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Vanderbilt Entrepreneur aims to provide a renewed sense of life to Parkinson’s disease patients

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Brian Laden
Brian Laden, Photo Credit: Appello Pharmaceuticals

Brian Laden's journey to becoming Appello Pharmaceutical’s CEO began with scientific passion and pivotal career experiences that ultimately brought him to Vanderbilt.

After earning degrees from Penn State and University of Michigan, and a Ph.D. in Toxicology from the University of Kentucky, Laden arrived at Vanderbilt in 2000 as a postdoctoral researcher in Biochemistry Professor Mike Waterman's laboratory. 

"While I enjoyed Dr. Waterman’s leadership, I found the lab environment isolating," Laden recalls. Laden eventually transitioned to the Center for Technology Transfer and Commercialization before the field was as established as it is today. "I was lucky to witness the early days of Vanderbilt's venture investment activity," he explains. "I learned a tremendous amount about startup formation and financing from mentors like Christopher Rand and Dr. Harry Jacobson."  

Later, Laden, Rand and Jacobson partnered to launch a separate venture fund, Tristar Health Partners, positioning him to recognize the potential in a promising Parkinson's disease treatment being developed at Vanderbilt. 

From the WCNDD Lab to the Market

At CTTC, Laden worked closely with Dr. Jeff Conn, who had just founded what is now the Warren Center for Neuroscience Drug Discovery. The WCNDD team was developing compounds targeting the mGlu4 receptor—the foundation for Appello's technology.  A large global pharmaceutical company initially licensed the program but later returned it. Laden, then at TriStar Health Partners, saw an opportunity. Laden states, “I didn’t initially set out to solve any particular problem; in this case, the problem more or less found me.”

"Jeff contacted me about promising compounds that were better suited for a startup environment," he states. Veteran pharmaceutical entrepreneur Joe Cook, Jr. recognized the potential and helped secure backing from Deerfield Management, quickly bringing Appello Pharmaceuticals to life.

Scientific Hurdles and Breakthroughs

Early challenges with drug solubility and COVID-19 complicated clinical trials, particularly in recruiting healthy volunteers aged 65 and older for a 14-day stay that involved tests like spinal taps. "Recruiting was very difficult, expensive, and slow,” Laden states. Despite these obstacles, the team secured FDA clearance for a Phase I clinical trial, enabling critical funding and validation for future Parkinson’s patient testing. 

Additionally, the existing biotech funding environment posed additional challenges, as fundraising became tougher as they sought financing for their Phase II trial. Laden emphasizes the importance of engaging investors early to address their data needs.  Laden and his team persevered through the fundraising challenges and has newly secured investment financing to fund the Phase II efficacy studies.  

Appello’s evolution from a Vanderbilt lab concept to therapeutic candidate highlights the university's dedication to translating research into impactful treatments. Throughout this process, CTTC served as a vital partner. Laden states, “CTTC has understood the unique challenges facing early-stage biotech companies and shown remarkable flexibility.”

Looking Ahead

Appello is nearing a critical milestone: dosing its first Parkinson's patient in a Phase II trial. "If clinical results match our preclinical success, this drug could greatly advance treatment for Parkinson's," says Laden, noting its potential to manage late-stage symptoms and slow disease progression. Despite sometimes facing more skeptics than supporters, Laden remains confident. "I'm honored by the trust from Drs. Conn, Lindsley, and the CTTC team," he adds, crediting the collaborative effort of the team and supportive investors for their progress in providing hope to Parkinson's patients.