A new partnership combining AI and rare disease drug discovery with a patient-centered treatment network is seeking to unlock drug cures for the world’s most intractable diseases.
AI-powered, patient-inspired technology company Healx – which is headquartered on Hills Road – has teamed up with US-based Mission: Cure, a non-profit coalition of patients, doctors, researchers, and entrepreneurs who have already invested significant resources in the search for new medicines.
Mission: Cure is a non-profit organization that aims to dramatically improve the health and well-being of people suffering from chronic and recurrent acute pancreatitis within 10 years, demonstrating a new model for curing disease. It was founded in 2017 by Megan Golden and her brother Eric Golden after they struggled for years to find treatment for Eric’s pain from chronic pancreatitis.
Today the Mission: Cure team is based in New York City, Los Angeles, and Chicago, with partners across the US and around the world. Mission: Cure partners with patients and their families, researchers, clinicians, pharmaceutical companies, and other stakeholders to discover life-altering therapies – including repurposed drugs – and bring them to patients. The initial hope is that the collaboration will discover and advance the world’s first treatments for chronic pancreatitis, a rare inflammatory condition that affects over a million people across the globe.
Megan Golden, president at Mission: Cure, said: “We are excited to move the needle forward for pancreatitis patients with the help of Healx. Their AI-powered drug repurposing model gives pancreatitis patients hope that they will see an effective treatment for their disease in the next five years.”
Meera Swami, drug discovery alliance manager at Healx, says: “Healx is incredibly proud to be working with Mission: Cure.
“They are an organization which shares our passion for, and investment in, patient-centered care and drug repurposing for rare diseases. We are looking forward to combining our expertise to accelerate novel therapies for chronic pancreatitis and we hope to have more to share in the next few months.”
The partnership will introduce Healx’s Rare Treatment Accelerator program (RTA) to Mission: Cure’s disease knowledge and preclinical, clinical, and patient networks to treat chronic pancreatitis.
RTA focuses on partnering with groups that have identified promising drug repurposing opportunities for rare diseases but need additional support to bring them to patients. RTA 1.0 was launched in October 2019, and the new liaison is the fourth such partnership. All the partnerships are part of Healx’s Rare Treatment Accelerator, an innovative partnership program that connects academic groups, patient groups, and early-stage biotechs with Healx’s team of experts to unlock the power of repurposed drugs for rare diseases – 95 percent of which are still without an approved treatment. RTA v2.0 was launched last November and is focused on partnering with groups that have identified promising drug repurposing opportunities for rare diseases but need additional support to bring them to patients. Recognizing the all-too-common lack of financial investment and clinical expertise needed to translate repurposed drugs from research to reality, Healx will use its AI, drug development, and clinical expertise, as well as significant financial resources, to evaluate, enhance and accelerate the repurposed drugs, with the aim of starting a clinical trial within six to12 months.
Healx has always believed in the importance of early and continued engagement of patient groups for all of the projects in its pipeline to ensure that the treatments it develops provide meaningful patient benefit. The partnership with Mission: Cure is the newest and builds on the team’s announcements last year that it would be working with the Children’s Tumor Foundation, Muscular Dystrophy UK and the Foundation for Angelman Syndrome Therapeutics to identify and progress treatments for their rare conditions.
Around 95 percent of 7,000 possible rare diseases have no treatment. Chronic pancreatitis is a sustained inflammation of the pancreas, the organ which helps digestion and regulates blood sugar. It can irreversibly affect the organ’s ability to function, leading to severe abdominal pain, scarring, digestive problems, and, often, diabetes. It is also linked to an increased risk of pancreatic cancer. For many patients, the only hope is the removal of the pancreas.
Healx was founded in 2014 in Cambridge by biochemical engineer Dr. Tim Guilliams and Dr. David Brown, co-inventor of Viagra and ex-global head of drug discovery at Roche.