Saturday, May 30, 2020
Friday, May 29, 2020
"“These three projects highlight three very different approaches to combatting the acute devastating health manifestations of Covid-19 as well as the debilitating sequelae that impact the ability to recover from the acute illness. Through this Covid funding opportunity, CIRM is enabling researchers to re-direct work they have already done, often with CIRM support, to quickly develop new approaches to Covid-19."
"If anything is known for sure about the scientific battle against the novel coronavirus, it’s that the quest for a vaccine has been unprecedentedly intense, with rapid development and speedy production the paramount goals."
"The so-called Cutter incident was 'one of the worst biological disasters in American history, exploded the myth of the invulnerability of science and destroyed faith in the vaccine enterprise,' observed Paul Offit, a vaccine expert at the University of Pennsylvania whose 2005 book is the definitive account of the episode."
"Public confidence in the vaccine was shattered. 'The summer of 1955 came and went with few children getting their shots,' historian David Oshinsky, a co-author of the JAMA article, recounted in his 2005 history of the epidemic. Major outbreaks occurred in Boston and Chicago. 'It seemed like old times, with beaches and movie theaters once again deserted and people fleeing the cities to escape the evil germs.'"
"Clinical trials won’t be the final hurdle in bringing vaccines to the world. Managing the public’s expectations will be crucial, since no vaccine for anything is 100% effective. So will ensuring that the manufacturing process is airtight."With anti-vaxxers and conspiracy theorists already spreading wild stories about the coronavirus, it would be a shame to give them more ammunition."'In this world, where there’s so much vaccine hesitancy and doubt,' Caplan said, 'you better get it right, out of the box. You can’t afford a failure. Even knowing that we’re in desperate need of a vaccine and the deaths are happening, I worry that if we don’t do it right, it’s going to be worse.'"
Thursday, May 28, 2020
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:
"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 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.
Wednesday, May 27, 2020
Tuesday, May 26, 2020
"We should be realistic about how that may look good on our website...but the reality is that this whole project (is) not going to have major, short-term impact barring some wonderful serendipity."And, and to come back to the original trust of the voters of California in CIRM, I really do think that we are deviating from our mission and ought not dilute our impact, which is so necessary and so vital."
"As an agency we're taking a much broader and much more sophisticated view of what our mission is. It's morphed. If we were being true to our original mission, we would only be doing embryonic stem cell research....(T)hat's what we the voters asked us to do in 2004."
"To sum up, I'm not really sure what the board is trying to accomplish with its Covid program. I'm not sure that the board has a clear, coherent view of the scope of CIRM's research. And I am not sure the board has a clear idea of what the scientific mission of CIRM should be in the event that new funding comes from the voters."
The discussion at the May 23, 2020, meeting of the governing board of the California stem cell agency ranged from Covid-19 and the agency's strapped financial condition to its mission and priorities. One of the directors, Jeff Sheehy, elaborated on the matters and more when he responded to an inquiry following the meeting from the California Stem Cell Report.
The California Stem Cell Report has a policy of running verbatim comments from stem cell agency board members and other interested parties. If other readers would like to submit their comments on this subject or other stem cell matters, please direct them to email@example.com.
Below is the verbatim text of Sheehy's comments.
"First, I felt a great deal of uncertainty around CIRM proposing a COVID program. Having worked towards stem cell therapies for HIV since I came onto the board in 2004, I felt some skepticism around stem cell approaches targeting infectious diseases (ID). Infectious disease has not been a particularly major focus of regenerative medicine efforts and many of the approaches we have tried have struggled. I think there could be a role, but we have a long way to go.
"I thought, in general, a focus on ID is a bit overdue, but I was very concerned about taking funds from sickle cell, where I think there is a strong case for believing we are on track for a cure using stem cell technology, and there are well constructed clinical trials such as the one we just funded.
"Given that CIRM is using its last of its funding, I was also concerned about the funding needed. One of the most telling moments in Friday's meeting was the descriptions by (CIRM Director) Dr. (Keith) Yamamoto (vice chancellor for science policy at UCSF) of the ready and ample availability of Covid funding from the NIH and other federal sources, not just in new funds but also the ability to repurpose existing grants towards Covid.
"Btw, I am not alone in my skepticism around stem cells for Covid-19. I offer first the tweet from Dr. Sean Morrison, former head of ISSCR on March 29:
"'There is no stem cell therapy for Covid19. We would not expect stem cells to have any therapeutic value for people with Covid19.
"Then a couple of weeks ago, Dr. Paul Knoepfler on his blog, the NICHE, offered this:
"'The idea of testing stem cells for Covid-19 may be music to the ears of some folks as an opportunity, but to me from the beginning it sounded mostly like a spaghetti on the wall road to trouble.
"'There is buzz out there that some kind of stem cells or other cells will help with COVID-19. The reality is that that’s probably not going to happen.
"'Even so, a whole range of people and firms are somewhat exaggerating and in a few cases outright hyping the odds of success. That is harming patients and the cell medicine field.'"
"I offer these two comments in support of my doubts. But, I also feel the urgency surrounding Covid. This is my second pandemic, having lived through HIV/AIDS and lost and continue to lose countless people that I care deeply about and living with the disease myself, having endured enormous stigma and outright hatred along with indifference from a president, seeing marginalized communities suffering death and disease disproportionately and indifference because people don't recognize the humanity of the populations most impacted, feeling triggered and re-traumatized by this second pandemic and the enormous stupidity from all points in trying to make a disease fit their self described perceptions of reality in the face of what is real and undeniable.
"I voted to support the COVID program despite my doubts due to urgency, but we know what tools work against infectious diseases -- antivirals and vaccines, and I became an enthusiastic and vocal supporter of the vital research opportunity for convalescent plasma. Convalescent plasma is about as far removed from CIRM as a project could be--a technology at least 100 years old with zero barriers to receiving funding from any source. But, in the absence of a vaccine or antivirals, it represented to me the best and most rational chance to make a difference in patients now. Plus, Zaia et al at City of Hope have the cell handling and manufacturing ability to address some of the factors limiting use and evaluation of convalescent plasma, e.g. potency and consistency of product to name a couple.
"After having participated in reviews, our two clinical projects are the convalescent plasma one and an ongoing $14 million trial for mesenchymal stem cells for ARDS, the lung complication suffered by critical COVID patients necessitating ventilator support. That UCSF/UC Davis trial is already treating COVID patients at SF General and in Houston to name a couple of sites, and it was supportable because we were availing more patients of access to that therapy like we are doing with convalescent plasma. But both approaches are well supported outside of CIRM and the $750K we gave each project.
"The $150K Discovery projects we funded are interesting, but they are very early in development and given the time and money it takes to move from discovery into translation into clinical trials, these antiviral products could likely be superseded by the host of antiviral products in trials around the world.
In that context, we did see a couple of interesting vaccine proposals that were very far afield and did not score in the fundable range. I had a feeling -- having looked at the vaccine trials underway (and I'm not an expert btw) -- that they seemed very much based in the most part on proven tech that a far-out, left field approach might be useful to explore. HIV has been stubbornly resistant to vaccine development, so I'm very cautious about that field. I was heartened to hear (CIRM Director) Dr. (Kristiina) Vuori from Sanford Burnham opine that she thought that current vaccine approaches were, to paraphrase, following usual pathways, and I thought I heard a suggestion that opening up CIRM to vaccine work might elicit something novel.
"However, the board thought otherwise and I defer to their wisdom.
"I would note that I was intrigued by the discussion around CIRM's mission. As Dr. (Gil) Sambrano (CIRM vice president for portfolio development) noted, stem cells are ubiquitous in the body, and I took that to mean that one could either be elastic or inelastic in accepting products. A vaccine, for instance, would almost necessarily impact T memory stem cells, which are a crucial part of the adaptive immune response to disease (and a vaccine). We have begun to recognize the limits of stem cell centric therapies through our VRO for gene therapy.
"I then asked myself, does CIRM hews to its original mission per Prop. 71? I looked up the original funding mission for the Grants Working Group :
"'(C) In order to ensure that institute funding does not duplicate or supplant existing funding, a high priority shall be placed on funding pluripotent stem cell and progenitor cell research that cannot, or is unlikely to, receive timely or sufficient federal funding, unencumbered by limitations that would impede the research. In this regard, other research categories funded by the National Institutes of Health shall not be funded by the institute....Notwithstanding subparagraph (C), other scientific and medical research and technologies may be funded by the institute if at least two-thirds of a quorum of the members of the Scientific and Medical Research Funding Working Group recommend to the ICOC that such a research proposal is a vital research opportunity.'
"With the federal ban ended, all of CIRM's research is really a vital research opportunity by default at this point. Per COVID and ample federal funding, any funding in that arena is about as far afield as one could get.
"Even with fetal tissue research, which, btw, has fallen off the radar in the media and with some in Congress and the Administration, I can guarantee no one is talking about banning that. You simple cannot develop vaccines or antivirals without mice with humanized immune systems created by using fetal tissue.
"To sum up, I'm not really sure what the board is trying to accomplish with its COVID program. I'm not sure that the board has a clear, coherent view of the scope of CIRM's research. And I am not sure the board has a clear idea of what the scientific mission of CIRM should be in the event that new funding comes from the voters.
"I would note as an aside that CIRM has never submitted its scientific program, including all grants made and their impact, to a rigorous, independent scientific review.
"In short, I don't think we really know at CIRM where we've been and where we want to go. We have anecdotes...."
(Sheehy’s own ellipses in the last paragraph)
Saturday, May 23, 2020
Friday, May 22, 2020
"All of my CIRM awards contributed to (Aspen). We used those funds to develop our genomics tools and databases and to develop improved methods for handling the cells."
Thursday, May 21, 2020
"Emergency" Meeting Tomorrow Morning; California Stem Cell Agency Wants to Expand Scope of its Covid Awards
Wednesday, May 20, 2020
"The current standard of care for severe sickle cell disease is a blood stem cell transplant, which is only available to patients who have a matched donor.
“However, the unique properties of ECT-001 cell therapy enable patients without a matched donor to receive treatment, with a greatly reduced risk of post-transplant complications. This study introduces a new indication into the ongoing evaluation of our ECT-001 technology, which we hope will make this life-saving therapy available to a far greater number of severely ill patients."
Monday, May 18, 2020
Saturday, May 16, 2020
Friday, May 15, 2020
The CIRM governing board rejected Li's and Celularity's applications, but they may resubmit them next Tuesday with modifications. Their scores and rankings can be found here along with the summary of their reviews, as well as summaries of all reviews.
The stem cell agency, officially known as the California Institute for Regenerative Medicine(CIRM), is expected to run out of cash this year. It is hoping that a $5.5 billion, refinancing proposal will qualify for the ballot this fall and be approved by voters. Currently, election officials are trying to determine whether the measure as the necessary number of signatures of registered voters to qualify.