Last
week I wrote about a pending lawsuit by the FDA against Regenerative Sciences,
a medical company that developed a stem cell treatment for orthopedic
injuries.
While adult stem cell therapies have been
fraught with concerns about safety and efficacy, this case is particularly
important not only because it is unprecedented — the FDA is attempting to
classify non-embryonic stem cells as drugs – but also because it brings into
focus the disconnect between the pace of scientific progress and the struggle
of regulators to keep up with rapidly evolving technologies.
I spoke with three people with a unique
perspective on what’s at stake – a lawyer who specializes in FDA regulation, a
lab director of an in-vitro fertilization clinic, and a bioethicist – to get a
sense of how the case is viewed in their industries. One common thread that
emerged from these conversations is that the FDA is conflicted, even confused,
about how to regulate stem cell-based products. The prolonged legal battle with
Regenerative (it dates back to at least 2008) is due largely to the fact that
the agency recognizes the absurdity of coming down with a one-size-fits-all
regulatory approach that would apply to every clinic offering such treatments.
To be sure, stem cell medicine has a terrible
reputation. Stem cell therapies that purport to treat everything from shot
knees and slipped disks to autism, multiple sclerosis, and Parkinson’s disease
are rightly regarded by many as modern-day quackery. That is because
unscrupulous doctors have seized on the promise of this exciting new science
and used it to peddle unproven treatments to desperate and ill-informed
patients.
In 2010, CBS aired a 60 Minutes special titled
21st Century Snake Oil, in which Doug Sipp, a researcher at the RIKEN Center
for Developmental Biology in Japan and a critic of stem cell fraud, who also
runs the watchdog blog Stem Cell Treatment Monitor, warned of the dangers of
blossoming stem cell medical tourism. That same year, NPR aired a story called
“Offshore Stem Cell Clinics Sell Hope, Not Science.” And in 2011, the journal
Nature reported on Texas governor Rick Perry’s crusade to soften the FDA’s
position on stem cell clinics, whose number in the U.S. is estimated to be
around a dozen. The International Society for Stem Cell Research (ISSCR), a
non-profit organization of medical doctors and scholars, also functions as a
myth buster for clinically untested treatments. On its website, the group
writes that the mesenchymal stem cell, which is at the heart of the FDA lawsuit
and is used by Regenerative in its procedures, has “become a cell of intense
interest” but “has not yet been shown to have a clear-cut advantage over
existing therapies, is not considered a standard of care for any condition and
does not have regulatory approval for the routine treatment of any disease.”
Against this background, the FDA is grappling
with a difficult decision – how to curb the proliferation of unproven stem cell
treatments hawked by unconscientious doctors, while recognizing that this
branch of medicine holds a legitimate promise whose full potential is only
beginning to be discovered.
Areta Kupchyk, a partner at the law firm Nixon
Peabody who counsels clients on FDA regulation and development of new
biotechnologies, including human tissue and stem cells, says cell-based products
are tough to regulate because they are individualized and manipulated to
different degrees for uses in individual patients.
“The FDA has been trying to figure out how to
regulate stem cell product development because it is so difficult — individual
hospitals, individual doctors are performing individual procedures. It has had
a very difficult time over the years trying to regulate something that’s not a
big manufacturing facility. It’s very hard to get your arms around something
like that as a regulatory agency,” Kupchyk told me in a telephone interview.
She noted that where minimal manipulation is
concerned, the FDA may face a special challenge. One of the arguments in the
agency’s case for regulating Regenexx, a procedure in which adult stem cells
are extracted, cultured in a special serum, and reinjected back into the same
patient, is that the cells are more than minimally manipulated.
“I can understand why the FDA is focusing on
minimal manipulation — that is something they have been focusing on for a
number of years. But they haven’t been able to articulate what minimal
manipulation means. They’re regulating on a case-by-case basis and they’re not
informing the public well on where they draw the line,” Kupchyk said. ”One good
reason they’re not coming down is that this area is rapidly evolving and there
are products created out of cells that have never been used or created before.
So they’re struggling with new products, they are not sure how they are going
to work in the body. This case could be very important in forcing the FDA to
define it more clearly.”
The number of scientists and clinics
developing or providing adult stem cell treatments is another reason the FDA
may be at a disadvantage trying to establish hard-and-fast new rules, Kupchyk
added. “If the FDA is successful in this case, it’s going to raise a lot of
questions for everyone out there who’s trying to develop new uses for stem
cells for patients. There may be a chilling effect, certainly, and it’s very
expensive to test new products [through the formal regulatory framework]. If
every single physician or clinic who was doing something similar applied, the
FDA would not be able to handle it. That’s another reason they’re not coming
down on every single case.”
I was curious to hear the perspective of
Marlane Angle, a lab director at a San Francisco in-vitro fertilization clinic
and a former technical expert for Irvine Scientific, a manufacturing company
that has produced the first FDA-cleared cell culture for use in such clinics. Angle,
who is a member of the American Society of Reproductive Medicine and one of the
first IVF lab inspectors credentialed by the College of American Pathologists,
testified last year on behalf of Regenerative that the safety protocols in
place for its procedures exceed those at many IVF clinics she has seen.
“If you interview the average person, I think
you would find that there should be some oversight. If you start to look at
this product as being the patient’s own stem cell, how can the FDA claim Regenerative
is manufacturing [cells] – they’re culturing them,” Angle told me in response
to a question about the agency’s rationale to sue the company on the claim that
its manipulation of stem cells is tantamount to manufacturing a new drug.
“They seem to have lost perspective on using
autologous stem cells. There’s just no way you could apply manufacturing
standards. I would love to have been a fly on the wall [during FDA's
deliberations],” Angle said. “It’s like they are trying to come up with a law
that takes into account every possible point of abuse that could happen.”
Unlike fertility clinics in the U.K. and other
European countries, IVF clinics in the U.S. are not nationally regulated. That
means the government has no say over how embryos are cultured or even how many
embryos can be transferred at the same time to a single female. (Remember
Octomom? She was an outlier and certainly a “misapplication of the technology,”
Angle said).
Angle added that risky procedures such as
heart transplants have a much higher potential health impact on a patient’s
life than either IVF or stem cell treatments. “The FDA does not come into a
cardiology practice and tell doctors how to do their surgeries or how to do
heart replacements. And yet they feel they can come into a stem cell clinic.”
The FDA’s decision to regulate Regenexx on the
grounds that the procedure does not satisfy the standards of Good Manufacturing
Practices, rather than Good Tissue Practices, which are observed in blood and
human tissue transplantation, is also puzzling to Kupchyk, a former FDA
employee in the agency’s division for drugs and biologics.
“I’m curious how they came to the conclusion
that the clinic is manufactuing. With stem cell therapy, it has to be based on
an individual patient, for each person [the physicians] are deciding whether
and how to use their stem cells,” Kupchyk said.
I also spoke with Christopher Scott, a scholar
at the Stanford University Center for Biomedical Ethics. He walked me through
the autologous stem cell controversy with a succinct but comprehensive
analysis.
“In principle, I agree with the concern going
into this slinging too wildly. This case has two edges: in this country and
others, we have an epidemic of transplant clinics offering so-called cures and
therapies to patients that have not been proven and have not passed muster with
federal agencies. So that’s one pressure coming into this debate – the reverse
is that we don’t want to overregulate therapies to the extent that we would
cause the field to stumble,” Scott told me via telephone.
He cited as an example the way gene therapy
was stalled by the FDA in its early stages. “There were concerns that the
science was moving too quickly, so the FDA stopped all trials and the field was
effectively shut down. There was such a severe reaction on the part of regulatory
agencies that the science was effectively moribund, and now it is slowly coming
back to viability.”
A second example worth considering, Scott
noted, is bone marrow transplantation, a medical procedure that is also largely
unregulated. Since most autologous stem cell treatments are nearly identical or
variations of bone marrow transplantation, he said, the challenge for
regulators and translational scientists is how to use what they understand from
the procedure’s history and literature to help create a regulatory structure
that would be efficient.
Stem cell medicine will learn to settle the
question with time, Scott believes. “However, that doesn’t resolve the issue
now about companies and clinics who want to do this to a desperately ill
patient on their doorstep. There are cases when physicians are justified
morally looking at a single patient and treating them with the best available,
most promising treatment.”
“The question for us as a society is when
something done at the edge of clinical care becomes something we want to
study,” Scott observed. “My feeling is that in many cases clinics are
scrambling to get approval, but don’t have the gravitas that the clinical
literature provides that would give comfort to a regulatory agency or even an
advisory board to say ‘go ahead and do it.’ So until we have a better idea of
what the adverse effects would be, these things are moving too quickly.”
“FDA understands this is very edgy science and
there’s a lot of hope into it.”
Gergana Koleva
Forbes
Business & Investment Opportunities
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