Developing
life-saving medicine
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.
“The pharmaceutical community 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 unheard of.”
But major scientific advances don’t come without significant investments. 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 drugs developed will enter clinical testing and go on to receive FDA approval.
Nevertheless, science continues to persist and prevail. 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.
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 of cancer research: Going after a target that was long considered “undruggable.”
Undruggable refers to cells – specifically proteins – in the body that play a role in disease and aren’t affected by medications. An estimated 85 percent of proteins are deemed undruggable, with some considered too risky to even pursue.
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 – who have exhausted other options - Lanman and his colleagues set out to develop a therapy targeting a common cancer-causing gene mutation called KRAS.
KRAS is what’s known as an oncogene, which is a mutated form of a protein that causes cells to grow and divide too quickly, and can lead to cancer. The National Cancer Institute says KRAS normally acts “like a light switch toggling between being ‘on’ and ‘off.’” When mutated, it stays “on” more than “off.” For decades, scientists have searched for ways to turn that 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 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. And it’s a lot harder to try to persuade nature to do what we want it to do than to create something wholly human-conceived.”
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 attach to. This discovery allowed them to design a drug that binds to certain types of KRAS proteins and locks them in that “off” position, which can impact tumor growth.
This development offers hope in an area of cancer that desperately needs it. Lung cancer is a leading cause of cancer deaths around the world, accounting for more deaths than colon cancer, breast cancer and prostate cancer, combined.
“As a scientist, I think there can be few things that are as rewarding as seeing some of the work you’ve done bring benefit to patients with a serious disease like cancer,” says Angela Coxon, who is vice president of oncology research at Amgen.
As a scientist, I think there can be few things that are as rewarding as seeing some of the work you’ve done bring benefit to patients with a serious disease like cancer
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 through other parts of the body, the five-year survival rate for people with non-small cell lung cancer is 7 percent. Amgen’s discovery could change that.
Advancements in targeted therapies are also enabling a new generation of cancer treatments. Coxon says it’s exciting to live in this era of biomedical research, with scientists making strides at an extraordinary speed. “We’re now at a point where innovation is moving so fast, at times it’s hard to keep up.”
Breakthroughs, like with KRAS, make the years of research trials and tribulations worth it, and as POLITICO Focus saw firsthand at Amgen’s Thousand Oaks headquarters, motivates the team to get up each day to go after the undruggable and continue to develop new drugs that will bring new hope to cancer patients everywhere.
Learn more about the work Amgen researchers are doing to take on the toughest to treat cancers by visiting Amgen.com.
Sickle Cell Disease
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 of cancer research: Going after a target that was long considered “undruggable.”
Undruggable refers to cells – specifically proteins – in the body that play a role in disease and aren’t affected by medications. An estimated 85 percent of proteins are deemed undruggable, with some considered too risky to even pursue.
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 – who have exhausted other options - Lanman and his colleagues set out to develop a therapy targeting a common cancer-causing gene mutation called KRAS.
KRAS is what’s known as an oncogene, which is a mutated form of a protein that causes cells to grow and divide too quickly, and can lead to cancer. The National Cancer Institute says KRAS normally acts “like a light switch toggling between being ‘on’ and ‘off.’” When mutated, it stays “on” more than “off.” For decades, scientists have searched for ways to turn that 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 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. And it’s a lot harder to try to persuade nature to do what we want it to do than to create something wholly human-conceived.”
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 attach to. This discovery allowed them to design a drug that binds to certain types of KRAS proteins and locks them in that “off” position, which can impact tumor growth.
This development offers hope in an area of cancer that desperately needs it. Lung cancer is a leading cause of cancer deaths around the world, accounting for more deaths than colon cancer, breast cancer and prostate cancer, combined.
“As a scientist, I think there can be few things that are as rewarding as seeing some of the work you’ve done bring benefit to patients with a serious disease like cancer,” says Angela Coxon, who is vice president of oncology research at Amgen.
As a scientist, I think there can be few things that are as rewarding as seeing some of the work you’ve done bring benefit to patients with a serious disease like cancer
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 through other parts of the body, the five-year survival rate for people with non-small cell lung cancer is 7 percent. Amgen’s discovery could change that.
Advancements in targeted therapies are also enabling a new generation of cancer treatments. Coxon says it’s exciting to live in this era of biomedical research, with scientists making strides at an extraordinary speed. “We’re now at a point where innovation is moving so fast, at times it’s hard to keep up.”
Breakthroughs, like with KRAS, make the years of research trials and tribulations worth it, and as POLITICO Focus saw firsthand at Amgen’s Thousand Oaks headquarters, motivates the team to get up each day to go after the undruggable and continue to develop new drugs that will bring new hope to cancer patients everywhere.
Learn more about the work Amgen researchers are doing to take on the toughest to treat cancers by visiting Amgen.com.
Epilepsy
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 of cancer research: Going after a target that was long considered “undruggable.”
Undruggable refers to cells – specifically proteins – in the body that play a role in disease and aren’t affected by medications. An estimated 85 percent of proteins are deemed undruggable, with some considered too risky to even pursue.
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 – who have exhausted other options - Lanman and his colleagues set out to develop a therapy targeting a common cancer-causing gene mutation called KRAS.
KRAS is what’s known as an oncogene, which is a mutated form of a protein that causes cells to grow and divide too quickly, and can lead to cancer. The National Cancer Institute says KRAS normally acts “like a light switch toggling between being ‘on’ and ‘off.’” When mutated, it stays “on” more than “off.” For decades, scientists have searched for ways to turn that 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 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. And it’s a lot harder to try to persuade nature to do what we want it to do than to create something wholly human-conceived.”
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 attach to. This discovery allowed them to design a drug that binds to certain types of KRAS proteins and locks them in that “off” position, which can impact tumor growth.
This development offers hope in an area of cancer that desperately needs it. Lung cancer is a leading cause of cancer deaths around the world, accounting for more deaths than colon cancer, breast cancer and prostate cancer, combined.
“As a scientist, I think there can be few things that are as rewarding as seeing some of the work you’ve done bring benefit to patients with a serious disease like cancer,” says Angela Coxon, who is vice president of oncology research at Amgen.
As a scientist, I think there can be few things that are as rewarding as seeing some of the work you’ve done bring benefit to patients with a serious disease like cancer
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 through other parts of the body, the five-year survival rate for people with non-small cell lung cancer is 7 percent. Amgen’s discovery could change that.
Advancements in targeted therapies are also enabling a new generation of cancer treatments. Coxon says it’s exciting to live in this era of biomedical research, with scientists making strides at an extraordinary speed. “We’re now at a point where innovation is moving so fast, at times it’s hard to keep up.”
Breakthroughs, like with KRAS, make the years of research trials and tribulations worth it, and as POLITICO Focus saw firsthand at Amgen’s Thousand Oaks headquarters, motivates the team to get up each day to go after the undruggable and continue to develop new drugs that will bring new hope to cancer patients everywhere.
Learn more about the work Amgen researchers are doing to take on the toughest to treat cancers by visiting Amgen.com.
