[Ip-health] At a UMass lab, a eureka moment

Alicia Mundy amundy at aliciamundy.com
Tue Jan 2 11:55:12 PST 2018

This is one more awful story about greed and the rejection of the concept of Public Health -- great read~

> On January 3, 2018 at 1:07 AM Kim Treanor <kim.treanor at keionline.org> wrote:
> https://www.bostonglobe.com/business/2017/12/16/spinrazasidecopy/CgWVLcXzZNI3b8nPAyWzHL/story.html
> At a UMass lab, a eureka moment
> Jonathan Saltzman and Robert Weisman in the Boston Globe on 17 December 2017
> An experimental drug is prepared for analysis at Alnylam Pharmaceuticals in
> Cambridge.
> By Jonathan Saltzman and Robert Weisman GLOBE STAFF DECEMBER 17, 2017
> Biogen Inc. bought the rights to Spinraza, guided it through final clinical
> trials, and then took it to market at a stunning price.
> But the Cambridge biotech giant didn’t invent it.
> That creation story began in a lab at the University of Massachusetts
> Medical School in Worcester, where scientist Ravindra Singh was trying to
> crack a genetic puzzle. It was 2004 and his tiny lab team of four — he, his
> wife, a postdoctoral student, and another collaborator — had spent three
> years methodically isolating and analyzing molecules in search of a way to
> attack the source of spinal muscular atrophy.
> Simply put — though there is really no way to put it simply — SMA occurs
> when the spinal cord has too little of a vital protein that controls muscle
> movement. Two genes — SMN1 and a nearly identical backup, SMN2 — make this
> protein in healthy people. But those with SMA have a mutation that deletes
> the first gene entirely. Their backup gene doesn’t produce enough protein
> to compensate. They eventually lose the ability to walk, eat, and,
> ultimately, breathe.
> Singh’s focus was on isolating the exact sequence of genetic code that sets
> off this deadly cascade. He and his team screened about 200 potential drug
> targets before their eureka moment.
> The culprit, they found, was a genetic sequence called ISS-N1 that prevents
> the backup gene from making more protein.
> Other labs could now get to work finding therapeutic compounds to block the
> devastating sequence. Thanks to the UMass scientists, they knew where to
> aim.
> Singh, who now operates a lab at Iowa State University, said he designed
> the experiment that identified the sequence ISS-N1, but had no idea that it
> would yield the desired result. “We were completely dumbfounded,” he said,
> with their findings in the lab one November night.
> “We were so excited, so happy,” he said, recalling the euphoria he and his
> wife, Natalia, felt. At home later that night, “we drank some wine,” he
> said.
> UMass licensed their discovery to a Carlsbad, Calif., biotech firm called
> Isis Pharmaceuticals. The deal gives UMass and Singh a total of 2 percent
> of net US sales of any resulting drug, according to Jennifer Berryman, a
> university spokeswoman. That’s potentially tens of millions of dollars. Of
> that sum, UMass will get a little over two-thirds, and Singh almost
> one-third.
> Isis — now known as Ionis — then built on the UMass work by developing a
> compound, with help from the Cold Spring Harbor Laboratory in New York,
> that could bind to and block ISS-N1.
> At last there was a potential treatment for SMA — if it would hold up
> through years of exhaustive clinical trials.
> Biogen, working with Ionis, helped bankroll the experimental drug and
> shepherd it through the approval process. Last year the firm spent $75
> million to buy exclusive worldwide rights to the drug, which won FDA
> approval a year ago this week, and gave it a new brand name: Spinraza.
> It also fell to Biogen to put a price on this prized new product — $750,000
> for each patient the first year and $375,000 every year after — and therein
> lies a tale that company executives are reluctant to discuss in detail.
> The costs of developing a treatment like Spinraza are obviously huge, but
> how exactly those costs fit into the market decision on price is unclear.
> Michel Vounatsos, who became chief executive in January after joining
> Biogen in April 2016 as a high-ranking executive, recently told the Globe
> that the company had invested “close to a billion” in developing the drug.
> But he declined to provide a breakdown.
> The lack of detail provided by Biogen has led others to try to piece
> together the drug-pricing process.
> Knowledge Ecology International, a Washington-based advocacy group that
> wants more openness in drug pricing, calculated the cost for Biogen and
> Ionis to run 10 clinical studies for Spinraza involving several hundred
> patients from 2011 to 2016. It concluded that the drug is wildly overpriced.
> After factoring in federal tax credits that the drug makers received for
> developing an orphan disease drug — plus financial risks they took — KEI
> came up with a total of $35 million for the cost of clinical trials,
> according to its analysis.
> That’s a fraction of the roughly $2.6 billion often cited by the
> pharmaceutical industry to develop a drug and take it through clinical
> trials to FDA approval.
> “The price [of Spinraza] has absolutely no relationship to what it cost to
> develop,’’ said James Love, director of KEI. “People are getting ripped off
> on Spinraza big time.”
> Vounatsos said he respects the work of KEI but its estimate of costs is
> “grossly” inaccurate. Furthermore, he said, Biogen based Spinraza’s price
> mostly on the fact that it was the first treatment to offer hope to
> desperately sick people with SMA, not how much it cost to develop.
> Biotech executives say a variety of factors go into the price of a new
> drug, including the value it brings to the health care system — a somewhat
> hazy concept — the cost of research and development, the price of
> comparable medicines, and the likelihood that other experimental drugs in
> the company’s pipeline will go bust. That last one is a grim reality of the
> drug business; less than one in 10 drugs that enter clinical trials
> ultimately make it to market, according to a 2016 study by the
> Biotechnology Innovation Organization, a national trade group.
> “There’s no industry that’s as capital-intensive, that has as much risk,”
> said John Maraganore, chief executive of Alnylam Pharmaceuticals Inc., a
> Cambridge biotech. Alnylam is working on therapies for orphan diseases
> ranging from hemophilia to acute hepatic porphyria, a rare metabolic
> disorder that some scholars theorized might have afflicted England’s King
> George III in the late 1700s.
> To illustrate the costs and risks, Maraganore cites his own company. He
> says Alnylam has invested $2 billion in drug development since it was
> founded in 2002. It has amassed a market value of roughly $12 billion based
> on enthusiasm over its experimental gene-silencing drugs, which rely on a
> technique called RNA interference to shut down dysfunctional genes.
> But in its 15 years of existence, the company has yet to sell a single
> commercial product, and Maraganore says profitability remains years away.
> Critics counter that the breathtaking prices reflect the fact that the
> government lets pharmaceutical companies set whatever price they want and
> gives them years of exclusive rights when marketing drugs for rare
> diseases, effectively creating a monopoly. As a result, they say, leaders
> of some drug companies try to make as much money as quickly as possible, in
> the hopes of boosting stock prices — and their own pay packages.
> “The industry is more and more just an arm of Wall Street,” said John
> Rother, head of the Campaign for Sustainable Rx Pricing, a nonpartisan
> coalition in Washington, D.C. “There’s nothing that restrains them from
> setting the price without regard to the impact on patients and the health
> care system in general.”
> And the potential impact of stratospherically priced orphan drugs on the
> health care system is staggering.
> The average price of a medicine for an orphan disease is nearly $119,000 a
> year, Dr. A. Gordon Smith, chief of neuromuscular medicine at the
> University of Utah School of Medicine, wrote in an April Harvard Business
> Review article on Spinraza.
> If a single drug was approved for that much for just 10 percent of the
> 7,000 rare diseases for which there now are no treatments, Smith wrote, the
> total would exceed $350 billion annually. That’s far more than the cost of
> care for the millions of people with — to name just two common conditions —
> diabetes or dementia.
> --
> Kim Treanor
> Knowledge Ecology International
> kim.treanor at keionline.org
> tel.: +1.202.332.2670 <(202)%20332-2670>
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