Biomanufacturing to reduce the cost of regenerative medicine

Mayo Clinic director on how progress in biomanufacturing is key to ensuring that regenerative medicine is made available to all.

The world-renowned Mayo Clinic, widely regarded as one of the world’s leading medical facilities and research institutions, has formed a strategic biomanufacturing collaboration with National Resilience Inc. to deliver novel biotherapeutics for rare and complex conditions. The aim is to establish Mayo’s Minnesota headquarters as a centre for biomanufacturing regenerative medicines, with the ultimate goal of bringing new cures to patients.

Longevity.Technology: As a non-profit organisation, the billions of dollars Mayo Clinic generates are invested back into pioneering research into many fields, including regenerative medicine. Beyond the fundamental research it conducts, the organisation is also focused on addressing some of the challenges of bringing regenerative medicine to patients. One such challenge is how to biomanufacture therapies derived from human sources — cells, blood, enzymes, tissues, genes, or genetically engineered cells — for use in medicines. We spoke to Dr Julie Allickson, director of Mayo Clinic’s Center for Regenerative Medicine, to learn more about the organisation’s work in biomanufacturing.

Speaking on the recent collaboration with biomanufacturing technology company National Resilience, Allickson said. “Mayo is making significant investments in facilities to create the world’s most advanced and innovative ecosystem for the development, manufacture and delivery of biotherapeutics. We are seeking to build a community of innovators to explore ideas, develop new products and create thriving biotherapeutic companies.”

 As a non-profit organisation, the billions of dollars Mayo Clinic generates are invested back into pioneering research into many fields
Dr Julie Allickson, director of Mayo Clinic’s Center for Regenerative Medicine

Having started her career working on bone marrow transplantation, one of the first examples of regenerative medicine in practice, Allickson has worked in the discipline for more than 30 years and has watched it grow from the beginning. A crucial moment came around 10 years ago, when the first cancer patients were receiving immunotherapy in clinical trials.

“We saw these stunning results that we’d never seen before, where you change the lives of 70% of those patients that were in the clinical trials, and FDA allowed us to fast track that into a licenced product,” says Allickson. “I feel like that was the point where we saw a difference and saw this was something that’s really making a difference in patients’ lives.”

Biomanufacturing is maturing

While there were only a very small number of Phase 1 clinical trials in regenerative medicine 10 years ago, Allickson is excited to see how things have moved forward since then.

“When you look now, probably half of those early stage biotherapeutics trials are now in Phase 2 trials, and a fair number are in Phase 3, so we’ve really matured with the therapies,” she says. “And progress in biomanufacturing is also making a difference because more people are now focused on that – trying to understand what we have to do to bring the costs down but also how to standardise the product.”

Biomanufacturing is maturing

Thanks to technological advances, biomanufacturing is also now maturing in a way that wasn’t possible in the early days of regenerative medicine.

“In the beginning, we just didn’t have the technology but now we have a lot of choices to be able to automate and bring things together,” says Allickson. “There’s a lot more published data on what occurs as you’re doing the biomanufacturing, so we’ve improved significantly in that space, and that’s really making a difference.

“Using platform technologies, we have the opportunity to bring the cost of regenerative biotherapeutics down. Because in healthcare not only do we want to be able to change people’s lives, but we want to make sure that everybody can afford it.”

Making regenerative medicine affordable

The quest to make regenerative medicine affordable and available to all is at the heart of Mayo’s recent collaboration with National Resilience.

“Resilience is a large technology development company, and they will be setting up a space in our Destination Medical Centre, where we will be working side-by-side with them to scale up our technology as you would see it an industry setting,” says Allickson, who wants to avoid the problems that can happen when early stage therapies move into commercialisation.

“When you consider all the time, money and effort it takes to get regenerative biotherapeutics to a Phase 1 trial, we want to set things up so that a therapy can always move to the next step – we want to make sure that we’re setting it up for success. Because without that it’s going to die – if a pharma company has to spend too much money to commercialise a therapy, it’s not going to happen.”

Having only been in her new role for a year, Allickson is excited about the opportunities for Mayo Clinic to collaborate with industry.

“There are so many people standing at the door waiting to see what we have in discovery,” she says. “Within regenerative biotherapeutics alone, we have about 100 discovery projects that we’re sorting through right now. We’re working on a strategic analysis to look at what are the most important things that we should be taking forward and investing in.”

Regenerative medicine applications

Outside of producing biotherapeutics for a range of cancers, Mayo is also working on a host of other regenerative medicine projects, including the ability to produce banks of induced pluripotent stem cells (cells derived from skin or blood cells that have been reprogrammed back into an embryonic-like state).

“That work allows us to be able to differentiate those cells into any cell that we want, and we’re focusing a lot of our research in that space looking at neurodegenerative diseases like Alzheimer’s,” says Allickson. “But it’s a very exciting area, because those cells really open up the opportunity to potentially treat in any space.”

Mayo is also doing a lot of work in orthobiologics (substances to help musculoskeletal injuries heal quicker) for applications such as cartilage regeneration.

“We have a programme called the Cartilage Bank, where if there’s excess cartilage in a surgery, we’re able to store that and use that for another patient,” says Allickson. “We also have a clinical trial where we’re taking a small amount of cartilage from a patient that has osteoarthritis, and we’re adding mesenchymal stem cells, which modulates the immune response in that area and then injecting those.”

As with many drug development operations, a key component of what may dictate whether a project moves forward is whether it has IP or not. But Mayo is also sometimes prepared to move forward with therapies that don’t have IP associated with them.

“If a therapy is for a rare disease, then we may have to think out of the box to figure out how to treat those patients,” says Allickson. “So sometimes we have to determine how we can bring those therapies in, even though they might not move out to a company. Because the real goal is to be able to have these therapies for rare and complex diseases – especially if patients can’t be treated anywhere else.”

Photographs: Mayo Clinic