New Discoveries in Neurodegenerative Diseases
Everyday is another day that can totally differ from the previous day with regard to increasing researchers’ capabilities of discovering new pathways of diseases and turning their new discoveries into far-reaching treatments. The tireless revolution in life sciences has uncovered treasures of knowledge that has been buried for centuries in the graves of the unknown. The wealth of knowledge being accumulated about the genes’ expressed proteins that impact the physiology and pathology of living beings is translated, in an unprecedented speed, into clinical methods and approaches towards conquering deadly diseases. Deadly diseases that have yet to find treatment are numerous, including neurodegenerative diseases, mental sicknesses, metabolic diseases and many other crippling and life-threatening diseases.
Great news has been the announcement that researchers in academia and biopharmaceutical companies are becoming confident enough to start zeroing in on diseases in extremely complicated body systems such as the nervous system. Recently, our enthusiasm swelled when we heard news about discoveries that helped scientists understand neurodegenerative diseases. Our swelled enthusiasm grew huger when we learned that one of the new discoveries has already led to the design and creation of a specific treatment for two deadly neurodegenerative diseases that have yet to find safe and effective treatments. Other discoveries were also announced by academic institutions – all have opened the door wide for scientists in biotechnology and biopharmaceutical firms to start creating safe and effective targeted treatments.
In the NEWS
Scientists working at Genentech, a unit of Roche (RHHBY), together with other scientists have discovered a protein that they called dual leucine zipper kinase (DLK). Blocking that protein, reversed the signaling from the pathway of amyotrophic lateral sclerosis (ALS) and Alzheimer disease (ED) – both recognized now as neurodegenerative diseases.
DLK activates two proteins including c-Jun N-terminal kinase (JNK), which is known to induce brain-cells’ death. The authors demonstrated that DLK/JNK signaling is elevated in both mice and people suffering from both AD and ALS.
The news was considered reliable only when the gene that makes DLK has actually demonstrated it protects against neuron loss in mouse models of ALS.
The Journal Science Translational Medicine, which published the discovery announced that drugs that inhibit the DLK protein will soon be in human clinical trials for ALS and other neurodegenerative diseases.
As a matter of fact, Genentech has launched a phase 1 study evaluating an oral DLK inhibitor called GDC-0134, in people with ALS.
It is well known that the complicated nervous system has failed hundreds of investigational drugs, disappointing many drug developing companies, millions of patients and thousands neurologists around the world. The disappointments have been in the management of the most morbid and deadly neurological diseases, including benign and malignant tumors, neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease; Parkinson’s disease and other diseases that can be diagnosed, but remained unfathomed and have yet to find treatments.
The great news represents one of many discoveries made in the past couple of months in neurodegenerative diseases, brain cancers and other central and peripheral nervous system diseases. Some of the discoveries are being made by universities and others by specialized companies that we will discuss one by one in various upcoming articles after validating their science and their trial results.
From Yale University we learned that its School of Medicine’s researchers have discovered that defects in the transport of lysosomes within neurons promote the buildup of protein aggregates in the brains of mice with Alzheimer’s disease (AD).
The study, which is published on August 7 in Journal od Cell Biology (JCB) suggests that developing ways to restore lysosome transport could lead to designing treatments for Alzheimer’s Disease.
Which drug developers will be the lucky ones to design, develop and sell these breakthrough drugs in the vast thirsty markets that are full of frustrated specialists and millions of their agonizing life-threatened patients whom they cannot rescue or even promise any help. Prohost will continue to follow up and update its readers about the selected firms.
Indeed, neurological diseases threaten the lives of millions of babies, children and adults of both gender.
Prohost will discuss this subject and we will present old and the new firms that are already developing novel targeted breakthrough therapeutics for neurological diseases.
We believe that Genentech’s success in discovering a target for treatment of neurodegenerative diseases and designing a product that blocks this target is the first of many large steps that the firm will be walking in the direction towards solving many of the nervous system problems.
The market is huge.
Roche is lucky to have Genentech.
Will we be lucky to add Roche to our portfolio?
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