Friday, October 20, 2017

LA Times: Does California's New Stem Cell Law Do Enough to Regulate Exploitation of Desperate Patients?

The Los Angeles Times this morning carried a piece that praised the state's first-in-the-nation disclosure law concerning unregulated stem cell treatments, but the article also questioned whether the law is tough enough for the task.

Business columnist Michael Hiltzik wrote that the law, which goes into effect in January, is "a major step to address an emerging public health crisis."  But, he continued,
"(T)here’s reason to ask whether California’s law goes far enough to regulate businesses exploiting the desperation of patients with intractable diseases."
The law targets the more than 100 clinics in California that sell what Hiltzik described as  "unlicensed, unproven — and sometimes disproven — stem-cell 'treatments.'" For the first time, such California clinics will be required to disclose to their customers that the treatments are not approved or regulated by the federal government. The notices will advise the customers to consult with their physicians prior to treatment.

Hiltzik,  however, questioned the optimistic wording of the disclosure which says that the treatments have "not yet" been approved by the Food and Drug Administration (FDA). He said,
"This suggests that FDA endorsement may only be a matter of time — that the treatments may be premature, but not fictitious. That’s wildly optimistic and may itself foster a false hope for the treatments."
Hiltzik, a Pulitzer Prize winner,  said the state's Medical Board, which is charged with enforcement, has not been provided with funding to take on the clinics. Plus, he said the board, at best, is a "reluctant regulator."

State Sen. Ed Hernandez, D-Azusa, authored the law. He told Hiltzik the measure is a first step. Hiltzik quoted the legislator as saying,
 “Because it’s so new, we’re trying to figure out the best way to start the conversation.”
Hiltzik concluded,
"But lawmakers and regulators may need to move faster. What will make a difference in California may not be how the conversation starts, but where it leads."
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Monday, October 16, 2017

CIRM-funded ALS Trial Enrolls First Patients; Therapy Produced by Israeli Firm

An Israeli firm backed with $16 million from the California stem cell agency today announced that it has enrolled the first patients in its phase three trial for a treatment for ALS, sometimes known as Lou Gehrig's disease.

The trial, the final one before possible approval for widespread use of the therapy, is expected to include about 200 patients. The California component is being conducted at UC Irvine for the firm, BrainStorm Cell Therapeutics. 

The $3 billion California stem cell agency, known formally as the California Institute for Regenerative Medicine (CIRM),  awarded the cash to BrainStorm last July. The agency's reviewers said of the application:

"Overall, the proposed phase 3 study is well-designed. If executed successfully, it will provide a very meaningful outcome, generating data to define the overall value of the program and the path to regulatory approval and marketing. 
"The investigators have now included slow vital capacity as a clinical measure in the revised clinical trial per (earlier reviewer) recommendations . 
"The current protocol will still not answer the question of whether the product is better than untreated autologous bone marrow-derived MSCs. However, reviewers felt this is not critical for the current study as a positive outcome in this trial may allow a demonstration of superiority over MSC alone in a future study."
The firm has matched the California award with another $16 million. Completion date of the trial is 2019.  The trial is one of two phase three trials backed by CIRM that are currently recruiting. The principal investigator at UC Irvine is Namita A. GoyalMore information on the trial can be found here. 

About 5,600 people in the United States are diagnosed annual with ALS, which has severely disabled British physicist Stephen Hawking. Sphere: Related Content

Thursday, October 12, 2017

Correction

An item earlier today said incorrectly that this week's Internet outage at the California stem cell agency was the first such significant occurrence. The agency, in fact, had another outage in September. Sphere: Related Content

Internet Outage for California Stem Cell Agency Earlier This Week: Cause Unknown

California's $3 billion stem cell agency went dark on the Internet earlier this week, the second time it has had a significant Internet outage in recent months.

The outage began on Monday afternoon. The agency was back up by Tuesday at about 7 p.m. The California Institute for Regenerative Medicine (CIRM), as the agency is formally known,. reported that the problem did not affect internal work but did affect public access to agency information. It also blocked any incoming email or outgoing email. However, all of the email went through after the problem was solved. 

In response to a question from the California Stem Cell Report, Kevin McCormack, senior director for communications, said,
"At about 1 p.m. on Monday 10/9/2017 our external Domain Name System (DNS) provider dropped our DNS records, that meant people looking for our website, blog, GMS(grant management system), and email servers were unable to access them as DNS converts names (like www.cirm.ca.gov) into an IP (internet protocol) address (like 192.168.2.3) so that the appropriate server may be accessed by your browser. 
"We are working with our DNS provider to find out the root cause of that event.  In the meantime, we brought up a new external DNS server with another provider to restore name resolution.
"The servers for www.cirm.ca.gov and grants.cirm.ca.gov were themselves unaffected in that they continued to operate as normal awaiting requests - but without external DNS converting names into IP addresses, no requests would come to those servers from outside of CIRM.
 "CIRM staff and the GWG (grant application reviewers) then meeting at CIRM were still able to continue as usual accessing the GMS since they were using internal DNS servers.  Similarly, all purely internal email within CIRM was unaffected - however, because we were without external DNS, any email coming into or out of CIRM was not delivered."
Editor's note: An earlier version of this incorrectly stated that the problem was the first significant outage for the agency. Sphere: Related Content

Monday, October 09, 2017

Probing California's Stem Cell Trials: New Tool Helps Public Navigate the Multi-Million Dollar Maze

Here's how the new clinical trial dashboard looks. You can slice and dice
the information by clicking on the black and blue boxes on the CIRM
website at the top of the page.
California's $3 billion stem cell agency this week is debuting a new, online guide to its burgeoning network of clinical trials aimed at  developing therapies ranging from acute myeloid leukemia to X-linked severe combined immunodeficiency (X-linked SCID).

The new tool is called the clinical trials dashboard. It is a major improvement in making information on the all-important trials more easily available to the public, patients, researchers and policy makers.

Clinical trials, which have three stages and can last years, are the final step before a therapy can be approved for widespread use by the Food and Drug Administration. The agency plans to spend about $400 million on clinical ventures from 2017 to 2020.

The new dashboard cuts through the online maze and allows readers to sort by disease areas, investigator and institution, among other things. It also shows quickly the status of each trial, whether it is recruiting and takes the reader to a contact if he or she is interested in participating in a trial.

As the main dashboard page shows, the agency, formally known as the California Institute for Regenerative Medicine (CIRM), has funded 40 trials. The dashboard allows you to see which are still active and which are still recruiting. If you are interested in cancer and solid tumors, filtering them out is simple. Just click on the blue box on the dashboard.

Want to know which institution has the most CIRM-backed trials? The answer is UCLA with five, a figure easily arrived at by using the new CIRM tools. That goes for investigators as well. The dashboard can show you that Donald Kohn of UCLA has the highest number of trial awards with three. A bunch of others have two each.

CIRM has long had a vast array of information on its website. As with any website, that presents a challenge in terms of making the information available to public. First, the public needs to know it is there. Next, they need to be able to actually find the relevant information and find it relatively easily. Removing barriers to online access is critical. The online tools also must fill a wide range of information needs.  A cancer patient or researcher is not likely to be interested in plowing through reams of eye disease information in order to gain access to the relevant cancer data.

The clinical trial dashboard was built by Karen Ring, CIRM's website manager and a former stem cell researcher, along with contributions from numerous others on the agency's team. It is a major step forward in the agency's effort to tell the CIRM story and how it has spent the billions provided by California taxpayers.

Here is a video that turned up this morning as I navigated my way through the clinical trial dashboard. Another note: I did not encounter any bugs this morning, but Ring says she will be tweaking the dashboard and improving it in the next couple of weeks.
Sphere: Related Content

Sunday, October 08, 2017

Preview of Bond Campaign: California Stem Cell Agency Described as 'Dismal,' 'Disappointing' and a 'Waste'

A couple of die-hard opponents of the $3 billion California stem cell agency minced no words this weekend. In an op-ed piece in the Orange County Register, they described the agency as nothing more than an "advanced high school science project."

The article was written by Jon Coupal, president of the Howard Jarvis Taxpayers Association in Sacramento, and state Sen. John Moorlach, R-Costa Mesa. 

They described the agency's efforts as "dismal" and declared that "this disappointing abuse of taxpayer dollars" should be terminated.

The print version of the article appeared in Sunday's paper, which has a circulation of about 312,000. Both the print and online version carried photographs involving stem cell research. But neither was from UC Irvine, which has received $108 million from the agency and has a member on the board of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known.  UC Irvine is located only 17 minutes away from the offices of the newspaper. 

The piece is a relatively tame preview of the rhetoric that will confront CIRM if it goes to the ballot in 2020 for more billions from California taxpayers. The article cited arguments from the 2004 Prop. 71 ballot measure campaign that created the agency. 

Coupal and Moorlach said, 
"According to the ballot pamphlet mailed to voters, proponents promised the bond proceeds would advance the 'cure and treatment' of  'cancer, diabetes, heart disease, Alzheimer’s, Parkinson’s, spinal cord injuries, blindness, Lou Gehrig’s disease, HIV/AIDS, mental health disorders, multiple sclerosis, Huntington’s disease, and more than 70 other diseases and injuries.' 
"But actual outcomes for these promised advances are speculative at best and nonexistent at worst. Similar benefits were promised to the California economy to 'generate millions of new tax dollars.' 
"With such a dismal record, this would be a good time to shut the spigot on issuing the remaining $345 million — meaning some $690 million would be saved by state taxpayers. That money could be better spent on pensions, schools, roads, housing or better basic medical care for our residents."
As for the high school project business, the article said CIRM "continues to operate as a kind of advanced high-school science project, instead of moving toward the cures promised to voters in Prop. 71."
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Thursday, October 05, 2017

Forty-three Percent Jump in Interest in California Stem Cell Internet Information


Interest in California stem cell matters spiked in mid-week but still is well below the interest in broader matters, such as stem cell therapies, according to Google search data this morning.

Searches using the term "California stem cells" shot up 43 percent in midweek, Google reported. Just exactly why more people were searching on the term is not clear, but the Google data shows a definite high point.

The scale of 1 to 100 on the charts with this item reflects the level of interest -- not numbers of readers or page views. One-hundred shows the peak interest level only.  Actual numbers were not disclosed by Google.

Readers can see the trends in the two Google-generated charts attached to this item, which have an important caveat. The charts are live and updated automatically by Google as long as they are posted.

My take on the information is that at this point it is more of a curiosity than of any major signficance. However, in the event of major stem cell news, the data might have more value.
Sphere: Related Content

Wednesday, October 04, 2017

California Moves to Protect Patients Seeking Unregulated Stem Cell Treatments

Beginning next year, California will have a new law that imposes the first-ever disclosure requirements on currently unregulated stem cell clinics that offer therapies that have not been approved by the Food and Drug Administration.

UC Davis stem cell scientist Paul Knoepfler, who for years has raised concerns about such clinics, wrote an item yesterday about the new law for his blog. It was headlined:
"Groundbreaking new California stem cell law gives consumer protections on clinics"
Ed Hernandez, Daily News photo
The measure is the first of its kind in California and probably the entire nation.  Authored by state Sen. Ed Hernandez, D-West Covina, it will require clinics to specifically notify its customers that he or she is about to undergo a treatment not approved by the FDA. The notification also must include advice encouraging customers to consult their primary care physician prior to undergoing a stem cell therapy.

A legislative analysis of the bill, SB512, cited a statement from the Center for Public Interest Law that said, 
"It is critical to patient safety that these individuals are not misled into believing they are partaking in an FDA-approved clinical trial and assuming serious health and financial risks in the process. The disclosure notices required by this bill are an important step in ensuring that patients have the information they need before making such monumental decisions about their treatment."
Paul Knoepfler, UC Davis photo
Knoepfler co-authored a research paper in 2015 that documented  the presence of nearly 600  unregulated clinics nationwide with California leading the way. He wrote on his blog, 
"Taken together, the provisions of this law will help consumers learn more about the stem cell clinic industry, make better decisions about their health and that of their loved ones, and delineate the difference between stem cell clinics versus compliant researchers conducting stem cell clinical trials with FDA approval.
"In the big picture, this new California law plus a more active FDA on the stem cell front together give me more hope that the wild west of stem cell clinics can be reined in sooner rather than later! Perhaps other states will follow suit with new laws and state medical boards will get more involved in overseeing stem cell therapies. With more 570 stem cell clinics in the US and more than 100 here in California alone, more efforts like these are needed on the consumer protection and educational outreach front."
Knoepfler deserves great credit for his efforts regarding unregulated clinics. It is fair to say that without his work, California would not have seen this law at this time. He was the first to document the size of the industry with the 2015 article, co-authored by Leigh Turner of the University of Minnesota.  Their work makes it abundantly clear that scientists can make a real difference on public policy issues if they are persistent and effective. One of Knoepfler's key tools was blogging, a practice that some scientists think is less than dignified. Nonetheless, his blogging helped to elevate the issue and served as a source for the news media as they looked into the matter. 

Like most new state laws, the law takes effect Jan. 1, 2018.
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