By Heather Main
Mesa, one of my first Spanish words since moving to California. The isolated flat-topped hill with steep sides where I have just had the pleasure of listening to some of the best basic and applied science. Mountains of various sorts formed the metaphor of CIRM 2.0 with Randy Mills (President/CEO of CIRM) describing the ‘giant boulder of love and happiness’ that academia pushes and industry pulls over the mountain of regulation, safety and efficacy bringing cures to patients. The streamlining of regulation makes the mountain smaller to decrease both the push and pull needed.
The meeting was a nice mix of academic and applied research, highly evident was the drive that CIRM funding has ignited in California. Of interest, after 5 years of 91% academic CIRM funding, the next 5 years is focusing towards critical funding towards industry translational projects…CIRM 2.0
Listening to Elaine Fuchs makes you question your worth in the field and when this woman will get a Nobel Prize for her contributions to stem cell sciences. The number of ‘Cell’ and ‘Nature’ references with her name on it in her talk demonstrate her unique ability to conquer the highest levels of science consistently throughout her career. A career spanning therapeutic skin stem cell application to stem cell niche determination, quiescent stem cell identification and now cancer stem cell quiescence, the lessons outlined by Elaine’s Keynote address form a foundation basis for stem cell sciences in both cellular therapy and cancer applications.
Stephanie Cherqui (at right), discussed the nanotubular highways for intercellular organelle transport with HSC cellular therapies that lead to macrophage differentiation and delivery of healthy lysosomes for Cystinosis disease rescue phenotypes.
Samuel Pfaff talked about his lab’s identification of miR218 as the most specific motor neuron marker known to date and the mechanistic possibility of its loss of activity, leading to an increase in gene expression deleterious to motor neuron survival, a possible mechanism for ALS.
Shyni Varghese (at left), gave a great talk on production of bone from endogenously recruited cells using hydrogels engineered to bind minerals. The minerals were sufficient to produce bone in acellular scaffolds, while absence of mineral loading lead to fat formation. There was even novelty in Isaac Newton shaped bone deposit.
Pilar Ruiz-Lozano discussed amazing results using FSTL1 loaded collagen matrix to rescue heart regeneration following infarction, from mouse to pigs. The specific activity of FSTL1 from epicardium was essential to this regenerative function, in contrast to non-regenerative glycosylated FSTL1 produced in response to injury by the myocardium.
Joseph Metzger discussed stabilization of Duchenne Muscular Dystrophy dystrophic muscle membranes. These Poloxamer 188 ‘band-aids’ strengthen weaknesses in dystrophic membranes to avoid catastrophic Calcium entry that leads to cellular death. While super interesting, this is really a band-aid treatment, as Joseph says, just like Type 1 Diabetes, there is still room for gene and cellular therapy based cures.
The most confusing part of the meeting for me was the Thursday night ‘public forum’. I’m not sure what this means in California but the public forums I have attended have a majority of audience members from the public and attempt to pass knowledge and excitement about stem cells to a lay audience. While there was some fantastic science, I don’t think I would have understood a word in regard to ‘The promise of regenerative medicine: are we there yet?’ if I was a member of the public.
As my first time at Stem Cells on the MESA I was really impressed by the attitude I sensed, that solid science combined with effective clinical and commercial translation strategies are essential to successes in our field. That basic research is not enough. Second, it was nice to see an equal and unbiased appreciation for material sciences applications, gene therapy treatments, cellular therapy applications and endogenous repair applications. Whatever gets to the post first should ‘win’ for the sake of the patient.