Scientist examines the result of a plaque assay.

Innovation in the stem cell field

Pri­or to 2005, the stem cell field was vir­tual­ly nonexis­tent. Stem cells had recent­ly been dis­co­ve­r­ed as a power­ful tool for under­stan­ding dise­a­se and deve­lo­ping new tre­at­ments, but the field lacked the fun­ding and talent nee­ded to capi­ta­li­ze on this tech­no­lo­gy. Becau­se of this, Sus­an L. Solo­mon laun­ched The New York Stem Cell Foun­da­ti­on with the mis­si­on of using stem cell rese­arch to acce­le­ra­te tre­at­ments and cures for the major dise­a­ses of our time. This cata­ly­zed the growth of the field, which now inclu­des thousands of sci­en­tists world­wi­de con­duc­ting ground­brea­king rese­arch across many diseases.

Clinical Impact

NYSCF’s pri­ma­ry goal has always been to acce­le­ra­te new tre­at­ments and cures for the major dise­a­ses of our time, and we have seen many exci­ting the­ra­pies made pos­si­ble by NYSCF moving into the cli­nic, inclu­ding 21 cur­r­ent­ly in cli­ni­cal tri­als. The­se suc­ces­ses demons­tra­te the power of stem cells for brin­ging about inven­ti­ve new tre­at­ments, and we look for­ward to see­ing many more move into the cli­nic soon.

Mitochondrial Replacement Therapy

A team of sci­en­tists from NYSCF and Colum­bia Uni­ver­si­ty Medi­cal Cen­ter deve­lo­ped a tech­ni­que cal­led mito­chon­dri­al repla­ce­ment the­ra­py that pre­vents the inheri­tance of devas­ta­ting mito­chon­dri­al dise­a­ses in child­ren. With this tech­ni­que, publis­hed in Natu­re, mito­chon­dria car­ry­ing dise­a­se-causing muta­ti­ons in a mother’s egg cell can effec­tively be repla­ced by healt­hy mito­chon­dria, pre­ven­ting her off­spring from inheri­ting fatal mito­chon­dri­al dise­a­ses. Mito­chon­dri­al repla­ce­ment the­ra­py is cur­r­ent­ly used in the United King­dom, whe­re it is pre­ven­ting child­ren from inheri­ting poten­ti­al­ly fatal diseases.

A Cell Therapy for Macular Degeneration

Our 20/20 cli­ni­cal tri­al is a cell the­ra­py that will pro­vi­de a las­ting cure for age rela­ted macu­lar dege­ne­ra­ti­on (AMD). This pionee­ring stem cell the­ra­py will use a patient’s stem cells to crea­te healt­hy reti­nal cells that then replace tho­se lost to AMD and res­to­re visi­on. We are lea­ding this effort and working in col­la­bo­ra­ti­on with world-renow­ned reti­nal sur­ge­ons inclu­ding Dr. Stan­ley Chang at Colum­bia Uni­ver­si­ty and stem cell sci­en­tists inclu­ding Dr. Kapil Bhar­ti at the Natio­nal Eye Insti­tu­te at the Natio­nal Insti­tu­tes of Health. This builds on pionee­ring work by NYSCF – Robert­son Stem Cell Pri­ze reci­pi­ent Dr. Pete Cof­fey, who­se cli­ni­cal tri­al of a stem-cell-based AMD the­ra­py has impro­ved visi­on in two AMD pati­ents. Con­struc­tion is now com­ple­te at the NYSCF Rese­arch Insti­tu­te for a sta­te-of-the-art “good manu­fac­tu­ring prac­ti­ce” (GMP) faci­li­ty in which we can crea­te cells that are safe for trans­plan­ta­ti­on into pati­ents.

Discovering a New ALS Drug

An epi­le­psy drug, initi­al­ly tes­ted using a stem cell model of amyo­tro­phic late­ral scle­ro­sis (ALS) deve­lo­ped with NYSCF sup­port (which was named Time and Sci­ence Maga­zi­ne’s #1 medi­cal bre­akthrough of the year), has suc­cess­ful­ly redu­ced motor neu­ron exci­ta­bi­li­ty – a hall­mark of the dise­a­se – in 65 ALS pati­ents par­ti­ci­pa­ting in a pha­se 2 cli­ni­cal tri­al. This work, led by NYSCF Sci­en­ti­fic Advi­sor Dr. Kevin Eggan and NYSCF – Robert­son Stem Cell Inves­ti­ga­tor Dr. Bri­an Wain­ger, is the first cli­ni­cal tri­al to result from a drug dis­co­ve­r­ed in a stem cell model, set­ting a pre­ce­dent for count­less future discoveries.

Clinical Trials for Cancer and Parkinson’s Disease

NYSCF – Robert­son Stem Cell Inves­ti­ga­tor Alum­nus Dr. Ravi Majeti of Stan­ford Uni­ver­si­ty, is con­duc­ting cli­ni­cal tri­als to test a class of can­cer the­ra­pies cal­led anti-CD47 tre­at­ments. The­se drugs strip away camou­flaging agents in leukemia-affec­ted cells, hel­ping the immu­ne sys­tem tar­get them more effectively.

Addi­tio­nal­ly, NYSCF – Robert­son Stem Cell Inves­ti­ga­tor Alum­na Dr. Malin Par­mar of Lund Uni­ver­si­ty has spent several years working with the phar­maceu­ti­cal com­pa­ny Novo Nor­disk on a cell the­ra­py for Parkinson’s dise­a­se that aims to replace lost brain cells with healt­hy ones deri­ved from a patient’s own stem cells.

Disease Research

The back­bone of our mis­si­on lies with using stem cells to advan­ce dise­a­se rese­arch, and our sci­en­tists have made many important dis­co­ve­ries that are paving the way for brigh­ter futures for pati­ents everywhere.

The First Personalized Embryonic Stem Cell Model of Disease

We crea­ted the first-ever dise­a­se-spe­ci­fic stem cell line using soma­tic cell nuclear trans­fer (SCNT). Our sci­en­tists used SCNT to crea­te embryo­nic stem cells from a pati­ent with type 1 dia­be­tes. This work mar­ked a major step for­ward in the effort to use stem cells to under­stand the deve­lo­p­ment of, and even­tual­ly tre­at, a ran­ge of chro­nic dise­a­ses affec­ting tens of mil­li­ons of people.

Creating All Cell Types in the Brain from Stem Cells

To under­stand brain dise­a­ses such as Alzheimer’s, Parkinson’s, and mul­ti­ple scle­ro­sis, it is important that sci­en­tists can exami­ne all cell types in the brain. NYSCF’s Dr. Valen­ti­na Fos­sa­ti has pionee­red methods for crea­ting a cate­go­ry of brain cells cal­led glia – sup­port cells in the brain that likely play signi­fi­cant roles in dise­a­se but are often over­loo­ked in rese­arch and drug deve­lo­p­ment. Dr. Fossati’s pro­to­cols for making glia (such as astro­cytes, oli­go­dend­ro­cytes, and micro­glia) are now used by sci­en­tists around the world, acce­le­ra­ting rese­arch into how inflamma­ti­on and the immu­ne sys­tem dri­ve neu­ro­lo­gi­cal dise­a­ses and ope­ning the door for ent­i­re­ly new clas­ses of therapies.

Personalized Bone Grafts from Stem Cells

In a step towards per­so­na­li­zed bone grafts to tre­at trau­ma­tic inju­ry or con­ge­ni­tal defects, NYSCF sci­en­tists led by Dr. Giu­sep­pe Maria de Pep­po dis­co­ve­r­ed in 2013 how to crea­te pati­ent-spe­ci­fic bone sub­sti­tu­tes from skin cells for repair of lar­ge bone defects. This advan­ce faci­li­ta­tes the deve­lo­p­ment of cus­to­miz­ab­le, three-dimen­sio­nal bone grafts on-demand, matched to fit the exact needs and immu­ne pro­fi­le of pati­ents who­se bodies may other­wi­se reject syn­the­tic grafts or requi­re fre­quent sur­ge­ries to get them replaced.

Disease Modeling and Drug Testing

NYSCF sci­en­tists have estab­lis­hed methods for advan­ced cell mani­pu­la­ti­on, allowing us to crea­te models of dise­a­ses that affect various human tis­su­es, inclu­ding the lungs, pan­cre­as, bone, and more. Becau­se of this, we can address a varie­ty of dise­a­ses inclu­ding rare and recent­ly emer­ging dise­a­ses like COVID-19, for which our rese­ar­chers are crea­ting lung cells to stu­dy infec­tion. The cells NYSCF crea­tes can also ser­ve as mate­ri­al for drug tes­ting and toxi­ci­ty eva­lua­tions, ensu­ring that the safest, most effec­ti­ve drugs are advan­ced toward the clinic.

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