Amazing Team Research at the California Stem Cell Program

By Don C. Reed

As high as $20 million each, the disease team grants are the most spectacular research projects offered by the California stem cell program.

–http://www.cirm.ca.gov/grants

The challenge is enormous: to succeed, each team of scientists must bring a therapy to human trials within just four years. At every step of the way milestones of progress must be met, or the funding stops—and every disease these teams will challenge has been declared chronic: incurable. A victory here would change medical history.

How are they doing? On March 19th, the next public meeting of the California stem cell program’s board of directors, there will be an update on the 25 teams in action now.

–http://www.cirm.ca.gov/sites/default/files/sites/default/files/2013_ICOC_Schedule.pdf

Here are the teams: may they all succeed.

1. ALZHEIMER’S DISEASE: Alexandra Capela, of Stem Cells Inc., wants to transplant neural stem cells directly into the hippocampus portion of the brain, attempting to “markedly restore memory and thus, quality of life, for patients.”

– http://www.cirm.ca.gov/Grant/restoration-memory-alzheimers-disease-a-new-paradigm-using-neural-stem-cell-therapy

2. BLINDNESS: Mark Humayun, USC, estimates that “by 2020, over 450,000 Californians will suffer from vision loss or blindness due to age-related macular degeneration…the loss of a layer of cells at the back of the eye…this can be overcome by transplanting (new) cells derived from embryonic stem cells…”

–http://www.cirm.ca.gov/content/stem-cell-based-treatment-strategy-age-related-macular-degeneration-amd

3. BLINDNESS: Henry Klassen, UC Irvine, hopes to use cells to treat retinitis pigmentosa, (RP) a severe disease of the eye. His approach is to “save the light-sensing cells of the eye… an application will be made to the FDA… following approval, a small number of patients with severe RP will be injected with cells in their worse-seeing eye.”

–http://www.cirm.ca.gov/Grant/retinal-progenitor-cells-treatment-retinitis-pigmentosa

4. BRAIN TUMORS: Karen Aboody, City of Hope, has built a stem cell bank to make her prospective attempt at destroying brain tumors widely available. Her techniques may also remove “solid tumors throughout the body”…” applicable to other deadly growths.

http://www.cirm.ca.gov/content/stem-cell-mediated-therapy-high-grade-glioma-toward-phase-i-ii-clinical-trineurological-diso

5. CANCER: Dennis Slamon, UCLA, points out that “ a minor population of cancer stem cells drives the growth of an entire tumor…” These trigger-like cells can bring back the cancer which appeared to be gone. Fortunately, says Slaymon, “our lead drug can inhibit the growth of cancer stem cells.”

– http://www.cirm.ca.gov/content/therapeutic-opportunities-target-tumor-initiating-cells-solid-tumors

6. CANCER: Judith Shizuru, Stanford, hopes to “enable chemotherapy free transplants”, with the use of a selected antibody. The same technique might also save the lives of children born with an immune-system defect, Severe Combined Immune Deficiency (SCID), who often die before the age of two.

–http://www.cirm.ca.gov/content/therapeutic-opportunities-target-tumor-initiating-cells-solid-tumors

7. CANCER: Antoni Ribas, UCLA, seeks to reprogram stem cells, hoping to “redirect the patient’s immune response to specifically attack the cancer….”

–http://www.cirm.ca.gov/Grant/genetic-re-programming-stem-cells-fight-cancer-0

8. CRITICAL LIMB ISCHEMIA: John Laird, UC Davis, hopes to benefit two million Americans currently at risk of leg amputation or death. Since a key problem in this condition is low oxygen level in the affected limbs, Laird has shown that stem cells can “migrate into the areas of lowest oxygen…wrap around the damaged…blood vessels…and secrete (helpful) factors where needed most…” A stem cell hug of life!

– http://www.cirm.ca.gov/Grant/phase-i-study-im-injection-vegf-producing-msc-treatment-critical-limb-ischemia

9. DIABETES: ViaCyte, Inc. is challenging diabetes, using embryonic stem cells to build “beta cells, which have been shown to cure experimental diabetes in mice and rats… could become the most significant stem cell-based medical treatment of the coming decade…”

– http://www.cirm.ca.gov/content/cell-therapy-diabetes

10. HEART ATTACK: Rachel Smith, of Capricor, Inc., wants to use stem cells to turn heart attack scars back into healthy tissue. As scarring makes the next heart attack more likely, Smith’s method may save lives.

–http://www.cirm.ca.gov/Grant/allogeneic-cardiac-derived-stem-cells-patients-following-a-myocardial-infarction

11. HEART ATTACK: Eduardo Marban of Cedars-Sinai Medical Center, says: “The adult human heart contains small numbers of cardiac stem cells that are able to partially repair the heart following a heart attack…we have developed a way to isolate these cells and grow them to large numbers…” Clusters of these cells could be injected into the heart– even in the middle of a heart attack.

– http://www.cirm.ca.gov/content/autologous-cardiac-derived-cells-advanced-ischemic-cardiomyopathy

12. HEART FAILURE: Robert Robbins, Stanford, intends to use “embryonic stem cell-derived (heart cells) for patients with end stage heart failure…transplanting cells rather than a whole heart….”

–http://www.cirm.ca.gov/Grant/human-embryonic-stem-cell-derived-cardiomyocytes-patients-with-end-stage-heart-failure-0

13. HIV/AIDS: Irving Chen, UCLA, believes that the process which can change the color of a flower (RNA interference) may “block the spread of HIV infection…”. Right now, surviving with AIDS means requires several dozen pills a day. But the Chen method could mean “a therapy that will require only a single treatment…”

– http://www.cirm.ca.gov/content/hpsc-based-therapy-hiv-disease-using-rnai-ccr5

14. HIV/AIDS: John Zaia, City of Hope, may have found a way to duplicate a “cure” of an AIDS patient who received a bone marrow transplant with a mutation called delta-32. This mutation apparently blocks entry of the AIDS virus. It may be possible to alter a patient’s cells so that they can also block the virus.

– http://www.cirm.ca.gov/content/therapeutic-opportunities-target-tumor-initiating-cells-solid-tumors

15. HUNTINGTON’S DISEASE: Vicki Wheelock, UC Davis, hopes to fight the fatal disease with stem cells as a delivery vehicle: bringing a nerve-growth factor called Brain Derived Neurotrophic Factor, (BDNF) to the at-risk nerve cells.

–http://www.cirm.ca.gov/Grant/msc-engineered-produce-bdnf-treatment-huntingtons-disease-0

16. LEUKEMIA: Catriona Jamieson and Dennis Carson of UCSD are fighting an after-treatment problem with the deadly disease; it often leaves small amounts of leukemia stem cells still alive, to come back stronger later. The Carson team hopes to make anti-leukemia drugs available soon.

– http://www.cirm.ca.gov/content/development-highly-active-anti-leukemia-stem-cell-therapy-halt

17. LEUKEMIA: Irv Weissman, Stanford. The body’s protective cells, macrophages, should “eat” the leukemia invaders—but they don’t. A marker cell called CD-47 on the leukemia cells acts like a “don’t eat me” sticker. But if the marker could be removed….

–http://www.cirm.ca.gov/content/development-therapeutic-antibodies-targeting-human-acute-myeloid-leukemia-stem-cells

18. LOU GEHRIG’S DISEASE: Larry Goldstein, UCSD, hopes to prevent the progression of the fatal disease (amyotrophic lateral sclerosis, ALS, or Lou Gehrig’s Disease) by using astrocytes (nerve support cells) developed from embryonic stem cells.

–http://www.cirm.ca.gov/content/stem-cell-derived-astrocyte-precursor-transplants-amyotrophic-lateral-sclerosis

19. LOU GEHRIG’S DISEASE: Clive Svendsen, Cedars-Sinai Medical Center, wants to use nerve “fertilizer” (Glial Derived Neurotrophic Factor, GDNF) to treat the deadly disease. Svendsen wants to use neural progenitor cells as “Trojan horses”, to carry the GDNF to the nerve cells, where it is needed.

–http://www.cirm.ca.gov/Grant/progenitor-cells-secreting-gdnf-treatment-als

20. MUSCULAR DYSTROPHY: Stanley Nelson, UCLA, is tackling the devastating and muscle-wasting disease. Nelson’s technique brings repairs “directly inside each muscle cell”… and is “predicted to lessen the disease severity…”

http://www.cirm.ca.gov/Grant/combination-therapy-enhance-antisense-mediated-exon-skipping-duchenne-muscular-dystrophy-0

21. OSTEOPOROSIS: Nancy Lane, UC Davis, wants to strengthen the bones of the elderly, and thereby lessen the number of bone fractures they suffer. In osteoporosis, bones can grow almost transparently thin and accordingly weak. Lane has developed a small molecule that directs stem cells to grow the skeleton stronger.

–http://www.cirm.ca.gov/Grant/treatment-osteoporosis-with-endogenous-mesenchymal-stem-cells

22. SICKLE CELL DISEASE: Donald Kohn, UCLA, offers a stem cell therapy for the agonizing and often fatal disease. The blood cells change shape into a c-shape like a sickle, said to cause pain like broken glass in the veins. Kohn’s approach will transplant “adult cells that are genetically corrected by…a gene that blocks sickling…”

–http://www.cirm.ca.gov/content/stem-cell-gene-therapy-sickle-cell-disease-0

23. SKIN DISEASE: Alfred Lane, Stanford, hopes to use induced Pluripotent Stem Cells (iPSC)s to combat a hideous disease, dystrophic epidermolysis bullosa, in which the skin literally rips apart. His method may treat other skin disorders as well.

– http://www.cirm.ca.gov/content/ips-cell-based-treatment-dystrophic-epidermolysis-bullosa

24. SPINAL CORD INJURY: Nobuko Uchida, Stem Cells Inc., wants to transplant neural stem cells into the spine to ease chronic cervical spinal cord injury. His method has restored motion in animal models, and “could potentially reverse paralysis and improve the patient’s quality of life.” For paralyzed people, even a small improvement is hugely positive: perhaps the difference between needing an attendant and not.

–http://www.cirm.ca.gov/Grant/neural-stem-cell-transplantation-chronic-cervical-spinal-cord-injury

25. STROKE: Gary Steinberg, Stanford, calls stroke “the number one cause of disability, the second leading cause of dementia, and the third leading cause of death in adults…” His team will use embryonic stem cells– to make neural stem cells– to “augment the body’s natural repair process after stroke.”

–http://www.cirm.ca.gov/content/embryonic-derived-neural-stem-cells-treatment-motor-sequelae-following-sub-cortical-stroke

Major Changes Accepted at California Stem Cell Program

Major Changes Accepted at California Stem Cell Program

By Don C. Reed

As a long-term patient advocate, I have attended probably more public meetings of the California stem cell program than anybody, staff included; and I have never ceased to be amazed at the dedication of all concerned.

The most recent meeting was a response to what I consider a grossly misguided study of our program by the Institute of Medicine. Google it for the full 166 page.

The study maintained the California Institute of Regenerative Medicine (CIRM) was rife with potential conflicts of interest, including, according to their view, patient advocates: people with a medical condition, or their family.

“…The committee believes that personal conflicts of interest arising from one’s own or a family member’s affliction with a particular disease…can create bias for board members..” ibid–section 3, page 14

Patient advocates are the people who organized, led, fought for, and supported Proposition 71, the citizens’ initiative which led to the California Institute of Regenerative Medicine. Bob Klein who inspired the program has a son with type one diabetes. My son Roman Reed is paralyzed from the shoulders down. Patient advocates stood on street corners for nearly a year gathering signatures to get the measure on the ballot. Without us, there would be no three billion dollar stem cell program.

Yet now, having done the work, we were not to be part of the decision-making?

Pioneering stem cell scientist Dr. Jeanne Loring said it best, in a letter she asked me to read into the public record:

“Patient advocates are extremely valuable to us researchers.

“With my first CIRM grant, I started meeting patient advocates, and now I can’t imagine pursuing a disease-related research project without them…They are wonderful sources of knowledge: Jeff Sheehy taught me about HIV/AIDS and patient activism, I learned about Parkinson’s disease from Joan Samuelson, autism from John Shestack, David Serrano-Sewell and Diane Winokur have educated me about MS and ALS.

“Advocacy makes CIRM-funded research breathtakingly relevant and uniquely powerful to change the course of medicine.”— Jeanne Loring, Ph.D. ,Professor and Director of the Center for Regenerative Medicine at the Scripps Research Institute in La Jolla, CA.

Even so, those three words, “conflict of interest”, are huge, whether applied to patient advocates, or to the board of directors: the Independent Citizens Oversight Committee (ICOC).

Virtually every critic of the program alleges conflicts of interest, noting (correctly) that many of the institutions which get research money are also on the ICOC. For example, the University of California system has many researchers; it is natural for it to be represented on the board—where else should the money go, if not to where the researchers are? And the members are not allowed to vote on anything which might benefit themselves.

Yet, like Caesar’s wife, CIRM must be above suspicion: held to the highest standards.

The Board listened to the IOM: and offered changes to address the criticism.

From the official CIRM statement:

“One of the big issues raised by the IOM report was that our current governing structure created a perception of conflicts of interest in how we fund research,” says Chairman Jonathan Thomas. “While no one has found any actual conflicts, these changes directly address the broad public perception.”

Translation: no actual conflicts of interest were found, only the possibility that some might develop.

This fit with earlier studies. Both the California legislature and the State Controller’s office had found no conflicts of interest in the program.

In fact, the CIRM’s: “conflict of interest rules are modeled on, but exceed, the standards established by the National Institutes of Health…”
–http://cirm.ca.gov/files/meetings/pdf/2013/012313_item_5.PDF

But perceptions, accurate or not, can be damaging. Chairman Thomas believed the board must answer the issue, or lose credit with the public. He offered a draconian solution:

“By having board members who belong to institutions that can get CIRM money abstain from all votes on funding we take even the perception of conflict of interest out of the picture….”

Thirteen members of the board of directors just had their right to vote taken away.

From now on, institutions which could receive funding from the program will no longer be able to vote–at all.

How significant were the changes? In a published e-mail to Chairman Thomas, the study’s leader, Harold Shapiro, former President of Princeton, commended the stem cell agency for its response, calling it:

“…very thoughtful and significant…will serve the long term interests of the citizens of California and the field of regenerative medicine. … dealing with financial conflicts of interest, enhancing the credibility and integrity of the scientific review process…”

With an essentially unanimous vote, (one abstention) the board voted to approve the changes (subject to a month’s public comment) for a trial period of one year. When that testing phase is done, the changes will either become permanent, or be altered.

And the patient advocates? Their vote was taken away on the Grants Working Group, which recommends research projects to the board—but patients on the board will still vote—and our right to participate in public comment has not been infringed.

“These are big potential changes, and important ones,” said Jonathan Thomas, JD, PhD, Chair of the Board. “We are proposing them to address the concerns of the IOM, and to demonstrate how seriously we take the recommendations detailed in their report….”

All change is painful, more so when its necessity is questionable.

But there can be no debate that the board took the high road. Even those who would be losing their right to vote still approved the changes; their belief in the program and the importance of its public perception was more important than their personal authority.

For the California stem cell program, the conflict of interest issue should officially be declared null and void.

GREATEST STEM CELL PROGRAM IN THE WORLD– attacked again

THE GREATEST STEM CELL PROGRAM IN THE WORLD—under attack, again!

By Don C. Reed

Few institutions are more beloved–and more often attacked– than the California stem cell research program, the California Institute for Regenerative Medicine (CIRM).

For patient advocates like my paralyzed son Roman Reed and myself, the $6 billion program is like nothing else on earth: an institute solely designed to bring cure to chronic conditions, often called incurable. Every time I attend a CIRM public meeting, I feel a sense of disbelief, that California was able to make this happen.

Not without opposition! Lawsuits from the Religious Right tried to shut it down; opponents of “big government” wrote countless op-ed pieces opposing our program– well-meaning legislators tried to micromanage it. A small cottage industry of critics emerged: a mixed blessing. The same endlessly-quoted handful of folks offer constant negativity, but also suggestions for improvement, some of which have been adopted.  

The most recent threat comes in the form of a study:  “Committee on a Review of the California Institute for Regenerative Medicine (CIRM): Health Sciences Policy; Institute of Medicine”, available at: http://www.nap.edu/catalog.php?record_id=13523.  

While lacking power in itself, the study could become the basis for a new law  to revise and re-do the California stem cell program, in defiance of the voters’ will.

The study begins politely enough, complimenting CIRM:

 ”… enhances California’s position as key international hub of activity… enriching regenerative medicine everywhere… impressive research portfolio…exemplary training program…   translational projects (ready) for industry involvement.”

Unfortunately, the study then recommends radical surgery! The proposed “improvements” would not only overturn the program’s structure, but might even violate state law. CIRM is in our state Constitution.– http://www.cirm.ca.gov/pdf/prop71.pdf)

The study objects to the program’s board of directors, the Independent Citizens Oversight Committee (ICOC), a 29-member panel of experts, appointed by public officials. These board members, the study believes, have an automatic conflict of interest, meaning they could use their votes to benefit the colleges or businesses for which they work.  

“They make proposals to themselves…regarding what should be funded. They cannot exert independent oversight.”—Harold Shapiro, Chair of the study.

This is provably false.

Members of the ICOC may NEVER “make proposals to themselves”. They are prevented by law from so doing: board members may neither discuss nor vote on projects which could benefit their parent organizations.

Here is what actually happens. 

First, California scientists offer their proposals. These are evaluated by the Grants Working Group, (GWG), a panel with a majority of out-of-state stem cell experts.

Each project is scored, from 1-100. The grant proposals fall into three categories: essentially YES, MAYBE, and NO. 

At a public hearing, the projects are listed in order of scoring, on a big screen in the auditorium.  The various grant proposals are also divided by color, so it is easy to tell which is recommended for funding.

The board has read the proposals, posted  on the public CIRM website (www.cirm.ca.gov). They have been reminded which projects they may not discuss.

The chairperson asks: does anyone want to move a proposal up or down?    

In most of the cases, probably 90%, the ICOC agrees with the recommendations. 

But if a board member challenges the placement of a grant, then the CIRM’s scientific staff will give its opinions, pro or con. The scientist applying for the grant may also speak. The board makes comments, as do we in the public—our only chance to do so.

Example:  a recent disease team proposal was offered, a new way to fight Alzheimer’s disease with its terrible memory loss.

http://www.researchstore.com/technologynetworks/News/stemcells_wins_20_million_from_cirm_for_alzheimers_disease_program?productid=EF6D220C-8225-4A37-A21F-126572F21FD3

It was a major grant, a $20 million dollar project by Stem Cells, Inc., led by Dr. Frank LaFerla of UC Irvine. It had been rejected by the Grants Working Group. But I had studied the proposal and it seemed groundbreaking– the scientists had achieved unprecedented memory return in the laboratory mice.  So, in the three minutes allowed me (as for any member of the public) I argued in support.

The debate lasted an intense 45 minutes. When all sides had been heard, the chair called for a vote by the board. One by one they declared themselves: those who were eligible.     

When the votes were counted, the Alzheimer’s project had won. It is going forward today. Will it work? I don’t know. But we are going to find out.

To me, this is America at our best:  everybody having their say: the board, the scientists, the patients, the public: transparent government, out in the open, no secrecy.

Unfortunately, this is what the study would destroy.

“The board…should not be involved in day-to-day management. (It) should delegate day-to-day management responsibilities to the President…” — Section 3, page 11

What do they mean by “Day-to-day management”? Nothing less than who gets the money.  Giving grants for stem cell research is not trivial housekeeping; it is our whole program. Take that away from the board, and they might as well go home.  

And who does the study suggest should now make these crucial decisions, instead of our 29-member board, interacting with scientists, staff, patient advocates and the public?

Two people would be in charge, one of whom works for the other.

“The Senior Vice President and the President…decide on a final slate of proposals to submit to the ICOC for a “yes” or “no” vote on the entire slate.

…the ICOC (Board)… should NOT (emphasis added) be empowered to evaluate individual applications…..”)– Section 4, page 18

“A “yes” or “no” vote on the entire slate?   

If the Board can only make a blanket decision on several dozen projects at once, all the research projects going forward or none of them—what kind of choice is that? Are they going to say NO, and block all the research?    

Instead of our open process, funding decisions would be made in private; the public would be denied meaningful participation: and our Board reduced to a rubber stamp.

Wednesday,  January 23rd, is an all-day workshop (10:30 AM to 6 PM) to consider the IOM study.   To attend, come to the Claremont Hotel, 41 Tunnel Road, Berkeley, CA 94705.  Or listen to the audiocast at (800) 700-7784.  Participant Access Code: 279280

Information at: http://www.cirm.ca.gov/Agenda_2013-01-23/icocgoverning-board

Opinions may be sent to the Board Chairman Jonathan Thomas, jthomas@cirm.ca.gov

Remove Patient Advocate Voices from the California Stem Cell Program>

REMOVE PATIENT ADVOCATE VOICES FROM THE CALIFORNIA STEM CELL PROGRAM?

By Don C. Reed

What group of people was Proposition 71, the California stem cell program, designed to help?

Patients with incurable disease or disability.

Who speaks for them? They do, and we do—the patients themselves and those who advocate on their behalf.  Patient advocates are people like my son Roman, who in his wheelchair, helped gather 1.2 million signatures to get the Initiative on the ballot.

Patient advocate Bob Klein inspired, led, and helped raise the $30 million to pay for the campaign; patient advocates defended the program at every step, including fighting for decent salaries for staff and leadership, so they would be paid well instead of adequately.

So why should anyone want to silence patient advocate voices now—especially when their support will be crucial if there is to be a continuation of the program?

January 23^rd, 9-5, an all-day meeting on the future of the California stem cell program will be held at the Claremont Hotel in Berkeley, California. Check with www.cirm.ca.gov for official announcements.

The meeting will focus on a recently completed “study” of the California stem cell program.

The entire 166 page report, “Committee on a Review of the California Institute for Regenerative Medicine (CIRM): Board on Health Sciences Policy; Institute of Medicine” is available at http://www.nap.edu/catalog.php?record_id=13523.

I consider both the study and its recommendations staggeringly misguided.

If its recommendations were enacted, they would silence stem cell patient advocate involvement, eliminate public debate on funding proposals, and delegate the real decisions to secret proceedings by an out-of-state-controlled board.

It would no longer be the program California voted into law.

Presently, patient advocates are both members of the board of directors, and welcome members of the public. Our voices are respected and routinely heard; we are welcome at the table when decisions are made.

This is something wonderful, which would be destroyed if the study’s recommendations were adopted wholesale.

The study was authored by a 13-member panel which had just one stem cell scientist, zero patient advocates, no biotech representatives, and precious little understanding of Proposition 71’s place in California law. http://voterguide.sos.ca.gov/past/2004/general/propositions/prop71text.pdf

It feels so one-sided: with opponents of the program featured prominently, and their opinions given credence.

Where are the 200-plus major organizations representing literally millions of Californians and Americans who support our program? Where are the viewpoints of the California Medical Association, the Coalition for the Advancement of Medical Research, the California Children’s Hospital, the Chambers of Commerce, Bay Bio, the International Society for Stem Cell Research, just to name a few?

The IOM’s objections to California’s program may be summed up in two sentences:

“They (the governing board) make proposals to themselves…regarding what should be funded. They cannot exert independent oversight.”—Walter Shapiro, Chair of the study.

This is demonstrably false.

Members of the governing board NEVER “make proposals to themselves”. They are prohibited by law from so doing. In fact, they may not even voice an opinion on any project which might benefit their parent organizations.

Here is how it actually works: scientists offer proposals, which are evaluated by the grants working group, (GWG), with  a majority of out-of-state stem cell experts.

After being scored, the projects are brought before the 29-member Independent Citizens Oversight Committee (ICOC) for a public hearing.

Typically the grant proposals fall into three categories:  recommended, maybe, and not.

The project names and scores are put up on a screen so everyone can see. Then the chair will ask: does anyone believe a particular grant proposal is in the wrong category?

In the overwhelming majority of cases, as high of 90% of the time,  the ICOC agrees with the recommendations.

But when a board member disagrees with a score, he or she may ask that it be publicly discussed.   Also, the grant applicants themselves can submit a petition pointing out errors in the scientific critique.  They are allowed three minutes to state their case.

The CIRM scientific staff gives its opinion, in support or opposition.

The board gives its comments– and we in the public are allowed to offer support or objections—our only opportunity to do so.

Underline that in your mind: this is the patient advocates’ only chance to influence a decision on what shall be funded. Take it away, and we might as well just go home—after doing the work to make the program possible, we would have been shut out.

After this, the board votes.

Instead, the IOM recommendations would silence both the governing board AND  patient advocates.

The board would only be allowed to vote yes-or-no on the entire slate of grants. What are they going to say? No, don’t let anything go forward? They would be reduced to a rubber stamp.

No public discussion would be allowed on individual grants.

Not only would this remove patient advocates, educators, biomedical authorities and ethicists from significant input, it would also violate California law: resulting in the real decisions being made in secret, by the out-of-state controlled Grants Working Group.

This is unacceptable.

The patient advocacy community provide real-life insights on current therapies and policies. No one knows better what patients need to sustain both life and the quality of life—and who better to be heard on the risk/benefit decisions when a new drug or therapy might be tried?

A few days ago, a grant to fight thalassemia and sickle-cell anemia was approved for funding: these diseases disproportionately affect African-American and Asian-American communities. What if that grant had been turned down? Should the public have no avenue to voice our concerns?

Yet the IOM proposal would deny our voice. Why?

The study labels the patient advocates as biased because we want cures for ourselves or our loved ones. Because we are affected, we are ineligible as part of the solution?

“No taxation without representation” would seem to apply here. Those who want our help must accept our intelligent  involvement.

There was a time when patients passively accepted doctoral decisions as from on high.

California today takes a different approach. Here, patients and advocates are active partners in the stem cell program we built.

Do others consider patient advocates useful? Listen to the U.S. Food and Drug Administration.

The “FDA includes Patient Representatives (with voting rights—dr) on advisory committee panels that review products and therapies related to serious and life-threatening diseases…” –http://www.fda.gov/ForConsumers/byAudience/ForPatientAdvocates/default.htm

The IOM report considers the governing board itself to be biased because its members were chosen from leaders of colleges, research institutions, the biomed industry, and other organizations, including ten patient advocates from various disease areas.

This is a convergence of expertise, not a conflict of interest.  Our Initiative was always intended to be led by experts from the scientific and medical fields. When we were sued by the Religious Right, this very issue was part of their attack—and it was defeated. Having experts on a board is nothing shocking. Scientists from prestigious research institutions serve on National Institutes of Health (NIH)  peer review panels. Are they biased too?  The IOM seems to want board members to have no connection with either science or chronic illness—should ignorance be considered an advantage?

The IOM study goes so far as to recommend “Elimination of the current process in which applicants rejected by reviewers appeal publicly to the governing board. …. Instead appeals would be heard only by staff BEHIND CLOSED DOORS.” (emphasis added)

More secrecy, instead of open argument?

The IOM would actually deny board membership to anyone with a “personal conflict”  meaning someone in their  family had an incurable illness, because that might influence their vote. What nonsense is that?

First, it is hard nowadays to find a person who does not a family member suffering a chronic disease or disability. My son is paralyzed, my sister has cancer, my brother has a permanently crippling leg injury—so I would be disqualified—three times!

We need people on the board who care passionately about fighting disease and disability, people who know what it means to have a loved one at risk—that is why we are fighting!

The report does admit the California program made tremendous achievements, saying:

“bold social innovation… enhances  California’s position as one of the key international hubs of activity in regenerative medicine…impressive research portfolio…enriches regenerative medicine everywhere…sets in motion a significant scientific enterprise… an impressive grant management structure… exemplary training program… a pipeline of intellectual property… translational projects primed for industry involvement.”

There we agree. The California stem cell program is indeed something fantastic; it works. So why mess with it, in such a brutal and insulting manner?

If you have an opinion on this subject, join us at the public workshop at the Claremont Hotel, Berkeley, January 23^rd, 9-5. If you want to be heard, be there.

At this all-day meeting, the proposals of the IOM will be considered, both in regards to California law (to this non-lawyer some of the changes appear illegal), and the voter-approved structure of Proposition 71, as written in the California Constitution.

As it does with all serious criticisms, the Independent Citizens Oversight Committee will study the IOM opinions carefully. Where improvements can be implemented without compromising the integrity of our program, they will be made.

But the open and interactive structure of this magnificent program must not be destroyed.

 

 

A Visit to the World Stem Cell Summit

LIKE TOUCHING TOMORROW… 8th Annual World Stem Cell Summit

By Don C. Reed

The just-concluded World Stem Cell Summit, December 3-5, left advocates excited, revitalized, updated—and cheerful!

It might seem odd that the battle against chronic disease, the most terrifying enemy in the history of the world, could sometimes be fun. Chronic disease touches every household. With an estimated 100 million Americans (one in three) suffering an incurable disease or disability—at a financial cost exceeding the annual instalment of the national debt—everyone is affected.

But those who take on this incredible challenge can be some of some of the warmest personalities you could imagine.

Like Bernie Siegel. Organizer of the World Stem Cell Summit, Bernie reminds me of the story of the argument between the Sun and Winter as to who was stronger? Old Man Winter pointed to a man and said, “I will make him take off his jacket”, and blasted him with icy winds. But the man clutched his jacket tighter. The Sun merely smiled, and the man grinned back, and took off his jacket.

That’s Bernie Siegel’s approach to life in general, and the Summit in particular. Inclusion is key to the efforts of the Executive Director of Genetics Policy Institute; he makes everyone welcome at the World Stem Cell Summit. It looks easy when he does it, but he works all year to make these three days welcoming and successful.

This is the premier event of our field, of the movement to empower patients, patient-advocates, scientists, scholars, lawyers and lawmakers—working together for cure.

More than “just” our bodies’ health; biomed is the economy’s well-being, and the two intertwine —as one panel showed—“Stem Cells and Regenerative Medicine as an Engine for Economic Growth”—in Ballroom B.

In that one panel were: Jonathan Thomas, chairman of the Board of the California stem cell program, Frank Stonebanks, of the Ontario Institute for Cancer Research, Glyn Stacey of the UK Stem Cell Bank; and Elona Baum, of the California Institute for Regenerative Medicine– you could build a convention around just those four!

As Gloria Reed, my wife of 43 years said to me: “We really needed another day to see it all!” (not to mention a separate suitcase to pack all the free stuff).

Walking up and down the aisles of the new industry was a burst of adrenaline—here were people staking their business lives on the success of the battle against cronic disease. When scientists succeed in a lab, the next step may be a new business to bring that success to patients in need. Little surprises everywhere—free totebags, quality publications, even a jar of gray and green candies labeled with an alleged joke: “We just hired a molecular biologist. Man, is he small!” Next door to the candy source, biotech’s AKRONBIO, was Liebert Publications, medical reporting, probably the most widely read such publications in the world.

Half an auditorium was devoted to new posters of research from all around the planet.

Everywhere was synergy, everyone benefiting from everybody else.

For me the highlight was Bob Klein’s speech on new ways to fund regenerative medicine. Bob is the inspirational leader of Prop 71, which became the $6 billion California stem cell program, and his keynote address was so jam-packed I will do a separate article on it.

Issues, arguments, and controversies were aired.

For instance, which is more promising, human embryonic stem cells (HESC) or Induced Pluripotent Stem Cells (IPSC)? One is made from throwaway blastocysts left over from the In Vitro Fertilization procedure; the other is made by reprogramming some of the patients’ own cells. The Religious Right tries to shoot down one by advancing the other—but is that the best way to find cures?

Anyone who attended stem cell pioneer Jeanne Loring’s panel saw presentations on the strengths of both approaches, each with value, neither excluding the other. And where did the panelists come from? Loring– Scripps Research Institute; Gary Smith, the University of Michigan; Outi Hovatta, Karolinska Institute, and Timothy J. Kamp of the University of Wisconsin Medical School—internationally esteemed institutions.

(My non-scientist’s opinion is that embryonic will be more powerful—and faster and cheaper—for the purpose of transplanting body tissues and organs; but the reprogrammed cells will be invaluable for drug-testing and disease models.)

A controversial panel inveighed on “Unproven Stem Cell Therapies and Deceptive Claims: hidden dangers to Patients”: stem cell tourism. A desperate patient might consider going to a small country where there are few if any medical regulations. There the owners might open your body and put in something, maybe not even stem cells. One “stem cell clinic” did spinal cord surgery with the patients lying on none-too-clean-looking beds, like a third-rate hotel!

Ann Tsukamoto of Stem Cells Inc., moderated that panel, ably assisted by Doug Sipp, Riken Center for Developmental Biology, James Guest, illustrious spinal cord expert from U of M—and Allan Wu of the Morrow Institute.

Wu brought the house down with a gross-out story: a would-be stem cell tourist kept saying a potential transplant was guaranteed harmless because it was autologous— taken from the patient’s own body—“It’s autologous, it’s autologous!”, he said.

Finally Dr. Wu said, “I could put a STOOL SAMPLE into your brain, and that would be autologous too—but would that make it safe?”

Experts were everywhere, on the platform and in the audience: 175 speakers and attendees from 41 nations.

The dangerous lawsuit, Sherley v. Sebelius, was updated by Attorney Beth E. Roxland. Ms. Roxland performed the seemingly impossible task of making the case interesting. Sherlev v. Sebelius is for me the very essence of a frivolous lawsuit. Two religious-minded researchers are suing America, trying to shut down government support of embryonic stem cell research, because they claim it is economic competition for them and their preferred method of adult stem cell research.

Attorney Roxland concluded by saying the court case will either go forward to the United States Supreme Court—or not—before January 4th. The articulate Roxland has a deep interest in the legal/ethical aspects of the biomedical field, AND she can describe it in exciting, understandable terms—can somebody please hurry up and offer her a job in California? (I’m serious! Contact her at http://www.linkedin.com/in/bethroxland for more info.)

The National Institutes of Health, the crown jewels of American medical research, sent Joshua Hunsberger to describe the NIH’s new Center for Regenerative Medicine.

The University of Miami Miller School of Medicine was ably represented by Dr. Dalton Dietrich of the Miami Project to Cure Paralysis, who made an impassioned defense of Schwann cells, as a way to alleviate spinal cord injury paralysis. Dr. Dietrich announced they had commenced a Stage One (safety) clinical trial.

And for those of us who view government as a necessary part of the solution, Bernie Siegel chaired the panel “Stem Cell Politics: the Post-Election Advocacy Agenda”.

(CIRM’s own Kevin McCormack did an excellent piece on the panel, seeable at: http://cirmresearch.blogspot.com/2012/12/if-they-feel-heat-they-see-light-role.html

As Bernie put it, “stem cells are not a Democrat or Republican issue, they are a human issue, and we need to put aside political partisan issues and work together on this.”

Amy Comstock Rick, chair of the Coalition for the Advancement of Medical Research, discussed why Congress is not immediately going forward with another Stem Cell Research Enhancement Act, like those which passed Congress in 2005 and 2007 before being vetoed by then-President George Bush. In her view, the Senate and House won’t push legislation until they see how Sherley v. Sebelius pans out. Ms. Rick is a fighter par excellence both for CAMR and Parkinson’s Action Network.

Paul Knoepfler, America’s only blogging stem cell scientist took the audience on a leaping tour through vital national science issues: because National Institutes of Health (NIH) funding is level (static) or down, some 90% of grants are not getting funded—and embryonic stem cell research funding is still decreasing, for political reasons, not scientific. Paul has a white paper on stem cells that has been translated into 22 languages….

Then it was my turn, to speak on state issues– a thousand things I wanted to say, and ten minutes to say them in.

Scientists need three things: freedom, funding, and friends.

Freedom: people in this audience had been part of the epic struggles for research freedom in Michigan (Proposal 2) and Missouri (Amendment 2); there we battled for the rights of research, and won. In California, we passed the first stem cell freedom bill in the country, AB 253 (Ortiz) to officially legalize embryonic stem cell research—but the freedom to research came with no money.

Funding: only a handful of states have stem cell research funding programs now—New Jersey, California, Maryland, New York, Connecticut and Illinois—pathetically few, compared to the need. Every state in the country should have a biomed funding program—do we not all struggle with chronic disease and disability?

And friends? I wish I could report that stem cell research was equally backed by both our major political parties. But sadly that is not the case right now.

If you want to really understand the difference between the two parties on this issue, look up the votes on HR 3, the Stem Cell Research Enhancement Act of 2005 and 2007. One statistic tells it all. In the House of Representatives there were 16 Democratic opponents–and 158 Republicans who voted against the research—almost ten times the opposition.

One of our Summit attendees was new Representative Patrick Murphy (D), who had run against incumbent Allen West (R). The Democrat supported advanced stem cell research; the Republican did not. But when the votes were counted (and re-counted) the supporter of stem cell research prevailed.

So much! At a special awards dinner, Florida’s own Sabrina Cohen received the GPI’s Stem Cell Action “Inspiration” Award. A very creative advocate, Sabrina sometimes has audiences remain motionless for sixty seconds, for a hint what it is like to be paralyzed.

Susan Solomon CEO of the New York Stem Cell Foundation received the Leadership Award for helping to “catapult NYSCF into a research juggernaut, raising approximately $100 million in the quest for cures”.

SIXTY MINUTES won the “Media Integrity” Award for their important TV series on stem cell rip-offs.

The hard-working Alliance for Regenerative Medicine (ARM) received the National Advocacy Award.

And the Nebraska Coalition for Lifesaving Cures won the Public Outreach and Education Award for “supporting the efforts…of Nebraska-based medical researchers, science educators, and young collegiate scientists.”—“GPI Stem Cell Action Award Honorees include CBS “60 Minutes and NYSCF’s Susan Solomon”, PR Newswire, 12/7/2012

As I walked backward out of the Grand Ballroom of the West Palm Beach Convention Center, dragged away by the remorseless schedule of the airplane ticket, the last thing I saw was an onscreen video of Christopher “Superman” Reeve talking about another wheelchair warrior, Danny Heumann, a motivational speaker. Patient advocates like Danny are the emotional muscle behind the biomedical revolution. The scientists are often reluctant to speak for themselves, and are sometimes hard to understand. Biomed businessfolk might be discounted because they are (as they should be!) selling something. But patient advocates embody the struggle for cure– and people listen.

Want to hear more? Check out the website, which includes keynote speeches (free!) from men and women who are changing the world through science for the public good.

http://www.worldstemcellsummit.com/

Above all, mark your calendar for next year.

In 2013, December 4-6, the World Stem Cell Summit will be held in San Diego, California, the heart of the biomed and stem cell research community. It will be an epic event, and you do not want to miss it.

It will be like touching tomorrow.

Disability Rights Treaty Blocked

http://www.huffingtonpost.com/don-c-reed/republicans-block-disability-rights_b_2246201.html

Breakthrough on Alzheimer’s? And a new threat…

BREAKTHROUGH  RESEARCH–AND A NEW THREAT    

By Don C. Reed

Two scientists’ beaming faces recently adorned a press release:

 ”New cell type developed for possible treatment of Alzheimer’s and other brain diseases”.–http://today.uci.edu/…

Why are they smiling– and what should we be worried about?

First, the good news. Using embryonic stem cells, California’s Edwin Monuki and Momoko Watanabe   may have found a way to answer Alzheimer’s disease. As you know, Alzheimer’s involves plaques and tangles in the brain, studded throughout the brain like raisins in oatmeal. If these plaques could be removed, it might help heal the devastating disease of memory loss, as well as perhaps easing Huntington’s disease and other neurological nightmares.

Alzheimer’s affects 4.5 million Americans today. A brutal condition, removing both memory and judgment from the sufferer, the financial costs are almost unbelievable, a staggering two hundred billion dollars a year… https://www.alz.org/…

How might cure work?

In a healthy brain, cerebrospinal fluid  (CSF) washes away the metabolic wastes which would otherwise become the plaques of Alzheimer’s; in addition, CSF “produce(s) many nutritive molecules…good things for the brain.”

And where does this cleansing and re-vivifying fluid come from?

   ”…the choroid plexus… tissue in the brain…produces cerebrospinal fluid (CSF).

And if the choroid plexus goes wrong?

“In Alzheimer’s… the choroid plexus (may) age prematurely, resulting in….decreased ability to flush out such debris as the plaque-forming proteins…of Alzheimer’s.”–personal communication, Ed Monuki -emonuki@uci.edu

To fix a non-working choroid plexus, it seems you need repair cells called the “choroid plexus epithelial cells” or CPECs.

“…For the first time we can use stem cells to…(make)… large amounts of (CPECs), … to treat neurodegenerative diseases,” said Monuki….

 ”These cells could be part of… disease treatments in at least three ways:

•    ….first… to flush out plaque-causing proteins from brain tissue, and limit disease progression;
•    Second, CPEC(s)….could transport therapeutic compounds to the…brain and spinal cord;
•    Third, the cells could be used to screen…drugs that….improve function.”

Dr. Monuki is a creative individual. He invented a new Petri dish on a grant from California’s Roman Reed Spinal Cord Injury Research Act, the law named after my paralyzed son. The Monuki petri dish can separate cells by their electrical potential, a job presently done by an expensive machine so big you have to rent it.

Ms. Watanabe is a graduate student with what would have to be described as an amazing future. The University of California at Irvine, and the Sue and Bill Gross Stem Cell
Research Center should take great pride in their accomplishments.

All good so far?

Now the problem: the project was only possible because of government funding: vital to scientists as air is to a deep-sea diver. Without grants, no work can be done.

Unfortunately, the threat of massive cuts hangs over the National Institutes of Health–America’s crown jewel of medical research.

It would be a tragedy if right now, just when we are starting to break through toward therapies for cures, that we should be blocked for lack of funds.

Want to help?

Here is Mary Wooley, the CEO of the non-partisan group Research!America with some thoughts on what you can do.  

“Research could be put on hold if we the stakeholder community allow ourselves to be ignored or taken for granted by a lame-duck congress as it wrestles with averting the fiscal cliff.  Massive cuts to federal research agencies could have devastating consequences.

“Accordingly, this week we began a “Save the Research!” campaign here in Washington with many partners in the science and patient community.

“We need cures not cuts! We are funding ads, and making visits, 60 visits yesterday.

“Research is a bi-partisan priority, but it is just not brought to the forefront sufficiently. Now is the time to do it.

“Visit our website and find out how you can make the difference.  

“http://www.saveresearch.org/

“The single most important thing you can do? Contact your own elected officials.

“Visit the website and sign on to the group letter of support, and do an easily clickable outreach to your elected officials.”  Mary Wooley, personal communication. (for verification contact  info@researchamerica.org
For media queries, contact Suzanne Ffolkes, 571.482.2710)

Think of it this way: if just one chronic disease costs America $200 billion, what is the price of all them put together?

I can’t even count that high.

But the Kaiser Medical Foundation estimates that in 2010, total health care cost America $2.3 trillion–of which 75% went to chronic (incurable) disease.

http://www.kaiseredu.org/…

We can either pony up the funds to pay for research–or watch our country go broke trying to pay impossible mountains  of medical cost.

Our choice, as Shakespeare said, (is) “to endure the slings and arrows of outrageous fortune, or to take arms against a sea of troubles, and by opposing, end them.”

Cure, or endure–and I am sick of enduring. My son Roman Reed has been paralyzed 18 years, since a football accident in 1994. I want to see him walk again, and close his fingers, and enjoy all the small luxuries of motion we take for granted.

The money Washington allocates to research funding will affect not only Roman’s fate, but that of approximately 109 million Americans with chronic conditions.– http://www.milkeninstitute.org/…

Don’t let a lame duck session of Congress do damage to our hopes for cure.