Research is a form of hope, and Seattle Children’s is on a quest to give every child the opportunity to benefit.
Havianna Hornish, 9, (with mom Suzanne Hornish) has been cancer-free for nearly two years, thanks to the cancer care team at Seattle Children’s. Because our doctors also conduct groundbreaking research, our patients have access to leading-edge therapies.
When Dr. Bob Sawin was training asa surgeon, one of his mentors wasthe late Dr. Joseph Murray – a Nobelprize–winning plastic surgeon whosecuriosity about skin grafting eventuallyled him to perform the first successfulkidney transplant.
“His interest in a clinical problemled to medical advances that vastlyimproved the lives of hundreds ofthousands of people,” notes Sawin,who is now Seattle Children’s surgeon-in-chief. “The impact of his curiosityand the research it sparked stillinspires me today.”
Integrating care and cure
The road to improved treatmentsusually starts with a medical questionfor which there isn’t yet a satisfyinganswer followed by efforts to findbetter ones. The cycle is often referredto as “bedside to bench to bedside.”
“We have a great opportunity tobring our care and cure enterprisescloser together for the benefit of ourpatients, and for children everywhere,”says Dean Allen, chair of Seattle Children's Research Institute AdvisoryBoard. “We want the insights of ourtop-notch clinicians to influenceand inspire the work of our incrediblescientists – that’s the way we’lldeliver on Children’s mission toprevent, treat and eliminatepediatric disease.”
Dr. Soheil Meshinchi’s research dramatically upped the chance of survival from 10% to 90% for kidswho relapse with an aggressive form of leukemia – fantastic results that are being tested nationallyto see whether the investigational treatment should become standard care.
The outlook for children with cancerhas improved dramatically in the last50 years because pediatric oncologistsacross the country work together todevelop new treatment approaches.It’s a model that provides patientswith the opportunity to participate inresearch and benefit from the mostcurrent thinking on therapies.
That constant striving to improvewas a lifesaver for Havianna Hornishof San Diego, who was visiting cousinsin Bremerton, Washington, the summer shewas 6. When her parents brought herto Children’s for what they thought wasa sinus infection, she was diagnosedwith a particularly aggressive type ofblood cancer that had horrible oddsfor survival.
“We almost returned to San Diegofor Havianna’s treatment,” recalls hermom Suzanne Hornish. “But when wediscovered that doctors at Children’swere researching and treating theexact type of cancer Havianna had,we made a last-minute decision tostay in Seattle.”
Dr. Soheil Meshinchi is renowned forhis research on acute myeloid leukemia (AML) with the specific gene mutationHavianna had. He had some compellingdata that sorafenib (a medication used to treat kidney cancer) might work forher type of leukemia.
His hunch was right. “The result wasfantastic. Kids who relapse with thistype of cancer typically have a 10%chance of survival,” Meshinchi explains. “With sorafenib, we’re seeing survivalrates of about 90%.”
Havianna was one of the first inthe nation to receive sorafenib totreat AML; about 20 more kids havereceived it since. The results havebeen so overwhelmingly positive thatChildren’s started a national clinicalresearch study last fall to test whethersorafenib should become part of thestandard care for AML with thismutation.
“With sorafenib, we’re seeing survivalrates of about 90%.”
“Because of the care Haviannareceived she’s been cancer-free fornearly two years and she’s even backon the soccer field,” says her mom. “We owe it all to Seattle Children’s.”
A bold step
Logan Lewis, 11, (with brother Peyton, 5) delivers holiday gifts to kids battling neuroblastoma – atradition he started after winning his own fight with the disease. Logan’s eight-month treatment atSeattle Children’s was followed by five more months in a clinical trial to rid his bodyof any remaining cancer cells.
Children’s quest to bring care and curecloser together would replicate successstories like Havianna’s throughout thehospital. A five-year strategic planninginitiative, led by Dr. Bonnie Ramsey andSawin, is a bold step toward helpingscientists integrate their researchstudies into the daily business ofpatient care – and giving families easyaccess to opt in or out of participating.
They envision a future wherepatients’ confidential medical recordsare connected to a secure database ofall the open clinical research studies atChildren’s – an electronic advancementthat would identify patients who areeligible for specific studies.
When patients and their familiescheck in for an appointment, they’d beinvited to participate in an appropriatestudy or even asked if they would allowclinicians to save any blood or tissuesamples for future studies.
Families who opt in would beprovided with information aboutthe study while they wait for theirappointment. If they have questions,a research associate would talk tothem right away.
The power of participation
Samuel Olszta, 3, (with mom Gosia Olszta and Dr. Margaret Rosenfeld) stays healthy with cysticfibrosis treatments developed, in part, through clinical research at Seattle Children’s. The Olsztasview their participation in research as the path to their son’s future wellness.
When Pawel and Gosia Olszta kissedtheir newborn son, Samuel, they couldtaste salt – which they learned was atelltale sign of cystic fibrosis (CF).
As recently as the 1980s, childrenwith CF lived to an average age of just15. Today, because of clinical researchdone at Children’s and other centersacross the country, the average lifeexpectancy of people with CF is 40 –a great advance, but still not nearlygood enough.
“We know the treatments that keepSamuel healthy today didn’t come outof the void,” says dad Pawel Olszta. “It’s all thanks to research and theparticipation of thousands of people.”
When Samuel was 2, the Olsztasjumped at the chance to enroll him ina clinical study to see if hypertonicsaline – which helps reduce respiratoryflare-ups in older patients with CF – was effective in children under 5.
Though the study ultimately showedthat hypertonic saline did not work for younger patients, the Olsztas remaincommitted to the research processand finding better cures.
“Research is a form of hope. Familiesexpect us to challenge the statusquo and develop new and bettertreatments; in fact, they demand it.”
“Research is a form of hope,”reflects Ramsey, who helped developan inhaled antibiotic that dramaticallyimproves life for children with CF. “Families expect us to challengethe status quo and develop newand better treatments; in fact, theydemand it. Being part of an institutionthat is committed to connectingresearch opportunities and clinicalcare is a giant magnet for familiesseeking the best possible care fortheir child.”
Research brings hope
Katelyn Mhyre, 4 (with dad Andrew Mhyre), was within three months of hospice care when anexperimental medication suggested by Seattle Children’s Dr. Russ Saneto reduced her seizures frommultiple times a day to only a few each month.
When Katelyn Mhyre was diagnosedwith mitochondrial disease almostfour years ago, Jennifer and AndrewMhyre – who are both neuropharmacologists – had to reach for a textbookto learn how this relatively unknowndisease prevents cells from making theenergy their infant daughter neededto live and grow.
Katelyn was having seizureshundreds of times per day when theMhyres met Dr. Russ Saneto, a pediatricneurologist nationally known for hisresearch on mitochondrial disease.About a year ago, Saneto suggestedKatelyn take EPI-743, a medicationthen only available as part of acompassionate use study for childrenwithin three months of hospice care.
“Before Katelyn started themedication, I wouldn’t even buy herclothes in the next size, because I didn’tknow if she would need them,” recallsher mom. “Now Katelyn’s seizures havedecreased and her general health hasimproved to the point that we can lookahead to a visit with my family in NewYork next year and feel confident thatKatelyn will be with us. Our constantanxiety has been replaced with hopefor the future, and for that we areincredibly grateful.”
You can support the researchthat starts in our own backyard andimproves the lives of children aroundthe world by becoming a researchchampion. For more information, orto tour Seattle Children’s Research Institute, visit our Research Champions page.
Published in Connection magazine, April 2013