Rogue Stem Cell Clinics and Covid: California Moves 'Sedately' on Regulation

The state of California and its top medical regulator remain mired in a go-slow posture on regulation of "snake oil" stem cell clinics that are currently riding the Covid crisis to peddle dubious treatments to desperate patients.

The marketing surge by the clinics has drawn increased attention nationally, including in prestigious scientific journals such as Cell Stem Cell whose usual fare deals with such things as "Stem Cell Hierarchy in Colorectal Cancer."

But one article published earlier this month was titled:

 "Preying on Public Fears and Anxieties in a Pandemic: Businesses Selling Unproven and Unlicensed 'Stem Cell Treatments' for COVID-19." 

The quotes around "stem cell treatments" are deliberate. No guarantee exists that the treatments actually include stem cells. 

Leigh Turner, an associate professor of bioethics at the University of Minnesota, authored the piece. He said,

"In the midst of a global public health emergency, some businesses are taking advantage of widespread fears by marketing purported stem cell treatments for Covid-19. 

"Such businesses target prospective clients with misleading claims, expose patients to potentially risky stem cell-based products, and undermine efforts to develop evidence-based treatments for Covid-19."

Nearly two years ago, California's State Medical Board said it was going to tackle the problem of the rogue clinics. But to this date little has occurred. State legislation to develop regulations was also sidelined.

The California Stem Cell Report queried the medical board earlier this month concerning the status of its effort and whether regulations had been drafted and when a draft would be ready. In response, the board last week released the following statement:

"The board is continuing to work toward the goal of providing recommendations on stem cell and regenerative therapies and developing some guidelines that California physicians and patients can follow that will include a sample informed consent document and educational materials for the public to present to the board for review and final approval. 

"In the meantime, pursuant to Business and Professions Code section 684, healthcare practitioners providing stem cell therapy not approved by the Food and Drug Administration (FDA) and who have not obtained approval or clearance for an investigational new drug or device exemption from the FDA for the use of human cells, tissues, or cellular or tissue-based products, must provide notice to their patients indicating the lack of FDA approval, and encouraging their patients to consult with their primary care physician before treatment. Individuals concerned about a physician’s stem cell practice should file a complaint with the Board for review and appropriate action."

The issues with the clinics has drawn attention in the Los Angeles Times from Pulitzer Prize winning columnist Michael Hiltzik  who wrote recently,

"The proliferation of stem cell clinics selling untested and unlicensed therapies has been a public health crisis for years. The Covid-19 pandemic will only deepen the crisis as clinics add the coronavirus to their menu of treatment claims."

The California stem cell agency has also pushed for tighter regulation and partnered to help pass the law for disclosure notices concerning lack of FDA approval. For patients considering stem cell treatments, the agency has posted as rundown on issues concerning the treatments or clinical trials. 

(After this item was posted, the agency posted a blog item dealing with Parkinson's. In addition to information on the search for a cure or treatment, the item carried the following advice for persons seeking some sort of unregulated treatment. Here is what the agency had to say in a Q&A format,

"If you go online you can find lots of stem cells clinics, all over the US, that claim they can use stem cells to help people with Parkinson’s. Should I go to them?

("In a word, no! These clinics offer a wide variety of therapies using different kinds of cells or tissues (including the patient’s own blood or fat cells) but they have one thing in common; none of these therapies have been tested in a clinical trial to show they are even safe, let alone effective. These clinics also charge thousands, sometimes tens of thousands of dollars these therapies, and because it’s not covered by insurance this all comes out of the patient’s pocket.

("These predatory clinics are peddling hope, but are unable to back it up with any proof it will work. They frequently have slick, well-designed websites, and  'testimonials' from satisfied customers. But if they really had a treatment for Parkinson’s they wouldn’t be running clinics out of shopping malls, they’d be operating huge medical centers because the worldwide need for an effective therapy is so great.

("Here’s a link to the page on our website that can help you decide if a clinical trial or “therapy” is right for you.")

California Parkinson's Group Strikes Out on Path to Stem Cell Therapy; Clinical Trial, For-profit Company Goals

A San Diego patient advocate group for Parkinson's Disease is making a major financial move as it heads toward creation of a for-profit company to develop a stem cell therapy for an affliction that affects about one million people in the United States.

The group is the Summit for Stem Cell Foundation, which has been deeply involved in seeking funds from the $3 billion California stem cell agency. The Summit organization supports research being conducted by Jeanne Loring, director of the Center for Regenerative Medicine at the Scripps Research Institute.

Summit says it has raised $5 million as of this spring from a variety of sources. It estimates it will need $8 to $10 million to reach the stage where the federal government approves the beginning of a clinical trial. It also expects to raise more cash by avoiding the high administrative charges involving many research organizations.

Reporter Bradley Fikes of the San Diego Union Tribune wrote yesterday about the latest developments. He said Summit is moving out of its space at Scripps as it prepares to apply for funds for clinical research. Fikes wrote,

"The group has leased about 5,000 square feet, doubling its space, and as a parting gift Scripps Research is donating equipment from its stem cell center, saving the foundation nearly $1 million in start-up costs. At the same time, its overhead is dramatically reduced, said Jeanne Loring, the group’s lead scientist and professor at Scripps Research. (Loring is serving as the unpaid director of research for Summit.)

"A $2.4 million grant the group received from California’s stem cell agency ran out earlier this year, Loring said. Now the group can step up fundraising and add staff. It’s also soliciting more donated equipment."

Fikes continued,

"The group also plans to establish a for-profit company to get funding from investors interested in generating returns, Loring said. By drawing investments as well as philanthropy, the project gains access to capital not otherwise available.

"The group was inspired by the example of the Cystic Fibrosis Foundation, Loring said. In 2000, the foundation gave San Diego’s Aurora Biosciences $40 million to develop a cystic fibrosis drug."

Fikes reported that separation from Scripps has financial advantages dealing with what called "indirect costs." 

"Raising (donations) has been difficult, in large part because of the overhead issue, Loring said.

"Scripps Research imposes overhead of up to 94 percent, meaning that every dollar donated to the group required an additional 94 cents for the institute, Loring said. At the new location close by on Torrey Pines Mesa, overhead is roughly 10 percent.

"Lower overhead means the group can now apply for philanthropic grants that previously weren’t practical, she said, because donors prefer as much as their money as possible go directly to the cause."

Here is a video of Loring describing her work. 

California Backs Research for Brain Cancer and Parkinson's with $9.5 Million

OAKLAND, Ca. -- The California stem cell agency today awarded $9.5 million for two late stage preclinical projects for development of therapies aimed at brain cancer and Parkinson's Disease.

A $5.8 million award for Parkinson's went to Krystof Bankiewicz of UC San Francisco and Clive Svendsen of Cedars-Sinai. A $3.7 million award for glioblastoma, a form of brain cancer, went to John Zaia of the City Of  Hope. Glioblastoma is the form of cancer that has afflicted U.S. Sen. John McCain.

In a news release from the agency, known formally as the California Institute for Regenerative Medicine (CIRM), its CEO, Maria Millan, said,

“Glioblastoma is the most common, and the most aggressive, form of brain cancer that led to the death of U.S. Senator Ted Kennedy and former Vice President Joe Biden’s son Beau Biden. 

"CIRM has supported a variety of stem cell-based approaches to target this devastating and currently untreatable condition.  The project approved by our board today is unique in that it seeks to use gene modified stem cells to allow patients to tolerate the high doses of chemotherapy while also making these tumors more susceptible to the chemotherapy.”

Regarding the Parkinson's award, the agency has already pumped $22.3 million into a phase one clinical trial being conducted by Svendsen for treatment of ALS with the same neural progenitor cell product that will be used in the Parkinson's research.

Here are links to the review summaries on the Parkinson's application and the glioblastoma application, plus additional information.

Application Number	Principal Investigator	Institution	Target	Amount Awarded	Public Summary of Review
CLIN1-10967	John Zaia	City of Hope	Glioblastoma	3,684,259	Ex Vivo Gene Engineering of Blood Stem Cells for Enhanced Chemotherapy Efficacy in Glioblastoma Patients
CLIN1-11059	Krystof Bankiewicz/ Clive Svendsen	UC San Francisco/ Cedars- Sinai	Parkinson’s Disease	5,811,340	MRI Guided Delivery of Neural Progenitor Cells Secreting GDNF for the Treatment of Parkinson's disease

Safety and Effectiveness Concerns Raised about $5.8 Million California Stem Cell Research Proposal for Parkinson's

A California stem cell scientist yesterday commented on an application before the state's stem cell agency for $5.8 million for research into a possible therapy for Parkinson's disease, which afflicts as many as 10 million persons worldwide.

Jeanne Loring, director of the Center for Regenerative Medicine at the Scripps Research Institute, made the remarks concerning a proposal that is likely to be approved June 28 by the governing board of the $3 billion agency.

Her comments came in an email to the California Stem Cell Report after it asked for her thoughts on the review summary of the application (CLIN1-11059), which has been already approved behind closed doors by the agency's scientific reviewers. The vote was 9-6-0 with nine reviewers voting to approve it and six reviewers saying it needed improvement and should be resubmitted. No reviewers voted outright against it. The summary of the review and application can be found here. 

The name of applicant is not known. The stem cell agency withholds the identity of applicants until an application is ratified by the full board in a public session.

As Loring notes in her email, she is also conducting research on Parkinson's and also has funding from the stem cell agency, formally known as the California Institute for Regenerative Medicine. Here is the text of her email along with links to the two research papers that she cites.

“Researchers have explored many approaches to treatment of Parkinson’s disease over the last several decades. The tremors and freezing in PD are caused by the progressive loss a specific type of dopamine-producing neuron in a part of the brain called the substantia nigra. Half of these neurons have died by the time the disease is diagnosed.

“The only approach that has resulted in long-term reversal of the symptoms of PD is transplantation of human fetal tissue containing the precursors of that specific type of dopamine neuron. 

“The therapy that we are developing is based on the success of those fetal studies; our particular approach is to use patient-specific dopamine neurons produced from their own induced pluripotent stem cells (iPSCs). The arguments in support of this therapy have been published (see below).

“We share this idea for neural replacement therapy with several other groups worldwide: Roger Barker in the UK, Malin Parmar in Sweden, Jun Takahashi in Japan, and Lorenz Studer in New York are using human embryonic stem cell-derived neurons or unmatched iPSCs. We are the only group among these that is using matched neurons so there will be no need for immunosuppression.

“These are my concerns about any therapy using undifferentiated neural stem cells and gene therapy:

“Safety concerns:

“Neural stem cells are dividing cells that have been known to make tumors; one patient in California had a tumor grow in his spine after receiving neural stem cells from a clinic that did not have FDA approval, to treat a stroke. A few years ago there was a proposed treatment for Alzheimer disease that used neural stem cells, and in that case there was clear evidence that those cells made tumors in animals; it was funded by CIRM. Our cells are not dividing, and we have done a one-year study in rats to make sure no tumors form.

“Addition of a transgene for a neural growth factor, GDNF, to the cells, will require thorough testing because transgenes can disrupt the genome, and the expansion of the cells increases the risk of other mutations arising, such as the p53 mutations that we discovered in hESCs a few years ago. p53 is a very bad actor; mutations in this gene are found in half of all cancers. The applicants should be planning to sequence the genome of the cells to be improve the chances that the cells are safe; we sequence our cells.

“Effectiveness concerns:

“Survival vs. replacement.

“There are two general approaches to cell-based treatment of Parkinson’s disease. The neural stem cell approach is not intended to REPLACE the dying neurons. It is intended to help the remaining dopamine neurons survive longer. The other approach, neuron replacement therapy, can reverse the symptoms long-term.

“Immunosuppression. It is necessary to suppress the immune system whenever a transplantation of unmatched cells or tissues is performed. Immunosuppression is a concern for neurologists who are treating PD patients, because it weakens the immune system and makes patients more susceptible to serious debilitating infections.

“The neural stem cells making GDNF may be a good solution for the debilitating neuronal loss in ALS, and CIRM is already funding their use for that disease. But please ask a few neurologists what they would advise their patients with Parkinson’s disease. Those I’ve asked would prefer replacement of dopamine neurons, rather than a treatment using transplantation of dividing cells pumping a nerve growth factor into the brain, that requires immunosuppression. Historically, this has not been a good idea.”

Here are two links to articles that Loring cited. See here and here. 

California Researchers Score $16 Million Plus in State Stem Cell Awards

The California stem cell agency today handed out $16.4 million in research grants seeking therapies for afflictions ranging from gum disease and cancer to vision loss and Parkinson's Disease.

The award for Parkinson's was relatively tiny -- only $150,000 -- but represented a rare case in which the agency's governing board overturned its reviewers, who make the de facto decisions on awards.

The reversal came after one board member, David Higgins, of San Diego, who has Parkinson's, noted that the most common drug that Parkinson's patients take is 70 years old. He told the board.

David Higgins, CIRM photo

“I’m a fourth generation Parkinson’s patient and I’m taking the same medicine that my grandmother took. They work but not for everyone and not for long. People with Parkinson’s need new treatment options and we need them now. That’s why this project is worth supporting. It has the potential to identify some promising candidates that might one day lead to new treatments.”

The award went to Zenobia Therapeutics, Inc., of San Diego, whose president and co-founder, Vicki Nienaber, had filed an appeal on the reviewers' decision.  Another applicant rejected by reviewers, Toshio Miki of USC, also filed an appeal with the board. His appeal was not discussed. Miki's application sought $5.9 million for research involving metabolic disorders.

The largest award today, $5.6 million, went to Anthony Oro of Stanford, who will be testing a therapy to treat an affliction that creates wounds that will not heal. Dan Kaufman of UC San Diego received $5.5 million to produce "killer cells" to help people with a form of leukemia. Catriona Jamieson, also of UC San Diego, received $2.7 million for leukemia research.

Here is a link to summaries of reviewer remarks, including scores, on the three winners and the 11 other applications that were rejected in the translational awards category. (Scroll down on the page to see the reviews.)

In addition to the "discovery" award for Parkinson's, here are the names of the other winners in that category:

•  DISC1-10603, Ngan F Huang, iPSC-Derived Smooth Muscle Progenitors for Treatment of Abdominal Aortic Aneurysm, Palo Alto Veterans Institute for Research, $172,621
• DISC1-10475, Semil P Choksi, Generation of human airway stem cells by direct transcriptional reprogramming for disease modeling and regeneration, U.C. San Francisco, $238,408
• DISC1-10643, Dmitriy Sheyn, IVD rejuvenation using iPSC-derived notochordal cells, Cedars-Sinai, $241,992 
• DISC1-10598, Alice F. Tarantal, Enhanced Branching Morphogenesis and Pluripotent Cell Lineage Differentiation for Pediatric Regenerative Therapies, U.C. Davis, $235,80
• DISC1-10583, John R Cashman, Human Pancreatic Cancer Stem Cells: Developing a Novel Drug for Cancer Eradication, Human BioMolecular Research Institute, $303,894 
• DISC1-10555, Hiromitsu Nakauchi, Optimizing self-renewal signaling kinetics to stabilize ex vivo hematopoietic stem cell expansion, Stanford, $235,836 
• DISC1-10620, David J. Baylink, Bone Marrow Targeting of Hematopoietic Stem Cells Engineered to Overexpress 25-OH-VD3 1-α- hydroxylase for Acute Myeloid Leukemia Therapy, Loma Linda University, $178,967 
• DISC1-10513, Guillem Pratx, Novel metabolic labeling method for tracking stem cells to irradiated salivary glands using PET, Stanford, $235,613 
• DISC1-10522, Gerald P Morris, Identification of antigenic neo-epitopes from in vitro reprogrammed human tissue precursors for regenerative therapy, U.C. San Diego, $193,500
• DISC1-10588, Julia J. Unternaehrer-Hamm, Targeting cancer stem cells with nanoparticle RNAi delivery to prevent recurrence and metastasis of ovarian cancer, Loma Linda University, $172,870 
• DISC1-10721, Karl J. Wahlin, An IPSC cell based model of macular degeneration for drug discovery, U.C. San Diego, $232,200 
• DISC1-10516, Alyssa Panitch, Development of treatments to improve healing of ischemic wounds, U.C. Davis, $235,800 
• DISC1-10718, Alireza Moshaverinia, Gingival mesenchymal stem cells as a novel treatment modality for periodontal tissue regeneration, U.C. Los Angeles, $194,483

Here is a link to all the review summaries and scores in the round, including the 28 rejected applications.  Here is a link to the CIRM press release. 

More California Millions Sought to Support the 'New Era' of Parkinson's Therapy Research

A "new era" in the search for a cure for Parkinson's disease was heralded this month in an article in a prominent scientific journal that explored research involving more than $52 million and an organization called GForce-PD.

The news was accompanied by a cry for more support for Parkinson's research from the $3 billion California stem cell agency, which has pumped $49 million in Parkinson's studies over the last 13 years.

Jeanne Loring, director of the Scripps Center for Regenerative Medicine in La Jolla, Ca., and also a participant in the GForce initiative, said this week that CIRM has not supported Parkinson's research at the level of the other enterprises involved in GForce.

In an item she wrote for The Niche, a blog published by UC Davis researcher Paul Knoepfler, she listed $52.3 million in support plus substantial backing from BlueRock Therapeutics, which is financed with $225 million from Bayer AG and Versant Ventures. BlueRock, a Cambridge, Mass., firm, says on its web site, “Our most advanced therapeutic candidate, for Parkinson’s disease, will enter the clinic in 2018.

Parkinson's is a devastating disease that afflicts 10 million people in the world and 125,000 in California. One of those persons, David Higgins of San Diego, currently serves on the board of the stem cell agency, formally known as the California Institute for Regenerative Medicine (CIRM). Another, Joan Samuelson, was one of the original board members in 2004 and a fervent but often frustrated voice for Parkinson's research at many CIRM board meetings.

Actor Michael J. Fox, who made TV ads for the 2004 ballot initiative that created the stem cell agency, is also among the those living with the disease. Others with the affliction included the late boxing champion Muhammad Ali and famed semiconductor pioneer Andy Grove. 

Loring wrote on The Niche about a meeting in Japan earlier this year dealing with the research teams in the GForce project. She said,

"The Kyoto meeting was unprecedented in my experience.  Instead of competing, the four groups cooperated and shared plans for their proposed clinical trials.  We agreed to harmonize our trials and stay in communication about our progress.  All of us plan to start clinical trials within two years."

Loring continued,

"Since my team has been recognized by the international GForce initiative devoted to safe effective therapy for PD, we hope that CIRM will follow the example of New York, the EU, and Japan, and invest more in our project to provide neuron replacement therapy for Californians with Parkinson’s disease.

"While we hope to gain more support from CIRM, we are determined to follow through with our clinical trial, with or without CIRM.  It will just be more difficult without their help. The patients and their advocates inspire us, and we won’t let them down."

Loring added more information on Sunday (Nov. 19) concerning CIRM funding. She told the California Stem Cell Report. 

"We started our pre-clinical PD studies in 2011, with funding from Summit for Stem Cell. The first and only funding we received from CIRM for PD research was in 2016. 

"Before 2011, CIRM invested $41,838,336. Since 2011, CIRM has invested $7,357,468. This means that the majority of the funding went to projects that didn’t lead to any translational or clinical applications.  

"There are currently three active grants working on Parkinson’s disease, for a total of $4.9 million. We have the only translational grant, and it will expire in March 2018. All but $650,000 runs out by the spring of 2018. 

"There are no more grants forthcoming for our work. The Scaled Biolabs grant awarded this year is a partnership with us. Birgitt Schuele’s grant is basic research, not a cell therapy. (Below are the three active grants identified by Loring.) 

"Parkinson's Institute, Birgitt Schuele, Quest - Discovery Stage Research Projects, CRISPR/dCas9 mutant targeting SNCA promoter for downregulation of alpha-synuclein expression as a novel therapeutic approach for Parkinson’s disease, $1,931,495 

"Scripps Research Institute, Jeanne Loring, Quest - Discovery Stage Research Projects, Autologous cell therapy for Parkinson’s disease using iPSC-derived DA neurons, $2,354,226 and later $4,285,721  

2017
"Scaled Biolabs Inc., Justin Cooper-White, Quest - Discovery Stage Research Projects, A tool for rapid development of clinical-grade protocols for dopaminergic neuronal differentiation of Parkinson’s Disease patient-derived iPSCs, $657,528 "

(Editor's note: An earlier version of this item contained incorrect figures on the GForce initiative.) 

Aussie Clinical Trial for California Firm's Stem Cell Therapy for Parkinson's Disease

A Southern California firm that repeatedly and unsuccessfully sought funding from the $3 billion California stem cell has received the go-ahead in Australia for human testing of a treatment for Parkinson’s Disease, it was reported today. 

In an article today in the San Diego Union-Tribune, reporter Bradley Fikes described the move involving International Stem Cell Corp. (ISCO) of Carlsbad as appearing to be a “medical first.” The company's stock price jumped nearly 16 percent today on the news.

Fikes wrote,

“If all goes according to plan, doctors will implant replacement brain cells into 12 Parkinson’s patients, probably in the first quarter of 2016, said Russell Kern, the company’s chief scientific officer. These are called neural precursor cells, a slightly immature kind of neuron. The cells will finish maturing in the brain into the kind of neurons destroyed by the movement disorder.

“The neural precursor cells are derived from the company’s parthenogenetic stem cells, which are produced from unfertilized human egg cells.”

International Stem Cell is a publicly traded firm whose researchers pitched proposals with some regularity to the stem cell agency a few years back. It was not known whether any of the applications involved the Parkinson's therapy. The firm’s personnel also attended CIRM board meetings with some frequency. (For earlier items on the company, see here, here, here, here and here.)

The day after Fikes' story appeared, the stem cell posted an item on its Stem Cellar blog about the effort.

Fikes wrote that the firm’s trial will be the first Parkinson’s trial  using replacement brain cells grown from stem cells, according to clinicaltrials.gov. The trial will be conducted by the firm’s Australian subsidiary, Cyto Therapeutics. 

Fikes reported that company’s effort is similar to other research in the San Diego area.

“That’s also the approach Summit for Stem Cell will take, said stem cell scientist Jeanne Loring, a leader of the Summit for Stem Cell project. The cells make proper connections with the brain better when they are still maturing, said Loring, who’s also head of the regenerative medicine program at The Scripps Research Institute in La Jolla.”

Loring is applying for an award from the stem cell agency but her research is at an earlier stage than that of the Carlsbad firm. Fikes wrote,

“Loring said she views ISCO as a partner in fighting Parkinson’s. One of her former students is working for the company, she said….

“ISCO’s choice of Australia for its streamlined regulatory process makes sense, Loring said. Her team, with U.S.-based academics and medical professionals, doesn’t have the same flexibility as ISCO in looking for clinical trial locations, she said.”

The firm’s stock closed at $5.00 today, up nearly 16 percent. Its 52-week high was $12.30 and low was $1.25. Here is a link to the company’s press release on the news today.

(The information concerning the agency posting an item on the San Diego news was added to this item 24 hours after it was first published.)

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.

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. 

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. 

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. 

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.)

Text of Comments by Sherrie Gould, Executive Director, Summit4stemcells

Here is the text of comments by Sherrie Gould, executive director of Summit4stemcell of San Diego, made in connection with changes in the stem cell agency board meeting Sept. 24. Gould made the remarks in response to a query from the California Stem Cell Report.

“We are both confused and disappointed that this PUBLIC meeting has
been essentially cancelled.  We were prepared to talk with the ICOC (the stem cell agency) board and answer all questions about our patient advocacy
(Summit4stemcells) movement and our research. The Summit4stemcell
project has been faithfully attending public meetings every other
month for the last two years.  We have flown in numbers to make sure
our voice is heard.  We have spoken to board members, and to the media
about our plight.  This San Diego meeting, now cancelled, was an
opportunity attend to discuss the funding possibilities for
Parkinson's disease and whether funding might be appropriate for our
project.  Our science is sound and we have a clinical arm to this
project. We believe CIRM could take ownership of this very exciting
translational research if we were indeed funded.

“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. And
although hope springs eternal, we are saddened by the repetitive
delays.”

Text of Remarks by David Higgins, Stem Cell Agency Board Member

Here is the text of comments by David Higgins, a member of the governing board (the ICOC) of the California stem cell agency, in connection with changes in the September meeting of that board. By law, Higgins is appointed as a patient advocate for Parkinson’s disease. He made his comments in response to questions from the California Stem Cell Report.

“It is my understanding that the September ICOC meeting was switched to a phone format because the modest agenda planned made the cost associated with having this meeting ‘on the road’ questionable.

“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. There will be differences in opinion on how best to allocate CIRM’s financial resources, but there are no difference of opinion on the importance of patient advocacy and the value of interactions between the public and the ICOC.”

Text of McCormack's Response to Questions

Here is the text of remarks today from Kevin McCormack, senior director of communications for the California stem cell agency, concerning changes involving this month's governing board meeting. He made the comments in response to questions by the California Stem Cell Report.  One of the questions involved the agency's strategic plan, which was scheduled to be discussed at the meeting this month.

"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 elimination of the dates in the Discovery and Translational programs is simply to give the CIRM Team more flexibility. As we say in the documents that will be presented to the Board – available on our website here https://www.cirm.ca.gov/sites/default/files/files/agenda/150905_Agenda_4_DTConcept%20Amendments%20--%20FINAL%20%2800256870xAEB03%29.pdf
  

"In  order  for  CIRM  to  operate  efficiently  and  to  respond  to  changing  circumstances  and  needs,  CIRM  must  be  nimble  and  flexible.   To  ensure  that  the  CIRM  team  has  the  ability  to  make  adjustments  to  the  program  schedule  to  meet  these  needs,  we  propose  to  eliminate  the  specificity in  the translation  and  Discovery  Program  concept  plans  regarding  the  schedule  for  the  submission  of  applications  in  response  to  program  announcements  issued  under these programs.”  (McCormack's boldface) 

"As for the Strategic Plan, we have already conducted extensive outreach, through both on-line surveys and in-person meetings, with Patient Advocates (including meetings in San Diego, Los Angeles and San Francisco), researchers, Board members and the public. We are now working on bringing all those thoughts and suggestions together in order to be able to present them to Board. 

"And the Board agenda will be posted ten days ahead of the meeting as usual." 

'Brutal Facts,' Parkinson's and the California Stem Cell Agency

Randy Mills in San Diego, Union-Tribune photo by K.C. Alfred

The $3 billion California stem cell agency took its strategic-plan road show to San Diego yesterday and heard impassioned pitches for development of a Parkinson’s therapy.

Reporter Bradley Fikes of the San Diego Union-Tribune wrote that “sometimes emotional” exchanges arose at the meeting.  And he reported that the agency’s president, Randy Mills, said one of the “brutal facts” is that that “drug and medical companies aren’t really interested in stem cell treatments.”

The hearing was one of three this week to gather information to help reshape the agency’s plan for spending its last $800 million over the next five years. The other hearings will be in Los Angeles today at noon and in San Francisco tomorrow at noon. (See here for addresses.)

At yesterday’s session, Fikes quoted Mills as saying,

"We do not have enough industry pull for our technology….We don't have enough buyers for our intellectual property."

The article continued,

“Stem cell therapies are still considered too risky by most companies, Mills said.”

 About 70 persons attended the meeting, according to Fikes. That is about 10 times the size of the audience at most meetings of the governing board of the California Institute for Regenerative Medicine or CIRM, as the San Francisco agency is formally known.

Many in the audience were there to pitch for Parkinson’s. Fikes wrote,

“Patient advocate Sherrie Gould asked Mills to support funding a potential Parkinsons' disease therapy being developed by a coalition of scientists, doctors and Parkinson's patients called Summit4StemCell. Dozens of coalition supporters attended the meeting.

“The group seeks to grow replacement brain cells to relieve symptoms of the movement disorder, derived from the patient's own skin cells. It's affiliated with the Parkinson's Association of San Diego.”

Fikes continued,

David Higgins, Linked In photo

“We are ready, the patients are ready, the cells are ready,’ Gould said. ‘Time is running out for these patients.’" 

The 29-member CIRM board has a Parkinson’s patient advocate as a member, David Higgins of San Diego, who is a former president of the Parkinson’s Association, according to CIRM.