BGU researchers find reformulated drug helps ALS patients

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May 23, 2017 20:14

Researchers praise a “breakthrough” treatment to improve brain function and longevity for patients with the incurable Lou Gehrig’s disease, also known as ALS.

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Doctor and patient

Doctor and patient (illustrative).. (photo credit: INGIMAGE)

Researchers at Ben-Gurion University of the Negev have suggested the use of a drug in what they call a “breakthrough” treatment to improve brain function and longevity for patients with the incurable Lou Gehrig’s disease, also known as amyotrophic lateral sclerosis, or ALS.

The proposed drug can also improve the conditions of patients with Parkinson’s and Alzheimer’s disease by helping the brain rid itself of toxins, the researchers said on Tuesday,.

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The drug improves the protective immune functioning of the central nervous system by promoting the recovery of a healthy CNS environment.

ALS is a progressive fatal neurodegenerative illness that attacks motor neurons. When motor neurons die, the ability of the brain to control muscle movement is lost, leading to paralysis. When muscles are unable to function, they atrophy.

Eventually, all muscles under voluntary control are affected and, in time, patients lose their ability to walk, talk, swallow and breathe.

During this inevitable process, the mind remains completely intact and aware of the loss of each function. When the diaphragm and chest muscles fail, patients stop breathing. The majority of people with ALS die of respiratory failure within two to five years of noticing the symptoms The odds of contracting the 100% fatal disease are one out of 1,000.

Every year, more than 100,000 people around the world are diagnosed with ALS and it is the cause of death of a similar number of individuals annually. Latest estimates of the number of people affected with ALS world-wide exceed 600,000. The non-contagious illness affects people from all ethnic backgrounds, usually between the ages 40 and 60, although cases of younger individuals with ALS are known. In Israel, at least 700 people are doomed to die from ALS – about twice as many as the annual death toll due to car accidents.

Dr. Rachel Lichtenstein, of the biotechnology engineering department in BGU’s Faculty of Engineering Science, focused her research on reducing negative immune response. “I found a way to prevent the glial cells from attacking and killing healthy cells in the brain,” she explained, referring to the “insulating” cells abundant in the central nervous system.

To do this, she and her colleagues collected the central functioning type of sugar in the fragment-crystallizable (Fc) tail of the antibody.

“We used the Fc component of MabThera, an existing drug given to patients with lymphoma that contains the same type of sugar. We managed to turn the Fc in Mab- Thera into a new molecule that meets the conditions for patenting for the treatment of ALS. The results of experiments on transgenic mice with ALS showed a significant rise on life expectancy,” Lichtenstein noted.

The technique she developed helps the weakened nervous system to improve its tasks of cleansing and removing the dead cells and waste products that harm the system. It enables the sick mice to live better and longer lives, Lichtenstein said.

Because the exact cause of ALS is unknown, the study focused on prolonging life expectancy after the onset of the disease, which currently ranges from two to five years in most patients.

“While this is only early research in mice, we believe that this new drug can have a significant impact on the lifespan of ALS patients,” said Dr. Ora Horowitz, senior associate director-general for business development at BGN Technologies, the university’s technology transfer company.

“It can have significant effects on the lifespan of patients with other neurodegenerative diseases, such as Alzheimer’s and Parkinson’s,” added Horowitz. “Our new drug proposal can be effective in stimulating the self-purifying mechanism of the human brain, thus improving the lives of millions of people.”

Only two ALS drugs have been approved by the US Food and Drug Administration, she concluded, and they have significantly helped patients. They are Riluzole (Rilutek) and edaravone (Radicava), which have been shown to extend survival by only three to six months.

“Since we use a component of existing and approved drugs, we believe we will need a limited amount of preclinical testing, and we can get to the clinical stage earlier than in other cases,” concluded Lichtenstein, who is now looking for a pharmaceutical company to cooperate in this field of research.


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