As laboratory breakthroughs using CRISPR gene editing technology advance from bench to bedside – including a lung cancer clinical trial from the Gene Editing Institute at ChristianaCare’s Helen F. Graham Cancer Center & Research Institute – federal policies and guidelines to ensure safe, effective therapies have lagged behind.
But last month they took a major leap forward when the Food and Drug Administration (FDA) issued six new guidances on gene therapy manufacturing and clinical development of products.
“The FDA guidances are really great news,” said Eric Kmiec, Ph.D., director of the Gene Editing Institute. “Putting these guidances in place gives us a bit of a roadmap to follow as we prepare for submission protocols to the FDA.”
Dr. Kmiec was among the experts in a panel discussion titled “Bringing Gene Editing Technology from Laboratory to Bedside” Feb. 6, hosted by ChristianaCare and Delaware BioScience Association. The event featured an insider look at the challenges and opportunities of translating what scientists are doing in their labs to create radical treatment products for diseases.
Speaking to an audience of roughly 200 people – including industry experts, policy makers, clinicians and students from local colleges – Dr. Kmiec and his colleagues explored the scientific, legal and regulatory hurdles of moving therapies from bench to bedside, and, once ready for market, the needs for democratic access to clinical trials and treatments.
“We must make these breakthrough technologies available to everyone,” said Dr. Kmiec. “At ChristianaCare, we put our patients first and are committed to making sure patients from all socio-economic backgrounds have access to the latest and best therapies. It’s part of our values of love and excellence.”
ChristianaCare is developing a clinical protocol for FDA review of a clinical trial using the CRISPR-based genome editing platform to treat squamous cell lung carcinoma.
Dr. Kmiec outlined in his presentation how his team has identified a DNA sequence alteration that occurs in certain kinds of tumor cells that enabled his research team to design a CRISPR/Cas gene editing system that will disable only the NRF2 gene in tumor cells but not in normal, non-tumor cells.
“This discovery perked the interest of regulators because it addresses the age-old question of the selectivity of a therapeutic to distinguish between tumor cells and normal cells,” he said.
Speakers spotlighted some of the challenges the gene editing industry has faced in moving gene editing therapies from bench to bedside.
“It has not been a breeze to get the products that are on the market approved,” said Maritza McIntyre, Ph.D., owner and operator of Advanced Therapies Partners. “As some of the products make it over the goal posts, scientists are coming up with new things – more complex or completely novel – that the FDA has to contend with. And sometimes they don’t have a framework to know which questions to ask to understand how these products work and can be administered safely.”
The FDA guidances, McIntyre agreed, are a giant step in the right direction.
Echoing her was Cartier Esham, executive vice president for emerging companies at the Biotechnology Innovation Organization. “We’re building the plane – as we’re flying it,” she said.
But imagine building a plane with no global standards to follow. This is one of the challenges of moving lab science to clinical trials to the more broadly available products, explained Samantha Maragh, Ph.D., from the Department of Commerce’s National Institute of Standards and Technology, a measurement agency that helps industry and science measure the veracity of data while developing scientific products.
Maragh heads the organization’s Genome Editing Consortium, which convenes experts to help address the measurements and standards needed to increase confidence and lower the risk of using gene editing technologies in research and commercial products. This includes developing measurements and controls, as well as standardizing data collection and reporting and terminology.
“We need universal agreement so when we say ‘gene editing,’ we can point to a definition, and we’re all saying it the same way and have a universal understanding of what we’re talking about,” she said.
Robert Oakes, principal in the Delaware office of Fish & Richardson, addressed legal roadblocks, specifically with CRISPR. “Going from bench to bedside requires a license and a license requires knowing who owns the technology, and we’re in the midst of figuring that out now.”
During a panel discussion moderated by Maiken Scott, host of WHYY’s weekly health and science show The Pulse, the gathered experts agreed one way to advance gene therapies is through awareness building among public policy leaders and the broader public about what gene editing is and the promise it holds.
“We need to raise the level of scientific understanding in the country,” McIntyre said.
“It’s great that we have the science but if we don’t have the public policy, we may not get the products in the hands of patients,” said Helen Stimson, president and CEO of Delaware BioScience Association.
Dr. Kmiec said education is key. He pointed to the Gene Editing Institute efforts to raise awareness among high school and college students through the first-ever gene editing kit and curriculum it developed with Delaware Technical Community College with grant funding from the National Science Foundation.
“Our educational program is designed to educate students who will be the ones making the decisions about gene editing in the future,” he said.