Attach to Form 990 or Form 990-EZ.
See separate instructions.| (i) Name of supported organization |
(ii) EIN |
(iii) Type of organization (described on lines 1- 9 above or IRC section (see instructions)) |
(iv) Is the organization in col. (i) listed in your governing document? |
(v) Did you notify the organization in col. (i) of your support? |
(vi) Is the organization in col. (i) organized in the U.S.? |
(vii) Amount of support? |
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|---|---|---|---|---|---|---|---|---|---|
| Yes | No | Yes | No | Yes | No | ||||
| Total | |||||||||
| Calendar year(or fiscal year beginning in) | (a) 2007 | (b) 2008 | (c) 2009 | (d) 2010 | (e) 2011 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 1 | Gifts, grants, contributions, and membership fees received. (Do not include any "unusual grants.") .... | 103,328,720 | 209,859,230 | 150,769,181 | 160,093,433 | 156,880,684 | 780,931,248 |
| 2 | Tax revenues levied for the organization's benefit and either paid to or expended on its behalf....... | ||||||
| 3 | The value of services or facilities furnished by a governmental unit to the organization without charge.. | ||||||
| 4 | Total. Add lines 1 through 3.. | 103,328,720 | 209,859,230 | 150,769,181 | 160,093,433 | 156,880,684 | 780,931,248 |
| 5 | The portion of total contributions by each person (other than a governmental unit or publicly supported organization) included on line 1 that exceeds 2% of the amount shown on line 11, column (f).. | 14,453,736 | |||||
| 6 | Public Support. Subtract line 5 from line 4. | 766,477,512 | |||||
| Calendar year(or fiscal year beginning in) | (a) 2007 | (b) 2008 | (c) 2009 | (d) 2010 | (e) 2011 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 7 | Amounts from line 4.. | 103,328,720 | 209,859,230 | 150,769,181 | 160,093,433 | 156,880,684 | 780,931,248 |
| 8 | Gross income from interest, dividends, payments received on securities loans, rents, royalties and income from similar sources.. | 3,955,763 | 1,995,011 | 1,354,102 | 2,037,946 | 4,402,201 | 13,745,023 |
| 9 | Net income from unrelated business activities, whether or not the business is regularly carried on.. | ||||||
| 10 | Other income. (Explain in Part IV.) Do not include gain or loss from the sale of capital assets.. | 303,840 | 286,205 | 343,630 | 907,754 | 370,643 | 2,212,072 |
| 11 | Total support (Add lines 7 through 10). | 796,888,343 | |||||






| Calendar year(or fiscal year beginning in) | (a) 2007 | (b) 2008 | (c) 2009 | (d) 2010 | (e) 2011 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 1 | Gifts, grants, contributions, and membership fees received. (Do not include any "unusual grants.") . | ||||||
| 2 | Gross receipts from admissions, merchandise sold or services performed, or facilities furnished in any activity that is related to the organization's tax-exempt purpose...... | ||||||
| 3 | Gross receipts from activities that are not an unrelated trade or business under section 513.. | ||||||
| 4 | Tax revenues levied for the organization's benefit and either paid to or expended on its behalf... | ||||||
| 5 | The value of services or facilities furnished by a governmental unit to the organization without charge.. | ||||||
| 6 | Total. Add lines 1 through 5. | ||||||
| 7a | Amounts included on lines 1, 2, and 3 received from disqualified persons... | ||||||
| b | Amounts included on lines 2 and 3 received from other than disqualified persons that exceed the greater of $5,000 or 1% of the amount on line 13 for the year. | ||||||
| c | Add lines 7a and 7b.. | ||||||
| 8 | Public Support (Subtract line 7c from line 6.) | ||||||
| Calendar year (or fiscal year beginning in) | (a) 2007 | (b) 2008 | (c) 2009 | (d) 2010 | (e) 2011 | (f) Total | |
|---|---|---|---|---|---|---|---|
| 9 | Amounts from line 6... | ||||||
| 10a | Gross income from interest, dividends, payments received on securities loans, rents, royalties and income from similar sources.. | ||||||
| b | Unrelated business taxable income (less section 511 taxes) from businesses acquired after June 30, 1975. | ||||||
| c | Add lines 10a and 10b. | ||||||
| 11 | Net income from unrelated business activities not included in line 10b, whether or not the business is regularly carried on. | ||||||
| 12 | Other income. Do not include gain or loss from the sale of capital assets (Explain in Part IV.) | ||||||
| 13 | Total support (Add lines 9, 10c, 11 and 12.). | ||||||




| Facts And Circumstances Test |
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| Explanation |
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| Software ID: | |
| Software Version: |
Attach to Form 990 or 990-EZ.| Identifier | Return Reference | Explanation |
|---|---|---|
| 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. IN THE AREA OF CANCER RESEARCH: SWARMING NANOPARTICLES FIGHT CANCER. RESEARCHERS HAVE BEEN WORKING FOR DECADES TO DEVELOP NANOPARTICLES THAT DELIVER CANCER DRUGS DIRECTLY TO TUMORS, MINIMIZING THE TOXIC SIDE EFFECTS OF CHEMOTHERAPY. A TEAM OF RESEARCHERS, INCLUDING DR. ERKKI RUOSLAHTI, DESIGNED A DELIVERY SYSTEM IN WHICH NANOPARTICLES HOME IN ON A TUMOR AND THEN CALL IN A MUCH LARGER SECOND WAVE OF NANOPARTICLES TO DISPENSE AN ANTI-CANCER DRUG. THIS COMMUNICATION BETWEEN NANOPARTICLES, ENABLED BY THE BODY'S OWN BIOCHEMISTRY, BOOSTS DRUG DELIVERY TO TUMORS MORE THAN 40-FOLD IN MOUSE MODELS. THE STUDY WAS PUBLISHED IN "NATURE MATERIALS." HOW CANCER CELLS SURVIVE STRESS. CANCER CELLS SURVIVE DURING TIMES WHEN NUTRIENTS ARE SCARCE USING A PROCESS CALLED AUTOPHAGY. A STUDY LED BY THE LABORATORY OF DR. JOHN REED FOUND THAT A PROTEIN KNOWN AS BI-1 REGULATES THIS MECHANISM. AUTOPHAGY, LITERALLY MEANING "SELF-EATING," IS A WAY FOR CELLS TO RE-USE THEIR INTERNAL COMPONENTS AND THUS MAINTAIN CRITICAL LEVELS OF ENERGETIC MOLECULES. THE STUDY, PUBLISHED IN GENES AND DEVELOPMENT, ALSO DEMONSTRATED THAT MICE WITH LOW BI-1 HAVE LOWER AUTOPHAGY RATES. IN ADDITION, EXPERIMENTALLY REDUCING BI-1 EXPRESSION IMPAIRED TUMOR GROWTH IN MICE. THE WORK UNCOVERED A NEW AREA RELEVANT TO THE UNDERSTANDING OF MECHANISMS OF CANCER DEVELOPMENT AND PROGRESSION. MOLECULAR SWITCH THAT ALLOWS MELANOMA TO RESIST THERAPY, IDENTIFIED. THE NATIONAL CANCER INSTITUTE (NCI) ESTIMATES THAT AS MANY AS ONE IN 51 MEN AND WOMEN WILL BE DIAGNOSED WITH MELANOMA-THE DEADLIEST FORM OF SKIN CANCER-AT SOME POINT DURING THEIR LIFETIMES. A RESEARCH TEAM LED BY DR. ZE'EV RONAI HAS BEEN STUDYING A PROTEIN NAMED ACTIVATING TRANSCRIPTION FACTOR 2 (ATF2), WHICH IS ASSOCIATED WITH POOR PROGNOSIS IN MELANOMA. THE FINDING PROVIDES THE BASIS FOR HIGH-CONTENT SCREENING, IN COLLABORATION WITH SANFORD-BURNHAM'S CONRAD PREBYS CENTER FOR CHEMICAL GENOMICS, WHERE RESEARCHERS ARE LOOKING FOR SMALL MOLECULES THAT CAUSE ATF2'S EXIT FROM THE NUCLEUS, WITH THE EXPECTATION THOSE WILL ALSO BE ABLE TO SENSITIZE MELANOMA TO THERAPY. THE WEEK IT WAS PUBLISHED, THIS STUDY WAS SELECTED BY THE NATIONAL CANCER INSTITUTE (NCI) AS ONE OF THE PAPERS HIGHLIGHTED ON ITS HOMEPAGE. TOWARDS PERSONALIZED BREAST CANCER THERAPY. DR. TIMOTHY OSBORNE AND COLLABORATORS RECENTLY FOUND A UNIQUE FINGERPRINT OF A SUBSET OF BREAST CANCERS. THIS STUDY, PUBLISHED IN THE JOURNAL CELL, FOUND THAT MUTANT FORMS OF THE PROTEIN P53 "PIGGYBACK" ONTO THE STEROL REGULATORY ELEMENT BINDING PROTEIN-2 (SREBP-2) TO REGULATE GENES REQUIRED FOR FAT SYNTHESIS IN SOME BREAST TUMOR CELLS. THE PRODUCTION OF FAT IS AN ESSENTIAL MECHANISM FOR THE UNCONTROLLED GROWTH OF TUMOR CELLS. MUTATIONS IN P53 ARE AMONG THE MOST COMMON CHANGES IN CANCER. THEREFORE, THIS PATHWAY REPRESENTS A TARGET FOR NEW THERAPIES THAT COULD BE HIGHLY USEFUL FOR PATIENTS WITH THIS SPECIFIC DISEASE MARKER-A PERSONALIZED CANCER TREATMENT APPROACH. SEARCHING FOR NEW CANCER DRUGS THAT INHIBIT THE SPREAD OF TUMORS. METASTASIS IS THE MOST COMMON REASON THAT CANCER TREATMENTS FAIL. TO METASTASIZE, SOME TYPES OF CANCER CELLS RELY ON INVADOPODIA, CELLULAR MEMBRANE PROJECTIONS THAT ACT LIKE FEET, HELPING THEM "WALK" AWAY FROM THE PRIMARY TUMOR AND INVADE SURROUNDING TISSUES. DR. SARA COURTNEIDGE AND SCIENTISTS IN THE CONRAD PREBYS CENTER FOR CHEMICAL GENOMICS SCREENED A COLLECTION OF CHEMICAL COMPOUNDS AND SUCCESSFULLY IDENTIFIED SOME COMPOUNDS THAT INHIBIT INVADOPODIA FORMATION AND SOME THAT ACTUALLY INCREASE THEIR NUMBER. ONE OF THE PRO-INVADOPODIA COMPOUNDS WAS THE CHEMOTHERAPEUTIC AGENT PACLITAXEL (TAXOL)-A FINDING THAT MIGHT HAVE IMPLICATIONS FOR THE DRUG'S CURRENT USE IN TREATING CANCER, IMPLYING THAT TAXOL MIGHT MAKE MATTERS WORSE BY ENCOURAGING METASTASIS! THESE FINDINGS WERE PUBLISHED IN "SCIENCE SIGNALING." CANCER DRUG'S SECRET TO RESISTANCE. DRUG RESISTANCE IS A SERIOUS PROBLEM FOR CANCER PATIENTS-OVER TIME, A THERAPY THAT WAS ONCE PROVIDING SOME BENEFIT SIMPLY STOPS WORKING. DR. MATTHEW PETROSKI AND COLLEAGUES RECENTLY DISCOVERED HOW CANCER CELLS DEVELOP RESISTANCE TO A DRUG CALLED MLN4924. THIS EXPERIMENTAL THERAPY IS CURRENTLY BEING TESTED IN A NUMBER OF PHASE I AND PHASE I/II CLINICAL TRIALS TO DETERMINE ITS EFFICACY AGAINST SEVERAL DIFFERENT TYPES OF CANCER, INCLUDING MULTIPLE MYELOMA, LEUKEMIA, AND LYMPHOMA. THE RESEARCH WAS PUBLISHED ONLINE MARCH 19 BY "CELL REPORTS." IN THE AREA OF NEUROSCIENCE AND AGING RESEARCH: GETTING TO THE ROOT OF ALZHEIMER'S DISEASE. ALZHEIMER'S DISEASE IS CHARACTERIZED BY ABNORMAL PROTEINS THAT STICK TOGETHER IN LITTLE GLOBS, DISRUPTING COGNITIVE FUNCTION. THESE STICKY PROTEINS ARE MOSTLY MADE UP OF BETA-AMYLOID PEPTIDE. A RESEARCH TEAM LED BY DR. STUART LIPTON FOUND THAT BETA-AMYLOID-INDUCED DESTRUCTION OF SYNAPSES-THE CONNECTIONS THAT MEDIATE COMMUNICATION BETWEEN NERVE CELLS-IS DRIVEN BY A CHEMICAL MODIFICATION TO AN ENZYME CALLED CDK5. THE TEAM FOUND THAT THIS ALTERED FORM OF CDK5 (SNO-CDK5) WAS PREVALENT IN HUMAN ALZHEIMER'S DISEASE BRAINS, BUT NOT IN NORMAL BRAINS. THESE RESULTS, PUBLISHED IN THE "PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES USA," SUGGEST THAT SNO-CDK5 COULD BE TARGETED FOR THE DEVELOPMENT OF NEW ALZHEIMER'S DISEASE THERAPIES. A SNAPSHOT ON VITAMIN A FUNCTION. DR. GREGG DUESTER'S LABORATORY HAS BEEN WORKING ON VITAMIN A FUNCTION FOR MORE THAN 20 YEARS AND HAS PROVIDED MANY OF THE BREAKTHROUGHS, PARTICULARLY ITS FUNCTION IN GROWTH AND DEVELOPMENT AFTER BEING METABOLIZED TO RETINOIC ACID. THE DUESTER LAB RECENTLY REPORTED ON RETINOIC ACID SIGNALING IN THE JOURNAL "CELL" THAT SUMMARIZED THE PATHWAY FOR VITAMIN A METABOLISM TO RETINOIC ACID AND THE GENES REGULATED BY RETINOIC ACID DURING GROWTH AND DEVELOPMENT. RETINOIC ACID IS A KEY AGENT OF CELLULAR DIFFERENTIATION NEEDED FOR GENERATION OF TISSUES FROM STEM CELLS DURING EMBRYONIC DEVELOPMENT, PLUS IT HELPS ADULTS REGENERATE DAMAGED TISSUES AND PREVENTS CANCER CELL GROWTH. STEM CELLS AND SPINAL CORD REPAIR. DR. EVAN SNYDER WAS INVITED BY THE "NEW ENGLAND JOURNAL OF MEDICINE" TO PROVIDE A FORMULATION OF THE STATE-OF-THE-ART OF STEM CELL BIOLOGY AS IT APPLIES TO SPINAL CORD INJURY. HIS CRITIQUE AND ADVICE FOR FUTURE DIRECTIONS IN THAT FIELD WERE PUBLISHED AS PART OF THE "JOURNAL'S" SERIES ON "CLINICAL IMPLICATIONS OF BASIC RESEARCH." AN EASIER (AND SAFER) ROUTE TO STEM CELL THERAPIES. RECENTLY, SCIENTISTS HAVE FIGURED OUT HOW TO GENETICALLY ENGINEER A SPECIAL KIND OF STEM CELL CALLED AN INDUCED PLURIPOTENT STEM CELL (IPSC) FROM ALMOST ANY TYPE OF ADULT CELL, SUCH AS A SKIN CELL. RESEARCHERS CAN THEN USE IPSCS TO STUDY HUMAN DEVELOPMENT OR TO CREATE "DISEASE IN A DISH," A TECHNIQUE THAT ALLOWS THEM TO MODEL AN INDIVIDUAL PATIENT'S SPECIFIC DISEASE AND SCREEN FOR PERSONALIZED TREATMENTS. DR. TARIQ RANA AND HIS TEAM UNCOVERED TWO NEW CHEMICAL COMPOUNDS THAT HAVE THE POWER TO DIAL UP SOME GENES AND TONE DOWN OTHERS IN A WAY THAT GENERATES IPSCS IN A SAFER AND MORE PREDICTABLE WAY. THESE FINDINGS, PUBLISHED IN STEM CELLS, SIGNIFICANTLY ADVANCES IPSC RESEARCH, BRINGING THESE SPECIAL CELLS CLOSER TO THERAPEUTIC APPLICATIONS. |
| PROGRAM SERVICE ACCOMPLISHMENTS CONTINUED | IN THE AREA OF DIABETES AND OBESITY RESEARCH: SUGAR-BURNING MOUSE. MUSCLE PERFORMANCE AND GENERAL FITNESS ARE PARTLY DETERMINED BY HOW WELL YOUR MUSCLE CELLS CAN USE SUGAR AS A FUEL SOURCE. CERTAIN DISEASE STATES SUCH AS OBESITY OR DIABETES REDUCE THE MUSCLE'S CAPACITY TO BURN SUGAR. DR. DANIEL KELLY AND HIS LABORATORY UNRAVELED A MECHANISM THAT INCREASES SUGAR BURNING IN MUSCLE. THE TEAM FOUND THAT TRIGGERING THIS EFFECT IN MICE MIMICS THE EFFECTS OF EXERCISE TRAINING. THESE FINDINGS, PUBLISHED IN THE JOURNAL "GENES & DEVELOPMENT," REVEAL NEW CANDIDATE DRUG TARGETS FOR COMMON METABOLIC DISEASES, SUCH AS OBESITY-RELATED DIABETES. HEART HORMONE SHAPES FAT METABOLISM. A NEW STUDY LED BY DR. SHEILA COLLINS AND HER LABORATORY SUGGESTS THAT THE HEART PLAYS A ROLE IN BREAKING DOWN FAT. THE STUDY, PUBLISHED IN THE "JOURNAL OF CLINICAL INVESTIGATION," DETAILS HOW HORMONES RELEASED BY THE HEART STIMULATE FAT CELL METABOLISM. THE HEART HORMONES TURN ON A PROCESS IN FAT TISSUE SIMILAR TO WHAT IS ACTIVATED WHEN THE BODY IS EXPOSED TO COLD, LEADING TO THE BURNING OF FAT TO GENERATE HEAT. THIS STUDY ADDS ANOTHER DIMENSION TO OUR UNDERSTANDING OF HOW THE BODY REGULATES FAT TISSUE AND COULD LEAD TO NEW WAYS TO REDUCE WEIGHT IN OBESE PATIENTS. THIS STUDY WAS NAMED "RECOMMENDED READING" BY THE FACULTY OF 1000 (F1000), A PRESTIGIOUS GROUP OF SCIENTISTS. UNRAVELING THE MACHINERY OF TISSUE GLUCOSE UPTAKE. ALL CELLS NEED GLUCOSE TO PRODUCE THE ENERGY THEY NEED TO SURVIVE. A STUDY LED BY DR. ZHEN JIANG IDENTIFIED A PROTEIN-CALLED CDP138-WHICH IS RESPONSIBLE FOR ENSURING THAT GLUT4, A GLUCOSE TRANSPORTER RESPONSIBLE FOR GLUCOSE UPTAKE FROM THE BLOODSTREAM, IS PROPERLY INSERTED IN THE CELLULAR MEMBRANE. THESE RESULTS, PUBLISHED IN THE JOURNAL "CELL METABOLISM," PROVIDE A NEW UNDERSTANDING OF GLUCOSE METABOLISM DURING NORMAL PROCESSES SUCH AS EXERCISE, AS WELL AS PATHOLOGICAL PROCESSES SUCH AS DIABETES. ALL WEIGHT GAIN IS NOT THE SAME. A STUDY BY DR. STEVEN SMITH AND COLLEAGUES PROVIDES NEW NUTRITIONAL INFORMATION THAT COULD SHAPE THE DEVELOPMENT OF PUBLIC HEALTH POLICY RECOMMENDATIONS. THE STUDY, PUBLISHED IN THE "JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION" (JAMA), EXAMINED THE IMPACT OF DIETS CONTAINING VARYING AMOUNTS OF PROTEIN ON WEIGHT GAIN, BODY COMPOSITION, AND ENERGY EXPENDITURE. THE RESULTS OF THE STUDY REVEALED THAT INCREASING THE PERCENT OF DIETARY PROTEIN WITH OVERFEEDING LED TO MORE LEAN BODY MASS RATHER THAN SOLELY EXPANDING FAT MASS. THIS WORK IS THE FIRST TO ANALYZE THE IMPACT OF DIETARY PROTEIN ON CALORIE EXCESS AND PROVIDES NEW GUIDANCE FOR DIETARY COMPOSITION RECOMMENDATIONS. CAN BAD FAT BECOME GOOD FAT? IN A STUDY PUBLISHED IN CELL METABOLISM, DR. DEV SIKDER'S GROUP DISCOVERED THAT OREXIN, A HORMONE PRODUCED IN THE BRAIN, ACTIVATES CALORIE-BURNING BROWN FAT IN MICE. INTERESTINGLY, OREXIN DEFICIENCY IS ASSOCIATED WITH OBESITY IN HUMANS, SUGGESTING THAT OREXIN SUPPLEMENTATION COULD PROVE EFFECTIVE AS A NEW THERAPEUTIC APPROACH FOR THE TREATMENT OF OBESITY. GIVEN THAT MOST CURRENT WEIGHT LOSS DRUGS ARE AIMED AT REDUCING A PERSON'S APPETITE, AN OREXIN-BASED THERAPY WOULD REPRESENT A NEW CLASS OF FAT-FIGHTING DRUGS THAT INCREASE ENERGY EXPENDITURE. A LINK BETWEEN REGULATION OF PROTEIN SYNTHESIS AND DIABETES. IN THE PRESTIGIOUS "NEW ENGLAND JOURNAL OF MEDICINE," DR. RANDAL KAUFMAN DESCRIBED NEW FINDINGS THAT DEMONSTRATE HOW ALTERATIONS IN PROTEIN SYNTHESIS IN THE PANCREAS' INSULIN-PRODUCING BETA CELLS CAN CAUSE DIABETES. THE INSIGHTS MAY LEAD TO NEW THERAPEUTIC STRATEGIES TO TREAT DIABETES. IN THE AREA OF CHILDREN'S HEALTH RESEARCH: EXCITING CLINICAL TRIAL NEWS FOR CHILDREN WITH INHERITED BONE DISEASE. FOR THE PAST 15 YEARS, DR. JOS LUIS MILLN AND COLLEAGUES HAVE STUDIED HYPOPHOSPHATASIA, AN INHERITED DISEASE THAT MAKES BONES DANGEROUSLY FRAGILE. BUOYED BY THE INITIAL RESULTS TESTED IN A MOUSE MODEL, ENOBIA PHARMA RESEARCHERS AND COLLABORATORS BEGAN TESTING ENB-0040 IN YOUNG PATIENTS WITH INFANTILE HYPOPHOSPHATASIA-THE MOST SEVERE FORM OF THE DISEASE. NOW, A PAPER PUBLISHED IN THE "NEW ENGLAND JOURNAL OF MEDICINE" REVEALS THE RESULTS OF THIS CLINICAL TRIAL WHICH HAS BEEN LIFE-CHANGING FOR SOME OF THE STUDY'S PARTICIPANTS. THREE YEAR-OLD CORINNA, FOR EXAMPLE, COULD NOT WALK WHEN SHE BEGAN TAKING ENB-0040. JUST EIGHT WEEKS LATER, SHE WAS RUNNING AND JUMPING. RARE BONE DISORDER REVEALS NEW INSIGHTS INTO AUTISM. CHILDREN WITH MULTIPLE HEREDITARY EXOSTOSES (MHE), AN INHERITED GENETIC DISEASE, SUFFER FROM MULTIPLE GROWTHS ON THEIR BONES THAT CAUSE PAIN AND DISFIGUREMENT. SOME PARENTS HAVE LONG OBSERVED THAT THEIR CHILDREN WITH MHE ALSO EXPERIENCE AUTISM-LIKE SOCIAL PROBLEMS. RESEARCHERS LED BY DR. YU YAMAGUCHI RECENTLY USED A MOUSE MODEL OF MHE TO INVESTIGATE COGNITIVE FUNCTION. THEY FOUND THAT MICE WITH A GENETIC DEFECT THAT MODELS HUMAN MHE SHOW SYMPTOMS THAT MEET THE THREE DEFINING CHARACTERISTICS OF AUTISM: SOCIAL IMPAIRMENT, LANGUAGE DEFICITS, AND REPETITIVE BEHAVIOR. THE STUDY, PUBLISHED IN THE "PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES," ALSO DEFINES THE MOLECULAR AND PHYSIOLOGICAL BASIS OF THIS BEHAVIOR, PINPOINTING THE AMYGDALA AS THE REGION OF THE BRAIN CAUSING AUTISTIC SYMPTOMS. IN THE AREA OF INFECTIOUS AND INFLAMMATORY DISEASES: LIGHTENING THE BURDEN OF LUNG FIBROSIS. PEOPLE WITH PERSISTENT INFLAMMATION IN THE LUNGS OFTEN DEVELOP DEBILITATING SCAR TISSUE THAT MAKES BREATHING DIFFICULT. DR. CARL WARE AND COLLABORATORS RECENTLY REVEALED A KEY ROLE FOR CYTOKINES-SIGNALING MOLECULES THAT MEDIATE INFLAMMATION-IN ASTHMA AND RELATED LUNG DISORDERS. THE RESEARCH DEMONSTRATES THAT NEUTRALIZING ONE PARTICULAR CYTOKINE CALLED LIGHT, WHICH WAS FIRST DISCOVERED BY DR. WARE, BLOCKS THE FORMATION OF LUNG SCARRING IN MICE. THESE RESULTS, PUBLISHED IN "NATURE MEDICINE," IDENTIFY CONDITIONS THAT MIGHT BE ALLEVIATED BY DRUGS THAT BLOCK LIGHT. NEW DRUGS FOR BRAIN INFLAMMATION. DRUG SCREENING EFFORTS BY DRS. FRAYDOON RASTINEJAD, PENGXIANG HUANG, AND COLLABORATORS, IDENTIFIED SMALL MOLECULES THAT ALTER A CRUCIAL PROTEIN CONTROLLING IMMUNE CELL GENES THAT CAUSE BRAIN INFLAMMATION. THE RESEARCHERS SHOWED THAT THE MOLECULE-CALLED CARDIAC GLYCOSIDE DIGOXIN-SPECIFICALLY INHIBITS A GENE REGULATOR CALLED RORYT. DIGOXIN, AND RELATED COMPOUNDS THAT ARE LESS TOXIC, DELAYED THE ONSET OF AND REDUCED INFLAMMATION IN THE BRAINS OF MICE. REPURPOSING DIGOXIN TO INHIBIT INFLAMMATION PROVIDES A NEW APPROACH TO TREATING AUTOIMMUNE DISEASES. THE STUDY WAS PUBLISHED IN "NATURE." HOW THE BODY FENDS OFF INFECTION. TO INVADE ORGANISMS SUCH AS HUMANS, BACTERIA MAKE USE OF A PROTEIN CALLED FLAGELLIN, PART OF A TAIL-LIKE APPENDAGE THAT HELPS THE BACTERIA MOVE ABOUT. NOW, FOR THE FIRST TIME, DR. ANDREI OSTERMAN AND COLLEAGUES DETERMINED THE 3D STRUCTURE OF THE INTERACTION BETWEEN THIS CRITICAL BACTERIAL PROTEIN AND AN IMMUNE MOLECULE CALLED TLR5, SHEDDING LIGHT ON HOW THE BODY PROTECTS ITSELF FROM SUCH FOREIGN INVADERS. THE STUDY, PUBLISHED IN "SCIENCE" ADVANCES KNOWLEDGE THAT'S KEY TO THE DESIGN OF NEW THERAPEUTICS. FLAGELLIN IS A COMPONENT IN SOME VACCINES AND A DERIVATIVE OF THIS PROTEIN IS CURRENTLY BEING DEVELOPED AS A MEDICAL COUNTERMEASURE TO RADIATION. TURNING THE FIGHT AGAINST BACTERIA INTO A FIGHT AGAINST RHEUMATOID ARTHRITIS. THE COMPLEMENT SYSTEM IS ONE OF THE IMMUNE SYSTEM'S MOST IMPORTANT WEAPONS FOR FIGHTING OFF INFECTION. COMPLEMENT CONSISTS OF NINE PROTEINS (C1 THROUGH C9) THAT DIRECTLY BIND BACTERIA AND PUNCH HOLES (PORES) IN THEIR MEMBRANES. USING X-RAY CRYSTALLOGRAPHY, RESEARCHERS LED BY DR. ROBERT LIDDINGTON SOLVED THE CRYSTAL STRUCTURE OF ONE CRITICAL PROTEIN C6 AND ANALYZED OTHER STRUCTURES, PUBLISHING THEIR FINDINGS IN THE "JOURNAL OF BIOLOGICAL CHEMISTRY." THIS INFORMATION COULD LEAD TO NOVEL THERAPIES FOR A VARIETY OF INFLAMMATORY DISEASES SUCH AS RHEUMATOID ARTHRITIS. | |
| PROCESS USED BY MANAGEMENT AND/OR GOVERNING BODY TO REVIEW FORM 990 | FORM 990, PART VI, LINE 11B | THE 990 IS PREPARED BY THE FINANCE DEPARTMENT. IT IS REVIEWED BY THE INSTITUTE'S TAX ADVISORS, SELECTED MEMBERS OF MANAGEMENT INCLUDING OFFICERS, AND THE AUDIT COMMITTEE OF THE BOARD OF TRUSTEES AND PROVIDED TO MEMBERS OF THE BOARD PRIOR TO FILING. |
| DESCRIPTION OF PROCESS TO MONITOR TRANSACTIONS FOR CONFLICT OF INTEREST | FORM 990, PART VI, LINE 12C | CONFLICT OF INTEREST STATEMENTS ARE GIVEN TO MANAGEMENT STAFF AND PRINCIPAL INVESTIGATORS ON A PERIODIC BASIS TO REPORT ON CONFLICTS OR LACK OF CONFLICTS. THE STATEMENTS ARE REVIEWED ANNUALLY BY THE SENIOR DIRECTOR, INTELLECTUAL PROPERTY AND THE SENIOR DIRECTOR, SPONSORED RESEARCH, WITH A SUMMARY OF THE REVIEW PROVIDED TO THE EXECUTIVE VICE PRESIDENT/CAO/CFO/TREASURER. AN ASSESSMENT IS MADE TO WHETHER A CONFLICT CAN BE MANAGED BY THE EXISTING DEPARTMENTS AND PRACTICES. CONFLICTS ARE BROUGHT TO THE BOARD OF TRUSTEES ON AN AS NEEDED BASIS. CONFLICTS AND POTENTIAL CONFLICTS THAT INVOLVE THE CEO OR THE PRESIDENT ARE BROUGHT TO THE BOARD OF TRUSTEES TO ADDRESS ANY ACTUAL OR POTENTIAL POSSIBILITY THAT THE DESIGNATED REVIEWERS (i.e., SENIOR DIRECTOR, INTELLECTUAL PROPERTY AND THE SENIOR DIRECTOR, SPONSORED RESEARCH, AND THE EXECUTIVE VICE PRESIDENT/CAO/CFO/TREASURER) COULD BE PRESSURED TO MAKE BIASED DECISIONS IN MANAGING CONFLICTS INVOLVING THE CEO OR PRESIDENT. AS TO RESTRICTIONS IMPOSED: THE INSTITUTE WILL EMPLOY THE MECHANISM THAT IS THE BEST FIT WITH THE FACTS AND CIRCUMSTANCES OF ANY IDENTIFIED CONFLICT OF INTEREST. METHODS TO MANAGE POTENTIAL CONFLICTS/ENFORECEMENT MECHANISM: REVIEWING MANAGEMENT AND THE TECHNOLOGY TRANSFER COMMITTEE OF THE BOARD OF TRUSTEES ARE RESPONSIBLE FOR MANAGING, REDUCING, OR ELIMINATING REAL OR POTENTIAL CONFLICTS OF INTEREST. EXAMPLES OF CONDTIONS OR RESTRICTIONS THAT MIGHT BE IMPOSED TO MANAGE, REDUCE, OR ELIMINATE CONFLICTS OF INTERESTS INCLUDE: -PUBLIC DISCLOSURE OF SIGNIFICANT FINANCIAL INTERESTS -MONITORING OF RESEARCH BY INDEPENDENT REVIEWERS -MODIFICATION OF THE RESEARCH PLAN -DISQUALIFICATION FROM PARTICIPATION IN THE PORTION OF FUNDED RESEARCH THAT COULD BE AFFECTED BY SIGNIFICANT FINANCIAL INTEREST -DIVESTITURE OF SIGNIFICANT FINANCIAL INTERESTS -SEVERENCE OF RELATIONSHIPS THAT CREATE ACTUAL OR POTENTIAL CONFLICTS -REVIEW OF COMMERCIAL SPONSORED RESEARCH AGREEMENTS FOR SCIENTIFIC MERIT BY THE PROGRAM PLANNING COMMITTEE -REMOVAL OF AN AFFECTED PARTY FROM NEGOTIATIONS WITH A COMPANY WITH HE/SHE HAS SIGNIFICANT FINANCIAL INTEREST. |
| PROCESS USED TO DETERMINE COMPENSATION | FORM 990, PART VI, LINES 15A AND 15B | ANNUALLY, THE INSTITUTE REQUIRES BOARD OF TRUSTEE APPROVAL OF COMPENSATION ADJUSTMENTS AND TOTAL COMPENSATION LEVELS FOR ALL OFFICERS AND OTHER "DISQUALIFIED PERSONS" (AS DEFINED BY IRC SECTION 4958). THE COMPENSATION COMMITTEE, A COMMITTEE DESIGNATED BY THE BOARD, IS RESPONSIBLE FOR THE PROCESS AND ENGAGING AN INDEPENDENT CONSULTING FIRM TO ASSIST IT IN REVIEWING THE TOTAL COMPENSATION PACKAGES (INCLUDING CASH AND NON-CASH BENEFITS) FOR THESE IDENTIFIED PERSONS. THE CONSULTANT REVIEWS THE VARIOUS TYPES OF DIRECT CASH COMPENSATION INCLUDING BASE SALARIES AND ANNUAL INCENTIVE AWARD OPPORTUNITIES AND PAYMENTS. IN ADDITION, THE CONSULTANT PROVIDES THE COMMITTEE AN ASSESSMENT OF TOTAL COMPENSATION INCLUDING THE CASH COMPONENTS MENTIONED ABOVE, PLUS EXECUTIVE BENEFITS AND PERQUISITES. THE CONSULTANT COLLECTS INFORMATION FROM VARIOUS SOURCES COVERING NOT-FOR-PROFIT AND FOR-PROFIT ORGANIZATIONS SIMILAR IN SIZE AND REFLECTING THE TALENT MARKETS FROM WHICH THE INSTITUTE DRAWS EXECUTIVE TALENT. THE NUMBER OF FOR-PROFIT ORGANIZATIONS USED IN THE SURVEY PROCESS DOES NOT EXCEED 50% OF THE TOTAL ORGANIZATION USED. THE COMPENSATION COMMITTEE UTILIZES THIS STUDY ALONG WITH PERFORMANCE DATA FOR EACH INDIVIDUAL TO SUPPORT THEIR DELIBERATIONS AND REVIEW OF PAY LEVELS IN RELATION TO THE EXECUTIVE COMPENSATION PAY PHILOSOPHY AND THE ORGANIZATION'S MISSION. THE DETERMINATION OF EXECUTIVE COMPENSATION IS ALSO DESIGNED TO MEET THE "REBUTTABLE PRESUMPTION OF REASONABLENESS" STANDARD AS OUTLINED IN THE TREASURY REGULATIONS. DECISIONS BY THE COMMITTEE ARE CONTEMPORANEOUSLY DOCUMENTED. THE COMMITTEE PRESENTS THE STUDY FINDINGS AND THE RESULTING DECISIONS TO THE ENTIRE BOARD OF TRUSTEES. THE INSTITUTE'S EXECUTIVE COMPENSATION PHILOSOPHY, WHICH IS REVIEWED ANNUALLY, IS AS FOLLOWS: TARGET EXECUTIVE BASE SALARY AND TOTAL CASH COMPENSATION IS BETWEEN THE 50TH AND 75TH PERCENTILES BY BLENDING DATA FROM SIMILARLY SIZED BIOTECHNOLOGY ORGANIZATIONS FROM THE NOT-FOR-PROFIT AND FOR-PROFIT SECTOR, RESEARCH INSTITUTES AND LARGE UNIVERSITIES. THE PROCESS WAS LAST COMPLETED ON JUNE 8, 2012 FOR THE FOLLOWING 8 POSITIONS: 1) CHIEF EXECUTIVE OFFICER; 2) PRESIDENT/CANCER CENTER DIRECTOR; 3) EXECUTIVE VICE PRESIDENT, CHIEF ADMINISTRATIVE OFFICER, CHIEF FINANCIAL OFFICER, AND TREASURER; 4) SCIENTIFIC DIRECTOR, SBMRI AT LAKE NONA; 5) CENTER DIRECTOR, NEUROSCIENCE AGING AND STEM CELL RESEARCH CENTER; 6) CENTER DIRECTOR, INFECTIOUS AND INFLAMMATORY DISEASE CENTER; 7) SENIOR VICE PRESIDENT, BUSINESS DEVELOPMENT; 8) SENIOR DIRECTOR OF INTELLECTUAL PROPERTY AND LEGAL AFFAIRS, AND SECRETARY OF THE BOARD |
| AVAIL OF GOV DOCS, CONFLICT OF INTEREST POLICY & FIN STMTS TO GEN PUBLIC | FORM 990, PART VI, LINE 19 | DOCUMENTS ARE AVAILABLE UPON REQUEST. |
| OTHER CHANGES IN NET ASSETS OR FUND BALANCES | FORM 990, PART XI, LINE 5 | NET UNREALIZED GAIN (LOSS) ON INVESTMENTS: $(1,212,803) UNREALIZED GAIN (LOSS) ON INTEREST RATE SWAP: $(1,773,062) ROUNDING: $ 3 ------------ $(2,985,862) |
| SCHEDULE K, PART I, ROW A, COLUMN (F) | REFINANCE 1999 BONDS AND FINANCE CAPITAL IMPROVEMENTS & EQUIPMENT. | |
| SCHEDULE K, PART I, ROWS A-C | 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, 2011. | |
| SCHEDULE K, PART III, LINE 4, COLUMN (C) | THE PERCENTAGE OF FINANCED PROPERTY USED IN A PRIVATE BUSINESS FOR THE ANNUAL MEASUREMENT PERIOD ENDING 12/31/2011 IS 5.54%. THE PRIVATE BUSINESS USE PERCENTAGE OVER THE LIFE OF THE BONDS THROUGH 12/31/2011 IS 3.27%. THE PROJECTED PRIVATE BUSINESS USE PERCENTAGE OVER THE LIFE OF THE BONDS IS 4.35%. |
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