California’s $70 Million ‘Alpha’ Stem Cell Clinic Plan Headed for Approval Next Week

By Dr. Matthew Watson

Alpha clinic organizational diagram
Graphic by CIRM

California's stem cell agency next week
is likely to approve a $70 million plan to build a taxpayer-financed
chain of “Alpha” stem cell clinics in what could be a major step
towards making California the stem cell capital of the world,

The proposal would create five centers
at existing institutions or businesses to be funded at up to $11
million each over five years. Also proposed is a coordination and
information center that would receive $15 million over five years.

A story in Nature Medicine said that
the Alpha clinics would be the first-ever “clinical trials network focused around a broad therapeutic platform.”

The clinics are expected to draw stem
cell projects from the around the world as well as those
funded by the $3 billion California stem cell agency. The proposal
would be one of the largest single research efforts funded by the
agency, formally known as the California Institute for Regenerative
Medicine (CIRM) and use about 10 percent of its remaining cash.

The Alpha clinics are aimed at creation
of a sturdy foundation for the stem cell industry in California,
capitalizing on the burgeoning, international lure of stem cell
treatments. Indeed, one of the objectives of the information center
is to divert people from dubious treatments elsewhere.

The plan would fill a “profound gap”
in quality information about stem cell treatments, according to a CIRM document, which said,

“By providing this resource, the public and potential patients
would be better educated and informed, whether or not they should opt
to enroll in clinical trials or approved treatments at any of the
Alpha clinics.”

The Alpha concept was first broached two years ago publicly by
CIRM President Alan Trounson, a pioneer in IVF research and the IVF
business. His proposal has received early and heavy
attention on the CIRM website with a video, blog items and a white paper.

The plan has received little critical
attention although a researcher from an institution that could be a
candidate for an Alpha clinic commented harshly in May on the California
Stem Cell Report, calling the proposal “an irresponsible waste”and a “boondoggle for some medical schools.” The researcher, who
asked that he/she not be identified, said,

“CIRM will pay for an unneeded
infrastructure that will be empty space and staff sitting on their
hands 99 percent of the time. Or worse yet, CIRM will pay but
the space will be used for other things, other clinic procedures paid
for by insurance.”

Elie Dolgin's July 8 piece in Nature
Medicine quoted Mahendra Rao, director of the Center for Regenerative
Medicine at the US National Institutes of Health (NIH), as applauding the concept. However, Rao said he doesn't
anticipate the approach being tried nationally soon.

Dolgin wrote,

“Rao regularly asks researchers
hoping to advance promising stem cell therapies whether they require
additional clinical infrastructure. 'So far, what they've told us is
they'll let us know if they need anything more than (the) programs
that we have already established,' he says.”

The question of the size of the demand
for Alpha clinics is not addressed in the CIRM concept plan. Also
absent is much discussion of the business aspects of the proposal. It
does mention “corporate sponsors” in passing. In a CIRM blog item
yesterday, Natalie DeWitt, special project director at the agency,
touched on business elements, declaring,

“(The proprosal) will yield better
clinical trial design, accelerated approval of high quality
treatments, and data and know-how to inform regulatory and
reimbursement decisions.”

Reimbursement is the industry euphemism
for creating ways to generate profits for stem cell firms.

The proposal said applicants would have
to bring substantial support from their own institutions and
“demonstrate the potential to bring in a pipeline of additional
stem cell-based therapeutic trials as well as future funding streams
to sustain the clinic.” Applicants would also be “evaluated in
their ability to create a positive 'brand' that would attract
clinical trials.”

Also up in the air was whether grant
reviewers, all of whom come from out-of-state, would have special
expertise to evaluate the business aspects of each applicant's
proposal along with their business track record.

What is before the CIRM directors July
25 at their meeting in Burlingame, Ca., is a request for approval of the concept, which
would be fleshed out for the RFA. The governing board almost always
approves staff concepts, although they may modify them slightly. A
number of directors come from institutions that are likely to be
applicants in the program. They can participate in voting on the
concept plan but would be barred from voting on any applications that
come in later. The two RFAs could go out as early as October with
approval of funding of applications one year from now.

In addition to the Burlingame meeting
site, members of the public can participate from two teleconference
locations in the Los Angeles area. The specific locations can be
found on the meeting agenda.

The California Stem Cell Report will
provide live coverage of the entire meeting, filing reports as
warranted based on the Internet audiocast.

Here are excerpts from CIRM's staff
document on the plan.

“To accelerate therapeutic
development and delivery of stem cell therapies, CIRM proposes
establishing the CIRM Alpha Stem Cell Clinics Network (CASC Network).
The network will be designed to support projects emanating from
CIRM’s funding pipeline, as well as scientifically outstanding stem
cell products being developed worldwide and brought to California.
Conceptually, the CASC Network is intended to be a sustainable
infrastructure designed to support academic- and industry-initiated
clinical trials, and delivery of therapies proven safe and
effective.”
“The major thrusts (of the overall
plan) will be:

• Development of clinical capacity
and associated resources designed to support the effective
implementation and execution of clinical trials and delivery of
registered stem cell therapies

• Compilation of data and
information concerning clinical trial experience and therapy outcomes
to further inform the research, regulatory, and general community
about the status of investigational stem cell interventions and
long-term outcomes

• Dissemination of information to
the public and counseling of patients and potential trial subjects
about therapeutic options and clinical trials involving stem cells in
the network and elsewhere.”

“The long-term vision is for the
Alpha Clinics to expand and accommodate a broad array of stem
cell-based clinical trials, where the trial meets the scientific,
clinical trial design and ethical standards set forth by the Alpha
Clinics Network, as well as FDA approved treatments.”
The coordinating and information center
would be expected to :
“Build relationships with
Accountable Care Organizations, and participate in initiatives for
informing coverage and payment decisions

“Design strategies to attract
investors and philanthropists to CASC network

“Create business plans, and marketing
and branding strategies for financial sustainability of the Alpha
Clinics Sites and (the coordination/information center)”.