What it Takes for New Drugs to be Tested and Reach Patients

What goes into researching and developing a cancer drug and what is required for approval by the Food and Drug Administration (FDA)? We sat down with Moshe Talpaz, M.D., Associate Director of translational research at the U-M Comprehensive Cancer Center, for a closer look at the FDA approval process.

Moshe Talpaz, M.D., has been involved in the FDA approval process of many cancer drugs. He leads translational research at the U-M Comprehensive Cancer Center.

Q: What is the general timeframe for a new drug to be approved by the FDA so it is available to patients?

From beginning to end, typical clinical drug development takes around 7-10 years. If a drug is showing remarkable activity and serves an unmet medical need, an accelerated process can go much quicker, maybe around 3 years after the onset of clinical trials.

Q: What is the first part of the process in developing a new drug?

The preclinical process is quite elaborate. Academic medical centers like U-M have the role of discovering what governs a disease, to discover the mutation that drives an illness like cancer. We first focus on the biology to show the full sequence of events in the body. We develop various laboratory approaches to demonstrate that shutting off the abnormal protein kills the cancer cell.

Drug companies then get involved. Medicinal chemists study many different molecules to identify one that has the characteristics of potentially becoming a drug.  This becomes the subject for extensive testing on animals to determine that it works safely in the body.

Q: When does research begin with actual patients?

Phase I testing in humans aims to find the maximum tolerated dose. We start with very low doses to check for toxicity. We end up with a safe dose to recommend for use in a Phase II study.

Phase II involves more extensive testing and is where we demonstrate whether the compound is effective and which tumors respond to it.

Phase III is a randomized study of large groups of patients that compares the potential drug to the current standard of care. If the compound reaches a significant goal, which is acceptable to the FDA, the FDA approves it.

Q: What about drugs that go through the accelerated process for FDA approval? Are there disadvantages when drugs go through the process quickly?

I believe the FDA approval process is streamlined and efficient. If a compound proves to be good and there is no question about it, it moves forward quickly in the approval process.

The FDA requires further testing on the drug once patients are using it, and the drug must meet additional milestones later.

Learn more about the clinical trials at the U-M Comprehensive Cancer Center.

Making a Difference in Breast Cancer Research

In honor of Breast Cancer Awareness Month, we’d like you to meet Kristine, a breast cancer survivor:

For more information about participating in clinical trials, please visit An Introduction to Clinical Trials.

Learn more about how you can make a difference in breast cancer research and treatment:

Contact the Cancer AnswerLine™ at 800-865-1125 for help finding a clinical trial that’s right for you.

 Have you participated in a clinical trial? Tell us what motivated your choice.


What is Triple- Negative Breast Cancer?

Did you know that there are many types of breast cancer?  Breast cancer is a disease made up of several subtypes, one of which is called triple negative breast cancer.  Approximately 10% to 20% of breast cancers are triple-negative.

Dr. Lisa Newman, who directs the U-M Breast Care Center, speaks to colleagues and patients in Ghana, Africa. Her research focuses on triple negative breast cancer in African-American women. In Ghana, she treats women with breast cancer and collects information to bring back here. By better understanding the disease in African women, Dr. Newman hopes to be able to improve treatment options for all women with triple-negative breast cancer.

Triple-negative breast cancer cells do not express three markers that can play a role in breast cancer.

  • Estrogen receptor(ER)
  • Progesterone receptor(PR)
  • HER2(human epidermal growth factor receptor 2)

These negative results mean that the growth of the cancer is not supported by the hormones estrogen and progesterone, or by the presence of too many HER2 receptors. Therefore, triple-negative breast cancer does not respond to hormonal therapy (such as Tamoxifen or aromatase inhibitors) that target estrogen and progesterone, or therapies that target HER2 receptors, such as Herceptin.

Who gets triple -negative breast cancers?

  • Younger people – Triple-negative breast cancer is more likely to occur before age 40 or 50, whereas other breast cancer types are more common in women; 60 or older.
  • African-American and Hispanic women -Triple-negative breast cancer most commonly affects African-American women, followed by Hispanic women. Asian women and non-Hispanic white women are less likely to develop this type of cancer.
  • People with a BRCA1 mutation. – When people with an inherited BRCA1 gene mutation develop breast cancer, especially before age 50, it is often triple-negative.

Researchers do not yet understand why premenopausal women and women in some ethnic groups have higher rates of triple negative breast cancer–than other groups of women. Continue reading

Collaboration and Innovation will defeat Cancer

logos for the U-M Comprehensive Cancer Center, the National Cancer Institute and the National Comprehensive Cancer NetworkIf you’ve visited the U-M Comprehensive Cancer Center’s website, or happened to read a news article about the Center; you may have noticed we have an association with the National Cancer Institute (NCI) and the National Comprehensive Cancer Network; (NCCN).  What you may not know is the importance of these associations – particularly as it relates to cancer treatment and research.

The NCI, part of the National Institutes of Health, supports and coordinates cancer research projects conducted by universities, hospitals, research foundations and businesses in the United States as well as world-wide.  In turn, the NCI collects and shares information about cancer treatment and research.  As an NCI-designated comprehensive cancer center – one of only 41 in the country — the U-M Cancer Center has the opportunity to represent the needs of our community in the national dialogue. We also have access to national and world-wide research collaborations – as well as the opportunity for our own research to receive funding.   Our NCI funding has allowed our doctors and researchers to explore promising new ways to make cancer care less toxic, more compassionate and more effective.

The NCCN is a not-for-profit alliance of 21 of the world’s leading cancer centers dedicated to improving the quality and effectiveness of cancer care.   One of the NCCN’s most significant projects is the clinical practice guidelines.  The guidelines offer treatment, prevention, detection and supportive care standards that can be used by patients, doctors and other health care decision-makers.  Our doctors are at the table helping to determine the best practices in cancer care. By standardizing these methods for treating and preventing cancer, the NCCN ensures quality care for cancer patients nationwide.

Becoming a U-M Cancer Center patient means your treatment will follow the latest and best guidelines that have proven effective over rigorous study. But more than that – your U-M health care team belongs to a collection of the best cancer providers in the country, providing the best care to you and your family.

Improving Breast Cancer Treatment Through Better Decisions

One of the biggest challenges facing doctors and their cancer patients is deciding on a treatment plan for a disease that has a relatively favorable prognosis.

As more breast cancers are found at an earlier stage, patients and doctors must consider the best way to treat the cancer without over treating the patient and causing unnecessary side effects and quality of life problems. For women with breast cancer, improving the quality of these treatment decisions has a high potential for improving the quality of their care.

Thanks to a $13.6 million grant, U-M breast cancer researchers and a national team will study how patients and doctors make breast cancer treatment decisions, and how to improve the process for better outcomes. The group expects to develop an online decision tool for patients to help improve the quality of their decision-making. This tool will be tested first in a clinical trial to measure its effectiveness.

The best treatment decisions are made when a patient and doctor really talk with each other. Still, the prospect of understanding your medical condition, what the treatment options are, the risks and benefits of each choice, and then deciding what treatment plan is right for you can seem overwhelming. Such things as underlying values, spiritual needs and family concerns are often just as important during the journey from treatment to recovery, but patients sometimes are not comfortable discussing these concerns with a stranger.

But there are good resources available to help patients prepare for the decisions ahead.

Continue learning how to become an empowered patient


Stand Up, on Friday, Sept. 7 at 8 PM

Why I Stand Up.

“The patient is the ultimate lab.” 

These words, spoken by the late Laura Ziskin, continue to resonate with all of us who receive funding from Stand Up to Cancer (SU2C). With these words Laura encapsulated a major challenge facing cancer researchers today: too many discoveries stay in the lab and fail to be translated into innovations in patient care.

In 2008, Laura, along with six other remarkable women from the entertainment industry, recognized this challenge and decided to tackle it head on by creating SU2C.

The funding commitments from SU2C are already having impact on moving research along. Through their Dream Team initiative, SU2C funds collaborative research that unites senior scientists across multiple institutions to rapidly advance new therapies into the clinic. Two researchers from the University of Michigan are part of SU2C Dream Teams –Dr. Arul Chinnaiyan leads the Prostate Cancer Dream Team and Cancer Center Director Dr. Max Wicha is part of the Breast Cancer Dream Team.

The second major funding commitment made by SU2C is their Innovative Research Grants Program.  With this initiative, SU2C targets investigators in earlier stages of their scientific careers and asks them to propose high-risk, high-reward research that has the potential to radically alter current thinking. Since 2009, SU2C has funded 26 Innovative Research Grants, including to two U-M scientists – me and Dr. Yali Dou, an associate professor of pathology. Continue reading