Every third Friday last fall, Brandie Sellers passed most of her day in a plush leather recliner. She reads, writes or talks to a friend — anything to distract her from the intravenous line hooked to a port under the thin skin at the base of her neck.
Sellers, 38, is in the “chemo room” at her oncologist’s office in McKinney receiving a six-hour treatment for breast cancer. The cancer is stage three, meaning that, although it hasn’t spread, much of the breast and lymph nodes, or skin, is involved.
Most of the drugs in her chemotherapy cocktail are typical and more than a decade old. Delivered one at a time, the treatment usually begins with Benadryl and moves to the cutting-edge anti-nausea medicine approved in 2003. There’s also one drug discovered in the 1950s and a couple more from the mid-1990s, all of which nauseate her. Even the smell of latex gloves, which she associates with the treatment, makes her gag.
Last August, when her doctors attempted to soften the idea of a mastectomy by explaining her insurance would cover breast reconstruction, Sellers remained stoic.
“I think they’ve refined the drugs they’re using, but they’ve been doing mastectomies for a really long time,” she says. “They package it all nicely … but you’re amputating body parts, for God’s sake.”
Over the last decade, the Food and Drug Administration has approved an average of 23 new drugs per year. During the five years prior, they approved an average of 37 new drugs per year. A National Institutes of Health report released last summer said the average time for a new drug to travel from a researcher’s petri dish to a patient’s pill cup or port line is about 13 years.
This protracted approval process is called the “valley of death” in medical circles.
Responding to the dwindling number of new drug approvals, the Patient Protection and Affordable Care Act created the Cures Acceleration Network. And, in 2010, the NIH proposed a plan to reshape how research breakthroughs become medicine by creating the National Center for Advancing Translational Sciences.
The center and the network are charged with accelerating the development of new drugs by coordinating the work of academic scientists and the pharmaceutical companies doing the clinical trials.
Amid tense debates within in the U.S. House of Representatives on how to best manage the 2012 budget cuts, both the new center and the network within the National Institutes of Health became official in late December. President Obama approved a 1 percent increase in the 2012 budget for the NIH, with $576.5 million allocated to the network — less than the $723 million requested. In the 2013 budget released earlier this month by the Obama administration, the NIH budget is proposed to remain flat at about $31 billion. However, the new center’s budget would increase 11 percent, with an jump from $10 million to $50 million next year in the network.
Less than a half-hour drive down the freeway from Sellers’ oncology treatments, researchers at the Cancer Immunology Center in Dallas search for a cure for cancer. Despite the physical proximity, Sellers and other chemotherapy patients are more than a decade displaced from the work of researchers down the road.
As professor and researcher, Dr. Ellen Vitetta, the immunology center’s director, has worked to discover new treatments for an array of incurable diseases for more than three decades. In 2000, after more than a decade of research to genetically engineer and modify a toxin to target tumor cells, her team realized the cancer research had brought them to the doorstep of a vaccine for something else — ricin, an extremely toxic protein found in the castor bean.
While continuing the tumor-targeting research, Vitetta’s team hoped government interest in the vaccine would lead to an increase in grant money that could be applied toward the lab’s primary goal of cancer research. For a while, it did.
But after 12 years, numerous papers and two clinical trials, both the vaccine and the cancer treatments that inspired it sit on freezer shelves in the Dallas lab — a sort of purgatory for promising research that falls out of favor, Vitetta says.
“If I can’t get money for [the research] and I can’t get money for the trials, there’s no choice but to hang it up and let it sit until it comes back into vogue again,” she says.
In the freezer room at Vitetta’s lab, half a dozen 6-foot-tall freezers sit side by side, housing decades of unfunded work at 80 degrees below zero, hoping at some point in the future to be resuscitated. Some drugs, like the poison vaccine, show promise in clinical trials but become victims of federal funding cuts that leave the center no choice but to consign them to the freezer.
For Vitetta, the force that curbs the flow of new drugs to market is money. The percentage of researchers approved for first-time research grants from the NIH decreased from 19.5 percent in 2002 to 9.5 percent last year, according to the agency’s website.
“It’s more than that because a lot of those investigators who could routinely run a lab at 20 percent funding have been thrown out of the pool now, and so the pool is much more competitive,” Vitetta says. “We’re down to the people who are the top in the field all competing for those nine grants out of a hundred.”
The implications of such a competitive field may do more than cause seasoned research veterans like Vitetta to search the high ground for funding, says Dr. Stephen Katz, a former chairman and current member of the NIH Scientific Management Review Board.
“The thing that wakes me up at night is, what happens to that assistant professor who has such talent,” Katz told the group in a recent presentation, “in whom we have invested in order to get them trained to be the next leader in science, the next Nobel Prize winner, and who is having trouble getting started?”
Teaching old drugs new tricks
With money hard to come by, researchers can turn to privately funded charity organizations like the Leukemia and Lymphoma Society, which funds research for rare blood cancers. The society’s chief mission officer, Louis DeGennaro, says many venture capitalists are reluctant to invest in pharmaceuticals whose markets are small, whose margins are thin or whose expired patents no longer protect a profit.
The new National Center for Advancing Translational Sciences seeks to reverse that trend by conducting tests of old drugs and research-abandoned drugs for their effectiveness against diseases they were not originally intended to treat, according to Kathy Hudson, the center’ acting deputy director.
“They [pharmaceutical companies] could provide their abandoned compounds and we could provide the neuronal energy … of all our scientists to basically crowd-source their compounds and find new indications,” Hudson says.
Only a few venture capitalists have journeyed into resuscitating off-patent pharmaceuticals for this purpose. But some of the new drugs that have grown out of repurposing old ones have proven successful. In the 1990s, the Leukemia and Lymphoma Society helped fund clinical studies on Imatinib, which initially had failed to treat prostate cancer. But years later, when another researcher tested the drug for treating chronic myeloid leukemia, it proved effective.
Patients “would have an expected lifespan of three to six years with a disease where there really were no good treatments,” DeGennaro says. “It’s taken that disease to a situation where 90 percent of the patients diagnosed with this disease are treated with this drug and their cancer goes into remission.”
More recently, the society has worked with NIH to develop a treatment for chronic lymphocytic leukemia.
“They tested this panel of existing generic drugs against [chronic lymphocytic leukemia] and found a drug that seemed to block the growth of samples taken from patients,” DeGennaro says.
Hudson is confident that the National Center for Advancing Translational Sciences and the Cures Acceleration Network, working with their partners ranging from medical researchers to venture capitalists, can exploit previously unexplored uses for old drugs. Part of the reason for that confidence is the new authority provided to NIH by the network that will allow them to spotlight high-need research breakthroughs and escort them through federal regulations for faster approval.
“The emphasis here is on creating new methods, technology and resources that will help everybody in the drug and therapeutics development phase accelerate their work,” Hudson says.
The waiting game
Vitetta and other scientists at the end of the funding chain are skeptical this new approach will accelerate the development of important new drugs to treat presently incurable diseases.
“I’ve heard this my whole career,” she says. “And first of all, you have to agree on the bottlenecks — nobody does. Second, you have to agree on how to fix the bottlenecks — nobody does. I think that having an intramural NIH community and an extramural non-NIH community can be useful, but we’ve never really struck a balance at how to distribute the money between the two sectors.”
There are others, such as former Food and Drug Administration fellow Henry Miller, a scholar at the Hoover Institution at Stanford University, who fear the NIH has overextended its resources.
“It’s just a poorly conceived idea,” Miller says. “[The Institutes of Health] could use some reforms, but that’s not it. I don’t think it will be cost effective and I don’t think it’ll be the wisest use of the federal research fund.”
As Vitetta and others wait to see whether these health care research reforms will reinvigorate the flow of new drugs into the market, Sellers is left to deal with her disease that’s being treated with the old standbys.
“I’m one of those people that’s saying, ‘This doesn’t work,’ but I don’t have a better solution,” Sellers says. “Some other people need to work out how this is going to get paid for, because I have cancer and I need to deal with that.”
IF YOU GO
• What: “Healthcare 101,” a nonpartisan, apolitical seminar on the new health care law
• Who: A panel of speakers will explain the health care reform law in a free event sponsored by the Denton Black and Hispanic chambers of commerce.
• When: 6 to 8 p.m. Tuesday, with a mixer preceding the seminar from 5 to 6 p.m.
• Where: Emily Fowler Central Library, 502 Oakland St., Denton