"There is No Plan B." ~ Spencer Bell

Company Takes Rare Adrenal Cancer Drug Co-discovered by a Patient into Clinical Trials

 

This story peaked our interest as it involves UM and a patient who helped work to find a cure :)

Raili Kerppola had a thriving career in the pharmaceutical industry when she was diagnosed with stage four adrenal cancer in 2011.

While seeking treatment at the University of Michigan, she began working with an endocrinologist named Dr. Gary Hammer and her husband, a professor of biological chemistry at UM, on researching and discovering new treatments for the rare disease.

Kerppola died this June. But her legacy lives on through a VC-backed company called Atterocor, for which she is credited as a co-founder. The University of Michigan spinoff is conducting a Phase 1 clinical study of a drug that it hopes could prolong the lives of patients with adrenocortical carcinoma, and cause fewer toxic side effects, than the current treatment option.

It’s a rare form of cancer that carries a short life expectancy after diagnosis.  That’s because it’s usually caught late-stage, when a patient presents with symptoms like unexplainable weight gain, hypertension, abdominal pain or other metabolic complications caused by excessive production of hormones by the adrenal glands.

Treatment starts with surgery, if the cancer is caught early enough. Then, doctors usually try to extend life with a cocktail of chemotherapy drugs including mitotane, a drug that reduces the amount of hormone produced by the adrenal cortex.

The problem? Mitotane, a derivative of the pesticide DDT, is terribly toxic. And its efficacy is questionable, too.  Atterocor co-founder Julia Owens said the FDA approved the drug decades ago, before the agency required drugmakers to demonstrate that it works before approval. She said that although it likely does have some benefits for patients, they usually have to take multiple doses a day for three to four months before their mitotane levels reach a therapeutic range. Meanwhile, they’re usually also taking chemotherapy drugs.

“One endocrinologist recently told me that the way you can tell if a patient is getting close to efficacious mitotane levels is when they can’t walk down the hallway without assistance,” she said.

Atterocor says its compound is highly specific to the cells of the adrenal cortex, which is where the cancer occurs. “We haven’t done human trials yet so we can’t make any claims, but based on toxicity studies, our effect seems much more specific, much more rapid, with a better (pharmacokinetic) profile,” she explained.

It’s received orphan drug designation in the U.S. and in Europe. The company started a Phase 1 trial over the summer at MD Anderson Cancer Center and the University of Michigan Comprehensive Cancer Center. Owens said the company should have some data on tumor responses and steroid markers in the next six to 12 months.

With six employees, Atterocor is a small company, but it’s attracted some big-name investors. Last year the Ann Arbor startup reined in a $16 million Series A from Frazier Healthcare, Osage University Partners and 5AM Ventures.

Owens said that, for a company developing an orphan drug, that capital makes all the difference. “We can move this compound forward ourselves without being at the mercy of a big pharma company (partner),” she said.  “We haven’t been around for two years yet and have taken this idea from the University of Michigan and run our toxicology studies, synthesize the drug and now are running the study. If we see encouraging results, we could go into a Phase 2 study and seek approval ourselves.”

You have to think that’s something Raili Kerppola would be proud of.”

Read more: http://medcitynews.com/2013/12/patients-research-rare-adrenal-cancer/#ixzz2pvQNaIpu

New Cancer Drug ‘Revs Up’ Immune System

Cancer Testing

Scientists at the University of Southampton have developed a drug to strengthen the immune system to destroy cancer.  Many caners can switch immune cells off which make it unable for them to attack the tumor and stop its growth. This new drug, ChiLob 7/4, helps to increase the immune system and switch the immune cells back on so it can attack cancerous tumors in difficult areas to treat such as the pancreas, head and neck.  A trial has already been done among 26 pancreatic cancer patients and the results are promising. Scientists will start a £5- million European Union funded trial of the treatment beginning next year.  The research of this new cancer drug is being led by Professor Martin Glennie, a cancer specialist at the University of Southampton.  The drug is apart of a cancer treatment known as immunotherapy which is an attempt to use the patient’s immune system to target cancerous tumors rather than chemotherapy or radiation. Currently, there is only one immunotherapy (called Ipilimumab)  which has been approved to be used in patients. Professor Glennie said he hoped ChiLob 7/4 could start being used widely in patients within the next five years if the clinical trials are successful. He also added “We know from our phase one trials that it produces symptoms like the flu, but this is relatively mild compared to the side effects of chemotherapy and disappears once the antibodies have gone away.”

 

For complete article, see:

http://life.nationalpost.com/2013/08/12/new-drug-targets-tough-to-treat-cancers-by-revving-up-the-immune-system/

University of Michigan Comprehensive Cancer Center Turns 25

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Max Wicha, M.D., director of the University of Michigan Comprehensive Cancer Center, reflects on the center’s first 25 years and what the future may hold.

How did the Cancer Center come about?

The idea actually started on a napkin. It began at a meeting of the American Society of Clinical Oncology — this was about 1985. At the time, I was the chief of the division of hematology/oncology and Dr. Allen Lichter was the chairman of the Department of Radiation Oncology. We started talking about creating a cancer center at the U-M. We then got together with Dr. Raymond Ruddon, chairman of Pharmacology and one of the most renowned cancer researchers on the U-M faculty at the time. In these initial discussions, we sketched out on the back of a napkin how we might establish multidisciplinary cancer programs that would bring together basic scientists, clinical scientists, radiation oncologists and surgeons.

What was the vision?

The goal of a cancer center was to design better strategies to link cutting-edge research and clinical care, and in fact, that has remained our goal throughout the last 25 years. It’s both a challenge as well as a goal — inking research to patient care and putting together multidisciplinary teams of researchers and clinicians in the most efficient way to facilitate patient care and research.

Research and care weren’t integrated back then?

The U-M, in fact, does have a long-standing history of being a center for interdisciplinary research. However, in the field of cancer, much of the research, as well as cancer care, was fragmented. Most of the physicians and scientists were organized according to their departmental appointments. This sometimes accentuated a “silo” mentality that inhibited interdisciplinary research. The Cancer Center provided a structure to organize multidisciplinary and interdisciplinary research between departments and schools at the university. We felt, and still believe, that this is a better way to advance research and patient care.

What was it like at the beginning of the Cancer Center?

When we started the Cancer Center, we were scattered throughout many buildings at the Medical Center and other schools in the university. There was, in fact, no Cancer Center building. We ran the administration of the center from a trailer behind the Simpson Memorial Institute. That was it — all we had was a trailer and a vision of what could be created in the future.

But even without a dedicated building, what was happening?

Even before we built the cancer center facility, we worked with Medical Center and departmental leaders to greatly expand the cancer research and treatment capabilities at the U-M. This included our recruitment of a number of stellar researchers, many of whom focused on moving basic research into the clinic. As a result our cancer program, which was unranked nationally, jumped into the top 20 over that decade. In fact, we have continued that trajectory and are now the No. 1 university-based cancer center in terms of National Cancer Institute grants to medical schools and No. 3 in the country for overall grants from NCI.

How is cancer research changing?

For much of the last 20 years, we thought that the most efficient way to organize cancer research was according to disease sites. We created research programs in each of these disease sites such as breast cancer, prostate cancer, colon cancer and pancreas cancer. This greatly facilitated our interdisciplinary research. However, science continues to advance and we now find that there are many similarities between cancers in different organs at the molecular and genetic level. Our challenge is now to integrate research across multiple tumor types so that we can draw from the strengths of these programs and develop specific treatments that target genetic pathways in cancer.

What are some of the current challenges?

Despite the tremendous advances in cancer research, national funding for cancer research is actually declining. As a result, only 7 percent of grants submitted to the NCI are now funded. Our core grant which funds the Cancer Center infrastructure was successfully renewed for the sixth time last year. Despite receiving an “Outstanding” priority score, however, NCI budgetary constraints have resulted in a freeze of all cancer center budgets.

The tremendous increase in cancer research technology has also been expensive. These increased expenses and great opportunities occurring in a context of decreased NIH funding means that we need to identify additional sources for research funding. One of these sources is the biotechnology and pharmaceutical industry. The Cancer Center has greatly expanded its interaction with these groups. This not only helps to support research, it helps to move discoveries into the clinic in an efficient manner. As an indication of this success, U-M Cancer Center investigators have spun-off 10 biotechnology companies, eight of which remain in Michigan. In addition to moving research forward, this helps to stimulate the local economy.

Another important source for funds to support research is philanthropy. We are privileged to have a loyal group of donors who have helped us develop our dream. We continue to work with the Health System to identify and work with major donors and in fact, to potentially attract a naming gift for the entire Cancer Center.

In the future, will oncologists be able to select a treatment based on the genetic mutations of each patient?

That is our hope. In fact, the Cancer Center is one of the leaders nationally at performing genetic analysis of individual tumors. The analyses have shown us that individual tumors may contain multiple mutations suggesting that successful cancer treatment may require the use of multiple targeted drugs. A good analogy of this is the treatment of AIDS. If you use a single drug against HIV, the virus rapidly becomes resistant. But if you use a cocktail of inhibitors, you can inhibit HIV growth turning AIDS into a chronic disease. That’s what we hope to accomplish with cancer treatment. Using a combination of molecularly targeted drug inhibitors, we can either eradicate cancer or at least make cancer a chronic disease.

The NCI’s most recent “Cancer Trends Progress Report” shows that the overall incidence and death rates for cancer are decreasing. Is there a danger that people will relax and say cancer is taken care of, let’s move on to something else?

It is true that we are making substantial progress in the treatment of cancer. However, we also are seeing an aging population and since cancer is more common as we age, this is leading to an increase in cancer incidence. As we continue to make progress in treating other major killers such as cardiovascular disease, cancer may become the No. 1 killer of Americans over the next decade. Furthermore, although we’ve made great progress in treating certain cancers, progress has been significantly more limited in the treatment of other cancers — and progress in the treatment of these cancers will depend on our continued research.

What are your dreams for the future of the Cancer Center in the next 25 years?

I would like the U-M Comprehensive Cancer Center to be seen as the place where some of the crucial breakthroughs were made that led to improved outcomes for patients with cancer. We also would like U-M to continue to enhance its reputation as a preeminent center for holistic patient center care, as well as groundbreaking research.

 

Respectfully taken from University of Michigan’s website. To see original article, go to  http://www.cancer.med.umich.edu/giving/wicha-remembers.html

Gene Test Aids in Thyroid Cancer Screening

biopsy

The current test for Thyroid cancer is a fine-needle aspirate biopsy. According to a new study done at the Pontifical Catholic University of Chile, between one-fourth and one-fifth of the results gathered are unclear. Unclear tests cause increased anxiety among patients along with more tests or surgery where cancer is only present in 25% of cases. Three out of every four patients end up having unnecessary thyroidectomies (removal of the thyroid) which leads to high treatment costs and risk of complications.

Investigators have created a gene signature test which identifies 18 genes that are associated with thyroid cancer. Ten of those genes are then used to develop a computer program which finds thyroid nodules with cancer. The study tested thyroid nodules from 300 patients by using a DNA-amplifying test which they call the polymerase chain reaction. So far, the test has accurately identified benign nodules in almost all of the samples that were tested along with identifying cancerous and noncancerous tissue in 96% of samples. The test could be helpful in the future to identify those patients who do not need surgery.

All information taken from:

http://www.drbicuspid.com/index.aspx?sec=sup&sub=orc&pag=dis&ItemID=313639

Parental Longevity and Cancer

centenarians

 

Most of the time when we think about our chances of developing a disease or condition, we look to our parents. If a man’s father, grandfather, and great grandfather were bald, there is a good chance he will eventually go bald too. If a woman’s mother, aunt, and grandmother all have lots of wrinkles, it is a pretty safe bet that she assumes she, too, will have wrinkles. What if the correlation between our parents’ longevity and our own was linked just like wrinkles or hair-loss? The study below suggests that people whose parents live past their life expectancy are more likely to live longer and are less likely to develop cancer. Looks like now there is another reason to hope our parents live to a ripe old age!

A study from 1992 to 2010, which included 9, 764 people in America, concluded that people who had a long- lived mother or father were more likely to live longer along with a 24% less chance of getting cancer. The study compared patients who had parents that lived longer compared to those who had parents who lived to an average age for their generation. Long-lived mothers were classified as those who lived past 91 years old, compared to those who reached an average of 77-91 years of age. Those who were long-lived fathers lived over the age of 87 compared to an average of 65 to 87 years. 

Mortality rated dropped up to 19% for each decade that a parent lived past the age of 65. For children whose mothers lived past 85 years mortality rates dropped 40% and those with fathers who also lived past 85 years mortality dropped 19% (More adverse lifestyle factors were believed to be more common in fathers).

There have been previous studies which have shown that children with parents who lived past 100 years old tend to live longer along with less heart diseases but this is the first study to show, with evidence, that children with long- lived parents are less likely to get cancer, diabetes or suffer a heart attack. Evidence also showed that the resistance of inheriting an age-related disease lowered as the parents got older.

The group of scientists included Kenneth Langa, M.D., Ph.D, a professor of internal medicine at U-M Medical School as well as the associate director of the U-M-based Health and Retirement study. There were also doctors from the University of Iowa such as Robert Wallace, Dr. Jean-Marie Robine from the National Institute for Health and Medical Research in France (Institut National de la Santé et de la Recherche Médical) and David Melzer of the University of Exeter Medical School.

 

All information taken from

http://www.cancer.med.umich.edu/news/long-lived-parents-and-cancer-2013.html

Bradley Lowery’s Fight Against Cancer

On January 7th, 2013 Bradley Lowery, of the UK, was diagnosed with stage 4 high risk neuroblastoma. Neuroblastoma is a form of cancer that forms in immature nerve cells in the body. It is commonly found in or around the adrenal glands and affects kids younger than 5 years of age; Bradley turned two on May 17th, 2013.

Bradley’s cancer was found in his adrenal glands and has now spread to his chest, lungs, lymph nodes, bone and bone marrow. As of May, Bradley gone through his 5th lot of chemotherapy. Doctors say that those with neuroblastoma have a 50/50 chance of survival along with an 80% chance he could relapse.

Bradley’s family is campaigning to raise £500,000 so he can fight any relapses he may have. There is no relapse treatment in the UK so he will have to be flown to the United States to be treated. More rounds of chemotherapy, radiotherapy, surgery and immunotherapy will be used to prevent a relapse.

 

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Follow Bradley’s story on is Facebook page, Bradley Lowery’s Fight Against neuroblastoma or follow him on Twitter at @Bradleysfight. There is also a video of fundraising efforts on YouTube (http://www.youtube.com/watch?v=DcpmqS_4W5k).

 

All information taken from: http://www.hartlepoolmail.co.uk/news/local/cancer-fighting-bradley-s-birthday-milestone-1-5691156

Gene Sequencing Helps Identify Cancer Mutations

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The recent discovery that gene sequencing, or the process of determining the order of nucleotides within a DNA molecule, could help identify cancer mutations began with a 44-year-old woman with solitary fibrous tumor, a rare cancer seen in only a few hundred people every year.

By looking at the entire DNA from this one patient’s tumor, researchers have found a genetic anomaly that provides an important clue to improving how this cancer is diagnosed and treated.

Researchers at the University of Michigan Comprehensive Cancer Center sequenced the tumor’s genome through a new program called MI-ONCOSEQ, which is designed to identify genetic mutations in tumors that might be targeted with new therapies being tested in clinical trials.

The sequencing also allows researchers to find new mutations. In this case, an unusual occurrence of two genes fusing together. This is the first time this gene fusion has been identified.

“In most cases, mutations are identified because we see them happening again and again. Here, we had only one case of this. We knew it was important because integrated sequencing ruled out all the known cancer genes. That allowed us to focus on what had been changed,” says Michigan Center for Translational Pathology’s lead study author Dan R. Robinson.

Once they found the anomaly, the researchers looked at 50 other tumor samples from benign and cancerous solitary fibrous tumors, looking for the gene fusion. It showed up in every one of the samples. 

“Genetic sequencing is extremely important with rare tumors,” says study co-author  and associate professor of internal medicine at University of Michigan Medical School Scott Schuetze, M.D. “Models of rare cancers to study in the laboratory are either not available or very limited. The sequencing helps us to learn more about the disease that we can use to develop better treatments or to help diagnose the cancer in others.”

This particular fusion may prove to be a difficult target for therapies, but researchers believe they may be able to attack the growth signaling cycle that leads to this gene fusion.

“Understanding the changes induced in the cell by the (NAB2-STAT6) gene fusion will help us to select novel drugs to study in patients with advanced solitary fibrous tumors. Currently this is a disease for which there are no good drug therapies available and patients are in great need of better treatments,” Schuetze says.

No treatments or clinical trials are currently available based on these findings. Additional testing in the lab is needed to assess the best way to target this specific gene fusion. it could also potentially be used to help identify solitary fibrous tumors in cases where diagnosis is challenging.

All information in this blog post has been taken from the University of Michigan Comprehensive Cancer Center website (http://www.cancer.med.umich.edu/giving/gene-sequencing.html). 


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