rend sees patients paying for their own clinical trials, but critics raise ethical concerns

As a bio-sciences start-up, Regenastem Inc. is beginning life in typically modest fashion. It has secured lab space at an incubator run by a small Ontario university, its website is rudimentary — and it has tapped into a surprising source to pay for two new clinical trials.The studies of cell-based arthritis and spine treatments — conducted with a Buffalo, N.Y., research company — are to be funded by the patients who take part in them.The firms are part of an intriguing — and controversial — new trend. Unable to access research dollars from governments, health charities or pharmaceutical companies, many developers of new treatments are turning to the people who want to participate in their studies for financial support.So-called “pay-to-participate” clinical trials have helped launch investigations of everything from impotence treatments to multiple sclerosis medication.

The stem-cell therapy hockey legend Gordie Howe underwent in Mexico triggered intense media attention earlier this year. What was largely overlooked is that Howe was part of a trial that charged other stroke patients $32,000 to join.“It’s a model of funding that I think we are going to see being proposed more and more in the coming years,” said Jonathan Kimmelman, a McGill University bio-ethicist.Patients are paying to be part of trials primarily of cutting-edge stem-cell and other cell-based treatments — relatively inexpensive for small players to develop — and therapies for rare diseases that get little attention from industry, he said.But the idea is triggering debate.Critics, including Kimmelman and colleagues in a new journal article, warn that such studies are rife with ethical problems that could harm patients and produce questionable science.

People who pay to be part of a study will naturally be unwilling to take the chance of receiving a placebo, the McGill professor said. And yet having a placebo “control” group and blinding patients and doctors to who is in which group is key to obtaining the most reliable data, said Kimmelman.Some patient-funded studies appear not to even be true research, but more a way to market unproven treatments, he said.“It’s co-opting the kind of scientific aura that we associate with clinical trials,” Kimmelman said. “(But) if your business model is to sell the trial itself, as opposed to the product, you don’t have strong incentives to design rigorous clinical trials.”Proponents respond that patient fees, though unconventional, make possible research that might otherwise never get off the ground.

If your business model is to sell the trial itself, as opposed to the product, you don’t have strong incentives to design rigorous clinical trials

“As funding becomes harder and harder to secure for all the good work that legitimate clinical trials seek to accomplish, society needs to be open to this evolving creative process,” said Dave McGuigan, vice president of San Diego’s Stemedica, the company whose treatment was administered to Howe.Patient-funded trials can also plug a gap that major pharmaceutical companies are unwilling to fill, testing stem cells and other products that are difficult to patent — and therefore unlikely to attract huge sums of money for traditional testing, said Rafael Carrillo, head of Stemedica’s Mexican partner, Novastem.

Even Kimmelman says he is not completely opposed, and in a paper coauthored with two American colleagues, proposes a series of measures to make the idea ethically workable, chiefly by requiring regulatory oversight.Finding money to run trials — the priciest part of medical research — has always been a challenge, and budgets for government funding bodies like the Canadian Institute for Health Research have generally been declining in real dollars.

In that demanding environment, there are now “numerous” trials around the world financed either entirely or partly by patients, said McGuigan.That includes 14 being run by Novastem, like the study of the stem-cell treatment for stroke survivors that Howe’s family claims had a dramatic effect on the NHL luminary. But the company is also helping fund those trials, McGuigan stressed, under a model he calls patient-assisted.

“Patient-assisted clinical trials — where all parties bear a portion of the financial risk — are an ideal way for society to advance clinical trial initiatives … when no other external financial resources are available,” he said.Patient-supported research is often not double-blind and controlled because “you can’t ask a person to pay an important amount of money” and give them a placebo, said Carrillo. But if the results are promising, they could lead to more scientifically rigorous clinical trials, he said.

Meanwhile, U.S. law specifically allows for patient-funded studies in certain circumstances.

A Florida company called Ageless Regenerative Institute has Food and Drug Administration (FDA) approval, for instance, to look at treating erectile dysfunction with stem cells derived from body fat. The trial is “patient-sponsored.”Regenastem, based at Brock University’s Biolinc facility in St. Catharines, says it has developed what it calls its own “unique” way to isolate stem cells from adipose — fat — tissue.It is working with the Buffalo Research Group to set up two trials that involve removing cells and blood from patients’ bodies, processing them and injecting them back to treat arthritis and disc-related pain.

Patients – all of whom are to receive the treatment – will be charged $7,500 each, which only just covers costs, and enables doctors to provide good care while also gathering scientific data, said Jason Kane, the company’s CEO.The process has been endorsed by Brock, the treatment itself is already approved in Australia, he said. Without patient funding, Regenastem “would not be able to finance a study of any size.”McGill’s Kimmelman, however, worries about more than just the scientific value of pay-for-participation studies, arguing the subjects’ own best interests may be at risk.

Experimental treatments are usually studied on as few people as possible until safety is assured. But when the subjects fund the study, there is financial incentive to enroll numerous patients, exposing more people than necessary — and possibly unsuitable ones — to possible harmful side effects, he warned.“This can tarnish a reputation of a new and emerging research area,” he warned. “There is some threat to actually undermining the progress in certain areas.”

---

source:National Post