Botox for Acne? and Other LSU Health New Orleans Inventions

In-no-vate

1: to introduce as or as if new 2: to effect a change in, to make changes: do something in a new way

How did a pediatric neurologist and an ENT (ear, nose and throat) physician invent a new technology poised to make a huge impact in the field of dermatology? Collegiality combined with a confluence of interests, collaboration and curiosity led to a partnership with far-reaching benefit.

Dr. Ann Tilton, Professor of Clinical Neurology and Chief of Child Neurology at LSU Health New Orleans School of Medicine, had been pursuing ways to improve function and the quality of life for her patients with movement disorders involving spasticity like cerebral palsy, a pursuit she continues today. One of the substances she was working with is botulinum toxin. Yes, that’s right, Botox®. A study she was conducting found the action of botulinum toxin at the neuromuscular junction improved stretch and reduced the likelihood of contracture in calf muscles. Since it worked there, Dr. Tilton and her colleague Dr. Dana Suskind, then an LSU Health pediatric otolaryngologist, wondered whether it would also be an effective treatment for the excessive drooling likewise associated with cerebral palsy. They combined their expertise – Dr. Suskind’s with the salivary gland and Tilton’s with the medication. Their seminal work was some of the first published on the subject. And the collaboration, driven by a common quest for answers for their patients, blossomed.

“We took what we knew about Botox and started figuring out other applications for it,” Tilton remembers. “And the one we decided to pursue was acne. Botulinum toxin stops inflammation, blocks androgen, a hormone associated with acne, and increases the motility of cells in the pores. It acts like a retinol, but without the side effects. We wrote out our idea, dated and sealed it.”

When they did preliminary research with the LSU Health Department of Dermatology, then LSU Health resident Dr. Mary Caire came on board. The research found a complete resolution of severe, intractable acne. At that point, they knew their results would have to be reproducible, by outside scientists, so Tilton negotiated a deal with Allergan for a pilot trial in Dallas. Unusually, the results remained the property of LSU Health and its three female inventors. They now had proof of concept to apply for a patent, which they were awarded for the use of botulinum toxin to treat or prevent acne. Despite the success of the pilot study, Allergan chose to pursue other applications for its Botox product, and in 2008, the LSU Health licensed its patented technology to Anterios, a clinical stage pharmaceutical company.

Fast forward to 2016. In an ironic twist, Allergan, now the world’s largest pharmaceutical company, acquired Tilton’s technology when it bought Anterios in a $90 million deal.

Tilton is one of LSU Health New Orleans faculty recognized recently for their innovation. Dr. Sunyoung Kim, Associate Professor of Biochemistry and Molecular Biology, is another.

On behalf of their tiny patients, LSU Health New Orleans neonatologists asked Dr. Kim, a basic science researcher, to develop a test to detect necrotizing enterocolitis (NEC) early.

“Despite many advances that have improved care for premature infants, NEC continues to be an unpredictable feared and sometimes fatal disease for these tiny babies,” says Dr. Brian Barkemeyer, LSU Health Professor and Head of Neonatology. "Developing a way to recognize this disease in a more timely manner has the potential to save lives.”

NEC, a gastrointestinal disease, is a one of the most catastrophic conditions associated with premature birth. It is one of the leading causes of illness and death in these fragile infants, and by the time it is diagnosed, many are already in danger. Kim developed a non-invasive protein test to determine whether an at-risk baby has the condition at a point before its course is irreversible.

“We know our test has a really high rate of identifying true positives (93%) as well as true negatives (95%) because of our pilot – the first human clinical trial,” notes Kim. “That’s double what the prior diagnostic can do, which is about 44%.”

“From a research perspective, we understand more about the pathophysiology and inflammatory mediators involved in NEC,” adds Dr. Barkemeyer. “Unfortunately, this knowledge has yet to significantly alter the incidence or severity of NEC. Dr. Kim's work may have more practical benefit than most other investigations.”

Kim’s spin-out company Chosen Diagnostics, started to support the commercialization of this life-saving technology, has won competitions by local economic development groups and support from the National Science Foundation’s Innovation Corps (I-Corps) Program. Her latest distinction is an invitation from the US State Department and the Small Business Administration to represent the United States at the Global Entrepreneurship Summit in India later this month.

Tilton and Kim are two of the LSU Health New Orleans faculty members working to solve some of society’s most difficult health problems who were honored by LSU Health New Orleans leadership.

“The impact of innovation is revolutionizing health care – what we deliver and how,” notes Larry Hollier, MD, Chancellor of LSU Health New Orleans. “New-era biosciences research not only saves lives and improves quality of life; it creates highly desirable, high-paying jobs that improve economic health. LSU Health New Orleans’ robust research enterprise is making major contributions, and we are proud of our faculty driving innovations in health.”

The Celebration of Innovation, sponsored by Baker Donelson, recognized the honorees’ dedication to improving human health, inventiveness in developing innovative approaches and success in translating their discoveries to benefit people in Louisiana and throughout the world. The event celebrated those whose technologies have been licensed to a company or been awarded a competitive Leverage Innovation for Technology Transfer, or LIFT2, grant or a US patent.

LSU Health faculty innovations funded by LIFT2 grants like Kim’s, include Nursing Assistant Professor and Program Director Dr. Stephanie Pierce’s online course for healthcare professionals covering the management and best practices of providing culturally sensitive care to vulnerable populations during disasters; and the science-education videos developed by Graduate Studies’ Dr. Fern Tsien and Public Health’s Martha Cuccia for elementary, middle and high school students; Dr. Rohan Walvekar’s work to develop a solution that dissolves salivary, and perhaps other, stones;` and Dr. Donna Neumann’s work on a class of novel and potent antifungal agents that pre-clinical research showed are highly effective against fungal infections resistant to currently marketed antifungal therapies.

In addition to Tilton’s, licensed technologies included the interactive gross and microscopic images teaching tool developed by Allied Health’s Angela Foley, MS, MT and Daniel Haun, MS, as well as the therapeutic compound with THC and a Cox2 inhibitor for Alzheimer’s and other neurodegenerative diseases developed by Dr. Chu Chen at the Neuroscience Center.

Among the patents recognized was Dr. Xiamoning Xu’s for the development of fluoride-releasing compositions incorporated into dental materials – work done at the School of Dentistry.

All in all, 30 LSU Health New Orleans faculty innovators were honored for their 21 LIFT2 grants representing $674,772 in proof-of-concept funds, seven exclusive license agreements and seven US patents over the past two years. Each honoree received a crystal award with a 3D etched light bulb symbolizing scientific discovery.

A complete list of LSU Health New Orleans faculty innovators and their technologies is available here.

“Celebrating the innovation of our faculty is important both to recognize their achievements and to reinforce that this activity is an invaluable pursuit at our institution,” says Patrick Reed, Director of Technology Management. “The innovators recognized at this event are helping build out the local health and biotech ecosystem, and it's exciting to be a part of that.”