Busting the Myths about Pancreatic Enzymes for Pancreatitis and EPI
Dr. Steven Freedman, MD, Ph.D., a worldwide expert on pancreatic disease and cystic fibrosis, presents Busting the Myths about Pancreatic Enzymes for Pancreatitis. In this webinar, the fifth in Mission: Cure’s Patient and Family Webinar Series, Dr. Freedman explains:
What causes exocrine pancreatic insufficiency (EPI)?
How is EPI diagnosed and treated?
When should you take PERT and how do you calculate the correct dose?
Is PERT helpful in treating the pain of chronic pancreatitis?
How does PERT affect patients with diabetes?
What new developments are in the pipeline to treat EPI?
Dr. Freedman address these questions and more in this webinar replay as he debunks the myths and solves the mysteries that surround pancreatic insufficiency and pancreatic enzymes, followed by a robust Q & A session with pancreatitis patients who participated in the live webinar.
On February 28, 2019, Rare Disease Day, an alliance of leading life-science companies, in partnership with Mission: Cure, announced the promising results of a 4-month “datathon” aimed at identifying potential treatments for chronic pancreatitis. Using artificial intelligence and statistical techniques, datathon participants identified 4 existing, approved drugs as repurposing candidates worth testing as possible cures for pancreatitis.
New Pancreatitis Treatment Candidates
The drugs identified are believed to address (target) specific defective genes or processes that lead to pancreatitis.
Lacosamide, targeting Cathepsin B
Dapsone, targeting the cystic fibrosis transmembrane conductance regulator (CFTR gene)
Megan Golden, Mission: Cure’s co-founder and co-director, says, “We look forward to taking the promising candidates to the next step where we hope they will help us find effective treatments for this difficult, rare disease.”
A Unique Public-Private Partnership
Mission: Cure would like to thank its collaborators in the datathon: Elsevier, a data analytics firm that supplied the innovative artificial intelligence framework used in the datathon, the Pistoia Alliance, a nonprofit consortium founded by major life science companies that works to lower barriers to innovation, and Cures Within Reach, a nonprofit leader in research to apply already-approved drugs to treat new diseases.
Two academic scientists played major roles in the project. Mission: Cure is grateful for their work and hopes to continue to collaborate with them.
Dr. Bruce Aronow is the John Hutton MD Professor of Biomedical Informatics and Co-director of the Computational Medicine Center at Cincinnati Children’s and has developed a computable data model network for chronic pancreatitis that helped determine which drugs were most likely to correct the biological problems causing pancreatitis.
Dr. Aleksandar Poleksic, Professor, University of Northern Iowa, used data science to predict which drug candidates would have the least side effects for patients.
Successful Collaboration Between Patients, Industry and Academia
The success of this project highlights the role that partnering with pharma and life sciences companies and academic institutions can play in bringing us closer to discovering a cure. It is by pooling our expertise that we will achieve our mission and bring new treatments to those who need it the most.
To get updates on Mission: Cure’s efforts to test these drugs and bring those that work to patients, sign up for Mission: Cure’s email list.
We had so many questions during our webinar on the genetics of pancreatitis, which can be viewed here, that we could not answer them all during the session. We didn’t want to leave questions unanswered, so here are Dr. Mark Haupt and Dr. David Whitcomb‘s answers to the questions we didn’t get to:
On December 8th, Mission: Cure hosted an informational webinar for chronic and recurrent acute pancreatitis patients, their families and clinicians on their new approach to curing pancreatitis. It featured co-directors Megan Golden and Linda Martin, who gave presentations on the gap Mission: Cure is working to address, their innovative financing model, and how patients and families can get involved and help! Attendees gained unique insights to the projects Mission: Cure is spearheading to provide promising pathways toward effective treatments and cures.
Mission: Cure believes that new innovations in medical research are key to achieving our goal of curing chronic pancreatitis. For this reason, we strive to leverage and support advances in genomics and precision medicine and aim to collaborate with forward-thinking and mission-driven organizations. Today, we are excited to announce our partnership with Perlara, a rare disease drug discovery platform company working with highly motivated families, foundations and drug developers. Together, we have formed a n=1 drug discovery PerlQuest pact with the goal of discovering promising compounds for rapid testing in patient-matched organoid models of pancreatitis to get treatments to patients faster.
In Stage One of the PerlQuest, Perlara will engineer ∆F508 and 26 other CFTR disease-causing variants in yeast YOR1 models of CFTR deficiency with Mission: Cure’s support. The project will then involve completing drug repurposing and drug discovery screens with the ∆F508 model and another mutant, e.g., class III or class IV.
In the words of Perlara founder and CEO Ethan Perlstein, PhD., we look forward to scientific firsts for rare disease drug discovery.
Mission: Cure aims to demonstrate a new model for curing disease, leveraging outcome-based financing and impact investing. Our first mission is to find a cure for CHRONIC PANCREATITIS, a painful and disabling disease that affects 150,000 people, including 10,000 children. Our goal is to accelerate the development of promising new treatments and find a cure. But we can’t do it alone.
Pancreatitis in the news
Yes, you read that right! The popular Swedish DJ Avicii died at age 28 in late April after suffering from pancreatitis for several years. All of the major news outlets covered it and “pancreatitis” trended on social media. Unfortunately, some reports made inaccurate statements that pancreatitis is usually caused by alcohol. These generalizations are harmful to patients. Mission: Cure is committed to educating patients, the medical community and others about the latest science and ending the stigma and denial of care that can result from misinformation.
Calling all people living with pancreatitis! Mission: Cure is launching a new awareness and education program. With support from the AbbVie Foundation and in partnership with the National Pancreas Foundation, we will hold a series of webinars where pancreatitis experts will share the latest research and answer patients’ and caregivers’ questions. Also, our blog is coming soon plus we regularly share news and updates on social media. Connect with us!
Our ambitions are BIG but we need your help! We are developing a patient survey to understand patients’ needs, preferences and priorities. Our goal is to help the FDA and other government agencies, medical researchers and healthcare professionals focus on what is important to those affected by pancreatitis. Watch for the survey this Fall. To increase our impact, we urge everyone to participate. Please spread the word to all patients and family members!
An effective treatment for pancreatitis may already be in your drug store… We have identified 36 approved drugs that scientists believe could treat chronic pancreatitis. These include some statins, rapamycin and drugs approved to treat cystic fibrosis. Since these drugs have already been approved for human use, clinical trials can happen quicker and much less expensively than new drugs. We are working with leading scientists and Cures Within Reach to design efficient clinical trials to test the most promising of these drugs. Stay tuned!
Pursuing a “Social Impact Bond” in England The innovative financing model Mission: Cure is planning originated in the United Kingdom in 2010. Called a Social Impact Bond (or SIB, even though it was not really a bond), the first of these was used to fund programs to prevent crime and recidivism. In 2016, Cures Within Reach began exploring whether a SIB could fund repurposing of generic drugs to cure rare diseases in England, with funding from Big Lottery and support from the National Health Service. Cures Within Reach and Mission: Cure are now working with a pro bono social innovation team at Medidata to apply this financing to curing pancreatitis in the UK.
The Nonprofit Finance Fund, which maintains a website for people interested in innovative financing, recently published a blog by co-founder Megan Golden on Mission: Cure’s model. You can read it here: http://www.payforsuccess.org/blog/outcome-based-financing-cure-disease
The Mission: Cure team is growing! Linda Martin joins Mission: Cure as co-director.
Before joining Mission: Cure, I spent over 30 years as a serial entrepreneur, launching and growing companies in the fast-paced mobile telecom industry. It was a challenging and rewarding career and I relish the many friendships that I made. However, my life instantly changed when my daughter was diagnosed with Chronic Pancreatitis. After months of research, I learned that little is understood about this disease and existing treatments are ineffective. I was frustrated but committed to changing the status quo and finding effective treatments for my daughter and everyone living with pancreatitis.
As co-director of Mission: Cure and the mother of a CP patient, I’m obsessed with the urgency to develop improved therapies and find a cure. There is much to do to accelerate research and drug discovery for those living with CP. Together we can raise awareness, drive new discoveries and impact lives. I hope you’ll join us!
Mission: Cure is grateful for the support of the National Pancreas Foundation, Cures Within Reach, the AbbVie Foundation and, especially, the many individuals who have donated to our cause. Every contribution, regardless of size, makes a difference and is an investment in education, research, and advocacy to find a cure for chronic pancreatitis. Mission: Cure depends upon your support to continue this important work.
Megan Golden presented Mission: Cure’s strategy on the Brookings “Impact Bonds for Health” Webinar on December 12th. View the presentation below. For more information please follow this link to the Brookings Website.
“Mission: Cure” Launched to Harness Innovative Financing and Impact Investing to Cure Disease; Pioneers New Approach by Attacking Pancreatic Disease
New York, New York, Tuesday, November 7, 2017—The new nonprofit Mission: Cure will convene the world’s leading pancreatic experts at the American Pancreatic Association meeting in San Diego on November 10th to devise a 10-year plan to cure chronic pancreatitis, a painful and costly pancreatic disease, using an innovative financing model that could be applied to cure other diseases.
Building on the global movement toward outcome-based financing, Mission: Cure will partner with healthcare payers and philanthropists to provide financial incentives for improving patient outcomes. Mission: Cure will work with researchers, clinicians, patients, providers, industry, and entrepreneurs to achieve those outcomes.
The strategies will include using advanced genetic testing and data aggregation tools (precision medicine), working with scientists and the FDA to efficiently move promising therapies into clinical trials, and repurposing drugs approved for other diseases. “Repurposing already-approved drugs is a faster and less expensive way to get new treatments to patients. Cures Within Reach has facilitated numerous successful repurposing efforts and will help Mission: Cure deploy this strategy as its fiscal sponsor,” said Cures Within Reach CEO Dr. Bruce Bloom.
Joe Titlebaum, Chair of the National Pancreas Foundation, said, “Mission: Cure’s groundbreaking approach is at the cutting edge of efforts to cure disease. The more than one million people suffering from chronic pancreatitis globally will benefit from their focused effort to bring new therapies to people in need.”
Mission: Cure will work with the growing group of impact investors who want their investments to produce social benefits in addition to financial return to create a new type of health investment where investors can make a quantifiable impact on people’s health while potentially receiving a return from patient outcome payments and the market.
About Mission: Cure
Mission: Cure is a nonprofit founded in 2017 by Megan Golden, an expert in innovative financing for social and health programs, and her brother Eric Golden, an entrepreneur, investment banker, and pancreatitis patient. Mission: Cure is partnering with the not-for-profit Cures Within Reach, which is serving as its fiscal sponsor, and the National Pancreas Foundation, to pioneer a new approach to curing disease using financing based on patient outcomes and partnerships with impact investors.