Once par­a­lyzed by tu­mor, teen is now ‘cured’ of can­cer

Baltimore Sun Sunday - - FRONT PAGE - By Sarah Mee­han

Eight years ago, Kait­lyn Dor­man was so sick with can­cer she was par­a­lyzed and her vi­sion blurred. De­pen­dent on a wheelchair, the lit­tle girl was con­fined to liv­ing on the first floor of her fam­ily’s home.

Now 17, Kait­lyn can walk and see clearly. And she’s star­ing down her fi­nal months at Lib­erty High School, weigh­ing col­lege ac­cep­tance let­ters and com­pos­ing tunes on her beloved pi­ano.

She came to Dr. Eric Raabe as a 9-year-old with an ag­gres­sive brain tu­mor, and to­day he doesn’t hes­i­tate when he de­scribes her can­cer out­come.

“We’re us­ing the c-word, for cure,” said Raabe, an as­so­ciate pro­fes­sor of pe­di­atric on­col­ogy at the Johns Hop­kins Kim­mel Can­cer Cen­ter. “It’s not com­ing back.”

In a mat­ter of months, treat­ment with an ex­per­i­men­tal drug melted Kait­lyn’s de­bil­i­tat­ing tu­mor from the size of a wal­nut to the size of a grain of rice, restor­ing the mo­bil­ity and sight she lost as the tu­mor grew. And eight years later, the mass has not re­turned.

The kind of treat­ment Kait­lyn re­ceived is what’s known as a tar­geted ther­apy — us­ing a drug that only works in cer­tain pa­tients, on spe­cific tu­mors by at­tack­ing par­tic­u­lar mol­e­cules that al­low the tu­mor to grow. It won’t work for all kids with the same kind of can­cer. Re­searchers at Hop­kins and across the coun­try are con­duct­ing an­other trial of the drug to learn from Kait­lyn’s out­come. They want to know how to make the treat­ment more ef­fec­tive for other pe­di­atric brain tu­mors.

Such highly tai­lored treat­ment is the future of can­cer care, doc­tors say.

While many chil­dren sur­vive the can­cer Kait­lyn had, a rare few en­joy the qual­ity of life she’s achieved — some­thing re­searchers are striv­ing for in other pa­tients with the same con­di­tion.

Low-grade gliomas, the cat­e­gory of brain tu­mor Kait­lyn de­vel­oped, are among the most com­mon brain tu­mors in chil­dren. Her spe­cific type of tu­mor — a pi­lo­cytic as­tro­cy­toma — is a slow-grow­ing mass that de­vel­ops in the sup­port­ing cells of the brain. It ac­counts for about one in five brain tu­mors in chil­dren, ac­cord­ing to the Amer­i­can Can­cer So­ci­ety.

About 95 per­cent of chil­dren who de­velop pi­lo­cytic as­tro­cy­tomas sur­vive at least five years from their di­ag­no­sis, the Amer­i­can Can­cer So­ci­ety re­ports. But the tu­mors can con­strict op­tic nerves and put pres­sure on other parts of the brain, caus­ing some pa­tients to go blind, have prob­lems with balance or de­velop other dis­abil­i­ties that can last a life­time if the tu­mors don’t re­cede.

“Right now there’s still a lot of pa­tients that have lost all their sight ... even though their tu­mor doesn’t grow any­more,” Raabe said. “What’s ex­cit­ing in Kait­lyn’s case, and in the case of a few other pa­tients where we’ve ac­tu­ally seen tu­mors re­ally shrink down, is that that loss of sight isn’t per­ma­nent . ... The eye is fine, the nerves are fine, the rest of the brain is fine.”

An on­looker would never know Kait­lyn was once par­a­lyzed in both legs and one arm. She re­cov­ered her sight and mo­bil­ity — down to her toes and, cru­cially, her fingers.

A pi­ano player from age 5, Kait­lyn con­tin­ued play­ing through her can­cer treat­ment, even when she could play only with one hand. Pi­ano lessons later be­came a form of phys­i­cal ther­apy as she re­gained dex­ter­ity in her left hand.

She now plays in a band called Psy­cho­so­matic, com­poses and records songs, and has a mini-al­bum in the works. Col­leges have taken no­tice of Kait­lyn’s mu­si­cal tal­ents — though she also talks of study­ing Ja­panese — and her ac­cep­tance let­ters are rolling in, in­clud­ing one from Bos­ton’s Berklee Col­lege of Mu­sic.

Other pa­tients aren’t so for­tu­nate. Though their tu­mors stop grow­ing, they of­ten don’t shrink, leav­ing pa­tients per­ma­nently dis­abled. Many such tu­mors are in­op­er­a­ble be­cause of their delicate po­si­tion in the brain — of­ten near the meet­ing of op­tic nerves, in the cere­bel­lum or, in Kait­lyn’s case, in the brain stem.

Af­ter Kait­lyn was di­ag­nosed, doc­tors placed her on a stan­dard chemo­ther­apy reg­i­men, which typ­i­cally ar­rests the can­cer’s de­vel­op­ment. But her tu­mor re­sisted the first line of drugs, con­tin­u­ing to grow more ag­gres­sively.

Due to the high sur­vival rate, there’s lit­tle re­search about low-grade glioma in chil­dren, Raabe said.

“That has been a prob­lem for pa­tients like Kait­lyn, whose tu­mor doesn’t read the text­book and doesn’t re­spond to the up­front ther­apy,” Raabe said.

More re­search is be­ing con­ducted now as fam­i­lies of pa­tients with low-grade gliomas have de­manded and funded al­ter­na­tive treat­ments, said Amy J. We­in­stein, direc­tor of pe­di­atric low-grade as­tro­cy­toma re­search and ad­vo­cacy for the North Carolina-based Pe­di­atric Brain Tu­mor Foun­da­tion. Everolimus, the drug Kait­lyn re­ceived, was one of the first tar­geted ther­a­pies to be tested in chil­dren with low-grade brain tu­mors, and that trial laid the ground­work for other drug tri­als, she said.

“Un­til everolimus came along there re­ally wasn’t any­body study­ing how can we stop these tu­mors from grow­ing and im­pact­ing the kids,” We­in­stein said.

At the time Kait­lyn was sick, Hop­kins was one of a dozen in­sti­tu­tions of­fer­ing a clin­i­cal trial us­ing everolimus to treat low-grade gliomas in chil­dren.

The oral drug was de­vel­oped by the Swiss phar­ma­ceu­ti­cal com­pany No­var­tis un­der the brand name Afin­i­tor. Everolimus is al­ready ap­proved for use in adults by the U.S. Food and Drug Ad­min­is­tra­tion to pre­vent re­jec­tion of or­gan trans­plants and to treat some can­cer pa­tients. For can­cer, it works by in­hibit­ing pro­teins that can­cer cells need to grow.

“The amaz­ing thing is that there are these drug agents that are al­ready on the shelves at phar­ma­ceu­ti­cal com­pa­nies that just haven’t been tested in tar­geted ways,” We­in­stein said.

The first study, in which Kait­lyn was treated, con­firmed the safety of everolimus in pe­di­atric pa­tients with low-grade glioma and found that some pa­tients re­sponded to the medicine. Now in a fol­low-on trial, re­searchers in the Pa­cific Pe­di­atric Neu­roOn­col­ogy Con­sor­tium are study­ing brain tis­sue from pa­tients to es­tab­lish which are the best can­di­dates for the drug based on the bi­ol­ogy of their tu­mors. Hop­kins is one of the 18 in­sti­tu­tions in the con­sor­tium.

Kait­lyn’s care­givers of­fered the ex­per­i­men­tal al­ter­na­tive af­ter stan­dard chemo­ther­apy was un­suc­cess­ful.

“All we re­ally wanted to know was that our daugh­ter was go­ing to get bet­ter,” said Mary Dor­man, Kait­lyn’s mom. “I wasn’t ap­pre­hen­sive about the clin­i­cal trial or even any­thing we had to do in terms of col­lect­ing ad­di­tional data or mon­i­tor­ing her. As a par­ent, I think we would have done any­thing to give her an op­por­tu­nity to sur­vive the tu­mor that she had.”

Kait­lyn’s out­look re­mained sunny. Hospi­tal vis­its didn’t seem like a heavy bur­den, she said, de­spite hav­ing ports placed in her body and shunts sur­gi­cally in­serted to drain spinal fluid.

“It was just a doctor’s ap­point­ment,” Kait­lyn said. “When I got a port … it didn’t re­ally feel like the se­ri­ous­ness of get­ting treated for can­cer. It just kind of felt like I was get­ting my ears checked or what­ever.”

But her con­di­tion was se­ri­ous, and she felt worst be­fore she be­gan tak­ing everolimus. Symp­toms that be­gan with a mild tremor in her hand de­te­ri­o­rated into the paral­y­sis of her arm and legs. Pres­sure from the tu­mor caused her right pupil to di­late, blur­ring her vi­sion. And she de­vel­oped a stom­ach in­fec­tion dur­ing a hospi­tal stay.

She was home-schooled from the Fe­bru­ary of her fourth-grade year to Novem­ber of fifth grade.

“I couldn’t walk and I couldn’t re­ally see,” she said. “Hear­ing that there was, like, some­thing else that could pos­si­bly do some­thing, could pos­si­bly make ev­ery­thing go away, I was like, ‘Awe­some. OK, let’s try it.’ ”

When she be­gan tak­ing the drug, she saw ben­e­fits al­most im­me­di­ately, she said. And it wasn’t long be­fore she re­gained mo­bil­ity.

“It was like a snap of the fingers — ev­ery­thing was just work­ing again,” she said. “The sec­ond it started hap­pen­ing I was like, ‘I need more of this.’ I need to, like, start mov­ing again, so then I could start walk­ing and start run­ning.”

Mary Dor­man re­called her daugh­ter sum­mon­ing the fam­ily to her bed­side to demon­strate she could lift her left arm above her head again — a trick she later showed Raabe dur­ing a hospi­tal visit.

“We didn’t even need the MRI picture to know that it was work­ing,” Raabe said.

Kait­lyn stopped tak­ing everolimus af­ter ex­pe­ri­enc­ing liver dam­age from the pills — a known side ef­fect. Still, her tu­mor con­tin­ued to shrink. And for the five years doc­tors tracked her re­cov­ery af­ter the treat­ment, it did not return.

Be­cause Kait­lyn had the best re­sponse of any pa­tient in the trial, re­searchers are look­ing to repli­cate her re­sults as they con­tinue study­ing everolimus. Oth­ers in the first trial also saw im­prove­ments, but some didn’t.

“How do we get more pa­tients to have a re­sponse to this drug that are like Kait­lyn?” Raabe said. “[We’re] fig­ur­ing out how to make the drug be the right drug for the right pa­tient at the right time.”

Raabe ex­pects the cur­rent everolimus trial to con­clude within the next year. He said future stud­ies could ex­am­ine how everolimus can make tra­di­tional chemo­ther­apy drugs more ef­fec­tive when they’re used si­mul­ta­ne­ously.

Dr. Daphne Haas-Ko­gan, chair of the ra­di­a­tion on­col­ogy de­part­ment at the DanaFar­ber Can­cer In­sti­tute in Bos­ton, said tar­geted ther­a­pies like everolimus that are tai­lored to an in­di­vid­ual’s can­cer — down to the mol­e­cules — will be trans­for­ma­tive for can­cer treat­ment.

Drugs like everolimus are par­tic­u­larly at­trac­tive be­cause they’re taken orally, re­quire fewer hospi­tal vis­its and are not toxic like tra­di­tional chemo­ther­apy, HaasKo­gan said. She ex­pects a com­bi­na­tion of chemo­ther­apy paired with tar­geted agents like everolimus to dom­i­nate can­cer care in the future.

Doc­tors have a “grow­ing ar­mada” of tar­geted agents that can be used to treat par­tic­u­lar tu­mors, Haas-Ko­gan said, but their grasp on which drugs will suc­ceed in par­tic­u­lar pa­tients re­mains lim­ited.

Stud­ies like the one at Hop­kins pro­vide doc­tors with a deeper un­der­stand­ing of which pa­tients are more likely to re­spond, she said.

“Ev­ery per­son’s brain tu­mor is unique — the ge­netic makeup and the mech­a­nism that led its de­vel­op­ment and its re­sponse to treat­ment,” Haas-Ko­gan said. “It’s per­son­al­iz­ing the care — whether it’s a child or an adult — that’s go­ing to be the most bang for the buck.”

We­in­stein cau­tioned that re­searchers have not yet doc­u­mented pos­si­ble longterm side ef­fects of drugs like everolimus be­cause the treat­ment is so new.

But, she added, “they give [pe­di­atric low-grade as­tro­cy­toma] pa­tients the great­est sense of hope for a nor­mal child­hood and for a nor­mal future.”

Kait­lyn Dor­man

JERRY JACK­SON/BAL­TI­MORE SUN PHO­TOS

Kait­lyn Dor­man, 17, poses with Dr. Eric Raabe at Johns Hop­kins Chil­dren’s Cen­ter. She came to Raabe when she was 9, seek­ing treat­ment for an ag­gres­sive brain tu­mor that left her par­a­lyzed and with blurred vi­sion. Raabe now says of Kait­lyn’s can­cer, “We’re us­ing the c-word, for cure.”

Liv­ing can­cer cells are pro­jected from a mi­cro­scope in a lab at Johns Hop­kins. As a young girl, Kait­lyn Dor­man dealt with low-grade gliomas, among the most com­mon brain tu­mors in chil­dren. Tar­geted ther­apy helped shrink the size of her tu­mor.

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