Mayo Researchers Score Another Neurodegenerative Disease Patent

Jacksonville team’s latest describes drug treatment for frontotemporal dementia.

A medical team led by a renowned Mayo Clinic researcher has patented a drug treatment method for frontotemporal dementia, a rare, genetic and rapidly progressive neurodegenerative brain disorder that can affect behavior, cognition, language and motor skills.
It’s the second notable dementia-related patent awarded in the last three months to Mayo researchers based at the clinic’s Jacksonville, Florida facility. 
The latest patent is for a novel type of molecular compound designed to stimulate the production of a key brain protein that is lacking in patients with frontotemporal dementia (FTD), and also covers its delivery to the brain via a virus.
One of the inventors cited is Mayo neuroscience professor Rosa Rademakers, whose lab has made several significant discoveries in fighting devastating neurological diseases such as FTD, Alzheimer's disease, amyotrophic lateral sclerosis (ALS), as well as Parkinson’s disease-related syndromes.
Also listed as inventors are fellow researchers at academic institutions in Belgium, the United Kingdom and Canada.
The method disclosed by Rademakers and her colleagues is based on a striking characteristic of those suffering from FTD as well as ALS: a deficiency of a protein called progranulin, or PGRN, which is essential for the proper functioning of brain neurons. Without sufficient quantities of PGRN, the brain’s frontal and temporal lobes gradually atrophy.
Rademakers’ research in genetic sequencing five years ago identified a mutation in the gene known as C9ORF72 as a key biomarker for FTD—an accomplishment which in April earned the 37-year-old educator the 2016 Potamkin Prize for Research in Pick’s, Alzheimer’s and Related Diseases, one of the highest honors in neuroscience.
There are currently no treatments for FTD, which strikes many patients in the prime of life, progresses rapidly and is ultimately fatal. One of the main avenues of research has been to seek ways to boost levels of PGRN in the brain through new kinds of drugs. That process, however, has yet to yield any successes. For instance, Massachusetts-based FORUM Pharmaceutics was working on a drug to boost PGRN levels, but went out of business this year when its other drug candidates for Alzheimer’s disease failed in clinical trials.
In 2013, Rademaker and her colleagues earned a patent on a method of genetically testing for FTD in patients who have a family history of the disease. It was based, in part, on their discovery that mutations within the PGRN gene’s nucleic acid are linked to dementias such as FTD.
The current patent extends on that intellectual property to disclose a new method of increasing PGRN levels by administering manufactured PGRN polypeptides (a polypeptide is a chain of linked amino acids). Also described in the patent is a method of delivering the drug to the brain by piggybacking it onto a benign bioengineered virus.
With the new patent, Mayo’s team of neurological researchers is on an IP hot streak. In September, the U.S. Patent Office granted them ownership of a new kind of genetic test targeting ALS and FTD invented by members of the clinic’s Neurodegenerative Diseases Lab, led by Leonard Petrucelli.
Petrucelli and his colleagues made news last year with the announcement that they had developed a mouse model that mimics the human neuropathological and behavioral features associated with the most common genetic form of ALS and FTD caused by the C9ORF72 gene mutation.
Their mouse model has been hailed as a breakthrough because the mice exhibit human pathologies such as hyperactivity, anxiety, antisocial behavior and motor deficits. The brains of the specially bred mice have key hallmarks of the disorders, including toxic clumps of ribonucleic acids (RNA) and TDP-43, a protein that has long been known to go awry in the majority of ALS and FTD cases.