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Archive for June, 2011

STEM CELL CHAMPION STEPS DOWN: new leadership for California program

By Don C. Reed

I am still a little in shock.

On June 23rd,  Bob Klein resigned the leadership of the California stem cell program—which he began. 

 

His initiative, Prop 71, the California Stem Cells for Research and Cures Act, became the California Institute for Regenerative Medicine (CIRM). In addition to having been the largest funder of the campaign (including taking out a loan on his house to help pay for it) Klein had led the oversight board as Chairperson for the first six and half years, most of that time without any salary at all. 

 

Now he was retiring from the leadership of that great agency.

 

Who would follow him?

 

Two superb individuals, Dr. Frank Litvack and Jonathan Thomas, were competing for the chairmanship. Either would be an outstanding leader for our group, but there was only one job to offer.

 

After studying their biographies, I contacted both men, who were kind enough to allow me to interview them at length, each for about ninety minutes. I asked them every question I could think of, brought up every objection that might be thrown against their candidacy. I also offered them both my best “free advice” (“Free advice costs nothing, and it’s worth the price”—Alan King) on how they could best present themselves to the 29-member governing board, which would decide.

 

Also, I personally had to decide. As a member of the public, I would be allowed three minutes to speak my opinion on who should lead.

 

I had no vote, of course. My opinion was important only to myself.

 

But (after my family) the CIRM is the most important thing in my life. I had fought to give it life, standing on wind-swept street corners asking for petition signatures, attended a gazillion meetings, editorialized thousands of pages, babbled endlessly to anyone who would listen.

 

And now there would be a new leader?  It would be wrong of me not to give my two cents’ worth on the most important decision for the program’s future.

 

It was actual pain, my stomach in knots for days, trying to decide which of the two men I would support.

I did not know which one I would back until the day before the meeting.  I studied each man’s position paper until I could have made his presentation for him.

 

When I finally made up my mind, I contacted each one, and told him. 

 

At the meeting, both men gave their speeches, which had been previously delivered at two subcommittees, so that everyone on the board had a chance to hear, and decide.

 

The two men were strikingly different in appearance.

 

Frank Litvack looks like a semi-retired weightlifter, white crewcut hair, very erect: he carries himself with genuine authority, a little intimidating.

 

Jonathan Thomas is slender, built for the bob and weave of basketball, darkhaired and cheerful, approachable, like someone you might ask directions from, if you were lost at the airport.

 

Their qualifications could not be more different. 

 

Frank Litvack is a heart doctor and a great medical entrepreneur, capable of recognizing an idea with potential and making it real; he developed biomedical companies, like the one which owned the medicated heart stent,  He could choose a stem cell therapy with the most potential, and drive it through to the patient’s bedside. A strong success like that might be our best hope to renew Prop 71.

 

Jonathan Thomas is a lawyer, investment counselor, and bond expert. He put together the funding for several multi-billion dollar government projects, like the highway connection for the Los Angeles harbor. If our funding source failed, he could use his experience to keep the CIRM alive; without funding, the best science in the world is meaningless.

 

Each man spoke for about 20 minutes.  Both men nailed their speech. Litvack  made me want to get up and do something to help the cause; Thomas gave me confidence that the financial battles could be won.

 

I remembered Henry Kissinger’s comment, that the most difficult decisions are always 51/49 percent.

 

And my friend Karen Miner’s. “If you are that torn, that conflicted, that means they are both good.”

 

Public comment was called for. I made the following statement:

 

“We are fortunate to have outstanding candidates for Chair. I contacted both, questioned them at length. 

 

“In personality, Frank Litvack is a strong leader, explosive, charismatic, fun to be around. His answers  to most questions were short, punchy, easy to follow.

 

“Jonathan Thomas on the other hand is quiet, not an exciting speaker. But he has a bulldog tenacity, which makes him unexpectedly persuasive. When I asked him a question, he would not only answer the question, but would come up with possible objections to his own answer, and answer those as well.  He would never be distracted from the issue at hand, but kept coming back to it again and again, until every objection had been completely met. He is focused, and determined, and he basically wore me down.

 

“Frank Litvack is a highly successful entrepreneur, the active chair of five companies. This is evidence of his success in the business world—but it may also be a possible negative. With the best will in the world, his  energies and commitments will necessarily be divided.

 

“Bob Klein also faced the problem of divided time; being chair of a real estate firm as well as leading the California  stem cell program. His answer was to routinely work to exhaustion, sometimes to the point where I feared for his health. That is not a tenable solution, especially when five companies are involved.

 

“Jonathan Thomas views the job of chair as a full time one, requiring his 100% effort and commitment, year round. That is to my mind the only realistic assessment of the job.

 

“Almost certainly, the new chair will face huge financial challenges to our program. I asked both candidates: if the worst happened, and there were no General Obligation Bonds, what will you do?

 

“Litvack said (and if I am mischaracterizing him I ask that he be allowed to correct me) without general obligation bonds, there was no way to fund the program at its current level, and anyone who said otherwise was blowing smoke.

 

“Asked the same question, Thomas said he would first implement a short-term plan to stop the bleeding, and then develop a long-term financial plan. He laid it out, in about a fifteen minute answer. I do not pretend to understand it, but it appeared to be a mix of bonds, donations from charities and foundations, revenues from biomed, national government contributions, and other sources of funding. He said that such fundraising challenges has been a major part of his life for the past thirty years, and he was prepared, if necessary, to do it every day of his tenure. He has shown the ability to raise massive amounts of funding: projects involving literally billions of dollars.

 

“In entrepreneurship, Frank Litvack is wonderfully successful, having developed for example a company which manufactured the medicated heart stent and other valuable products.

 

“But Jonathan Thomas has also demonstrated the ability to pick a winner in the biomedical world. Many years ago, when stem cell research as an industry was just getting started, he showed foresight by picking  just one company to support: Advanced Cell Technology.  Jonathan Thomas helped Mike West raise the funding he needed. From that humble beginning, the field of biomedicine began.

 

“In a time when complicated financing may mean success or failure for the California stem cell program—Jonathan Thomas’s unique skillset makes him the essential choice.

 

“As a patient advocate, I strongly recommend Jonathan Thomas for the Chairmanship of the Independent Citizens Oversight Committee of the California stem cell program. ”

 

The board went into closed session for three  hours; it seemed like days.

 

When they returned, the votes were counted.

 

By a narrow majority, 14-11,  Jonathan Thomas was elected chair of  the California stem cell program.

 

Bob congratulated Jonathan Thomas on “beginning a journey you will never forget”. The man who prefers to be called JT told a  self-deprecating  story about basketball great coach John Wooten, whose talentless son tried to play but mainly rode the bench—but was able to come in and help at an important moment. I don’t understand basketball, but his meaning was clear.

 

Old chair and new shook hands.

 

There was a long line of people who wanted to speak to Dr. Litvack afterward. Everyone knew this was a giant in the field.

 

I was nervous to approach him, but did anyway, and when it was my turn, I blurted out that I really respected his greatness and I hoped he would help us, because the program really needed him.

 

Fortunately, he was as large in spirit as he was in accomplishment, and was gracious. 

 

It is my hope that a way will be found to get him on the board, because his knowledge of making products succeed would be enormously valuable.

 

 

 

The next day was the official transfer of authority.

 

As one of his last acts in office, Bob asked that his wife Danielle be acknowledged. An advocate for many causes, and a strong force for positive change, Danielle is pretty like a movie star, and the love just shines  between the two of them.

 

It broke me down when Bob Klein read the swearing-in oath, and Jonathan Thomas raised his right hand and swore to uphold the Constitution of the United States and the State of California. I was typing, but I could not see the keys anymore.  My stomach was shaking, wracked with hopefully silent sobs—but nobody noticed, because every person in the room was having similar difficulties.

 

And then Bob gave his last official remarks.    

 

“It has been the privilege of my life working with this board and the staff of CIRM. We are on a mission for all our families, in our state, our country, and the world.

 

“With the tremendous outpouring of dedication and effort of all involved, including especially the patient advocate groups, I believe we will be successful beyond our wildest dreams.

 

“It is the dedication of the people in this room, the staff and the empowered scientists, who will see this dream of California through: to reduce human suffering.

 

“And so I thank you,” he said, and stopped.

 

The first action of the new leadership of Jonathan Thomas was an ICOC vote that Bob Klein would receive the title, “Chair Emeritus” , to honor his contribution forever.

 

On behalf of all who suffer, and their families, I would like to add my appreciation, with this small poem.

 

HOW DO YOU SAY “THANK YOU”– TO SOMEONE WHO CHANGED THE WORLD?

The California stem cell program has the glory of a flag unfurled.

But how do you say “Thank you!”—to someone who changed the world?

 

Like a farmer who plants a field in new ways, growing food from exhausted land,

You brought life to a new field of medicine, though others did not understand.

 

On a radio show once, an enemy of the research came on the air to insult you,

But you would not let the anger take over, talked to him as if he would consult with you;

 

Vilely, he compared you to a Nazi, Joseph Mengele, the angel of death,

But you said, “thank you for raising that important question”, without even pausing for breath;

 

You explained to him what the research really was, the hope of cure, dawn of new day,

And at the end the enemy said, “I never thought of it that way.”

 

But it is not enough to think anew, to have a little bit of hope,

Concrete relief for suffering is needed, like throwing a drowning man a rope.

 

You raised a billion for diabetes, long before Prop 71;

But three billion for stem cells? That will help everyone.

 

Many people gave their energies to Prop 71, nothing happens by itself,

But an idea without a leader– gathers dust upon the shelf.

 

You dreamed and designed it, raised money to help it grow;

Exhausting days of labor– no one would ever know;

 

The voters said yes, overwhelmingly, fifty-nine point six per cent,

California supported cure research, the message was plainly sent.

 

The battle was not over; obstacles confronted us, every day,

But every day the community fought back; our initiative was here to stay.

 

The magnificent board of governors, Independent Citizens Oversight Committee,

Struggles with near-impossible decisions, in the light of day, where all can see.

 

At the California Institute for Regenerative Medicine, narrow opening to the King Street address,

The staff of C.I.R.M. brings the program to life, quiet heroism, muscling toward success.

 

And twelve new buildings have sprung up, shining centers of tomorrow,

Where scientists work to save lives, ease suffering, and to diminish sorrow;

 

There is no way to thank you properly, for this shining Prop 71,

Except to pledge we will protect it, continuing what was so nobly begun.

 

You brought a state together, Bob, your voice an inspiring call;

The people in this room stand united—we love you, one and all.            –June 22, 2011

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

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CANADIAN  DELIGHTS—a Patient Advocate’s Perspective on the 9th Annual International Society of Stem Cell Research Conference

By Don C. Reed

“You’ve got to see Niagara Falls while you’re here,” said my flight mate, “It is gorgeous from the Canadian side.”

“I couldn’t without my wife Gloria,” I said, “It is on our must-do-together list—plus I will be too busy at the stem cell conference!”

“Stem cells? We’re going there too!”

It seemed like half the plane turned around: patient advocates and scientists, and everyone excited.

The sparkling clean city of Toronto, lovely mix of old and new, made us welcome. If you have ever been to Boston, with its blend of carefully preserved past and shining modernity, you would feel at home.

Canada’s magnificent newspaper, THE GLOBE AND MAIL, did an in-depth coverage of the International Society for Stem Cell Research
convention, including a full story on the opening of a website* about stem cell research.

Why? In the arena of international stem cell research, the country of the Maple Leaf is a major player. Canada understands. Maybe the rest
of the world was consumed with Stanley Cup hockey fever, but the Premiere of Ontario, Dalton McGuinty, was attending a press conference on regenerative medicine.

Of course, he had to acknowledge the game:

“Tonight we will see if Canada’s Canadian hockey players—are better than America’s Canadian hockey players,” said the Premiere at the press conference for the new website,

Stem Cell City, the electronic presence of the McEwen Center for Regenerative Medicine. (www.joinstemcellcity.com).

I was in the audience, one of the lucky patient advocates who had their way paid to the convention.  Not only did we get free admission (not cheap!) but the California Institute for Regenerative Medicine (CIRM) flew us up and even helped with the hotel bill!
Check out the CIRM website, http://www.cirm.ca.gov, and follow the action.

Premiere McGuinty spoke of Canada’s pioneering giants, James Edgar Till
and Earnest Armstrong McCulloch, first to prove the existence of stem cells. In
2001, Canada’s Stem Cell Network began, which has since trained over 1,300 new
stem cell scientists.   In 2007, California Governor Arnold Schwarzenegger, Bob
Klein of CIRM, and Premiere McGuinty met to create the international Cancer Stem
Cell Consortium. And in 2011, Dr. Mick Bhatia of McMaster University converted
skin cells directly into blood cells… Think what that miracle could mean, to the
always short supply of usable blood.

The ultimate patient advocate, Bob
Klein, author of Prop 71, received the first-ever ISSCR Public Service Award.
He took the opportunity to thank Canadian researchers for developing insulin–
which keeps his diabetic son Jordan alive.

The entire convention was like
that, surprises built on the hard work of researchers.

It was also (for me at least) a struggle to understand what the scientists were saying.

The purpose of the event was for scientists to share information
with each other and encourage the research of the world, so they talked pure
“science-talk” all the way, giant latin words, which is right and proper when
they chat among themselves.  Like auto-mechanics, they are masters of a world
the rest of us may never know.

I am no scientist, just an educated supporter, so everything I say here should be taken with a grain of salt.

My favorite presenter?  Shosei Yoshida of Japan, who spoke on the topic
of “Sperm Stem Cells in Mouse Testis”. This was not a subject to which I had
previously given much thought, and probably won’t in the future.

But if we had an impersonator, someone like Rich Little who could imitate movie stars,
Shosei Yoshida would be someone worth watching.  He was so happy and animated,
full of the joy of helping, as if he had won the lottery and was giving the
money to the world.

But a question dogged my mind. Why was this important?

And when another presenter, Johns Hopkins’ own Daniella
Drummond-Burbosa, spoke on: “Control of stem cells by diet and systemic factors
in the drosophila ovary”, I turned to the scientist beside me, and asked:

“Why does this matter? I mean—houseflies?”.

“For reprogramming cells,” she said, “A fly’s insides are easier to understand. If we go back to
the beginning stages of living tissue,” she said, “we can figure out how each step works.”

The whole conference was like that:  information overload,
confusion of new knowledge, and here and there a glimmer of understanding,
something shining and extraordinary.

As you know, my son Roman has been paralyzed seventeen years, and I hate paralysis with all my might. I wish it was
a person I could physically attack.

So imagine making a hollow spinal
cord column—and stuffing it with stem cells?
Robert S. Langer, MIT professor
with more than 1,100 published articles, and with approximately 760 issued and
pending patents, spoke of working with top embryonic stem cell scientists to do
just that.

It sounded impossible, but there it was on the screen before
us, and monkeys which had been paraplegic were galloping on a treadmill. There
were gasps in the audience.

Greatness of the future? Robert Blelloch
received the ISSCR Outstanding Young Investigator Award for his work on the
signals regulating both embryonic and induced Pluripotent stem cells.

A room full of junior researchers heard successful scientists talk about how to
get grants. Naturally I had to chime in about  the California stem cell program,
how every new scientist should make the CIRM website their homepage, so they
could find out what new grants were coming.

It irritated me that the California stem cell program was not systematically represented throughout the
program—but when I griped about it to someone from CIRM,  he pointed out that
the program was made by the ISSCR, and neither Canada nor California could
dictate to it. And, with the CIRM chair receiving the first ever ISSCR Public
Service Award, and a young investigator award going to a CIRM grantee (who
credited CIRM for his success), the agency was hardly invisible. (Also, CIRM
communications director Don Gibbons put on a public outreach program I did not
know about until later.)

In the elevator, naturally I was talking stem cells to everyone I met. You can say a lot between floors.

“Here for the stem cells?” I asked one distinguished-looking early-riser. He said yes, and we
shook hands.

“I’m Rudy Jaenisch,” he said.

“Whoa!”, I said, and jumped back a little—then I shook his hand again and thanked him for doing so
much to help the field. He was speaking on “Stem Cells, Pluripotency and Nuclear
Reprogramming..”

At the ‘MEET  THE EXPERTS LUNCH” you could sit with a
favorite scientist and eat a good basic box lunch and ask anything you wanted. I
got to sit with Margaret Goodell of Baylor College of Texas, and what a charming
person she was. In addition to being an expert on hematopoietic stem cells, she
was one of those people one has no choice but to like—so glad she is in our
field!

Irv Weissman  spoke of the dangers of “stem cell tourism”, where
patients went overseas and tried potentially unsafe procedures, spending their
life savings on something which might not help at all, and which might endanger
their lives.

Shinya Yamanaka! The famous Japanese scientist who came up
with induced Pluripotent Stem cells (iPS) (which may one day replace embryonic
stem cells, although I personally am unsure about that). He was ready to answer
several scientific articles, which had been critical of his new
method.

Everything boiled down to the comparison between embryonic to
iPS, and he talked about it for 26 minutes, give or take a paragraph. I wanted
to understand.

Having thoughtfully located a seat next to the microphone,
I was ready for the announcer to ask for questions. I also knew everybody in the
room would want to ask them as well. Halfway through the magic word “Question”,
I bounced up as from a trampoline, and was talking before the echo of the word.

“As a patient advocate, I want to understand—did you say there was a
difference in the—immunogenicity (I struggled with the word) —of iPS compared to
embryonic?”

And he said—something. I thanked him politely, and sat down
to think about what I thought I heard.

If I understood his remarks
correctly, he said there were differences, but they might be good ones as well
as “bad”.  An earlier slide had shown overlap between the two kinds of stem cell
research. Many embryonic stem cells are like many  iPS cells, he said, and vice
versa. A good difference was that the immune response might be less because the
cells were made from the patient’s own body. Also, cells of all kinds may
vary…

More research needs to be done, he said, comparing and
analyzing.

The man is honest. He defends his research, which is right and
proper, citing the studies that appeared to shine negatively on
it.

And he made everyone feel proud of Japan, which is hosting the
ISSCR meeting next year, despite the disaster which so riveted the
world.

“It is safe, it is beautiful– and you will like the food!” he
said, and the audience burst into applause.

There were endless choices to be made. Multiple “tracks” made possible an organized presentation of
various approaches.

Freda Miller: fighting paralysis with skin cells,
which may be turned someday into useful nerves.

Elly Tanaka: how a
salamander regenerates its severed spinal cord by first growing a living “tube”
for the cord to grow inside.

Amy Wagers of Harvard spoke about
regenerating muscle function for the aged—wow, that made me sit up and take
notice! I had always heard critics of the research say we were all hunting for
some fantasy fountain of youth, and this made no pretentions of being that—but
to my aged and aching muscles, it sounded very good indeed.

And speaking
of Harvard, Brock Reeve walked by.  Naturally I had to jump up and run over and
shake the hand of the director of Harvard’s stem cell program, and brother of
Christopher Reeve.

It felt like a Hall of Fame for research for
cure.

Fred Gage of Salk, Sally Temple of the New York Neural Stem Cell
Institute, Elaine Fuchs, chair of the ISSCR itself—each one of them deserving of
a book or two about their research–and the exhibit halls? Huge. I could have
spent a week in there, and never come to the end.Two phone book-sized volumes
gave brief descriptions of the thousands of posters.

I walked up to two
Chinese scientists, and asked them where the most stem cell research was in
China, and they said: “Beijing, Shanghai, and Guangdong”—we chatted a bit. I
tried out my baby-talk Mandarin on them, and they were polite enough to pretend
I was understandable—and it turned out one of them, Gang Li, was now in Mountain
View, California, and we had a mutual friend, Deepak Srivastava, who recently
published an article on turning heart scars into useful tissue, which may be of
enormous significance.

Hans Keirstead! The Canadian/California scientist
was his usual cheerful self, there for   another piece of groundbreaking
research. He, Tanya Watt and Gabriel Nistor were presenting work on: “Does human
motor neuron stem cell-derived neurotrophic support enhance the development of
the NMJ in Spinal Muscular Atrophy”—a stem cell therapy aimed at saving the
lives of children who may otherwise die before the age of two.

And over
there was Bernie Siegel of Genetics Policy Institute. He and Alan Fernandez are
working their heads off getting the World Stem Cell Summit together, and I
really did not think they would have the time to attend the ISSCR, but there
they were, maybe a little exhausted, but spreading the word and advancing the
field like they always do.

Don’t forget,  the World Stem Cell Summit will
be in Pasadena, California, this year, October 3-5—a must see for every patient
advocate! (If finances are a barrier, check with both Bernie Siegel of the
Summit, who has reduced rates for advocates—and also check out the CIRM website,
because they may help pay the way for some advocates!) The World Stem Cell
Summit is very different from the ISSCR conference, being designed to bring
together not only scientists and biomedical entrepreneurs, but above all the
advocates, the emotional muscle behind regenerative medicine.

Visiting
the GPI booth was Aaron Levine of Georgia Tech, expert on stem cell battles in
the states—great to say hello to friends you have only met on the email
circuit.

Lorraine Stiehl, patient advocate coordinator for the CIRM, told
me that Meri Firpo was going to be there.  Dr. Firpo has been working so hard to
keep Minnesota’s research freedoms alive. I had brought my stem cell phone book
along (I thought) and I wanted to sit down with her and see if we knew the same
people—but I brought the wrong phone book! Argh…

George Daley of
Boston’s Children’s Hospital, great scientist and communicator… he could be a
convention by himself! I tried to sit at his table during a meet the scientist
lunch, but the scheduler laughed—Daley’s  table was booked solid before the
conference began.

One huge overall impression? Despite all the talk about
new methods, embryonic stem cell research is increasingly the number one
research choice. I walked up and down the research poster aisles every day for
at least a couple hours, and it was astonishing to see how even in countries and
states officially against the research, there were people who were studying it
anyway.

And the two biggest names in non-embryonic stem cell research?
The world is abuzz with the iPS work of Shinya Yamanaka, just as it was with
adult stem cell research of Catherine Verfaille when it seemed she might have a
method which was just as good as embryonic—both of them had experiments underway
in embryonic stem cell research.

The hall of biomedicine in action was a
continual surprise—every time I walked down an aisle, there was something else
to explore, new and exciting.

Like Kowasaki, the famous Japanese
motorcycle company, now heavily into biomedicine.

Or Xyclone, with their
laser, which may help Somatic Cell Nuclear Transfer (SCNT, sometimes called
therapeutic cloning) become a practical reality.  So far, SCNT has endured
tsunamis of political attacks, but even when it was allowed, success seemed far
away. SCNT involves removing the nucleus of a woman’s egg, and putting a bit of
skin inside. Set in a dish of salt water, shock with electricity, wait 5 days and take it apart for stem cells. But—every time the needle goes into the egg, seems like it automatically collapses.  If one person’s cell lines takes several hundred eggs, it will never be practical. Human eggs are scarce and precious. So SCNT (though I support it) may never work.

Unless a way could be found to make an ultra-small hole in the egg—and that may be what the Xyclone laser can do.

Conversations and companies, theories and therapies, champions of the past and future.

Thank you, ISSCR for bringing together the greatest stem cell researchers in the world.

Thank you, CIRM, for making it possible for patient advocates to take a look into the future.

And thank you, Canada, stem cell champion, and our beloved neighbor to the North.

* “New website rallies stem cell advocates”, Carolyn Abraham, The Globe and Mail, June
16, 2011 (for more information, please visit: http://www.joinstemcellcity.com .

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CIRM SENDS PATIENT ADVOCATES TO INTERNATIONAL SOCIETY OF STEMCELL RESEARCHERS CONFERENCE!

By Don C. Reed

A few hours from now, I will sit in an astonishingly narrow airplane seat, and shove my briefcase full of reading materials under the seat in front of me. The jet will taxi around, and then—and then—the thrust of acceleration, the thrilling roar of leaving earth, bending the laws of gravity.

A few hours of travel and a passport check, and Canada will open its doors to another happy visitor.

In Toronto the stem cell conference awaits—three days with the world’s best stem cell scientists.

Once again, the California stem cell program is sending patient advocates to Canada for the International Society of Stem Cell Researchers meeting.  This is both a wonderful gift to the advocates who volunteered and were lucky enough to be chosen—and highly appropriate.

Patient advocates like my son Roman Reed are the emotional muscle behind research for cure. It is not easy for a paralyzed person to travel, but he will be there, listening and learning, and speaking.

Because there will be funding fights ahead, both nationally, and in every state.

When a politician says, “We can’t afford to fund the research”—someone must be there to say: “We cannot  afford NOT to fund the research—and here’s why.” That someone is us. If it is just the scientists talking, it is all too easy for politicians to ignore them.

Think of he numbers. There are only a handful of top research scientists. Politically, they are negligible. If they only talk to each other, they might as well pack up their test tubes and go home, because they will never get public funding.

But there are millions of patients and family– patient advocates. Working together, we cannot be ignored.

At my first scientific convention (a spinal cord injury conference at Asilomar, California), I asked so many questions they took away my microphone privileges! The man in charge said the meeting was for scientists only, so they could learn from each other.

Fortunately, that archaic attitude is no more. Scientists may learn best when they can shift into the experts-only mindset, just like auto mechanics or any other specialist—but for them and us to succeed, we must work together. Researchers want funding; we want them to have it. So we have to understand, at least a little, what they are trying to do.

The California Institute of Regenerative Medicine (CIRM, the California stem cell program) sprang from the citizen initiative Prop 71. Last year, the CIRM funded more stem cell research last year than any other organization on earth. And who made that funding possible?  Patient advocates, led by Bob Klein, patient advocate extraordinaire.

Personally, I am a dues-paying member of the ISSCR, chipping in at the non-scientist level, $70 a year.  (My wife Gloria does not know about this little expenditure, so please do not enlighten her.  Fortunately for me, she does not read all of my stem cell scribblings.)

For three days I will sit through lectures till my eyes glaze over, and then I will sit some more, taking notes to try and stay awake while giant-brained researchers explain in big words things I can never fully grasp, but must  understand—at least well enough to fight for funding.

I will be sitting near the microphone, having learned long ago that if you want to ask questions, you  need to be early in the line. Being at an age when everything reminds you of something else, I  invariably have a comment or a question, and if they do not unplug the mike I will be up there.

The ISSCR conference will be an adventure of the mind, as well as a chance to meet the champions.  We will make new friends and find ways to work together, on the universal problem of chronic disease.

Because it is not just America with one-third our population suffering incurable illness and/or disability.

Every country on earth faces such challenges, and no medical system can pay such extraordinary costs.

You have heard the statistics: how chronic disease cost America $1.65 trillion in 2009 (the most recent year I have statistics for) as much as the year’s installment of the national debt ($1.60 trillion). We spent more on caring for folks with an incurable disease than all federal income taxes ($1.2 trillion) combined.

We need an international effort to solve the epidemic of chronic disease.

The ISSCR conference brings the scientists together—and once again, thanks to the foresight of the CIRM, the patient advocates will be there too.

P.S. Are you going to be in SAN DIEGO on Wednesday the 22nd for the next big meeting of the California stem cell program?  The governing board of the program will be choosing the next Chairman. Bob Klein, the man who began Prop 71,  is retiring from the active leadership—who would you want to take his place?

Two outstanding candidates—Frank Litvak and Jonathan Thomas– have been nominated by the Governor of California, the Lieutenant Governor, the Treasurer and the Controller.

If you want to hear the candidates speak, and offer your opinion as to which one is more qualified,  please join us on Wednesday, June 22nd.  Go to www.cirm.ca.gov, and click on “MEETINGS” for details.

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CALIFORNIA VERSUS THE VALLEY OF DEATH

by Don C. Reed

There is a place you will not find on any map. It has no physical location. But it is real nonetheless, and a danger to all mankind.

It is the Valley of Death, where new medicines die.

May 4th, 2011. Gloria and I were sitting in the meeting hall of Covel Commons, the University of California at Los Angeles, (UCLA), attending a meeting of the California stem cell program. (Want to join us next time? Just go to www.cirm.ca.gov, click on “meetings” for details.)

The most important item on the agenda was a question: should the California stem cell program loan a company $25 million– to help with the enormous costs of human clinical trials? 

This was the Valley of Death, the series of tests every new medicine or treatment must undergo, before the Food and Drug Agency (FDA) approves it for public use. These tests (clinical trials) are staggeringly expensive. If the company runs out of money during the trials, the new product is gone. The Valley of Death claims another victim—a potential cure lost to the world.

PHONE CALL TO THE FDA

A Menlo Park company, Geron, Inc. was being considered for a $25 million loan to help with the stem cell human trials of its new product, GRN-OPC1, a proposed treatment for new paralysis.

The product was developed from Hans Keirstead’s work–  originally funded by the California law named after my paralyzed son, the Roman Reed Spinal Cord Injury Research Act of 1999.

Geron had bought the rights to Keirstead’s procedure. Since then the small California company had been in constant negotiations with the FDA, performing seemingly endless tests to meet their concerns. Every objection the FDA raised, Geron met. An astonishing 24,000 pages of correspondence had passed between them.

And this was before the clinical trials (with people) even began!

Was the delay… political?

I called the FDA to ask. (They have an ombudsman for public questions.)  He said he would get back to me. I figured  that was the brushoff, but it wasn’t.

A phone conference was set up with Dr. Steven Bauer, Chief of the Cellular Tissue and Therapy branch, who was in charge of the clinical trials. I had seventeen questions typed out waiting, and we talked for an hour.

At the end of the hour, I was convinced he was a scientist, not a politician. His decision to go slow and careful on the testing was from reasons of caution. 

But if my head was convinced, my heart was not. Delay is agonizing. A balance is needed, between rational caution and choking stagnation.

At some point we must say to the patient: here are the risks, it is up to you now. 

Geron’s courage and determination earned them permission to go ahead. Patients (two so far) have begun to receive GRN-OPC1, the actual product developed from the stem cells.

But this is no giant corporation with bottomless pockets.  If they went broke in the tests, all hopes were dashed. 

The valley of death was before us. I could almost see it, a shadowed abyss, full of monsters waiting to devour our hopes and dreams.

If we find no way to bridge this gap, the world will suffer.

A NERVOUS LUNCH, AND WHY

The board went into closed session. Every so often some of the information to be discussed was “proprietary”, meaning it might involve trade secrets, and so the members of the Independent Citizens Oversight Committee (ICOC) took their box lunches into another room. (If you are curious what the executives ate, I had one once, and it included a sandwich, apple, juice, chips and a cookie.)

 Gloria and I went out to lunch, at a little snack shop in hollering distance of the hall.    

I could not stop thinking about the loan—twenty-five million dollars…  But it came with the condition that Geron had to raise another twenty-five million on its own, so that the state would benefit doubly. All the money had to be spent in California, unless there was some rare supply item that could only be gotten somewhere else, and that would have to be justified. Every dollar spent meant the biomedical industry was advancing.

So much was involved…

In a way, the entire field of embryonic stem cell research depended on these trials. If they succeeded, the procedure might be applied to fighting many more diseases and disabilities for which there was no cure….

This could affect everybody in the world.

Chronic (incurable) disease is a gigantic drain not only on the economy of Earth. I don’t have planetary statistics, but I know about my country—and I suspect sick people are the same the world over. In the United States:

We spend more on chronic disease than on all federal income taxes. In 2009, chronic disease cost America $1.65 trillion compared to $1.2 trillion for all federal income taxes. 

Half of all bankruptcies and foreclosures are caused by inability to pay medical costs—and 75% of medical costs are due to chronic disease.

 Chronic illness now costs more than the national debt. (In 2009 the deficit was $1.60 trillion, less than the $1.65 trillion cost of chronic disease for that same time.)

 No medical program on earth can handle such costs; only cures can bring down the expense.

HOW WASHINGTON (AND ONE  CANCER SURVIVOR) MAY ANSWER

We need a federal program to help bridge the Valley of Death.

Fortunately, there is such a program—if we can get it funded.

It is called the Cures Acceleration Network, proposed by Senator Arlen Specter, a cancer survivor and a beloved friend of research for cure.

The Cures Acceleration Network is specifically designed to help cures survive the Valley of Death. It is an amendment to President Obama’s Patient Protection and Affordable Care Act.

A panel of experts would pick the most promising clinical trials for new cures or treatments, and recommend them for funding.

The Cures Acceleration Network was originally proposed as a $2 billion dollar program, then it was chopped to $500 million, then $100 million–  then suddenly the money for it was removed. 

We must not let it disappear.

Here is Amy Comstock Rick, CEO, Parkinson’s Action Network (PAN):

“PAN supports the goal of the Cures Acceleration Network and is hopeful that the President’s request of up to $100 million for FY 2012 is approved.  We are working to secure funding for CAN in the FY 2012 budget because we believe the government must do more to build a bridge across the scientific “Valley of Death”.…  Creating a funding source dedicated to accelerating the development of such cures and treatments will maximize the impact of both private and public funding, and will foster a flexible, expedited review process that will fund promising research as quickly as possible.”

Find out more about it at:  http://www.parkinsonsaction.org/federal-initiatives/national-institutes-health/cures-acceleration-network.

But for now, there was only the California stem cell program, and this moment, this decision.

If the Geron tests failed through lack of funding, other corporations would be discouraged. They would say, “Geron did everything right. They spent ten years cooperating with the FDA—and then they ran out of money in the clinical trials—why should we try the same thing?” 

I heard a noise—“They’re back!”– and was running.

BOB,  ALAN,  ELLEN—AND THE VOTE.

“I oppose this trial”, said one of the board members, “It is not the right time”.

What? Ten years of testing… and it was not time?

Chairman Bob Klein sat at the head table, leaning on his elbows. His eyes were shut.

He looked exhausted, and with good reason. In 2003 he began the campaign to pass Proposition 71, which in 2004 became the California stem cell program. Since then, he had led it.

In the next few weeks, his successor would be decided, and Bob would “retire” to private life. Bob’s idea of retirement may be different than most folks; I am guessing he will use the time to get ready for the greatest campaign for stem cell research ever—Proposition 71, part two…

The speaker went on for what seemed like eternity, maybe ten minutes. Bob did not move.   

I thought: please just be gathering your energies; you have to be at your best right now.

And then he spoke.

Usually Bob Klein says a lot in one burst, several subjects in one paragraph, so that I might be still thinking about subject number one while he has gone on to topics three or four.

But today his speech was short and clear.

The preliminary testing had been done in depth, he said. It was carried out with unprecedented thoroughness, and every conceivable objection had been met. 

President Alan Trounson spoke, saying that a decision to go with human trials was never absolutely clear-cut, black or white. It was always a balancing of the head and the heart, the desire to heal against the possibility of harm: risk and reward: and the hope of cure.

Ellen Feigal, new Vice President of Research and Development for CIRM, noted that moving into clinical trials meant considering safety for patients, the need for the intervention, the underlying science and data, the experience of the team in conducting first-in-human studies, and the development plan itself. At the end of the day, she said, it must be a carefully deliberative judgment call, based on experience and expertise.

The loan award would be disbursed in installments, not a single check.  There would be a plan to assess progress, based on a series of mutually acceptable milestones accomplished by Geron.

Several more scientists of CIRM spoke. All tried to stay objective, as was right and proper.

But I got the feeling they were internally leaning forward, holding their breaths, wanting this to go forward as much as I did.

Public comment was called for: Roman and I spoke, as can anyone who attends.

This was Proposition 71 in action, where everything came together. Here was why hundreds of patients and friends had stood on street corners, gathering 1.2 million petition signatures; why scientists labored day after day, why the staff of CIRM routinely put in unpaid overtime, everybody working toward exactly this moment: when patients could actually be helped.

Then it got quiet. The time for words was done.

On the audience chair next to mine, Jane Lebkowski sat, veteran Chief Scientist of Geron. Her normally cheerful face seemed chalk-pale, drained of blood.

“Melissa King, will you call the roll, please?” said the Chairman. One by one, she called the names.

“No…” said the speaker who had opposed the vote, and the word seemed to echo in the room.

But the next vote was yes. And then—yes—and yes—and yes and yes and yes– one after another, until every person in the room who was eligible to vote had registered their decision.

The vote was 16 “yes”, and 1 “no”.  Jane Lebkowski of Geron breathed again.

“Supporting the Geron trial is a landmark step for CIRM,” said Chairman Klein. “However, we must remember that there will be successes and interim failures as human trials proceed through the refinements necessary to achieve a successful human therapy. We need to be prepared to stand beside the heroic patients and the companies as they face these challenges and solve the problems that stand in the way of the recovery of patients from paralysis.

“When the people of California voted for   Proposition 71, they did so with the hope of seeing new therapies for disabling diseases like Alzheimer’s disease, Parkinson’s disease, diabetes and other chronic diseases and injuries.

“By funding this trial, CIRM is taking a major step toward making that hope a reality.”

 

 

 

 

 

 

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