The Human Organs, Physiology, and Engineering (HOPE) program is a $50 million effort that brings experts in the life sciences and engineering together from across universities, foundations, and companies to develop human tissues, organoids, organs, and platforms that accelerate and scale new treatments.
We need better models of human physiology, in particular, functional organs and immunological responses.A mouse is not a human and dialysis is not a kidney, after all.
Today, drug development relies on preclinical animal studies to test the efficacy and safety of potential treatments, but 90 percent of treatments that succeed in animal studies fail in human trials. A mouse is not a human being, after all. And, in particular, each species has an exquisitely tuned immune system, which impacts many results. This high attrition rate − approximately 50% of failures for lack of efficacy and 30% for toxicity − raises the cost per new drug up to $2.8 billion according to recent estimates. Success in the HOPE program would give researchers the ability to run pre-clinical trials on representative human organs and systems in the lab rather than on animals, which would reduce the attrition rate and in turn, decrease the cost of new drugs.
We need to bioengineer a change.
“If we can transform the time, cost, and risk associated with drug development, we can help spark a new Health Age,” said Regina E. Dugan, CEO of Wellcome Leap. “And that’s exactly what we aim to do.”
The success of the HOPE program would also have transformative impact on organ transplant, particularly for patients with end stage renal failure, who have to spend an average of 5 years on dialysis while waiting to receive a kidney from a living donor.
Of the more than 2 million patients with late-stage chronic kidney disease that receive dialysis replacement therapy, only 35% will survive the wait.
For example, fewer than 25,000 transplants happen each year in the United States. As a result, kidney disease is a top ten killer in the United States, where it is responsible for more deaths than breast cancer. Breakthroughs in our ability to construct non rejected, bio or bio/synthetic kidneys could alleviate the need for a waitlist altogether.
The HOPE program leverages a decade of advances across multiple areas: stem cells and organoids, gene editing, advances in immunoengineering, 3D bioprinting; biomaterials; modular microfluidic systems; and the continued shrinking of feature sizes in semiconductor microelectronic biosensors and devices, which together make it possible to combine targeted biological self-assembly with scalable engineering systems.
The program will focus on bioengineering human physiological systems to achieve two goals: One, a multiorgan platform that recreates human immunological responses with sufficient functional fidelity to double the predictive value of a preclinical trial for therapeutic interventions targeting tumor immunity, autoimmune and infectious diseases. Success for these therapeutics requires a new level of fidelity with respect to our ability to represent and replicate the complex responses of the human immune system. Two, demonstrate the advances necessary to double the 5-year survival rate of patients with end-stage renal disease and point to a fully transplantable, non-rejected kidney within 10 years.
“In the next decade, we have the potential to create fully functioning human organs that pose low rejection risk. This program will fund the breakthrough technologies necessary to enable that bold ambition, precisely the type of effort for which Leap was founded.”
— Jay Flatley, Chair of Wellcome Leap