First-ever biomarker qualified for Parkinson’s is a vital step toward improved clinical trials

FOR IMMEDIATE RELEASE

Contact:
Kissy Black
615.298.1144
kissyblack@lotosnile.com

 

Tucson, AZ — July 26, 2018 – The Critical Path Institute’s (C-Path) Critical Path for Parkinson’s Consortium (CPP), in partnership with Parkinson’s UK, announces that the European Medicines Agency (EMA) has issued a positive qualification opinion on an imaging test (biomarker) as a tool to enrich Parkinson’s clinical trials. The purpose of this enrichment biomarker is to serve as a measurement that can be used to select people with Parkinson’s who are most suitable to take part in clinical trials.

The biomarker is used to determine the presence of dopamine transport deficiency in the brain, and has been qualified as an enrichment biomarker for clinical trials targeting early stages of Parkinson’s soon after diagnosis. The qualified biomarker involves the intravenous injection of a small amount of a radioactive tracer before the brain images are acquired, and can be done at any one of many specialist imaging centers. The imaging agent binds very specifically to dopamine transporter sites on the neurons that are lost in Parkinson’s disease. The use of this biomarker can help better identify patients that are more likely to exhibit significant progression in their motor signs and symptoms, thus helping select patients for clinical trials. The CPP consortium is a global public-private partnership consisting of industry, academics, advocacy organizations, and government agencies collaborating to develop solutions to optimize drug development for Parkinson’s. This qualification represents a major milestone in this regard as the first biomarker for Parkinson’s to receive such a regulatory designation. In 2015, the U.S. Food and Drug Administration issued a letter of support for the use of this imaging biomarker for use in Parkinson’s clinical trials.

“This endorsement from the European Medicines Agency represents many years of hard work and incredible collaboration among companies, universities, and charities facilitated by the Critical Path Institute,” says Dr. Diane Stephenson, Executive Director of CPP, who led the work. “These brain scans in themselves are not new, but until now there has not been a clear consensus that they can and should be used to select participants for clinical trials. Through our global project, we’ve been able to bring all the data and expertise together to make a powerful case, so we’re delighted that this endorsement from the EMA will improve the quality and chances of success for future trials of Parkinson’s treatments. This success is just the first in a suite of new tools that we hope to deliver for Parkinson’s.”

Studies suggest that up to 15% of individuals taking part in clinical trials for new Parkinson’s treatments may not exhibit a measurable progression in motor signs and symptoms over the course of such trials. Furthermore, the rate of uncertainty in predicting disease progression is higher at earlier stages of the condition. These individuals are extremely unlikely to benefit from the new therapies being tested, and their inclusion can affect both the trial results and, ultimately, the future of the potential treatment.
Professor David Dexter, Deputy Research Director of Parkinson’s UK, which funds CPP, comments, “Scientific breakthroughs mean that there is now a new wave of exciting treatments that genuinely could slow, stop, or reverse the condition, but it’s crucial that we’re able to test them properly in clinical trials. Being able to rule out individuals who are unlikely to have Parkinson’s could be the difference between a successful trial and failure. This is a vital step forward in our mission to deliver, as quickly as possible, better treatments, and one day a cure, to people living with Parkinson’s.”

Because Parkinson’s is a progressive condition caused by the gradual loss of cells in the brain, the best chance to intervene with treatments that can slow, stop, or reverse damage is during the earliest stages of the condition. During these early stages, however, signs, and symptoms tend to be mild, which makes selecting the right people to participate in trials very difficult.

“The use of these brain scans is already being included in new clinical trials at Biogen.” said Dr. Michael Ehlers, Executive Vice President of Research and Development at Biogen. “We believe that this new approach will introduce greater efficiency in terms of cost and speed, while ensuring that the right patients are being included in our trials.”


About the Organizations:

C-Path (Critical Path Institute) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include over 1,500 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path is headquartered in Tucson, Arizona, with additional staff in multiple remote locations. For more information, visit www.c-path.org.

Every hour, someone in the UK is told they have Parkinson’s.

It affects 127,000 people in the UK – which is around one in 500 of the population.

Parkinson’s is a degenerative neurological condition, for which there currently is no cure. The main symptoms of the condition are tremor, slowness of movement and rigidity.

Parkinson’s UK is the UK’s leading charity supporting those with the condition. Its mission is to find a cure and improve life for everyone affected by Parkinson’s through cutting edge research, information, support and campaigning.

For advice, information and support, visit www.parkinsons.org.uk or call our free, confidential helpline on 0808 800 0303.

Parkinson’s UK Contact:
Molly Horsburgh
Senior Media and PR Officer
0207 963 9351
mhorsburgh@parkinsons.org.uk
Out of hours: 07961 460248

Parkinson’s UK Announces Major Pharma Companies Sign Up to CPP

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Major pharma companies sign up to groundbreaking Parkinson’s consortium

Seven of the world’s largest pharmaceutical companies have signed up to a groundbreaking consortium aimed at accelerating the development of safe and effective therapies for Parkinson’s.

The Critical Path for Parkinson’s consortium will bring together leading academics; industry members AbbVie, AstraZeneca, Biogen, Eli Lilly and Company, Merck Sharp and Dohme (known as Merck & Co. Inc. in the United States and Canada), Pfizer, and UCB; and founders the Critical Path Institute (C-Path) and Parkinson’s UK, to share data, expertise and resources to promote and develop new treatments for Parkinson’s.

The consortium, which was launched in October 2015, was formed by Parkinson’s UK and C-Path to increase investment into research and development of new Parkinson’s treatments.

Dr Arthur Roach, Director of Research at Parkinson’s UK, the principal funder of the consortium, says:

“Despite significant advances in our understanding of the genetics, biochemistry and pathology of Parkinson’s, the development of new treatments has not kept pace. New treatments are desperately needed to deal with the devastating effects of this progressive condition.

“Investing in clinical trials for brain disorders currently carries a high cost and high risk of failure. As the world’s largest patient-led Parkinson’s charity, we know that people living with conditions such as Parkinson’s have often been disappointed when drugs that showed significant promise early on failed in late stage testing.

“We see the consortium as a crucial part of strategies to develop new treatments that work at the earliest stage of the condition, with the goal of slowing its progression, and eventually finding a cure.”

Parkinson’s affects 127,000 people in the United Kingdom, and approximately 7 million people worldwide. In 2012/13, the National Health Service (NHS) spent more than £212 million on caring for people with Parkinson’s in England (1), with the cost of the condition in the United States approximately US$25 billion per year (2). With the baby boomer generation becoming older, the number of people with neurodegenerative conditions, including Parkinson’s, is likely to increase and will become one of the biggest issues faced by healthcare.
The International Federation of Pharmaceutical Manufacturers & Associations (IFPMA) estimated that the cost of developing a single drug was US$1.3 billion, rising from US$138 million in 1978 (3), a tenfold increase attributed to a multiplicity of factors faced by drug research.

Diane Stephenson, PhD, Executive Director, Critical Path for Parkinson’s consortium, C- Path, says:

“With the increase in the costs of getting a drug to market, the design of a clinical trial is a crucial part of a drug’s success. There is a strong realisation from the industry that collaboration among industry, academia, and worldwide regulatory agencies, along with the sharing of data, has the potential to create a more efficient development process. This recognition is evidenced by the fast pace at which members of this new consortium have joined.”

Parkinson’s UK has committed over £1million to the Critical Path for Parkinson’s consortium.

For more information, and to donate to Parkinson’s UK research and the work with the Critical Path Institute, visit http://parkinsons.org.uk/research


 

Media enquiries

Please contact: Hanna Kilpin, Senior Media & PR Officer on 020 7963 9311 or email: hkilpin@parkinsons.org.uk

Out of hours: 07961 460248 Notes to editors

  1. Parkinson’s and the NHS in England: the cost of poor care. Parkinson’s UK. October 2013.
  2. Statistics on Parkinson’s. Parkinson’s Disease Foundation.
  3. The Pharmaceutical Industry and Global Health: Facts and Figures 2015.
    International Federation of Pharmaceutical Manufacturers & Associations.

 

About Parkinson’s UK

Every hour, someone in the UK is told they have Parkinson’s.

It affects 127,000 people in the UK – which is around one in 500 of the population.

Parkinson’s is a degenerative neurological condition, for which there currently is no cure. The main symptoms of the condition are tremor, slowness of movement and rigidity.

Parkinson’s UK is the UK’s leading charity supporting those with the condition. Its mission is to find a cure and improve life for everyone affected by Parkinson’s through cutting edge research, information, support and campaigning.
For advice, information and support, visit http://www.parkinsons.org.uk or call our free, confidential helpline on 0808 800 0303.