With the 21st Century Cures Act, the future of regenerative medicine is to “inject and see’

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If Sammy Jo Wilkinson had a spirit animal, it would be Marty McFly. In the past four years, the 51-year-old native of California, is using stem cell therapy to beat her secondary-progressive multiple sclerosis back in remission. Gone is the paralysis on the left side of her face and the numbness in her fingers. In February she walked for the first time in years. “I live in a future in which everyone will one day,” says Wilkinson, who co-founded the patient rights of the group Patients for Stem cells. “We try to tell everyone, the solution is here now, we just need a logical way to these patients sooner rather than later.”

According to the Congress, that the only logical way in the 21st Century Cures Act, a labyrinthine piece of legislation that would make the most significant changes in the coming decades, and how medical treatments are tested and brought to market. Politicians are working overtime to give for the new year— it is the number one priority for the lame duck session, passing the House on Nov. 30 and advancing through the Senate last night. Final passage is expected to follow later this week.

In some ways it is the legislation lives up to its name: It includes ambitious objectives to advance biomedical science, and will inject $4.8 billion into a long stagnation of the National Institute of Health budget. But attached to those promises is a roadmap for the exit from the gold standard of medicine in the advantage of an accelerated “ground” for medicines, medical devices and regenerative therapies. Critics say that the deregulation in sheep’s clothing, and ensure that both the science and the patients are going to suffer.

Whether you applaud or decry the legislation, it is almost certain to pass and be signed into law, if not by President Obama, which are then used by the incoming administration. What does regenerative medicine is on the way to prime time. Welcome to the era of ” inject and see.”

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Medicine from the Wild West

In 2012, Wilkinson flew to Houston for her first stem cell treatment at Celltex Therapeutics. Engineers have extracted adult stem cells from her fat tissue, then cultured them for 13 days, allowing the population to expand for the injection of the cells back to Wilkinson. The effects, she says, were immediate. They had less pain, more energy. But a few months later, the Food and Drug Administration (FDA) has ruled that Celltex was violating a rule 2006 change, allowing the FDA to regulate expanded cell populations as drugs. They would have to get agency approval before being used in treatments.

These actions became the most visible confrontation in a bitter decade-long battle between regulators and patients regarding the legality of regenerative therapies. While some companies, such as Celltex, their residence in Mexico skirt regulations, other small clinics with lower profiles in a hurry to fill the void. The FDA has been slow to examine whether the distribution of these clinics and the treatments that they market. Today there are almost 600 companies in America, the sale of stem cell solutions for everything from deafness to Alzheimer’s disease, and autism, all without the approval of the FDA, according to a study published in February by Paul Knoepfler and Leigh Turner.

Turner, who is a bioethicist at the University of Minnesota, was surprised by the size of the exploitative behavior he found. “Anyone can buy a domain name and create a website and make great therapeutic claims that have no basis in reality whatsoever,” he says. The situation has led scientists and policy wonks dub stem cell clinics “medicine of the wild west.” The 21st Century Cures Act will change—not by reining in unproven, unregulated treatments, but by providing a direct path to medical acceptance. On the basis of the law, the FDA would have the authority to grant an accelerated approval for regenerative medicines, skip the right of animal models and safety studies on the efficacy testing in humans, after the evaluation of the market. The new law would also force the FDA to update its regulations for such products.

That, Turner says, would be a disaster for the traditional stem cell research. “If you identify these therapies, and companies can commercialize, then it is difficult to recruit persons for the actual phase 3 clinical studies,” he says. Clinical studies have guidelines about who does and does not participate— these inclusion/exclusion criteria helps to produce reliable results. People who go on the market and get a non-approved therapy is not able to participate because their inclusion criteria is affected. “They are lost for the science,” says Turner.

The Dark Echo

Knoepfler, a stem cell researcher at UC Davis, is worried about a more disturbing outcome: What happens if people get hurt by these therapies, both physically and financially? Phase 3 studies typically include a much larger number of participants to enable the statistical assessment of clinical benefit and the detection of any unusual risks that are not discovered in smaller studies. To avoid this step places those risks and uncertainties with both legs on the shoulders of the paying patients. Because the treatments are not covered by public or private insurers, patients may have a fortune in their quest for a remedy (Wilkinson has spent $90,000 out-of-pocket). Or worse, the development of a tumor, is blind, or suffers a stroke and dies.

“I think we’re going to hear a lot more about that in the coming years,” says Knoepfler. That is unfortunate timing, because that is when he expects the real, rigorously tested therapies that are online. Stem cell treatments that help diabetics in their new insulin-making pancreatic cells, stop the progression of AS, and the strengthening of the heart muscle cells in people who suffer from heart failure are just some of the therapies advancing steadily down the full clinical trial path. “There really is hope,” he says. “And my concern is that the dark echo of the clinics will negatively impact on the perception of the whole stem cell arena.”

But people like Wilkinson does not have that kind of time. “Facebook is more like an in memoriam page for a few days, and I’m just tired of watching my friends die,” she says. Her organization was instrumental in obtaining a record number of comments during the FDA hearings on the draft guidances for stem cell treatments in September. She says that she is not paid by Celltex or someone else to be a voice for the technology, and really only wants the FDA to stop telling her what she can and can’t do with her own cells.

Not so fast

While the patients and the regenerative medicine investors celebrate and researchers raise alarms, top FDA officials are holding their ground. In an article published In the New England Journal of Medicine last Wednesday, FDA Commissioner Robert Califf expressed skepticism about the safety and efficacy of stem cell treatments. “The current excitement about the possibilities for stem cell therapy to improve the outcome for the patient and even cure the disease is understandable,” he wrote. “However, to ensure that this new area meets the potential promise to the patient, we must first understand the risks and benefits and the development of therapeutic approaches based on sound science.”

The timing of the article suggests that the FDA will not have the authority to accelerate approvals any time soon. Not, or maybe not. The 21st Century Cures Act is not to know exactly how it will provide the necessary resources for the implementation of that requires of the agency. Currently, strict conflict of interest rules and a stringent application process impedes the agency’s ability to attract top talent. “The FDA has the hiring of a shortage problem for more than a decade,” says David Gortler, a former FDA senior medical officer. He says there are small ways to the speed of the review process here and there, but nothing on the scale people imagine. “Mark my words. Nothing will happen,” he says.

If so, the agency will live up to his reputation as a cracking thorn in the side of progress. But according to Turner, the FDA is just a convenient scapegoat. The thorn is just the reality of the man. “We try to develop truly safe and effective cell-based therapies for what is in the human history, are intractable diseases,” he says. “The human body is the challenge, not bureaucratic inertia.”

And not even a plutonium charged Delorean can change that.

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