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: https://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.”