Top Stories

Published on Thursday, April 11, 2019

Moderated discussion: “Advancing the innovation ecosystem: Working together for patients,” in Washington, DC

Moderated discussion: “Advancing the innovation ecosystem: Working together for patients,” in Washington, DC

On April 3, 2019, Francis Collins, MD, PhD, director of the National Institutes of Health (NIH), Mikael Dolsten, MD, PhD, chief scientific officer and president, Worldwide Research, Development and Medical, Pfizer, joined in a discussion led by Susan Dentzer, visiting fellow at the Robert J. Margolis Center for Health Policy, Duke University, in Washington DC.

Photo: Francis Collins, MD, PhD, director of the NIH (left) and author Alejandro Hernandez, editorial manager of the Alpha-1 Foundation (right)

For 60 minutes, Collins, Dolsten, and Dentzer engaged in a conversation with the theme of: “Advancing the innovation ecosystem: working together for patients,” where they emphasized the need for continued collaboration between the private and public sectors, along with the patient community, in order to advance new and emerging technologies and innovative approaches for potentially life-saving treatments and new outcomes in medicine.

“We are currently living in remarkable times with amazing things happening in areas such as cancer immuno-therapy, gene therapy, and gene editing. The things that are happening right now, I had no idea they would even be possible just five or six years ago,” expressed Collins when asked about the current position “the ecosystem,” as they described the collaboration between the private and public sectors with small biotech companies; an ecosystem with symbiotic characteristics where all contributors play an important role in a tremendous team effort, in medical research.

Collins and Dolsten agreed on the importance of advocacy groups and organizations as well as the patient community, in being part of the process for better outcomes and the ability to bring new and potential treatments for various conditions to the clinic, including rare diseases, such as Alpha-1 Antitrypsin Deficiency (Alpha-1). 

“We need you, the patient community, you are the reason we have gotten this far,” said Collins.

“One of the most important resources needed for this ecosystem to strive is you, the patient. We need to line-up our resources and keep the ecosystem going, including the patient community. There is no one better to bring together what others (physicians) can do than patients: you inspire all of us,” expressed Dolsten.

The Alpha-1 Foundation applauds and supports these types of initiatives, in which patients become partners and are of vital importance for therapeutic development. 

The panel also mentioned that it is critical to spread awareness and education in the medical community, and being able to have early diagnosis and detection, while mentioning the Accelerating Medicines Partnership (AMP), a public-private partnership between the NIH, the U.S. Food and Drug Administration (FDA), multiple biopharmaceutical, and life science companies, as well as non-profit organizations to transform the current model for developing new diagnostics and treatments by jointly identifying and validating promising biological targets for therapeutics.

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

“Patient participation is critical for AMP, especially to remind us what we are doing and why-and-who we are doing it for,” said Collins, who also mentioned the Foundation for the National Institutes of Health (FNIH) as being instrumental in bringing together funds and resources from both the private and public sectors, to work in mutual collaboration in research projects that will ultimately benefit the patient community.

The Alpha-1 Foundation applauds these types of initiatives that support research and bring innovative approaches to the table for the benefit of the patient community, as we are committed to finding a cure for Alpha-1 and improving the lives of Alphas, and those affected by the condition, worldwide.

For more information, please visit the Collaboration Ecosystem website and the AMP website.


Foundation presents Building Friends for a Cure awards

Monday, June 14, 2010
ORLANDO, FL—The Alpha-1 Foundation presented its Building Friends for a Cure awards Friday evening during the Alpha-1 Association’s annual educational conference. The Alpha-1 Foundation presented awards to the following. All-Star...

Alpha-1 protein to be tested as treatment for Type 1 diabetes

Wednesday, June 9, 2010
MarketWire DENVER, CO—Omni Bio Pharmaceutical, Inc. announced that the Barbara Davis Center for Childhood Diabetes has received regulatory clearance from the U.S. Food and Drug Administration (FDA) to initiate a Phase I and II clinical...

Childhood liver disease studies enrolling patients; some travel funds available to eligible families

Tuesday, June 8, 2010
The Childhood Liver Disease Research and Education Network (ChiLDREN) is enrolling children now for vital research studies on cholestatic liver diseases of children. In cholestatic liver disease, the flow of bile from the liver into the digestive...

Grifols, Talecris press release on planned acquisition

Monday, June 7, 2010
PR Newswire BARCELONA, Spain and RESEARCH TRIANGLE PARK, NC—Grifols a global healthcare company and leading producer of plasma protein therapies, and Talecris, a US-based biotherapeutics products company, today announced that they have...

Spanish company Grifols to acquire Talecris for $3.4 billion

Monday, June 7, 2010
Reuters Spain’s Grifols has agreed to buy US-based Talecris Biotherapeutics, which makes plasma-based protein therapies, for $3.4 billion in a bold move to expand its business in blood products. Talecris is the manufacturer of Prolastin,...

Marta Strock dies; one of first AlphaNet coordinators

Monday, June 7, 2010
Marta Strock was one of the first AlphaNet coordinators, and proud of it. “As an AlphaNet coordinator,” she told a magazine writer in 2004, “I have the opportunity to bring a wealth of knowledge about our disease to many Alphas...

Anti-seizure drug improves liver disease in Alpha-1 mice

Friday, June 4, 2010
A note from Robert A. Sandhaus, MD, PhD, clinical director of the Alpha-1 Foundation and medical director of AlphaNet: In the interest of presenting the latest information to our Alpha-1 community, the following press release has been posted...

Whom to test, whom to treat, for Alpha-1?

Friday, June 4, 2010
An examination of the current issues in testing and treating for Alpha-1 has been published by Robert A. Sandhaus, MD, PhD, in the journal Seminars in Respiratory and Critical Care Medicine. Sandhaus is clinical director of the Alpha-1...

Article Search

Enter keywords in text box and click Search button for results.