“Stem cell research and regenerative medicine are in an extremely exciting phase right now. We are gaining knowledge very fast and many companies are being formed and are starting clinical trials in different areas,” says Dr Jonas Frisen.

As an example, a first-in-human study was just initiated for Parkinson’s disease patients with the drug product, sNN0031, from the Swedish company NeuroNova. The drug, which is administered into the fluid-filled cavities of the brain, has shown long lasting recovery and formation of new cells in animal models of Parkinson’s disease. Last year, a treatment for ALS entered the clinical trial phase.

Disorders in the brain and nervous system result in more hospitalizations than any other disease group, and treatments entail large costs to society. The research field of neuroscience is one of Sweden’s finest. This had resulted in achievements within numerous areas of basic science with considerable scope to direct clinical applications. These include research advances concerning the origin and repair of nerve cell damage following stroke and spinal cord injury, as well as research into major degenerative diseases such as Parkinson’s and Alzheimer’s.

Dr Frisen is one of Sweden’s leading stem cell researchers, since many years with a focus on nerve stem cells. Among his most recent publications is an article in Science, April 3rd, 2009 where evidence is shown for renewal of heart muscle cells in humans, a result that can be used to develop therapeutic strategies for cardiac pathologies.

NeuroNova AB is a Swedish biopharmaceutical company working with neurogenesis and neuroprotection for treatment of several currently incurable neurodegenerative diseases. Dr Jonas Frisen is the scientific founder of NeuroNova.

Source:
Sabina Bossi

Karolinska Institutet

Genzyme Corp. (NASDAQ: GENZ) and Isis Pharmaceuticals Inc. (NASDAQ: ISIS) today announced that the phase 3 study of mipomersen in patients with homozygous familial hypercholesterolemia (hoFH) met its primary endpoint, with a 25 percent reduction in LDL cholesterol after 26 weeks of treatment, vs. 3 percent for placebo (p<0.001). This study also met each of its three secondary endpoints of reduction in apolipoprotein B, total cholesterol and non-HDL cholesterol (all p<0.001).

Although the patients were on maximally tolerated statins and other lipid-lowering therapies, their average LDL-C at baseline was greater than 400 mg/dL. The reductions observed in the study were in addition to those achieved with the patients’ existing therapeutic regimen. Full data from the study will be presented at a future medical meeting.

“These are promising results for a very high-risk patient population that is in great need of new treatment options,” said Genzyme Chief Medical Officer Richard A. Moscicki, M.D. “This is one of the largest studies of hoFH patients ever conducted, and we are very encouraged by these robust data and the emerging profile of the drug. With these results, we remain on-track with our development plan for mipomersen.”

Consistent with previous studies evaluating mipomersen, the most commonly observed adverse events were injection site reactions, flu-like symptoms and elevations in liver transaminases. Of the 34 patients treated with mipomersen, 28 completed the study. One patient discontinued due to elevations in liver transaminases.

“The results announced today are good news for patients with hoFH,” said John J. P. Kastelein, M.D., Ph.D., Professor of Medicine and Chairman of the Department of Vascular Medicine at the Academic Medical Centre Amsterdam. “The currently available treatments do not provide the magnitude of lipid lowering that these patients need, leaving them at extraordinarily high risk of cardiovascular events. Mipomersen has the potential to change the standard of care for hoFH patients, whose life expectancies are limited due to the severity of this disease.”

“This is a historic moment for Isis and antisense technology as this study represents the first successful phase 3 trial with a systemically delivered antisense drug,” said Isis Pharmaceuticals Chairman and CEO Stanley T. Crooke, M.D., Ph.D. “This drug exemplifies the potential of the antisense drug discovery platform pioneered by Isis. We look forward to benefitting patients in need with mipomersen and other drugs in our pipeline.”

The trial was a randomized, double-blind, placebo-controlled study that enrolled 51 hoFH patients, aged 12 and older. Seven patients were aged 12 to 17. Patients were randomized 2:1 to receive a 200 mg dose of mipomersen or placebo via weekly injections for 26 weeks. The trial was conducted at 10 sites in seven countries in North America, Europe, Asia, South America and Africa.

Data from this phase 3 study of mipomersen in patients with hoFH will form the basis of Genzyme’s initial regulatory filing for marketing approval, which is anticipated in the second half of 2010.

About Familial Hypercholesterolemia (FH)

FH is a genetic disorder in which patients are unable to properly metabolize LDL cholesterol, resulting in elevated LDL-C levels. FH patients experience a markedly increased risk of premature cardiovascular disease (CVD) and CVD-related death. There are two forms of FH: homozygous (hoFH), where the same defective gene is inherited from both parents, or heterozygous (heFH), where the defective gene is inherited from only one parent so that some function is preserved.

The homozygous form of FH is a very rare condition estimated to affect approximately one in a million people. HoFH patients can have LDL-C levels greater than 600 mg/dL and are at very high risk for early coronary events and sudden death. Because many patients are resistant to the lipid-lowering effects of currently available therapies, effective treatment of hoFH patients is difficult. HeFH is a more common form of the disorder, with a prevalence of approximately one in 500, and results in untreated LDL cholesterol levels of approximately 300 mg/dL, double those of the general population.

About Mipomersen

Mipomersen is a first-in-class apo-B synthesis inhibitor currently in late-stage development. It is intended to reduce LDL-C by preventing the formation of atherogenic lipoproteins. Genzyme and Isis are currently conducting several clinical trials of mipomersen, including:

- A phase 3 study in patients with heterozygous FH;
- A phase 3 study in patients with severe hypercholesterolemia;
- A phase 3 study in hypercholesterolemic patients at high risk for coronary heart disease; and
- A phase 2 study in high-risk, high-cholesterol patients who are intolerant to statins.
Data from these trials are expected to be available at the time of the initial hoFH regulatory filing, and will continue to build the body of clinical evidence around the treatment’s value in managing high-risk, high-cholesterol patients.

In 2008, Genzyme and Isis completed a licensing agreement that provides Genzyme with exclusive worldwide rights to mipomersen, which was discovered and initially developed by Isis.

Source
Isis Pharmaceuticals Inc.

Summaries of several developments related to health care reform appear below.

• Rising costs without reform: A failure to overhaul the U.S. health care system could result in 66 million U.S. residents being uninsured and individual and family spending on health care increasing by 68% by 2019, according to a recent study prepared by the Urban Institute, CQ HealthBeat reports. The study, funded by the Robert Wood Johnson Foundation, examined three different scenarios that could occur if the U.S. does not reform its health care system. The study assumed that 49 million U.S. residents currently are uninsured, based on 2007 figures adjusted for coverage losses from the recent rise in unemployment. Under the best-case scenario, 53.1 million people would be uninsured in 2019 and individual and family spending would increase by at least 46%. The 66 million uninsured U.S. residents and the 68% increase in health spending were estimates under the worst-case scenario. In addition, inaction on health care reform could nearly double government expenditures as more U.S. residents become eligible for programs such as Medicaid and CHIP, according to the study. According to RWJF, the total costs from uncompensated care also would increase, thus “putting a tremendous strain on health systems, hospitals, providers of clinical care and local municipalities” (Reichard, CQ HealthBeat, 5/21). The study is available online.
  
  
• AARP: AARP on Thursday called for health care reform legislation to include provisions to close Medicare Part D’s so-called “doughnut hole” coverage gap, CQ HealthBeat reports. In addition, the group said that efforts to lower prescription drug costs should address increased spending on biologics, such as vaccines or blood serums, which often are difficult and costly to produce as generics. AARP said older U.S. residents particularly are affected by the increased spending on biologics because such treatments often are prescribed for conditions such as cancer, arthritis and multiple sclerosis. CQ HealthBeat reports that Congress is expected to take up a bill this year that would accelerate approval for generic biologics through the FDA (CQ HealthBeat, 5/21).
  
  
• Insurance market: Liberal advocacy group Health Care for America Now on Wednesday released a report that found that few insurance companies dominate the market in several states and that the concentration of insurers has led to an increase in the cost of premiums, the Raleigh News & Observer reports. According to the report, premiums on average rose by 75% from 2000 to 2007, while personal income rose only by 14% during the same period. The report also profiled marketplace conditions in several states using 2007 data from the American Medical Association. According to the report, Blue Cross and Blue Shield of North Carolina and UnitedHealth Group control 73% of North Carolina’s private insurance market (Avery, Raleigh News & Observer, 5/21). In Florida, Blue Cross and Blue Shield of Florida and Aetna control a 45% share, the report found (Quintero, Orlando Sentinel, 5/20). In addition, BCBS and UnitedHealthcare control 68% of Texas’ insurance market. According to the Houston Chronicle, the report states that a government-sponsored public plan would not undermine the private insurance market (Ackerman, Houston Chronicle, 5/21). The report is available online.
  
  
• Reform forum: Four “political heavyweights” on Wednesday participated in a forum on health care reform at the Georgia World Congress Center as part of a biotechnology conference, the Atlanta Journal-Constitution reports. At the session, former Senate Majority Leader Bill Frist (R-Tenn.) said that a government-sponsored health insurance plan would hamper innovation and competition and lead to tax increases. Karl Rove, former chief adviser to former President George W. Bush, added, “A public plan is a government-run plan. And a government-run plan is bad.” However, Howard Dean, former chair of the Democratic National Committee, said he supported a government-sponsored public insurance plan that would not eliminate the private sector. Dean said, “We need a public insurance option to give people a choice.” Former Senate Majority Leader Tom Daschle (D-S.D.) said that a public plan would lower health care costs, adding that U.S. residents want Congress to “get something done” on health care and “are very concerned about the implications if we do nothing” (Schneider, Atlanta Journal-Constitution, 5/21).
  
  
• Faith-based campaign: Faith-based groups Faithful America and Cover All Families have launched a campaign on Christian radio stations advocating for health care reform, the Wall Street Journal’s “Washington Wire“ reports. The ads, titled “Abundant Life,” feature a voice saying, “All Americans should be able to get the care they need for their families, when they need it. God desires abundant life for all people. It’s time we step up, ask our politicians to move the debate forward, so we can get the reform we desperately need.” The campaign also urges listeners to contact their congressional representatives about health care reform. The ads are running in seven states — Arkansas, Colorado, Florida, Indiana, Louisiana, Missouri and Nebraska — which have “representatives and senators who may well determine the fate of health reform,” according to CAF (Mundy, “Washington Wire,” Wall Street Journal, 5/21).

Reprinted with kind permission from http://www.kaisernetwork.org. You can view the entire Kaiser Daily Health Policy Report, search the archives, or sign up for email delivery at http://www.kaisernetwork.org/dailyreports/healthpolicy. The Kaiser Daily Health Policy Report is published for kaisernetwork.org, a free service of The Henry J. Kaiser Family Foundation.

© 2009 Advisory Board Company and Kaiser Family Foundation. All rights reserved.