FDA blocks trials that send US cells to 'hostile' countries

The FDA has launched a review of clinical trials that plan to send American citizens' living cells to 'hostile' countries – including China – for genetic modification.

The just-announced initiative is focusing on cell therapies that are harvested from patients, modified to turn them into therapeutics such as CAR-Ts, and then re-infused into patients.

The rationale for the move is that the international transfer and genetic engineering may have been carried out without patient knowledge or consent and "may have exposed Americans' sensitive genetic data to misuse by foreign governments, including adversaries," said the FDA.

China has emerged as a force in cell therapy development in recent years, by some estimates having the second largest clinical pipeline in this area after the US, thanks to recent regulatory initiatives that have made it easier to carry out studies.

The crackdown ties in with other anti-China measures that have been proposed in the US, notably the BIOSECURE Act, which has not yet been enacted into law despite clearing a House of Representatives vote last year, but would prohibit federal agencies and businesses in the US from contracting with various Chinese firms on national security grounds.

It is also in line with two executive orders signed by President Donald Trump that seek to prevent the bulk transfer of sensitive personal data to countries of concern and so-called 'gain-of-function' research that can change the properties of biological agents and pathogens.

The FDA claimed the Biden administration has allowed the cross-border transfer of this type of cellular material by allowing an exemption in recently updated data security legislation for trial participants' biological samples – including DNA – that are processed overseas as part of FDA-regulated clinical trials.

The agency said it is actively reviewing all relevant trials that relied on this exemption and will require companies to demonstrate "full transparency, ethical consent, and domestic handling of sensitive biological materials."

Meanwhile, new trials that cannot meet these standards will not be allowed to proceed, and the regulator said it was working with the NIH to ensure that no federally funded research is "compromised" by these practices.

"The previous administration turned a blind eye and allowed American DNA to be sent abroad — often without the knowledge or understanding of trial participants," said FDA Commissioner Marty Makary in a statement.

"The integrity of our biomedical research enterprise is paramount," he added. "We are taking action to protect patients, restore public trust, and safeguard US biomedical leadership."

Shake-up in FDA's cell and gene therapy leadership

The new policy was announced as it emerged that two senior figures at the FDA's Office of Therapeutic Products (OTP), director Nicole Verdun and deputy director Rachael Anatol, have been placed on administrative leave.

The two have been credited with modernising the FDA's regulatory approach to cell and gene therapy (CGT) over the last couple of years. The news has been criticised by the Alliance for Regenerative Medicine (ARM), which pointed out that there are five regulatory decisions pending on CGT products this year, plus a pipeline of nearly 2,000 CGT clinical trials ongoing globally.

Given that level of activity, "it is critical that CBER and OTP operate at the highest level of productivity, efficiency, and scientific rigour," said the organisation.

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