ROADMAP TO A CURE

Mission: Cure has worked tirelessly since 2017 to bring stakeholders together in search for a cure for chronic pancreatitis. We held two meetings of scientific experts at the 2017 annual conference of American Pancreatic Association ( APA) and PancreasFest 2019. We have also identified prominent advisors with substantial drug development experience (including developing several widely used drugs and vaccines). 

Below is an overview of our multi-pronged strategy to cure chronic pancreatitis.

Accomplished

Get government support for and assistance with developing therapies for pancreatitis patients

  •  Organized & led ​patient panel​ ​at 2018 National Institute of Diabetes and Digestive and Kidney Diseases ​meeting to expedite therapies for pancreatitis 
  •  Co-sponsored patient-focused drug development meeting with FDA & contributed to Voice of the Patient Report
  •  Won support for including chronic pancreatitis in FDA-funded Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP)
  • Participated in Rare Disease Day on Capitol Hill and Digestive Disease National Coalition meetings with Congressional staff to educate members about pancreatitis and the needs of patients
    • Submitted grant to NIH with partners to test repurposed drugs for pancreatitis. Not funded due to COVID. 

Partner with Life Sciences and Biotech Companies to Pursue Pancreatitis Therapies

  • Presented to more than 10 biotech and pharmaceutical companies on the urgent need for therapies and commercial opportunity

Advance patient-focused drug development

Identify and test drugs approved for other diseases (Drug Repurposing)

  • Invited to join Healx’s Rare Treatment Accelerator Program (RTA) with the aim of starting a clinical trial and eventually develop a treatment for pancreatitis. 
  • Identified​ 60 potential drug repurposing candidates​ ​that have evidence of action on pancreatitis targets. 
  • Awarded ​Innovative Practices ward for Elsevier/Pistoia Alliance Datathon​, an international data science competition that generated 300 drug repurposing candidates for chronic pancreatitis

Move drug candidates through development process

  • CFTR
  • PRSS1
  • SPINK1
  • CA 19-9
  • Stem cell therapy

Identify new targets and drug candidates

  • Applied and got approved to be part of the FDA-funded Rare Disease Cure Accelerator-Data and Analytics Platform (RDCA-DAP)
    • Identified and met with researchers studying innovative therapeutic targets and drug candidates
  • Monitored scientific developments in other diseases that could apply to pancreatitis; met with researchers to determine whether they will work for pancreatitis

Establish an efficient drug development process

  • Participated in American Pancreatic Association committee on animal models; identified researchers developing in vitro models; brought impact investor funding to support development of models
  • Identified researchers developing in vitro models; brought impact investor funding to support development of new preclinical models
  • Identified experts in trial design to serve as advisors, partners for potential clinical trial

In Progress

Build Government Support

  • Helping C-PATH get data from researchers for RDCA-DAP
  • Identifying and applying for additional government grants to fund translational research

Partner with life sciences and biotech companies to pursue pancreatitis therapies

  • Working with life sciences companies (e.g. Vertex) to make the case for chronic pancreatitis work and to design feasible clinical trials

Advance patient-focused drug development

  • Incorporating patient priorities in clinical trial design
    • Consulting with trial design experts on most cost-effective, convenient way to gather data on relevant patient outcomes

Identify and test drugs approved for other diseases (drug repurposing) 

  • Partnering with Healx on moving repurposing candidates through preclinical testing
  • Develop efficient trial design (aggregated n of 1 trials or adaptive trial) to test repurposing candidates in partnership with researchers and trial design expert

Identify new targets and candidates

  • Working with RDCA-DAP to get data from multiple sources
  • Working with researchers to prioritize most promising targets and drug candidates (e.g. Johns Hopkins work on specific pain mechanisms)
  • Working with researchers to secure funding for most promising new approaches
  • Continuing to monitor new research in pancreatitis and new therapeutic approaches for other diseases
  • Working with Machine learning/bioinformatics team to develop a disease model that integrate big data from multiple data sources (e.g. single cell atlas); securing funding to implement that model

Establish an efficient drug development process

  • Helping raise funds and coordinate efforts to develop in vitro models; test those models as a way to screen possible therapies
    • Drafting protocol for “n of 1” trials with experts
  • Seeking funding for n of 1 trials of approved (repurposed) drugs

Future Steps

Build government support

  • Conduct analyses of RDCA-DAP data to inform therapy development
  • Meet with FDA’s Center for Drug Evaluation and Research to discuss patient perspective and requirements for clinical trials

Partner with life sciences and biotech companies to pursue pancreatitis therapies

  • Partner with companies testing therapies to use patient-friendly clinical trial designs; inform patients of clinical trials

Advance patient-focused drug development

  • Provide input into recruitment strategies, instruments, and design of clinical trials for new therapies
  • Inform patients of clinical trials; troubleshoot recruitment problems with researchers

Identify and test drugs approved for other diseases (drug repurposing)

  • Partner with Healx on clinical trials of Healx-discovered therapies
  • Clinical trials of other repurposing candidates in partnership clinical trial sites such as CPDPC sites

Identify new targets and drug candidates

  • Analyze data to learn more about the disease and identify targets and potential treatments
  • Advance innovative therapies for new targets (such as CA 19-9) through drug development pipeline.

Establish an efficient drug development process

  • Support use of in vitro models to identify and develop therapies
  • Implement clinical trials using efficient designs to test approved drugs and new therapies