News & Updates

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.

The Accelerating Medicines Partnership (or AMP) programmes are public-private partnership between the NIH, the U.S. Food and Drug Administration (or FDA), multiple biopharmaceutical/life science companies, and non-profit organizations. The goal of these efforts are “to transform the current model for developing new diagnostics and treatments by jointly identifying and validating promising biological targets for therapeutics”.

And the ultimate goal of the AMP programmes is to “increase the number of new diagnostics and therapies for patients and reduce the time and cost of developing them“.

Launched in February 2014, the first AMP programmes focused on three disease areas:

• Alzheimer’s disease
• Type 2 diabetes
• Autoimmune disorders of rheumatoid arthritis and systemic lupus erythematosus (lupus)

In January 2018, an AMP programme focused on Parkinson’s was launched, called the Accelerating Medicines Partnership for Parkinson’s disease (or AMP-PD).

It involved nine partners:

• The NIH (Government agency)
• The National Institute of Neurological Disorders and Stroke (or NINDS – Government agency)
• The FDA (Government agency)
• GlaxoSmithKline (biopharmaceutical company)
• Pfizer (biopharmaceutical company)
• Sanofi (biopharmaceutical company
• Celgene (biopharmaceutical company)
• Verily (life science company)
• The Michael J. Fox Foundation (non-profit organization)

The AMP-PD research plan encompasses a deep molecular characterisation of longitudinal clinical data from well characterised cohorts, and analysis of existing biosamples with the goal of identifying (and validating) diagnostic, prognostic and disease progression biomarkers for Parkinson’s.

The critical component of the AMP-PD partnership is the extensive sharing of the collected data and analyses with the biomedical community, with the very simple goal of advancing research in PD.

The clinical cohort data collected so far has come from:

• The Michael J. Fox Foundation and NINDS BioFIND study – 213 participants
• The Harvard Biomarkers Study (HBS) – 877 participants
• The NINDS Parkinson’s disease Biomarkers Program (PDBP) – 1,599 participants
• The Michael J. Fox Foundation Parkinson’s Progression Marker Initiative (PPMI) – 1,610 participants

But the goal is to include data collected from clinical trials as well (Source). Initial additional data will come from two Phase III clinical trials which have conducted long-term (multi year) assessments of Parkinson’s progress:

• The Study of Urate Elevation in Parkinson’s Disease (SURE-PD3; NCT02642393) – a double-blind, placebo controlled study assessing inosine in Parkinson’s
• The Efficacy of Isradipine in Early Parkinson Disease (STEADY-PD3; NCT02168842) – a double-blind, placebo-controlled study evaluating Isradipine in Parkinson’s

The data collection for both of these studies has been completed, and the results are currently being analysed. The SURE-PD study was stopped early as no effect was seen by the trial’s independent Data and Safety Monitoring Board (Click here to read more about this), and the STEADY-PD trial found no significant difference between the treatment and placebo groups after 36 months treatment (Click here to read more about this).

I’m not sure who said it (someone please help me), but I read a quote some time ago which went something like “the best ideas come from what others do with your data“, and I loved this concept. Humbling and utilitarian, it embodies the idea that more can be gained by sharing.

The AMP-PD is a wonderful idea. Pulling together as much data as we can and providing researchers with access to it is exactly what we need. Opening up vast datasets will hopefully bring about new discoveries, new ideas, new testable hypotheses and all of this will lead to new knowledge, new biomarkers, and (fingers crossed) new therapies for Parkinson’s.

It is an ambitious project in it’s scale and goal, but also in its attempt to change attitudes. Open data, open science, open access are all very popular concepts and they get a lot of lip service at the moment, but the biggest challenge with all of them is having an open mindset.