Cancer vaccine ready for commercialization as part of growing MUSC startup community

In the 12 years since its inception, Leukogene Therapeutics Inc. has had one goal: to take an idea from the academic lab into the real world where it can benefit patients. 

With a new cancer vaccine platform designed to stimulate the immune system and attack cancer, Leukogene offers a therapeutic solution for acute myelogenous leukemia, or AML, and pancreatic cancer. And while it is currently in the preclinical phase, Nathan Dolloff, Ph.D., founder and chief scientific officer of the company, as well as a researcher for the MUSC Hollings Cancer Center, said that with the right strategic partner, they could be ready for clinical trials in 18 months. 

Current treatment for AML and pancreatic cancer relies on chemotherapy drugs, which can have bad side effects and often don’t extend a patient’s life significantly in these cancers. Newer immunotherapy drugs have revolutionized the treatment of some cancer types but are largely ineffective in others like AML and pancreatic cancer. 

Leukogene aims to fill the gap in treatment options with its cancer vaccine. By directing immune cells to attack specific proteins on malignant cancer cells as if they were foreign antigens, the vaccine harnesses the natural power of the body’s immune system and redirects it.

Results from preclinical studies in mice have Dolloff excited for the vaccine’s clinical future. “We’ve seen some remarkable anti-tumor effects in the lab,” he said. “And in some cases, we’re even curing mice in certain tumor models where that’s really difficult to do.” 

Dolloff and his team hope to get this therapy into the hands of clinicians and patients as soon as possible. “Our product is safe and works incredibly well,” he said. “We have the pathway to get it to patients, but our greatest challenge right now is funding.”

MUSC startup companies like Leukogene will soon be housed in the new Blue Sky Labs on the hospital’s Charleston campus. In addition to its own company growth, Leukogene is a good example of MUSC’s expanding innovation and entrepreneurship ecosystem. It’s that opportunity for growth that first brought Dolloff from Pennsylvania to Charleston in 2014. 

“One of the reasons I came down here was because I wanted to have an impact on the startup community at MUSC,” he said. “I’ve always believed in the importance of entrepreneurship in academia. It’s essential if we are serious about taking ideas from the lab and getting them out into the world, so to speak.”

Dolloff said MUSC inspires and encourages innovation by creating an environment with programs that promote collaboration between researchers. Through a partnership with the College of Charleston, Carol Feghali-Bostwick, Ph.D., a professor and researcher at MUSC with a passion for mentoring, encourages women to pursue entrepreneurship skills. She recruits researchers and clinicians for her Coaching and Resources for Entrepreneurial Women program, or CREW, to be mentored and advised by seasoned entrepreneurs like Dolloff. 

Jesse Goodwin, Ph.D., MUSC’s chief innovation officer, was part of the founding of CREW with Feghali-Bostwick, and she points to MUSC’s unique position as an academic medical center, which provides both a hospital and education opportunities to its community with research and innovation at the heart of its mission. 

“One of our primary jobs in our innovation ecosystem,” she said, “is helping entrepreneurs grow their companies to the point where they can be attractive to venture capital, established industry partners or customers, depending on the technology. Blue Sky Labs and our Innovation District will provide a physical home for our companies and also offer opportunities to network with such partners.”

Leukogene Therapeutics Inc. received a small business grant from the National Cancer Institute in 2022, and since then, Dolloff said the team has generated more data and even more confidence in their platform. “We’ve gone from an interesting concept with a little bit of data to something that is potentially paradigm-shifting with a lot of data to support it,” he said. 

Dolloff is looking forward to moving ahead and seeing their drug platform reach patients in the clinic. “That’s the reason we do this. Getting to the clinic and having an impact would be a major milestone for us,” he said.

He also said none of this would be possible without basic science. He noted that decades of great basic science enabled their research and paved the way for new drugs and medical products. He’s seeing basic science and its commercialization intersect at MUSC, which, for Dolloff, makes the opening of Blue Sky Labs pivotal to that process and to companies like Leukogene.