The disclosure involves Stanford University researcher Judith Shizuru, severely ill babies, toxic chemical treatments, delays in clinical trials and stem cell agency board members who are acutely aware of the $3 billion agency's rapidly dwindling resources.
Photo by Flynn Larsen, Ludwig Institute
The agenda nominally contains one item, an application by Shizuru for $6 million from CIRM. But the issues reach back to 2013 and provide insight into difficult research pathways and how the agency manages its programs.
At the top of the matter is the fact that the state agency expects to run out of cash for new awards by the end of this year. It is pinning its hopes on a proposed $5 billion bond measure on the November 2020 ballot. Meanwhile, directors are trying to raise privately $200 million to tide it over until then.
Avoiding Toxic ChemotherapyThat was the backdrop Wednesday Jan. 30 when directors convened to consider application number CLIN2-11431 by Shizuru for $6 million. She is seeking a way to avoid chemotherapy treatments and their toxic side effects in the case of a rare genetic affliction often referred to as the bubble baby syndrome.
In December, CIRM's grant reviewers, meeting behind closed doors, approved Shizuru's application, an action that nearly invariably is rubber-stamped by directors. But last month was different, and a bit of CIRM history was brought up.
Shizuru received a $19 million CIRM award in 2012 which has led to a phase one clinical trial with positive results for her therapy. Cost per each of the six patients so far averages $917,000, according to a CIRM document.
However, additional patients are needed, along with more funding, before the potential product can reach the marketplace. The average cost of per patient will run about $333,333 during the final portion of the phase one trial. Her latest application is aimed providing assistance with those costs.
Questions about Delays and Co-FundingLast month, questions arose among directors about months of delays in the clinical trial and a current shortfall in co-funding, among other things.
"It's not clear to me that they're being frugal with the money that they have been given."Juelsgaard also said,
"Delay doesn't necessarily add up to more money. They obviously spent the money on something that they didn't anticipate or under-budgeted or something. There's something more to it."Another director, Jeff Sheehy, chair of the board's Science Committee, said,
"Financially, it seems very muddy to me."Other directors weighed in as well, ultimately leading to a motion to delay action on the application to provide more time to find answers to questions.
At that point, Shizuru, who was in the audience, rose to respond.
"I understand CIRM's concerns, and I can see you're very thoughtful about how this money is being spent," she said, according to the transcript.
The Sheehy-Shizuru ExchangeThe Stanford researcher said that without additional funding the trial would have to suspend enrollment of additional patients, prompting this exchange between her and Sheehy.
"Shiruzu: Budgetarily we're better off using it (the remaining funding) to continue to follow the patients that we've already transplanted. From that budgetary standpoint, we should delay the trial. We should delay treating any more patients on the trial.
"Sheehy: So Stanford won't front you 1.6 million to (treat) patients if we don't give you the money today?
"Shiziru: I hesitate to say what they would do.
"Sheehy: To Stanford...they would actually put patients at risk?
"Shiziru: I'm not at liberty to say what Stanford would do."
"I want to acknowledge fellow board members (for) their continued concern about spending taxpayer's money wisely because I think this is a great example of that."
(The public can listen to and participate in the Thursday meeting via the Internet. Instructions are on the meeting agenda.)