The world of ALS patients and their families focuses on the most current research, theories, clinical trials taking place, potential treatment opportunities and symptom relief availability. While a great deal of information is available on this website, current updates with the most recent information can be read here. At times, brief statements are offered as well as links to other websites for in depth explanations. Steve will do his best to keep information current as he combs the internet, visits with researchers, explores bio-medical facilities and meets with patients and their families.
THE WEIZMANN INSTITUTE
While in Israel this past April, we had the privilege of meeting with Professor Michal Schwartz at the Weizmann Institute of Science, an extraordinary research institution based in Rehovot, Israel near Tel Aviv. She is a member of the Department of Neurobiology and the Maurice and Ilse Katz Chair of Neuroimmunology. We met again on May 22, 2013 at the Women in Science luncheon at the Peninsula Hotel in Chicago where Professor Schwartz was honored for her outstanding achievements.
Professor Schwartz has demonstrated that the immune system is essential for the adult brain's functional plasticity. She has shown how the immune system plays a key role in maintaining, protecting and healing the central nervous system in health, disease, and aging. Her "protective autoimmunity" theory links immunity to proteins expressed within the brain to the brain's self-maintenance activities, and for the trafficking of the selective immune cells that are needed for brain repair. This approach attributes a revolutionary role to the immune system in support of cognitive function, mental stability, stem cell renewal and repair. Her studies provide a basis for immune-based therapies and therapeutic vaccinations for spinal cord injury and chronic neurodegenerative conditions such as ALS, Alzheimer's, glaucoma and mental disorders.
Professor Schwartz shared with us the focus of her most recent research: a long-awaited ALS vaccine, which will hopefully delay the development of ALS and dramatically slow down disease progression in ALS patients. This is a groundbreaking development in ALS research with the potential to come as close to a cure as we have ever seen.
Currently, the vaccine has only been trialed on SOD1 mice, and there is always the potential for mouse success but human failure. In order to test the vaccine's efficacy in humans, Professor Schwartz, together with her outstanding team, will undertake multi-location preclinical studies. The results of her work have been submitted for publication. We will provide updated information as it becomes available both through the ALS WORLDWIDE website and directly from the Weizmann Institute.
The Weizmann Institute of Science is a world leader in multidisciplinary scientific research, from the life sciences, chemistry and physics to mathematics and computer science. The Institute was established in 1934 upon the initiative of Dr. Chaim Weizmann, a renowned chemist who was the first President of the State of Israel. Today, there are five faculties - Mathematics and Computer Science, Physics, Chemistry, Biochemistry and Biology. These in turn are divided into 17 scientific departments, 50 multidisciplinary centers, 1000 scientists and scientific staff, 1100 research students, 220 postdoctoral fellows and 400 administrative employees. Their work stimulates activity in a multiplicity of fields, including brain research, cancer research nanotechnology, renewable energy sources, experimental physics, biological physics, environmental studies, autoimmune diseases, plant sciences, photosynthesis and genetics. Achievements include Copaxone for the treatment of Multiple Sclerosis; a vaccination for Hepatitis B; the 2009 Nobel Prize in Chemistry, awarded to the Institute's Professor Ada Yonath for her work with ribosomes and antibiotics. Scientists have pioneered cancer research in Israel and designed and built the first electronic computer in the country-- one of the first in the world.
On this beautifully landscaped campus, groundbreaking medical and technological applications have emerged from basic research that include amniocentesis, sophisticated laser systems, hybrid seed production preventing transmission of disease, affinity chromatography and many other accomplishments.
As one of the leading institutions in the world, the Weizmann Institute encourages mathematicians, physicists, chemists and biologists to collaborate, create new research fields and lift science to new heights.
TRIAL RESULTS CONFIRM NEURALSTEM EFFICACY
After years of scrupulous research initiated under the aegis of Chief Scientific Officer Karl Johe and CEO Richard Garr, Neuralstem clinical trial results have recently been released. Their efforts were acknowledged on May 16, 2013 when the Washington DC chapter of the ALS Association honored Neuralstem's significant accomplishments with an outstanding achievement award, accepted by Richard Garr on behalf of the entire organization.
On May 17, 2013, Dr. Eva Feldman, Principal Investigator, presented updated data on all fifteen patients from the Phase I Neuralstem clinical trial at the Neuro Diabetes Special Medical Symposium in Bucharest, Romania. She detailed the trial design, procedure and then focused on the results. Using the ALSFRS (functional rating scale) to assess degrees of disability in ALS patients, Dr. Feldman reported that six of the fifteen patients have a stable, very slowly progressing or improved disease course at more than 700 to 850 days post-surgery. They had no bulbar features of ALS, a form of the disease that destroys motor neurons in the corticobulbar area of the brainstem in the early stages and typically progresses faster than the limb-onset ALS. Additionally, these patients all received stem cell transplantation early in the course of their disease (at an average of 2 years, 1 month after symptom onset). Two of the patients showing stabilization or improvement were among the three to receive transplants in both the lumbar and cervical regions.
Of the nine remaining patients in the trial, three subjects had a long disease course (5.6, 11.6 and 12.7 years of known disease), at the time of their transplantation, likely representing atypical ALS, and have had little change in the trajectory of their disease. Finally, Dr. Feldman reported, six of the trial patients died of their disease, 7-to-30 months after surgery. Two of these patients had features of bulbar ALS at the time of their transplantation.
Dr. Feldman summed up these results stating, "Collectively, these preliminary results suggest that intraspinal stem cell transplantation of ALS subjects, with no bulbar symptoms, early in the course of their disease, could slow disease progression and even allow for functional improvement." This stability has been achieved through minimal cell implantation. We can only imagine what the results will be when Neuralstem implants a greater number of cells into an even more effective location (higher in the spinal cord) as is now planned in its continuing clinical trials in the US and elsewhere.
The trial is under the direction of Dr. Feldman, Director of the A. Alfred Taubman Medical Research Institute and Director of Research of the ALS Clinic at the University of Michigan Health System and is being administered by Site Investigator Dr. Jonathan Glass at Emory University, Atlanta. Also essential to the procedure's success are the surgical skills of Dr. Nicholas Boulis, also of Emory. Neuralstem has received FDA approval for a Phase II clinical trial, which will be expanded to additional institutions within and outside of the U.S. There is hope of a compassionate use protocol during 2014. More about Dr. Feldman's presentation in Bucharest can be read at the Wall Street Journal.
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