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BIDMC Researchers Identify Possible Biomarker for Parkinson’s Disease

Although Parkinson’s disease is the second most prevalent neurodegenerative disorder in the U.S., there are no standard clinical tests available to identify this widespread condition. As a result, Parkinson’s disease often goes unrecognized until late in its progression, when the brain’s affected neurons have already been destroyed and telltale motor symptoms such as tremor and rigidity have already appeared.

Researchers Identify a Metabolite as a Biomarker of Diabetes Risk

Type 2 diabetes (T2D) is the most common form of diabetes and is associated with many complications. T2D is preventable through weight control and exercise; however, many individuals are unaware that they are at risk and do not change their lifestyle in time to avoid disease.

Mayo Clinic Researchers Identify Biomarker for Smoker’s Lung Cancer

Mayo Clinic researchers have shown that a specific protein pair may be a successful prognostic biomarker for identifying smoking-related lung cancers. The protein — ASCL1 — is associated with increased expression of the RET oncogene, a particular cancer-causing gene called RET. The findings appear in the online issue of the journal Oncogene.

“This is exciting because we’ve found what we believe to be a ‘drugable target’ here,” says George Vasmatzis, Ph.D., a Mayo Clinic molecular medicine researcher and senior author on the study. “It’s a clear biomarker for aggressive adenocarcinomas. These are the fast-growing cancer cells found in smokers’ lungs.”

ASCL1 is known to control neuroendocrine cell development and was previously linked to regulation of thyroid and small cell lung cancer development, but not smoking-related lung cancer. The research also showed that patients with ASCL1 tumors with high levels of the RET oncogene protein did not survive as long as ASCL1 patients with low levels of RET.

When researchers blocked the ASCL1 protein in lung cancer cell lines expressing both genes, the level of RET decreased and tumor growth slowed. This leads researchers to believe this mechanism will be a promising target for potential drugs and a strong candidate for clinical trials.

The co-authors of the study include Farhad Kosari, Ph.D.; Cristiane Ida, M.D.; Marie Christine Aubry, M.D.; Lin Yang, Ph.D.; Irina Kovtun, Ph.D.; Janet Schaefer Klein; Yan Li, M.D.; Sibel Erdogan; Sandra Tomaszek, M.D.; Stephen Murphy, Ph.D.; Lynn Bolette; Christopher Kolbert; Ping Yang, M.D., Ph.D.; and Dennis Wigle, M.D., Ph.D., all of Mayo Clinic.

The research was supported by a Waterman Biomarker Discovery grant and by the Mayo Clinic Center for Individualized Medicine.

Study: ASCL1 and RET expression defines a clinically relevant subgroup of lung adenocarcinoma characterized by neuroendocrine differentiation [Oncogene]

Source: Mayo Clinic

A Roadblock to Personalized Cancer Care?

There’s a major roadblock to creating personalized cancer care.

Doctors need a way to target treatments to patients most likely to benefit and avoid treating those who will not. Tumor biomarker tests can help do this.

The problem, according to a new commentary paper, is that, unlike drugs or other therapies, cancer biomarker tests are undervalued by doctors and patients. The authors say that inconsistent regulatory rules, inadequate payment and underfunded tumor biomarker research has left us in a vicious cycle that prevents development and testing of reliable biomarker tests that could be used to personalize clinical care of patients with cancer.

“Right now biomarkers are not valued nearly to the extent that we see with therapeutics. But if a tumor biomarker test is being used to decide whether a patient should receive a certain treatment, then it is as critical for patient care as a therapeutic agent. A bad test is as dangerous as a bad drug,” says Daniel F. Hayes, M.D., clinical director of the breast oncology program at the University of Michigan Comprehensive Cancer Center.

Hayes led a blue-ribbon panel of experts from universities, corporations, insurance and advocacy organizations to outline the issues in a commentary published recently in Science Translational Medicine.

Tumor biomarker tests look at the genetic or molecular make-up of a tumor to determine whether the cancer is likely to progress, and if so, if it is likely to respond to treatment. If the test is good, it can help doctors decide when a patient can safely skip further therapy, or it can be used to direct which drug might be most likely to help. The result: “personalized medicine,” which means patients get treatments that benefit them specifically and they avoid treatments – including their costs and side effects – that are not likely to make a difference for them.

The regulatory process, the research funding, the reimbursement, even the standards for journal publications for tumor biomarker tests are all meager compared to the robust support for drug development, the authors say.

This creates a vicious cycle in which researchers and drug companies don’t invest in tumor biomarker research, tests are not fully evaluated in clinical trials, and tests with uncertain value in terms of predicting the success of treatment are published. This in turn means that few of these tests are included in evidence-based care guidelines, leaving health care professionals unsure of whether or how to use the test, and third-party payers unsure of how much to pay for them.

The authors outline five recommendations and suggest that all five must be addressed to break the vicious cycle:

  1. Reform regulatory review of tumor biomarker tests
  2. Increase reimbursement for tumor biomarker tests that are proven to help determine which therapies will or are working
  3. Increase investment for tumor biomarker research so it’s comparable to new drug research
  4. Increase the rigor for peer review of tumor biomarker publications
  5. Include only proven biomarker tests in evidence-based care guidelines

“These recommendations are not about creating more regulation; they are about creating an even playing field that allows tumor biomarker tests to be developed and proven clinically relevant. We want to stimulate innovation yet hold investigators and clinicians to the highest scientific standards – as we now do for therapeutics,” Hayes says. “We need to change the way we value tumor biomarkers in this country.”

Study: Breaking a Vicious Cycle [Science Translational Medicine]

Source: University of Michigan Health System

ASU Scholars Advocate Innovation in Regulatory, Payment Pathways for Personalized Medicine

Two innovative programs designed to improve the availability of emerging medical technologies that can help patients receive more effective, efficient and personalized health care are advanced in a commentary written by a team of scientists and policy experts, including seven from Arizona State University, and published today in Science Translational Medicine.

The March 13 article, “Regulatory and Reimbursement Innovation,” explores the benefits of coverage with evidence development (CED) and parallel review for the regulation and reimbursement of molecular diagnostics. Molecular diagnostics include tests that aid in better prediction, diagnosis, prognosis and treatment of disease through the use of DNA, RNA and proteins.

The U.S. Food and Drug Administration (FDA) requires certain diagnostic tests to provide reasonable assurances of safety and effectiveness before they can be marketed. The Centers for Medicare and Medicaid Services (CMS) determines whether such products are “reasonable and necessary” before they can be covered by Medicare. The FDA and CMS currently are reviewing CED and parallel review for more widespread use, according to the article.

One of the co-authors, ASU Regents’ Professor Gary Marchant, Faculty Director of the Center for Law, Science & Innovation (LSI) at ASU’s Sandra Day O’Connor College of Law, said these tests are expected to improve health outcomes by giving providers optimal treatment choices for their patients.

“It’s a new paradigm of health care,” said Marchant, who founded LSI’s Program on Personalized Medicine, Law & Policy, the nation’s first law-school program that fosters the discipline’s study through collaborative, multidisciplinary evaluation of critical issues. “And it’s a critical time for both of these innovative programs, because agencies of the federal government are actively considering expanding them.

“These tests produce complex algorithms that can help your physician direct your health care, where you should go for treatment and what medications can – and cannot – help you,” he said. “People are dying unnecessarily, and we need to get these diagnostics out more quickly and with better data.”

Regulatory and reimbursement roadblocks hinder the tests’ development and slow their integration into routine care decisions, according to lead author, LSI Research Director Rachel Lindor. A graduate of the law school’s J.D. program who is now completing her M.D. degree at Mayo Medical School, Lindor began researching these issues during an externship in 2011 at the U.S. Department of Health and Human Services.

An LSI-hosted workshop in April 2012, “Potential Solutions to Regulatory and Reimbursement Barrier for Molecular Diagnostics: Parallel Review and Coverage with Evidence Development,” followed. It drew notable experts from government, industry and academia to brainstorm solutions to these barriers; their input led to the now-published article.

“Overall, the group seemed to agree that both of these policies were good in theory, but there were pieces of each that made people skeptical they would actually work,” Lindor said. “We came up with a few changes at the workshop that we thought would make them more attractive for developers who may have products coming up through the pipeline.”

Parallel review enables developers to meet with both CMS and FDA early in a product’s review process, in order to clarify the agencies’ evidentiary expectations and reduce inefficiencies. CED allows CMS to temporarily cover new products not yet supported by sufficient evidence to meet its “reasonable and necessary” coverage threshold while additional data are evaluated.

“Our recommendations on CED focused on trying to streamline the process so developers wouldn’t see it as a hurdle to getting paid for their products – things like smoothing out the way that FDA and CMS work together when they review the same product and shortening the time it takes for CMS to actually start a CED,” Lindor explained. “The group was also concerned about CMS’ hints that CED may be used by local Medicare contractors, so one of our recommendations was that CMS provide some more logistics on how exactly that would work.”

Lindor said tweaks to the parallel review program could make it more amenable to diagnostic test developers and speed the tests’ access to insurers, care providers and patients.

“Our group recommended that CMS give developers some assurance that there will be some benefits to participating, which could be done by shortening the time it takes to go through the process, or by providing more flexibility about what type of payment decisions would come from the review,” Lindor said. “We also recommended that the policy be made available to a broader range of products than it’s open to now.”

Dr. Denis Cortese, Director of ASU’s Health Care Delivery and Policy Program, and a co-author of the article, said “The primary goals of these two programmatic recommendations are to more quickly and accurately determine the appropriate role for new medical technologies in medical practice and patient care.”

Another co-author, Dr. George Poste, Chief Scientist of ASU’s Complex Adaptive Systems Initiative, said molecular diagnostics have enormous potential to increase diagnostic accuracy and increase the efficacy and safety of drugs in multiple diseases.

“Current ambiguities in regulation and reimbursement policies for these new tests are a major barrier to corporate investment and R&D innovation,” Poste said.

Study: Regulatory and Reimbursement Innovation

Source: EurekAlert!