Biotech CEO Launches Moonshot Ideas to Create Reproducible Human Organs

United Therapeutics Corp wants to conquer the global shortage of organs.  

CEO Martine Rothblatt, during a 2015 TED discussion, posed the idea that an unlimited supply of transplantable organs could keep people living indefinitely the same way interchangeable parts keep cars and planes from becoming inoperable.   

She envisions offering custom-designed organ transplants to patients with life-threatening illnesses.  

Specifically, United hopes to achieve that goal by using 3-D printers to manufacture human lungs for patients with chronic or terminal lung ailments.  

In May of this year, United successfully printed a replica of the upper part of a human airway: the windpipe and the first two bronchi. Using collagen, a protein that maintains our body’s structures, the company took the first step toward creating organs in unlimited quantities.  

Within a few years, the company hopes to generate a solid lung structure which includes the descending branches of the airway, the alveoli, and the entire network of capillaries.  

The effort cannot stop there, though, because a collagen lung isn’t sufficient to replace a living lung. The next step, then, is to impregnate the structure with human cells in hopes that they’ll attach and burrow into the lung, bringing it to life.  

Derek Morris, project leader in United’s organ manufacturing group, equates it to building little stick houses for cells to live in.  

Organ Strategies 

One strategy United employs is xenotransplantation, or transplanting an animal organ into a human. For its efforts, the company uses genetically engineered pigs. 

United acquired a company to help it produce those pigs and then enlisted the help of Synthetic Genomics Inc. to edit pig genomes so that the organs will be accepted by the human body rather than rejected.  

Finally, United owns a laboratory that will ultimately be capable of producing up to 1,000 sets of healthy pig lungs, or xenolungs, from genetically engineered pigs.  

United’s separate efforts at building organs involve tissue regeneration. The effort begins when scientists strip the cells from a donor pig lung and use the remaining collagen as an organ “scaffold.”  

The scaffold is infused, or recellularized, with billions of human cells, replacing the pig collagen with human DNA-directed collagen that is capable of breathing. The final product, she said, would be a completely human lung.   

United has created an assembly line capable of decellularizing more than 500 scaffolds a year, as well as a similar assembly line for recellularizing.  

The technology is promising, but like other options, it faces a major obstacle: the need for billions of human cells to infuse into the scaffold.  

Of all the options, the 3-D effort would be easier to scale, and far less expensive. Additionally, 3-D printing would allow the generation of lungs that specifically fit the patient’s chest cavity.  

Although bioprinting tissue has been done before in the form of human skin and even retinas, the method has been limited to very small tissues that lack blood vessels. In fact, most experts believe that bioprinting is the least challenging part of printing new organs. 

Unusable Organs 

Rothblatt also discovered through her work that more than half of Americans elect to be organ donors in the event of death. Paired with the knowledge that about 2,000 lung transplants occur each year, she wondered what happened to the rest of the organs? 

It turns out that most lungs are deemed unusable because they carry cancer or infectious disease. In other cases, they were filled with fluid and mucus at the time of death, so they were no longer usable by a patient whose health was already compromised. 

United’s subsidiary Lung Bioengineering refurbishes human lung donations by infusing warm solution into them to keep them usable. More than 250 people have benefitted from the revitalized lungs that otherwise would have been discarded. 

Career Change 

If it seems like Rothblatt is all-in for lung research, she is. She left her engineering career in the 1990’s where she was the founding CEO of Sirius Satellite Radio after doctors diagnosed her daughter with a rare lung disease.  

At the time, pulmonary hypertension patients didn’t survive beyond five years of their diagnosis. Rothblatt rallied the efforts of people she knew to develop a medicine to address the condition and then gained FDA approval for the medicine. 

Since then, the number of patients with pulmonary hypertension has ballooned from 2,000 to 40,000, because patients are surviving the diagnosis now.  

Better yet, they’re living normal lives.  

Rothblatt refers to her work as turning moonshots into earthshots, and she predicts that much of United’s technology could become reality within the next five years. Although the process is slow, the company anticipates gaining federal approval to employ its manufactured organs by the year 2025.