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Bloomberg – Anxious Lawyers Hindering Cutting-Edge Drugs, Scientists Say

Bloomberg – Anxious Lawyers Hindering Cutting-Edge Drugs, Scientists Say

A risk-averse culture at universities and cancer centers holds back a field of potentially life-saving cell transplantation therapies, scientists said.


“Patients are waiting. These therapies are curative. They’re important for the field,” Ellen V. Sigal, chairperson and founder of Friends of Cancer Research, said during a May 17 meeting on the future of cell therapies.


Universities and other research institutions require scientists to meet onerous commercial-grade drug manufacturing requirements in the first phase of human trials. Researchers say that’s unnecessary because they’re not developing commercial products. Upcoming Food and Drug Administration guidance could ease those concerns.


Cell-based therapies—treatments in which stem cells are modified into the specific cell type required to repair damaged or destroyed cells or tissues—have the potential to treat diseases ranging from autoimmune diseases to cancer.


Richard Klausner, a former National Cancer Institute director and the current chief executive of Lyell Immunopharma, called cell therapies the third great modality of science-based medicine, following small molecules that created the pharmaceutical industry and recombinant DNA discoveries that spurred the biotechnology industry.


Novartis broke open the cutting-edge CAR T-cell market in 2017 with Kymriah, which treats a type of leukemia by training a patient’s T-cells to fight off cancer cells. But Sigal said challenges lie ahead in the manufacturing, accessibility, and affordability that could hold back these therapies.

New Tests Needed

Realizing the potential will require breaking out of the traditional ways of testing experimental therapies, scientists said.


“Our efficacy models are so-so, and our safety models are downright poor,” Crystal L. Mackall, a pediatric oncologist at Stanford University, said about pre-clinical testing. “We spend $1 million dollars and 18 months to rule out a thing that is not going to be the major risk for patients.”


The FDA wants to be flexible in regulating early-stage human testing of cell therapies, Peter Marks, director of the agency’s Center for Biologics Evaluation and Research, said.


“The individual institutions are so risk-averse they’re not allowing the flexibility the FDA already provides,” said Steven A. Rosenberg, whose research led to the first effective immunotherapies for selected patients with advanced cancer.


Rosenberg, who’s the chief of the surgery branch at the NCI, part of the National Institutes of Health, said he’s heard those concerns as he has traveled the country.


“It’s not unlikely we’ll see some draft guidance around CAR T-cell therapies or perhaps T-cell therapies,” Marks told Bloomberg Law.


The biggest challenge for universities and cancer centers is making “their legal departments comfortable with the idea” that commercial-grade manufacturing standards aren’t necessary, Anne Keane, vice president of regulatory affairs for Lyell Immunopharma, said.

White Paper Addresses Concerns

Friends of Cancer Research and the Parker Institute, which organized the meeting, called for easing the manufacturing requirements in a white paper addressing roadblocks for cell-based therapies.


Marks said he’s open to a number of proposals listed in white paper, including one allowing a sponsor to test different constructs and improvements of a treatment without submitting an investigational new drug application for each construct, which the agency currently requires.


“We’re going to take back a lot of from what we heard here and think about whether there are things that we can move forward,” Marks said.


“Ultimately, we want to see important treatments that are safe and effective get to people,” he said. “At FDA, we have to be careful that when we do that, we don’t ever go too far that people get hurt in the process. So I think we’ll balance things.”…