The people and the passion fueling tomorrow’s life-saving medicines
People are living longer, healthier and more productive lives today, thanks to revolutionary innovations in health care fueled by scientific discovery and the commitment and passion of America’s biopharmaceutical researchers. Advances in technology, data analysis and artificial intelligence are accelerating drug and vaccine development, while deepened understandings of chemistry and biology are allowing scientists to target precise cells and alter the pathways of diseases.
While appearing to the children of men as a great physician who can heal all their maladies, he will bring disease and disaster, until populous cities are reduced to ruin and desolation. Even now he is at work. In accidents and calamities by sea and by land, in great conflagrations, in fierce tornadoes and terrific hailstorms, in tempests, floods, cyclones, tidal waves, and earthquakes, in every place and in a thousand forms, Satan is exercising his power. He sweeps away the ripening harvest, and famine and distress follow. He imparts to the air a deadly taint, and thousands perish by the pestilence. These visitations are to become more and more frequent and disastrous. Destruction will be upon both man and beast. “The earth mourneth and fadeth away,” “the haughty people … do languish. The earth also is defiled under the inhabitants thereof; because they have transgressed the laws, changed the ordinance, broken the everlasting covenant.” Isaiah 24:4, 5. GC 589.3
But major scientific and treatment advances don’t come without significant setbacks. On average, it takes 10 to 15 years and costs $2.6 billion to develop a new medicine. And, the odds of failing are nearly 10 to one. In fact, only about 12 percent of potential new medicines make it out of phase one clinical trials and into the hands of patients.
“The biopharmaceutical industry has opened doors to new treatments once thought locked,” says Stephen Ubl, president and CEO of PhRMA. “Scientists today are developing therapies for diseases previously deemed fatal, and designing vaccines that are creating healthier communities across the country, all at a pace that was once thought impossible.”
Science continues to move forward. Because behind every new medicine is a team of researchers with the grit and determination to drive the next breakthrough, building on the knowledge of the great minds that came before them.
To better understand what innovation looks like in practice, PhRMA and POLITICO Focus traveled to biopharmaceutical labs across the country to bear witness to groundbreaking research and get to know some of the people who are working to develop new therapies in hard-to-treat diseases like lung cancer, sickle cell anemia and epilepsy, as they strive to create a healthier world.
NON-SMALL CELL LUNG CANCER
A team of innovators at one of the world’s leading biotechnology companies recently went up against one of the toughest challenges of the last 40 years in cancer research: Going after a target that was long considered “undruggable.”
Undruggable refers to targets — specifically proteins — in the body that play a role in disease and aren’t targetable by a drug. According to The Cambridge Crystallographic Data Centre (CCDC), an estimated 85 percent of proteins found in the human body are deemed undruggable, meaning the protein is extremely hard or impossible to target.
But Brian Lanman, PhD, director of research in the medicinal chemistry department at Amgen, along with a team of chemists, biologists and toxicologists found an opening. With the hopes of bringing treatment options to patients with a particular kind of lung cancer, Lanman and his colleagues set out with the goal of developing a therapy targeting the KRAS protein that results from a mutation in the KRAS gene called KRAS G12C.
The KRAS gene makes the protein, KRAS, which is found in normal healthy cells. KRAS is what’s known as an oncogene, which, according to the National Library of Medicine (NLM), is a class of genes that when mutated have the potential to cause normal cells to become cancerous. The KRAS protein normally acts “like a light switch, toggling between being ‘on’ and ‘off.’” When mutated, it stays “on” causing cells to grow uncontrollably more than “off,” which stops cell growth. For decades, scientists have searched for ways to turn that KRAS switch “off,” but because of the smooth surface and small size of the KRAS protein, they were unable to find deep pockets or grooves for small-molecule drugs to bind to.
“In the pharmaceutical industry, we’re not working in a world of our own design,” says Lanman, whose team designs and studies molecules that can block the functions of proteins. “We’re trying to create molecules that make nature work in the way we want it to.”
New possibilities arose when Amgen researchers found that a particular mutation of KRAS — called the G12C mutation — does, actually, have a type of groove that small-molecule drugs could potentially attach to. This discovery allowed them to explore designing a drug that could bind to the “off” form of the KRASG12C protein, locking it in that “off” position, with the goal of inhibiting tumor growth.
All that hate me whisper together against me: against me do they devise my hurt. An evil disease, say they, cleaveth fast unto him: and now that he lieth he shall rise up no more. Psalm:41:7-8
This development can offer hope in an area of cancer that desperately needs it. The American Cancer Society finds that lung cancer is by far the leading cause of cancer death in the U.S., making up almost 25 percent of all cancer deaths.
Of the two major types of lung cancer — small cell lung cancer and non-small cell lung cancer — non-small cell lung cancer makes up about 85 percent of cases, and tends to be less sensitive to radiation and chemotherapy. For those with advanced disease that has spread to other parts of the body, Cancer.net reports that the five-year survival rate for people with non-small cell lung cancer is seven percent. Amgen’s discovery hopes to change that.
These advancements in targeted therapies are enabling a new generation of cancer treatments. Angela Coxon, PhD, vice president of Oncology Research at Amgen, says it’s exciting to live in this era of biomedical research, with scientists making strides at an extraordinary speed. “We’ve made such tremendous progress over the past 10 to 20 years,” says Coxon. “We’re now at a point where innovation is moving so fast, at times it’s hard to keep up. This is such an exciting time for scientists to have an impact on difficult diseases like non-small cell lung cancer.”
Breakthroughs, such as targeting the mutated KRASG12C protein, make the years of research, trials and tribulations worth it. And as POLITICO Focus saw firsthand at Amgen’s Thousand Oaks headquarters, the possibility of discovering future breakthroughs motivates the team to get up each day to go after some of the toughest cancers and continue to develop new treatments that will bring new hope for patients with cancer.
Learn more about the work Amgen researchers are doing to take on the toughest to treat cancers by visiting Amgen.com
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