Tuesday, December 23, 2014

In Search of True Energy Independence

Published on Huffington Post

Americans are thanking their lucky stars that gas prices are falling to levels not seensince July 2010.
The root causes of the fall in oil prices are many: increasing production from sources such as U.S. shale, and declining consumption, due to improved vehicle fuel economyhere at home and stagnating economies abroad. At the same time, OPEC nations have agreed -- for the time being at least -- to allow oil prices to fall in an effort to drive high-cost oil producers such as U.S. shale operations bankrupt.
At the moment, though, everything is coming up roses: consumers are happy, OPEC is nervous, and America is producing a greater share of the oil we consume than at any time in the last 30 years. Could it be that the United States is on the verge of energy independence?
Not hardly, and here's why: as long as the United States remains dependent on the global market for oil -- even if an increasing share of that oil is produced domestically -- we remain subject to wild swings in price for a fossil fuel that is becomingincreasingly expensive to produce.
Oil price volatility hurts the economy, lowering consumer confidence and making businesses less willing to invest. For fear of future price fluctuations, everyone slows their spending. That decreases consumer demand, which has ripples throughout the economy.
Increasing domestic oil production does not solve the problem. Oil produced in America does not necessarily stay in America; rather, oil is a globalized commodity, which is why revolution or war in far-off lands has a rapid impact on gas prices here at home. While increasing production at home adds to global supply, the effect on prices is no different than if production were to increase in Saudi Arabia or Venezuela.
And a "drill baby drill" approach to maximizing oil production brings its own risks, including that of environmental damage that threatens precious natural places and warms the planet. Oil booms also have the potential to create big economic problems when they go bust, as U.S. shale oil producers may find out soon and as world leaders such as Vladimir Putin are becoming keenly aware.
There is only one true path to energy independence, one that frees our economy of its ties to volatile world oil markets and our environment of the damage and risk that comes from oil production: cutting our dependence on oil entirely.
America is actually making good progress in cutting our reliance on oil. This stunningBloomberg News data visualization shows that the U.S. is "shaking off its addiction to oil," with America using about a third less oil per dollar of GDP than we did in the mid-1970s. Americans are driving less, and what driving we do is in cars that are more fuel-efficient than in the past. New technologies -- such as electric vehicles -- areshowing strong promise for displacing oil use in the transportation sector.
In the 1980s and 1990s, America responded to lower oil prices by slacking off on efforts to reduce our consumption of oil -- fuel economy standards for cars stagnated, SUVs proliferated, progress toward alternative-fuel vehicles stalled and auto-dependent sprawl spread across the land, leaving us all, and the American economy, to pay the piper in higher fuel prices over the last decade.
Let's not make the same mistake again. By using oil more efficiently and continuing progress toward electric vehicles, Americans can finally enjoy the lasting economic, environmental and health benefits of true energy independence.

Wednesday, August 13, 2014

The Challenger: Can Shenna Bellows ’97 stun the political establishment by upsetting an entrenched incumbent and winning a U.S. Senate seat in Maine?

Published in Middlebury Magazine

Shenna Bellows ’97 is cold. This is not the sort of brief chill that passes now and again: It’s the deep, bone-shaking kind that both racks her slight frame and causes others nearby to shiver in sympathy. I am surprised for two reasons: First, Shenna knows Maine weather well. She grew up here, in a house that didn’t get electricity or running water until she was in fifth grade. When she and her siblings got home from school, their chore was to relight the woodstove. As often as not, though, they would huddle under the bedclothes, doing their homework until their parents got home. It is also surprising because the feeling she usually exudes is not contagious cold but rather infectious, friendly warmth.
Right now, though, Shenna is freezing. We have just gotten in the car after an election-campaign visit to Mount Desert Island Biological Laboratory, right on the Maine coast. She didn’t check the weather this morning and didn’t bring a coat on this blustery, sprinkling spring day.
As the heat comes on in the car, she settles into the backseat; her staffer-driver and I ride up front to give her space to work. She stops shivering, takes off her shoes, tucks her feet under her, and resumes her most frequent activity: typing on her iPhone.
Sending texts and emails whooshing into the ether is key to Shenna winning what she admits is “an uphill battle” against United States Senator Susan
Collins, a three-term Republican incumbent with a big bankroll, who is widely expected to win handily. Even more crucial will be Shenna’s idea of what a candidate and a campaign can be.
Her vision has paid off before. While heading the American Civil Liberties Union of Maine, she co-led the 2012 statewide same-sex marriage referendum that made Maine the first state to approve marriage equality at the ballot box. (Maryland did the same that day.)
The ACLU of Maine lobbied for and won major legislative victories too: passing a first-in-the-nation law requiring police to get a warrant before tracking suspects with cell-phone data, defending transgender Mainers’ rights, limiting police use of drones, and protecting women’s reproductive health rights.
She is still campaigning—and gaining national media attention and donations—for civil-rights protections, economic opportunity and justice, constitutional freedoms, and environmental protection. She is bringing together what she calls “an unusual coalition” of people who, like her, hold positions that transcend stereotypical political divides.
On the left of most Democrats, she backs universal single-payer health care, legalizing marijuana, “bold, visionary action” on climate change, and student-loan debt reform as a means to boost the economic prospects of young college graduates, who face the toughest job market in decades.
In the middle, she supports Internet neutrality and equal pay for women.
And well to the right—at times on turf occupied only by the libertarian wing of the Republican Party—she insists on ending the National Security Agency’s domestic-spying program and repealing the USA PATRIOT Act.
We have been friends for years; when she asked me what I thought would be the hardest thing about her running, I told her she would have to ask people for money not to support a cause, but to back her.
As a Peace Corps volunteer in Panama from 1999 to 2001, Shenna helped give microloans to artisans in a remote community. And, more recently, as executive director of the ACLU of Maine for more than eight years, she was responsible for raising its annual budget of around $750,000, as well as for contributions to the various campaigns the group joined.
But even now, Shenna is not running for herself. She says that she wants her candidacy to be viewed as a revolutionary rethinking of how campaigning—and politics—can and should work. And her plan for victory is very much like her previous public-service efforts, most notably that 2012 same-sex marriage campaign.
Then, supporters had hundreds of thousands of individual conversations with their friends, neighbors, and communities, making personal connections to explain the importance of marriage equality. The strategy gave backers strong talking points they could repeat in their own words, multiplying the effectiveness of the campaign’s direct appeals to the broader public.
Shenna’s fundamental idea, one adopted by few on the left (though many on the far right), is that elections can be won by the power of human connection and fidelity to one’s ideals. (And integrity. Having spent years working for marriage equality, she refused to get married until all Maine couples could; her September 2013 emailed wedding announcement ended with a request: “And please… no gifts. For real,” with a link to the Federal Election Commission’s campaign-finance rules.)
And she believes that beyond being funded by small, individual donations for regular-person candidates (which Tea Party candidates espoused), campaigns should be staffed by actual humans, who have real lives and families amidst the fray.
Hearing Shenna talk about both unseating Maine’s senior senator and upending a broken electoral system is strange not because it’s so divergent from cynical national political punditry, nor because critics might call it naïve, but because it’s oddly empowering. It sounds rational, reasonable, possible. It’s an idea whose time, like Shenna’s, may at long last have come.
The execution of Shenna’s impossibly possible candidacy is being shepherded by campaign manager Katie Mae Simpson ’02, a native of rural Washington County, Maine. She’s the type of person for whom political science was too philosophical a major; religion was not.
A lifelong progressive activist and campaigner, Katie Mae was on maternity leave with her second child when Shenna came to her house to ask her to run the campaign. She declined; an all-consuming job running a campaign for U.S. Senate was not compatible with meeting the needs of her young family.
So Shenna went to work employing those skills of connection and persuasion that would go on to make her such an attractive candidate. She told Katie Mae that she wanted her to embody the campaign’s beliefs, to be the standard bearer for a candidacy based on fairness and equality and concern for the everyday Mainer. (To that end, all Shenna’s campaign workers are paid above minimum wage and have health-insurance benefits.)
“The pop culture picture you get of politics is not right for 99 percent of people,” Katie Mae says. So Shenna’s campaign manager (yes, she was quickly persuaded to join the campaign) has set out to reclaim it for the rest of us, which can include breastfeeding her son while conducting an interview. Two other campaign staffers are working mothers whose partners also work full time. Striking a balance is important—teammates who are exhausted from the spring’s battles are of little use come fall’s crunch time.
The same is true of the candidate herself. Her husband, Brandon Baldwin ’98, initially shielded himself from the campaign, hoping to create a refuge where Shenna could escape from the 12-plus-hour-days, seven-days-a-week effort. It wasn’t that he didn’t want Shenna to run— quite the opposite. He’s been supportive of her ambitions ever since she revealed on their fourth or fifth date in 2009 that she was going to run for U.S. Senate some day, possibly soon.
A watershed moment came in February 2012. With the announcement that Olympia Snowe would not run for a fourth Senate term that fall, Maine’s political circles went into a frenzy, handicapping potential successors to the state’s senior senator.
Shenna took a week of unpaid leave from the nonpartisan ACLU to explore her options. But then former independent governor Angus King joined the race (ultimately winning Snowe’s seat handily). Shenna went back to the ACLU, but with a new fire inside.
What surprised her husband about that week was how enthusiastic he felt about the prospect of her being a candidate. It was a powerful realization. “It didn’t feel like it was taking you away from me,” he tells her during a joint interview.
Though removed from the campaign at first, Brandon has slowly become more involved. “It’s tapped into everything that I hold to be important,” he says, admitting that her candidacy has awakened his “somewhat dormant love of politics.” He spoke on Shenna’s behalf at caucuses and house parties, where he would write “campaign spouse” on his nametag sticker. Crucially, he has found himself able to disengage, too. So has she.
“We take one day a month that is our day,” Shenna says. They hike, read, watch movies, sleep in, relax. It’s a private—and vital—aspect of the campaign they both say.
During this joint interview, Shenna suddenly stands up and walks to the fridge, retrieving a card that has been hanging there since she started campaigning. On the front is an inspirational quote: “Be the change you wish to see in the world.” It is a note from Shenna to herself.
Brandon is surprised: He has seen the card daily for months, without realizing there was a message inside. Shenna wrote herself resolutions as she began the campaign. Highlights include “have fun,” “be your authentic self,” and, finally, what Shenna declares as “the most important rule of this race: ‘Love matters most.’”

Love is a strange value to want to bring to Washington, D.C. Congressman Mike Michaud, a Democrat now running to be Maine’s next governor, says that Shenna would do well in the nation’s capital, working hard to forge connections in spite of the heightened partisanship that has infected the city.
Shenna says that she’d bring activist organizing tactics to Congress, meeting with lawmakers as a colleague, while facilitating conversations between legislators and regular citizens. She wants other legislators to hear about the mother Shenna met who is a student and has a full-time job, but still can’t cover all of her bills. “She eats one meal a day so her son can eat three,” Shenna says, tearing up.
At a recent political rally, a woman told Shenna her house had just been foreclosed upon. And there she was, offering to volunteer for Shenna’s campaign, hoping for change. “There are people who are persevering in really horrible situations,” Shenna says. “That is humbling.”
Shenna’s personal touch goes a long way. On an evening in Lewiston, in a room full of Democratic supporters, Shenna makes sure to talk to each one, working the room with a smile, a ready hug for friends, and a laugh audible across the gathering. At one point she leaps with joy and shouts “Yay!” The next second, she regains composure and says soberly, “I’m sorry. That wasn’t very senatorial.” But she is beaming: She has just learned that she was endorsed by the Maine People’s Alliance, a statewide progressive grassroots organization.
Her support is broader, though. Chuck Quintero, the director of the Maine Democratic Party’s coordinated statewide campaign, says of Shenna’s appeal across the political spectrum: “She kind of puts a wrench in our partisan politics. . . .We have to reach out to lots of people we might not otherwise.”
Just hours before Quintero’s half-complaint, some party faithful were lamenting—to Shenna’s face—her insistence on meeting with people she often disagrees with. Shenna had begun the day sparring amiably with popular Maine conservative Ray Richardson on his talk-radio show. Long a fan of Shenna’s, particularly on privacy and limited-government issues, he has announced on the air that he is not voting for Collins. (Cagily, he hasn’t endorsed Shenna—yet.)
Alienation and division get in the way of the important work she wants to do, Shenna says. When she learned that independent U.S. Senator Angus King was about to endorse Collins, Shenna was gracious, but worked to defuse any partisan angst. Moving quickly, she set up interviews on early morning TV and radio programs, so viewers and listeners would hear the news from her. It defused a problem political commentators might have harped on for months.
While still firmly in underdog territory, Shenna’s campaign is on solid footing. She outraised Collins in the first quarter of the race and has already hit $1 million in total fundraising, with the vast majority of that money coming from Maine residents giving under $100.
It’s late May, and Shenna and I are heading south on I-295 when we spot a “Shenna Bellows – U.S. Senate” bumper sticker on a car ahead of us.  As we pass it, she looks carefully at the driver and exults, half-kidding: “And I don’t even know him!” The driver must be a big fan: Our rear-view mirror shows a Bellows sticker on the front bumper too.

Though Shenna is still a relative unknown, those who know her like her—indeed, many Mainers agree with her positions on key issues, and, perhaps more important, disagree with Collins. Tasked with becoming better known every day, she is giving a nod to a state political tradition of sorts: She has planned a month-long, 350-mile walk across Maine to draw media attention and connect more directly with all stripes of voters.
Being the underdog energizes her. “The only way to win is to outwork my opponent. The only way to do that is to organize and inspire,” she says.
Brandon chimes in: “Then if you win, it’s an endorsement” of that approach by the voters. “It’s the best possible way to win.”
It is a winning strategy, whether Shenna beats Collins or not, says Geoffrey Skelley, associate editor of Sabato’s Crystal Ball, an election analysis website at the University of Virginia’s Center for Politics that calls Collins’s seat safe.  Skelley points out in an email that though Maine leans Democratic at the federal level and despite Republicans’ national image problems, Collins’s reelection margins have been high. “In a midterm cycle with a Democrat in the White House, Republicans are naturally positioned to do better,” he says. “About the only way to change that would be for a scandal to develop that centrally involves Collins.”
Nevertheless, Skelley sees a silver lining for Shenna. “While she may deny this, a failed Senate run against a powerful politician like Collins may serve as a stepping stone for her political future,” he says. “If Bellows acquits herself well enough in a difficult race where she stands little chance to win, she might get another electoral shot in the future in more favorable circumstances.”
For the long term, Shenna believes her campaign is on “the right side of history” on the crucial issues facing the nation and the world. But the short term is here. And she is hoping two parallels in Maine’s political history are on her side. In 1964, the unsinkable Republican senator Margaret Chase Smith was beaten by Democrat Bill Hathaway. And more recently, Angus King entered the governor’s race as a relative unknown, and beat, yes, Susan Collins.
“A lot can happen between June and November,” Shenna says.

Wednesday, April 9, 2014

Making clinical trials more diverse

Published in Drug Discovery News

WASHINGTON, D.C.—Hoping to expand both statistical validity of clinical trials and access to experimental therapies beyond their current confines, major medical-industry players have united to launch a campaign called “I’m In,” encouraging minorities and their doctors to find out about, and participate in, clinical medical-research trials.
While encouraging African Americans, Asian Americans, Hispanics and members of other ethnic groups to join medical research, speakers at a March 12 press event announcing the initiative were cognizant of the shadow of the Tuskegee syphilis experiments, an unethical and long-condemned study of the spread of untreated syphilis in African-American men that ran from the 1930s to the 1970s.
While Dr. Carlos Cardenas, board chairman of Doctors Hospital at Renaissance in Edinburg, Texas, alluded more vaguely to the idea of “removing any and all stigma that is associated with being part of a clinical trial,” another speaker, Averl Anderson, a breast-cancer survivor and participant in clinical trials, mentioned Tuskegee by name as a reason “people have a lot of mistrust in medical research”—particularly in the African-American community, she said.
But they and others representing a range of partners in the new campaign stressed the importance of broadening participation.
“These breakthroughs do not happen on their own,” said John Castellani, president and CEO of Pharmaceutical Research and Manufacturers of America, but rather rely “very heavily on volunteer participation in clinical trials.”
Years of work by the pharmaceutical industry to increase diversity in trials have not borne the hoped-for fruit: “African Americans, Asian Americans, and Hispanics are still dramatically underrepresented in clinical trials,” he said.
As Castellani said, “the future of medicines is going to be aimed more and more at genetically homogeneous populations,” which means drawing more from minority populations to properly test drugs—especially those aimed at diseases those populations suffer from disproportionately.
For example, prostate cancer is twice as fatal for African Americans as Caucasians, according to material distributed in advance of the campaign kickoff—and yet only 4 percent of prostate-cancer clinical trial participants are African American. Cancer is the top cause of death for Asian Americans, but just 2.8 percent of cancer trials patients are Asian American. And despite the higher prevalence of diabetes in the Hispanic population, only 1 percent of all trial members are Hispanic.
Based on early glimpses, the campaign is centering on personal connections to family members and the wider community of each minority group. A video promoting the effort included lines like “It’s not enough to wait for someone else to act” and “We all have a responsibility to each other and future generations.”
Gary Puckrein, president and CEO of the National Minority Quality Forum, noted that by 2020, more than half of Americans will be members of groups now called “minorities,” leading him to argue that “underrepresentation of minorities affects everyone.”
He said this is a campaign to help test “medicines for a biodiverse America.”
Cardenas offered an example: When he started practicing medicine, there was just one medication for hypertension; he noticed, though, that it didn’t work the same in people with Hispanic backgrounds as it did in those with non-Hispanic heritage.
“How are we to know how our patients will respond to these medications?” he asked. “It’s something we should not leave to chance.”
Anderson, for her part, said she is a five-year breast-cancer survivor. She was diagnosed in 2009 with stage three, triple-negative cancer, “a very aggressive form of breast cancer that’s common in African-American women.” Her doctor suggested she participate in a trial, which she credits with her survival.
That’s another key element beyond encouraging individuals to participate, said Dr. Ho Luong Tran, founding president and CEO of the National Council of Asian Pacific Islander Physicians.
“As physicians we must recognize our role as trusted healthcare providers,” she said. Doctors “owe it” to their patients to “share all possibilities” for treatment, including clinical trials. She said more than two-thirds of Americans report being likely to join a clinical trial if their doctor suggests it, but only 22 percent of people say they have had such a conversation.
Citing statistics that 38 percent of Hispanics, 36 percent of Asian Americans, 33 percent of African Americans and 42 percent of non-Hispanic whites say doctors have the greatest impact and the greatest responsibility to talk about trials and research, Tran called on her fellow doctors to learn about, support and encourage their patients to join trials. “If we don’t talk to them about clinical research, few others will,” she said.
Puckrein said the website for I’m In, at www.JoinImIn.org has options to register as a member of the public, an interested doctor, and even as a trial researcher, to allow all three groups to connect with each other more efficiently.

Merck HIV therapies make big news at CROI

Published in Drug Discovery News

NEW YORK—Two separate HIV therapies manufactured by Merck, one new and the other a groundbreaking favorite from the past, had big announcements at the 21st Conference on Retroviruses and Opportunistic Infections in early March.
It was just a peek at the newcomer, doravirine (MK-1439), looking at data from the first 24 weeks of the first part of a two-segment 96-week trial comparing doravirine to the current standard of care, efavirenz (Bristol-Myers Squibb’s Sustiva).
“This study is really the first data we have,” apart from a very small, very short monotherapy study, said Dr. Hedy Teppler, executive director, infectious diseases at Merck Clinical Research, who was also an investigator on the trial.
This was a Phase 2 trial (Phase 3 will come later in 2014), in which a range of once-daily doses, 25 milligrams, 50 mg, 100 mg and 200 mg, were used in combination with once-daily doses of emtricitabine/tenofovir disoproxil fumarate, Gilead Sciences’ Truvada.
“It certainly performed at least as well as efavirenz,” she tells DDNews. Merck is hoping doravirine will be better in terms of both tolerability and potency that existing drugs, and with less central nervous system toxicity.
“Each dose compared well,” according to Teppler—in fact, the data didn’t distinguish one dosage as being more effective than the others. Additional information, though, led to the choice of 100 milligrams to be the dose for the remainder of the 96-week trial and other further studies.
Teppler says those factors included providing a reasonable dosage level to discover potential negative drug interactions, and to explore the drug’s overall usefulness.
“Choosing the 100-milligram dose should protect against needing to do any dosage adjustment,” Teppler says.
For those not candidates for efavirenz (such as women of child-bearing age or people with central nervous system issues), another study offers hope. Merck’s drug Isentress (raltegravir), the first integrase inhibitor approved by the FDA (in 2007), featured in a new study by the AIDS Clinical Trials Group. It compared Isentress given twice daily against atazanavir (Bristol-Myers Squibb’s Reyataz) and darunavir (Tibotec’s Prezista), both given once daily. (All patients also got Truvada daily.)
While Merck itself had looked at Isentress versus efavirenz, and found positive results, the ACTG “felt there was a need to look at options in patients who were not candidates for efavarinz,” said Randi Leavitt, senior director, clinical research, global clinical development—infectious disease at Merck, such as women of childbearing age or people with central nervous system issues.
The 96-week ACTG study (ACTG 5257) looked at 1,800 treatment-naive patients, a quarter of whom were women, 34 percent non-Hispanic white, 42 percent non-Hispanic black and 22 percent Hispanic.
The study found encouraging news for Merck, Leavitt tells DDNews. First, “all three regimens were basically equivalent with regard to efficacy.” But when combining that result with tolerability, “raltegravir was superior to the other two regimens.”
And while Isentress has been criticized for being a twice-daily medicine up against once-daily competitors, the results showed that “if the drugs are well tolerated, people will be compliant with a twice-a-day regimen,” Leavitt said.
Nevertheless, the company is working on a reformulation to make a once-daily 1,200-mg dose available. That follows on the 2011 report of the failure of tests of an 800-mg daily dose; Leavitt said those results “provided a lot of information” that have improved expectations for the new formulation.

Smash and grab

Published in Drug Discovery News

LA JOLLA, Calif.—Scripps Research Institute scientists have devised two highly specific methods to create new drugs, one that flings a single atom as a wrecking ball and another that can find therapy targets in tiny folds of microRNA.
A paper published in Nature in March describes research by Scripps chemistry professor Jin-Quan Yu that builds on his earlier groundbreaking development of using a weak chemical bond in molecule frameworks previously thought to be an obstacle, instead turning it into a powerful advantage when building drug compounds.
To attach function groups to chemical frameworks, a C-H bond must be broken—and it must be a specific, and possibly different, one for each potential intended attachment option.
“The best way to make a molecule is to replace a C-H bond directly,” Yu tells DDNews.
Some C-H bonds, though, don’t react, and are far away from attachment points of potential catalysts, rendering them difficult to break. With previous methods, “you cannot make certain types of molecules,” Yu says.
Yu’s insight, which he has been developing since 2002 at the University of Cambridge, was that he could install nitrile groups—weak connections that were dismissed in the past as hurting the structure of a framework—and use that weakness to facilitate the swinging of a catalyst across the molecular distance to a remote C-H bond, allowing it to be broken.
At that point, just as with other broken C-H bonds, functional groups that are building blocks for drugs can be attached, Yu said.
He originally published about the technique for what is called “meta” C-H activation inNature in 2012; the most recent paper has both simplified the process and allowed it to be applied more specifically to targeted C-H bonds.
“The key is to tune the shape of the template to create a subtle bias towards the targeted carbon hydrogen bond,” Yu said in Scripps’s announcement of the paper. “At the same time the template’s movement towards the target site has to be exploited effectively by a super-reactive catalyst.”
The chemical reagent involved will be available through Bristol-Myers Squibb’s catalog for order by laboratories, so that other researchers may use it in their own work, including targeting compounds common in drug discovery, such as tetrahydroquinoline, benzooxazines, anilines, benzylamines, 2-phenylpyrrolidines and 2-phenylpiperidines. “All these are commonly used in medicinal chemistry either as final drug compounds or intermediate compounds from which the final compounds are made,” Yu notes.
And in the future, he expects to further refine the technique so that the nitrile groups can be used catalytically, rather than needing to be installed and later removed.
The other approach, developed by Matthew Disney at the Scripps Florida campus, also inverts a standard method of searching for binding opportunities, this time in microRNA folds. Where previously researchers had to take RNA structures and do high-throughput screenings to find binding opportunities, Disney has built a database of potential types of bonds between RNAs and small-molecule function groups.
Then, by comparing given RNA sequences—not structures—to the database, Disney’s method can pinpoint possible opportunities. Only then does attention turn to the structures themselves, he tells DDNews: “Once we identify these interacting partners, could we find them … and drug it?”
Every disease has a relation to RNA, he said, because proteins play key roles in the process.
“If there’s some toxic protein … we can potentially target the RNA that makes that protein,” Disney says. (Alternately, if a disease causes too little of a protein to be produced, his technique can boost production.)
As a test case, and proof of concept, Disney and his team identified a druggable target—and its corresponding drug—in MiR-96 microRNA, which is believed to delay cell death by obstructing apoptosis, a natural cell-death process that begins when cells begin to grow in ways that are otherwise uncontrollable.
“People think that RNA can’t be drugged with a small molecule,” Disney says, but his approach proves that belief wrong. And it offers the prospect of very tightly targeting cells, in a way much narrower than the broad targeting approach taken today, where non-disease-related cells are also affected by therapies.
Next, Disney will go after diseases without current cures, such as Ebola, as well as orphan diseases that may need therapeutic-research attention.
While resistance to microRNA-targeting drugs is possible, Disney said his approach would help respond: “If resistance were to happen, and the RNA structure were to change,” then they could go back to the database and find binding matches for the new structure, he says.