Targeted Drugs

Targeted Drugs

Breakthroughs in our understanding of the biology of cancer cells are driving development of a new generation of drugs that are targeted to the unique genetics of each tumor and patient. By blocking or switching off the molecular defects that cause cancer to grow and spread, these agents attack cancer cells, but leave healthy cells largely untouched, resulting in better cancer control and better quality of life.

While targeted drugs are relatively new, some have already become staples of cancer treatment. Recently, targeted drugs have been shown to extend the lives of people with advanced melanoma, long one of the most difficult cancers to treat.

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2017

New chemotherapy regimen improves pancreatic cancer survival

New chemotherapy regimen improves pancreatic cancer survival

A new standard of care for pancreatic cancer treatment is set after data from a clinical trial show that adding a second chemotherapy drug, capecitabine (Xeloda), to gemcitabine (Gemzar) modestly improves survival after surgery. For the first time in 20 years, patients with pancreatic cancer – which is difficult to diagnose and often not found until it’s too late to be removed through surgery – have a new option for treatment after surgery.

2016

Pembrolizumab extends NSCLC survival, with fewer side effects

Pembrolizumab extends NSCLC survival, with fewer side effects

Studies find that pembrolizumab (Keytruda) improves survival and causes fewer side effects than standard chemotherapy for patients with advanced non-small cell lung cancer (NSCLC) and high levels of PD-L1 protein in the tumor. These trials include patients who were newly diagnosed and previously treated with other therapies, leading the FDA to approve the drug for both populations of patients. These approvals establish a new treatment paradigm for this type of lung cancer, with immunotherapy becoming the preferred treatment over chemotherapy regimens for PD-L1-positive cancers. 

First new treatment for bladder cancer in three decades

First new treatment for bladder cancer in three decades

The FDA approves atezolizumab (Tecentriq) for treatment of advanced urothelial carcinoma, the most common form of bladder cancer. With this approval, atezolizumab becomes the first new bladder cancer treatment in 30 years and the first PD-L1 immune checkpoint inhibitor approved for any use. The approval follows an early clinical trial in which atezolizumab rapidly shrank tumors in patients with previously treated advanced urothelial cancer, particularly those with high levels of PD-L1 in the tumor and immune cells.

2015

Immunotherapy helps patients with treatment-resistant Hodgkin lymphoma

Immunotherapy helps patients with treatment-resistant Hodgkin lymphoma

2015-2017

A growing body of research shows that for patients with Hodgkin lymphoma that do not respond to standard treatment, including autologous stem cell transplantation, immunotherapy with PD-1 inhibitors can have significant benefits. Clinical trials suggest that treatment with the PD-1 inhibitors nivolumab (Opdivo) or pembrolizumab (Keytruda) can lead to remissions in patients who had received many previous cancer treatments, leading the FDA to grant accelerated approval to both drugs for this indication in 2016 and 2017, respectively.  

A new class of breast cancer treatment introduced

A new class of breast cancer treatment introduced

2015-2016

Palbociclib (Ibrance) becomes the first in a new class of medicines called cyclin-dependent kinase (CDK) inhibitors, which block key proteins that control cell division. In clinical trials, addition of palbociclib to standard hormone therapy extended the time until the cancer worsened by a median of 11 months when given as initial therapy and by about 5 months for women with previously treated cancer. In 2015, the FDA grants accelerated approval to palbociclib in combination with letrozole (Femara) as initial hormone-based therapy in post-menopausal women with hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced breast cancer. In 2016, the FDA grants accelerated approval to palbociclib with fulvestrant (Faslodex) to treat advanced breast cancer that worsens following initial hormone therapy.  

Nivolumab improves survival and receives FDA approval for kidney cancer

Nivolumab improves survival and receives FDA approval for kidney cancer

The FDA approves nivolumab (Opdivo) for patients with advanced renal cell carcinoma, the most common type of kidney cancer in adults. The approval follows a clinical trial that found patients who received nivolumab lived about five months longer compared to those treated with the previous standard treatment, everolimus (Afinitor). This marks the second FDA-approved therapy proven to extend survival in renal cell cancer, following temsirolimus’ (Torisel) 2007 approval.

Adjuvant ipilimumab lowers recurrence risk for earlier-stage melanoma

Adjuvant ipilimumab lowers recurrence risk for earlier-stage melanoma

A large clinical trial finds that the immunotherapy ipilimumab (Yervoy), when given after surgery, lowers the risk of cancer recurrence and extends survival for patients with stage III melanoma who have a high risk for recurrence. This marks the first study to show the effectiveness of ipilimumab against earlier-stage melanoma (ipilimumab was first approved in 2011 for advanced melanoma). Ipilimumab attaches to a protein on cancer-killing T cells called CTLA-4, which acts as a brake on the immune system. This unleashes the immune system to attack the cancer. 

Three immunotherapies introduced for most common type of lung cancer

Three immunotherapies introduced for most common type of lung cancer

Three new immunotherapies are approved by the FDA for patients with advanced non-small cell lung cancer (NSCLC) that worsened during or following chemotherapy. In clinical trials, the three drugs, nivolumab (Opdivo), atezolizumab (Tecentriq), and pembrolizumab (Keytruda), were each shown to extend survival and cause fewer severe side effects than chemotherapy. These treatments target the so-called immune checkpoint proteins PD-1 and PD-L1, respectively, helping the immune system fight cancer.

2014

New targeted treatment helps people with advanced stomach cancer live longer

New targeted treatment helps people with advanced stomach cancer live longer

The FDA approves ramucirumab (Cyramza), for treatment of advanced cancers of the stomach and gastroesophageal junction. This marked the first FDA-approved treatment for patients with advanced stomach cancer that worsened during or after chemotherapy. The approval follows results from a clinical trial showing that ramucirumab both alone and combined with the chemotherapy paclitaxel (Abraxane) modestly extends patient survival. Anti-angiogenic drugs like ramucirumab block the growth of blood vessels to the tumors.

Four new drugs transform CLL treatment

Four new drugs transform CLL treatment

In a span of eight months, the FDA approves four new treatments for chronic lymphocytic leukemia (CLL), a blood cancer that most commonly develops in older adults. Obinutuzumab (Gazyva) and ofatumumab (Arzerra), approved as initial treatments for CLL, delay cancer worsening by about a year when given with chemotherapy. Targeted drugs idelalisib (Zydelig) and ibrutinib (Imbruvica) were approved for use in patients with CLL that became resistant to or relapsed after standard treatment. In clinical trials, both slowed cancer worsening and ibrutinib also helped patients live longer.  All four new therapies are easier for patients to tolerate than prior therapies, making treatment possible for more patients than ever.  

First targeted therapy combination approved for high-risk melanoma

First targeted therapy combination approved for high-risk melanoma

FDA grants accelerated approval for combined modality therapy with trametinib (Mekinist) and dabrafenib (Tafinlar) in patients with advanced or inoperable melanoma that harbor certain BRAF gene mutations. The approval is based on studies demonstrating that the new regimen leads to long-lasting responses in most patients. The two drugs target different molecules in a pathway involved in melanoma growth. 

FDA approves two immunotherapies for melanoma

FDA approves two immunotherapies for melanoma

FDA grants accelerated approval to pembrolizumab (Keytruda) and nivolumab (Opdivo) for patients with advanced melanoma that cannot be removed with surgery who no longer respond to other treatments.

These therapies, known as “immune checkpoint inhibitors,” work by blocking a pathway called PD-1, that prevents the body’s immune system from attacking cancer cells.

Approval comes following research showing that the therapies shrank tumors in up to 40% of patients. In one clinical trial, nivolumab extended survival compared to targeted therapy for patients with BRAF mutations. 

2012

Two new targeted drugs available
“Armed-antibody” drug improves survival for women with resistant HER2 positive cancers

“Armed-antibody” drug improves survival for women with resistant HER2 positive cancers

A clinical trial shows that a next-generation targeted drug called trastuzumab emtansine (T-DM1;Kadcyla) extends survival in women with HER2-positive breast cancers that progress despite other standard therapies. This drug combines two anticancer drugs – the HER2-targeted drug trastuzumab and the chemotherapy drug emtansine. The drug is designed to bind to the HER2 protein on cancer cells and directly deliver these drugs to the tumor, minimizing adverse effects to healthy tissue in the body.

The FDA approves T-DM1 for breast cancer in 2013.

New treatment option for advanced prostate cancer

New treatment option for advanced prostate cancer

Enzalutamide (XTANDI) is proven to extend survival in men with advanced prostate cancer that persists despite all other standard treatments, including hormone therapy and chemotherapy. The drug is approved later in the year.

Enzalutamide works by blocking testosterone from binding to the androgen (male hormone) receptor, a process that would otherwise help fuel prostate cancer growth.

Two targeted drugs together are more potent than one for HER2-positive breast cancer

Two targeted drugs together are more potent than one for HER2-positive breast cancer

A study shows that initial treatment with two drugs that target the HER2 protein – pertuzumab (Perjeta) and trastuzumab (Herceptin) – together with standard chemotherapy substantially slows cancer growth in women advanced with breast cancer that over-produces the HER2 protein.

The results add new momentum to research exploring the effectiveness of combining two or more drugs that target the same molecular pathways.

2011

FDA approves axitinib for persistent kidney cancer

FDA approves axitinib for persistent kidney cancer

A study finds that axitinib (Inlyta) is more effective than a current standard therapy, sorafenib (Nexavar), for slowing cancer growth in patients with advanced kidney cancer that gets worse despite prior therapy. The FDA approves axitinib in 2012, offering a new treatment for patients with a challenging form of kidney cancer.

Second targeted drug extends survival for advanced melanoma

Second targeted drug extends survival for advanced melanoma

The FDA approves the targeted drug vemurafenib (Zelboraf), based on data showing the drug shrinks tumors and improves survival in advanced melanoma patients whose tumors have a specific mutation in a gene known as BRAF. In many cases, patients experience dramatic reductions in pain and tumor shrinkage within days of starting this therapy. However, research is ongoing to determine why the cancer typically stops responding to this novel drug after several months.

2010

Adding rituximab extends survival for chronic lymphocytic leukemia

Adding rituximab extends survival for chronic lymphocytic leukemia

A large, long-term trial establishes that adding the targeted drug rituximab (Rituxan) to initial treatment with the standard drug fludarabine (Fludara) slows the progression of chronic lymphocytic leukemia, and improves survival. This is the first drug regimen ever found to significantly extend the lives of patients with the disease. The trial found that more than 10 percent of patients treated with this regimen had no disease progression after ten years.

New targeted drug approved for certain advanced prostate cancers

New targeted drug approved for certain advanced prostate cancers

FDA approves abiraterone acetate (Zytiga) in combination with the drug prednisone for patients with advanced prostate cancer whose disease progresses despite prior hormone therapy and standard chemotherapy with docetaxel. Abiraterone blocks the production of male sex hormones, including testosterone, which fuel prostate tumor growth. Since only one other agent, cabazitaxel (Jevtana), has been shown to prolong survival in this population of patients, the treatment represents a much-needed new option.

Bevacizumab significantly delays progression of advanced ovarian cancer

Bevacizumab significantly delays progression of advanced ovarian cancer

A large study finds that adding the targeted drug bevacizumab (Avastin) to initial chemotherapy treatment, and then using it as longer term "maintenance" therapy, significantly slows the spread of the disease in women with cancer in their ovaries and surrounding tissue. Bevacizumab is designed to interfere with the growth of blood vessels needed to fuel a tumor's growth – a process called angiogenesis. The drug continues to be studied to determine its optimal use and whether it actually extends the lives of women with ovarian cancer.

Drug aimed at a newly identified target causes dramatic tumor shrinkages

Drug aimed at a newly identified target causes dramatic tumor shrinkages

The drug crizotinib (Xalkori), which targets the anaplastic lymphoma kinase (ALK) pathway, is shown to cause tumor shrinkage in a high percentage of patients whose tumors had an abnormality in the ALK gene. This ALK gene mutation only occurs in about 4 percent of lung cancers, and is more common in patients who never smoked. The development of crizotinib exemplifies the future of lung cancer therapy, in which tumors will be analyzed for their molecular make-up and then treated based on those findings.

In 2011, the drug is approved for patients with advanced lung cancer whose tumors harbor the ALK gene mutation based on longer-term data.

Tyrosine kinase inhibitors provide additional options for CML

Tyrosine kinase inhibitors provide additional options for CML

Two studies show that dasatinib (Sprycel) and nilotinib (Tasigna) may be more effective than the current standard drug, imatinib (Gleevec), for the initial treatment of chronic myeloid leukemia. Though all three drugs target the same active genetic pathway on the "Philadelphia chromosome," studies suggest the newer drugs possibly elicit faster, stronger responses and cause fewer side effects than imatinib.

First drug shown to improve survival for patients with advanced melanoma

First drug shown to improve survival for patients with advanced melanoma

In a Phase III study, the targeted drug ipilimumab (Yervoy) – which boosts a specific component of the immune system – is found to improve survival and delay disease progression in patients whose advanced melanoma progresses despite other therapies. The drug is approved for this use in early 2011.

Soon after, a second trial finds that treatment using a combination of ipilimumab and dacarbazine (a chemotherapy drug commonly used in melanoma) extends survival compared to dacarbazine treatment alone.

Targeted drug denosumumab helps prevent common bone-related complications in advanced breast cancer

Targeted drug denosumumab helps prevent common bone-related complications in advanced breast cancer

About 80 percent of women with metastatic breast cancer experience cancer spread to the bone, a complication that causes pain, bone weakening and other side effects that affect quality of life. In 2010, a large study shows that the targeted drug called denosumumab (Xgeva) can prevent or significantly delay bone metastases. Other drugs, such as zoledronic acid (Zometa), have previously been used to prevent and treat these bone-related complications, but this study suggests denosumumab may be more effective.

2009

Targeted drug helps children with hard-to-treat leukemia

Targeted drug helps children with hard-to-treat leukemia

Adding the targeted drug imatinib (Gleevec) to standard, intensive chemotherapy is found to dramatically improve outcomes for patients with acute lymphoblastic leukemia (ALL) who have a genetic mutation known as the Philadelphia chromosome. After three years, 80 percent of patients who received both therapies in the study were alive, compared to 30 percent who received chemotherapy alone. While further research is needed, the results offer hope to children with this hard-to-treat form of cancer.

Pazopanib approved for advanced kidney cancer
Bevacizumab approved for renal cancer after multiple studies confirm benefit
PARP inhibitors show promise for difficult-to-treat breast cancers

PARP inhibitors show promise for difficult-to-treat breast cancers

Researchers share the first encouraging findings on a new class of targeted drugs – PARP inhibitors – for difficult-to-treat "triple-negative" breast cancers and those that involve BRCA1 and BRCA2 gene mutations. PARP inhibitors are designed to disable key enzymes that cancer cells use to repair DNA damage, including damage inflicted by chemotherapy, and to promote cancer cell death. They may also make cancer cells more sensitive to other chemotherapy agents. Triple-negative breast cancers lack receptors for estrogen, progesterone and HER2, and therefore do not respond to targeted drugs that block these proteins.

Further long-term, randomized clinical trials are needed to determine whether PARP inhibitors truly benefit patients with breast cancer, and if so, which specific types of breast tumors are most likely to respond to the drugs.

Everolimus approved for renal cell carcinoma if other drugs fail

Everolimus approved for renal cell carcinoma if other drugs fail

FDA approves everolimus (Afinitor) for treatment of renal cell carcinoma in patients whose disease returned or progressed despite prior therapy with sunitinib (Sutent) and/or sorafenib (Nexavar). Everolimus targets the mTOR protein, a pathway that is particularly active in renal cell carcinomas, and was found to help patients live twice as long without their cancer progressing, compared to placebo and standard supportive care.

2008

Bevacizumab (Avastin) approved for advanced breast cancer

Bevacizumab (Avastin) approved for advanced breast cancer

In 2008, the FDA grants accelerated approval for bevacizumab (Avastin) in combination with paclitaxel (Taxol) for women with newly diagnosed advanced breast cancers. Bevacizumab is an "anti-angiogenic" drug, meaning that it works by interfering with the growth of the blood vessels that tumors need to grow and spread. The approval is based on studies showing this regimen delays cancer growth. However, later, longer-term studies show that bevacizumab does not extend survival. Debate continues about the use of this drug for breast cancer, although it has an established role in the treatment of other common cancers, such as lung and colorectal cancers.

Advanced prostate cancers respond to new treatments in early studies

Advanced prostate cancers respond to new treatments in early studies

Two new targeted drugs appear promising in early studies among patients with advanced stages of prostate cancer. In one study, a drug called abiraterone acetate (Zytiga) reduced levels of prostate-specific antigen (PSA) by up to 90 percent in men who no longer responded to hormone therapy. A second trial shows that adding the drug custirsen to standard chemotherapy significantly reduced PSA and pain levels, compared to an alternative combination. (Custirsen is designed to make prostate cancer cells more sensitive to chemotherapy.) In 2011, the FDA approves abiraterone acetate, in combination with prednisone, for men whose advanced prostate cancer progresses despite other therapies, including docetaxel (Taxotere) chemotherapy.

First drug approved specifically for T-cell lymphoma treatment

First drug approved specifically for T-cell lymphoma treatment

A study shows that the new targeted anticancer drug pralatrexate (Folotyn) shrank tumors in nearly one-third of patients with peripheral T-cell lymphoma that persisted or returned after conventional therapy. Tumors disappeared completely in more than 10 percent of patients in the study. In 2009, the FDA grants accelerated approval for pralatrexate for peripheral T-cell lymphoma that has relapsed or has not responded well to other forms of chemotherapy. This is the first drug to be specifically approved to treat T-cell lymphoma, and this accomplishment reflects a movement toward increased clinical research coordination among lymphoma researchers in the U.S. and worldwide to identify new therapies for this rare but often aggressive disease.

Circulating tumor cells in the blood can help track treatment response in select patients

Circulating tumor cells in the blood can help track treatment response in select patients

Recent advances in lab technologies have made it possible to detect cancer cells that have broken free of the tumor, called circulating tumor cells, using a simple blood sample. In a new study, researchers show that tracking the number of these circulating tumor cells is useful for assessing how well a patient with non-small cell lung cancer is responding to therapy.

2007

Bevacizumab (Avastin) receives FDA approval for glioblastoma

Bevacizumab (Avastin) receives FDA approval for glioblastoma

Two early-stage trials suggest that giving the targeted therapy bevacizumab (Avastin), alone or with the chemotherapy drug irinotecan (Camptosar), may cause tumor shrinkage in patients with glioblastoma whose disease progresses after previous therapy. Based on these findings, the FDA grants accelerated (or early, conditional) approval for bevacizumab to treat glioblastoma. Bevacizumab is an "anti-angiogenic" drug, meaning it works by interfering with the development of blood vessels that tumors need to grow and spread. This marks the first new drug approved for treating brain tumors in a decade, and studies are ongoing to determine if initial treatment with bevacizumab improves overall survival.

Study supports expanded use of dasatinib for CML

Study supports expanded use of dasatinib for CML

Research shows that dasatinib (Sprycel) – initially approved for use only when treatment with imatinib fails – should also be an option for initial treatment of early-stage chronic myelogenous leukemia. The FDA approves the drug for this broader indication in 2010, giving patients with CML multiple options for controlling their cancer.

Lapatinib approved for patients whose HER2-positive breast cancer no longer responds to trastuzumab

Lapatinib approved for patients whose HER2-positive breast cancer no longer responds to trastuzumab

The targeted therapy lapatinib (Tykerb) is approved by the FDA for use in combination with the drug capecitabine for patients with advanced breast cancer whose tumors overproduce the HER2 protein. Lapatinib is used to slow disease progression in cancers that no longer respond to trastuzumab (Herceptin). In 2010, the drug is also approved as an initial therapy in combination with the aromatase inhibitor letrozole (Femara) for patients with HER2-positive cancer.

2006

Two new targeted drugs are proven effective in major trials; FDA approvals follow

Two new targeted drugs are proven effective in major trials; FDA approvals follow

Sunitinib (Sutent) is approved in January 2006 for advanced renal cell cancer in previously untreated patients, based on studies showing it shrank more tumors and resulted in slower disease progression compared to the previous standard treatment, interferon.

In May 2007, FDA approves another targeted drug, temsirolimus (Torisel), based on studies showing that it prolonged median overall survival of patients with advanced renal carcinoma by more than 3 months (or about 50 percent), compared to standard interferon treatment. This drug targets the mTOR protein, which regulates cancer cell growth and division.

New drug helps CML patients who become resistant to imatinib

New drug helps CML patients who become resistant to imatinib

Researchers identify a second targeted treatment, dasatinib (Sprycel), for patients with chronic myelogenous leukemia who cannot tolerate or develop resistance to imatinib (Gleevec). The drug is approved based on study results showing that more than 90 percent of these poor prognosis patients had no evidence of their cancer following dasatinib treatment. Dasatinib targets the same mutated protein as imatinib, but through a different mechanism.

2005

First targeted drug approved for pancreatic cancer

First targeted drug approved for pancreatic cancer

A major clinical trial finds that adding the targeted drug erlotinib (Tarceva) to standard gemcitabine (Gemzar) chemotherapy extends the lives of patients with inoperable pancreatic cancer, compared to gemcitabine alone. This is the first trial to identify a survival benefit with a new drug for patients with advanced pancreatic cancer since gemcitabine was introduced nearly a decade earlier. The FDA approves erlotinib for pancreatic cancer later the same year. The new drug targets a protein called the epidermal growth factor receptor (EGFR), which fuels the growth of some types of cancer cells.

Two targeted drugs approved for advanced colon cancer

Two targeted drugs approved for advanced colon cancer

The drugs cetuximab (Erbitux) and panitumumab (Vectibix) are approved to treat colon cancer that has spread to other parts of the body (metastatic disease). These drugs attack tumors that express the epidermal growth factor receptor (EGFR) protein, which is involved in cancer cell growth. Later, a coordinated analysis of multiple studies shows that cetuximab and panitumumab are effective only in patients with the normal form of a gene known as KRAS. This discovery helps physicians ensure that the drugs are used only for patients who stand to benefit, while eliminating unnecessary treatment and costs for patients who will not.

First FDA approval of a targeted drug for kidney cancer

First FDA approval of a targeted drug for kidney cancer

FDA approves the new treatment sorafenib in December 2005, based on studies showing it delays tumor progression in patients with advanced renal cell carcinoma whose cancer recurs or persists despite previous immunotherapy. It is the first treatment found to benefit patients with this advanced, pre-treated form of the disease.

Bortezomib shrinks tumors in mantle cell lymphoma

Bortezomib shrinks tumors in mantle cell lymphoma

Two studies show that the drug bortezomib (Velcade) shrinks tumors in nearly half of patients with mantle cell lymphoma, one of the rarest forms of non-Hodgkin lymphoma. The drug is approved the next year for patients who have relapsed after prior treatment. Bortezomib blocks the proteasome, a protein complex inside the cell that destroys proteins that are no longer needed and cleanses the cell of abnormal proteins. Researchers continue to explore other drugs that target the proteasome.

U.S. launches effort to map cancer genomes

U.S. launches effort to map cancer genomes

The National Cancer Institute and the National Human Genome Research Institute team up to launch The Cancer Genome Atlas project. In its initial phase, the project aims to develop a comprehensive atlas of the genomes of three common cancers – lung, ovarian, and glioblastoma, a form of brain cancer. By gaining a deeper understanding of the genetic pathways involved in the development and growth of these cancers, researchers hope to identify molecular targets that can guide development of effective new treatment.

Anti-angiogenic drug bevacizumab extends survival for advanced lung cancer

Anti-angiogenic drug bevacizumab extends survival for advanced lung cancer

A major study shows that adding the targeted drug bevacizumab (Avastin) to standard chemotherapy extends survival compared to chemotherapy alone for select patients with advanced lung cancer. In 2006, the FDA approves bevacizumab in combination with standard chemotherapy as an initial therapy for inoperable, non-squamous non-small cell lung cancer that has spread within or outside the lungs, or that has recurred. Bevacizumab is the first of a new generation of targeted drugs called "anti-angiogenics," which attack cancer by blocking the tumor's ability to develop the blood vessels it needs to grow.

2004

FDA approves second targeted drug, erlotinib, for non-small cell lung cancer

FDA approves second targeted drug, erlotinib, for non-small cell lung cancer

Erlotinib (Tarceva) is approved for patients with advanced non-small cell lung cancer that has worsened after at least one regimen of chemotherapy. The approval is backed by data showing that erlotinib improves survival by two months, compared to placebo. A follow-up analysis shows that erlotinib also improves patients' quality of life, reducing pain and allowing better physical function. Erlotinib is administered in pill form – a convenient alternative to traditional chemotherapy, which is generally administered intravenously in a doctor's office or hospital.

Epigenetic drugs approved to prevent cancer in patients with myelodysplastic syndromes

Epigenetic drugs approved to prevent cancer in patients with myelodysplastic syndromes

The FDA approves azacytidine (Vidaza) in 2004 and decitabine (Dacogen) in 2006 for treatment of myelodysplastic syndromes (MDS), a group of serious blood disorders that predispose a person to acute myelogenous leukemia. These targeted drugs interfere with the control of genetic processes that enable cancer cells to develop and multiply. Both drugs are shown to reduce or eliminate the need for blood transfusions – a common MDS treatment that can impair patients' quality of life.

Studies show that specific EGFR mutations are associated with response to gefitinib

Studies show that specific EGFR mutations are associated with response to gefitinib

A series of major studies shows that lung cancer tumors with specific mutations of the EGFR (epidermal growth factor receptor) protein are highly responsive to EGFR-targeted drugs. EGFR is an important signaling pathway that stimulates cancer cell division, and these findings provide critical information for identifying a subset of patients most likely to respond to gefitinib (Iressa) and erlotinib (Tarceva). The relevant mutations are found most commonly in lung cancer patients without a significant smoking history.

FDA approves first "anti-angiogenic" drug, bevacizumab

FDA approves first "anti-angiogenic" drug, bevacizumab

Bevacizumab (Avastin) is the first of a new generation of targeted drugs, called anti-angiogenics, that attack cancer by blocking the growth of blood vessels that tumors needs to grow. First approved to treat colorectal cancer, in 2004, the drug has since become an important treatment for patients with advanced lung, ovarian and kidney cancers, and for certain brain tumors, who have few other effective options.

2003

First targeted drug approved for non-small cell lung cancer

First targeted drug approved for non-small cell lung cancer

The FDA approves the first targeted therapy for non-small cell lung cancer, gefitinib (Iressa), following studies showing it shrinks tumors in some patients with advanced cancer that progresses despite other therapies. Gefitinib targets the epidermal growth factor receptor (EGFR), a protein on cell surfaces that drives lung cancer growth and spread. Use of gefitinib is restricted by the FDA in 2005 to patients who had already benefited from the drug or were receiving it through a clinical trial, after data from larger trials show gefitinib therapy did not extend patients' lives. However, later research suggests that gefitinib has value as a first-line treatment, and is more effective than chemotherapy in patients whose tumors carry an EGFR mutation.

Scientists decode the human genome

Scientists decode the human genome

Scientists announce that they have successfully mapped the 3 billion DNA letters in the human genome. This marks the completion of the Human Genome Project, an unprecedented international collaboration between researchers in the U.S. in and six other countries, funded primarily by the U.S. government. Results of the 13-year effort are made freely available to scientists around the world, paving the way for research to identify the genetic defects that fuel cancer, and for new ways of screening for and treating the disease.

2002

Key molecular pathways in liver cancer offer new insight

Key molecular pathways in liver cancer offer new insight

Caption

Researchers discover genetic pathways that govern liver cancer cell growth and development. The findings suggest that liver cancers develop as a result of disruptions along multiple pathways, resulting in many genetically varied forms of liver cancer. These findings explain why the development of gene-targeted drugs has proven so challenging over the past decade, but also provide researchers with a number of potential treatment targets to explore.

Radioimmunotherapy introduced to treat lymphoma

Radioimmunotherapy introduced to treat lymphoma

This new therapeutic approach becomes available to patients with the FDA approval of the drug ibritumomab tiuxetan (Zevalin). The drug acts as a "smart bomb" against the tumor by combining a radioactive material with a protein that locates and binds to a select receptor on cancer cells called CD-20. The radioactive material kills the cells with which it comes in contact, while sparing the healthy surrounding tissue. In 2003, a similar drug called tositumomab (Bexxar) is approved for the same use.

Adding rituximab to "CHOP" chemotherapy boosts survival

Adding rituximab to "CHOP" chemotherapy boosts survival

The targeted drug rituximab (Rituxan), when added to standard CHOP chemotherapy (cyclophosphamide, doxorubicin, vincristine and prednisone), is shown to boost survival for older patients with diffuse B-cell lymphoma. Subsequent trials confirm the results in patients of all ages, and the "R-CHOP" combination soon becomes standard treatment for this type of non-Hodgkin lymphoma.

2001

Imatinib found highly effective against rare gastrointestinal tumor

Imatinib found highly effective against rare gastrointestinal tumor

Just weeks after being approved to treat chronic myelogenous leukemia, the targeted drug imatinib is shown to be effective against a rare abdominal tumor called GIST (gastrointestinal stromal tumor). Prior to imatinib, available drug treatments had little effect on GIST tumors. Imatinib works like a "circuit breaker" to block enzymes that can send faulty signals to trigger tumor cell growth. In GIST, it blocks the growth signal of a gene called c-Kit.

New targeted therapy transforms treatment for rare leukemia

New targeted therapy transforms treatment for rare leukemia

The FDA approves imatinib (Gleevec) after just three months of review – the fastest approval in FDA history – based on data showing the drug halted the growth of chronic myelogenous leukemia (CML) in the majority of patients. Imatinib is the first drug proven to counteract a molecular defect on the so-called "Philadelphia chromosome," first discovered in 1960. It has since become the standard of care for this disease, and its effectiveness and easily-administered pill form enables most patients to live with CML as a manageable, chronic disease.

1999

First targeted anti-breast cancer drug, trastuzumab (Herceptin), has major impact on care

First targeted anti-breast cancer drug, trastuzumab (Herceptin), has major impact on care

The FDA approves the groundbreaking drug trastuzumab (Herceptin) after research shows that adding the monoclonal antibody to chemotherapy dramatically increases survival for women with advanced breast cancer that over-produces a protein called HER2. In 2006, the drug is also approved as part of adjuvant therapy (after surgery) for women with early-stage HER2-positive breast cancer, after two major trials show that it reduces the risk of recurrence by more than 50 percent, an unprecedented result.

About 25 percent of breast cancer patients have HER2-positive disease, and prior to the introduction of trastuzumab, there were no effective treatments for these cancers, which were considered some of the most aggressive, deadly forms of the disease. Recently, trastuzumab was also FDA-approved to treat patients with stomach cancers that have a similar over-production of the HER2 protein.

1997

FDA approves first-ever targeted cancer drug, rituximab

FDA approves first-ever targeted cancer drug, rituximab

The FDA approves the first molecularly targeted cancer drug, rituximab (Rituxan), to treat patients with B-cell non-Hodgkin lymphoma that no longer responds to other treatments. Rituximab is in a new class of drugs called monoclonal antibodies, and targets a protein on the surface of immune cells known as B cells, interfering with the development of cancer. It is later combined with other cancer therapies to boost cure rates and increase survival.

1987

Scientists discover key genetic vulnerability in tumor cells – EGFR

1960

Researchers link "Philadelphia chromosome" to leukemia

Researchers link "Philadelphia chromosome" to leukemia

Investigators in Philadelphia identify a chromosomal abnormality linked to many leukemias. A decade later, researchers discover that this abnormality results when parts of two chromosomes – chromosomes 9 and 22 – switch places in a phenomenon called translocation. It later becomes the target of one of the first-ever targeted cancer treatments, imatinib (Gleevec), which transforms treatment of chronic myelogenous leukemia and other cancers.