ALS Safety Trial Suggests Potential For Stem Cell-Gene Therapy

Nikki Attkisson | Last Updated : September 9, 2022

Stem cells and ALS gene therapy is under many studies in the medical world and this article will discuss A-Z information about it.

ALS, also known as Lou Gehrig’s Disease, is a neurological disorder affecting the body’s overall functions. This will cause the patient to move, lower muscle strength, and even breathe and eat. The affected can only last life for 5 years of life as per medical reports. The high usage of fats for activities and metabolism could badly influence the weight of the person. So, he or she may not sustain a healthier body for so long.

The earlier symptoms may include, 

  • Struggles with eating and swallowing
  • Difficulties to move sometimes
  • Pains in the body
  • Muscle twitches
  • Not being able to properly speak

There are many reasons for having ALS, like age, ethnicity, gender, genetics, etc. 

What Are The Stem Cells?

Stem cells are the primary cells of the human body which can change into whatever cells are needed for the body.  Every type of cell has its own properties and they could reproduce their own kind of cells only. But the cells like RBC could not divide or reproduce. But stem cells are the key to providing cells to the body or repairing the cell damage to the body. It is able to reproduce multiple variants of cells from neurons, blood cells, etc. Embryonic stem cells, adult stem cells, and induced pluripotent stem cells are the important three types of stem cells. 

ALS Safety Trial Suggests Potential For Stem Cell-Gene Therapy

Many of the researches have shown that stem cells are able to rejuvenate the cells internally and externally when the body is facing any kind of problem. Stem cells are visible in the embryo mainly as well as in the adult body. 

Stem cell therapy

According to studies, stem cells are capable of generating any cells of the body or organs. Stem cells are used for cancer patients and thalassemia patients for replacing the blood stem cells destroyed at the time of treatment. And also major damages from accidents can be also get corrected by stem cell therapy. Stem cells can be generated, preserved, and stored in laboratories and also, and modern medicine could produce stem cells for nerves, blood, and other organs too. So, the treatment can be done for the cause itself. 

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Stem cell therapy and ALS

Studies are happening around the world to find the scope of stem cell therapy to effectively for treating patients with ALS. Stem cells are potent to provide all proteins for the neurons of the body to maintain and stable the body. It can support and generate motor neurons which help with the movements of the body. The cultured stem cells will be placed into the spines and help to sustain the motor neurons. This will help the patients who are paralyzed to come to normal life again. 

Svendsen’s laboratory produced the stem cells for the experiments known as GDNF (glial cell line-derived neurotrophic factor) can tackle the “brain-blood” barrier according to the scientists and releases the proteins essential for the motor neurons. In experiments, it is shown improvement in treated patients has shown more results than the non-treated ones. And also GDNF is transplanted to the brain, in another trail, to the motor cortex which is able to help with the movement of hands. This trail is successful among the affected ones. This transplantation of stem cells can last up to 3 years and is a new era of modern medicine. 


The hope that is given to patients with ALS by stem cell therapy is big and promising. ALS is one of the major diseases and treatment is very hard. The patient cannot move or even properly eat. But, stem cell-gene therapy can transplant the engineered stem cells into the spinal cord and certain areas of the brain. This will help to maintain the motor neuron cells of the body. And also, it is completely safe for the patients. Now, the researchers are in the stage of spreading the trial to more patients and it is proven completely side effect free. 


🔵National Library Of Medicine (n.d) Role of Stem Cells in Treatment of Neurological Disorder (Available Online):

🔵Science Direct (n.d) Stem Cell Gene Therapy (Available Online):

Nikki Attkisson

With over 15 years as a practicing journalist, Nikki Attkisson found herself at Powdersville Post now after working at several other publications. She is an award-winning journalist with an entrepreneurial spirit and worked as a journalist covering technology, innovation, environmental issues, politics, health etc. Nikki Attkisson has also worked on product development, content strategy, and editorial management for numerous media companies. She began her career at local news stations and worked as a reporter in national newspapers.

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