SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE: Form 990, tax year 2012

View the entire original 990 at ProPublica ↗

Organization: SANFORD BURNHAM PREBYS MEDICAL DISCOVERY INSTITUTE (EIN 51-0197108)
Tax period: 2012-07-01 to 2013-06-30
Filed: 2014-05-12
IRS object ID: 201411329349303161
Schema version: 2012v2.3
Source: Download XML · Original 990 at ProPublica

Every field in the e-filed return. Faint text is the IRS element name, for citing.

Return header
Form 990
Schedule A
Schedule B
Schedule C
Schedule D
Schedule F
Schedule G
Schedule J
Schedule K
Schedule L
Schedule M
Schedule O

Return header ReturnHeader

Field	Value	IRS element (for citing)
Timestamp	2014-05-12T14:08:39-05:00	Timestamp
Tax Period End Date	2013-06-30	TaxPeriodEndDate

Preparer Firm PreparerFirm

Preparer Firm Business Name PreparerFirmBusinessName

Field	Value	IRS element (for citing)
Business Name Line1	ERNST & YOUNG US LLP	BusinessNameLine1

Preparer Firm US Address PreparerFirmUSAddress

Field	Value	IRS element (for citing)
Address Line1	4370 LA JOLLA VILLAGE DRIVE STE 500	AddressLine1
City	LA JOLLA	City
State	CA	State
ZIP Code	92122	ZIPCode

Field	Value	IRS element (for citing)
Return Type	990	ReturnType
Tax Period Begin Date	2012-07-01	TaxPeriodBeginDate

Filer Filer

Field	Value	IRS element (for citing)
EIN	510197108	EIN

Name Name

Field	Value	IRS element (for citing)
Business Name Line1	SANFORD-BURNHAM MEDICAL RESEARCH INSTITUTE	BusinessNameLine1

Field	Value	IRS element (for citing)
In Care Of Name	% GARY F RAISL EDD EVP CAO CFO	InCareOfName
Name Control	SANF	NameControl
Phone	8586463100	Phone

US Address USAddress

Field	Value	IRS element (for citing)
Address Line1	10901 NORTH TORREY PINES ROAD	AddressLine1
Address Line2	Suite	AddressLine2
City	LA JOLLA	City
State	CA	State
ZIP Code	92037	ZIPCode

Officer Officer

Field	Value	IRS element (for citing)
Name	GARY F RAISL EDD	Name
Title	EVP/CAO/CFO	Title
Date Signed	2014-05-06	DateSigned

Preparer Preparer

Field	Value	IRS element (for citing)
Phone	8585357200	Phone

Field	Value	IRS element (for citing)
Tax Year	2012	TaxYear
Build TS	2015-11-30 17:44:51Z	BuildTS

Form 990 IRS990

Field	Value	IRS element (for citing)
Name Of Principal Officer Person	KRISTIINA VUORI MD PHD	NameOfPrincipalOfficerPerson

Address Principal Officer US AddressPrincipalOfficerUS

Field	Value	IRS element (for citing)
Address Line1	10901 N TORREY PINES ROAD	AddressLine1
City	LA JOLLA	City
State	CA	State
ZIP Code	92037	ZIPCode

Field	Value	IRS element (for citing)
Gross Receipts	162967633	GrossReceipts
Group Return For Affiliates	false	GroupReturnForAffiliates
Organization501c3	X	Organization501c3
Web Site	WWW.SANFORDBURNHAM.ORG	WebSite
Type Of Organization Corporation	X	TypeOfOrganizationCorporation
Year Formation	1976	YearFormation
State Legal Domicile	CA	StateLegalDomicile
Activity Or Mission Description	SEE SCHEDULE O	ActivityOrMissionDescription
Nbr Voting Members Governing Body	20	NbrVotingMembersGoverningBody
Nbr Independent Voting Members	17	NbrIndependentVotingMembers
Total Nbr Employees	1130	TotalNbrEmployees
Total Nbr Volunteers	22	TotalNbrVolunteers
Total Gross UBI	915264	TotalGrossUBI
Net Unrelated Business Txbl Income	144833	NetUnrelatedBusinessTxblIncome
Contributions Grants Prior Year	156880684	ContributionsGrantsPriorYear
Contributions Grants Current Year	146497371	ContributionsGrantsCurrentYear
Program Service Revenue Prior Year	774874	ProgramServiceRevenuePriorYear
Program Service Revenue CY	1077105	ProgramServiceRevenueCY
Investment Income Prior Year	1048418	InvestmentIncomePriorYear
Investment Income Current Year	1074044	InvestmentIncomeCurrentYear
Other Revenue Prior Year	3393059	OtherRevenuePriorYear
Other Revenue Current Year	4061676	OtherRevenueCurrentYear
Total Revenue Prior Year	162097035	TotalRevenuePriorYear
Total Revenue Current Year	152710196	TotalRevenueCurrentYear
Grants And Similar Amnts Prior Year	0	GrantsAndSimilarAmntsPriorYear
Grants And Similar Amnts CY	0	GrantsAndSimilarAmntsCY
Benefits Paid To Members Prior Year	0	BenefitsPaidToMembersPriorYear
Benefits Paid To Members CY	0	BenefitsPaidToMembersCY
Salaries Etc Prior Year	82248203	SalariesEtcPriorYear
Salaries Etc Current Year	79343211	SalariesEtcCurrentYear
Total Prof Fundrsng Exp Prior Year	196445	TotalProfFundrsngExpPriorYear
Total Prof Fundrsng Exp CY	33225	TotalProfFundrsngExpCY
Total Fundrsng Exp Current Year	2463153	TotalFundrsngExpCurrentYear
Other Expense Prior Year	75933118	OtherExpensePriorYear
Other Expenses Current Year	69599654	OtherExpensesCurrentYear
Total Expenses Prior Year	158377766	TotalExpensesPriorYear
Total Expenses Current Year	148976090	TotalExpensesCurrentYear
Revenues Less Expenses Prior Year	3719269	RevenuesLessExpensesPriorYear
Revenues Less Expenses CY	3734106	RevenuesLessExpensesCY
Total Assets BOY	316383458	TotalAssetsBOY
Total Assets EOY	315253706	TotalAssetsEOY
Total Liabilities BOY	109159237	TotalLiabilitiesBOY
Total Liabilities EOY	100215489	TotalLiabilitiesEOY
Net Assets Or Fund Balances BOY	207224221	NetAssetsOrFundBalancesBOY
Net Assets Or Fund Balances EOY	215038217	NetAssetsOrFundBalancesEOY
Info In Schedule O Part III	X	InfoInScheduleOPartIII
Mission Description	SANFORD BURNHAM MEDICAL RESEARCH INSTITUTE CONDUCTS WORLD-CLASS COLLABORATIVE RESEARCH DEDICATED TO FINDING CURES FOR HUMAN DISEASE, IMPROVING THE QUALITY OF LIFE, AND THUS CREATING A LEGACY FOR ITS EMPLOYEES, PARTNERS, DONORS, AND COMMUNITY.	MissionDescription
Significant New Program Services	false	SignificantNewProgramServices
Significant Change	false	SignificantChange
Expense	129077713	Expense
Grants	0	Grants
Revenue	1502152	Revenue
Description	SEE SCHEDULE O	Description
Total Program Service Expense	129077713	TotalProgramServiceExpense
Described In501 C3	true	DescribedIn501C3
Schedule B Required	true	ScheduleBRequired
Political Activities	false	PoliticalActivities
Lobbying Activities	true	LobbyingActivities
Subject To Proxy Tax	false	SubjectToProxyTax
Donor Advised Funds	false	DonorAdvisedFunds
Conservation Easements	false	ConservationEasements
Collections Of Art	false	CollectionsOfArt
Credit Counseling	false	CreditCounseling
Term Or Permanent Endowments	true	TermOrPermanentEndowments
Report Land Bldg Equip	true	ReportLandBldgEquip
Report Invest Oth Securities	false	ReportInvestOthSecurities
Report Prog Rel Invest	false	ReportProgRelInvest
Report Other Assets	false	ReportOtherAssets
Report Other Liabilities	false	ReportOtherLiabilities
Report Fin48 Footnote	true	ReportFin48Footnote
Independent Audit Financial Stmt	true	IndependentAuditFinancialStmt
Consolidated Audit Financial Stmt	false	ConsolidatedAuditFinancialStmt
School	false	School
Foreign Office	true	ForeignOffice
Foreign Activities	true	ForeignActivities
More Than5000 K To Organizations	false	MoreThan5000KToOrganizations
More Than5000 K To Individuals	false	MoreThan5000KToIndividuals
Professional Fundraising	true	ProfessionalFundraising
Fundraising Activities	true	FundraisingActivities
Gaming	false	Gaming
Hospital	false	Hospital
Grants To Organizations	false	GrantsToOrganizations
Grants To Individuals	false	GrantsToIndividuals
Schedule J Required	true	ScheduleJRequired
Tax Exempt Bonds	true	TaxExemptBonds
Invest Tax Exempt Bonds	false	InvestTaxExemptBonds
Escrow Account	false	EscrowAccount
On Behalf Of Issuer	false	OnBehalfOfIssuer
Excess Benefit Transaction	false	ExcessBenefitTransaction
Prior Excess Benefit Transaction	false	PriorExcessBenefitTransaction
Loan To Officer Or DQP	true	LoanToOfficerOrDQP
Grant To Related Person	false	GrantToRelatedPerson
Bsnss Rltnshp With Organization	false	BsnssRltnshpWithOrganization
Bsnss Rltnshp Thru Family Member	true	BsnssRltnshpThruFamilyMember
Officer Entity With Bsnss Rltnshp	true	OfficerEntityWithBsnssRltnshp
Deductible Non Cash Contributions	true	DeductibleNonCashContributions
Deductible Contributions Of Art	false	DeductibleContributionsOfArt
Terminated	false	Terminated
Partial Liquidation	false	PartialLiquidation
Disregarded Entity	false	DisregardedEntity
Related Entity	false	RelatedEntity
Related Org Controlled Entity	false	RelatedOrgControlledEntity
Transfers To Exempt Non Chrtbl Org	false	TransfersToExemptNonChrtblOrg
Activities Conducted Partnership	false	ActivitiesConductedPartnership
Schedule O Required	true	ScheduleORequired
Number Forms Transmitted With1096	222	NumberFormsTransmittedWith1096
Number W2 G Included	0	NumberW2GIncluded
Compliance With Backup Witholding	true	ComplianceWithBackupWitholding
Number Of Employees	1130	NumberOfEmployees
Employment Tax Returns Filed	true	EmploymentTaxReturnsFiled
Unrelated Business Income	false	UnrelatedBusinessIncome
Foreign Financial Account	false	ForeignFinancialAccount
Prohibited Tax Shelter Trans	false	ProhibitedTaxShelterTrans
Taxable Party Notification	false	TaxablePartyNotification
Non Deductible Contributions	false	NonDeductibleContributions
Quid Pro Quo Contributions	true	QuidProQuoContributions
Quid Pro Quo Disclosure	true	QuidProQuoDisclosure
Form8282 Property Disposed Of	false	Form8282PropertyDisposedOf
Funds To Pay Premiums	false	FundsToPayPremiums
Premiums Paid	false	PremiumsPaid
Indoor Tanning Services	false	IndoorTanningServices
Info In Schedule O Part VI	X	InfoInScheduleOPartVI
Nbr Voting Governing Body Members	20	NbrVotingGoverningBodyMembers
Number Independent Voting Members	17	NumberIndependentVotingMembers
Family Or Business Relationship	false	FamilyOrBusinessRelationship
Delegation Of Management Duties	false	DelegationOfManagementDuties
Changes To Organizing Docs	false	ChangesToOrganizingDocs
Material Diversion Or Misuse	false	MaterialDiversionOrMisuse
Members Or Stockholders	false	MembersOrStockholders
Election Of Board Members	false	ElectionOfBoardMembers
Decisions Subject To Approval	false	DecisionsSubjectToApproval
Minutes Of Governing Body	true	MinutesOfGoverningBody
Minutes Of Committees	true	MinutesOfCommittees
Officer Mailing Address	false	OfficerMailingAddress
Local Chapters	false	LocalChapters
Form990 Provided To Governing Body	true	Form990ProvidedToGoverningBody
Conflict Of Interest Policy	true	ConflictOfInterestPolicy
Annual Disclosure Covered Persons	true	AnnualDisclosureCoveredPersons
Regular Monitoring Enforcement	true	RegularMonitoringEnforcement
Whistleblower Policy	true	WhistleblowerPolicy
Document Retention Policy	true	DocumentRetentionPolicy
Compensation Process CEO	false	CompensationProcessCEO
Compensation Process Other	false	CompensationProcessOther
Investment In Joint Venture	false	InvestmentInJointVenture
States Where Copy Of Return Is Filed	AR	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	CA	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	CT	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	KS	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	ME	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	MD	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	MA	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	MI	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	NH	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	NJ	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	NY	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	NC	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	OH	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	OR	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	PA	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	RI	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	UT	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	VA	StatesWhereCopyOfReturnIsFiled
States Where Copy Of Return Is Filed	WA	StatesWhereCopyOfReturnIsFiled
Upon Request	X	UponRequest

The Books Are In Care Of TheBooksAreInCareOf

Field	Value	IRS element (for citing)
Name Person	GARY F RAISL EDD EVP CAO CFO	NamePerson
Telephone Number	8586463100	TelephoneNumber

Address US AddressUS

Field	Value	IRS element (for citing)
Address Line1	10901 N TORREY PINES ROAD	AddressLine1
City	LA JOLLA	City
State	CA	State
ZIP Code	92037	ZIPCode

Form990 Part VII Section A Form990PartVIISectionA (31 entries)

TitleTitle	Average Hours Per WeekAverageHoursPerWeek	Individual Trustee Or DirectorIndividualTrusteeOrDirector	Reportable Comp From OrganizationReportableCompFromOrganization	Reportable Comp From Related OrgsReportableCompFromRelatedOrgs	Other CompensationOtherCompensation	OfficerOfficer	Average Hours Per Week RelatedAverageHoursPerWeekRelated	Highest Compensated EmployeeHighestCompensatedEmployee
LORENZO BERHO
TRUSTEE	1.0	X	0	0	0
JAMES BLAIR
TRUSTEE	1.0	X	0	0	0
SHEHAN DISSANAYAKE
TRUSTEE	1.0	X	0	0	0
DANIEL EPSTEIN
TRUSTEE	1.0	X	0	0	0
WAIN FISHBURN
TRUSTEE/CHAIRMAN	1.0	X	0	0	0	X
PAULINE FOSTER
TRUSTEE	1.0	X	0	0	0
PATRICK GERAGHTY
TRUSTEE	1.0	X	0	0	0
ALAN GLEICHER
TRUSTEE	1.0	X	0	0	0
JEANNE HERBERGER
TRUSTEE	1.0	X	0	0	0
BRENT JACOBS
TRUSTEE	1.0	X	0	0	0
JAMES JARDON
TRUSTEE	1.0	X	0	0	0
ROBERT LAUER
TRUSTEE	1.0	X	0	0	0
BERNARD MACHEN
TRUSTEE	1.0	X	0	0	0
NICOLAS NIERENBERG
TRUSTEE	1.0	X	0	0	0
HENRY NORDOFF
TRUSTEE	1.0	X	0	0	0
DOUGLAS OBENSHAIN
TRUSTEE	1.0	X	0	0	0
PETER PREUSS
TRUSTEE	1.0	X	0	0	0
DUANE ROTH
TRUSTEE	1.0	X	0	0	0
JAN TUTTLEMAN
TRUSTEE	1.0	X	0	0	0
ANDREW VITERBI
TRUSTEE	1.0	X	0	0	0
ALLEN WEISS
TRUSTEE	1.0	X	0	0	0
GAYLE WILSON
TRUSTEE	1.0	X	0	0	0
JOHN C REED MD PHD
CEO	50.0	X	680710	0	400613	X
KRISTIINA VUORI MD PHD
PRESIDENT/INTERIM CEO	50.0	X	614402	0	336101	X
GARY F RAISL EDD
EVP/CAO/CFO/TREASURER	50.0		432812	0	48941	X
MARGARET DUNBAR JD
SECRETARY/SR DIRECTOR IP	50.0		165701	0	15321	X	0.0
STUART LIPTON MD PHD
CENTER DIRECTOR	50.0		448146	0	52739			X
DANIEL KELLY MD
CNTR DIR./SCI. DIR. AT LK NONA	50.0		446021	0	50001		0.0	X
CARL WARE PHD
CENTER DIRECTOR	50.0		347871	0	34808			X
CYRIL DOUCET
VP, ADMIN. AT LAKE NONA	50.0		307670	0	35312			X
JORGE MOSCAT PHD
PROGRAM DIRECTOR	50.0		279856	0	45878			X

Field	Value	IRS element (for citing)
Total Reportable Comp From Org	3723189	TotalReportableCompFromOrg
Total Reportable Comp Frm Rltd Orgs	0	TotalReportableCompFrmRltdOrgs
Total Other Compensation	1019714	TotalOtherCompensation
Number Individuals GT100 K	128	NumberIndividualsGT100K
Formers Listed	false	FormersListed
Total Comp GT150 K	true	TotalCompGT150K
Compensation From Other Sources	false	CompensationFromOtherSources

Contractor Compensation ContractorCompensation (5 entries)

Address Line1AddressOfContractor/AddressUS/AddressLine1	CityAddressOfContractor/AddressUS/City	StateAddressOfContractor/AddressUS/State	ZIP CodeAddressOfContractor/AddressUS/ZIPCode	Description Of ServicesDescriptionOfServices	CompensationCompensation
Pabst Patent Group
1545 Peachtree St NE Suite 320	ATLANTA	GA	30309	LEGAL	498923
DLA Piper US LLP
PO BOX 64029	BALTIMORE	MD	21264	LEGAL	459291
Deloitte Touche
200 South Orange Suite 200	SAN DIEGO	CA	92130	AUDIT	297488
Berman Property Management
100 W Lucerne Circle Suite 403	ORLANDO	FL	32801	Janitorial	215592
Jones Day
12265 El Camino Real Suite 200	SAN DIEGO	CA	92130	Legal	200927

Field	Value	IRS element (for citing)
Number Of Contractors GT100 K	7	NumberOfContractorsGT100K
Fundraising Events	1090045	FundraisingEvents
Government Grants	110044226	GovernmentGrants
All Other Contributions	35363100	AllOtherContributions
Noncash Contributions	1063332	NoncashContributions
Total Contributions	146497371	TotalContributions

Program Service Revenue ProgramServiceRevenue

Field	Value	IRS element (for citing)
Description	Shared Services revenue	Description
Business Code	541900	BusinessCode
Total Revenue Column	1077105	TotalRevenueColumn
Related Or Exempt Function Income	1077105	RelatedOrExemptFunctionIncome

Field	Value	IRS element (for citing)
Total Program Service Revenue	1077105	TotalProgramServiceRevenue

Investment Income InvestmentIncome

Field	Value	IRS element (for citing)
Total Revenue Column	919164	TotalRevenueColumn
Exclusion	$919,164	ExclusionAmount

Income From Invest Bond Proceeds IncomeFromInvestBondProceeds

Field	Value	IRS element (for citing)
Total Revenue Column	44371	TotalRevenueColumn
Exclusion	$44,371	ExclusionAmount

Royalties Revenue RoyaltiesRevenue

Field	Value	IRS element (for citing)
Total Revenue Column	2582193	TotalRevenueColumn
Exclusion	$2,582,193	ExclusionAmount

Gross Rents GrossRents

Field	Value	IRS element (for citing)
Real	454672	Real

Rental Income Or Loss RentalIncomeOrLoss

Field	Value	IRS element (for citing)
Real	454672	Real
Personal	0	Personal

Net Rental Income Or Loss NetRentalIncomeOrLoss

Field	Value	IRS element (for citing)
Total Revenue Column	454672	TotalRevenueColumn
Exclusion	$454,672	ExclusionAmount

Gross Amount Sales Assets GrossAmountSalesAssets

Field	Value	IRS element (for citing)
Securities	10003466	Securities

Less Cost Oth Basis Sales Expenses LessCostOthBasisSalesExpenses

Field	Value	IRS element (for citing)
Securities	9892957	Securities

Gain Or Loss GainOrLoss

Field	Value	IRS element (for citing)
Securities	110509	Securities

Net Gain Or Loss Investments NetGainOrLossInvestments

Field	Value	IRS element (for citing)
Total Revenue Column	110509	TotalRevenueColumn
Exclusion	$110,509	ExclusionAmount

Field	Value	IRS element (for citing)
Gross Income Fundraising Events	49000	GrossIncomeFundraisingEvents
Cntrbtns Rprtd Fundraising Events	1090045	CntrbtnsRprtdFundraisingEvents
Fundraising Direct Expenses	364480	FundraisingDirectExpenses

Net Income From Fundraising Events NetIncomeFromFundraisingEvents

Field	Value	IRS element (for citing)
Total Revenue Column	-315480	TotalRevenueColumn
Exclusion	-$315,480	ExclusionAmount

Net Income From Gaming NetIncomeFromGaming

Field	Value	IRS element (for citing)
Total Revenue Column	0	TotalRevenueColumn

Net Income Or Loss NetIncomeOrLoss

Field	Value	IRS element (for citing)
Total Revenue Column	0	TotalRevenueColumn

Other Revenue Misc OtherRevenueMisc (3 entries)

DescriptionDescription	Business CodeBusinessCode	Total Revenue ColumnTotalRevenueColumn	Unrelated Business RevenueUnrelatedBusinessRevenue	Related Or Exempt Function IncomeRelatedOrExemptFunctionIncome
RESEARCH	541900	915264	915264
REAGENTS -BY PRODUCT OF EXEMPT RESEARCH	541900	168950		168950
SYMPOSIUM FEES	900099	71888		71888

Miscellaneous Revenue MiscellaneousRevenue

Field	Value	IRS element (for citing)
Total Revenue Column	184189	TotalRevenueColumn
Related Or Exempt Function Income	184189	RelatedOrExemptFunctionIncome

Field	Value	IRS element (for citing)
Total Other Revenue	1340291	TotalOtherRevenue

Total Revenue TotalRevenue

Field	Value	IRS element (for citing)
Total Revenue Column	152710196	TotalRevenueColumn
Related Or Exempt Function Income	1502132	RelatedOrExemptFunctionIncome
Unrelated Business Revenue	915264	UnrelatedBusinessRevenue
Exclusion	$3,795,429	ExclusionAmount

Grants To Domestic Orgs GrantsToDomesticOrgs

Field	Value	IRS element (for citing)
Total	0	Total

Grants To Domestic Individuals GrantsToDomesticIndividuals

Field	Value	IRS element (for citing)
Total	0	Total

Foreign Grants ForeignGrants

Field	Value	IRS element (for citing)
Total	0	Total

Benefits To Members BenefitsToMembers

Field	Value	IRS element (for citing)
Total	0	Total

Comp Current Officers Directors CompCurrentOfficersDirectors

Field	Value	IRS element (for citing)
Total	1841699	Total
Program Services	2115745	ProgramServices
Management And General	-274046	ManagementAndGeneral

Comp Disqual Persons CompDisqualPersons

Field	Value	IRS element (for citing)
Total	191138	Total
Program Services	191138	ProgramServices

Other Salaries And Wages OtherSalariesAndWages

Field	Value	IRS element (for citing)
Total	60510538	Total
Program Services	50837956	ProgramServices
Management And General	8237101	ManagementAndGeneral
Fundraising	1435481	Fundraising

Pension Plan Contributions PensionPlanContributions

Field	Value	IRS element (for citing)
Total	4167689	Total
Program Services	3388695	ProgramServices
Management And General	689405	ManagementAndGeneral
Fundraising	89589	Fundraising

Other Employee Benefits OtherEmployeeBenefits

Field	Value	IRS element (for citing)
Total	8906179	Total
Program Services	7220690	ProgramServices
Management And General	1521434	ManagementAndGeneral
Fundraising	164055	Fundraising

Payroll Taxes PayrollTaxes

Field	Value	IRS element (for citing)
Total	3725968	Total
Program Services	3053466	ProgramServices
Management And General	583449	ManagementAndGeneral
Fundraising	89053	Fundraising

Fees For Services Management FeesForServicesManagement

Field	Value	IRS element (for citing)
Total	1464931	Total
Program Services	1119204	ProgramServices
Management And General	345480	ManagementAndGeneral
Fundraising	247	Fundraising

Fees For Services Legal FeesForServicesLegal

Field	Value	IRS element (for citing)
Total	379952	Total
Program Services	44781	ProgramServices
Management And General	335171	ManagementAndGeneral

Fees For Services Accounting FeesForServicesAccounting

Field	Value	IRS element (for citing)
Total	305901	Total
Management And General	305901	ManagementAndGeneral

Fees For Services Lobbying FeesForServicesLobbying

Field	Value	IRS element (for citing)
Total	123133	Total
Program Services	245	ProgramServices
Management And General	122888	ManagementAndGeneral

Fees For Services Prof Fundraising FeesForServicesProfFundraising

Field	Value	IRS element (for citing)
Total	33225	Total
Fundraising	33225	Fundraising

Fees For Services Invst Mgmnt Fees FeesForServicesInvstMgmntFees

Field	Value	IRS element (for citing)
Total	12288	Total
Management And General	12288	ManagementAndGeneral

Fees For Services Other FeesForServicesOther

Field	Value	IRS element (for citing)
Total	756085	Total
Program Services	575134	ProgramServices
Management And General	164463	ManagementAndGeneral
Fundraising	16488	Fundraising

Advertising Advertising

Field	Value	IRS element (for citing)
Total	53399	Total
Program Services	220	ProgramServices
Management And General	15239	ManagementAndGeneral
Fundraising	37940	Fundraising

Office Expenses OfficeExpenses

Field	Value	IRS element (for citing)
Total	9308668	Total
Program Services	8628160	ProgramServices
Management And General	589168	ManagementAndGeneral
Fundraising	91340	Fundraising

Information Technology InformationTechnology

Field	Value	IRS element (for citing)
Total	1315093	Total
Program Services	1009512	ProgramServices
Management And General	281248	ManagementAndGeneral
Fundraising	24333	Fundraising

Royalties Royalties

Field	Value	IRS element (for citing)
Total	0	Total

Occupancy Occupancy

Field	Value	IRS element (for citing)
Total	13284137	Total
Program Services	11802321	ProgramServices
Management And General	1364933	ManagementAndGeneral
Fundraising	116883	Fundraising

Travel Travel

Field	Value	IRS element (for citing)
Total	785657	Total
Program Services	678326	ProgramServices
Management And General	85927	ManagementAndGeneral
Fundraising	21404	Fundraising

Travel Entrtnmnt Public Officials TravelEntrtnmntPublicOfficials

Field	Value	IRS element (for citing)
Total	0	Total

Conferences Meetings ConferencesMeetings

Field	Value	IRS element (for citing)
Total	1194284	Total
Program Services	844290	ProgramServices
Management And General	319367	ManagementAndGeneral
Fundraising	30627	Fundraising

Interest Interest

Field	Value	IRS element (for citing)
Total	3710284	Total
Program Services	3367833	ProgramServices
Management And General	307574	ManagementAndGeneral
Fundraising	34877	Fundraising

Payments To Affiliates PaymentsToAffiliates

Field	Value	IRS element (for citing)
Total	0	Total

Depreciation Depletion DepreciationDepletion

Field	Value	IRS element (for citing)
Total	19065947	Total
Program Services	16654008	ProgramServices
Management And General	2263256	ManagementAndGeneral
Fundraising	148683	Fundraising

Insurance Insurance

Field	Value	IRS element (for citing)
Total	85144	Total
Management And General	85144	ManagementAndGeneral

Other Expenses OtherExpenses (4 entries)

DescriptionDescription	TotalTotal	Program ServicesProgramServices	Management And GeneralManagementAndGeneral	FundraisingFundraising
RESEARCH SUPPLIES & EXPENSES	10479721	10479721
SCIENTIFIC COLLABORATIONS	6616691	6616691
RECRUITMENT	385513	330049	30955	24509
MISCELLANEOUS	272826	119528	48879	104419

Total Functional Expenses TotalFunctionalExpenses

Field	Value	IRS element (for citing)
Total	148976090	Total
Program Services	129077713	ProgramServices
Management And General	17435224	ManagementAndGeneral
Fundraising	2463153	Fundraising

Cash Non Interest Bearing CashNonInterestBearing

Field	Value	IRS element (for citing)
BOY	2011199	BOY
EOY	1669367	EOY

Savings And Temp Cash Investments SavingsAndTempCashInvestments

Field	Value	IRS element (for citing)
BOY	7878644	BOY
EOY	17237397	EOY

Pledges And Grants Receivable PledgesAndGrantsReceivable

Field	Value	IRS element (for citing)
BOY	32920556	BOY
EOY	28941590	EOY

Accounts Receivable AccountsReceivable

Field	Value	IRS element (for citing)
BOY	230956	BOY
EOY	503732	EOY

Receivables From Officers Etc ReceivablesFromOfficersEtc

Field	Value	IRS element (for citing)
BOY	1142218	BOY
EOY	1186166	EOY

Receivables From Disqual Persons ReceivablesFromDisqualPersons

Field	Value	IRS element (for citing)
BOY	0	BOY
EOY	0	EOY

Other Notes Loans Receivable Net OtherNotesLoansReceivableNet

Field	Value	IRS element (for citing)
BOY	6173667	BOY
EOY	6113457	EOY

Inventories For Sale Or Use InventoriesForSaleOrUse

Field	Value	IRS element (for citing)
BOY	629240	BOY
EOY	448178	EOY

Prepaid Expenses Deferred Charges PrepaidExpensesDeferredCharges

Field	Value	IRS element (for citing)
BOY	8065520	BOY
EOY	8412047	EOY

Field	Value	IRS element (for citing)
Land Buildings Equipment Basis	288293600	LandBuildingsEquipmentBasis
Land Bldg Equipment Accum Deprec	133199334	LandBldgEquipmentAccumDeprec

Land Buildings Equipment Basis Net LandBuildingsEquipmentBasisNet

Field	Value	IRS element (for citing)
BOY	167593019	BOY
EOY	155094266	EOY

Investments Pub Traded Securities InvestmentsPubTradedSecurities

Field	Value	IRS element (for citing)
BOY	86413448	BOY
EOY	92491582	EOY

Investments Other Securities InvestmentsOtherSecurities

Field	Value	IRS element (for citing)
BOY	0	BOY
EOY	0	EOY

Investments Program Related InvestmentsProgramRelated

Field	Value	IRS element (for citing)
BOY	0	BOY
EOY	0	EOY

Intangible Assets IntangibleAssets

Field	Value	IRS element (for citing)
BOY	0	BOY
EOY	0	EOY

Other Assets Total OtherAssetsTotal

Field	Value	IRS element (for citing)
BOY	3324991	BOY
EOY	3155924	EOY

Total Assets TotalAssets

Field	Value	IRS element (for citing)
BOY	316383458	BOY
EOY	315253706	EOY

Accounts Payable Accrued Expenses AccountsPayableAccruedExpenses

Field	Value	IRS element (for citing)
BOY	20849521	BOY
EOY	17867974	EOY

Grants Payable GrantsPayable

Field	Value	IRS element (for citing)
BOY	0	BOY
EOY	0	EOY

Deferred Revenue DeferredRevenue

Field	Value	IRS element (for citing)
BOY	11772199	BOY
EOY	10516399	EOY

Tax Exempt Bond Liabilities TaxExemptBondLiabilities

Field	Value	IRS element (for citing)
BOY	76537517	BOY
EOY	71831116	EOY

Escrow Account Liability EscrowAccountLiability

Field	Value	IRS element (for citing)
BOY	0	BOY
EOY	0	EOY

Loans From Officers Directors LoansFromOfficersDirectors

Field	Value	IRS element (for citing)
BOY	0	BOY
EOY	0	EOY

Mort Notes Pybl Secured Invest Prop MortNotesPyblSecuredInvestProp

Field	Value	IRS element (for citing)
BOY	0	BOY
EOY	0	EOY

Unsecured Notes Loans Payable UnsecuredNotesLoansPayable

Field	Value	IRS element (for citing)
BOY	0	BOY
EOY	0	EOY

Other Liabilities OtherLiabilities

Field	Value	IRS element (for citing)
BOY	0	BOY
EOY	0	EOY

Total Liabilities TotalLiabilities

Field	Value	IRS element (for citing)
BOY	109159237	BOY
EOY	100215489	EOY

Field	Value	IRS element (for citing)
Follow SFAS117	X	FollowSFAS117

Unrestricted Net Assets UnrestrictedNetAssets

Field	Value	IRS element (for citing)
BOY	74517313	BOY
EOY	77369944	EOY

Temporarily Restricted Net Assets TemporarilyRestrictedNetAssets

Field	Value	IRS element (for citing)
BOY	120682803	BOY
EOY	125635591	EOY

Permanently Restricted Net Assets PermanentlyRestrictedNetAssets

Field	Value	IRS element (for citing)
BOY	12024105	BOY
EOY	12032682	EOY

Total Net Assets Fund Balances TotalNetAssetsFundBalances

Field	Value	IRS element (for citing)
BOY	207224221	BOY
EOY	215038217	EOY

Total Liab Net Assets Fund Balances TotalLiabNetAssetsFundBalances

Field	Value	IRS element (for citing)
BOY	316383458	BOY
EOY	315253706	EOY

Field	Value	IRS element (for citing)
Info In Schedule O Part XI	X	InfoInScheduleOPartXI
Reconcilation Revenue Expenses	3734106	ReconcilationRevenueExpenses
Reconciliation Unrealized Invest	2773536	ReconciliationUnrealizedInvest
Reconcilation Other Changes	1306354	ReconcilationOtherChanges
Method Of Accounting Accrual	X	MethodOfAccountingAccrual
Accountant Compile Or Review	false	AccountantCompileOrReview
FS Audited	true	FSAudited

FS Audited Basis FSAuditedBasis

Field	Value	IRS element (for citing)
Financial Statement Separate	X	FinancialStatementSeparate

Field	Value	IRS element (for citing)
Audit Committee	true	AuditCommittee
Federal Grant Audit Required	true	FederalGrantAuditRequired
Federal Grant Audit Performed	true	FederalGrantAuditPerformed

Schedule A IRS990ScheduleA

Field	Value	IRS element (for citing)
Publicly Supported Org170b1 Avi	X	PubliclySupportedOrg170b1Avi

Gifts Grants Contrib Received170 GiftsGrantsContribReceived170

Field	Value	IRS element (for citing)
Current Tax Year Minus4 Years	209859230	CurrentTaxYearMinus4Years
Current Tax Year Minus3 Years	150769181	CurrentTaxYearMinus3Years
Current Tax Year Minus2 Years	160093433	CurrentTaxYearMinus2Years
Current Tax Year Minus1 Year	156880684	CurrentTaxYearMinus1Year
Current Tax Year	147266803	CurrentTaxYear
Total	824869331	Total

Tax Rev Levied For Org Benefit170 TaxRevLeviedForOrgBenefit170

Field	Value	IRS element (for citing)
Total	0	Total

Value Srvc Fclts Furnished By Govt ValueSrvcFcltsFurnishedByGovt

Field	Value	IRS element (for citing)
Total	0	Total

Total170 Total170

Field	Value	IRS element (for citing)
Current Tax Year Minus4 Years	209859230	CurrentTaxYearMinus4Years
Current Tax Year Minus3 Years	150769181	CurrentTaxYearMinus3Years
Current Tax Year Minus2 Years	160093433	CurrentTaxYearMinus2Years
Current Tax Year Minus1 Year	156880684	CurrentTaxYearMinus1Year
Current Tax Year	147266803	CurrentTaxYear
Total	824869331	Total

Field	Value	IRS element (for citing)
Amounts Subst Contributors Total	23709484	AmountsSubstContributorsTotal
Public Support Total170	801159847	PublicSupportTotal170

Gross Investment Income170 GrossInvestmentIncome170

Field	Value	IRS element (for citing)
Current Tax Year Minus4 Years	1995011	CurrentTaxYearMinus4Years
Current Tax Year Minus3 Years	1354102	CurrentTaxYearMinus3Years
Current Tax Year Minus2 Years	2037946	CurrentTaxYearMinus2Years
Current Tax Year Minus1 Year	4402201	CurrentTaxYearMinus1Year
Current Tax Year	4133944	CurrentTaxYear
Total	13923204	Total

Net UBI NetUBI

Field	Value	IRS element (for citing)
Total	0	Total

Other Income170 OtherIncome170

Field	Value	IRS element (for citing)
Current Tax Year Minus4 Years	286205	CurrentTaxYearMinus4Years
Current Tax Year Minus3 Years	343630	CurrentTaxYearMinus3Years
Current Tax Year Minus2 Years	907754	CurrentTaxYearMinus2Years
Current Tax Year Minus1 Year	370643	CurrentTaxYearMinus1Year
Current Tax Year	425047	CurrentTaxYear
Total	2333279	Total

Field	Value	IRS element (for citing)
Total Support	841125814	TotalSupport
Gross Receipts From Admissions Etc	2283629	GrossReceiptsFromAdmissionsEtc
Public Support Pertcentage170	0.95249	PublicSupportPertcentage170
Public Support Pertcent Prior Year	0.96180	PublicSupportPertcentPriorYear
Thirty Three Percnt Tests CY170	X	ThirtyThreePercntTestsCY170

Schedule B IRS990ScheduleB

Contributor Info ContributorInfo

Field	Value	IRS element (for citing)
Contributor Number	RESTRICTED	ContributorNumber

Contributor Name Business ContributorNameBusiness

Field	Value	IRS element (for citing)
Business Name Line1	RESTRICTED	BusinessNameLine1

Contributor Address US ContributorAddressUS

Field	Value	IRS element (for citing)
Address Line1	RESTRICTED	AddressLine1
Address Line2	RESTRICTED	AddressLine2
City	RESTRICTED	City
State	RESTRICTED	State
ZIP Code	RESTRICTED	ZIPCode

Field	Value	IRS element (for citing)
Aggregate Contributions	RESTRICTED	AggregateContributions

Schedule C IRS990ScheduleC

Field	Value	IRS element (for citing)
Total Grassroots Lobbying		TotalGrassrootsLobbying
Total Direct Lobbying		TotalDirectLobbying
Total Lobbying Expenditures		TotalLobbyingExpenditures
Other Exempt Purpose Expenditures		OtherExemptPurposeExpenditures
Total Exempt Purpose Expenditures		TotalExemptPurposeExpenditures
Lobbying Nontaxable		LobbyingNontaxableAmount
Grassroots Nontaxable		GrassrootsNontaxableAmount
Tot Lobbying Grassroot Minus Non Tx		TotLobbyingGrassrootMinusNonTx
Tot Lobby Expend Mns Lobbying Nontx		TotLobbyExpendMnsLobbyingNontx
Volunteers	false	Volunteers
Paid Staff Or Management	true	PaidStaffOrManagement
Media Advertisements	false	MediaAdvertisements
Mailings Members	false	MailingsMembers
Publications Or Broadcast	false	PublicationsOrBroadcast
Grants Other Organizations	false	GrantsOtherOrganizations
Direct Contact Legislators	true	DirectContactLegislators
Direct Contact Legislators	$134,978	DirectContactLegislatorsAmount
Rallies Demonstrations	false	RalliesDemonstrations
Other Activities	true	OtherActivities
Other Activities	$123,133	OtherActivitiesAmount
Total Lobbying Expenditures IIB	258111	TotalLobbyingExpendituresIIB
Not Described In501c3	false	NotDescribedIn501c3

Form990 Schedule C Part IV Form990ScheduleCPartIV

Field	Value	IRS element (for citing)
Identifier	LOBBYING ACTIVITIES	Identifier
Return Reference	SCHEDULE C, PART II-B	ReturnReference
Explanation	LINE 1G - CONTACT AND MEET WITH FEDERAL AND STATE LEGISLATORS AND THEIR STAFF TO SEEK POTENTIAL RESEARCH FUNDING. LINE 1I - DUES TO RESEARCH AND BIOMEDICAL ORGANIZATIONS, A PORTION OF WHICH ARE CONSIDERED LOBBYING. THE INSTITUTE CONTRACTS WITH HURT NORTON AND ASSOCIATES FOR REPRESENTATION IN WASHINGTON, DC, TO INVESTIGATE AND SEEK POTENTIAL FEDERAL RESEARCH FUNDING OPPORTUNITIES OUTSIDE THE TRADITIONAL NIH PROCESS.	Explanation

Schedule D IRS990ScheduleD

Field	Value	IRS element (for citing)
Art Public Exhibition Amts		ArtPublicExhibitionAmts
Held Works Of Art Amts		HeldWorksOfArtAmts

Current Year CurrentYear

Field	Value	IRS element (for citing)
Beginning Of Year Balance	21200471	BeginningOfYearBalance
Contributions	9000	Contributions
Investment Earnings Or Losses	2517312	InvestmentEarningsOrLosses
Other Expenditures	921772	OtherExpenditures
End Of Year Balance	22805011	EndOfYearBalance

Current Year Minus1 Year CurrentYearMinus1Year

Field	Value	IRS element (for citing)
Beginning Of Year Balance	21602848	BeginningOfYearBalance
Contributions	29575	Contributions
Investment Earnings Or Losses	961439	InvestmentEarningsOrLosses
Other Expenditures	1393391	OtherExpenditures
End Of Year Balance	21200471	EndOfYearBalance

Current Year Minus2 Years CurrentYearMinus2Years

Field	Value	IRS element (for citing)
Beginning Of Year Balance	19352544	BeginningOfYearBalance
Contributions	1050000	Contributions
Investment Earnings Or Losses	1656322	InvestmentEarningsOrLosses
Other Expenditures	456018	OtherExpenditures
End Of Year Balance	21602848	EndOfYearBalance

Current Year Minus3 Years CurrentYearMinus3Years

Field	Value	IRS element (for citing)
Beginning Of Year Balance	15181323	BeginningOfYearBalance
Contributions	2983403	Contributions
Investment Earnings Or Losses	1912336	InvestmentEarningsOrLosses
Other Expenditures	724518	OtherExpenditures
End Of Year Balance	19352544	EndOfYearBalance

Current Year Minus4 Years CurrentYearMinus4Years

Field	Value	IRS element (for citing)
Beginning Of Year Balance	17485831	BeginningOfYearBalance
Contributions	55002	Contributions
Investment Earnings Or Losses	-2359510	InvestmentEarningsOrLosses
Other Expenditures	0	OtherExpenditures
End Of Year Balance	15181323	EndOfYearBalance

Field	Value	IRS element (for citing)
Board Designated EOY Balance	0.43580	BoardDesignatedEOYBalance
Permanent Endowment EOY Balance	0.52760	PermanentEndowmentEOYBalance
Term Endowment EOY Balance	0.03660	TermEndowmentEOYBalance
Endowments Held By Unrelated Orgs	false	EndowmentsHeldByUnrelatedOrgs
Endowments Held By Related Orgs	false	EndowmentsHeldByRelatedOrgs

Land Land

Field	Value	IRS element (for citing)
Other Cost Or Other Basis	25793087	OtherCostOrOtherBasis
Book Value	25793087	BookValue

Buildings Buildings

Field	Value	IRS element (for citing)
Other Cost Or Other Basis	162682250	OtherCostOrOtherBasis
Depreciation	55177762	Depreciation
Book Value	107504488	BookValue

Field	Value	IRS element (for citing)
Leasehold Improvements		LeaseholdImprovements

Equipment Equipment

Field	Value	IRS element (for citing)
Other Cost Or Other Basis	99269550	OtherCostOrOtherBasis
Depreciation	78021572	Depreciation
Book Value	21247978	BookValue

Other Land Buildings OtherLandBuildings

Field	Value	IRS element (for citing)
Other Cost Or Other Basis	548713	OtherCostOrOtherBasis
Book Value	548713	BookValue

Field	Value	IRS element (for citing)
Total Of Book Value Land Buildings	155094266	TotalOfBookValueLandBuildings
Federal Income Tax Liability	0	FederalIncomeTaxLiability
Total Of Amounts	0	TotalOfAmounts
Footnote In Part XIII	X	FootnoteInPartXIII
Tot Revenue Etc Audited Fincl Stmt	155472000	TotRevenueEtcAuditedFinclStmt
Net Unrealized Gains Investments	2773536	NetUnrealizedGainsInvestments
Other Revenues	556	OtherRevenues
Revenue Not Rptd F990	2774092	RevenueNotRptdF990
Revenue Subtotal	152697908	RevenueSubtotal
Investment Expenses Not Included	12288	InvestmentExpensesNotIncluded
Revenue Not Rptd On Fin Stmt	12288	RevenueNotRptdOnFinStmt
Total Revenue Per Form990	152710196	TotalRevenuePerForm990
Tot Expenses Etc Audited Fincl Stmt	147658000	TotExpensesEtcAuditedFinclStmt
Other Expenses Included	-1305802	OtherExpensesIncluded
Expenses Not Rptd F990	-1305802	ExpensesNotRptdF990
Expenses Subtotal	148963802	ExpensesSubtotal
Investment Expenses Not Included2	12288	InvestmentExpensesNotIncluded2
Expenses Not Rptd On Fin Stmt	12288	ExpensesNotRptdOnFinStmt
Total Expenses Per Form990	148976090	TotalExpensesPerForm990

Form990 Schedule D Part XIII Form990ScheduleDPartXIII (4 entries)

IdentifierIdentifier	Return ReferenceReturnReference	ExplanationExplanation
INTENDED USES OF THE ORGANIZATION'S ENDOWMENT FUNDS	SCHEDULE D, PART V, LINE 4	THE INSTITUTE'S ENDOWMENT FUNDS ARE ESTABLISHED FOR VARIOUS PURPOSES: SCIENTIFIC RESEARCH, ALS RESEARCH, CEO CHAIR, PRESIDENTIAL CHAIR, CANCER RESEARCH CHAIR, AND UNRESTRICTED AS TO INCOME.
FIN 48 FOOTNOTE	SCHEDULE D, PART X, LINE 2	THE INSTITUTE HAS NO MATERIAL DEFERRED TAX ASSETS OR DEFERRED TAX LIABILITIES RECORDED AS OF JUNE 30, 2013 AND 2012. THE INSTITUTE DID NOT RECOGNIZE A CHANGE IN LIABILITY FOR UNRECOGNIZED TAX BENEFITS (THE DIFFERENCE BETWEEN A TAX POSITION TAKEN, OR EXPECTED TO BE TAKEN, IN A TAX RETURN AND THE BENEFIT RECOGNIZED AND MEASURED IN THE FINANCIAL STATEMENTS). THE INSTITUTE'S LIABILITY FOR UNRECOGNIZED TAX BENEFITS WAS $0 AT JUNE 30, 2012 AND 2013.
DETAIL OF AMOUNT	SCHEDULE D, PART XI, LINE 2D	ROUNDING - 556
DETAIL OF AMOUNT	SCEDULE D, PART XII, LINE 2D	UNREALIZED GAIN ON INTEREST RATE SWAP $1,306,042 ROUNDING (240) ----------- TOTAL $1,305,802

Schedule F IRS990ScheduleF

Accts Actvs Out US Table AcctsActvsOutUSTable (8 entries)

RegionRegion	Number Of EmployeesNumberOfEmployees	Type Of Activities ConductedTypeOfActivitiesConducted	Specific Services ProvidedSpecificServicesProvided	Total ExpendituresTotalExpenditures
Europe (Including Iceland and Greenland)	54	Program Services	SEE PART V	108839
East Asia and the Pacific	28	Program Services	SEE PART V	59718
North America	2	Program Services	SEE PART V	1082
South Asia	3	Program Services	SEE PART V	2503
Middle East and North Africa	4	Program Services	SEE PART V	618
South America	5	Program Services	SEE PART V	9960
Central America and the Caribbean	4	Program Services	SEE PART V	10370
Sub-Saharan Africa	5	Program Services	SEE PART V	14880

Field	Value	IRS element (for citing)
Subtotal Number Of Employees	76	SubtotalNumberOfEmployees
Total Number Of Employees	105	TotalNumberOfEmployees
Subtotal Amount Spent	207970	SubtotalAmountSpent
Total Amount Spent	207970	TotalAmountSpent
Transfer To Foreign Corp	false	TransferToForeignCorp
Interest In Foreign Trust	false	InterestInForeignTrust
Own Foreign Corp	false	OwnForeignCorp
Passive Foreign Invest Co	false	PassiveForeignInvestCo
Foreign Partnership	false	ForeignPartnership
Boycott Countries	false	BoycottCountries

Form990 Schedule F Part V Form990ScheduleFPartV (3 entries)

IdentifierIdentifier	Return ReferenceReturnReference	ExplanationExplanation
SUPPLEMENTAL INFORMATION	SCHEDULE F, PART I, LINE 3	SANFORD-BURNHAM MEDICAL RESEARCH INSTITUTE DOES NOT MAINTAIN ONGOING OPERATIONS IN FOREIGN JURISDICTIONS. HOWEVER, THE INSTITUTE'S SCIENTISTS DO TRAVEL TO VARIOUS SCIENTIFIC CONFERENCES IN OTHER COUNTRIES TO SHARE THE INSTITUTE'S SCIENTIFIC DISCOVERIES, AS WELL AS TO LEARN FROM OTHER SCIENTISTS ACROSS THE WORLD.
SCHEDULE F, PART I, LINE 3, COLUMN E		SPECIFIC PROGRAM SERVICE ACTIVITY IN ALL REGIONS IS AGENTS ATTENDING AND/OR SPEAKING AT SCIENTIFIC CONFERENCES AND COLLABORATIONS.
ACCOUNTING METHOD	SCHEDULE F, PART I, LINE 3, COLUMN F	THE ACCRUAL METHOD OF ACCOUNTING WAS USED TO DETERMINE THE AMOUNTS IN PART I, LINE 3, COLUMN (F).

Schedule G IRS990ScheduleG

Field	Value	IRS element (for citing)
Mail Solicitations	X	MailSolicitations
Email Solicitations	X	EmailSolicitations
Phone Solicitations	X	PhoneSolicitations
In Person Solicitations	X	InPersonSolicitations
Solicitation Of Non Govt Grants	X	SolicitationOfNonGovtGrants
Solicitation Of Govt Grants	X	SolicitationOfGovtGrants
Special Fundraising Events	X	SpecialFundraisingEvents
Agreement Ref Fundraising	true	AgreementRefFundraising

Fundraiser Activity Information FundraiserActivityInformation (2 entries)

Name Of IndividualNameOfIndividual	ActivityActivity	Fundraiser Control Of FundsFundraiserControlOfFunds	Amount Retained By ContractorAmountRetainedByContractor	Net To OrganizationNetToOrganization
SEXTON BLUM AND ASSOCIATES	CONSULTING	false	22000	22000
JEROLD PANAS LINZY PARTNERS	CONSULTING	false	11225	11225

Field	Value	IRS element (for citing)
Total Retained By Contractors	33225	TotalRetainedByContractors
Total Net To Organization	33225	TotalNetToOrganization
Licensed States	AL	LicensedStates
Licensed States	AK	LicensedStates
Licensed States	AR	LicensedStates
Licensed States	CA	LicensedStates
Licensed States	CT	LicensedStates
Licensed States	DE	LicensedStates
Licensed States	FL	LicensedStates
Licensed States	ID	LicensedStates
Licensed States	IN	LicensedStates
Licensed States	IA	LicensedStates
Licensed States	KS	LicensedStates
Licensed States	ME	LicensedStates
Licensed States	MD	LicensedStates
Licensed States	MA	LicensedStates
Licensed States	MI	LicensedStates
Licensed States	MO	LicensedStates
Licensed States	MT	LicensedStates
Licensed States	NE	LicensedStates
Licensed States	NV	LicensedStates
Licensed States	NH	LicensedStates
Licensed States	NJ	LicensedStates
Licensed States	NY	LicensedStates
Licensed States	NC	LicensedStates
Licensed States	OH	LicensedStates
Licensed States	OR	LicensedStates
Licensed States	PA	LicensedStates
Licensed States	RI	LicensedStates
Licensed States	SD	LicensedStates
Licensed States	TX	LicensedStates
Licensed States	UT	LicensedStates
Licensed States	VT	LicensedStates
Licensed States	VA	LicensedStates
Licensed States	WA	LicensedStates
Licensed States	WY	LicensedStates

Events Information EventsInformation

Field	Value	IRS element (for citing)
Name Of Event1	GALA	NameOfEvent1
Gross Receipts Event1	1007243	GrossReceiptsEvent1
Charitable Contributions Event1	980243	CharitableContributionsEvent1
Gross Revenue Event1	27000	GrossRevenueEvent1
Food And Beverage Event1	57830	FoodAndBeverageEvent1
Entertainment Event1	96025	EntertainmentEvent1
Other Direct Expenses Event1	78823	OtherDirectExpensesEvent1
Name Of Event2	BRING IT	NameOfEvent2
Gross Receipts Event2	114700	GrossReceiptsEvent2
Charitable Contributions Event2	92700	CharitableContributionsEvent2
Gross Revenue Event2	22000	GrossRevenueEvent2
Rent Facility Costs Event2	4000	RentFacilityCostsEvent2
Food And Beverage Event2	33821	FoodAndBeverageEvent2
Entertainment Event2	67190	EntertainmentEvent2
Other Direct Expenses Event2	26791	OtherDirectExpensesEvent2
Total Number Of Other Events	0	TotalNumberOfOtherEvents
Gross Receipts Total	1121943	GrossReceiptsTotal
Charitable Contributions Total	1072943	CharitableContributionsTotal
Gross Revenue Total Events	49000	GrossRevenueTotalEvents
Rent Facility Costs Total Events	4000	RentFacilityCostsTotalEvents
Food And Beverage Total Events	91651	FoodAndBeverageTotalEvents
Entertainment Total Events	163215	EntertainmentTotalEvents
Other Direct Expenses Total Events	105614	OtherDirectExpensesTotalEvents
Direct Expense Summary Events	364480	DirectExpenseSummaryEvents
Net Income Summary	-315480	NetIncomeSummary

Schedule J IRS990ScheduleJ

Field	Value	IRS element (for citing)
First Class Or Charter Travel	X	FirstClassOrCharterTravel
Club Dues Or Fees	X	ClubDuesOrFees
Written Policy Re T And E Expenses	true	WrittenPolicyReTAndEExpenses
Substantiation Required	true	SubstantiationRequired
Compensation Committee	X	CompensationCommittee
Independent Consultant	X	IndependentConsultant
Form990 Of Other Organizations	X	Form990OfOtherOrganizations
Written Employment Contract	X	WrittenEmploymentContract
Compensation Survey	X	CompensationSurvey
Board Or Committee Approval	X	BoardOrCommitteeApproval
Severance Payment	true	SeverancePayment
Supplemental Nonqual Retire Plan	true	SupplementalNonqualRetirePlan
Equity Based Comp Arrangement	false	EquityBasedCompArrangement
Comp Based On Revenue Of Filing Org	false	CompBasedOnRevenueOfFilingOrg
Comp Based On Revenue Related Orgs	false	CompBasedOnRevenueRelatedOrgs
Comp Based Net Earnings Filing Org	false	CompBasedNetEarningsFilingOrg
Comp Based Net Earnings Relate Orgs	false	CompBasedNetEarningsRelateOrgs
Any Non Fixed Payments	true	AnyNonFixedPayments
Initial Contract Exception	false	InitialContractException

Form990 Schedule J Part II Form990ScheduleJPartII (9 entries)

TitleTitle	Base Compensation Filing OrgBaseCompensationFilingOrg	Comp Based On Related OrgsCompBasedOnRelatedOrgs	Bonus Filing OrgBonusFilingOrg	Bonus Related OrgsBonusRelatedOrgs	Other Compensation Filing OrgOtherCompensationFilingOrg	Other Compensation Related OrgsOtherCompensationRelatedOrgs	Deferred Comp Filing OrgDeferredCompFilingOrg	Deferred Comp Related OrgsDeferredCompRelatedOrgs	Nontaxable Benefits Filing OrgNontaxableBenefitsFilingOrg	Nontaxable Benefits Related OrgsNontaxableBenefitsRelatedOrgs	Total Compensation Filing OrgTotalCompensationFilingOrg	Total Compensation Related OrgsTotalCompensationRelatedOrgs	Comp Report Prior990 Filing OrgCompReportPrior990FilingOrg	Comp Report Prior990 Related OrgsCompReportPrior990RelatedOrgs
JOHN C REED MD PHD
CEO	658712	0	0	0	21998	0	375000	0	25613	0	1081323	0	0	0
KRISTIINA VUORI MD PHD
PRESIDENT/INTERIM CEO	613442	0	0	0	960	0	325000	0	11101	0	950503	0	0	0
GARY F RAISL EDD
EVP/CAO/CFO/TREASURER	429072	0	0	0	3740	0	23319	0	25622	0	481753	0	0	0
STUART LIPTON MD PHD
CENTER DIRECTOR	444521	0	0	0	3625	0	25000	0	27739	0	500885	0	0	0
DANIEL KELLY MD
CNTR DIR./SCI. DIR. AT LK NONA	435055	0	0	0	10966	0	25000	0	25001	0	496022	0	0	0
CARL WARE PHD
CENTER DIRECTOR	344246	0	0	0	3625	0	18738	0	16070	0	382679	0	0	0
CYRIL DOUCET
VP, ADMIN. AT LAKE NONA	214715	0	0	0	92955	0	19810	0	15502	0	342982	0	0	0
JORGE MOSCAT PHD
PROGRAM DIRECTOR	277405	0	0	0	2451	0	25000	0	20878	0	325734	0	0	0
MARGARET DUNBAR JD
SECRETARY/SR DIRECTOR IP	163906	0	0	0	1795	0	14434	0	887	0	181022	0	0	0

Form990 Schedule J Part III Form990ScheduleJPartIII (5 entries)

IdentifierIdentifier	ExplanationExplanation	Return ReferenceReturnReference
SUPPLEMENTAL COMPENSATION INFORMATION	FIRST CLASS TRAVEL FIRST CLASS TRAVEL IS GENERALLY NOT PERMITTED, EXCEPT UNDER VERY LIMITED AND RARE CIRCUMSTANCES AND CONDITIONS, SUCH AS A MEDICAL CONDITION REQUIREMENT AND URGENT CIRCUMSTANCES. THIS WAS NOT TREATED AS TAXABLE COMPENSATION. HEALTH OR SOCIAL CLUB DUES HEALTH OR SOCIAL CLUB DUES ARE NEGOTIATED IN THE HIRING PROCESS FOR CERTAIN EMPLOYEES. THE AMOUNTS WERE TREATED AS TAXABLE COMPENSATION.
SEVERENCE PAYMENT OR CHANGE OF CONTROL PAYMENT	CYRIL DOUCET WAS PAID $83,261 FOR SEVERANCE DURING THE YEAR.	SCHEDULE J, PART I, QUESTION 4A
SUPPLEMENTAL NONQUALIFIED RETIREMENT PLAN	THE PARTICIPANTS OF THE 457(F) DEFERRED COMPENSATION PLAN ARE JOHN REED AND KRISTIINA VUORI. VESTING FOR JOHN REED OCCURS THE EARLIEST OF 1/1/2019, OR DISABILITY, OR INVOLUNTARY TERMINATION WITHOUT CAUSE, OR THE PARTICIPANT'S RESIGNATION FOR GOOD CAUSE, OR THE DATE OF TERMINATION OF THE PLAN. VESTING FOR KRISTIINA VUORI OCCURS THE EARLIEST OF 4/1/2015, OR INVOLUNTARY TERMINATION WITHOUT CAUSE, OR PARTICIPANT'S RESIGNATION FOR GOOD CAUSE, OR THE DATE OF TERMINATION OF THE PLAN. DISTRIBUTIONS FOR BOTH PARTICIPANTS IS A ONE-TIME LUMP SUM PAYMENT UPON VESTING OF 100% OF THE VALUE OF THE PARTICIPANT'S INVESTMENT (COMPRISED OF INSTITUTE CONTRIBUTIONS TO THE CORPUS PLUS ANY INVESTMENT GAINS OR LOSSES THEREON).	SCHEDULE J, PART I, QUESTION 4B
NON-FIXED PAYMENTS	SOME SENIOR EXECUTIVES HAVE EMPLOYMENT AGREEMENTS STIPULATING A BONUS WILL BE PAID TO THEM PROVIDED CERTAIN GOALS OR ACCOMPLISHMENTS ARE ACHIEVED.	SCHEDULE J, PART I, QUESTION 7
RETIREMENT AND OTHER DEFERRED COMPENSATION	THE AMOUNT REPORTED IN SCHEDULE J, PART II, COLUMN (C) FOR JOHN REED INCLUDES AN ACCRUAL OF $350,000 RELATED TO THE 457(F) PLAN DESCRIBED ABOVE AND FOR KRISTIINA VUORI AN ACCRUAL OF $300,000 RELATED TO THE 457(F) PLAN DESCRIBED ABOVE. THESE AMOUNTS ARE NOT VESTED AND ARE SUBJECT TO A SUBSTANTIAL RISK OF FORFEITURE.	SCHEDULE J, PART II, COLUMN (C)

Schedule K IRS990ScheduleK

Form990 Schedule K Part I Form990ScheduleKPartI (3 entries)

Bond ReferenceBondReferenceCd	Issuer EINIssuerEIN	CUSIP NumberCUSIPNumber	Date IssuedDateIssued	Issue PriceIssuePrice	Description Of PurposeDescriptionOfPurpose	DefeasedDefeased	On Behalf Of IssuerOnBehalfOfIssuer	Pool FinancingPoolFinancing
COUNTY OF SAN DIEGO
A	956000934	797391G60	2006-02-01	60572867	SEE PART VI	false	false	false
COUNTY OF SAN DIEGO
B	956000934	797391K99	2012-11-15	18885000	FINANCE PROPERTY AND CAPITAL EQUIP	false	false	false
CALIFORNIA STATEWIDE COMMUNITIES DEVELOPMENT AUTH
C	956000934		2008-08-01	15300000	PURCHASE EQUIPMENT	false	false	false

Form990 Schedule K Part II Form990ScheduleKPartII (3 entries)

Bond ReferenceBondReferenceCd	Total ProceedsTotalProceeds	Amount In Reserve FundAmountInReserveFund	Refunding EscrowRefundingEscrow	Issuance Costs From ProceedsIssuanceCostsFromProceeds	Capital ExpendituresCapitalExpenditures	Other Spent ProceedsOtherSpentProceeds	Year Of Subst CompletionYearOfSubstCompletion	Current RefundingCurrentRefunding	Advance RefundingAdvanceRefunding	Final Allocation MadeFinalAllocationMade	Adequate Books And Recds MaintaindAdequateBooksAndRecdsMaintaind
A	60572867	3958500	46326377	745537	9542453	0	2000	false	true	true	true
B	18885000	0	0	0	18885000	0	2012	false	false	true	true
C	15300000	0	0	300000	13869914	1130086	2012	false	false	true	true

Form990 Schedule K Part III Form990ScheduleKPartIII (3 entries)

Bond ReferenceBondReferenceCd	Owning Bond Financed PropertyOwningBondFinancedProperty	Any Lease ArrangementsAnyLeaseArrangements	Mngment Cntrct Bond Financd PrprtyMngmentCntrctBondFinancdPrprty	Engage Bond Counsel ContractsEngageBondCounselContracts	Any Research AgreementsAnyResearchAgreements	Engage Bond Counsel ResearchEngageBondCounselResearch	Prcntge Prvt Business Use By OthersPrcntgePrvtBusinessUseByOthers	Total Private Business UseTotalPrivateBusinessUse	Bond Iss Meet Prvt Security Pay TestBondIssMeetPrvtSecurityPayTest	Change In Use Bond Financed PropChangeInUseBondFinancedProp	Remedial Action TakenRemedialActionTaken	Non Qualified Bond Remed ProcdrsNonQualifiedBondRemedProcdrs
A	false	true	true	true	true	true	0.03060	0.03060	false	false	false	false
B	false	false	false		false	true	0.01980	0.01980	false	false	false	false
C	false	true	true	true	true	true	0.05610	0.05610	false	false	false	false

Form990 Schedule K Part IV Form990ScheduleKPartIV (3 entries)

Bond ReferenceBondReferenceCd	Form8038 T FiledForm8038TFiled	Variable Rate IssueVariableRateIssue	Hedge Identified In Books And RecdsHedgeIdentifiedInBooksAndRecds	Gross Proceeds Invested In GICGrossProceedsInvestedInGIC	Business Name Line1NameOfGICProvider/BusinessNameLine1	Gross Proceeds InvestedGrossProceedsInvested	Written Proc To Monitor ReqsWrittenProcToMonitorReqs	Rebate Not Due YetRebateNotDueYet	Exception To RebateExceptionToRebate	Term Of HedgeTermOfHedge	Superintegrated HedgeSuperintegratedHedge	Hedge TerminatedHedgeTerminated
0
A	true	false	false	false	0	false	false
BANK OF AMERICA
B	false	true	true	false	0	false	false	true	false	25.	true	false
0
C	false	false	false	false	0	false	false	true	false

Form990 Schedule K Part V Form990ScheduleKPartV (3 entries)

Bond ReferenceBondReferenceCd	Proc To Undertk Corrective ActionProcToUndertkCorrectiveAction
A	false
B	true
C	false

Form990 Schedule K Part VI Form990ScheduleKPartVI (3 entries)

IdentifierIdentifier	Return ReferenceReturnReference	ExplanationExplanation
SCHEDULE K, PART I, ROW A, COLUMN (F)	0	REFINANCE 1999 BONDS AND FINANCE CAPITAL IMPROVEMENTS & EQUIPMENT.
SCHEDULE K, PART I, ROWS A-C	0	FOR THE BOND ISSUES LISTED IN PART I, THE ALTERNATIVE PERIOD USED FOR THE REPORTING REQUIREMENTS IN THIS SECTION IS THE TWELVE MONTHS ENDED DECEMBER 31, 2012.
SCHEDULE K, PART III, LINE 4, COLUMN (C)	0	THE PERCENTAGE OF FINANCED PROPERTY USED IN A PRIVATE BUSINESS FOR THE ANNUAL MEASUREMENT PERIOD ENDING 12/31/2012 IS 5.61%. THE PRIVATE BUSINESS USE PERCENTAGE OVER THE LIFE OF THE BONDS THROUGH 12/31/2012 IS 3.81%. THE PROJECTED PRIVATE BUSINESS USE PERCENTAGE OVER THE LIFE OF THE BONDS IS 4.19%.

Schedule L IRS990ScheduleL

Loan Table LoanTable (4 entries)

Relationship With OrganizationRelationshipWithOrganization	Purpose Of LoanPurposeOfLoan	Loan From OrganizationLoanFromOrganization	Original PrincipalOriginalPrincipalAmount	Balance DueBalanceDue	DefaultDefault	Approved By BoardApprovedByBoard	Written AgreementWrittenAgreement
DANIEL KELLY
EMPLOYEE	QUAL. EMPLOYEE RELOC	X	$500,000	454167	false	true	true
STUART LIPTON
EMPLOYEE	QUAL. EMPLOYEE RELOC	X	$400,000	171915	false	true	true
GARY RAISL
OFFICER	QUAL. EMPLOYEE RELOC	X	$225,000	225000	false	true	true
JORGE MOSCAT
EMPLOYEE	QUAL. EMPLOYEE RELOC	X	$200,000	86863	false	true	true

Field	Value	IRS element (for citing)
Total Balance Due	937945	TotalBalanceDue

Form990 Schedule L Part IV Form990ScheduleLPartIV (2 entries)

RelationshipRelationship	Amount Of TransactionAmountOfTransaction	Description Of TransactionDescriptionOfTransaction	Sharing Of RevenuesSharingOfRevenues
LOCKTON
TRUSTEE - DOUG OBENSHAIN	202078	INSURANCE SERVICES	false
PHILIP GRAHAM - SON OF TRUSTEE
TRUSTEE - GAYLE WILSON	191138	COMPENSATION	false

Schedule M IRS990ScheduleM

Field	Value	IRS element (for citing)
Works Of Art		WorksOfArt
Art Historical Treasures		ArtHistoricalTreasures
Art Fractional Interest		ArtFractionalInterest
Books And Publications		BooksAndPublications
Clothing And Household Goods		ClothingAndHouseholdGoods
Cars And Other Vehicles		CarsAndOtherVehicles
Boats And Planes		BoatsAndPlanes
Intellectual Property		IntellectualProperty

Securities Publicly Traded SecuritiesPubliclyTraded

Field	Value	IRS element (for citing)
Non Cash Checkbox	X	NonCashCheckbox
Number Of Contributions	10	NumberOfContributions
Noncash Contribs Rptd F990	1059634	NoncashContribsRptdF990
Method Of Determining Revenues	FMV	MethodOfDeterminingRevenues

Field	Value	IRS element (for citing)
Securities Closely Held Stock		SecuritiesCloselyHeldStock
Securities Prtnrshp Trust Intrsts		SecuritiesPrtnrshpTrustIntrsts
Securities Miscellaneous		SecuritiesMiscellaneous
Qualified Contrib Hist Struct		QualifiedContribHistStruct
Qualified Contrib Other		QualifiedContribOther
Real Estate Residential		RealEstateResidential
Real Estate Commercial		RealEstateCommercial
Real Estate Other		RealEstateOther
Collectibles		Collectibles

Food Inventory FoodInventory

Field	Value	IRS element (for citing)
Non Cash Checkbox	X	NonCashCheckbox
Number Of Contributions	1	NumberOfContributions
Noncash Contribs Rptd F990	1198	NoncashContribsRptdF990
Method Of Determining Revenues	COST/SELLING PRICE	MethodOfDeterminingRevenues

Field	Value	IRS element (for citing)
Drugs And Medical Supplies		DrugsAndMedicalSupplies
Taxidermy		Taxidermy
Historical Artifacts		HistoricalArtifacts
Scientific Specimens		ScientificSpecimens
Archaeological Artifacts		ArchaeologicalArtifacts

Other Non Cash Contributions Table OtherNonCashContributionsTable

Field	Value	IRS element (for citing)
Non Cash Checkbox	X	NonCashCheckbox
Description	MINOR EQUIPMENT	Description
Number Of Contributions	1	NumberOfContributions
Noncash Contribs Rptd F990	2500	NoncashContribsRptdF990
Method Of Determining Revenues	COST/SELLING PRICE	MethodOfDeterminingRevenues

Field	Value	IRS element (for citing)
Any Property That Must Be Held	false	AnyPropertyThatMustBeHeld
Review Process Unusual NC Gifts	false	ReviewProcessUnusualNCGifts
Third Parties Used	false	ThirdPartiesUsed

Form990 Schedule M Part II Form990ScheduleMPartII

Field	Value	IRS element (for citing)
Identifier	CONTRIBUTIONS RECEIVED	Identifier
Return Reference	SCHEDULE M, PART I, COLUMN(B)	ReturnReference
Explanation	COLUMN B IS REPORTING A COMBINATION OF THE NUMBER OF CONTRIBUTIONS RECEIVED AND THE NUMBER OF ITEMS RECEIVED.	Explanation

Schedule O IRS990ScheduleO

General Explanation GeneralExplanation (8 entries)

IdentifierIdentifier	Return ReferenceReturnReference	ExplanationExplanation
ORGANIZATION'S MISSION	FORM 990 - PART I, LINE 1 & PART III, LINE 1	SANFORD BURNHAM MEDICAL RESEARCH INSTITUTE CONDUCTS WORLD-CLASS COLLABORATIVE RESEARCH DEDICATED TO FINDING CURES FOR HUMAN DISEASE, IMPROVING THE QUALITY OF LIFE, AND THUS CREATING A LEGACY FOR ITS EMPLOYEES, PARTNERS, DONORS, AND COMMUNITY.
PROGRAM SERVICE ACCOMPLISHMENTS	FORM 990, PART III, LINE 4A	SANFORD-BURNHAM MEDICAL RESEARCH INSTITUTE IS DEDICATED TO DISCOVERING THE FUNDAMENTAL MOLECULAR CAUSES OF DISEASE AND DEVISING THE INNOVATIVE THERAPIES OF TOMORROW. SANFORD-BURNHAM TAKES A UNIQUE, COLLABORATIVE APPROACH TO MEDICAL RESEARCH AND HAS ESTABLISHED MAJOR RESEARCH PROGRAMS IN CANCER, NEURODEGENERATION, DIABETES, AND INFECTIOUS, INFLAMMATORY, AND CHILDHOOD DISEASES. CANCER RESEARCH BREAKING A VICIOUS CYCLE IN PANCREATIC CANCER. PANCREATIC CANCER, WHICH TYPICALLY KILLS PATIENTS A YEAR AFTER DIAGNOSIS, GENERATES TUMORS COMPRISED MOSTLY OF FIBROUS TISSUE. THIS TISSUE OBSTRUCTS BLOOD FLOW, WHICH MAKES IT DIFFICULT FOR DRUGS TO REACH INSIDE TUMORS. WORK PUBLISHED BY DR. GARTH POWIS IN "CANCER RESEARCH" REPORTS THAT HYPOXIA MAY CAUSE SIGNALING THAT LEADS PANCREATIC CANCER CELLS TO RELEASE A PROTEIN THAT IN TURN CAUSES SURROUNDING NORMAL PANCREATIC TISSUE TO PRODUCE LARGE AMOUNTS OF FIBROUS TISSUE. POWIS IDENTIFIED A MOLECULE INCREASED DURING HYPOXIA CALLED HYPOXIA INDUCIBLE FACTOR (HIF) THAT TRIGGERS THE PROTEIN THAT DRIVES FIBROSIS. BY DEVELOPING A DRUG THAT INHIBITS HIF, HE BROKE THE CYCLE OF HYPOXIA AND FIBROSIS-OFFERING A POTENTIAL NEW TREATMENT. NEWLY IDENTIFIED THERAPEUTIC TARGET FOR PROSTATE CANCER. THE LABS OF DRS. MARIA T. DIAZ-MECO AND JORGE MOSCAT HAVE IDENTIFIED HOW AN ENZYME CALLED PKCZ SUPPRESSES PROSTATE TUMOR FORMATION. THE FINDING, WHICH ALSO DESCRIBES A MOLECULAR CHAIN OF EVENTS THAT CONTROLS CELL GROWTH AND METASTASIS, COULD LEAD TO NOVEL WAYS TO CONTROL DISEASE PROGRESSION. THIS STUDY, PUBLISHED IN "PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES," SUGGESTS THAT RESTORING PKCZ COULD PROVIDE A NEW APPROACH TO TREATING PROSTATE CANCER. STOPPING MELANOMA BY CHANGING A PROTEIN'S LOCATION. IN A STUDY WITH THE CHEMICAL GENOMICS GROUP, DR. ZE'EV RONAI COMPLETED A PROOF-OF-CONCEPT STUDY THAT IDENTIFIED SMALL MOLECULES THAT PROMOTE THE EXPORT OF A PROTEIN CALLED ATF2 FROM A CELL'S NUCLEUS. EARLIER WORK FROM DR. RONAI'S GROUP SHOWED THAT THE PROTEIN, WHILE INSIDE THE NUCLEUS, PROMOTES CANCER. THE PRESENCE OF ATF2 OUTSIDE THE NUCLEUS IN MITOCHONDRIA, HOWEVER, PROMPTS MELANOMA CANCER CELLS TO SELF-DESTRUCT. IN THEIR NEW STUDY, DR. RONAI'S TEAM SCREENED 3,800 COMPOUNDS TO IDENTIFY SMALL MOLECULES THAT PROMOTE THE MOVEMENT OF ATF2 TO THE CELL'S CYTOPLASM OUTSIDE THE NUCLEUS. TWO COMPOUNDS WERE FOUND TO REDUCE CELL VIABILITY, INHIBIT THE FORMATION OF CELL COLONIES, INHIBIT CELL MOTILITY, AND LEAD TO OTHER DAMAGING EFFECTS. THE STUDY, PUBLISHED IN "CLINICAL CANCER RESEARCH," POINTS THE WAY TOWARD IDENTIFYING SMALL MOLECULES THAT BLOCK THE CANCER-CAUSING FUNCTION OF ATF2. SMIP004 AS A NOVEL TREATMENT FOR ADVANCED PROSTATE CANCER. A MAJOR HALLMARK OF CANCER CELLS IS THEIR ABILITY TO SURVIVE UNDER STRESSFUL CONDITIONS. A NEW STUDY SPEARHEADED BY DR. DIETER WOLF AND HIS TEAM REVEALS HOW A PROMISING ANTICANCER COMPOUND CALLED SMIP004 SPECIFICALLY KILLS PROSTATE CANCER CELLS BY COMPROMISING THEIR ABILITY TO WITHSTAND ENVIRONMENTAL STRESS. THE STUDY, RECENTLY PUBLISHED IN "ONCOTARGET," UNCOVERS NOVEL MECHANISMS OF ANTICANCER ACTIVITY AND COULD LEAD TO THE DEVELOPMENT OF MORE EFFECTIVE THERAPIES FOR ADVANCED AND HARD-TO-TREAT FORMS OF PROSTATE CANCER, AS WELL AS OTHER TYPES OF CANCER. WHY THE SHAPE OF NANOPARTICLES MATTERS. CONVENTIONAL TREATMENTS FOR DISEASES SUCH AS CANCER CAN CARRY HARMFUL SIDE EFFECTS-AND THE PRIMARY REASON IS THAT SUCH TREATMENTS ARE NOT TARGETED SPECIFICALLY TO THE CELLS OF THE BODY WHERE THEY'RE NEEDED. DR. ERKKI RUOSLAHTI AND COLLABORATORS AT UC SANTA BARBARA FOUND THAT THE SHAPE OF NANOPARTICLES CAN ENHANCE DRUG TARGETING. THE STUDY, PUBLISHED IN "PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES," FOUND THAT ROD-SHAPED NANOPARTICLES APPEAR TO ADHERE MORE EFFECTIVELY TO THE SURFACE OF ENDOTHELIAL CELLS THAT LINE THE INSIDE OF BLOOD VESSELS. THE FINDINGS HOLD PROMISE IN THE DELIVERY OF DRUGS SUCH AS CHEMOTHERAPEUTICS, WHICH ARE HIGHLY TOXIC. A NEW APPROACH TO TREATING BRAIN CANCER. MEDULLOBLASTOMA IS THE MOST COMMON MALIGNANT BRAIN CANCER IN CHILDREN. ALTHOUGH MANY PATIENTS CAN BE CURED WITH SURGERY, RADIATION, AND HIGH-DOSE CHEMOTHERAPY, SURVIVORS OFTEN SUFFER SEVERE LONG-TERM SIDE EFFECTS SUCH AS COGNITIVE AND DEVELOPMENTAL DISABILITIES DUE TO THE AGGRESSIVE TREATMENT. A RESEARCH TEAM LED BY DR. ROBERT WECHSLER-REYA DEVELOPED A NEW EXPERIMENTAL APPROACH TO TREATING MEDULLOBLASTOMA BY TARGETING CANCER STEM CELLS-THE CELLS THAT ARE CRITICAL FOR MAINTAINING TUMOR GROWTH. THE FINDINGS, DESCRIBED IN A RECENT PAPER IN "CANCER RESEARCH," STRONGLY SUPPORT THE NOTION OF COMBINING CONVENTIONAL CHEMOTHERAPY WITH CELL-CYCLE INHIBITORS TO IMPROVE THE EFFECTIVENESS OF THERAPY FOR MEDULLOBLASTOMA PATIENTS. DIABETES, OBESITY AND CARDIOVASCULAR RESEARCH DIFFERENCES BETWEEN "MARATHON MICE" AND "COUCH POTATO MICE" REVEAL KEY TO MUSCLE FITNESS. DR. DANIEL KELLY AND COLLEAGUES DISCOVERED THAT SMALL PIECES OF GENETIC MATERIAL CALLED MICRORNAS LINK THE TWO DEFINING CHARACTERISTICS OF FIT MUSCLES: THE ABILITY TO BURN SUGAR AND FAT, AND THE SWITCH BETWEEN SLOW- AND FAST-TWITCH MUSCLE FIBERS. THE TEAM USED TWO COMPLEMENTARY MOUSE MODELS-THE "MARATHON MOUSE" AND THE "COUCH POTATO MOUSE"-TO MAKE THIS DISCOVERY. THE FINDINGS, PUBLISHED IN "THE JOURNAL OF CLINICAL INVESTIGATION," SUGGEST MICRORNAS COULD BE TARGETED FOR THE DEVELOPMENT OF NEW MEDICAL INTERVENTIONS AIMED AT IMPROVING MUSCLE FITNESS IN PEOPLE WITH CHRONIC ILLNESS OR INJURY. PROGRESS TOWARD A CURE FOR DIABETES. IN BOTH TYPE 1 AND TYPE 2 DIABETES, LOSS OF THE PANCREATIC BETA-CELLS THAT PRODUCE INSULIN IN RESPONSE TO GLUCOSE IS A CRITICAL FEATURE OF THE DISEASE. IN TWO PAPERS PUBLISHED IN THE JOURNAL "STEM CELLS," DR. FRED LEVINE'S LAB DEMONSTRATES PROGRESS TOWARD CREATING FUNCTIONAL BETA CELLS. IN THE FIRST PAPER, THEY DEVELOPED A NEW MODEL OF RECONSTITUTING A NORMAL MASS OF FUNCTIONAL BETA CELLS IN MICE WHERE ALMOST ALL BETA CELLS HAD BEEN ELIMINATED. IN THEIR SECOND PAPER, THEY SHOW THAT A DRUG THEY DEVELOPED POTENTLY INDUCES BETA CELLS TO REPLICATE. THEY ARE WORKING ON A DRUG THAT CAN INDUCE BETA-CELL NEOGENESIS THAT, WHEN COMBINED WITH THE DRUG THAT INDUCES BETA-CELL REPLICATION, WILL PROVIDE A POTENT MIX TO INDUCE THE RECOVERY OF A NORMAL BETA-CELL MASS IN DIABETIC PATIENTS. FULL VISIBILITY OF NOVEL FACTORS IMPORTANT FOR INSULIN SECRETION. DR. PAMELA ITKIN-ANSARI AND COLLABORATORS AT THE SCRIPPS RESEARCH INSTITUTE HAVE REPORTED THE FIRST COMPREHENSIVE MAP OF THE PROTEIN INTERACTIONS THAT ENABLED CELLS IN THE PANCREAS TO PRODUCE, STORE, AND SECRETE INSULIN. THE MAP, CALLED AN "INSULIN-INTERACTOME" MAP, PROVIDES FULL VISIBILITY OF THE INTRICACIES OF THE INSULIN-SECRETION PROCESS. THE STUDY, PUBLISHED IN "CELL REPORT," HAS ALREADY YIELDED NOVEL FINDINGS, INCLUDING THE KEY ROLE OF A PROTEIN CALLED TMEM. THE STUDY IS IMPORTANT BECAUSE IT PROVIDES A DEEPER SCIENTIFIC UNDERSTANDING OF THE INSULIN-SECRETION PROCESS, WHICH MAY YIELD NEW TARGETS FOR THERAPEUTIC INTERVENTION IN DIABETES. NEW OBESITY DRUG REDUCES "BAD" BELLY FAT. BELLY FAT, ALSO CALLED VISCERAL FAT, IS LINKED TO AN INCREASED RISK OF HEART DISEASE, DIABETES, AND STROKE. IN A PAPER PUBLISHED IN "DIABETES, OBESITY AND METABOLISM," DR. STEVEN SMITH AND HIS COLLABORATORS TESTED WHETHER A NEW OBESITY DRUG IN LATE STAGE CLINICAL TESTING CALLED "CONTRAVE" MIGHT REDUCE "BAD" ABDOMINAL FAT. "BAD" FAT IS ASSOCIATED WITH COMPLICATIONS OF OBESITY SUCH AS DIABETES AND CARDIOVASCULAR DISEASE. THEY FOUND THAT THE DRUG, IN COMBINATION WITH OTHER THERAPIES, DECREASED "BAD" FAT, WITH SOME PATIENTS SHOWING UP TO A 40 PERCENT REDUCTION. CONTRAVE IS NOW BEING TESTED IN A LARGE MULTI-CENTER STUDY FOR ITS AFFECT ON CARDIOVASCULAR EVENTS, SUCH AS HEART ATTACKS. DISCOVERY OF A NEW MECHANISM LEADING TO ATHEROSCLEROSIS. IN A PAPER PUBLISHED IN "ARTERIOSCLEROSIS, THROMBOSIS, AND VASCULAR BIOLOGY," DR. DWIGHT TOWLER AND COLLEAGUES DESCRIBED A PATHWAY THAT LEADS TO HARDENING OF ARTERIES (ATHEROSCLEROSIS). THIS WORK ZEROED IN ON A PARTICULARLY DELETERIOUS PROTEIN CALLED DKK1. WHEN FUNCTIONING NORMALLY, DKK1 IS IMPORTANT FOR AIDING IN WOUND REPAIR. BUT INFLAMMATORY RESPONSES TRIGGERED INSIDE ARTERY WALLS ASSOCIATED WITH DISEASES LIKE DIABETES CAN TRIGGER PROLONGED AND DESTRUCTIVE DKK1 ACTIVITY. TOWLER AND HIS TEAM WILL CONTINUE TO STUDY DKK1 TO IDENTIFY POTENTIAL DRUG TARGETS TO PREVENT HARDENING OF ARTERIES IN PATIENTS WITH ATHEROSCLEROSIS.
PROGRAM SERVICE ACCOMPLISHMENTS CONTINUED		PATIENTS' OWN SKIN CELLS ARE TRANSFORMED INTO HEART CELLS TO CREATE "DISEASE IN A DISH." MOST PATIENTS WITH ARRHYTHMOGENIC RIGHT VENTRICULARDYSPLASIA/CARDIOMYOPATHY (ARVD/C) DON'T KNOW THEY HAVE A PROBLEM UNTIL THEY'RE IN THEIR EARLY20S-WHEN THEY'RE DIAGNOSED AT ADVANCED STAGES OR AFTER SUDDEN DEATH. A TECHNIQUE CALLED "DISEASE IN A DISH" MIGHT BRING NEW HOPE TO ARVD/C PATIENTS. DR. HUEI-SHENG VINCENT CHEN AND HIS TEAM RECENTLY TOOK SKIN SAMPLES FROM TWO ARVD/C PATIENTS. THEN, USING STEM CELL-BASED TECHNOLOGY THAT GARNERED A 2012 NOBEL PRIZE, THEY CONVERTED THE SKIN CELLS INTO HEART CELLS. IN A PAPER PUBLISHED IN "NATURE," THE RESEARCHERS UNVEILED THE FIRST MATURATION-BASED "DISEASE IN A DISH" MODEL FOR ARVD/C. THE MODEL WAS CREATED IN COLLABORATION WITH DR. DANIEL KELLY, AND IS LIKELY MORE RELEVANT TO HUMAN ARVD/C THAN OTHER MODELS AND BETTER SUITED FOR STUDYING THE DISEASE AND TESTING NEW TREATMENTS. INFECTIOUS AND INFLAMMATORY DISEASE THWARTING INFLAMMATION. VIRUSES HAVE EVOLVED TO EVADE THE IMMUNE SYSTEM, WHICH IS WHY THEY CAUSE INFECTIONS. HOWEVER, SOME VIRUSES, LIKE THE SMALLPOX VIRUS (NOW ERADICATED WORLDWIDE), HAVE MANY TRICKS TO THWART THE IMMUNE SYSTEM. A STUDY FROM DRS. REED'S AND LIDDINGTON'S LABORATORIES DEMONSTRATED HOW VACCINIA VIRUS, THE VACCINE STRAIN USED AGAINST SMALL-POX VIRUS, SUPPRESSES INFLAMMATION. PUBLISHED IN "PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCE," THE RESULTS DEMONSTRATE A VIRAL PROTEIN CALLED F1L THWARTS INFLAMMATION BY ALTERING A KEY HOST FACTOR KNOWN AS NLRP1. INTERESTINGLY, A SMALL PEPTIDE FROM F1L BLOCKS INFLAMMATION, SUGGESTING IT MAY HAVE POTENTIAL AS A DRUG TO TREAT ARTHRITIC CONDITIONS. PREVENTING AUTOIMMUNE DISEASES. B-CELL PRODUCTION IN BONE MARROW GENERATES A VAST REPERTOIRE OF ANTIBODY-PRODUCING CELLS THAT COLLECTIVELY RECOGNIZE VIRTUALLY ANY FOREIGN ENTITY, INCLUDING PATHOGEN COMPONENTS-BUT IT CAN ALSO PRODUCES B CELLS THAT IN TURN PRODUCE AUTOANTIBODIES THAT CAN RECOGNIZE "SELF-ANTIGENS," LEADING TO AUTOIMMUNE DISEASES. IN THE "JOURNAL OF IMMUNOLOGY," DR. ROBERT RICKERT'S LAB INVESTIGATED THE CELLULAR PROCESSES THAT CONTROL B-CELL TOLERANCE, WHICH PREVENTS B CELLS FROM PRODUCING ANTIBODIES TO SELF-ANTIGENS. THEY DEVELOPED A NOVEL MODEL TO SHOW THAT FOLLICULAR DENDRITIC CELLS (FDCS) KEY TO ELIMINATING AUTO-REACTIVE B CELLS TO SELF-ANTIGENS. THE FINDINGS PROVIDE A NEW PERSPECTIVE ON HOW AUTOIMMUNE DISEASE MAY BE PREVENTED. THE RESEARCH WAS RECENTLY RECOGNIZED TO BE OF SPECIAL SIGNIFICANCE BY THE FACULTY OF 1000. ONE IN A MILLION. THE IMMUNE SYSTEM PROTECTS US AGAINST INFECTIONS. BUT, SOMETIMES IT TURNS AGAINST US CAUSING AUTOIMMUNE DISEASE. IN RHEUMATOID ARTHRITIS, IMMUNE CELLS TERMED LYMPHOCYTES, SECRETE TNF (TUMOR NECROSIS FACTOR), A PROTEIN THAT CAN PROMOTE DESTRUCTION OF THE JOINTS, CAUSING PAIN AND SEVERE BONE EROSION. ANTI-TNF THERAPY IS SUCCESSFULLY TREATS SOME PATIENTS WITH AUTOIMMUNE DISEASE. IN THE "JOURNAL OF IMMUNOLOGY," DRS. CARL WARE AND SALVO ALBANI REPORT ON AN ATYPICAL LYMPHOCYTE THAT CONTINUOUSLY SECRETES TNF DETECTED AT ABOUT ONE IN A MILLION CELLS IN THE BLOOD OF NORMAL PEOPLE. THESE ATYPICAL LYMPHOCYTES ARE DRAMATICALLY INCREASED IN THE BLOOD OF PATIENTS WITH RHEUMATOID ARTHRITIS. THEIR GOAL IS TO UNDERSTAND WHY THESE CELLS ARE ELEVATED IN AUTOIMMUNE DISEASE. NEUROSCIENCE, AGING AND STEM CELL RESEARCH NOVEL DRUG REVERSES LOSS OF BRAIN CONNECTIONS IN MODELS OF ALZHEIMER'S. DR. STUART LIPTON AND HIS TEAM HAVE DEVELOPED THE FIRST EXPERIMENTAL DRUG TO BOOST BRAIN SYNAPSES LOST IN ALZHEIMER'S DISEASE. THE DRUG, NITROMEMANTINE, COMBINES TWO FDA-APPROVED MEDICINES, NITROGLYCERIN AND MEMANTINE, INTO A NOVEL SINGLE AGENT. TOGETHER, THEY STOP THE DESTRUCTIVE CASCADE OF CHANGES IN THE BRAIN THAT DESTROYS THE CONNECTIONS BETWEEN NEURONS, LEADING TO MEMORY LOSS AND COGNITIVE DECLINE. THE RESEARCH, PUBLISHED IN "PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES," FOCUSES ON A DOWNSTREAM TARGET TO TREAT ALZHEIMER'S, RATHER THAN ON AMYLOID BETA PLAQUES AND NEUROFIBRILLARY TANGLES-APPROACHES THAT HAVE SHOWN LITTLE SUCCESS IN THE PAST. UNRAVELING THE MOLECULAR ROOTS OF DOWN SYNDROME. DR. HUAXI XU'S LABORATORY HAS FOUND THAT PROTEIN CALLED SORTING NEXIN 27, OR SNX27, IS REDUCED IN HUMAN DOWN SYNDROME BRAINS. THE STUDY, PUBLISHED IN "NATURE MEDICINE," SHOWS THAT SNX27 HELPS KEEP GLUTAMATE RECEPTORS ON THE CELL SURFACE IN NEURONS. NEURONS NEED GLUTAMATE RECEPTORS IN ORDER TO FUNCTION CORRECTLY. WITH LESS SNX27, MICE HAD FEWER ACTIVE GLUTAMATE RECEPTORS AND THUS IMPAIRED LEARNING AND MEMORY. FURTHER STUDY REVEALED THAT CHROMOSOME 21 ENCODES ONE PARTICULAR MICRORNA CALLED MIR-155. IN HUMAN DOWN SYNDROME BRAINS, THE INCREASE IN MIR-155 LEVELS CORRELATES ALMOST PERFECTLY WITH THE DECREASE IN SNX27. THROUGH THIS MECHANISM, LEARNING, MEMORY, AND BEHAVIOR ARE IMPAIRED. TRANSPLANTED NEURAL STEM CELLS TREAT ALS IN MOUSE MODEL. AMYOTROPHIC LATERAL SCLEROSIS (ALS), ALSO KNOWN AS LOU GEHRIG'S DISEASE, IS UNTREATABLE AND FATAL. A CONSORTIUM OF ALS RESEARCHERS AT MULTIPLE INSTITUTIONS, LED BY DR. EVAN SNYDER, TESTED TRANSPLANTED NEURAL STEM CELLS AS A TREATMENT FOR THE DISEASE. IN 11 INDEPENDENT STUDIES, THEY FOUND THAT TRANSPLANTING NEURAL STEM CELLS INTO THE SPINAL CORD OF A MOUSE MODEL OF ALS SLOWS DISEASE ONSET AND PROGRESSION. THE NEURAL STEM CELLS HELP BY PRODUCING FACTORS THAT PRESERVE THE HEALTH AND FUNCTION OF THE HOST'S REMAINING NERVE CELLS. THESE FINDINGS, PUBLISHED IN "SCIENCE TRANSLATIONAL MEDICINE," DEMONSTRATE THE POTENTIAL NEURAL STEM CELLS HOLD FOR TREATING ALS AND OTHER NERVOUS SYSTEM DISORDERS. A KEY PROTEIN THAT MODULATES ORGANISMAL AGING. A STUDY DIRECTED BY DR. MALENE HANSEN IDENTIFIED A KEY FACTOR THAT REGULATES THE AUTOPHAGY PROCESS WHERE CELLULAR DEBRIS IS DIGESTED TO PROTECT CELLS FROM DAMAGE, AND WHICH IN TURN MODULATES AGING. HER LAB AND COLLABORATORS IDENTIFIED A TRANSCRIPTION FACTOR - AN ON/OFF SWITCH FOR GENES - CALLED HLH-30 THAT INDUCES AUTOPHAGY IN MODEL ORGANISMS, INCLUDING THE WORM C. ELEGANS. SINCE A SIMILAR TRANSCRIPTION FACTOR, TFEB, REGULATES AUTOPHAGY IN MAMMALIAN CELLS, THESE FINDINGS, PUBLISHED IN "NATURE COMMUNICATIONS," COULD LEAD TO THE DEVELOPMENT OF NEW THERAPIES FOR AGE-RELATED DISORDERS THAT ARE CHARACTERIZED BY A BREAKDOWN IN THIS PROCESS, FOR EXAMPLE, IN NEURODEGENERATIVE AND CARDIOVASCULAR DISEASES. CHILDREN'S HEALTH DISCOVERING AN ORAL DRUG TO PREVENT METABOLIC SYNDROME. DR. JOS LUIS MILLN'S LABORATORY AND COLLABORATORS AT MASSACHUSETTS GENERAL HOSPITAL AND HARVARD MEDICAL SCHOOL HAVE ADVANCED STUDIES SHOWING THAT ORAL ADMINISTRATION OF INTESTINAL ALKALINE PHOSPHATASE (IAP)-A GUT MUCOSAL DEFENSE FACTOR IS BENEFICIAL IN MOUSE MODELS OF CHRONIC COLITIS. IN "PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES," THE RESEARCHERS NOW SHOW THAT ORAL ADMINISTRATION OF IAP INHIBITS THE ABSORPTION OF LIPOPOLYSACCHARIDES, A MAJOR COMPONENT OF THE OUTER MEMBRANE OF BACTERIA. THE ABSORPTION OF LIPOPOLYSACCHARIDES OCCURS IN TANDEM WITH THE ABSORPTION OF DIETARY FAT. IAP, THEREFORE, IMPROVES THE LIPID PROFILE IN MICE THAT ARE FED A STANDARD, LOW-FAT DIET. THE FINDINGS MAY LEAD TO NEW WAYS TO PREVENT THE ONSET OF METABOLIC SYNDROME, A PRE-DIABETIC CONDITION. A NEW APPROACH TO PREVENTING LIVER FAILURE IN CHILDREN. WHEN CELL PROTEINS ARE MISFOLDED, THEY AGGREGATE IN THE CELL AND CAUSE CELL DEATH. IN A REPORT PUBLISHED IN "MOLECULAR BIOLOGY OF THE CELL," DR. RANDAL KAUFMAN'S LAB DESCRIBED HOW THE ENZYME "UGGT1" IS REQUIRED TO PREVENT PROTEIN AGGREGATION. THE UGGT1 ENZYME, CODED FOR BY A GENE THAT WAS FIRST ISOLATED BY KAUFMAN YEARS AGO, WAS INITIALLY THOUGHT TO PREVENT THE RELEASE OF MISFOLDED PROTEINS FROM THE ENDOPLASMIC RETICULUM. THE CURRENT STUDY USED MUTANT FORMS OF THE ?1-ANTITRYPSIN (?1-AT) LIVER PROTEIN TO SHOW THAT UGGT1 ACTUALLY PREVENTS AGGREGATION. SINCE MUTATIONS IN THE ?1-AT GENE THAT CAUSE PROTEIN AGGREGATION ARE THE MOST COMMON CAUSE OF LIVER FAILURE IN CHILDREN, THESE FINDINGS MAY GUIDE AVENUES TO TREAT AND/OR PREVENT THE MOST COMMON DEVASTATING LIVER DISEASE IN CHILDREN. GENOME-SEQUENCING DIAGNOSTICS FOR RARE DISEASES: A CAUTIONARY TALE. PROPERLY DIAGNOSING A CHILD BORN WITH CONGENITAL DISORDERS OF GLYCOSYLATION, OR CDG, AND PINPOINTING THE EXACT SUGAR GENE THAT'S MUTATED PROVIDES DOCTORS WITH USEFUL INFORMATION TO DEVELOP A THERAPEUTIC APPROACH. WHOLE-EXOME SEQUENCING, AN ABBREVIATED FORM OF WHOLE-GENOME SEQUENCING, IS INCREASINGLY USED AS A DIAGNOSTIC FOR CDG. BUT RESEARCHERS LED BY DR. HUDSON FREEZE RECENTLY DISCOVERED THREE CHILDREN WITH CDG WHO ARE MOSAICS-ONLY SOME CELLS IN SOME TISSUES HAVE THE MUTATION. FOR THAT REASON, STANDARD EXOME SEQUENCING INITIALLY MISSED THEIR MUTATIONS, HIGHLIGHTING THE TECHNIQUE'S DIAGNOSTIC LIMITATIONS IN SOME RARE CASES. THESE FINDINGS WERE PUBLISHED IN THE "AMERICAN JOURNAL OF HUMAN GENETICS."
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OTHER CHANGES IN NET ASSETS OR FUND BALANCES	FORM 990, PART XI, LINE 5	UNREALIZED GAIN (LOSS) ON INTEREST RATE SWAP: $1,306,042 ROUNDING: $ 312 ----------- $1,306,354