About a year ago, researchers at the UW Carbone Cancer Center received clearance from the FDA to begin testing a first-in-human cell therapy to treat a common side effect of throat radiation treatment: xerostomia. Or, in plain terms: dry mouth.
For many drug developers, finding a therapeutic solution for radiation-induced dry mouth wasn’t exactly on the top of the priority list. But for the University of Wisconsin’s Program for Advanced Cell Therapy, it was an opportunity to directly address and act on an issue adversely affecting patients.
“We’re really trying to help make a difference in the lives of those who may not have any other options,” said PACT director and associate dean for therapeutics development Jacques Galipeau, MD.
The Program for Advanced Cell Therapy, or PACT, was launched in 2016 to develop personalized cell technologies for improving health outcomes in children and adults with unmet medical needs, such as xerostomia, and testing them through first-in-human clinical trials.
While the clinical trials themselves are fairly unique, it’s what happens in the lead-up to the testing that really stands out. The cellular therapies – created from a patient’s own cells or from a related donor – are entirely manufactured at the University of Wisconsin.
Frequently, new drugs and therapies are either physically developed by or in conjunction with pharmaceutical companies. But thanks to a partnership with PACT, academic researchers and UW Carbone members are able to draw upon in-house resources to create new cell therapies without having to leave campus.
“We’re like a clinical stage pharmaceutical company or biotechnology startup, but in house,” Galipeau said. “What we do here, it’s very unique.”
Since its launch, PACT has worked with UW researchers to launch four unique clinical trials with homegrown therapies, with some having direct implications for cancer patients. For example, PACT’s very first study is examining the safety and effectiveness of a new cell therapy in treating cytomegalovirus (CMV) infection in bone marrow transplant recipients, including some cancer patients.
The study is led by UW Carbone member and PACT medical director Inga Hofmann, MD, who is using virus-specific white blood cells to treat post-transplant CMV infections. It’s estimated that up to 50 percent of bone-marrow transplant recipients may experience this viral infection, which can be fatal, so the need for an effective therapeutic option is great.
A similar clinical trial, also launched through PACT, is testing the same therapy in kidney transplant patients.
“We’re very invested in cancer immunotherapy, but also other disorders that are important to other patients outside of the cancer world,” Galipeau said.
For example, nephrologist Sandesh Parajuli, MD, recently worked with PACT to obtain FDA clearance to test a new therapy aimed at helping kidney transplant patients suffering from post-transplant BK virus infections. Another upcoming project with Randy Kimple, MD, looks to use marrow stem cells to treat xerostomia.
Most recently, Hofmann launched a clinical trial to test a new stem cell therapy aimed at treating – and potentially curing – a number of non-cancerous blood disorders, like sickle cell disease, a debilitating and lifelong condition that lacks a consistently safe and effective treatment method.
With any brand new therapy – especially when cell manipulation is involved – there are frequently regulatory hurdles to clear and complex protocols to follow before a clinical trial can take place. Study investigators and cell manufacturers must apply for and receive investigational new drug (IND) and investigational device exemption (IDE) licenses from the FDA before work can truly begin.
To help with this, PACT recently promoted Kimberly McDowell, MD, PhD, as PACT’s Director of Regulatory Affairs. Galipeau calls her a “concierge” between researchers and the FDA.
“These applications are incredibly complex and each one is different,” Galipeau said.
Pharmaceutical companies usually have a brigade of individuals who do this full time, but with her experience and her deep knowledge of what we do here, Kim is a highly capable ‘fixer’ in tackling this complex work for us.”
And for the PACT Team, the work is just getting started. New cell therapies targeting head and neck cancer could soon be coming, pending approval from the FDA, leading to even more clinical trials.
“Most of the elite cancer centers are involved in cell therapy nowadays, and we’re amongst those elite,” Galipeau said. “But we’re trying to distinguish ourselves by going harder and bolder, and really developing unique technologies that truly move the needle.”
At the end of the day though, being able to help patients – whether they have cancer or another condition – is what drives the team to keep going.