Photo by Daniel Paquet
Researchers from University of British Columbia’s brain research centre believe they have created an antibody that successfully targets cellular mutations caused by Lou Gehrig’s disease.
The illness, also known as ALS, has been difficult to treat since its discovery in 1869 due to a lack of understanding surrounding its causes.
Biomedical researcher Leslie Grad says the antibodies his team has developed target only the disease-causing proteins, which become mis-folded and spread their mutations throughout the body.
“In our lab we look at ways to stop the protein mis-folding,” Grad said.
“One way to do this is to create molecules called antibodies that specifically recognize diseased forms of the protein.”
The disease affects approximately one in 1000 people, with 80 per cent dying from the disease within two to five years. A disease of the nervous system, ALS alters motor neurons causing paralysis of the limbs, speech, and ultimately respiratory systems.
The guilty protein is known as SOD1 and is found in every cell of the human body. This protein can be affected by over 150 unique mutations, all of which can lead to the disease.
These mutations cause the protein to mis-fold. The researchers believe this disorder can then be transmitted to healthy proteins across cells and neurons.
The actual chemical structure of the protein itself remains unaffected. The disease is instead caused by the way the proteins are folded. The antibodies created by Grad’s team target only these specific mis-folded proteins, while ignoring healthy ones.
“The disease form can be secreted from the cell. Experiments have shown how diseased proteins impose their will on healthy ones,” Grad said.
According to him, SOD1 mutations are responsible for roughly two per cent of ALS cases. The team is also working to explore a general SOD1 connection in all instances of the disease, a topic he describes as “highly controversial” at this time.
Should Grad’s suspicions that SOD1 be linked to other forms of the disease prove valid, a treatment for most forms of the disease could be a possibility.
“We are in the process of humanizing the antibodies,” Grad said.
The researchers hope to make an antibody that the human body will not reject as a foreign agent, leading to possible preventative treatments for the disease.
“A human version of the antibody could essentially be taken as a vaccine,” added Grad.
The first thing researchers need to do, before they can offer their visions of a possible cure or vaccine, is demonstrate how the disease works.
The team is currently working on two studies. Their first aim is to show how the mutated proteins spread throughout a cell by influencing other healthy proteins to mis-fold.
Grad said the second study aims to show how these mutations can be transmitted outside the cell to healthy parts of the body, ultimately affecting our motor neurons and causing the disease.
It is the hope of the researchers that their antibody will block the transmission of these mutations, leading to a vaccine that could be administered in order to prevent ALS from developing.
For the immediate future, however, ALS will remain a vicious and uncontrollable killer.
“Unfortunately, ALS is a death-sentence,” said Grad. “A quick death-sentence.”