A total personalized medicine approach might be far from becoming a definite reality in the U.S. at the moment, but the method, which could eliminate the current one-size fits all philosophy in administering care, is rapidly gaining ground.

From the discussions on Capital Hill about comparative research over studying disease affects on a general population, to greater effort and funding being put into molecular diagnostics- the case for personalized medicine is moving forward.

“We’re seeing more interest and less skepticism toward [Personalized Medicine],”Ed Abrahams, executive director of the Personalized Medicine Coalition (Washington), told Biomedical Business & Technology. “We’re clearly seeing movement . . . greater investments and a larger number of products available on the market.”

Recently the Personalized Medicine Coalition released the 2nd edition of its 2006 report, The Case for Personalized Medicine, in hopes to further bolster the need to truly turn to this method and perhaps to enter into the current healthcare discussion.

The robust report, which is a living document and subject to more changes in the future, remains true to its namesake and builds a case for personalized medicine, at a time when the nature and future of healthcare are center stage in what promises to be a contentious political debate.

“We hope that the [report] captures the attention of policy makers and that they see what they do now has an impact on healthcare,” Abrahams said.

The report specifically focuses on where the country has come and where it could go given the current interest in personalized medicine. It also shows what advances have been made since the 2006 report was released.

Since that time, the number of prominent examples of personalized medicine treatments and diagnostics has increased from 13 products (69% of which were for cancer) to 37 products (56% of which are for cancer).

Some of those products such as Caris Diagnostics’ (Irving, Texas) Target Now, an oncology testing service and Applied Biosystems’ (Carlsbad,California) SOLiD system, which identifies methylation patterns across the genome, were discussed at length during the 100th annual meeting of the American Association for Cancer Research (AACR; Philadelphia) in Denver.

The report gives other real-world examples of personalized medicine in action and has insights from leading members of industry and government.

Geoffrey Ginsburg, MD, PhD, director of the Center for Genomic Medicine at the Duke Institute for Genome Sciences & Policy (Durham, North Carolina), remarked on how such practices and approaches impacted research at Duke.

“Personalized medicine approaches are beginning to affect patients, researchers, and clinicians in very significant ways,” Ginsburg said. “At Duke, we are conducting a series of genome-guided clinical trials that rely on the genomic fingerprints of disease to predict the best course of treatment for each patient with lung, prostate, and breast cancer. As described in this landmark report from the Personalized Medicine Coalition, such individualization of treatment will become more commonplace in coming years, leading to improvements in clinical outcomes.”

Contraceptive may prevent HIV transmission, pregnancy

Women may someday have a new birth control option designed to not only prevent unplanned pregnancy but also block the transmission of the HIV virus. Researchers from Weill Cornell Medical College (New York) have published results showing that a new contraceptive device may do just that. The results were published in the journal AIDS.

The new device is a vaginal ring that releases multiple types of non-hormonal agents and microbicides, which would prevent conception as well as sexually transmitted HIV infection, according to the researchers.

Worldwide, there are about 5 million new infections and 3 million deaths a year due to HIV/AIDS. If proven successful in future clinical trials, the new device could empower women to effectively and conveniently protect themselves from unintended pregnancy and sexually transmitted infection. The ring may also someday represent a novel method to prevent sexually transmitted infections for those with aversion to currently available methods, with hormonally derived active agents, or with allergies to latex condoms, the researchers said.

“This device is a new approach to birth control, because it avoids the long-term use of hormonal methods that have been associated with increased risk of certain cancers,” said Brij Saxena, MD, lead author and the Harold and Percy Uris Professor of Reproductive Biology and professor of endocrinology in the department of obstetrics and gynecology at Weill Cornell Medical College. “At the same time, this is the first device to simultaneously offer the possibility to prevent unintended pregnancy and HIV transmission.”

Saxena told BB&T that long-term use of hormones have side effects. Women are always at risk for infection with HIV, he said, because it is transmitted during heterosexual contact. With this vaginal ring, “women will have control themselves to protect them [from] unintended pregnancy and HIV infection.

He said the device can provide sustained release of both contraceptives and microbicide simultaneously in vitro at the concentration “which is efficacious and significantly below the toxic levels.”

The vaginally inserted ring is incorporated with multiple antiviral drugs that prevent HIV infection and are time-released over a period up to 28 days. The compounds tested were a newly developed anti-HIV agent, Boc-lysinated betulonic acid, TMC120 (dapivirine), PMPA, and 3’-azido-3’-deoxythymidine (AZT or zidovudine), which, when combined, were found to block infection in human cells exposed to the virus in a laboratory setting.

“No one has ever conquered a viral epidemic with treatment, so prevention is the most effective option. Ideally, an HIV vaccine is the most desirable method, but that is not foreseeable in the near future,” said Jeffrey Laurence, MD, co-author of the study and attending physician at NewYork-Presbyterian Hospital/ Weill Cornell Medical Center. “The next best thing would be something that would prevent infection and put the power in the susceptible female partner’s control. That’s the potential a device such as this can offer.”

When asked to provide a crystal ball guess as to when this new device might be available to women in the U.S., Saxena said it may be a couple of years. He said the compounds used in the device are natural materials that are already FDA approved for human use. However, the device itself may still be subject to FDA approval, he noted.

“The combination of these antiviral drugs has proven to be potent agents that may block infection by the HIV virus,” Saxena said.

Some progress, much hype in stem cells

A new era for stem cell research could lie ahead in the U.S., thanks to President Barack Obama’s lifting of the ban on stem cell research and the subsequent FDA decision allowing Geron Corp. to begin the first embryonic stem cell-based clinical trial in the U.S. But this good news may have to be taken with a large grain of salt.

Instead of a talk on cell fate in stem cells, one talk at the BIO International Convention in Atlanta in May focused more on the fate of stem cell research. According to Steve Burrill, CEO of Burrill & Company, “Most everything we are talking about doing is still being done in the academic world.” Public expectation of what stem cells can do in the clinic has gotten ahead of reality, he said. “The public has never been as engaged in anything.”

He added, “The regulatory world, all over the world, is very unsettled about what stem cell therapies are or should be.”

Some countries are more permissive than others and clinical use of stem cells is often not based on rigorous, evidence-based science. In the U.S., “pharmacovigilance trumps approval,” said Burrill. “That will be a barrier,” he added. The U.S. “will then be a late adaptor” compared to other nations. Given that stem cell research is in the early, high-risk stage, Burrill said, “It’s an unlikely place for VCs to invest,” although “the VCs as a whole, like stem cells.”

Rich Gregory, senior vice president and head of research at Genzyme (Cambridge, Massachusetts), said, “I believe the well-controlled system is good.” However, anything that could accelerate product development would help increase investor interest, he said. He said he believes stem cells “will probably remain a cottage industry for a long time,” adding “The industrialization of that process is what I think will take years.”

In a recent Marks & Clerk (London) survey of international biotech executives, there was widespread belief that the Obama stem cell policy reform would improve the regenerative playing field for the U.S.; however, 40% also believed there is significant risk of further biotech failures, particularly companies in early development.

Lamentably, most stem cell companies fall into this category. According to Genzyme, there currently are about 100 stem cell companies, globally. Although few in number, their influence on the future of healthcare may be incalculable.

Stem cells have already had a tremendous impact. Although human stem cells were first isolated in 1998, they have been used in humans for decades longer. At another BIO session, Andrew Balber of Duke University (Durham, North Carolina), said, “Bone marrow transplant is the father of stem cell therapy,” since it has been around for 50 years, and use of umbilical cord blood for 21 years. Genzyme has had the autologous chondrocyte therapy Carticel for 10 years.

Nicholas Chronos, president of St. Joseph’s Translational Research Institute (Atlanta), said capillaries or even fully functional blood vessels are being grown. Collapsed vessels in advanced diabetics lead to $40,000 limb amputations as the only option. In these critical limb ischemia patients, infusing bone marrow helps blood vessels reconform, so circulation improves. Also, skeletal myoblasts have been taken from a French patient’s leg and injected into her damaged heart, successfully repairing myocardial tissue.

Autologous cell therapy using keratinocytes for burn victims has also taken place. Ian Ratcliffe, CEO of Stemgent, cited the example of a recent $600,000 windpipe reconstruction, adding, “To make it an efficient health care vehicle…we are a long way from that.” Genzyme’s Gregory said, “Cell therapies are one of the most complex…on the other hand, the potential is even greater.”

Jan Nolta, director of the Stem Cell Program at the University of California (Berkeley), presented preclinical data on adult stem cells. These “paramedics of the body” have robust hematopoietic capacity and automatically migrate to hypoxia-damaged or inflamed tissues, while control cells lacking high levels of aldehyde dehydrogenase (ALDH), a conserved stem cell marker in mice and men, do not.

To confirm findings, glowing iron nanodust was added to the stems. Tracked by imaging, they went specifically to a hypoxic area of murine femoral artery ligation. Moreover, in a week there was blood flow and by 21 days, all appeared normal.

Andrew Balber, CSO of Aldagen, currently has four products in the clinic: Two are derived from cord blood, good for hematopoiesis; and two for bone marrow. In a mechanistic inquiry, the ALDH bright (br) cells were confirmed as the active component. Marrow is similar to cords, but half the ALDHbr cells differ, providing tissue repair capacity: ALD-301 will treat critical limb ischemia and ALD-201 is for heart repair.

Balber said, “In FDA jargon, these are minimally manipulated cells” since they are treated, but not cultured or manipulated, and re-implanted within 36 hours.

Ndeadline to define term ‘meaningful user’

The Office of the National Coordinator for Healthcare Information Technology (ONCHIT) has published a plan for making use of the funds provided in the American Recovery and Reinvestment Act of 2009 (ARRA), detailing both funding sums and timelines for specific objectives. However, one of the key questions for doctors and hospitals, that of the definition of “meaningful user,” does not come with a deadline in the document.

The meaningful user question has surfaced frequently over the past few weeks as physicians and hospital administrators attempt to plan their HIT purchases. During a session at the April edition of the World Health Care Congress in Washington, Kelly Cronin, the director of the office for programs and coordination at ONCHIT, said that the definition of meaningful user is not likely to be static, but made no mention of when ONCHIT might provide a definition. According to the ONCHIT document, the definition “will be determined through a process that will include broad stakeholder input and discussion.

Of the $2 billion allocated to ONCHIT’s efforts, $300 million will go toward bolstering the operations of regional HIT exchanges, also known as regional health information organizations (RHIOs). The National Institute of Standards and Technology will use another $20 million to help the Department of Health and Human Services craft rules that guide standards for electronic health records (EHRs), with a deadline of Dec. 31 to publish a proposed final standard in the Federal Register.

Roughly $1.6 billion of the total amount is allocated to “unspecified” uses, no doubt some of which will go toward bolstering IT infrastructure in hospitals and doctors’ offices. One of the measures ONCHIT is employing to gauge its success is to get one of every four physician offices to have adopted EHRs by February 2010. The document states that 21% of physician practices have EHRs in 2008. For small physician practices, a term that is not defined, the objective is to have 12% working with EHRs, a substantial percentage increase from the current 8%, although the document does not indicate the sheer numbers involved.

DoJ, HHS ‘HEAT’ things up

The Department of Justice and the Department of Health and Human Services have reported a plan to boost efforts to combat Medicare and Medicaid fraud, concentrating the effort “among a group of key stakeholders at the highest levels of DoJ and HHS,” according to May comments by Attorney General Eric Holder.

Fraud has been the bane of Medicare and Medicaid for a number of years, and the Office of Inspector General (OIG) at HHS has put substantial resources into the problem. All the same, the thousands of providers of durable medical equipment and the antiquated nature of much of the information technology infrastructure at the Centers for Medicare & Medicaid Services has made it difficult for authorities to chase down and prosecute fraudulent billings.

Holder described the program as “part of the administration’s ongoing commitment to fiscal responsibility and accountability,” and noted that since 1997, “when Congress established the Health Care Fraud and Abuse Control program, our departments have returned more than $14.3 billion to the federal government.”

HHS and DoJ, Holder said, are “launching a new effort with increased tools, resources and a sustained focus by senior-level leadership” in the form of a task force dubbed the Health Care Fraud Prevention & Enforcement Action Team (HEAT), that is geared toward improving coordination and data sharing between the two departments’ investigators and prosecutors. However, HHS will have to keep DoJ up to date on approvals and indications for drugs and devices in order to properly leverage planned boosts to information technology and employee training.

Holder mentioned that inter-department coordination in the form of Medicare fraud strike forces have cleaned up things in the state of Florida substantially. He said that efforts in the Sunshine State allowed prosecutors in Miami, Florida, to file 87 indictments for 159 defendants in 2007. This, Holder said, led to a drop in billing for durable medical equipment of $1.75 billion in claims and $334 million in payments, adding that the action has had a deterrent effect. A similar effort the following year in California led to indictments of 37 alleged perpetrators of fraud.

Holder said the HEAT group will consult with the FBI and the Office of Inspector General at HHS in addition to state offices of attorneys general.

HHS Secretary Kathleen Sibelius said at the press conference held on the task force announcement that the federal government is “turning up the heat on perpetrators who steal from the taxpayers and threaten the future of Medicare and Medicaid,” adding that “we cannot and will not allow billions of dollars to be stolen from Medicare and Medicaid through fraud, waste and serious abuse of the system.” She said that the improvements to the IT base in pursuit of fraud will “bring the fight . . . into the 21st century and put the resources on the streets and out into the community to protect the American taxpayers and lower the cost of healthcare.”