For the past four years, Sammy Jo Wilkinson has battled her secondary progressive multiple sclerosis back to remission.
Utilizing stem cell therapy, Wilkinson no longer suffers from paralysis in her face, or the numbness in her fingers. Her stem cell treatments have been so effective, she even walked for the first time in years last February.
“I’m living in a future that everybody will have some day,” says Wilkinson. “We’re trying to tell everybody the solution is here now; we just need a logical way to bring this to patients sooner rather than later.”
And that logical way may’ve been illuminated in early December with Congress agreeing to the 21st Century Cures Act. The labyrinthine, 1,000-page bill will make critical changes in how regenerative medicine & treatments are tested, and then brought to market.
Aside from shrewd re-gifting and practicing Christmas carols, the U.S. government made getting the Act passed before the New Year a top priority, which was met with overwhelming bipartisan support. The House voted 392 to 96 in favour of the Cures Act; the Senate passed the bill in a 94 to 5 vote; and President Obama has already stated he looks forward to seeing the proposal on his desk.
WHAT IS THE 21ST CENTURY CURES ACT?
The encompassing bill certainly lives up to its name, detailing lofty goals that’ll advance biomedical sciences – like stem cell research – through a $4.8 billion injection into the forever-stagnant National Institute of Health (NIH) budget.
Widely-publicized portions of the bill reveal that the total will be spread out over 10 years. The grant also includes Joe Biden’s $1.8 billion ‘cancer moonshot’ project, which experts believe is also accessibly-titled (re: success is a moonshot). Another $1.8 billion off the total will go towards researching brain diseases like Alzheimer’s.
Having said that, skeptics of the bill see a path outlined from the bill, a path that leads away from the gold standard of medicine, instead moving towards an expedited ‘middle ground’ for all drugs, medical devices, and regenerative medicines.
In other words, we’re looking at deregulation in sheep’s clothing.
THE ‘INJECT-AND-SEE’ ERA
Wilkonson received her first stem cell treatment at Celltex Therapeutics in 2012. Extracting adult stem cells from her fat tissue, the scientists cultured her cells, allowing them to grow in population before being injected back into her body. The effects were instantaneous – less pain, more energy. She had found a worthwhile solution, even if it was a temporary one.
But the U.S. Food & Drug Administration (FDA) ruled Celltex was violating a 2006 ruling change that put the agency in charge of regulating expanded-cell populations as drugs. Celltex needed approval to use the company’s own treatments.
That was just one of many similar tug-of-wars occurring over the past-decade between bitter regulators and weary patients regarding regenerative therapy approval. Some companies, like Celltex, found wading through regulations and policy makers too cumbersome, moving their operations to countries like Mexico.
But like an annoying pest infestation, when the large drug companies left town, waves of small clinics sprung up in their stead. Unlike the FDA hastily dropping the hammer on big corporations like Celltex, they were slow to investigate the validity of these clinics, or the legality of their treatments. Today, there are close to 600 businesses in the U.S. selling stem cell treatments that cure everything from Alzheimer’s to autism – all without FDA approval.
This odd causality is what pushed scientists and policy makers to dub stem cell clinics, ‘medicine’s wild west’.
WHAT THE CURES ACT – SHOULD – SOLVE
By rolling back the regulatory authority of the FDA, as well as their guidelines to approve new drugs, the 21 Century Cures Act was supposed to change the gun-slinging ways of the regenerative medicine community.
But it won’t do it in the way that ‘seems’ logical to us: by reigning in the aforementioned unproven, unregulated treatments offered by small clinics. Instead, the Act will create a clear path to medical acceptance for virtually any treatment.
Under the Cures Act, the FDA would have the ability to grant accelerated approval for regenerative medicines. That means new, untested treatments could go straight from animal models and clinical trials to post-market review – leapfrogging testing in humans, which seems pretty important.
A that could spell trouble for legitimate, traditional stem cell research.
“If you legitimize these therapies and allow businesses to commercialize them, then it becomes difficult to recruit individuals for actual phase 3 clinical trials,” explains Leigh Turner, bioethicist at the University of Minnesota.
Clinical trials require particular participants; specific inclusion/exclusion criteria are necessary to produce reliable results. With the new Act, people going into the marketplace and getting an unapproved treatment would make them ineligible for study, as their ‘inclusion criteria’ becomes compromised.
“They’ll be lost to science,” says Turner.
WAIT AND SEE
Other researchers think that’s just the tip of the iceberg; the consequences can be more dire.
For instance – what happens if people get hurt by these therapies, either physically or financially? With the loss of Phase 3 testing, which usually involves the largest number of human participants to ensure no unusual risks slipped by in smaller studies, those dangers and uncertainties are placed on paying patients.
And because these treatments aren’t covered by the government or private insurers, patients will dip into their own vault for a cure they may desperately need (Wilkson’s paid $90,000 from her own pocket). In addition to losing money over shoddy and untested treatments, the medicines could cause serious side effects – what if the patient developed a tumor, went blind, or even had a stroke and died?
“I think we’re going to hear a lot more about that in the next few years,” says Paul Knoepfler, a stem cell researcher at UC Davis.
It couldn’t come at a worse time, Knoepfler worries, as he expects the real, diligently-tested therapies to go public in the near future. Stem cell technology has led to potentially incredible treatments: helping diabetics grow insulin-generating pancreatic cells, stopping ALS progression, and strengthening cardiac muscles in people with heart failure are all on the long path of true clinical testing.
“There’s real hope,” continued Knoepfler. “My worry is that the dark echo of the [small, unregulated] clinics will negatively impact the perception of the whole stem cell arena.”
But, just because the FDA has the power of accelerated approval, doesn’t mean they’ll exercise it. FDA Commissioner Robert Califf made that evident in a recent interview, cautioning ‘to ensure that this emerging field fulfills its potential promise to patients, we must first understand its risk and benefits and develop therapeutic approaches based on sound science.’
Considering the timing of the interview and the Cures Act being at the apex of its popularity, it’s safe to assume the FDA will take a wait-and-see approach.
Patients and researchers alike can probably agree that it beats the inject-and-see method, anyway.
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