A new form of gene therapy is currently tested on various children who are suffering from spinal muscular atrophy and the results are positive. The disease affects infants and is a death sentence in all cases. Symptoms range from muscle atrophy to respiratory failure and their severity can prevent patients from surviving more than two years of age. Children who do survive would require constant respiratory assistance and general help to move. There are treatments, one of them being a drug called Spinraza made by Biogen. Spinraza costs almost a million dollars for the first year and then several hundred thousand dollars if you decide to continue. But another company is breaking new ground with another option.
Enter AveXis, a Biotech Company that is pioneering a currently unnamed treatment that comes in the form of gene therapy. It consists of a one-time shot of a specially designed virus that isn’t dangerous for humans but can extend to inside cells. Once injected, the virus will infect every cell in the body and generate a working copy of the SMN1 gene. Sources state that the new gene does not become part of the child’s DNA, but it acts as a supplemental gene.
All children who are affected by SMA lack a certain gene called SMN1 which produces a protein of the same name. Without the protein, the brain is unable to send messages to the neurons linking the muscles.
Both therapies can make the patient’s body produce the SMN protein, but their means of doing so are very different.
Spinraza can generate SMN protein while the new gene therapy option can add the missing SMN1 gene. However, it is not clear if a single dose will last indefinitely and the price for this has not been set yet.
Image Source: PublicDomainPictures