TURN­ING ILL­NESS INTO AC­TIVISM

Re­source­ful val­ley en­tre­pre­neur fights dis­ease with ad­vo­cacy

The Mercury News - - Front Page - By Lisa M. Krieger lkrieger@ba­yare­anews­group.com

Newly di­ag­nosed with a deadly ge­netic dis­ease, Palo Alto en­tre­pre­neur Ben LeNail walked into a con­fer­ence of fel­low pa­tients and was stunned by what he saw: a tableau of young boys in wheel­chairs, pro­foundly dis­abled and hope­less.

In shock, he turned and walked back out. Then he stopped, and re­turned. And vowed to make a dif­fer­ence.

“‘Go back and make friends,’ I told my­self. ‘It’s the same dis­ease,’ ” he said. “I re­al­ized that we’re not so far apart.”

With lim­ited time but enor­mous tal­ent and Sil­i­con Val­ley know-how,

War­rior: “With his back­ground, he is so well-equipped to tackle the ALD world head on. He’s been a guid­ing light, some­one I can look up to and ad­mire, help­ing me stay hope­ful and pos­i­tive.” — Justin D’Agostino, 33, an ALD suf­ferer

LeNail has shifted from grief to ac­tivism for those less for­tu­nate, mo­bi­liz­ing his 25 years of tech­nol­ogy and startup ex­pe­ri­ence to ac­cel­er­ate di­ag­no­sis, treat­ment and aware­ness of a rare and lit­tle-known neu­rode­gen­er­a­tive dis­ease. It's called adrenoleukodys­tro­phy, or ALD.

ALD is one in a fam­ily of ge­netic ill­nesses that de­stroys the myelin, or in­su­la­tion, that al­lows brain cells to com­mu­ni­cate with one an­other. Af­fect­ing 1 in 16,000 males, there are few treat­ment op­tions, as dra­ma­tized in the 1992 movie “Lorenzo's Oil.”

About half of cases ap­pear in child­hood, caus­ing nor­mal healthy boys to quickly go blind and deaf, lose mo­tor con­trol, de­velop seizures and de­men­tia — dy­ing within three to five years. The only ther­apy is stem cell trans­plan­ta­tion, and it's risky.

The other half of cases typ­i­cally oc­cur in young adults, ages 20 to 30s, and progress more slowly.

Then there's LeNail.

A prob­lem to solve

Now 51, he didn't ex­pe­ri­ence his first symp­toms un­til age 38 — time enough to build an im­pres­sive re­sume and Rolodex. He ex­er­cises, men­tors, en­ter­tains friends and di­rects busi­ness de­vel­op­ment at Alta De­vices, a Sun­ny­vale com­pany that man­u­fac­tures thin flex­i­ble so­lar cells. He was pre­vi­ously a dis­tin­guished vis­it­ing scholar at Stan­ford Univer­sity and se­nior di­rec­tor of cor­po­rate de­vel­op­ment at Sun Mi­crosys­tems.

A life­long ath­lete, he was puz­zled when his legs be­gan to feel leaden while jog­ging the leafy streets of Palo Alto. He prided him­self on sum­mit­ing Mount Rainier and Mount Shasta, play­ing squash with friends and ski­ing with fam­ily in Switzerland.

In­creas­ingly, he stum­bled. His gait changed.

“You think you're stressed out or tired. Then it pro­gresses and you start catch­ing your feet in rugs,” he said.

A long and frus­trat­ing di­ag­nos­tic odyssey, which in­cluded bunion surgery and other failed remedies, ended on Christ­mas Eve. The news was un­ex­pected — and dev­as­tat­ing.

“Our fam­ily col­lapsed for about a year. We were just in shock,” re­called his wife Lau­rie Yoler, an in­vestor, board mem­ber and strate­gic ad­viser with the ro­bot­ics mo­bil­ity com­pany Zoox.

“You think: ‘What are we go­ing to do?'” she said. “There's no good route for­ward.”

Then LeNail saw some­thing he rec­og­nized: a chal­lenge. Prob­lem-solv­ing was some­thing he was good at, sick or not.

He has no train­ing in medicine. Born and ed­u­cated in France, he stud­ied con­sti­tu­tional law, with an eye to­ward be­com­ing a French civil ser­vant un­til

vis­it­ing and fall­ing in love with Seat­tle, Yoler and tech­nol­ogy.

But he's not alone; other Sil­i­con Val­ley lead­ers are reach­ing be­yond tech to seek cures. Sergey Brin, the bil­lion­aire co-founder of Google, has con­trib­uted money and his DNA to a large study to re­veal the ge­netic un­der­pin­nings of Parkin­son's, a dis­ease for which he has a hered­i­tary pre­dis­po­si­tion.

Kris­ten and Matt Wilsey, a Stan­ford-ed­u­cated tech en­tre­pre­neur and mem­ber of San Fran­cisco's prom­i­nent Wilsey fam­ily, are fund­ing re­search into their daugh­ter Grace's rare ge­netic dis­ease. The fam­ily of Ray Dolby, the sound en­gi­neer and founder of Dolby Labs, do­nated to­ward Alzheimer's re­search af­ter their fa­ther's fa­tal di­ag­no­sis.

In­no­va­tions ex­pand their reach. Con­nected through so­cial me­dia, pa­tient com­mu­ni­ties rep­re­sent a new par­a­digm for con­duct­ing med­i­cal re­search, link­ing suf­fer­ers and sci­en­tists to make an im­pact. And gene

se­quenc­ing, once too ex­pen­sive for com­mon use, is now read­ily avail­able, help­ing re­veal a ge­netic cul­prit of mys­tery dis­eases.

LeNail has im­mersed him­self in med­i­cal texts, learn­ing all he can about why it's im­pos­si­ble for him to me­tab­o­lize cer­tain types of fats, caus­ing a toxic ac­cu­mu­la­tion of fatty acids on nerve cells — and a cas­cade of neu­ro­log­i­cal prob­lems.

‘My clock is tick­ing’

He stud­ies ge­net­ics and gene ther­apy, deep­en­ing his un­der­stand­ing of ALD's com­plex in­her­i­tance pat­tern. It's caused by a mu­ta­tion in a gene called ABCD1, lo­cated on the X chro­mo­some. This means that women tend to be just car­ri­ers, and have a 50 per­cent chance of giv­ing it to each child. Men, who trans­mit it to all of their daugh­ters but none of their sons, are al­ways af­fected.

LeNail se­quenced parts of his genome, and dis­cov­ered the likely se­cret to his slow pro­gres­sion: some ”re­sis­tance” genes. “Ben's been dealt some good cards,” said Yoler.

There are of­ten road­blocks in di­ag­no­sis, LeNail has found, due to doc­tors' un­fa­mil­iar­ity and of­ten non­spe­cific symp­toms. The re­search com­mu­nity has been small and frag­mented. And pa­tients' fam­i­lies of­ten suf­fer alone, lack­ing a way to find each other.

Early progress was made through serendip­ity and old-fash­ioned net­work­ing, such as cof­fee chats and friendly ref­er­ences. He found an ex­pert in France, who re­ferred him to a physi­cian in Bos­ton.

Even as his body weak­ens, LeNail is build­ing mo­men­tum.

Seek­ing West Coast ex­per­tise, he de­tected in­ter­est from a ris­ing star at Stan­ford's Lu­cile Packard Chil­dren's Hos­pi­tal: Dr. Keith Van Haren, a young as­sis­tant pro­fes­sor of neu­rol­ogy. He took Van Haren out to cof­fee, de­scribed the power of part­ner­ships, then out­lined the po­ten­tial of a foun­da­tion that could help sup­port Van Haren's re­search.

“The unique thing about hav­ing Ben on your side is you have some­one who un­der­stands the dis­ease in many ways bet­ter than spe­cial­ists do,” said Van Haren. “He brings with him a whole host of in­sights, in terms of what mat­ters to pa­tients, what things might be rel­e­vant for clin­i­cal tri­als and a lot of wis­dom about how to ex­e­cute these things.

“And then you have the re­ally im­por­tant com­po­nent: A deep sense of ur­gency,” Van Haren said. “It is ‘My clock is tick­ing' — not ‘Wouldn't it be neat some­day?' but ‘Let's get this done now.' “

LeNail helped or­ga­nize eight pa­tient ad­vo­cacy groups, then part­nered them with seven aca­demic cen­ters across the U.S. to form a con­sor­tium called ALD Con­nect to im­prove care — and ul­ti­mately erad­i­cate — the dis­ease.

“With his back­ground, he is so well-equipped to tackle the ALD world head on,” said Justin D'Agostino, a 33-year-old from Phoenix who is bat­tling the dis­ease. “He's been a guid­ing light, some­one I can look up to and ad­mire, help­ing me stay hope­ful and pos­i­tive.”

LeNail suc­cess­fully lob­bied for leg­isla­tive change, get­ting X-ALD added to new­borns' blood screen­ing panel. He pro­duced two videos, one ed­u­ca­tional and the other show­ing ex­er­cises. He speaks at con­fer­ences, such as Stan­ford's Medicine X.

Now he's vis­it­ing new biotech com­pa­nies, as­sur­ing them that there's a well-or­ga­nized com­mu­nity of pa­tients ready to share valu­able clin­i­cal in­for­ma­tion.

New suc­cess

There was promis­ing news last month in a gene ther­apy trial by Blue­bird Bio, sta­bi­liz­ing the dis­ease in 15 of 17 young pa­tients dur­ing more than two years of fol­low-up.

The arse­nal of ther­a­peu­tics must ex­pand, he said. “You lit­er­ally have hun­dreds of small biotechs that are look­ing for an ill­ness to cure,” he said. “They're hun­gry to try some­thing.”

But one of the big­gest trans­for­ma­tions has been per­sonal.

Be­fore his di­ag­no­sis, “he'd go into this room and close the door of his of­fice and read,” said Yoler. “That was who he was on the week­ends — an in­tro­verted guy. Now our house is full all the time.”

LeNail said he's had to let go of the life he planned to suc­ceed in this new life.

“I'm not the ath­letic guy any more. But I can't be an am­bas­sador for a dis­ease if I'm just go­ing to cling to that loss,” he said.

“I can take this hor­ri­ble di­ag­no­sis and turn it into some­thing much big­ger than me,” he said, “cre­at­ing some­thing much more pow­er­ful.”

JOSIE LEPE — STAFF PHOTOGRAPHER

A chal­lenge: Ben LeNail suf­fers from the rare neu­rode­gen­er­a­tive dis­ease called adrenoleukodys­tro­phy, or ALD, but con­tin­ues to ex­er­cise and has be­come some­thing of an am­bas­sador for those who have it.

GARY REYES — STAFF PHOTOGRAPHER

A part­ner: Ben LeNail, left, tapped Dr. Keith Van Haren at Stan­ford’s Lu­cile Packard Chil­dren’s Hos­pi­tal to cre­ate a foun­da­tion for ALD pa­tients.

JOSIE LEPE — STAFF PHOTOGRAPHER

Ben LeNail has be­come an ad­vo­cate for find­ing a cure for ALD, lob­by­ing to get X-ALD added to new­borns’ blood screen­ing, speak­ing at con­fer­ences and con­nect­ing pa­tients.

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