Wednesday, September 30, 2015

Connecting With the Public and Stem Cell Stakeholders: A New and Inexpensive Tool

For years the California stem cell agency has given the public access to the meetings of its governing board via a one-way audiocast over the Internet and by phone.

But patients, advocates, scientists and business folks have not had the opportunity to comment and make suggestions re the doings of the 29-member board of directors through the same method. The public could only listen unless they were on the scene.

However, that could change if the $3 billion stem cell enterprise follows the lead of the state Treasurer John Chiang. This summer he began opening up meetings of the many important boards in his office to live, two-way, teleconference connections with the public.

Chiang also uses the same sort of service and company as used by the stem cell agency. But he has added an inexpensive touch that genuinely opens up the meetings to the public.

We recently stumbled across Chiang's press release on the move, which may well be unique in California state government. We queried Drew Mendelson, a spokesman in the treasurer’s office, for more details. 

Mendelson said,
“Participants on the phone are in listen only mode, but when the meeting chair calls for public comments, callers can press a set of numbers to indicate they want to speak.  The conference call is monitored online by (treasurer’s) staff and the call monitor must click on the caller in the queue to allow the person to speak.”
 Mendelson continued,
“Under our state contract with AT&T, we determined that an hour long meeting with 10 callers would cost less than $15.  The cost increases significantly if you have AT&T provide conference monitoring services.  We chose to monitor the calls ourselves.  What we are using is a basic conference call service with an online component where the monitor sees the list of callers to know how many people are on the line and when someone indicates they want to speak.”
Mendelson said that the move was generated by Chiang's desire to enhance public access and his “desire to increase public participation in and increase awareness of the many boards, commissions and authorities" that he chairs.

On-the-scene public participation in CIRM meetings is slim. One of the reasons is that the sessions often require travel and overnight stays that pose barriers for many persons, particularly patients and their advocates. The agency would make it easier for its stakeholders and enhance its reputation for openness and transparency by instituting this inexpensive practice. It also fits with the goal of Randy Mills, the president of the agency, to provide more clarity in what the agency does. 
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Tuesday, September 29, 2015

Oversight Panel of California Stem Cell Agency Schedules First Meeting in 21 Months

The only governmental body specifically charged with oversight of the $3 billion California stem cell agency, an enterprise that operates beyond normal state controls, announced yesterday that it would meet in just three days.

The session of the oversight panel comes 21 months after the last meeting of the Citizens Financial and Accountability Oversight Committee(CFOAC). The panel is required by state law to meet annually. Its last meeting in January 2014 saw the agency criticized harshly by one of the members of the oversight panel.

The committee is chaired by the state controller, who currently is Betty Yee. At the time of the last meeting, the controller was John Chiang, who is now state treasurer.

The agenda for this Thursday's meeting contains no items that would seem to be controversial, only a review of a routine audit and a presentation by the agency itself.

In January of 2014, Jim Lott, one of the members of CFAOC, made it clear at some length that he was not pleased with the agency's performance. He said in part:
“What can we say we've done to advance to a cure or to cures? It's fine that we've got all -- we've contributed to all. What can you say that we've actually done? We don't really have any -- I'm going to just say this because it's a bias and I know it's a bias. We don't have any tangible specific and measurable results that I can point to.”
The $3 billion state stem cell agency is exempt from the usual state budgetary controls. It receives its funds directly from bond proceeds without intervention by the governor or the legislature. The agency’s independence was authorized by voters when they created the agency in 2004 through a ballot initiative that altered the state constitution.

Two sites are available for the public to observe and participate. One is in Emeryville in the San Francisco area and the other is in Los Angles. Addresses can be found on the agenda. The public can also listen to an audiocast of the proceedings. Directions are also on the agenda. 



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Friday, September 25, 2015

Building a 'Beautiful Machine:' California's Last $900 Million for Stem Cell Research

Intense, passionate, zealous – some of the words that could have applied yesterday to the CEO of a California enterprise devoted to the dispassionate world of scientific research.  

Randy Mills, president of $3 billion California stem cell agency, started with what could have been a mind-numbing overview of a revision of the agency’s strategic plan – a subject that draws yawns from many.
Randy Mills at an earlier strategic plan event in La Jolla.
San Diego UT photo

Plus he was not talking directly to his key audience – the directors of the governing board of the stem cell agency, who were listening to him from 18 different teleconference locations.

Mills eased into his presentation, backed by 30 pages of PowerPoint slides. He was speaking by phone from room 3803 in the Sanford Consortium in La Jolla, a facility partially financed with $43 million in agency funds.

His pace and fire escalated as talked about transforming the agency and fabricating a “great, beautiful machine.”

He told the directors,
“We have created very beautiful pieces – but they have existed as pieces”
Now is the time, he said, for California to create a “giant engine that will accelerate the research in a way that exists nowhere else in the world”  – a “giant coordinated stem cell machine.”

Mills said the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, is “by far the largest regenerative institute in the world.”  At the same time, it “needs to be better appreciated.” He promised to generate a “dramatic increase” in awareness of the agency.

Mills, who has been with the agency for about 16 months, skimmed over the numbers: $2 billion out the door, $900 million left for research awards, $400 million to go to clinical research, 50 new clinical trials.

Mills has already increased the speed of funding for clinical programs, dropping the time from years to months. But he wants to do more for what is called the translational stage – bringing developments out of  basic research into clinical phases in three years instead of eight.

Mills’ efforts yesterday were limited by time, however. The meeting was scheduled for only 90 minutes and the board had other business as well.

Individual board members had brief comments, including Jeff Sheehy, a communications manager from UC San Francisco. He raised questions about Mills’ plan to clear out obstacles at the FDA. Sheehy wanted to know more about how much it would cost and how it would be pursued.

Another asked whether there was a sufficient research base in California to achieve all that Mills proposed, a risk that Mills had identified in his PowerPoint slides. Yes, Mills said, it could be that CIRM will not be able to do 50 new clinical trials.

Mills’ report was a midpoint update on how the agency plans to spend its final $900 million in what could well be its last strategic plan.

The proposal will be examined once again by the Science Subcommittee of the agency’s governing board in November before it goes to the full board in December for approval.

The plan is likely to have a major impact on stem cell research in California and on the lives of hundreds of researchers. Only one scientist was present to comment yesterday at the meeting, Jeanne Loring of the Scripps Institute. She told Mills that his presentation was “terrific,” which sparked applause from about 20 patient advocates in the room at the Sanford Consortium.

As for the fate of the agency after 2020, when funds for new awards run out, CIRM Chairman Jonathan Thomas said that would be discussed at the December board meeting.

But as Mills noted, 
“We don’t have a lot of time left.” 
The agency is open for more comment from the public. Mills said suggestions, criticism, etc., can be sent to kmccormack@cirm.ca.gov.

More information about the strategic direction of the agency can be found here and here.

Those directly affected by CIRM spending – or who want to be affected by it -- would be well advised to listen to Mills’ presentation on a recording of the teleconference meeting. The recording can be found on the audiocast link on the meeting agenda, beginning at about 38 minutes into the meeting.
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Thursday, September 24, 2015

Stock of California Firm Jumps 17 Percent on News of Golden State Award

Google chart
ImmunoCellular Therapeutics’ stock price shot up 17 percent today in the wake of formal approval of a $20 million award from the California stem cell agency for a clinical trial involving a rare brain cancer.

The price has been climbing since last week when the California Stem Cell Report first disclosed that the company was set to secure the funding for the phase three clinical trial, the last step before widespread commercialization.

In an interview today, Andrew Gengos, president of the Calabasas, Ca., firm, confirmed that the trial is expected to cost $40 million to $50 million. The company has said it will add $34.4 million to the $20 million from the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

Gengos said the five-year trial is expected to get underway in November with the enrollment of the first patients. He said it will probably start in the United States with expansion to Canada and Europe. About 400 patients are expected to participate.

ImmunoCellular has not had any revenues since 2010. Asked about the source of the $34 million, Gengos said the company had $31 million on hand at the end of June with no debt. He added that the company can access additional cash through the sale of more stock.

In reponse to a question, Gengos said he first became aware of the possibility of funding the clinical trial with the help of CIRM as the result of a conversation with a colleague at the J.P. Morgan health care conference in San Francisco last January.

Gengos followed up by asking an ImmunoCellular co-worker to look into the process. As the result, the company made its initial application which CIRM sent back with suggestions for improvement. In the second review, reviewers voted 9-0 to fund the project.

Gengos told the CIRM board today that the trial would take five years to complete.
“Frankly, this time period is outside the interest of most public market investors in terms of their investment horizon and therefore, in their eyes, handicaps our project compared to other projects that can execute in a shorter time frame.
“The result is that investment capital is hard to come by for these types of promising and highly innovative therapies when the investment horizon is long, and a small company without product revenues is at the helm.”
Gengos continued,
“I do not think it is an overstatement to say that without CIRM’s support, this program would not go forward. California’s innovative biotechnology community needs institutions like CIRM. Clearly – we need CIRM. Brain cancer patients need CIRM.”
Here is the text of the company’s press release today.

Here is the text of Gengos statement to the CIRM board.
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Coming Up

We expect to post a piece tomorrow on the impassioned presentation today by Randy Mills, CEO of the California stem cell agency, of his proposed strategic plan for the $3 billion research engterprise. He envisions a "great machine" that will set a new standard for the world stem cell community. Look for the article tomorrow. Sphere: Related Content

Faster Action Approved in California on Stem Cell Therapy for Parkinson's

To cheers and applause, directors of the $3 billion California stem cell agency this morning moved to speed action on a much-lobbied effort to fund research on a therapy for Parkinson's disease.

Randy Mills, president of the agency, said the action would mean that a request for applications would be posted within the next seven days instead of sometime next spring.

He spoke from the Sanford Consortium in La Jolla during a teleconference meeting with 18 locations. Parkinson's advocates at his site cheered and applauded the news.

A giddy Jenifer Raub, one of the leaders of the Summit4StemCells group, told agency directors,
"I feel like a kid at Christmas."
It was a feeling echoed by other members of the group, which had protested the delay at a CIRM board meeting in July and also earlier this month. About 17 were in attendance at Mills' location this morning.

The San Diego Parkinson's group is backing research by Jeanne Loring, head of stem cell research at the Scripps Institute in La Jolla. (See here and here.)

The unanimous vote by the board gave Mills more flexibility in scheduling upcoming rounds of awards involving basic and translational research. Loring's research fits in the translational area. The basic research round will now apparently be put off until next spring. Sphere: Related Content

California Stem Cell Meeting Concludes: More Upcoming on Parkinson's Research, Creation of a 'Stem Cell Machine' and ImmunoCellular

Directors of the California stem cell agency concluded their meeting today at 11:43 a.m. They gave the go-ahead to speedier action on funding for Parkinson's disease research and heard an impassioned presentation from President Randy Mills on his plan to create a "great machine" that will "change regenerative medicine in the world." We will have more on both subjects later along with an interview with the president of ImmunoCellular Therapeutics, which received a $20 million award today. Sphere: Related Content

California Stem Cell Agency Backs Calabasas Firm with $20 Million in Search for Brain Cancer Therapy

Directors of the California stem cell agency today unanimously approved a $20 million award to help boost into the marketplace a therapy for a rare brain cancer known as glioblastoma.

The funds will go to ImmunoCellular Ltd, a tiny, publicly traded firm based in the Los Angeles suburb of Calabasas, which has close ties to Cedars-Sinai Medical Center.

Andrew Gengos, president of the firm, told the directors,
"Without (the agency's) support this program would not go forward. Brain cancer patients need CIRM."
It is the second, phase three clinical trial for the agency, formally known as the California Institute for Regenerative Medicine (CIRM). The $3 billion research enterprise is pushing to develop a commercial therapy before funds for new awards run out in about four years.  (See here for the CIRM  press release on the award.)

CIRM is also proposing to award $400 million over the next four years to support clinical level research and participate in 65 clinical trials.

The ImmunoCellular trial is expected to produce preliminary results by the end of 2019.  The trial will involve 414 patients and  begin recruiting in November of this year. (See here for recruitment and other information.)

Despite some caveats, the agency’s blue ribbon reviewers concluded that the proposed therapy “could become a new standard of care and extend survival of glioblastoma patients.” Other firms, however, are competing in the field, the reviewers noted. Earlier this year, reviewers approved the award during closed door proceedings on a 9-0 vote, an action that virtually assured formal approval today.

Immunocellular’s closing stock price has risen 13 percent since the California Stem Cell Report disclosed on Sept. 17 that the firm was set to receive the $20 million award. Today, the stock opened at 48 cents. At the time of this writing today it stood at 52 cents.

Information presented to the CIRM board today did not indicate potential cost of therapy to consumers. Nor did the information discuss the financial condition of the company other than to state that it would provide $34.4 million in co-funding. There was little discussion this morning of the application by the CIRM board. 

The company’s filings with the Security and Exchange Commission show that the firm has reported no revenues since 2010 and a loss of $9.4 million in 2014. It reported that it had $23.2 million in cash or cash equivalents in 2014. In February of this year, it reported raising $14.6 million from Roth Capital Partners, LLC.

Sabby Healthcare Master Fund, Ltd. and Sabby Volatility Warrant Master Fund, Ltd., both registered in the Cayman Islands, hold 9.22 percent of the company’s stock, according to company documents.

Capital Ventures International, another Cayman Island firm, owns 5.54 percent. John S. Yu, chairman and chief scientific officer of the ImmunoCellular and a neurosurgeon at Cedars-Sinai, owns 6.65 percent. The CIRM governing board has one member from Cedars-Sinai who was not permitted to vote on the award.

ImmunoCellular has six fulltime employees, Gengos said today.

The public summary of the review said,
“In the initial review of this application, reviewers were enthusiastic as glioblastoma represents a serious unmet medical need. Additionally, the applicant presented strong preclinical and clinical data to support the proposed Ph3 registration trial and there was a reasonable plan to obtain regulatory approval of the proposed therapeutic. However, reviewers had feasibility concerns regarding the applicant’s ability to enroll the trial and maintain the reagent supply chain to support manufacturing of the product.

“Reviewers also had concerns with the trial design, which centered around selection of the patient population targeted in the registration trial and the lack of immune monitoring proposed in the trial. The applicant was provided the opportunity to address these concerns in a revised application, and their responses and modifications to the proposal reassured reviewers that the applicant could enroll and conduct the Ph3 trial as proposed and that, if endpoints are met, the trial design could support licensing approval by FDA.”
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New Reviewers Appointed by California Stem Cell Agency

The governing board of the California stem cell agency this morning approved the consent calendar on the meeting's agenda. Those are matters that have no controversy. Today they included appointments of new scientific grant reviewers. Sphere: Related Content

Directors of California Stem Cell Agency Open Meeting

Today's meeting of the California stem agency began this morning at 10:04 a.m. PDT with the pledge of allegiance and a roll call. Sphere: Related Content

Wednesday, September 23, 2015

A $6 Billion Question: Affordability of California's Stem Cell Therapies

Imagine this headline popping up on the Internet and in the New York Times in about four years:
“$6 Billion for ‘Price Gouging' Stem Cell Therapy” 
The possibility of such a news story is not all that fanciful -- given the growing tension and anger over exorbitant drug prices being set by a variety of Big Pharma firms. The likelihood of such a report and its impact is a matter for careful consideration by the 29 directors of the $3 billion California stem cell agency.

They are presiding over an effort that will cost the Golden State’s taxpayers roughly $6 billion by 2020.  Nominally, the agency is giving out $3 billion in research awards. However, the cost is approximately doubled because the state is borrowing money to finance the research, a move approved by voters in 2004.

No therapies for widespread use have yet emerged from the agency, formally known as the California Institute for Regenerative Medicine (CIRM). But the agency is pushing hard. It could have a therapy on the market within four years. The question is whether it will be something that is perceived as affordable or something available only to the wealthy few.

CIRM is now expecting to be involved in 65 clinical trials by 2020. One of those trials is currently in phase three, which puts the proposed treatment on the cusp of commercialization.

A second phase three trial – with a $20 million taxpayer investment -- is all but certain to be approved tomorrow during the CIRM directors meeting. The session also includes a strategic discussion about how the agency will spend its last $890 million – for basic research, clinical trials, infrastructure and so forth.

Not on the table, however, is what those therapies are likely to cost consumers. The spending plan does not address the question of whether a publicly financed agency should back development of therapies that could cost, for example, $512,000. (See here and here.) Or whether it should focus on more affordable treatments.

The issue of high cost of drug therapies has roiled the medical community and the public for some years. The matter has now moved to a higher level and is not likely to vanish during the next five years, as it has in the past. It is tied to the income inequality debate and the upsurge in patient advocacy. (See here and here.)

The issue surfaced this week in the presidential campaign. Democratic aspirant Hillary Clinton used words like “gouging” and “outrageous,” causing biotech stock prices to drop. Republican presidential candidates used the problem to attack Obamacare. Folks in the scientific community said they were “shocked.” 

This afternoon, a Google search on the term “high drug prices” turned up 33.7 million results. “Outrage,” “huge spike,” “4,000 percent” and “insanely high” were some of the words in the headlines.

The affordability question is not new to the California stem cell agency. It popped up in the ballot campaign that created the agency in 2004 but has largely died down in recent years. (See here, here and here.)

It could surface again in the California legislature and elsewhere as lawmakers see a public desire to curb perceived greedy pharmaceutical companies that prey on the weak and afflicted.

It is not a prospect that would be pleasant for the California stem cell agency. And it would put a major damper on efforts to continue funding of the agency from both public and private sources.

As they consider their strategic direction, the CIRM directors of the agency may be well advised to move earlier rather than later on the question of high prices and affordability. As Ben Franklin’s medical homily goes,
"An ounce of prevention is worth a pound of cure."
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Dismayed San Diego Parkinson's Group Receives More Media Coverage

Jenifer Raub, Bill Maddox and Cassandra Peters (left to right),
members of the Summit4StemCells group. San Diego UT photo
The San Diego Union Tribune this week spotlighted a Parkinson’s patient group that is pushing the $3 billion California agency to speed up financing on research that the group says is promising.

Reporter Bradley Fikes provided the update on the activities of the group, Summit4StemCells, and its dismay about the slow funding by the stem cell agency.

Earlier this month, the group was perturbed by the cancellation of the scheduled, face-to-face meeting in San Diego tomorrow. It was changed to a teleconference meeting that is set for only 90 minutes. However, the group is expected to make its views known by teleconference from the Sanford Consortium in La Jolla, where agency President Randy Mills will be based.

The face-to-face session was cancelled, the agency said, because it was expected to be brief and because of the expense involved in bringing together the 29 members of the governing board and some agency staff.  Mills told Fikes that many of the board members would not have been able to attend in San Diego, but the meeting would have cost about $30,000 to stage. 
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Tune in Here Tomorrow for All the Stem Cell Action in California

The California Stem Cell Report will provide most of what you need to know live from tomorrow’s meeting of California’s stem cell research agency, which is set to award $20 million to a tiny Calabasas, Ca.,firm.

During the 90-minute, teleconference session, the agency’s governing board is also set to discuss the pros and cons of a $890 million spending plan that will take the agency to its termination date.

The California Stem Cell Report will monitor the session via the Internet and bring readers updates and more as warranted by action during the session.

Those interested in participating in the meeting and offering suggestions and criticism can do so from 18 locations throughout the state, but also in Atlanta. Here are the other teleconference sites and number of sites in each location: San Franisco (3), Los Angeles (3), La Jolla (2), Stanford, Davis, Napa, South San Francisco, Sacramento, Fresno, Berkeley, Los Gatos and Irvine. Specific addresses can be found on the agenda.

Randy Mills, president of the agency, will be addressing the board from La Jolla at the Sanford Consortium, an enterprise heavily financed by the stem cell agency.

For those who want to listen in via the audiocast, directions are also on the agenda. The PowerPoint presentations being used are available via Webex. To get something close to a complete understanding of what is going on, readers should log into both the audiocast and Webex. Comments during the meeting can also be emailed to the board via mbonneville@cirm.ca.gov or kmccormack@cirm.ca.gov.

A reminder: Agenda items are not necessarily taken in numerical order. 
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Tuesday, September 22, 2015

Nearly $1 Billion California Stem Cell Plan: 50 Clinical Trials, Therapy Cost to Consumers Not Addressed

The CIRM view of the dynamics of stem cell research over the next five years
Highlights
$575 million for clinical, translational
FDA big impediment
Patients want greater voice
Therapy cost to consumers not mentioned

The California stem cell agency will spend the bulk of its remaining $890 million for more advanced research, including as many as 50 new clinical trials, according to a plan released today.

Randy Mills, president of the $3 billion state research effort, is expected to discuss the proposal Thursday during a teleconference meeting of the agency’s governing board. The public can participate from 18 locations, including one in Atlanta.

The figures represent projected spending by CIRM.
Mills expects to spend $400 million on clinical ventures and about $175 million on translational research, the effort to transform basic discoveries into potential clinical use. Basic research would receive about $180 million between now and 2020. That’s when money for new awards would run out under Mills’ plan, which did not address the fate of the agency beyond that date.

The proposal was unveiled in part this week as a discussion item on the agenda for a meeting that comes in less than two days. The information was offered in a 30-page PowerPoint presentation with no supporting documents.

The plan also offered an overview of past activities of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.

Mills indicated that he had received input from 217 “stakeholders” as part of developing the proposal. The number referred to the persons who responded anonymously to an online survey. 


One of his conclusions from the input: Patient advocates “want a more active role.” He also reported, 
“Many of CIRM’s programs are still not widely known to all potential partners.”
Seventy percent of the stakeholders viewed the FDA “as the biggest impediment to the development of stem cell therapies.” Mills said his plan would “drive implementation of a new FDA paradigm.”

Mills also identified five key risks. They were:
  • “There may be an insufficient number of meritorious therapeutic candidates to reach our goals.
  • “There may be insufficient interest from qualified applicants to participate in key competitions.
  • “The current limited funding of the agency could affect the ability to retain or attract personnel.
  • “Investors may be uninterested in stem cell therapies.
  • “The FDA may be unwilling to improve the regulatory environment. 
Some key parts of Mills’ strategic plan are well underway, such as the start of CIRM 2.0, the name for the effort to step up the funding pace for clinical awards.  At Mills’ request this summer, the board allocated $100 million this fiscal year for clinical work. He is proposing $85 million for next year.

By the end of this week, the agency will be involved in 15 clinical trials. The goal of taking part in 50 new trials does not include those efforts.

Basic research (termed discovery by CIRM) is scheduled for $15 million this year and $50 million in both 2017 and 2018.

Missing from Mills’ presentation is a discussion of the cost to consumers of potential therapies backed by the agency or whether the agency might want to focus on less costly efforts. That question was an early one for the agency. In 2006, the Consumer Watchdog group said,
 “Cures and treatments must be priced so all Californians can afford and benefit from them, not just a wealthy few.”
The issue is back in the news again with national stories highlighting huge drug costs and price increases. Reuters reported today,
“Americans are paying way over the odds for some modern cancer drugs, with pharmaceutical companies charging up to 600 times what the medicines cost to make, according to an independent academic study.” 
Democratic presidential candidate Hillary Clinton, who supports CIRM efforts, said this week she intended to eliminate “excessive profiteering” in the drug industry, triggering a sell-off in drug stocks.

Another strategic issue involves whether the agency should make choices between research that directly involves only a few thousand people versus research that would directly affect millions. Also missing is an overview of the agency’s move away from research involving human embryonic stem cells(hESC), the controversial human tissue that is freighted with moral and ethical questions. As of February, the agency reported that about 240 of its 667 awards involved hESC. 

The desire to fund research involving human embryonic stem cells was the motivating force behind creation of the agency in 2004 through a ballot initiative. The $36 million campaign used former President Bush's restrictions on federal funding for hESC research to build support. Bush's action had stirred an outcry among scientists and many patient advocates. Biotech companies also shied away from publicly backing hESC research because of the controversial issues.

Mills’ strategic plan is well along the way to completion and may well be approved by the board later this year with few significant changes. The public can weigh in, however, with comments on the proposal during the Thursday meeting from the 18 teleconference locations.  The addresses can be found on the agenda.

Comments, criticism and suggestions for the CIRM board can be emailed to Kmccormack@cirm.ca.gov.

The strategic plan revision is set for discussion only this week. Presumably board approval could occur at meetings in October, November or December, the next non-teleconference session.

(Editor's note: An earlier version of this item said 20 teleconference locations were available.)
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Sunday, September 20, 2015

Parkinson's and the California Stem Cell Agency: Advocates Cite 'Overwhelming' Need to Air Unhappiness

The California stem cell agency has posted a letter online from Parkinson’s patient advocates that says that an emotional outpouring at a July stem cell meeting grew out of an “overwhelming” need to protest agency delays in funding research.

The letter was signed by Sherrie Gould and Jenifer Raub, leaders of Summit4StemCell, a San Diego group, and was sent to the agency's board. Gould and Raub wrote about the July 23 meeting of the board during which the patients protested after they learned that no funding for the research they were backing would be available until about 2017. (See here for full text of the protest.)

The link to the Aug. 2 letter is part of the agenda for this Thursday’s teleconference meeting of the directors of the $3 billion research effort. It said, 
“Our group at Summit4StemCell (has) been regularly attending the CIRM meetings for the past 18 months. Out of respect for Jeanne Loring PhD, we have typically shown up in ‘silent solidarity’ but this meeting was different and many of us had an opportunity to speak publicly about our project, our progress and our desperate need for funding.
“The outpouring on Thursday (July 23) was spontaneous and unexpected. We at Summit feel strongly that we communicate with all board members that our presence at any of the CIRM board meeting is not orchestrated by Jeanne Loring, Dr. Melissa Houser (of the Scripps Clinic) or anyone from the Loring lab.
“We are a passionate group of dedicated Californians that believe strongly in the probability of success of this research using pluripotent stem cell technology. Dr. Loring has always counseled respect and brevity. We believe she would prefer that we be the face of the issue and not the strident voice.
“When confronted with the possibility that there could be no help (or hope) for over a year, the need to speak and be heard was overwhelming. We sincerely believe that you and the board heard us and will do all you can to find an exception or some way to help.” 
The reference to Loring, who is head of the stem cell program at the Scripps Research Institute in La Jolla, has to do with the Summit group’s support of her research. It also seems to be linked to an anonymous comment filed on the California Stem Cell Report’s account of the July protest.

The writer indicated that Loring had submitted an application earlier that was scored unfavorably by the agency, based on a conversation that the writer had overheard at the Oakland meeting. In response, Loring said no such conversation took place. She said an earlier application in another round did not meet CIRM criteria and was withdrawn. The agency does not publicly comment on such grant review processes, which are conducted in secret. It also does not release the names of unsuccessful applicants. (See Loring's and the writer's complete comments at the end of this item.)

Summit4StemCell is also not happy with teleconference nature of this week’s meeting. Earlier this month, they said their voice was “diminished” because of the inability to speak to the governing board face-to-face.

The California Stem Cell Report item on the teleconference/Parkinson’s flap is the most widely read post this month, exceeding the readership of the item dealing with the agency’s announcement concerning its $32 million stem cell bank. 
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Text of Parkinson's Protest at July Meeting of California Stem Cell Board

Here is the text of remarks made by Parkinson’s patients at the July 23 meeting of the governing board of the California stem cell agency. We should note that the words do not convey the impassioned feelings that were expressed. The comments come from the agency’s transcript of the meeting.
I'm Ron Evans. I'm an ophthalmologist from Placerville, and my wife has Parkinson’s disease. Specifically I was noticing in an earlier talk, I think Dr. Mills presented some of the funding schedules, and for 20 -- well, for this year and up to, I think, 2016, they had zero dollars slated to fund translational projects. Is there a reason why there's a delay in that and not moving of that $40 million forward? 
(Randy) Mills (president of the stem cell agency, CIRM): Yes. So the funding for the $40 million here is on a -- starting as soon as it's approved by the board today. The numbers I put up on that slide were for the fiscal year 2016. So our fiscal year started July 1st and will end June 30th of next year. Between now and June 30th of next year, we will -- this program will be started, but we won't be through an entire cycle. Meaning we have to issue the program announcement, then there has to be time to complete the applications, and the applications come in and they go through the review process, and then after the review process, they come back to the board and the board approves them. We will not have gone through all of that cycle yet by June 30th. So the actual first awards that will come out of this will be in fiscal year 2017.
Evans: so essentially there has to be a certain legal process that it goes through before the funds are available or procedural if legal is not a good term.
Mills: Correct.
Evans: And maybe an amendment someone might even think of is the president, if he sees an exceptional project, could seek to sidestep that or make an exception. Thank you.
(Agency) Chairman Jonathan Thomas: other comments from members of the public? Please state your name.
Roos: My name is Ben Roos. I'm a young onset Parkinson’s at age 31. I think I want to see some action. I understand the procedures and policies, but this is a billion dollars of money that I voted for and the public voted for, and I’d like to see translation put into effect ASAP. And I’ve waited 15 years. I'm tired of waiting. This is a golden opportunity. I think you guys should -- the people at Scripps (Research Institute) here have a program which is under way. Ten million, for example, would help them get into the next stage of translation. And I would give it to them this year, not in 2016. Also I would urge my fellow Parkies to speak up. This translation thing is where the rubber hits the road. So we've all come a long way. Speak up.
(applause.)
Hoover: Chris Hoover. I'm a Parkinson's patient. I've addressed the board twice before, but the last one was with Dr. (Jeanne) Loring (head of the Scripps stem cell program) at the Claremont (Hotel), and we were the only ones there because There was a rainstorm of the year. We call that divine intervention. But I think what I want to do is summarize. We kind of feel like we're a round peg in a square hole. We came to you and we had our discovery stage pretty much done by ourselves. We've raised the money, we've done the research, and we’ve got the science. So clearly we're in the translational stage, and that's where we're at. They suggested that we pull our old application because where we're at didn't fit the old CIRM and that it would fit the new CIRM 2.0 better. I was here almost every meeting, and CIRM talked about doing a 60-day turnaround on applications under the 2.0. So I’m not hearing anything about that now. We're talking now June of the following year. And that's kind of -- we've been waiting a while here. We've attended every meeting. So I’m wondering if there's any way, when you approve the translational, that we can speed that process up. And my question is what happened to the 60-day turnaround that you talked about initially with the 2.0 presentation.
Chairman Thomas: Dr. Mills.
Mills: So the 60-day funding decision is for the clinical stage programs, which includes the last part of translational and the enabling Studies that are done. There would be no way to do it for the high volume applications though.
Hoover: Where do we fit in your process because we didn't to come to you in the discovery stage? We're where we are now. Our science is done. We're need funding to get FDA approval. I guess my question to the board is: Is there any way to accelerate the process? Then you'd have us out of your hair. Thank you.
Chairman Thomas: Mr. (Jeff) Sheehy(a patient advocate, CIRM board member).
Sheehy: I think that's a question for Dr. Loring. I'm not sure where you would be in the science in terms -- we had our train analogy. I don’t know. I don't know what pieces need to be done and how that fits.
Hoover: I guess what I meant was when can we get our application in? We just want our application in to get reviewed. That's what we're looking to do. And we've been at this for about a year. Thanks.
Loring: So, Jeff, what's the question?
Sheehy: You don't qualify for the preclinical that's currently open?
Loring: It's not open now. There isn’t another round open right now. I'm sorry. I'm confused. There's no RFA (request for application) that I know of that is open right now that would be appropriate for us. If there is, I’ll apply it.
Sheehy: So there's not an RFA. So you’re really in the middle of the translation stage.
Loring: That's right. We are. We call it preclinical, but it really is translation as you define it.
Chairman Thomas: additional public comment?
Raub: I’m Ray Raub. My wife has Parkinson's, and we've been involved with Dr. (Melissa) Houser and Sherrie (Gould). At any rate, here's where we are. And if there's an exception that can be made, here's why we need it. We have scraped together almost $2 million. We've done it ourselves. We've done it on the backs of our friends. We don't have a lot of those left. They don't answer the phone anymore when we call because they know why we're calling. But we've done this and we've gotten awfully far. We've accelerated what we're doing. We're trying to get through the FDA. We've got functioning sets of dopaminergic neurons. We're ready to put them into a lot of the people that you've heard from today. We won't be able to do it without your help. We're tapped. And we've accelerated what we’ve been trying to get through the FDA. We've probably got about three months more of funding left. We need help, and we can't wait till the end of the fiscal year 2016, till next year. We'll find a way to do it. If you can't help, we'll find a way somehow. Anything you can do. And as I said, if there was ever a situation when exception was warranted, I swear this is it. Thanks.
(Marie) Duliege (CIRM board member): I just want to clarify. First of all, I just want to say how moved we are with all of your coming and your testimony about what you are going through and what you've done on your side to accelerate the process. But I want just to clarify one simple thing that I didn't get clear answer. Is there currently an opportunity to apply for a grant for the research that Dr. Loring is trying to do, or is there not? It's a very simple answer. In which court is the ball?
Mills: For translational activities, that's what we're voting on now.
Duliege: I’d like for everybody. Is there an opportunity for Dr. Loring to apply for a grant for the research that she's trying to do?
Mills: If we approve this, then there will be a program announcement issued, and that's what this funding is for.
Duliege: Thank you very much.
Chairman Thomas: Additional comments from members of the public?
Radunsky: My name is Mike Radunsky, and I’m involved in Dr. Loring's program. You've heard from me several times. And I feel a little bit like the counsel members' statements are a way to say, well, we gave you a chance to apply, but you’re telling us that we have to wait another year before the funding comes in. That's what we saw, a big zero in the 2016 funding. And there were three digits in all the columns for everything else. And the fact that it's going to take nine months to review a grant that we've been discussing with you for a long period of time is a little bit onerous. I think, again, as Ray Raub asked, if there's ever a time for an exception, this is it. I have two small children that I’m taking care of with Parkinson’s disease. And if I don't find something that's going to help me with this, I don't know if I'm going to get to the end of this without severe financial hardship. And I’m extremely lucky to be involved in this program, but I would like to ask you guys to help me stay lucky by getting there, Helping us get the money faster so that I can continue to work and supply for my family.
Chairman Thomas: additional comments from members of the public?
Raub : Hello. My name is Jenifer Raub, and my husband is going to make me cry. I just ask you to please speed this up. Tell us when we can apply, not September.
Chairman Thomas: Dr. Mills.
Mills: For the scheduling, so everyone knows, the concept plan that's before you for translation has the first application deadline, meaning the application would have to be in, is in March, which means the application would post this year. There would be time to obviously be responsive to the application and the application available in March.
Chairman Thomas: any other comments from members of the public? 
Raub: Only me. My name is Jenifer Raub, and that's outrageous.
Burkes: My name is Dennis Burkes. I'm a patient, Parkinson’s patient. And this reminds me of the old saying, "Water, water everywhere and not a drop to drink." Down in San Diego we sit amongst other research institutes that are sitting on hundreds of millions of dollars. Guess what they're doing? They’re trying to figure out how they're going to dispense that money, and they’re just dragging that out and dragging that out. And it's so frustrating to all of us. If there's something wrong with the science here, if you know something or some reason this shouldn't proceed, then tell us. Don't let bureaucracy and policy and procedure stop this project from moving forward and taking another year because we're going to run out of money very soon. Ray is right. Maybe we can come up with more money, but we're not the most well-heeled group. And our list in our Rolodex has been used up. And it's going to be tough. And we've hired new people, we have new researchers at work now, and it would just be tragic to see that come to an end with all the funds that are there and with such a promising project as this one. So I just ask that you please take that into consideration. Thank you.
Chairman Thomas: Other comments from members of the public?
Roos: This -- I don't know which of you this is addressed to, but why was there zero dollars in this year's budget for translation?
Chairman Thomas: Dr. Mills.
Mills: So the schedule was set up because we were going to be launching all of these initiatives, not just the translational phase. So we have multiple discovery reviews to do, we have the translational review to do, we have the educational reviews to do, and we have all the clinical reviews to do. So we just have slots that we can have these things fall into. The reason that March was picked as the first application deadline for the translation phase was because we just did an award review in February.  It was the most recent award that we had. We hadn't done a discovery award in a while. So when we were ordering them between discovery and translation, because we had just completed the translational phase award in February, we slotted discovery first.
Roos: And the follow-up question is why can't you approve a project like this now? Is this procedure really necessary, to wait a year?
Mills: I would just – James (Harrison) is legal counsel here.
Harrison: So Prop. 71 (the ballot measure that created the agency) requires that we go through a competitive selection process which entails review by the grants working group. And backing up, that involves releasing a program announcement, which will be done expeditiously assuming the board approves the concept proposal. We then need time to process the applications, Schedule a GWG (grant review group) meeting for the review, and then bring those recommendations to the board for its final consideration.
Roos: You guys (unintelligible). Can you do it in three months? I just don't understand why it has to wait a year. Thank you.
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Thursday, September 17, 2015

Brain Cancer Targeted: Calabasas Biotech Firm Set to Win $20 Million in California Stem Cell Funding

John S. Yu, chairman, ImmunoCellular -- Cedars-Sinai video 
Highlights
Second CIRM bet on phase three trial
$34 million in co-funding
Application initially rejected

California’s stem cell agency next week is expected to award $20 million to a tiny California firm to help finance a phase three clinical trial of a therapy for a rare brain cancer.

The move would be the $3 billion agency’s second investment in a phase three clinical trial, the last major hurdle before a therapy can be offered for widespread public use. The first was an $18 million bet this spring on NeoStem, Inc., now known as Caladrius.

The two trials offer the best likelihood of fulfilling -- in relatively short order -- the 2004 campaign promises made to voters when they created the Golden State's stem cell research effort. 

The firm involved is ImmunoCellular Therapeutics, Ltd., a publicly traded enterprise headquartered in Calabasas, Ca., a suburb northwest of Los Angeles.  Calabasas is better known for its entertainment personalities, such as the Kardashian family, than biotech.

In addition to the state funds, the trial would be backed by $34.4 million in co-funding, according to the California Institute for Regenerative Medicine(CIRM), as the agency is formally known. Earlier this year, ImmunoCellular estimated the total cost of the trial could be about $50 million.

The firm's proposed therapy targets glioblastoma, a fast-moving cancer that CIRM said is “resistant to current standards of care."  It has a survival rate of slightly more than one year. Immunocellular said that about 10,000 new patients are diagnosed each year in the United States.

The treatment is called ICT-107. Immunocellular reported on its Web site that the treatment is “an autologous (patient-derived) dendritic cell immunotherapeutic that targets six different antigens (peptides that are tumor markers) associated with glioblastoma.”

The CIRM summary of reviewer comments said that the phase one trial for the treatment produced a “tremendous survival response.” The summary said,
“Though the survival response observed in the Ph1(phase one) trial was not fully replicated in the Ph2 trial, results are supportive of continued clinical development and of the Ph3 trial as designed.”
ImmunoCellular has been around with its current focus since 2006 when it acquired its cellular-based technology from Cedars-Sinai in Los Angeles. John S. Yu, director of surgical neuro-oncology at Cedars, is listed as the founder of the firm. He is now chairman of the board and chief scientific officer.

The firm has four full-time employees and four part-time, including Yu, according to February 2015 filings with the Securities and Exchange Commission. The firm said,
“We have a number of consulting agreements for clinical development, regulatory affairs, investor relations and business development. We outsource all of our drug discovery research, process development, manufacturing and clinical development to third parties with expertise in those areas."
ImmunoCellular reports no revenues for the past several years. CIRM said, however, the firm has promised $34.4 million in co-funding for the clinical trial.

In an August closed-door session, CIRM’s scientific reviewers scored the ImmunoCellular application as having “exceptional merit” on a 9-0 vote. 

Ratification of that decision will occur in public next Thursday in a teleconference meeting of the agency’s 29-member governing board, which includes one representative from Cedars-Sinai.

The board almost never rejects a positive recommendation from its reviewers. The agency also does not release the names of successful applicants in advance of a board vote. The California Stem Cell Report identified ImmunoCellular through other means.

The firm's original application was deemed by CIRM reviewers to need improvement on a 1-9-2 vote and was sent back to ImmunoCellular for revision. The August review summary said,
“(R)eviewers had feasibility concerns regarding the applicant’s ability to enroll the trial and maintain the reagent supply chain to support manufacturing of the product. Reviewers also had concerns with the trial design, which centered around selection of the patient population targeted in the registration trial and the lack of immune monitoring proposed in the trial.
“The applicant was provided the opportunity to address these concerns in a revised application, and their responses and modifications to the proposal reassured reviewers that the applicant could enroll and conduct the Ph3 trial as proposed and that, if endpoints are met, the trial design could support licensing approval by FDA.”
The price of ImmunoCellular stock closed at $0.425 today, up nearly one cent. The 52-week high of the stock was $1.03 and the low 35 cents.
Google chart
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California's $3 Billion Stem Cell Agency Releases More Information for Next Week's Meeting

The California stem cell agency yesterday made public significant background information concerning its board meeting one week from today, including an application for a $20 million clinical trial award.

Release of the information is a step forward in openness and transparency for the agency, which has slipped in that area recently. (See here and here.)

The $20 million award is the only application up for approval next Thursday, just four business days away. The California Stem Cell Report will have more on the proposal later today.

Items with fresh, additional information include new conflict of interest standards for the agency’s blue-ribbon grant reviewers, changes in the scheduling of upcoming rounds for basic and translational research and changes in loan award policies.

Still missing is information involving the agency’s plans to correct deficiencies identified in a $230,000 performance audit and the agency’s ongoing revision of its plans for spending its last $1 billion.

Next Thursday’s meeting will be a teleconference session with 17 remote locations, including Atlanta, where the public can participate and listen in.  All are places from which board members log in for the meeting. The locations include San Francisco (3 locations), Fresno, Davis, Los Angeles(3 locations), Napa, Stanford, Sacramento, Davis, Fresno, Berkeley, Los Gatos, Irvine and La Jolla (2 locations). Specific addresses can be found on the agenda.

The meeting will also be audiocast on the Internet, but no public participation is available by that means.

Comments to the board can be sent in advance of the meeting to mbonneville@cirm.ca.gov.
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Tuesday, September 15, 2015

California's Stem Cell Agency Meets This Month to Award Millions, Discuss Performance Improvements

This month’s meeting of the governing board of the $3 billion California stem cell agency, already the subject of some unhappiness from patient advocates, will be staged from 12 different teleconference locations on Sept. 24.

All but one site is in California. One board member, Lauren Miller, will be logging in from Atlanta for what is expected to be about an hour-long meeting.

This month’s session was originally scheduled for San Diego, where board members and staff would have been available for face-to-face discussions with the public. But the San Francisco-based agency switched the meeting to a teleconference session because of estimates that it would last only an hour.

The move upset Parkinson’s patient advocates in San Diego, who planned to attend the session in their area to speak about the need for funding for research into a possible cure for the affliction. They already have protested delays in funding at the July meeting of the board in Oakland.

On this month’s agenda are mostly routine matters considered earlier by board subcommittees that are expected to be quickly approved. Also on the agenda is consideration of presumably multi-million dollar applications for clinical stage research proposals. The board nearly invariably rubber-stamps the actions of its blue-ribbon panel of application reviewers. No information is yet available on those applications.

Up for discussion is a plan to correct unspecified deficiencies identified in a $230,000 performance audit of the agency.  The study, commissioned by the agency and required by law, said the agency needed to improve its procedures involving conflicts of interest and grant application reviewers. Also identified as an area needing work is the tracking of potential royalties. No board action is expected to be necessary this month to implement the plan.

The agency’s president, Randy Mills, is additionally scheduled to give an update on the agency’s strategic plan, which is being reformulated. The board has roughly $1 billion left before its cash for new applications runs out in 2020.

No background information has yet been posted on the meeting agenda for any item to be discussed or acted on. The California Stem Cell Report will carry items on the material as it becomes available. If interested parties would like to submit comments to the board in advance of the meeting, they can be addressed to mbonneville@cirm.ca.gov.

The teleconference locations where the public can participate in the meeting and comment can be found on the agenda.  The session is scheduled to be audiocast on the Internet as well. Directions will be placed on the agenda. 
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Thursday, September 10, 2015

Parkinson's Advocates Dismayed by California Stem Cell Agency Action

This is a Summit4stemcell video prepared following an emotional protest at the July meeting of the board of  the California stem cell agency.

Highlights
Lack of speed, openness protested
Agency cites cost issues
Patient-CIRM relationships unique
A group of Parkinson’s patients from San Diego is less than pleased today with a move by the $3 billion California stem cell agency that the group says will stall efforts to secure much-needed funding for a possible cure for the disease. 

The events this week follow an emotional protest at an agency board meeting in Oakland in July and highlight the often intense relationship between patient advocates and the stem cell agency – not to mention the pressure on the agency to move fast to find cures.

Anger and the Parkinson's symptoms of trembling and speech difficulties marked the protests. One patient advocate, Jenifer Raub of San Diego, said it was "outrageous" that an award round being discussed at the meeting would not provide funding until possibly 2017.

The patient advocate group, Summit4stemcell, had planned to address the stem cell agency board again at a Sept. 24 meeting scheduled to be held in San Diego. However, yesterday the group was told that the meeting would not be held in their city. Instead the session was being turned into a teleconference meeting and would be only about one hour long. Most in-person board meetings last much of the day.

Responding to questions from the California Stem Cell Report, Sherrie Gould, executive director of the Summit group and a nurse practitioner, said in an email,
Sherrie Gould, Scripps Health photo
“We are both confused and disappointed that this PUBLIC (Gould’s capitalization) meeting has been essentially cancelled.  We were prepared to talk with the ICOC (the agency board) and answer all questions about our patient advocacy movement (Summit4stemcells) and our research….
“Unless we can be present in front of the CIRM board, Summit's strong voice is diminished.  Being physically present in human form is always more effective than a teleconference. Delays, which we realize are a part of any government associated agency, are devastating to people suffering from the progressive symptoms of Parkinson's disease.” 
Jeanne Loring, head of the Scripps stem cell program in La Jolla, also voiced her displeasure in an email to Kevin McCormack, senior director of communications for the California Institute for Regenerative Medicine(CIRM), as the agency is formally known.

She wrote,
“CIRM has rather pulled the rug out from under the patient advocates. These meetings are supposed to be public, so how is the public going to participate if there is no access?…. I'm disappointed and the Parkinson's patients are exceedingly disappointed.  The Parkinson's group is trying to find out the resolution, if any, of the timing of the grant applications.”
The Summit group has raised $2 million to support Loring’s research and to match what CIRM might award for her effort.

Asked for comment today, McCormack said in an email,
“The reason we switched from an in-person to a teleconference board meeting is simple, the agenda for this meeting consisted of just a few items that could probably be handled in one hour. We can’t justify the cost of an in-person meeting in San Diego – which includes transportation for all those going there, plus hotel rooms, meals and the cost of the ballroom for the day - for one hour’s worth of business. As a state agency we are always mindful of how we spend our money so we made the change.” 
The next in-person meeting of the agency board is not scheduled until Dec. 17. But, in addition to the Sept. 24 meeting, two more teleconference meetings are scheduled between now and Dec. 17. Both are expected to be brief, routine approval of awards, if they are held at all.

Under the agency’s procedures and state law, members of the public can listen in and speak during teleconference meetings but only from the specific location from which an individual board member is participating.  

On the other hand, the in-person meetings offer an opportunity to speak face-to-face with all board members, chat with them informally during breaks and to converse directly with CIRM staff. The meetings have been an important tool for many patient advocate groups to press their cases. At times, however, their lengthy presentations have tried the patience of the agency.

The relationship between patient advocates and CIRM is notable. One of the unique features of the agency is the influence of patient advocates, particularly on its board.  Twelve sit on the 29-member panel and often play important roles. Sometimes they are the only way the board can approve awards because most of the other members have legal conflicts of interest and cannot vote.

David Higgins of San Diego is one of the patient advocate members of the board, which has a
David Higgins
specific slot for a Parkinson’s disease advocate. He was interviewed in July following the protest of the many-months delay in possible funding for Parkinson’s research. His remarks were recorded on a Summit-produced video(see at top of this item). Higgins, who is a scientist and has Parkinson's, said,
“It’s tough, it’s really tough, to be involved in the Parkinson’s community, especially to have Parkinson’s disease, and to watch things go in what seems like slow motion.”
Contacted today for additional comments, Higgins said,
“In my year at CIRM I have only encountered enthusiasm and support for patient advocacy - both from the staff as well as the board. Patient and patient advocate participation in the process is welcomed. CIRM is first and foremost about patients.”
Following the protests at the July meeting, CIRM President Randy Mills told the board he would see if there is anything he could do to speed funding. He said he would report back to the board later.

McCormack did not directly respond to a question today about whether recent, proposed changes in the award scheduling would have an impact on the Parkinson’s research.  He said only that the changes would provide more flexibility.

(The text of remarks by the various parties follow this item.)
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