World Intellectual Property Organization

Dr. Anthony Atala, MD: New body parts – the shape of things to come?

December 2013

Imagine a world where if the need arose you could order a spare body part to replace a diseased or dysfunctional one, where doctors could cure rather than simply manage chronic, life-threatening diseases. Could this ever become a reality? Dr. Anthony Atala, MD, Director of the Wake Forest Institute for Regenerative Medicine and the W.H. Boyce Professor and Chair of the Department of Urology at Wake Forest Baptist Medical Center, a pioneer in regenerative medicine, believes it can.

Every 30 seconds a patient dies from diseases that could be treated with tissue replacement.

Watch on YouTube.

Dr. Atala is driven by a deep-rooted desire to offer his patients the best possible treatments. “As a surgeon, there is nothing more devastating than being in an operating room and having to replace a piece of tissue or an organ and not having one to replace it with or not having the ideal treatment,” he said. “To create these tissues and organs outside in the laboratory and to have them available would be a really good option for some patients. That’s what has inspired our work.”

Dr. Atala and his multi-disciplinary team of researchers are adopting parallel strategies to find ways to grow the solid organs people need. Using 3-D printing technology the team is working on projects to create biodegradable scaffolds to produce bone, muscle, cartilage and in the longer-term to print a kidney. The team also re-uses discarded organs which after a washing process are repopulated with a patient’s own cells. (Photo: Wake Forest Baptist Medical Center)

Regenerative medicine offers the potential to transform the medical landscape and patients’ lives, offering new treatments for previously incurable conditions. “The ultimate promise of regenerative medicine is not just to help manage disease but to really improve the lives or even provide a cure,” Dr. Atala said.

Unlike established medical practice, regenerative medicine is patient-specific and targets the underlying cause of a disease by repairing, replacing or regenerating damaged cells. While the idea was aired as far back as the 1930s, “it has taken us several decades to get where we are today,” Dr. Atala notes. Just 30 years ago, he explains, it was not possible to grow most human cell types outside the body. “Today we are at a point where we know how to grow human cells, and we know how to expand them outside the body. We are not yet at a stage where we are implanting solid organs but we are implanting flat; tubular; and hollow, non-tubular organs in patients.”

Levels of complexity

Regenerative medicine recognizes four levels of organ complexity. “Flat structures, like skin, are the least complex, made up mostly of one cell type. They are not as complex as a tubular organ, like a blood vessel or a windpipe which has two cell types and architecturally is a little more complex as it remains open. It is really just a tube acting to allow fluid or air to go through it at a steady state within a defined range,” Dr. Atala explained. Hollow, non-tubular organs such as the bladder, offer a third level of organ complexity in terms of cells, shape and function. In 1999, Dr. Atala led a research team that successfully implanted the world’s first laboratory-grown bladder into a patient who today is living a normal, active life. Solid organs such as the kidney, liver and heart are the most complex organ type. With these organs “there are a lot more cells per centimeter and they require massive amounts of blood vessel supply and involve many more cell types,” he explained.

While the first three types of organs - flat; tubular; and hollow, non-tubular organs – have been successfully implanted in patients, using a combination of cells and/or scaffolds made from biodegradable materials, “the goal is to keep increasing the number of organs that we implant and someday to be able to implant solid organs. Every day we are getting closer,” Dr. Atala said.

Growing need for human organs

Regenerative medicine is evolving in response to a real need. The demand for human tissue is growing. “Every 30 seconds a patient dies from diseases that could be treated with tissue replacement,” Dr. Atala observed. Organ transplant waiting lists continue to grow; every 10 minutes someone is added to the transplant list in the US alone. This is a major problem. “Over a period of a decade the actual number of transplants went up by about one percent but the number of patients on the wait list has doubled,” Dr. Atala noted. “We have a major crisis right now because we are living longer and there’s more chance of organs failing. There is really a need to have organs available so we don’t have to wait until someone dies to be able to transplant one.”

One of the major advantages of regenerative medicine is that by harnessing the body’s innate potential to heal and replacing damaged tissues and organs with new ones grown from a patient’s own cells, organ rejection is all but eliminated. Moreover by focusing on the underlying cause of the disease, the aim is to cure a patient rather than simply manage symptoms or stem a disease’s progression. This promises significantly improved quality of life for patients and enormous financial savings for national healthcare systems.

Without intellectual property protection people will not invest in the technology, so if we want to see these technologies used for patients we need to have intellectual property protection. The technologies depend on it.

A scaffold for a bladder seeded with a patient’s cells. In 1999, Dr. Anthony Atala led a research team that successfully implanted the world’s first laboratory-grown bladder into a patient who today is living a normal, active life. (Photo: Wake Forest Baptist Medical Center)

Strategies for creating new solid organs

Dr. Atala and his multi-disciplinary team of 300 researchers are adopting parallel strategies to find ways to grow the solid organs patients need.

3-D printing organs

Using computed tomography (CT) images and computer aided design (CAD) software, researchers have developed 3-D printers that are designed to engineer new organs. “Our printing machines are very much like an inkjet printer but instead of using ink we are using cells in the cartridge and they are laying the cells down one layer at a time where they are needed to create three-dimensional structures that can lead to functionality,” he explained. The team is working on projects such as bone, muscle, cartilage and a long-term project to print a kidney.

Re-using discarded organs

Researchers are also using discarded organs. These are taken to the laboratory where all the existing cells are washed away using mild detergents leaving the three-dimensional structure of the organ intact. “We would then use the structure as a mold to repopulate it with the patient’s own cells,” Dr. Atala explained. “The idea is to take a small piece of tissue from the patient’s diseased organ, isolate the normal cells and put them back into the organ which would then be put back into the patient.”

The constant search for solutions

For Dr. Atala, innovation is a way of life. “The first step to innovation is just to try because if you don’t try you will never find a solution,” he said. “Anytime we see a barrier we have to find ways to get around it,” he noted, underlining the need to constantly re-examine accepted truths and develop new approaches on the basis of the new knowledge and tools available.

“Our job as scientists,” he notes, “is really to develop the technologies. If we can create technologies that are transformational and will make patients better then healthcare providers will want to use them. Then someone will need to invest in the technology and make sure the intellectual property is there. If all these pieces come together, the technology will be produced and it will be used and distributed for patients and their benefit. But it all starts and ends with having a technology that is transformational for our patients.”

Despite significant breakthroughs, regenerative medicine is still in its infancy. “We still have a lot of challenges. So many things have to happen for so many different organs. When you start expanding the number of organs you can engineer, you expand the indications, there are new uses, new inventions, new methods, new processes. The field is really wide open. It is an area where innovation can really take hold,” he said.

The role of intellectual property

A veteran-user of the patent system – he has applied for or received over 200 patents worldwide - Dr. Atala is a firm believer that IP has a key role in enabling and advancing medical technologies and ensuring they benefit patients. “Intellectual property is so important. The bottom line is that unless there is intellectual property present we don’t have a tool to commercialize these technologies,” to make them widely available and bring their cost down. “Without investment the technology will never be transferred to patients. It takes literally hundreds of millions of dollars to produce and distribute these technologies around the world,” he said. “People need to know that they’re going to get a return on their investment. Without intellectual property protection people will not invest in the technology, so if we want to see these technologies used for patients we need to have intellectual property protection. The technologies depend on it.”

The IP system also enables researchers to “put a stake in the ground” making it possible for the research community to keep pace with the state of technological development. “When you know where the technology is from an innovation standpoint you can build on that and create more innovation. This sharing of information is very useful in advancing towards the future,” he said.

Dr. Atala urged policymakers, to explore ways to bring down the costs associated with obtaining global IP protection. “To get world protection is a very expensive proposition, so you don’t want to eliminate someone from using the IP system because the cost is too high,” he said. Dr. Atala also urged policymakers to streamline regulatory processes to help reduce lengthy timelines and contain costs. “Safety is paramount but you can shorten the timeline by taking some of the bureaucracy out of the system,” he said.

Collaborative research

Regenerative medicine is a complex field drawing on multiple disciplines. Researchers at the Wake Forest Institute for Regenerative Medicine share a lab and rigorously test tissues at every stage of development. “Patient safety is of paramount importance to us,” Dr. Atala said. “We are dealing with patient’s lives so whatever we do we have to make sure that at the very least we do no harm and then that we create a benefit,” he said.

Beyond Wake Forest Baptist Medical Center, the Institute is involved in numerous research collaborations (more than 100 national and more than 50 international). “The goal is to create an international network to distribute these cells allowing these technologies to be worked on by many different scientists,” Dr. Atala explained. Such collaboration is also enabling the Institute to build an international network of clinical trial sites. “At the end of the day, this will help advance these technologies for everyone.”

The next big thing

“We are constantly looking out for potential breakthroughs,” he noted, explaining that the next big thing in regenerative medicine is a series of little things. “We are looking at so many different areas, there are so many small challenges to overcome, small victories to achieve to make the next big advances. It all points to implanting solid organs into patients. That is really going to be a major thing,” he said.

“You should never say never,” he reflected. “If a salamander can re-grow a damaged limb, why can’t we? The potential is there in biology to initiate these systems. The question is how can we make it happen and a better question is when? One thing is certain. These technologies do have the potential to make patients better. For us it is not really about the cells we use, or the technologies we choose, it is really all about making our patients better.”
 

Regenerative medicine is ground-breaking because:

  • it promises to save lives and improve those of patients suffering from debilitating chronic diseases;
  • it signals a move from a one-size-fits-all model to a patient-specific model of healthcare;
  • it eliminates the risk of organ rejection;
  • it focuses on harnessing the body’s innate capacity to heal and the cause of a disease and could potentially cure certain life-threatening chronic conditions;
  • it opens up a new world of medical treatments;
  • it has the potential to transform the healthcare landscape and promises to significantly reduce the healthcare costs associated with treating an increasingly aging and ailing population.

Explore WIPO