The likely future of the $3 billion California stem cell agency
was unveiled this week, and it envisions an enterprise no longer tied
to state funding and much more closely linked with industry as a
collaborator and “bundler” of resources.
Gone would be the $300 million a year in cash that the state borrows so
that agency can award grants to academics and occasionally to
business. Likely to be missing are faculty recruitment awards, non-
business training programs and perhaps most of the agency's basic
research effort.
Instead, the
California Institute for Regenerative Medicine(CIRM),
as the agency is formally known, would build a relatively small
number public-private partnerships to back projects close to turning
out commercial therapies. It would ally itself with the
Alliance for
Regenerative Medicine,
a national lobbying and industry group in
Washington, D.C. And the agency's funds would come possibly from
foundations, philanthropists, investors, biopharma, the health
insurance industry and federal agencies such as the
NIH and
Medicare.
That is part of the scenario painted in
a $150,000, 69-page report
by consultant
James Gollub of Tiburon, Ca. CIRM earlier this year
commissioned the report because the agency will run out of state
funds for new grants in 2017.
The governing board of the stem cell agency will hear a report on
the Gollub's recommendations at its
Dec. 11 and Dec. 12 meeting in
Los Angeles. The report will come as part of consideration of
proposals by the agency's new
Scientific Advisory Board that
the agency should sharpen its focus on six to eight projects to push
them closer to bearing commercial fruit. The proposed “strategic
roadmap” also comes as the agency is
looking for a new president to
replace
Alan Trounson, who is leaving to rejoin his family in
Australia.
Trounson has prepared
an outline of a plan on how to start implementing the proposals.
Gollub made three major recommendations, one of which would require a $50
million investment from CIRM with another $50 million to $100 million
coming from other sources, including wealthy individuals such as
businessman
Denny Sanford who recently gave
UC San Diego $100 million
for stem cell research.
The report said additional costs, including
those for outside consultants, could be absorbed by CIRM or funded
through awards on a charge-back basis. In others words, a grant would
include funds that a researcher would have to pay to the agency for
its “internal program management services.” CIRM is limited by
law to an operating budget that can total only 6 percent of its $3
billion, which keeps its overhead quite lean.
Gollub noted that that CIRM has already moved partially into the
areas of his three proposals, all of which would begin in the next year or so. They are:
- Create public-private partnerships to move projects into early
clinical trials, focusing on specific disease areas. Co-funders providing at least $50 million would
screen and select the projects from those presented by CIRM. This
could be scaled up from a pilot project next year and possibly
involve creation of a nonprofit group by CIRM.
- Create a “regenerative medicine accelerator” to provide
“commercial readiness services” to each(Gollub's italics) grant
recipient whether academic or business. The accelerator effort would
be linked to agency's proposed $70 million Alpha Clinic plan. The
accelerator also would assure that the “clinical trials structure
meets pharmaceutical industry expectations.”
- Create a pre-competitive regenerative medicine R&D program
that would organize collaborative efforts to break through barriers
to development of therapies. This would involve production but also
what the biotech industry calls “reimbursement,” which is a catch
word for making a profit from development of a therapy. One of the
issues in the industry is the expense of some medical treatments, and
stem cell therapies are expected to be very high. The idea is to
work with the insurance industry and the federal government to be
sure the appropriate cost is supported by Medicare and insurance
programs. Partnering with the Alliance for Regenerative Medicine would
enter into this effort, which would be ultimately funded by both
CIRM, the new non-profit and other partners.
The general direction of the recommendations is to “de-risk” stem cell therapy development and provide a focal point for overcoming many of the regulatory and manufacturing obstacles facing a new medical technology.
The report said CIRM has as a “strong innovation feedstock” of
more than 90 projects that are close to moving into clinical trials.
Some of those projects are likely to appeal to Big Pharma, which has
been increasingly looking outside of its own companies for R&D.
For donors willing to pay for the privilege, Gollub's “strategic
roadmap” recommended that CIRM create a group that would have
“'a seat at the table' to see early
stage research, discoveries and clinical performance.”
The report said the CIRM's new world would require a “robust,
dedicated fundraising group” within the agency, which now has minimal
capacity in that area. However, the study envisions members of the governing
board, many of whom are top notch fundraisers, as making a major
effort to raise cash.
Scaling up the initial public-private partnerships, accelerators,
etc., would also require additional staff. Gollub's report did not
present costs beyond the initial pilot project stage.
