Virus therapy to counter motor neuron diseases?

The areas of life sciences, technology, bio-medicine and medical devices has scaled dizzying heights in the past few years which has led to a high level of coverage for diseases, disorders and infections.

By Prashant Vadgaonkar (TECH TALK)

Published: Sat 24 May 2014, 11:48 PM

Last updated: Fri 3 Apr 2015, 10:18 PM

However, this growth has still kept the cure for few diseases out of bounds of human and technological brilliance and research. The classic cases of such diseases include HIV, Parkinson’s syndrome and also ALS for which, till date, there has been no proven and demonstrated remedies and medication available. A new US based start-up now plans to target this greenfield area and intends to use gene therapy to counter the menace of ALS and Parkinson’s disease.

For the uninitiated, Gene therapy is essentially the practice of using DNA as a drug to heal diseases by injecting therapeutic DNA into a patient’s cells. In simple terms, Gene therapy techniques involves swapping a mutated gene with a healthy version or switching off a gene that leads to disease or disorder. Gene therapy involves using the DNA to replace a mutated gene or could involve DNA that encapsulates therapeutic protein drug as a form of treatment.

Parkinson’s disease is a degenerative disorder of the central nervous system wherein the motor functions are such as shaking slowness, difficulty in walking, overall gait are impacted initially. These culminates into thinking and behavioural issues and dementia and acute depression at later stages as the disease aggravates. Parkinson’s disease usually happens on account of the demise of thdopamin generating cells in the region of the mid-brain, the cause for which is still a mystery to science. It is believed that about 8 to10 million people across the globe suffer from Parkinson’s disease which typically strikes at the age of 50 or above and is more prevalent tin me than women.

Amyotrophic lateral sclerosis (ALS) is also motor neuron function related disease which develops as a rapid progressive weakness due to muscle atrophy and muscle spasticity and leads to trouble in breathing, speaking, and swallowing. About 2 of 100,000 people are believed to succumb to ALS annually across the globe.

Boston, Cambridge based company Voyager Therapeutics is planning to use new gene therapy which will infect patients in an intentional manner to find the solution to these issues. The firm intends to use a type of virus named Adeno-associated virus (AAV) as transporters to deliver viral proteins to the human brain. The virus infections which are pre-meditated are totally harmless to humans and thus they form the perfect transport models for moving proteins through the human body without any side effects.

The AAV is a small virus which once injected does not cause disease but invokes a mild immune response and acts without integrating into the genome of the host cells thereby making it an ideal vehicle for treatment. Voyager intends to use the AAV virus to deliver a missing protein for treatment of Parkinsons disease while in case of ALS it will ensure the decimation of a harmful protein.

Though the solution is still at the drawing broad level and will take time to be released, it highlights the confidence of the scientific fraternity that use of gene therapy may provide an effective cure for the world most challenging diseases.

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