Breakthrough FDA Ruling

The Food and Drug Administration (FDA) granted accelerated approval to the immunotherapy pembrolizumab for patients with solid tumors that have one of two specific genetic features known as mismatch repair deficiency and high microsatellite instability. The approval covers adult and pediatric patients whose cancer has progressed despite prior treatment and who have no alternative treatment options.

This is the first time that FDA has approved a cancer treatment based solely on the presence of a genetic feature in a tumor, rather than the patient’s cancer type.

“I think this is a step forward for precision medicine.”

–James Gulley, M.D., Ph.D., head of the immunotherapy section of NCI’s Center for Cancer Research

Having a biomarker to identify patients who are most likely to respond is “an area we have widely anticipated as being the next step in understanding how to better use immunotherapies,” said Dr. Gulley. “It’s a welcome first step, and there’s much more yet to be done.”

For the full article, visit the National Cancer Institute page here.

Key to Metastasis?

A team at Johns Hopkins has discovered the biochemical mechanism that tells cancer cells to break off from the primary tumor and spread throughout the body, a process called metastasis. Some 90 percent of cancer deaths are caused when cancer metastasizes.

“…what we came up with through our studies was this drug cocktail that could potentially inhibit the spread of cancer.”

— Hasini Jayatilaka, post-doctoral fellow at Johns Hopkins

Typically, cancer research and treatment has focused on shrinking the primary tumor through chemotherapy or other methods. But, the team said, by attacking the deadly process of metastasis, more patients could survive.
Check out the whole article in the Baltimore Sun by clicking here.

Institute for Protein Innovation Launched

The Institute for Protein Innovation will work to pursue new therapies for currently intractable diseases by bringing together leaders with backgrounds in academic research, the biopharma industry, and biomedical investing.

The Institute plans to curate information related to DNA sequence, protein expression, and functional validation of these reagents, including source code, in a publicly-accessible web portal, with the goal of accelerating the development of new drugs and supporting existing large-scale research efforts, citing the Human Cell Atlas.

The Institute said it will recruit researchers focused on directed evolution, cell-line development, and biophysical protein characterization, in addition to glycoprotein expression and antibody discovery expertise.

“Despite their pivotal importance in research and medicine, proteins lag behind DNA and RNA in institutional research support and funding.”

–Timothy A. Springer, Ph.D., Founder and Latham Family Professor at Harvard Medical School and Boston Children’s Hospital

Read the full article at Genetic Engineering and Biotechnology News here.

New Article in GEN

Tissue Chips Matter!

tissue on a chip

Check this video from NCATS

How can we speed new drugs to market? One of the cool innovations coming to trials is the concept of “tissue on a chip”. Basically, scientists are mimicking human organs and testing drug compounds on these 3-D platforms. Check out this video for a quick explanation.

To find out more, go to the NCATS “Tissue on a Chip” website.

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National Center for Advancing Translational Sciences main website

Promise of Gene Therapy Being Realized?

Gene therapy has finally made it over the hump. After decades of research and some devastating setbacks, major technical hurdles have been overcome, opening up the long-anticipated promise of this field. A new approval has buoyed interest, pipelines are bubbling with new candidates and big investments are being made.

News about promising gene therapies in the clinic is sprouting up all the time. Scientific challenges remain considerable, though, and pricing will clearly be one of the biggest hurdles for gene therapies going forward.

“Today’s challenges are all around building a viable business model”

— Geoff MacKay, President and CEO of AvroBio

For the complete article at BioPharma Dive, click here.

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Exciting BioPharma News

Predictive Gene Expressions

Qiagen has reported that the company and Bristol-Myers Squibb will explore the use of NGS to develop gene expression profiles as predictive or prognostic tools for use with several BMS immuno-oncology therapies. The companies also plan to enter into a further agreement to develop diagnostic products using the jointly developed profiles to expand the use of NGS technology with other BMS immuno-oncology therapies.

“Greater precision in the treatment of cancer may enable faster decision making to identify which patient populations are most likely to derive benefit from our immuno-oncology agents.”

–Fouad Namouni, Bristol-Myers Squibb

This development is an example of the trend towards companion diagnostics, in which the use of predictive biomarkers identify individually-based therapies.
Read the announcement here.

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Get more genomic news at GenomeWeb

Precision Medicine’s All of Us Kicks Off

All of Us, the Precision Medicine Initiative that aims to revolutionize the path to improving health, has launched its beta phase.
The All of Us Research Program is a historic effort to gather data from one million or more people living in the United States to accelerate research and improve health. By taking into account individual differences in lifestyle, environment, and biology, researchers will uncover paths toward delivering precision medicine.
The program intends to get at least 1 million volunteers to sign up to contribute information on their genetic makeup, environmental factors, lifestyle choices, medical histories, and personal device and sensor data.

“By providing information about their health, lifestyles, and environments over the course of many decades, these volunteers will be important partners in helping create an unprecedented research resource to drive future discoveries.”

–Program Director Eric Dishman
The full announcement of the beta is available here.

Finding Brain Cancer 5 Years Early

the ohio state university logo

New research from academia

Interactions among proteins that relay information from one immune cell to another are weakened in the blood of brain cancer patients within five years before the cancer is diagnosed, said lead researcher Judith Schwartzbaum of The Ohio State University.

“this research could pave the way for techniques to identify brain cancer earlier and allow for more-effective treatment”

Read the entire article from The Ohio State University here.

From Point of Care to Point of Clinic

Within developed world healthcare systems, there is a growing awareness that the turnaround time of the central laboratory cannot always provide the speed to enable effective and economic decision making.

See full article

Point-of-clinic diagnostic services bring analytical testing from the laboratory to the clinic with the aim of providing prompt, high-quality results.
This is an exciting area of technology that has the potential to impact the lives of many patients and play a significant role in supporting sustainable healthcare systems. Stratified medicine and more individualized approaches to care will become a focus moving forward, and diagnostics will aid the selection of the most effective medicines.

“This facilitates early diagnosis and treatment of health conditions…”

Check the full article at Qmed here.

New Sensor for Early Cancer Detection

A new plasmonic sensor developed by researchers at the University of Illinois at Urbana-Champaign will serve as a reliable early detection of biomarkers for many forms of cancer and eventually other diseases.

Research from University of Illinois

“…instead of needing a vile of blood for a test, a simple finger prick will do.”

For now, detection methods for cancer biomarkers are being implemented in high-risk patients, especially cancer patients in remission. They take time, specialized equipment, and are labor-intensive.
In the future, however, because of the portability and inexpensive nature of this method, it can be more easily administered to any patient at routine check-ups. This would allow those with an elevated concentration of CEA to be treated even before cancer cells spread in the body.
Read the full article at the University of Illinois Engineering site.