ALSWW focus: A NEW LOOK
The search for effective treatment opportunities is ongoing at ALSWW--from new, cutting-edge biotechnology procedures and pharmaceuticals to nutritional supplements and dietary support.
We recently created a new, updated treatment option format called ALSWW focus. Each spotlights one medication or device with detailed information about what it is, how it works and where to obtain it.
What's most important for patients is to thoroughly discuss all options with their primary physician or neurologist before use. An easy way to accomplish this is to bring a copy of the ALSWW focus sheets to medical appointments. There will be regular additions as information about other potentially effective treatments become available. We encourage you to check the ALS WORLDWIDE website regularly for updates.
BIOGEN IDEC LAW SUIT UPDATE
You may recall that Knopp Neurosciences Inc. is a nine-year-old Pittsburgh biotechnology company formed specifically to develop treatments for ALS. Their post-trial analysis of the Phase I and II R+ Pramipexole clinical trial found that certain subgroups of the patient population could notably benefit from the drug. They need the blood samples from the Phase III trial for further exploration. Biogen Idec has declined to produce the samples, claiming plans to do further research and invoking patient privacy.
The stakes of the lawsuit are high for ALS patients and families across the globe. ALSWW hopes Biogen Idec returns the samples so that biopharmaceutical scientists can build on the research done by both companies. As the Knopp suit states, "Failure to preserve the Empower bio-samples will deprive ALS sufferers of potential life-altering treatments. Holding the [data] is the prerequisite to Knopp being legally permitted to conduct any post-Empower [dexpramipexole] clinical research or even approach the FDA or other regulatory authorities about such research." More detailed information is available at the Boston Globe.
NEURALSTEM'S PLANS FOR 2013
Having finally received much-deserved approval from the FDA, stem cell company Neuralstem is proceeding with five trials throughout the world in 2013 to further test their stem cell line, NSI-566. Two Phase II trials in the U.S. will study the effects in ALS patients. The U.S. sites include Emory University and University of Michigan, both under the direction of investigator Dr. Eva Feldman. The results of the Phase I trial for ALS that concluded in February 2013 are now available.
Karl Johe, PhD, Neuralstem Chairman and Chief Scientific Officer stated that the trial "is an extremely important confirmation of the safety and tolerability of both the route of administration and our cells."
Richard Garr, J.D., Director, CEO and President of Neuralstem, expressed particular thanks to the ALS community for their passionate participation in the February 2013 FDA hearing. "The outpouring of support... clearly had an impact which trickled down to the reviewers. They responded before they had to and provided us with a very aggressive trial, which of course is exactly what is needed for this most aggressive disease." The FDA's swift approval of Neuralstem's Phase II trial and the tenacity, determination and vision of the ALS community helped make it happen.
BRAINSTORM CLINICAL TRIAL U.S. EXPANSION
In March, 2013, Israeli-based BrainStorm Cell Therapeutics reported positive results from their Phase I/II clinical trial. Principal Investigator Prof. Dimitrios Karussis, Head of the Neuroimmunology Laboratory at Hadassah Medical Center in Jerusalem, stated that six of the patients who received an intrathecal stem cell injection experienced a significantly slower decline in clinical and respiratory function in the six months following treatment, as compared to the three months preceding treatment. Results also included positive indications for safety, the principal goal for all Phase I clinical trials.
This news is a heartening reminder of the potential that stem cell technology holds for ALS patients. BrainStorm is continuing its development of NurOwn technology. Alon Natanson, NurOwn Chief Executive Officer, stated, "We are excited by these data and the potential of NurOwn to positively impact the lives of patients with ALS. We look forward to continuing to advance this potentially important therapy. To that end, we have begun a Phase IIA dose-escalating trial at Hadassah and plan to launch a multi-center Phase II trial in the USA later this year in order to further validate the results that were presented today."
BrainStorm's US expansion of clinical trials are expected to begin mid-2013. Study sites include University of Massachusetts, Massachusetts General Hospital and Mayo Clinic, Rochester, MN. Patients and caregivers should carefully and thoroughly explore these trials as a means of both contributing to ALS research and hopefully alleviating symptom progression.
Further plans include partnering with the Dana-Farber Cancer Institute in Boston, MA for the Phase II ALS trial. BrainStorm's press release states that "the Connell and O'Reilly Cell Manipulation Core Facility at Dana-Farber will produce NurOwn for the MGH and UMass Hospital clinical sites."
Our April 2013 visit with CEO Alon Natanson, Dr. Karussis and several of their colleagues from both BrainStorm and Hadassah Medical Center in Ein Kerem, Israel, gave us an even greater level of confidence in their capacity to develop and expand this promising research. More details will be available soon both in an upcoming newsletter and on the ALSWW website.
2013 WORLD STEM CELL SUMMIT AND NEWSLETTER
ALSWW continues to be closely associated with the World Stem Cell Summit and Genetics Policy Institute, developers of the annual forum for stem cell industry scientists, advocates, industry leaders and the patient community. This year's Summit will be held in San Diego, CA December 4-6. We encourage you to review the newly implemented 360 Newsletter from GPI, a quick and effective scan of recent developments in the global stem cell and regenerative medicine sector.
GROUNDBREAKING UW-MADISON STEM CELL STUDY
In more stem cell news-this time, close to home for us at Madison-based ALSWW-scientists at the University of Wisconsin-Madison turned a rhesus monkey's skin cells into early brain cells, then successfully implanted them in the monkey's brain. The experiment, published March 14, 2013 in Cell Reports, worked so well that the reprogrammed cells grafted onto the brain and appeared identical to the cells already there. Researchers were only able to identify the new cells because they had been tagged with a glowing green fluorescent protein. What makes this study even more applicable to ALS patients is the fact that the three monkeys in the study were engineered to simulate the effects of Parkinson's disease, a neurogenerative disease that shares some characteristics with ALS.
Su-Chun Zhang, MD, PhD of the Waisman Center and head researcher of the study, expressed hope that the procedure could also work in humans. "We designed this experiment specifically for mimicking future therapy in patients. That's why we used monkeys."
Supported by the National Institutes of Health, Zhang's lab is now carrying out a long-term study of the monkeys that received injections of reprogrammed cells to assess whether the cells improve symptoms. If the monkeys do show improvement, the next step would be to carry out clinical trials with human patients.
Dr. Zhang is one of the preeminent stem cell scientists working today. In fact, it was his work, in conjunction with that of Dr. Clive Svendsen, now Director of Regenerative Medicine at Cedars-Sinai Medical Center, that drew us to Madison more than eight years ago. More about the UW trial can be read at JSO Online.
PRECISION STEMCELL INVESTIGATION
From our beginning, ALS WORLDWIDE has identified, reviewed and evaluated the efficacy and safety of many stem cell procedures available throughout the world. It is safe to say that at this time, all valid stem cell protocols for ALS are currently in clinical trial status. Many others have proven to be ineffective, unsafe, misguided or grossly misrepresented.
In February 2013, Peiman Hematti, MD, ALSWW Independent Safety Medical Monitor, and co-founder Stephen Byer visited Precision StemCell in Gulf Shores, Alabama, at the request of numerous patients and neurologists. Due to the publicity and interest in Precision StemCell from in the ALS community, we felt the need to release the results of this investigation immediately.
The Precision StemCell procedure is both ineffective and potentially dangerous. Our recommendation is that patients should not consider pursuing this procedure. Following our on-site investigation, Dr. Jason Williams stated his intention to discontinue his U.S. facility. However, he plans to move his operation to Bogota, Colombia. We are hopeful Dr. Williams will cease performing this procedure in all locations. Please read our full investigative report, Precision StemCell, which discusses in detail the issues that led to this conclusion.
AMORFIX DEVELOPS FIRST DIAGNOSTIC BLOOD TEST FOR ALS
Amorfix Life Sciences has furthered the development of the first effective blood test to diagnose ALS. The test uses ultra-high affinity antibodies to detect a misfolded version of the enzyme SOD1. Dr. Neil Cashman, Amorfix CSO, Co-founder and Chairman and a neurologist-neuroscientist at McGill University in Toronto, wants to "get this much needed diagnostic test in the clinic and on the market as soon as possible." Right now, there is no diagnostic test available for ALS, so physicians must use a combination of clinical findings and indirect testing to make an ALS diagnosis. This delay frequently reduces a patient's ability to seek treatment or secure placement in clinical trials. Misdiagnoses can also occur causing patients to undergo unnecessary surgery.
Further developments are anticipated for therapeutic and vaccine treatment for ALS. A collaboration between Biogen-Idec and the Amorfix therapeutic antibody program continues to advance through preclinical animal models. Amorfix has also partnered with Pan-Provincial Vaccine Enterprises Inc. (PREVENT) to assist in the development of a vaccine against misfolded SOD1.
In a world where ALS research and the clinical trial process can feel interminably slow-moving, the ALS program at Amorfix is a heartening example of a company eager to develop and implement new ways to diagnose and treat ALS. More can be read at Canada Newswire.
Meanwhile, another researcher, Robert Bowser, Ph.D. of the Barrow Neurological Institute in Phoenix, Arizona, is also attempting to create diagnostic tests for ALS by searching for a "protein signature" of ALS in the cerebrospinal fluid (the fluid within the central nervous system that helps to nourish the brain and spinal cord.) More about Dr. Bowser's work can be found at the Phoenix Business Journal.
NEW TECHNOLOGY TRACKS EYE MOVEMENT
Tobii Technology AB, a company that is leading the way in programming for disabled users--and whose excellent eye technology products are so appropriate for ALS patients--are beginning to expand into the mass consumer market. Tobii has developed a REX device that attaches to a computer through a USB port, which includes a microprojector that emits infared light to generate a reflection pattern on the eyes--and can therefore gauge the direction of a users' eye within a millimeter. While CEO and co-founder Henrik Eskilsson expects the REX to be incorporated into gaming, it is also designed to enable Windows 8 users to do more routine tasks, such as mouse pointing and scrolling-which could make it an ideal device for ALS patients. Read more at Computing Looks to the Eyes.
But Tobii is not the only company to utilized eye-tracking: Samsung Electronic Co. and LG Electronics Inc. have both implanted eye-tracking technology in their mobile devices. These new phones are able to detect users' eye movement, then pause videos when the user is looking away from the screen or scrolling through a webpage. The technology hasn't been perfected, but with time, these phones could be eminently useful for ALS patients.
DENMARK PATIENT REGISTER
While in Denmark this June, one of our projects will be to review the Danish National Patient Register, initiated in 1977. Since then, the registry has gradually expanded and at the present time, it is considered to be the finest and most complete patient database worldwide. Since 2007, this registry has contained information on all patients in Danish Hospitals. By reviewing the Register, we hope to see whether or not there is a clear coalescence between records of the Danish ALS/MND community and those from many other parts of the world, which we have aggregated via our own patient records and the ALSWW patient survey. Over the past 12 years, we have worked with nearly 10,000 patients, and the survey has been active since 2011. The Danish National Patient Register presents an exciting opportunity to compare and analyze data in the hope of further understanding this disease. The University of Copenhagen has already conducted a study that focuses on the financial and emotional burden ALS imposes on the entire family structure using the DNPR, the results of which are relevant to providing meaningful care for ALS patients and their families.
ELECTRONIC PEDALER HELPS ALS PATIENTS TO RETAIN MUSCULARITY
Several patients have shared their positive experiences with electronic pedalers, devices that enable ALS patients to exercise their lower limbs and retain muscle function. ALS patients need to exercise for as long as possible to enhance their muscle memory and maintain strength. We recommend an electronic pedaler that we know has been effective for ALS patients, made by FootSmart. It helps improve blood flow, muscle strength, coordination and joint range motion when used for a suggested 15 minutes per day. For those ALS patients with compromised mobility, a handheld remote allows the user to control each workout. Less expensive models are also available through Amazon.
AN OASIS IN THE DESERT OF ALS
The ALS journey begins with strange symptoms, many visits to many doctors and finally, the diagnosis no one wants to hear and the isolation and confusion that often follows. This was our experience and unfortunately, is still way too common. Our job is to lessen the stress, the sense of loss and confusion and help direct families to their best options. We've come to identify ourselves in part as a conduit between patients, researchers, neurologists, scientists, opportunities, clinical trials - the full gamut of possibilities. When a newly diagnosed patient contacts us, we are available to calm nerves, suggest clinical trial opportunities, help create a supportive familial atmosphere, provide guidance and hope. This was our intent in the very beginning, and 12 years later, it continues to guide our efforts. Your phone calls, emails and Skype contacts are always welcomed. We encourage you to seek our assistance, which is offered free of charge as a service provided by ALS WORLDWIDE, a not for profit organization.
Our trip to Israel and Istanbul was filled with extraordinary and exciting meetings on current research, clinical trials and more. During much of the month of April, we were in both Turkey and Israel with medical and scientific colleagues and many patients. We reviewed, discussed and learned much more about the continuing advances being made at institutions such as Tel Aviv University, the Weizmann Institute, Hadassah Medical Center, Sheba University, Hebrew University, and through laboratories and pharmaceutical companies such as Teva, Brainstorm Cell Therapeutics, and Immunity Pharma. We're writing our experiences as fast as we can. Look for a newsletter from us in the next few weeks.
JOIN THE ALSWW ORGANIZATION
Donations to ALS WORLDWIDE support patients directly through our in-home patient and family visits, ALS clinic reviews, extensive research analysis and investigation, continuing patient consultation, and providing treatment opportunities to those in financial need. Our time, energy and funds are spent totally on behalf of ALS patients and their families. Please donate through the ALSWW website or by mail. All contributions are greatly appreciated and tax deductible.
Best to you,
Barb and Steve
April 30, 2013
PLEASE COMPLETE THE ALS WORLDWIDE SURVEY TODAY