Tuesday, October 29, 2019

California's Big Research Tent: Beyond Stem Cells and Into VROs

California's $3 billion stem cell research program is unprecedented in state history, and it is now ready to mark another first: Backing a couple of clinical "VROs"  with $13.5 million.

VRO is a term that only a policy wonk could love or perhaps a researcher seeking funding under the rubric. It is not exactly posted on the home page of the California Institute for Regenerative Medicine (CIRM), as the agency is formally known. It is nestled comfortably among the 10,000 words of the 2004 ballot initiative that created CIRM.

VRO stands for vital research opportunity. It is fair to say that few persons are aware of the language or know that CIRM's funding authority is something of a big tent that can go well beyond stem cells. Or that CIRM's program could be more expansive under a new, proposed $5.5 billion ballot initiative.

CIRM's governing board set the stage for the awards last November when it approved a VRO process for gene therapy research that did not involve stem cells but involved an aspect of regenerative medicine.

According to the meeting transcript, an application for an award would be considered a VRO if, among other things, "the approach is intended to replace, regenerate, or repair the function of aged, diseased, damaged, or defective cells, tissues, and/or organ. This basically constitutes the definition of regenerative medicine and brings that as a requirement."

A VRO designation also requires a two-thirds vote of the grant reviewers, who conduct their meetings behind closed doors.

On Thursday, the board is expected to approve its first two VRO proposals. One for $8 million targets Parkinson's disease. The summary of the review said the research has "the potential to slow disease progression and provide amelioration of motor symptoms."

The other is a $5.5 million award for treatment of a rare autoimmune disease called IPEX. The summary of the review said the research offered "a valuable alternative to the current standard of care options, which have significant toxic side effects."

VRO awards are permitted under the 2004 ballot measure, which says:
"The institute shall have the following purposes: (a) To make grants and loans for stem cell research, for research facilities, and for other vital research opportunities to realize therapies, protocols, and/or medical procedures that will result in, as speedily as possible, the cure for, and/or substantial mitigation of, major diseases, injuries, and orphan diseases."
Currently on file with state election officials is a proposed ballot initiative that would provide an additional $5.5 billion for the agency, which is soon expected to run out of cash for new awards.

The measure would make a number of changes in CIRM, including permitting the agency to venture even farther afield than permitted under the 2004 language.

The measure would impose a new requirement that the agency support training programs for "careers in stem cell research and other vital research opportunities." It would establish experience in "other vital research opportunities" as acceptable criteria for the selection of governing board members and the chair of CIRM. The criteria would apply also to the grant review group and appointments to a new scientific advisory board.

The proposed ballot measure also defines a VRO as including personalized medicine, genetics and aging. Here is the proposed, new language:
"Vital research opportunity means scientific and medical research and technologies, includinq but not limited to qenetics, personalized medicine, and aqinq as a patholoqy, and/or any stem cell research not actually funded by the institute under paragraph (3) of subdivision (c) of Section 125290.60 which provides a substantially superior research opportunity, vital to advance medical science as determined by at least a two-thirds vote of a quorum of the members of the Scientific and Medical Research Funding Working Group (reviewers) and recommended as such by that working group to the ICOC (the governing board),or as determined by the vote of a majority of a quorum of members of the ICOC. Human reproductive cloning shall not be a vital research opportunity."
The language still can be modified by the initiative backers prior to being certified for the November 2020 ballot. Here is how that mechanism works. 

1 comment:

  1. Anonymous9:29 AM

    In other words...no longer a stem cell agency. Now it's the West Coast, California taxpayer-funded NIH.


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