INNOVATORS
A collection of just a few of the amazing minds and organizations that made and continue to make progress possible for people with neurodegenerative disease.
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In 1981, William K. Summers first administered intravenous tacrine, first developed as an intravenous antiseptic in 1940 by Australian chemist Adrian Albert, to twelve Alzheimer’s Disease victims. Previously, Summers had used tacrine as a treatment for drug overdose comas and delirium. However, he suspected that tacrine might be effective in the treatment of Alzheimer’s Disease based on research conducted by scientist Peter Davies, head of the Litwin-Zucker Research Center for The Study of Alzheimer’s Disease and Memory Disorders. The tacrine worked as an
acetylcholinesterase inhibitor by increasing the levels of acetylcholine within the brain. The twelve patients given tacrine showed noticeable improvement, and Summers went on to develop and demonstrate the effectiveness of orally administered tacrine alongside a team of researchers at UCLA, as intravenous treatment for a chronic disease was deemed impractical. Though the findings of these studies sparked much controversy within the scientific community, further studies conducted on a much larger scale were able to come to the same conclusion about oral tacrine as an effective Alzheimer’s treatment. The drug was approved by the U.S. FDA in 1993, and sold under the name Cognex. [S5.8, S5.9]
William K. Summers
William K. Summers, M.D. [I6.6]
This group of Mexican neurosurgeons reported success in a 1988 trial during which they transplanted fetal stem cells into the brains of patients afflicted with Parkinson’s Disease. Although the study was later considered inadequate proof of improvement in the condition of Parkinson’s patients after stem cell transplantation, trials such as these laid the foundation for studies regarding stem cell transplantation to cure neurodegenerative disease, which quickly became a widespread topic of study. [S6.1]
Madrazo I., León V., Torres C., Aguilera M. C., Varela G., Alvarez F., Fraga A., Drucker-Colín R., Ostrosky F., and Skurovich M.
A study published by Elan Pharmaceuticals in 1999 reported success with the injection of beta amyloid peptide to prevent amyloid plaque buildup within the brains of transgenic mice models of Alzheimer’s Disease. The mice were first treated with a mutant form of the human amyloid precursor protein, which allowed the mice to develop a form of Alzheimer’s which
Elan Pharmaceuticals, Inc.
closely replicated the expressions of AD in humans. The groups of mice, inoculated at different ages and stages of Alzheimer’s development, showed remarkable improvement in the amount of amyloid plaques within the brain when injected with the amyloid B protein. The treated mice also showed less susceptibility to glial cell damage and neural atrophy. The scientists involved in this study concluded that the vaccine not only had the potential to halt the progression of Alzheimer’s, but could also serve as an effective means of reversing some of the negative effects of the amyloid plaque on the brain, as the tested mice seemed to improve in condition after the amyloid B protein treatments. [S6.2, S6.3]
"The brain of a vaccinated mouse (right) shows less plaque formation than the control (left.)" [I6.7]
An article published just a year later, in 2000 described the results of a similar trial conducted by two groups, the groups of the Howard L. Weiner and Dennis J. Selkoe laboratories. Led by Cynthia Lemere of the Selkoe group, and Ruth Maron of the Weiner group, the study sought to discover the effects of a nasally administered beta-amyloid peptide in transgenic mice models of Alzheimer’s Disease. This study group reached the same conclusions as the one released by Elan Pharmaceuticals, the sole difference in the studies being the method of administering the amyloid B protein vaccine. [S6.4]
The Howard L. Weiner and Dennis J. Selkoe Laboratories
Sangamo Biosciences, Inc., a biopharmaceutical company that develops and researches therapeutic treatments of monogenic diseases, introduced the first presentation of data regarding their zinc finger DNA-binding protein (ZFP) gene regulation technology and its role in the treatment of Huntington’s Disease. Their unique ZFP Therapeutic was said to be able to selectively repress the mutant gene, leaving the expressions of other genetic material untouched. The Sangamo team engineered the zinc finger transcription factors to suppress the production of the huntingtin gene (HTT)’s mRNA, decreasing gene expression of the mutant gene, and leaving the remaining normal HTT unharmed. [S6.5]
Sangamo Biosciences, CHDI Foundation, Inc.
A project known as BrainCAV, conducted by researchers at the CNRS Institute of Molecular Genetics at Montpellier and led by Eric Kremer, involved the use of a viral vector, canine adenovirus-2 (CAV-2), which selectively infected the neurons. Scientists were able to use the non-human adenovirus as an ideal means of transporting healthy genetic material into the nervous system, a more practical means of gene therapy for those with neurodegenerative conditions than the more invasive surgical alternative. The BrainCAV team developed a monkey model of Parkinson’s Disease using CAV-2 loaded with Parkinson’s-causing genetic material, their theory being that if the primate began to develop Parkinsonian symptoms, then reversing the effects with the healthy genetic material should work in the same manner. [S6.6]
CNRS Institute of Molecular Genetics
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Biogen, Inc. are developers and manufacturers of therapies for the treatment of various conditions, including neurodegenerative diseases. Biogen’s investigational treatment for Alzheimer’s Disease shows promise, and was recently granted Fast Track designation by the U.S. FDA. The developed treatment compound known as aducanumab, an antibody with the ability to selectively attack amyloid plaque deposits, was able to successfully bind and reduce the amount of plaque aggregates. Researchers reported the slowing of neural degeneration, as well as the reduction of soluble oligomers and insoluble fibrils, commonly found in the amyloid plaque deposits. [S6.7]
Biogen, Inc.
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A five-year study conducted by researchers at Leicester University has reportedly found a means of reversing the symptoms of neurodegenerative disease, specifically Alzheimer’s and Parkinson’s Disease. The team targeted specific metabolites in the kynurenine pathway believed to be toxic to neural cells, using fruit flies as their subjects. Using a form of gene therapy to inhibit the activity of the TDO and KMO enzymes in the kynurenine pathway, researchers found that the level of the aforementioned metabolites was greatly reduced, and loss of nerve cells in the fruit fly models ceased. In the fly models of Parkinson’s and Alzheimer’s specifically, scientists also noticed a visible improvement, including the repossession of lost motor skills, and an extended lifespan. [S6.8]
Leicester University
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Neuralstem, Inc. seeks to use their neural stem cell technology to manufacture stem cell therapies in large quantities, in order make stem cell therapy easily available to patients struggling with neurological disease. They seek to improve the conditions of patients with neurodegenerative disease with cell therapy and drugs, such as their potential ALS treatment which involves transplanting specialized stem cells by injecting them into the affected patients’ spinal cords. [S6.9]
Neuralstem, Inc.
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The California Institute for Regenerative Medicine, a leading contributor to the field of stem cell research, currently has many ongoing projects seeking to find a way to prevent or reverse neurodegeneration. Among other innovative scientific ventures, CIRM has funded a research project that is currently being conducted by a UC Davis research team, that seeks to use MSCs from bone marrow to halt the degenerative trend in the nervous systems of patients with Huntington's Disease. [S7.4]