California Biomedical Industry - California Healthcare Institute

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Stem cell science: Putting the tools to excellent use“Problems in human medicine andhuman biology are just plain hard,” saidLawrence S.B. Goldstein, Ph.D., directorof the UC San Diego Stem Cell Programand a key leader in the San DiegoConsortium for Regenerative Medicine.“They take many years of effort to solve.They take many scientists and staffpeople working together to solve them.And they take money.”For all of those reasons, Goldsteinbelieves that California is the place to befor stem cell researchers today.Goldstein was co-chairman of thescientific advisory committee forthe campaign to pass California’sProposition 71, and he said that CIRMhas delivered on its potential. “For thefirst five years, CIRM was tool building,”he said. “The second five years — now —is tool utilization.”The institute has accelerated stem cellresearch — and cemented California asthe innovative leader in the emergingfield — in four key ways, Goldstein said.“First, CIRM is absolutely supportingscientific projects that would not haveotherwise been supported,” he said.“Stem cell research projects are soinnovative that they would not haveattracted federal funding.” For the NIHand other granting federal agencies,“Innovative equals controversial, andcontroversy splits votes on reviewpanels.” Given the intense competitionfor NIH grants, it is understandableand perhaps even appropriate forthe institutes of health to select moretraditional and incremental projects tosupport.Among the projects to receive CIRMgrants has been under John Zaia’sdirection at City of Hope. His teamplans to modify hematopoietic stemcells from HIV patients to make thecells more resistant to HIV. Whenre-infused, these altered cells mayreconstitute the patients’ immunesystems. A similar project throughMichele Calos’ lab at Stanford willattempt to add a missing gene to musclestem cells from boys with Duchenemuscular dystrophy (DMD). The goalis to create new, healthy muscle cellsin the bodies of DMD patients, whosedisease is invariably fatal. If successful,the technique could be applicable inthe treatment of other degenerativedisorders and, perhaps, in degenerativeconditions that occur in normalaging. Another CIRM-funded project,conducted by J. William Langston’slab at The Parkinson’s Institute, aimsto develop iPS cells from patientswith Parkinson’s disease and use theresulting cell lines for Parkinson’sresearch and drug discovery.These investigations, and many othersunderway, are expected to yield newscientific knowledge that could proveuseful to all stem cell researchers.More importantly, CIRM is drivingtranslational development projects tomake breakthrough new treatmentsavailable to patients as soon as is feasible.The second way CIRM has catapultedstem cell research forward has beenthrough a number of financing roundsdesigned to draw new researchers to thefield and to provide the training theyneed to not only get up to speed but tolead the science forward. In fact, theinstitute’s first round of funding was fortraining grants that enabled Californiapublic colleges and universities andnon-profit academic and researchinstitutions to provide stem cellresearch training to pre-doctoralstudents, post-doctoral students andclinical fellows. The programs were tobe available and applicable to studentsfrom scientifically and ethnically diversebackgrounds. Further, the programswere to offer one or more classes instem cell biology as well as a requiredcourse in the social, legal and ethicalimplications of stem cell research.Another round of training financingunderwrote stem cell techniquescourses. Two new faculty roundsfocused on bringing new instructorsinto stem cell programs. The firstfocused on funding young medicalstudents and doctoral scientists asindependent investigators and asfaculty members establishing their ownlabs. The second round paid particularattention to recruiting new physicianscientistsinto the field.Not only are these recruits inspiringundergraduate and graduate studentsto pursue stem cell research, Goldsteinsaid, “These are people who are going tostart and run companies.”Goldstein was instrumental in UC SanDiego being awarded a $1.2 millionCIRM grant to train the next generationof stem cell researchers. The programis providing interdisciplinary trainingin stem cell biology and medicine for16 scientists — six doctoral students,four postdoctoral fellows and six clinicalfellows — enrolled at the School ofMedicine, Division of Biological Sciences,Division of Physical Science, SkaggsSchool of Pharmaceutical Sciences,Jacob’s School of Engineering at UCSD,or at the Salk or Burnham Institutes.94 | California Biomedical Industry 2011 Report
“Only by lowering the barriers to entryinto this important field, can we attractthe very best scientists, ethicists,students, physicians, and fellows tostem cell research,” Goldstein said.At the same time, Goldstein is workingon a research project team with aphysician scientist who moved fromprivate practice to research by wayof a CIRM New Faculty grant. “She isworking at the very nexus of science andclinical practice,” Goldstein said, whichis where she wants to be. Further, hercontributions and insights already areproving invaluable to the team’s work.Third among CIRM’s catalyst roles,Goldstein said, was the formationand issuance of Disease TeamResearch Awards. Established to fundmultidisciplinary teams, these grantsenable teams to conduct the researchand regulatory activities necessaryto prepare and file a complete, wellsupported investigational new drugapplication (IND) with FDA.Goldstein is co-principal investigator ona Disease Team Award aimed at treatingamyotrophic lateral sclerosis (ALS)(see sidebar). He said that the teamsubmitted their idea when it really wasjust an idea. On the wall of his office atUCSD is a Gantt chart that illustratesevery key task to move that idea to theIND delivery date, four years from theproject’s start.“We’re working within incrediblyaggressive timelines,” Goldsteinadmits. “But sometimes things areas slow as you allow them to be.”There is no allowance for slow on theDisease Team projects: should theteam miss a milestone, or run into aninsurmountable research problem, thegrant ends and the remaining fundsrevert to CIRM. If the project succeedsand results in a commercializedtherapy, the grant converts to a loan, tobe paid back to CIRM through royalties.Either outcome ensures some fundingas well as invaluable data for futureresearch programs.The grant’s requirements ensurethat the teams have the businessand management tools they need tosucceed. For instance, CIRM insiststhat the Disease Team Award recipientshire experienced project managers.Goldstein said his team secured onewho insisted that they start talkingto the FDA as early as possible.Considering the agency’s needs andconcerns in the earliest stages of theresearch planning both guides the teamin addressing real-world considerationsand informs the FDA on issues to beprepared for as the programs reachfruition.The interactions have been sovaluable, Goldstein said, that he hasencouraged CIRM to work with thetop administrators at the FDA, toshare collective experiences and beginassembling the regulatory frameworkfor stem cell-related products.The final way that CIRM is ensuringthe commercial future of stem cellbreakthroughs, Goldstein said, isputting researchers and companiestogether on the same teams. Although“that piece is just getting started,”he said, it is a vital component to theemerging sector’s success.“Just getting started” is how Goldsteinalso describes the current state of stemcell research as a field. “There is nocomparison between the early daysand now,” he said, “and we’ve onlyscratched the surface.”The said the technology has becomevery powerful. It will need to be toenable scientists to reach their goal:“To take stem cells and command themto become something and producethem in quantity and at quality toallow for the safe and efficacious use bypatients.”He said the researchers are “gettingcloser” to meeting that goal. Goldstein’sresearch is focused on understandingthe molecular mechanisms ofmovement inside brain cells and howfailures in the movement systems maylead to neurodegenerative diseases. Hislaboratory has discovered importantlinks between transport processesand diseases such as Alzheimer’s andHuntington’s diseases. Given his focus,he may be biased when he says, “Thebrain is really where you see enormousprogress and potential for stem celltechnologies.”Yet given the list of neurologicaldisorders covered by the brain —Alzheimer’s disease ALS, Parkinson’sdisease, Huntington’s disease, lupus,multiple sclerosis, muscular dystrophy,myasthenia, autism, chronic pain,dyslexia, traumatic brain injury, and somany more — one hopes Goldstein iscorrect.Beyond the difficulties inherent infiguring out human biology and waysto use human stem cells to correctmedical problems, Goldstein sees somechallenges yet to be addressed. Yet theone that concerns him most is funding.“It’s always about the money supply,”he said. “As a nation, we woefullyunder-invest in scientific research. Wespend far more on things of far lessconsequence to ourselves. We spendmore on cigarettes as a nation than wespend on scientific research.”For this moment in time, however,California is demonstrating all thatcan be accomplished when expertise,passion, curiosity and, yes, cash, areleveraged to solve scientific puzzles.California Biomedical Industry 2011 Report | 95
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In analyzing California’s biomedi
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Figure 1: Distribution of employmen
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state operations (Figure 16). For t
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of competing products worldwide wer
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Sacramento area shows growth and pr
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Figure 38: California has largest c
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OncologyCancers in California, 2010
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Diagnostics advancementsNew ovarian
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Patient profileBill Walton striding
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families themselves; to institution
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HIV and AIDS statisticsby stateAn e
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Researcher profileDr. Samuel So add
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Immunology and inflammatory disorde
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Cardiovascular diseaseAs is the cas
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