. Dr. Tracie Caller, a researcher at Dartmouth Hitchcock, spoke about the current research taking place on the epidemiology of ALS in New England, specifically in New Hampshire.

Prize4Life believes that this is an important advocacy effort, and we were honored to participate. Following the introductory speeches, Prize4Life met with State Senator Patricia Jehlen, as well as Senator Anthony Petruccelli and his aid Donna LoConte. We also spoke with Representative Frank Smizik's staff, and left materials for Representative Lida Harkins. Additionally, Prize4Life’s Chief Scientific Officer, Melanie Leitner, was fortunate to speak with representatives from the Department of Public Health, including epidemiologist Dr. Christine Fischetti, who is working closely on the ALS registry (she signed up for our ALS Forum! Have you?) All meetings discussed the importance of supporting ALS research through this Registry effort, and the importance of focusing resources on finding a cure. Above: Brooke Larimer and Meghan Kallman with Senator Patricia Jehlen.

Organizations and individuals approached their meetings differently. One woman brought along a photograph of her mother, who had passed away from ALS, to show her elected officials, explaining through her tears why she cared so deeply about the project. Others spoke about the loved ones they had lost to the disease, while still others, ALS patients themselves and aided by canes or ventilators, arrived to advocate for themselves.

Be aware that the National ALS Advocacy Day and Public Policy Conference, is coming up, from May 9-11 in Washington, DC. May is also ALS Awareness Month—what will you do to help us find a cure?

Tuesday, April 6, 2010

ALS Clinical Trials Update

Back in January, as part of our coverage of the International ALS/MND Symposium in Berlin, Prize4Life blogged briefly about ALS clinical trials. We also featured clinical trials coverage here by Dr. Amber Dance, the science writer we sponsored through the ALS Forum, our collaboration with the Alzheimer Research Forum. And you all are wondering what has happened since then on the clinical trials front, right? Well, wonder no longer. Here’s the latest (in alphabetical order):

Ceftriaxone is an antibiotic used to treat Lyme disease, however it has also been shown to affect uptake of the neurotransmitter glutamate (which means, in English, that it may help protect brain cells which can be killed by too much glutamate) The Ceftriaxone Phase III study continues to enroll , and there is a potentially promising pre-clinical study looking at the drug’s effects for patients with spinal muscular atrophy (an infant-onset disease which shares many similarities with ALS). A 2005 study suggested that Ceftriaxone could increase lifespan, strength, and neuron survival in ALS model mice (for more information, see this review).

San Francisco, CA based Cytokinetics recently initiated a Phase IIa “Evidence of Effect” (EoE) clinical trial of their small molecule CK-2017357 for patients with ALS. The drug is reported to act on fast skeletal muscles to make them more responsive to signals from motor neurons. This means that the drug could, in essence, help muscles stay stronger and delay the loss of muscle control that accompanies ALS. In March 2010, Cytokinetics was granted orphan-drug designation for this compound by the United States Food and Drug Administration (FDA). The orphan-drug designation carries a number of benefits to help the drug reach the market sooner, including tax credits, and scientific and financial support.

Isis Pharmaceuticals in Carlsbad, California is using antisense technology to try to reduce the amount of a protein, superoxide dismutase (SOD1) that is mutated in some ALS patients. Antisense is a method by which scientists can reduce levels of a given protein and thus theoretically help in diseases thought to be related to mutant proteins, such as ALS. This approach was found to be effective in animal models (data will be presented at the upcoming American Academy of Neurology (AAN) Meeting next week) and the first patient was just enrolled in a Phase I clinical trial in March. When completed, this trial will enroll 32 subjects, and safety results will hopefully be completed in 2011.

Knopp Neurosciences
Knopp is working on a small molecule drug that is thought to protect the function of mitochondria, the energy source for all cells of the body (motor neurons are relatively huge cells so they are very sensitive to reduced energy supply). Nothing new has been reported by Knopp since the results presented at the Berlin Meeting, however, the company is scheduled to attend the AAN meeting next week. So stay tuned!


Neuralstem, Inc. of Rockville, Maryland has begun a Phase 1 safety trial utilizing stem cells derived from fetal tissue in ALS patients. The goal of the trial is to test a novel surgical method, developed by surgeon Nick Boulis, to safely deliver cells into the spinal cord. They have currently enrolled 2 patients and are planning on enrolling a dozen participants total (see ARF related news story).

TCA Cellular
TCA Cellular of Covington, Louisiana, has recently enrolled the first of 6 anticipated patients in a Phase I safety trial of its stem-cell based therapy.

While this progress on the clinical trial front looks promising, Prize4Life believes we still need other potential therapeutics in the pipeline. This is why we are excited about the impressive teams competing for the ALS Treatment Prize and why we are so focused on translational research (that is, the process of translating scientific discoveries into practical application, from the ‘bench’ to the ‘bedside’. See a full blog post on it here.) Those interested in the challenges and promise of translational neuroscience and the drug companies at the forefront of the field may be interested in an upcoming meeting happening in Boston in May:

The 2nd Annual Drug Development for Neurodegenerative Diseases conference will be held May 18-19. Convened by marcus evans, an internationally known conference organizer targeting senior industry decision-makers, the conference will feature a wide-ranging and high quality program covering innovations in pre-clinical studies and clinical trial design, biomarker discoveries, and target identification for therapeutic and drug discovery journey.

The conference will feature speakers from major drug companies, including Novartis, Merck, Pfizer, and BiogenIdec as well as some up-and-comers including Allon Therapeutics, Acorda Therapeutics, and EnVivo Pharmaceuticals (EnVivo is also one of the teams competing for the Avi Kremer ALS Treatment Prize). While the meeting will not focus exclusively on ALS, it will cover many topics and speakers in related fields that will hopefully “translate” into the ALS space. Prize4Life will be blogging live from this conference, so make sure to follow the blog for some hot-off-the-press info!