Today the US National Institute of Health (NIH) announced the opening of the data portal for the Accelerating Medicines Partnership for Parkinson’s disease (or AMP-PD) initiative.
This research program is a MASSIVE collaborative effort between the NIH, multiple biopharmaceutical/life sciences companies, and non-profit organisations.
It involves a pooling together of “well characterized cohorts with existing biosamples and clinical data”, and making this data available for researchers in order to identify and validate diagnostic, prognostic, and progression biomarkers.
In today’s post, we will look at what the AMP-PD initiative is, and consider how it could help to accelerate the development of novel therapies for Parkinson’s.
The US National Institute of Health Clinical Research Center, Bethesda, Maryland. Source: Wikipedia
In the late 1870s, the US ongress allocated funding for the investigation of the causes of specific epidemics (such as cholera and yellow fever). An urgent need was recognised and the US congress acted.
In doing so, however, it not only created the National Board of Health, but it also made medical research an official government initiative.
The National Board of Health was re-designated several times and in 1930 the US National Institutes of Health (or NIH) was born.
The NIH has gone on to become one of the largest funders of medical research in the world. And some of the numbers are really staggering (Source):
- The NIH invests nearly $39.2 billion annually in medical research
- More than 80% of the NIH’s funding is awarded to over 50,000 competitive grants.
- These grants fund 300,000 researchers at more than 2,500 universities/research institutions in every US state and around the world.
- About 10% of the NIH’s budget supports projects conducted by nearly 6,000 scientists in its own laboratories
It is an institution that can seriously change the landscape for medical research. And recently it has been trying to do this via AMP programmes.
What are AMP programmes?
Biotech firm Denali announced the dosing of the first person in their Phase Ib clinical study of their experimental treatment for Parkinson’s called DNL201.
DNL201 is an inhibitor of a Parkinson’s-associated protein called Leucine-rich repeat kinase 2 (LRRK2).
In Parkinson’s, there is evidence that LRRK2 is over activate, and by inhibiting LRRK2 Denali is hoping to slow the progression of Parkinson’s.
In today’s post, we will discuss what LRRK2 is, what evidence exists for DNL201, and what the new clinical trial will involve.
Founded in 2013, by a group of former Genentech executives, San Francisco-based Denali Therapeutics is a biotech company which is focused on developing novel therapies for people suffering from neurodegenerative diseases. Although they have product development programs for other condition (such as Amyotrophic Lateral Sclerosis and Alzheimer’s disease), Parkinson’s is their primary interest.
And their target for therapeutic effect?
The Parkinson’s-associated protein called Leucine-rich repeat kinase 2 (or LRRK2).
What is LRRK2?
New research published in the last week provides further experimental support for numerous clinical trials currently being conducted, including one by the biotech company Sanofi Genzyme.
Researchers have demonstrated that tiny proteins which usually reside on the outer wall of cells could be playing an important role in the protein clustering (or aggregation) that characterises Parkinson’s.
In today’s post we will look at this new research and discuss what it could mean for the on going clinical trials for Parkinson’s.
The proverb ‘When the cat is away, the mice will play’ has Latin origins.
Dum felis dormit, mus gaudet et exsi litantro (or ‘When the cat falls asleep, the mouse rejoices and leaps from the hole’)
It was also used in the early fourteenth century by the French: Ou chat na rat regne (‘Where there is no cat, the rat is king’).
And then Will Shakespeare used it in Henry the Fifth(1599), Act I, Scene II:
Westmoreland, speaking with King Henry V, Gloucester, Bedford, Exeter and Warwick
“But there’s a saying very old and true,
‘If that you will France win,
Then with Scotland first begin:’
For once the eagle England being in prey,
To her unguarded nest the weasel Scot
Comes sneaking and so sucks her princely eggs,
Playing the mouse in absence of the cat,
To tear and havoc more than she can eat”
Interesting. But what does any of this have to do with Parkinson’s?