One of the hottest areas in cancer research right now is the use of CAR-T treatments. These use the patient’s own re-engineered immune system cells to target and kill the tumor. But the thing that makes it so appealing – using the patient’s own cells – also makes it really complicated and expensive. Creating a custom-made therapy from each patient’s own cells takes time and costs a lot of money. But now a new approach could change that.
Fate Therapeutics has developed an off-the-shelf therapy (thanks to CIRM funding) that could, theoretically, be stored at hospitals and clinics around the country and used whenever it’s needed for anyone who needs it.
Equally impressive is that 42 days after being treated with FT516, the man showed no signs of leukemia in either his bone marrow or blood.
FT516 is designed to provide a one-two combination attack on cancer. It’s made up of the wonderfully named natural killer (NK) cells, which are a critical part of our immune system defenses against cancer. These NK cells are created by using the iPSC process and have been genetically modified to express a protein that boosts their cancer-killing abilities.
Because these cells are manufactured they can, if effective, be produced in large numbers and stored for whenever needed. That would not only dramatically reduce costs but also make them more widely available when they are needed.
This is only one patient and the follow-up is still relatively short. Even so, the results are encouraging and certainly give hope that Fate is on to something big. We’ll be keeping track and let you know how things progress.
What do you do when the supposed solution to a problem actually turns out to be a part of the problem? That’s the situation facing people who want to direct patients to scientifically sound clinical trials. Turns out the site many were going to may be directing patients to therapies that are not only not scientifically sound, they may not even be safe.
The site in question
is the www.clinicaltrials.gov
website. That’s a list of all the clinical trials registered with the National
Institutes of Health. In theory that should be a rock-solid list of trials that
have been given the go-ahead by the Food and Drug Administration (FDA) to be tested
in people. Unfortunately, the reality is very different. Many of the trials
listed there have gone through the rigorous testing and approval process to
earn the right to be tested in people. But some haven’t. And figuring out which
is which is not easy.
The issue was highlighted by a terrific article on STAT News this week. The article’s title succinctly sums up the piece: “Stem cell clinics co-opt clinical-trials registry to market unproven therapies, critics say.”
The story highlights how clinics that are offering unproven and
unapproved stem cell therapies can register their “clinical trial” on the site,
even if they haven’t received FDA approval to carry out a clinical trial.
Leigh Turner, a bioethicist at the University of Minnesota and a long-time foe of these clinics, said:
“You can concoct this bogus appearance
of science, call it a clinical study, recruit people to pay to participate in
your study, and not only that: You can actually register on clinicaltrials.gov
and have the federal government help you promote what you’re doing. That struck
me as both dangerous and brilliant.”
At CIRM this is a problem we face almost every day. People call or email us asking for help finding a stem cell therapy for everything from cancer and autism to diabetes. If we are funding something or if there is one underway at one of our Alpha Stem Cell Clinics we can direct them to that particular trial. If not, the easiest thing would be to direct them to the clinicaltrials.gov site. But when you are not sure that all the programs listed are legitimate clinical trials, that’s not something we always feel comfortable doing.
As the STAT piece points out, some of the “trials” listed on the site
are even being run by companies that the FDA is trying to shut down because of
serious concerns about the “therapies” they are offering. One was for a Florida
clinic that had blinded four people. Despite that, the clinic’s projects remain
on the site where other patients can find them.
Being listed on clinicaltrials.gov gives clinics offering unproven therapies
an air or legitimacy. So how can you spot a good trial from a bad one? It’s not
One red flag is if the trial is asking you to pay for the treatment.
That’s considered unethical because it’s asking you to pay to be part of an
experiment. Only a very few legitimate clinical trials ask patients to pay, and
even then, only with permission from the FDA.
Another warning sign is anything that has a laundry list of things it
can treat, everything from arthritis to Alzheimer’s. Well-designed clinical
trials tend to be targeted at one condition not multiple ones.
We have put together some useful tools for patients considering taking
part in a clinical trial. Here is a link
to a video and infographic that tell people the questions they need to ask,
and things they need to consider, before signing up for any clinical trial.
So why does the NIH continue to allow these clinics to “advertise”
their programs on its website? One reason is that the NIH simply doesn’t have
the bandwidth to check every listing to make sure they are legit. They have
tried to make things better by including a warning, stating:
“Listing a study
does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for
details. Before participating in a study, talk to your health care provider and
learn about the risks
and potential benefits.”
The bottom line is
that if you are in the market for a stem cell therapy you should approach it
the way you would any potentially life-changing decision: caveat emptor, buyer