Showing posts with label patient advocacy. Show all posts
Showing posts with label patient advocacy. Show all posts

Wednesday, October 07, 2020

Head-to-Head on Proposition 14: 'Fatally Flawed,' Life-Saver or Both?

2012 CIRM video of Sandra Dillon (on right) and Catriona Jamieson of UC San Diego

Sandra Dillon says she owes her life to the California stem cell agency, and she wants California voters to give it $5.5 billion more to help save the lives of many more, including someone they love. 

Dillon made her comments in a piece carried last month in the San Diego Union-Tribune. Her article was paired with another commentary in the paper that strongly opposed Proposition 14 on this fall's ballot. It was written by Jeff Sheehy, a patient advocate member of the board of the California Institute for Regenerative Medicine (CIRM), the official name of the stem cell agency.  

Together, Dillon's and Sheehy's commentaries capture the essence of the arguments for and against the ballot initiative and highlight what is at stake this fall. And that is the continued existence of the stem cell agency, which was created in 2004 by another ballot initiative that provided it with $3 billion in money borrowed by the state. That funding is all but gone. If voters reject Proposition 14, CIRM will begin closing its doors this winter. 

(The actual cost to taxpayers, it should be noted, is about $4 billion in the case of the 2004 measure and an estimated $7.8 billion for Proposition 14.)

When Dillon was 28, she was diagnosed with a rare form of blood cancer called myelofibrosis. She wrote in the San Diego paper, 

"Upon learning of my cancer diagnosis, I was left feeling helpless, afraid and searching for answers about how this disease would affect my everyday life, what I could do to fight it, and how much time I had left to live....

"It is because of California’s forward-looking leadership in passing Proposition 71 in 2004 — hoping to find treatments and cures for chronic diseases and illnesses — that has allowed me to still be here today." 

Dillon concluded,

"The passage of Proposition 71 helped save my life. It is unimaginable to think that Californians would vote to discontinue this amazing effort — I don’t know where I would be or what condition I would be in if it wasn’t for the investment Californians made nearly two decades ago."

Dillon's experimental therapy involved research by Catriona Jamieson of UC San Diego, who has received $17.3 million from the stem cell agency.

In the other commentary, Sheehy, who has served on the CIRM board since 2004 and was chairman of its science subcommittee, was deeply critical. He was the only member of the CIRM to votes against endorsing the measure. 

The California Stem Cell Report carried a piece last month summarizing what Sheehy had to say in his piece, which was headlined,  "Why Prop. 14 is unaffordable, unnecessary, fatally flawed and unsupportable."

But to refresh readers, Sheehy, whose article was solicited by the newspaper, said, 

"It must seem odd that someone who has spent countless hours over the last 15 years as a member of the governing board of the California Institute for Regenerative Medicine (CIRM) would oppose Proposition 14, which seeks to provide $5.5 billion in new funding for the stem-cell agency. While I value CIRM and its work to date, Proposition 14 commits California to spending money it does not have — $7.8 billion including interest for research that is already well-funded. Plus, CIRM’s pre-existing flaws are actually exacerbated by new provisions in the measure."

"And after spending all of that money, not a single U.S. Federal and Drug Administration-approved product has materialized on which CIRM’s funding played an important role."

"Proposition 14 will add at least another $260 million a year in annual repayments. That means California taxpayers will be on the hook for $587 million a year for stem-cell research. Remember state imperatives such as education, health care and housing are not only chronically under-resourced but with a looming deficit, will be starved for funding because bonds must be repaid first. Cuts have already happened and more are likely on the way. Critical needs will go unfunded."

Monday, August 27, 2018

State Lawmakers Hear Story of a Life-Saving Treatment and the California Stem Cell Agency

Pawash Kashyap and son, Ronnie
A father from Folsom, Ca., earlier this month told a state legislative committee about how his baby boy was saved through a clinical trial that was being financed by the Golden State's $3 billion stem cell agency.

"It was a blessing day for us," said Pawash Kashyap, who appeared before the Assembly Select Committee on Biotechnology,  which held a hearing Aug. 15 dealing with the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. Kashyab referred to the occasion when he and his wife, Upasana, received a call from UC San Francisco warning him of the immune deficiency that afflicted their baby, Ronnie.

Researchers told them that the child could well die if he suffered from even a slight infection. The immune deficiency is sometimes known as the "bubble baby" disease, which has sometimes meant children were encased in plastic bubbles to stave off infections.

The problem was detected by routine newborn screening. Kashyap told legislators of their concern after learning the bad news.
"We Googled it, and nothing good was coming out of it."
Ultimately, the Ronnie was treated successfully. His father said that Ronnie has experienced potentially infectious crowds in malls and elsewhere and that he is doing "fantastically well."

Ronnie is now the "cover baby" on CIRM's annual report, which was praised by one lawmaker, Assemblyman Todd Gloria, D-San Diego. He said the report told the nearly 14-year, CIRM story in understandable and compelling language.

The full hearing can be seen here and downloaded. Kashyap's brief remarks begin at 55:36 into the video (he spoke without a prepared text). An audio file is also available at the same URL.

Here are links to the remarks at the hearing by CIRM CEO Maria Millan, CIRM Vice Chair Art Torres, patient advocate Don Reed, Jan Nolta, head of the UC Davis stem cell program, and David Jensen, publisher of this web site.

Friday, August 04, 2017

'A Reliable Voice of Reason:' A Look at a California Stem Cell Researcher/Blogger


Paul Knoepfler's TED talk has drawn more than one million views

The journal Science this week profiled UC Davis stem cell scientist and blogger Paul Knoepfler, describing him both as a consumer watchdog and a "dogged voice of caution."

In the article by Kelly Servick, George Daley,  dean of the Harvard Medical School, also called Knoepfler a "reliable voice of reason."  Daley noted that academics are "often more comfortable being provincial and insular, and not … mixing it up in the public debates."

Knoepfler began blogging seven years ago after -- at the age of 42 -- he was diagnosed with prostate cancer and given a roughly 50-50 chance of survival. He told Servick that he wanted to "expand how I had impact, beyond just the pure science." From the start, his blog went beyond science to patient advocacy.

Readers of the California Stem Cell Report are likely to be familiar with Knoepfler's blogging efforts, which are often reported here. We have followed his efforts since their inception. Over the years, his voice has grown to be heard in publications across the country. Just this week, he was widely quoted in coverage of the ground-breaking research into gene editing of human embryo.

(An aside from this writer, who is quoted in the Servick piece: One of the reasons that Knoepfler is quoted widely is that he returns media phone calls and can "speak English" -- meaning that he can explain science in terms that most persons can understand, at least those who read newspapers. That could have something to do with having an undergraduate degree in English.)

The Science article focused heavily on Knoepfler's writing about unregulated stem cell clinics, including research by him and Leigh Turner of the University of Minnesota that documented for the first time the size of that particular industry in this country. Their work counted 570 clinics with the most in California.

Why the focus on the unregulated treatments? Knoepfler told Science:
"'They were just saying, ‘Screw the rules, we're just going to set up shop and put up a website and start injecting people with stem cells....I saw that as a threat, first to patients, but to the field as well.'"  

Tuesday, June 28, 2016

California Stem Cell Hooha: Testy Media Exchange Triggered by Criticism of the State's Agency

A sharp exchange erupted this week on a mainstream California media site after the state's $3 billion stem cell agency was taken to task for lobbying the federal government to ease its regulation of research on stem cell therapies.

The flap involved allegations of lies and questionable motives. Along the way certain death was mentioned, and one writer was accused of "stealing hope" from seriously ill persons. Personal animus charges were raised along with questions about ill-informed rhetoric.

The hooha was set off by an op-ed piece by UC Davis stem cell scientist Paul Knoepfler who wrote an op-ed in the San Francisco Chronicle (see here and here). It chastized the agency and its president, Randy Mills, for a risky and "political" effort to change regulations by the federal Food and Drug Administration. Knoepfler said patients could be put at risk.

The piece triggered harsh comments that were carried by the Chronicle at the end of the article. In addition to Knoepfler, individuals involved in the exchange included Jeanne Loring, head of the stem cell program at the Scripps Institute in La Jolla; Judy Roberson, a Sacramento patient advocate for Huntington's disease, and a person identified only as "Jeff." He wrote,
"What's Knoepler's real beef?....Poor Paul is not getting the money from CIRM he used to get. So his attitude is 'screw patients,' I want my lab funded like it used to be. Nauseating!!!"
CIRM is the abbreviation for the formal name of the state stem cell agency, officially known as the California Institute for Regenerative Medicine. Its board includes Jeff Sheehy, who told the California Stem Cell Report that he is not the Jeff commenting on Knoepfler's article.

The anonymous "Jeff" said, 
"Has any of (Knoepfler's) research led to a development candidate that might lead to a treatment in the future? Or is he just a stem cell scientist opining about something he knows very little or nothing about(the FDA)? Maybe he 'heard' something from somebody and that gave him just the hook he needed to attack CIRM and Mills, both of whom he obviously has personal animus towards."
Loring largely sided with Knoepfler. She also wrote,
"Jeff, why are you questioning Paul's motives? What are your motives? Do you work for CIRM? As you know, a person's profession doesn't define his/her sphere of knowledge. If something is important to you, you can research it and gain expertise."
Roberson, who favors the CIRM effort to loosen regulations to bring therapies into widespread use, said patient safety is important. She also said,
"But for patients and their families with a 100 percent fatal genetic disease like Huntington's disease, we can withstand some risk because we are facing a gut-wrenchingly long, gruesome death anyway. We have no chemotherapy, no surgery, no radiation and sadly, no hope. We have NOTHING! And we'll continue to have nothing for many more decades if the FDA continues doing business as usual."
Another reader, William Barnes, said,
"I suggest that Dr. Knoepfler consider how it feels to be suffering and near certain death and be denied a possible cure because Dr. Knoepfler doesn't think it's in their best interest....Let the individual patient decide! It’s their life! Why should the decision about someone’s life, possibly your loved one, be made by a distant and dispassionate academic?"
In addition to his research, Knoepfler produces a blog, The Niche, on stem cell science, regulation and policy. He took up the blog after a nasty bout with prostate cancer when he was in his 40s. Knoepfler is also the author of "Stem Cells: An Insider's Guide," a book aimed at helping patients understand stem cell science. 

Monday, June 27, 2016

The Case of the FDA's $330,000 Stem Cell Pigs

Judy Roberson at podium, Bob Klein, former chairman of the California stem cell agency at left, Claire Pomeroy, former
UC Davis vice chancellor and agency director at right. UC Davis photo
Judy Roberson is a registered nurse from Sacramento, Ca., who has long been active with the California stem cell agency on behalf of patients with Huntington's disease. She lost her husband to the always fatal, inherited brain disease, and members of her family are at risk.

Roberson appeared before the governing board of the $3 billion California Institute for Regenerative Medicine (CIRM), as the agency is formally known, earlier this month to applaud its efforts and those of its CEO, Randy Mills, to persuade the federal Food and Drug Administration to ease its regulation of proposed stem cell therapies. Here is the text of her statement. 

"The Northern California Huntington's disease (HD)  advocacy community says BRAVO to President Randy Mills for his editorial directed at the FDA, "Give Us Our Cures."

"Over 40 HD advocates joined the CIRM Stem Cell Champion campaign in April, promoted by Kevin McCormack (the agency's director of communications), in the hopes that pressure from affected families will prompt the FDA to become open to stem cell therapies and allow the increased risks that naturally go along with new therapies.

"For people with Huntington's disease, which is 100 percent fatal and has zero therapies, we are willing to take on more risk since we're dying anyway.

"The FDA has delayed the fully enrolled, CIRM funded, first-in-human clinical trial using adult stem cells at UC Davis with Drs. Vicki Wheelock and Jan Nolta. The NIH RAC committee enthusiastically approved their novel therapeutic clinical trial. Then the FDA asked for additional animal studies, this time with three pigs at a cost of over $330,000 plus two years of additional research; costs will approach $1 million.

"One HD family from New York has funded one of the pigs, but this gap in funding has shelved this promising research!

"Devastated patients and their families see this add-on research requirement as a delay from the FDA, which means that this fully enrolled trial, with 24 patients who meet today's criteria will progress and may not qualify in two to four years even if this project later receives FDA approval to begin a phase one trial. FDA delays are killing us!

"We need a new FDA 2.0 because doing NOTHING is doing harm!"

Tuesday, March 15, 2016

Going for the Gold: Pitches for $12.5 Million in Rejected Stem Cell Research Applications

Highlights
New scoring system
IOM team member appeal
New information on application

Olympic gold medalist, one of “America’s Top Doctors” and the head of the Scripps Institute’s stem cell program are lobbying the California stem cell agency this week to fund requests for $12.5 million in research grants. 

The two different grants have been rejected by the agency’s blue-ribbon reviewers, who meet behind closed doors and and make decisions without disclosing publicly their financial and professional interests. However, the proposals will come before a public meeting tomorrow of the governing board of the $3 billion agency for official ratification of reviewer actions.

Directors of the agency, officially known as the California Institute for Regenerative Medicine(CIRM), have been loath to override reviewers’ decisions, especially in the past couple of years. Plus this week's applications were considered under a new scoring system, which cuts off funding at a scientific score of 85. In the past, the agency has approved awards that were scored as low as 61.

Jeanne Loring, director of the Center for Regenerative Medicine at Scripps, wrote the board to seek funding for her $8 million application (TRAN1-08468) for a treatment involving Parkinson's disease. The proposal was scored at 70.

But first, here are details on the other application (TRAN1-08527). It seeks $4.5 million for research on a treatment for tendon and ligament injuries, something that the agency has not yet funded, according to the three letters supporting the application. The proposal was scored at 83, two points below the cut off. In the past, board members have noted that such small scoring definitions are statistically insignificant.

The identity of the applicant has not been released by the agency. Its practice is to withhold that information until the board acts, although there are notable exceptions to that policy.
Cato Laurencin, UConn photo

One of the letters of support came from Cato Laurencin, an eminent orthopedic surgeon at the University of Connecticut and who is listed as one of “America’s Top Doctors.” Laurencin also served on the Institute of Medicine’s team that conducted a $700,000 study of the California stem cell agency. 

In the letter dated yesterday, he said hundreds of thousands of persons suffer from tendon and ligament injuries. (All letters are clumped under the same URL.)  Laurencin wrote, 
"This seems to me like a marvelous opportunity to support an excellent study with tremendous potential clinical impact on patients in California and throughout the United States."
Jason Lezak, an Olympic swimmer with four gold medals, said in his letter that there is a "clear need" for legitimate research and treatment for such injuries, given the appeal of untested stem cell treatments attracting patients here and abroad.
 
CIRM document shows that while the application had an overall score of 83, its median score was 85 with scoring ranging from 75 to 92. 

In her March 11 letter, Loring focused on "signicant new information" concerning her application that
Jeanne Loring, Scripps photo
was submitted Nov. 20 of last year. The proposal was not reviewed until Feb. 11.

Loring said, 
"Between November and February, we generated new information that was not available to the GWG(grant review group). Importantly, we also received guidance from the FDA that alleviates the major concerns of the reviewers."
Her nine-page letter itemized reviewer concerns and provided her responses. She wrote, 
"In summary, based on our new data, our DNA sequencing publication, the recent approvals of two of our quality control-focused CIRM grants, and feedback from our meeting with the FDA, we believe that we are ready to proceed on our pilot studies to inform our IND-enabling studies. Some of the GWG concerns conflict with the guidance given by the FDA, and had the GWG been aware of the feedback we had received from the FDA, many of their concerns would have been addressed."
Loring's scientific and median scores were identical: 70. Scoring ranged from 60 to 80. 
David Higgins
 Parkinson's Association photo
The San Diego-based Summit4StemCell group has strongly supported Loring's research and raised funds for it. Representatives from the group have attended a number of CIRM board meetings, laying out the urgency of their needs. One meeting last year became emotional and left some CIRM representatives uneasy and irritated. (See here and here.)

The 29-member CIRM board includes one patient advocate from the Parkinson's community, David Higgins of San Diego. 

Of the $1.9 billion that the agency has handed out, $44 million has gone for Parkinson's. The relatively meager rate of funding was a long a sore point for the first Parkinson's patient advocate on the CIRM board, Joan Samuelson, who has since left the board as her affliction advanced. 

Monday, October 07, 2013

Brown Veto of Spinal Research Funds Triggers Ire

California Gov. Jerry Brown's veto of legislation backed by some stem cell patient advocates has triggered a sharp and negative reaction. One person said he was “disgusted” by the veto. Another person called the governor a “stingy rat.”

It was Roman Reed, a California patient advocate, who said he was disgusted and now planned to run for the state legislature to help find cures for paralyzed persons. Reed said in an interview with the blog ipscell.com:
“Curing the paralyzed and finding medical cures is the most important task of our lives for the health of our loved ones and the economy.  Some fights we just have to win and together we can.”
The legislation in question is AB714 by Assemblyman Bob Wieckowski, D-Fremont. It would have provided $1 million to UC Irvine's Reeve-Irvine Research Center, which has received $15.1 million from similar state appropriations over past years. The money has gone for research into spinal cord injuries and paralysis and has involved human embryonic stem cells. However, funding of the act has expired.

Brown said in his veto message that the measure “strives to do only good.” But the governor said that he has proposed $511 million in additional support for the University of California over the next four years. He indicated that it is now up to the UC system to decide whether it wants to use some of that additional cash for the spinal cord research.

UC Davis researcher Paul Knoepfler wrote about the veto on his blog and carried Reed's remarks. One of the persons commenting on the item identified himself as Brian Sanderson and said,
“I recall reading that Christopher Reeve(the actor who played Superman) said 'I wish I was a rat' when he heard of the progress that had been made repairing spinal cords of rats. Personally, I think that the Governor is behaving like a stingy rat.”
Reed was named as the Knoepfler Lab Stem Cell Person of 2012 for his lobbying efforts here and in Alabama as well as for his mentoring of other advocates. Reed is the son of another stem cell activist, Don Reed, who wrote about the veto on his blog, Stem Cell Battles. The legislation involved in the governor's veto is named after the younger Reed, who was paralyzed as the result of a football accident some years ago.

The legislation indirectly involves directors of the California stem cell agency. The head of the UCI Reeve Center, Oswald Steward, sits on the agency's governing board, along with Sue Bryant, interim provost of UCI, and Sherry Lansing, a former chair of the UC Board of Regents. Roman Reed also came up with the motto of the stem cell agency: “Turning stem cells into cures.”

Sunday, August 11, 2013

'Paradigm Shift' -- Researchers as Patient Advocates?

It is not easy for a stem cell scientist to break into “print” in that bastion of American capitalism, Forbes magazine. Much less one from that perceived antithesis of free enterprise, Reed College.

But researcher Paul Knoepfler of UC Davis has done just that.

John Farrell of Forbes wrote about Knoepfler on Friday in the wake of the announcement Knoepfler will be honored with a “national advocacy” award by the Genetics Policy Institute.
Paul Knoepfler -- Advocate for a "new ethos"
UC Davis photo

Knoepfler, who received a degree in English literature from Reed in 1989, is being recognized for his activities as a stem cell blogger since 2009.

Farrell quoted Knoepfler as saying in an email,
“With many stumbles, face plants, land mines, and even a few threats of litigation and career retaliation along the way for the last three and half a years, I turned my crazy idea into a reality.”
Farrell continued,
“But it was only possible, (Knoepfler) added, with guidance from many patient advocates and bloggers in other fields who generously helped him learn the ropes of blogging.
“'I see this award as a validation of the notion that advocacy by scientists has become a valued part of the stem cell field,' he said.
“'My hope is to catalyze a continuing paradigm shift whereby stem cell scientists and biomedical scientists more generally have a new ethos that not just accepts, but also deeply values advocacy.'”

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