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Testimony to California State Assembly,

Select Committee on Biotechnology


August 15, 2018

Maria T. Millan, M.D.


President & CEO, CIRM:

Introduction:

Mr. Chairman & Speaker and Members of the California State


Assembly, Select Committee on Biotechnology, it is an honor to
be here today to provide an update on behalf of the California
Institute for Regenerative Medicine (CIRM). At CIRM, we never
forget that we were created by the people of California. With the
approval of Proposition 71 in 2004, CIRM was formed to
accelerate stem cell and regenerative medicine treatments to
patients with unmet medical needs.

The human stem cell was first isolated in 1998 (just 20 years
ago). Because the stem cell has the unlimited capacity to
proliferate and to give rise to every cell in the body, prominent
scientific leaders and pioneers proposed that the stem cells could
therefore be used to solve the most elusive & devastating human
diseases. At that time, 1998-2000, there were there very few
scientists doing stem cell research and very little funding to do so.
Because of the vision of the citizens of California, patient
advocates and researchers, some of whom you will hear from
today, the California Institute for Regenerative Medicine was
formed in 2004 and California has played a critical role as a
leader in a medical revolution called---REGENERATIVE
MEDICINE.

With its team of highly trained and experienced professionals,


CIRM has developed a way to partner with the best academic and
industry projects to fast track their development into promising
therapeutic approaches. With our timely and efficient funding
process, expert reviewers, advisory resources, infrastructure and
in-house expertise, we have been able to fund the highest quality
programs and accelerate their progression to testing potential
curative treatments for patients with a broad range of diseases for
which there are currently no treatments or cures.

CIRM has funded over 800 projects at over 70 institutions and is


the largest single funder of clinical research for stem cell
regenerative medicine. In 2016, we launched a bold 5-year
strategic plan to cut the time in half for the development of a
regenerative medicine therapeutic and to markedly increase the
number of clinical trials. To date, CIRM has funded 49 FDA
regulated clinical trials and has funded the treatment of over 900
patients in these clinical trials for a broad range of indications
including fatal pediatric orphan diseases, stroke, ALS, spinal cord
injury heart disease, chemotherapy resistant advanced cancer,
diabetes, blinding eye disease, Sickle Cell Disease.

Clinical Trials:

In the next few minutes, I will highlight examples of how CIRM


funds the most innovative, high risk but high reward programs:

(1) We are funding clinical trials for three different forms of


the genetic disease, SCID “bubble baby disease.” These
babies do not have functional immune systems and can
suffer fatal consequences from even the common cold.
The programs we are funding are using the baby’s own
blood stem cells, correcting these cells for the gene
defect, returning the cells to the baby where the stem cells
engraft in their usual home (the bone marrow) & giving
rise to a normal immune cell.
You will hear from Baby Ronnie’s family – Baby Ronnie is on the
cover of our 2017 Annual report- he was recently treated in a
CIRM funded trial at UCSF for X Linked SCID… The cover of our
2016 Annual Report has a picture of Evie, who is cured of
another form of SCID (ADA SCID) and she is 5 years out from
this one-time curative treatment. This ADA-SCID project is in its
“pivotal” phase, the step before it is eligible to getting marketing
approval from the FDA.

The encouraging results with SCID provides a proof of principle


for a major paradigm shift in the medical treatment of SCID---this
represents a curative one-time treatment using the patient’s
own stem cells. This gives true hope for the estimated 10,000
other human diseases that are due to a single gene mutation that
could possibly be treated by a similar approach.

(2) In a different therapeutic approach, we funded Dr.


Mackenzie, a Pediatric Surgeon and researcher at UCSF.
in a trial for babies with alpha thalassemia, a fatal blood
disease that often leads to miscarriage. She is treating
the fetus while still in the mother’s womb where the baby
is most likely to accept the mother’s blood stem cell
transplant as its immune system is still developing. The
goal is to get the baby to full term delivery and to cure the
baby of its disease. This trial along with a picture of the
first baby enrolled (3-week-old baby Elianna, pink and
wide eyed) was featured in the May 25 issue of the
NYTimes. This trial is still in progress, but this is the first
step.

(3) While these previous examples are with blood stem cells,
we are also funding trials using stem cells that are not
from the patient such as the use of gene-modified
allogeneic (from donor tissues) neural stem cell therapy
for ALS (Lou Gehrig’s disease) – in a trial being
conducted at Cedars Sinai

(4) Many of you may have heard of the excitement around


curative immunotherapy and CAR T for Cancer. CART
are immune cells which have been gene modified to
express a powerful and specific docking device, so they
can seek & selectively destroy the cancer. CIRM has
been funding the next generation CART using stem cells
for solid cancers. The program at City of Hope targeting
Glioblastoma, a deadly brain cancer, is the first program
to yield the first royalty check ($190K) to the State in
December 2017.

(5) A variety of definitive or curative stem cell technologies is


being developed by CIRM investigators for prevalent
diseases as well such as stroke, diabetes, heart disease,
blinding eye disease, HIV-AIDS,

Every single trial represents a tremendous breakthrough


approach that was unimaginable and even the subject of science
fiction just years ago.

The Next Wave of Stem Cell Technologies.

As the aforementioned innovative treatments are making amazing


progress, the next wave of stem cell technologies will require the
same type of early stage investment and partnership that CIRM
has uniquely provided.

Shinya Yamanaka received the Nobel Prize in 2012 for his


discovery that stem cells (induced pluripotent stem cells that
mimic the behavior of the embryonic stem cell) can be created
from regular skin cells or cells from a blood draw. The key
advance with this technology is that there is the potential to
reliably grow an unlimited number of stem cells derived from
mature tissues that are readily available.

Dr. Yamanaka continues to do work to optimize this technology so


it could be safely developed into human therapeutics & CIRM has
funded his research at the Gladstone Inst, SF.

We do not yet have a clinical trial with iPSC in the US but the
promise for these iPSC cells is tangible & will take a few more
years to mature – for instance in taking a skin biopsy, turning it
into dopamine secreting neurons and treating Parkinson’s
Disease. That will require the type of early investment, expertise
in translational research and specialized systems that CIRM has
put in place.

CIRM’s Value proposition has been recognized by:

(1) Industry: Early research in academia suffering from the


“valley of death”. CIRM supports and de-risks through this
stage, where industry and traditional investors are not yet
ready to come in. By accelerating development and allowing
these early stage trials to proceed with CIRM funding, we have
enabled the projects to gain visibility and interest leading to an
increase in industry investments. Industry investment into
these “high risk’ programs, in many cases, occurred only after
CIRM supported them in the early stages. As an example, a
project that targeted Cancer Stem Cells was supported by
CIRM when the idea of “Cancer Stem Cells” was still being
debated …. now the concept that cancer stem cells play a role
in cancer biology is widely accepted and the project that we
funded to get into clinical trials, was spun out and licensed into
a company (47 Inc) and went on to raise $150M additional
financing and, just a little over a month ago, that company
successfully went public with its IPO in late June.

This is just one example of how CIRM dollars have been


leveraged to bring in additional investments into CIRM funded
programs. Just a bit over the halfway mark this year alone, we
have already seen $$575 M in industry investment into our
programs bringing the total Industry investment to approx.1.2B.
In fact, the $2.7B in CIRM awards has been leveraged to bring in
approximately $2.7B from other funding sources.

(2) By Federal Agencies The NIH- NHLBI have launched a


Cure Sickle Cell initiative and have signed an MOU with CIRM
because they recognize the value of CIRM’s expertise,
infrastructure and systems designed to increase the chances
of accelerating the science in the cell and gene therapy field to
finding a cure for this devastating condition. The FDA has
granted a disproportionately high number of a special
breakthrough / accelerated pathway called RMAT designation
for our CIRM funded projects (among the first to obtain in the
country)

(3) By the Scientific & Policy Community At prominent


national and international conferences, CA and CIRM are
recognized as leaders in the field and the field is looking to us
to remain in this role and have been seeking ways to partner
with us to globalize the assets and value proposition that we
have developed.

(4) Medical Community: CIRM has facilitated the integration


of novel regenerative medicine technologies into top notch,
accredited medical centers, by forming the Alpha Clinics
Network- the first & only stem cell clinical network composed
of top academic centers 5 programs (7 institutions)- designed
to be efficient, serve the unique challenges of these
treatments, accelerate while providing the highest quality care,
specialized nursing, shared/ efficient resources and a learning
environment for the benefit of patients. This Network,
composed of UCSD, City of Hope, UCLA/UCIrvine, UCSF-
Benioff Children’s Hospital and UCDavis, has conducted 56
stem cell and regenerative clinical trials (including 35 of the 49
CIRM funded trials).

Conclusion:
California is the birthplace of biotech, an industry built on the use
of the natural biology of microorganisms to produce therapies,
California is now also in the lead in the new field of regenerative
medicine, a field that harnesses the natural biology of the human
cells to produce therapies that were previously unimaginable.