MEDICALBREAKTHROUGHS

Every year, wewade through reamsof medical research, attend physician confer-

ences, pick scientists’ brains, take doctors to lunch and even scrub up for the OR—all

to find the most amazing discoveries, devices, tests and potential cures out there.

In the past, we watched doctors repair a heart with tiny tools inserted through three

small incisions, with no chest cracking. We witnessed a big blood clot being pulled out of

the brain of a stroke victim almost like a cork from a bottle of wine, followed by a com-

plete recovery. And we saw a paralyzed man draw pictures on his computer, change the

channels on his TV and open his curtains—just by using his thoughts. These advances

seemed mind-boggling at the time, but they’re becoming state-of-the-art treatments.

This year brings more excitement. From skin cells transformed into tissue beating

like a human heart to a simple checklist that’s saving thousands of lives, it was another

banner year for breakthroughs. Some are available now; some need more work before

they’re ready for prime time. But all will make you say “wow.”

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New hope forcancer, diabetes,dementia and more

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’08

A new look atthe brain

Better breast exams

An art if ic ia l lung

“Younger” vaccines

24 /7 bloodsugar monitor

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BREAKTHROUGHS ’08

• In Germany,researchers have devel-oped a new technique tosee theentire neuralnetwork of amousebrain in 3-D for the firsttime—without having toslice it apart with a scalpeland reconstruct it on acomputer. The method,which uses fluorescentmolecules and lasers, mayhelp shed light on how welldrugs work against degen-erative nerve diseasessuch as Alzheimer’s. Itmay also provide usefulinformation about howhuman brains changeover time and in response

to disease. Now• With the world’s most

powerful magnetic reso-nance imaging (MRI)machine, doctors maysoon be able to tell in days,instead of weeks,howwell a cancer treatmentisworking. The device,called the 9.4 Tesla anddeveloped at the Univer-sity of Illinois at Chicago,can show whether cellswithin a brain tumor aredying long before thetumor itself begins toshrink. 3-5 years

Neena Samuel

SEEING INTOTHE BRAINMEMORYKEEPERSSome 1,200Americans

develop Alzheimer’s everyday. Dozens of drugsto fight the devastatingeffects are in clinicaltrials. Two look especiallypromising:

•PRX-03140Designedto treat memory loss andother symptoms, this drugfrom EPIX Pharmaceuti-cals changed the brainwave activity in trial pa-tients. In just two weeks,they scored better onmemory tests and weremore engaged. One waseven able to speak in fullsentences again. 5 years

•FlurizanThis one,now finishing clinical trials,may attack the underlyingcauses of the disease.From Myriad Genetics,it reduces proteins thatform plaque buildup be-tween brain cells, possiblyslowingor even stoppingfurther damage. 1 year

Researchers expectfuture treatments to bea combination of drugsthat attack the disease atdifferent stages, much likethe current approach tofighting AIDS and cancer.

Bridget Nelson Monroe

AMANDASIERRASAAVEDRA,STONYBROOKUNIVERSITY

The birth ofbrain cel ls

•Believe it or not, youmake new brain cells allthe time. It’s called neuro-genesis, and for the firsttime, scientists havewatched a living humanbrain growing thesecells.The discovery wasmade by researchers atStony Brook University

Medical Center, Brook-haven National Laboratoryand Cold Spring HarborLaboratory using high-tech magnetic resonancespectroscopy. Watchingthe process could oneday help diagnose andtreat disorders like Par-kinson’s disease, multiplesclerosis and depression,in which neurogenesis isdisrupted. Now

NANOTUMORKILLERSSoon theremay be a treatment for liver cancer

thatworks from inside the tumorwithoutdamaging neighboring tissue, thanks to tinynanotubes. In early experiments with rabbits,scientists at the University of Texas and Rice

University implanted the carbon tubes into livertumors. (They’re so small that 75,000 of them lined

up side by side would be about the width of a humanhair.) Then the rabbits were exposed to radio waves.

The nanotubes produced heat, which ultimately destroyed the tumor. The researchers hopethis method has potential to treat other types of cancer too. 5+ years Kathryn M. Tyranski

FINDINGOVARIANCANCER EARLIERThe symptomsof ovar-

ian cancer can be vague,and there’s no early screen-ing test. Some 15,000women die from it eachyear, and the survival ratehasn’t changed in 30 years.But a simple urine test

now in development couldchange that. Researchersat the University of SouthFlorida and GeoPharmafound that the level of aprotein in urine calledBcl-2 is ten times higher inwomen with ovarian can-

cer than it is in healthywomen. Plus, levels in-creased with the growth ofcancer and declined aftertumors were removed.3-5 years Nancy Coveney

HEART-SAVINGGADGETSWhen it comes to the heart, two devices could

mean thedifference between life and death:• The CPRGlove by Atreo Medical talks you through

giving CPR, telling you how hard and fast to compressthe chest and when to give breaths. It even remindsyou to call 911. 3-4 years

• The Chronicle IHM (Implantable Hemodynamic Monitor)stays on alert 24/7 for patients who suffer from congestive heartfailure or other chronic cardiac conditions. If your heart getsinto trouble—from, say, increased fluid accumulation—the IHM will send a message wirelessly via the Internet to yourdoctor, who can adjust your treatment accordingly. 5+ years

Kathryn M. Tyranski

Nanotubesand heat canzap cancer cel ls .

The CPRGlove

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24/7BLOODSUGAR TESTDiabeticsmay be able

to say buh-bye to finger-pricking when checking

blood sugar. New monitors,like the one being developed by

Gerard Cote at Texas A&M, requireno blood and offer more certainty. In Cote’s model,

a sheath of tiny fluorescent particles, smaller than a strandof human hair, is inserted into a diabetic patient’s wrist. The sheath is

invisible, but when you shine a small laser on it, it glows and changes colors inresponse to deviations in blood sugar. A wristwatch-like device provides a digital

readout of glucose levels and alerts the person to dangerous dips or spikes. Houston-based BioTex is also developing a model using similar technology. 5 years Tara Conry

YOUNGERVACCINES•FluMistRecently approved for the ages 2 to 5 crowd,

the nasal spray vaccine takes the sting out of flu preven-tion. That’s a good thing, as 226,000 people are hospital-ized each year, and 36,000 die. Now

•MenactraThough rare, bacterial meningitis can bedeadly. The FDA recently expanded the age range for thisvaccine to include children 2 to 10 (original approval in 2005was only for people 11 to 55). Now Deirdre Casper

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TARGETED ASSAULT ONMELANOMAThe skin cancer

melanoma is treatablewhen caught early butoften deadly if it hasspread. New hopes:

• Doctors at the Na-tional Cancer Institute re-move cancer-fighting cellsfrom late-stage patients,

grow more and then givethem back. So far, this im-munotherapy has shown itcan shrink tumors in about75% of the patients. Now

• The myeloma drugVelcade seems to put skincancer cells in overdrive sothey self-destruct, accord-

ing to docs at the Univer-sity of Michigan. Evenbetter, itkills only thecancerous cells.Next up:seeing how actual patientsreact to Velcade, as thisresearch was done only ina petri dish. 5+ years

Patricia Curtis

The watch“reads” animplant andshows glucoselevels .

COURTESYSANOFLIPASTEUR

Meningit isprotect ionfor kids

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>>BREAKTHROUGHOF THE YEAR

STEM CELL STUNNERIn November,Univer-

sity of Wisconsin biologistJames Thomson, PhD, andhis research team amazedthe world by creating cellswith the same chameleon-like characteristics asembryonic stem cellsbutderived fromhumanskin.With this discovery,achieved simultaneouslyby Japanese researchers,Thomson may have short-

circuited the very debatehe helped trigger. Yes,Thomson was the firstto extract stem cells fromhuman embryos back in1998, unwittingly kickingoff the heated controversythat has bedeviled scien-tists, politicians and theAmerican people for adecade.

The new inducedpluripotent stem (iPS)cells, as they’re dubbed,

were created simply byidentifying four genesthat, when inserted intoskin cells, reprogrammedthe DNA. Then the iPScells began reproducingsteadily, which means thatscientists should be ableto produce an unlimitedsupply. The first task isto create cells carryingdiseases to learn how toprevent and cure them.

The ultimate goal is to usethe cells to heal diseasedand damaged organs.

The Japanese team,led by Shinya Yamanaka,demonstrated that with alittle coaxing, the iPS cells,like embryonic stem cells,could transform them-selves into different typesof human tissue. First theymanipulated a cluster tocreate nerve cells. Thenthey tried for heart cells.

Just 12 days after mixinga sample of iPS cells witha cocktail of proteins in apetri dish, the scientistswatched as clumps ofnewly formed cardiaccells started beating likea human heart (see anamazing video of it atrd.com). The iPS cellseven have an advantageover embryonic: Sincethey’re derived from the

patient’s own cells, there’sno risk of rejection.

Amid the heady opti-mism unleashed by theirdiscoveries, Thomson andYamanaka caution thatmore work needs to bedone before iPS cells canbe put to widespread use.Until then, research onembryonic stem cells, andthe controversy, will mostlikely continue. 5+ years

Joseph K. Vetter

(1) A layerof skin cel lsfrom a36-year-oldwoman;(2) twocolonies ofiPS cel lsderived from the skin cel ls ; (3) a magnif ied view showing individual iPS cel ls .

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is cancer (harmless cystsand calcifications areusually soft). Elastogramson 635 women predictedcancer 98.6% of the time,helping to avoid biopsies.That’s better than mam-mograms or MRIs, sayslead study author RichardG. Barr, a radiologist atNortheastern Ohio Uni-versities Colleges of Medi-cine and Pharmacy. Now

• A combination oflow-radiation CT scanand nuclear molecu-lar imaging (the kindused to look atblood flow in theheart) can provide

3-D images of thebreast without

compression andfindlesions half the size ofthose typically foundinmammograms.Thepatient lies facedownwith her breasts througha cutout on the exam table,and the hybrid machinecircles underneath. MartinTornai, PhD, of Duke Uni-versity, says this technol-ogy could also be used tomonitor how well chemodrugs are shrinking atumor. 5 years

Cynthia Dermody

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BETTER THANMAMMOCanadian andU.S. sci-

entists are excited aboutearly results of an experi-mental vaccine calledBHT-3009 for multiplesclerosis. Some 400,000people in the United Stateshave this incurable degen-erative disease of thecentral nervous system.

The injection producesan immune response thatseems to disable cells thatattack myelin, the protec-tive sheaths around thenerves of the brain andspinal cord. The smallstudy showed that thevaccine was well toleratedandproduced beneficialchanges to the immunesystem.MRI scans alsoshowed reduced inflam-mation. Lead researcherAmit Bar-Or, MD, of McGillUniversity’s MontrealNeurological Institute,cautions that more re-search is needed; a trialwith 290 patients is nearlyfinished. Researchers arehopeful that the approachcould also be used to treatother autoimmunedisorders, such as type 1diabetes. 5+ years

Deirdre Casper

TAKING ASHOT ATMS

3-D way tospot tumors

The days of squashingyour size-Cs into a pancakeand enduring invasivebiopsies that turn out to befalse alarms may be ending.Two new medical tech-nologies promise to makebreast cancer screeninga lot more accurate—and ouchless:

• In a new, souped-upultrasound procedurecalled elasticity imaging,doctors move a probe overa suspicious lump, creatingmultiple images and send-ing the data through spe-cial software.Thecomputeranalyzes the pictures anddetermines if the mass issoft or hard, a good indica-tion of whether the lump

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AN ARTIFICIAL LUNGAnewdevicemay offer hope to patients in

desperate need of a lung transplant. Robert Bartlett,MD, a University of Michigan professor emeritus ofsurgery, and his team have developed the BioLung,an artificial lung that works with the heart’s ownpumping action to manage oxygen and blood flowthroughout the body. Because the deviceuses theheart rather thanmechanics to pump blood,the patient can stay active at home instead of beingsedated and hooked up to a machine in the hospital.The device can even remain in place followinga transplant, until the donor lungs are fullyoperational. 5 years Ann DiCesare

PARKINSON’SPATCHAmajor hurdle for

treating Parkinson’s dis-ease is getting medicationto work consistently sothat a patient’s symptomsdon’t get worse before it’s

time to take the next pill.Now a company called UCBhas developed Neupro, aneasy-to-use dopamine-style patch that keeps thedrug working for 24 hours.

It improves movement andlets those with early-stageParkinson’s stay more ac-tive during the day—andsleep better at night. Now

Susan Doremus

NEW BONEBUILDERS

BioLungcan keeppatientsbreathing.

Fractures from fragile bones affect half of women over 50. Two newmedications can help repair them—and reduce the risk.

•Forteo If you’ve broken a bone, Forteo (teriparatide) may be thedrug for you. Unlike bisphosphonates (such as Fosamax and Boniva),which inhibit the cells that cause bone loss, Forteo stimulates the cellsthatmake bones larger and harder.The result: a greater increase ofbone mineral density in a shorter time. Researchers agree that bisphos-phonates are beneficial in treating moderate osteoporosis. But after afracture, Forteo’s bone-building prowess can be a real boost.

•Reclast Some pills for osteoporosis can be complicated to take(and remember). Now one IV dose of Reclast (zoledronic acid) justonce a year can strengthen bones and protect against fractures. It’sgiven via a 15-minute intravenous infusion. Now Fran Lostys

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BREAKTHROUGHS ’08

MAPPING OUROWNGENESBiologist J. Craig Ven-

ter, PhD, founder of theInstitute for GenomicResearch, made headlineslast September when hedecoded his genomeand published it on theInternet. He spoke to RDabout what his achieve-ment means for medicine.

RD:Help us mere mortalsunderstand this process.Venter: It’s similar tosolving a jigsaw puzzle:You pick up one piece andcompare it to all the oth-ers until you have a match.We shattered the six

billion base pairs of DNAin my genome into evensmaller pieces. Then acomputer assisted incomparing each tiny pieceof DNA to the next untilthere was a match.

RD:But why do it?Venter: I believe the fu-ture of medicine dependson individual genomics.Doing research on singlegenes isn’t enough. Weneed to look at the wholegenome and the wholeperson in order to preventand treat disease better.

RD: So just one genomeisn’t enough?Venter:That’s right. Weneed a large data set tounderstand how diseaseswork. Our website hasbeen inundated withthousands of volunteers

already, even thoughwe haven’t asked

for any yet.

RD:You found you carrygenes that predispose youto Alzheimer’s, diabetes,blindness and heart dis-ease. Does this worry you?Venter:Not really. I’vetried to change my dietand get more exercise.And I started taking astatin to lower my choles-terol. This is one of thechallenges of medicinegoing forward: learningto prevent diseases versustreating them later.

RD:When do you think anaverage person could goto her doc and ask for agenome analysis?Venter:Within a decade,you will be able to haveyour genome sequencedfor a few thousanddollars.

RD:What’s next?Venter: I’d certainly like tosee this enable preventivemedicine. Another goalis to make sure we havea chance of longer-termsurvival here by changing

what we’re doing to theenvironment before

it’s too late.Kathryn M.

TyranskiGenomics isour future, saysJ. Craig Venter, PhD.

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NEW WAYS TOSEE INSIDE•Remember the doctoron Star Trek

waving a handheld scanner over a patient inthe sick bay? Now there’s something similar:the first pocket-size ultrasound machine.

The Siemens Medical Solutions AcusonP10 saves critical minutes by letting

doctors, nurses and EMTs screen patientsanywhere quickly and administermedical care

on themove.GE Healthcare is working on a similar device. Now•Doctorsmay soonbe able to locate tumors betterwith a new

ultraminiature endoscope that promises to boldly go where no one hasgone before. Developed at Massachusetts General Hospital, the deviceuses light from a single optical fiber through a probe about the width ofa human hair. That will allow it to navigate and showcase delicate bodystructures, such as the middle ear or ventricles in the brain, with less tissuedamage. It also reduces the need for anesthesia. 3 years Neena Samuel

COLON CANCER SCREENINGMADE EASIERCould a simpleblood

test replace the dreadedcolonoscopy? Johns Hop-kins researchers found theblood proteins CCSA-3 andCCSA-4 in every patientwith colon cancer and

most with precancerouspolyps. The proteins, whichappear to be cast off fromcancer cells,didnotappear in thebloodofhealthy subjects,explainsstudy author Robert

Getzenberg, PhD. A patientwhose blood tested posi-tive would probably stillneed a colonoscopy. Butthe test may become obso-lete as a screening tool.3-5 years Nancy Coveney

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Pocket-s izeultrasound

FINALLY, A WEAPONAGAINST ALSLouGehrig’s disease (amyotrophic lateral sclerosis, or ALS) is a heartbreaking illness

that causes degeneration of the nerve cells that control muscles, and robs its victims ofspeech, movement and eventually their lives. There’s no cure. But researchers at WakeForest University School of Medicine have discovered a way to “keep muscle and nervecells talking to each other, which keeps the cells healthier longer,” says Carol Milligan,PhD, senior investigator of the study. A protein called Hsp70, which is made naturallyin the cells of animals and humans, was injected into mice with ALS. The treatmentdelayed the onset of symptoms and extended their livesby about 10 percent.Someday it may do the same in humans. 5+ years Deirdre Casper

(RIG

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Fiber-opticscope

Last August, Jeffrey Cadeddu, MD,at the University of Texas SouthwesternMedical Center, removed a patient’skidney through the belly button. Thefirst-of-its-kind procedure was donewith only one inch-long incision and leftthe patient with anearly invisible scar.

Dr. Cadeddu is now working on a systemthat will use magnets outside the abdomen toattract and guide other magnets attached to tinysurgical tools inside the abdomen. This MagneticAnchoring and Guidance System (MAGS) may lead to improvements in many minimallyinvasive surgeries—and that means less pain and disfigurement. 2-3 years Ed Goralski

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BREAKTHROUGHS ’08

Magnets toguide surgicaltools .

ONE BLOOD TYPE FITS ALLGive someonewith type A blood a batch of type B and it

could be fatal. That’s why type O is always in demand: Itworks for anyone. Now ZymeQuest, in Beverly, Massachu-setts, is working on technology to convert all types toO,by removing the sugar molecules that dictate blood type. Ifit works, it will put an end to deadly mistakes and help preventblood shortages. 3-5 years Patricia Curtis

UNIVERSALCANCER CURE?What if therewere

a treatment that couldtarget more than 90% ofcancers? Scientists at theUniversity of Pennsylva-nia’s Wistar Institute maybe getting close.Allcancers have somethingin common: an enzymecalled telomerase. Essen-

tial in protecting DNAduring cell replication,telomerase helps us growto adulthood, and then itshuts off. But in cancerpatients, telomerase isturned back on, causinguncontrolled cell growth.

Emmanuel Skordalakes,PhD, was able to decode

part of the structure oftelomerase and translateit into a 3-D image. Beingable to see what the en-zyme looks like for the firsttime will help scientistsfind a molecule that canturn off its damagingeffects. 5+ years

Kathryn M. Tyranski

SCAR-LESSSURGERY

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LIFESAVING LIST

>>NO-BRAINERBREAKTHROUGH

See video of someof these amazing

discoveries at rd.com/breakthrough.

Working in thetrenchesof the intensivecare unit at Johns Hop-kins, Peter Pronovost, MD,noticed something. Often,amid the chaos and stressof the busy ICU, hiscolleagues would failto follow basic guide-lines for preventingbacteria from invadingthe tubes, or lines,that deliver medicineand fluids directly tomajor blood vessels.

Each year, hospitalssee roughly 80,000cases of line infec-tions, which causelonger stays in thehospital, spiralingcosts and sometimeseven death. Figuringthese complicationscould be avoided, Dr.Pronovost institutedsomething so simple, hiscolleagues initially found itinsulting. Following thebasic recommendationsmade by the CDC, hedevised a concise checklistthat all doctors must fol-low when inserting centrallines (see box, center).

Should any doctor fail tocomplete any one of these

instructions, a nursewould be nearby (withchecklist in hand) to cor-rect the physician. From2001 to 2002, the hospi-tal’s rate of line infectionsdropped from 11 a day to

zero and saved an esti-mated $1.8 million.

Soon after, the stateof Michigan adopted Dr.Pronovost’s checklist andsaw infection rates slashedby 60 percent within thefirst three months. Almosttwo years later, the Michi-gan Health and HospitalAssociation estimatesthat the project has saved1,600 lives and $165 mil-

lion. “Better and safercare, faster recovery,improved comfort forpatients and lower healthcare costs all come fromreducing and preventinginfections,” says MHA

President SpencerJohnson.

Five years on, whyisn’t this standardpractice in every U.S.hospital? Good ques-tion. If adoptednationwide, the simpleprocedure could save18,000 lives and over$2 billion each year.That’s enough toprovide health insur-ance to 800,000uninsured people.

Dr. Pronovost rec-ommends contactingyour local health care

association. “Ask for yourhospital’s infection ratesand then find out what it’sdoing to eliminate them.”A full list of all hospital as-sociations can be found atamericanhospitals.com.Now Tara Conry

Dr. Pronovost’s checklist

1.Wash your hands.

2.Clean the patient’s skin withchlorhexidine antiseptic.

3. Place sterile drapes over thepatient’s entire body.

4.Wear a sterile gown, hat,mask and gloves.

5. Put a sterile dressing overthe catheter site once the lineis inserted.