Pioneering research recognized by the 2018/19 Gates Grubstake Awards underscores promising strides in recent years in guiding basic science at the CU Anschutz Medical Campus toward commercialization and tangible benefits for patients.
Kenneth Liechty, MD
The Grubstake Fund, with multiple awards of about $350,000 each year, is meant to bridge the “valley of death” funding gap that separates a promising medical concept from production and a business model that will make a difference in human lives.
It’s working.
The Gates Center for Regenerative Medicine and the CU Innovations Office, its Grubstake Fund collaborator, report that through early 2019, the cumulative $3.5 million in Grubstake awards had already produced nearly $20 million of follow-on funding from partners seeking to help develop and commercialize the concepts.
The more-than “5X” return is remarkable in any financial setting. For the Gates Center, it is confirmation that the mission of collaboration and entrepreneurship in the name of medical advancement is now producing results.
“The Grubstake Fund program has been so game-changing as far as impact and getting research to patients,” said Kim Muller, executive director of CU Innovations. “We are looking at using that model for all therapeutic areas around campus. We almost feel we have an ethical obligation to do it.”
This year's winners
The 2018/19 Gates Grubstake Fund winners of $350,000 grants were selected from 15 applicants representing CU Anschutz schools, centers and institutes, CU Boulder, CU Denver and Children’s Hospital Colorado. They are:
- Kenneth Liechty, MD, who is developing an inhaled formulation for the prevention and treatment of pulmonary fibrosis. Acute lung injury leading to Acute Respiratory Distress Syndrome (ARDS) causes 150,000 adult ICU admissions and 75,000 deaths annually in the US. Liechty and his team use nanoparticle therapy to regenerate healthy lung tissue in a mouse model of acute lung injury. The award will support an inhaled therapy and safety studies required prior to a clinical trial in ARDS patients.
- Kunhua Song, PhD, for the regeneration of functional heart tissue from tissue damaged in a heart attack. Eight million Americans suffer a heart attack annually, and up to 40 percent of those develop heart failure. Despite the development of various therapies and medical devices, the five-year survival rate has not changed in the past 20 years. Song and his team have discovered a way to regenerate healthy cardiac cells from the fibrotic tissue causing heart failure using a gene therapy approach. The Grubstake Award will allow them to complete their animal studies and begin preparing to file an IND to enter clinical trials.
- Michael Verneris, MD, for the generation of engraftable hematopoietic stem cells for use in bone marrow transplants. Bone marrow transplants are a high risk, high cost procedure used to treat leukemia, lymphoma, and genetic diseases. Autologous donation is the safest and quickest method. Recently, scientists have discovered how to create induced pluripotent stem cells (iPS cells) from a patient’s own cells, expand them, and convert them to bone marrow cells. So far these cells have not engrafted into the bone marrow of mouse models. Verneris and his team have discovered that adding a small molecule during the conversion of the iPS cells allows engraftment. The Grubstake Award will allow them to confirm their initial finding in animal models, and to determine the most promising small molecule for clinical and commercial use in humans.
Kunhua Song, PhD
To date, there are 105 ongoing regenerative medicine projects on campus with potential to compete for Grubstake awards, of which 25 have reached the product development stage. As word spreads of the program’s opportunities for researchers, being selected for the handful of awards has become increasingly competitive.
The Grubstake Fund goes a significant way toward addressing a gap between academia and “the real world” that has existed since the founding of medical schools. Researchers are constantly trying to broaden the impact of their work to help more than an individual patient. Yet when they have a promising concept, their scientific training lacks the background in regulatory approvals and how to access the capital required to prove a concept’s commercial value.
Intersecting with the private sector
“They’ve never had to intersect with the private sector,” Muller explained. “There’s no education in medical school or at work that helps them prepare for that.”
Members of the public who read casually about venture capital (VC) in hotbeds like California’s Silicon Valley may assume there are financiers with suitcases full of ready cash hovering around all PhDs. But that kind of capital tends to move in when an idea is ready for mass commercialization, innovation experts said. Before that stage, scientists need money for lab assistants and regulatory help to show that something seen under a microscope has real promise as a treatment.
“When you are at the proof-of-concept translational stage, that’s where most VCs aren’t going to invest,” said the Gates Center’s Entrepreneur in Residence Heather Callahan, who also serves as portfolio manager at CU Innovations, bringing the expertise of an Executive MBA, a law degree and a PhD in biochemistry. “That’s why this was the perfect place in the trajectory of invention to start putting money.”
The money and the mentoring have moved a growing portfolio of projects toward initial clinical trials in the next two to three years.
Michael Verneris, MD
“The Gates Center has been phenomenally helpful in accelerating our work,” said Liechty, who received a previous Grubstake award in anti-inflammatory research. The Grubstake model and the Gates Biomanufacturing Facility address three keys to the drug commercialization process, he said: “It’s one thing to say, ‘Okay, we can manufacture things.’ Or another thing to say, ‘We have people who know the ins and outs.’ And another to say, ‘How do you pay for it?’ We’re a relatively new campus for that kind of idea, and this is what’s needed to really jumpstart this.”
Applications due at the end of August
The annual Grubstake award process has helped everyone involved with the Gates Center and wider campus research efforts keep an eye on the true significance of the scientific pursuit, said Gates Center Associate Director J. Mark Petrash, PhD.
“This is an important part of the mission of the university, to not only do good research but to make it of practical use to society,” he said.
Written applications for the 2020 round of Grubstake Awards are due at the end of August. Interested applicants are encouraged to reach out to the Gates Center’s Entrepreneur in Residence Heather Callahan for information.
Guest contributor: Michael Booth
