Vertex Hep C Treatment Passes Midstage Milestone

Vertex Hep C Treatment Passes Midstage Milestone
Drug Discovery & Development - March 31, 2011

CAMBRIDGE, Mass. (AP) - Vertex Pharmaceuticals Inc. said that a potential hepatitis C treatment was safe and tolerable in a midstage study.
The study involved a combination of telaprevir and VX-222 with pegylated-interferon and ribavirin and focused on people with genotype 1 chronic hepatitis C who were new to treatment. Pegylated-interferon and ribavirin are already used to treat hepatitis C.
Previously, the company halted other arms of its telaprevir and VX-222 study because the combination wasn't working.
Telaprevir is Vertex's primary drug candidate.
Date: March 31, 2011
Source: Associated Press

Phase 3 Study Begins for ALS Treatment

Phase 3 Study Begins for ALS Treatment
Drug Discovery & Development - March 31, 2011

Biogen Idec and Knopp Biosciences announced enrollment of the first patient in EMPOWER, a multi-national Phase 3 study evaluating the efficacy, safety and pharmacokinetics of dexpramipexole in patients with amyotrophic lateral sclerosis (ALS). ALS, also known as Lou Gehrig’s disease and motor neuron disease (MND), is a rapidly progressive and degenerative disease of motor neurons in the brain and spinal cord. It causes muscle atrophy and spasticity, resulting in weakness, progressive paralysis and ultimately death, usually by respiratory failure.
“ALS is a serious disorder that affects patients in the prime of life and for which there is an urgent need for new and effective treatments," said Merit Cudkowicz, M.D., MSc, Director of the MDA ALS Clinic at Massachusetts General Hospital and Principal Investigator in the EMPOWER study. "In the past several years, there has been an explosion of research in ALS that has helped us better understand the disease and potentially pave the way for the discovery and development of new treatments for ALS.”
EMPOWER is a Phase 3, randomized, double-blind, placebo-controlled, parallel-group, multi-center study that will assess the safety and efficacy of dexpramipexole in people with familial or sporadic ALS. Approximately 804 patients will be randomized in a one-to-one ratio to receive either dexpramipexole 150 mg twice daily or placebo and will be followed for a period of at least 12 months. Investigators also have the option to maintain treatment with the current standard-of-care in ALS for all patients enrolled in the study. The primary objective will be assessed using a joint ranking of functional outcomes adjusted for mortality on the ALS Functional Rating Scale, Revised (ALSFRS-R), a validated rating instrument used by physicians for monitoring the progression of disability in patients with ALS.
“Biogen Idec is committed to working with the ALS community to find new treatment options for this deadly disease and to improve the lives of people with ALS,” said Alfred Sandrock, M.D., Ph.D., Senior Vice President of Development at Biogen Idec. “Based on promising data from earlier clinical trials, we believe that dexpramipexole has the potential to be a significant advance for people suffering from ALS, and we will fully explore its potential as a new treatment for this devastating disease.”
“Knopp Biosciences is very pleased to see dexpramipexole entering Phase 3 development with the initiation of the EMPOWER study,” said Michael Bozik, M.D., President and CEO of Knopp. “The promise of dexpramipexole has brought together a motivated multi-national network of investigators and study coordinators committed to determining if dexpramipexole can fulfill its potential as a treatment for patients with ALS.”
Dexpramipexole was well-tolerated in three Phase I studies in healthy volunteers who received dexpramipexole in single doses up to 300 mg or multiple doses of up to 150 mg twice daily for four-and-a-half days. Dexpramipexole was also well-tolerated in a two-part Phase II study (CL201) that evaluated about 100 ALS patients treated for up to nine months and in a subsequent Phase II extension study (CL211) that has continued to follow ALS patients for about two additional years. In the first part of CL201, dexpramipexole at 150 mg twice daily showed a trend toward slowing functional decline over a 12-week period compared to placebo. Following re-randomization in the second part of CL201, dexpramipexole at 150 mg twice daily again showed a trend toward slowing functional decline and also showed a trend toward improving survival compared to low dose over a six-month period. In CL201, the joint ranking, which incorporates both function and survival and which is the primary endpoint of the Phase 3 trial, significantly favored dexpramipexole 150 mg twice daily compared to 25 mg twice daily.
Biogen Idec has agreed with the U.S. Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for the design of the Phase 3 clinical trial of dexpramipexole. Under an SPA, the FDA evaluates a clinical trial protocol to assess whether it is adequate to meet current scientific and regulatory requirements for a potential application to market a new drug.
The first patient dosed in the EMPOWER study triggers a $10 million milestone payment from Biogen Idec to Knopp.
Date: March 31, 2011
Source: Biogen Idec http://www.biogenidec.com/

Clinical Trial Electronic Data Capture Solutions: Elevating Efficiency and Insight

Clinical Trial Electronic Data Capture Solutions: Elevating Efficiency and Insight
Simon Brooks, Senior Director Product Strategy, Oracle Health Sciences
Drug Discovery & Development - March 31, 2011

The clinical trial process has reached new levels of complexity. Trials are longer, more expansive, more complex in design, increasingly global, and require new types of data. These changes mean that investigative sites must grapple with more subjects, visits, forms, and data validation, as well as multiple applications. Trials are also more costly, driving the need for faster insight into trial and site progress so that sponsors can make more informed decisions about the path forward.
Electronic data capture (EDC) technology has become the de facto standard for gathering and recording clinical trial data. The technology has matured over the years to keep pace with these rapidly changing clinical trial requirements, but it is also important to keep pace with the increasing proficiency and expectations of EDC end users.
EDC solution providers are redoubling efforts to develop solutions that are more efficient for this new generation of front-line users and also expand data visibility to cover all areas of study progress. Helping trial managers and sponsors expedite collection analysis of all their clinical data allows deeper insights and more timely decisions, speeding the all-important time to market.
Helping the front-line
Improved efficiency and usability are key concerns for EDC users on the front lines of data collection and management. Clinical research professionals are looking for systems that do more than simply present data; they need solutions that quickly and easily direct them to key tasks and help complete them successfully—whether registering patients, managing and cleaning data, or monitoring.
EDC solution providers are working to answer this call.  For example, Oracle Health Sciences’ InForm GTM EDC solution has a redesigned user interface that helps users make quicker decisions, reduce errors and simplify navigation.
In creating the new InForm GTM interface, three usability design principles were applied:
• Signal-to-noise ratio, ensuring that important information stands out from peripheral information on a screen
• Information proximity, making certain that information needed to complete a task is grouped together on a screen for greater efficiency
• Streamlined workflow, simplifying the progression of documents and forms, as well as task delegation, from employee to employee or from sponsor to site
Leveraging these principles, InForm GTM’s interface features updated icons that make follow-up actions quicker to recognize and new controls that make it easier to navigate and filter. It also features improved query workflow that simplifies response exchange to speed query resolution; the user-friendly page layout also allows for more information to be viewed per page without scrolling.
Improving insight for managers and sponsors
With the cost of clinical trials continuing to escalate, study managers and sponsors need timely data on study progress to enable informed decisions about potential mid-study changes. Real-time reliable data and analytics are essential; InForm GTM provides on-demand and scheduled data extracts, as well as integrated advanced reporting that uses real-time data. These capabilities enable sponsors and managers to perform both ad hoc review and structured analysis earlier, and to more accurately spot trends and/or identify anomalies and move quickly to address them.
Electronic data capture solutions have hit their stride and are an indispensible clinical trial data management tool. A concerted focus to deliver expanded efficiency for front-line users—and real-time insight for trial managers and sponsors—will continue to fuel adoption and success.

Medicare to Cover Pricy Prostate Drug

Medicare to Cover Pricy Prostate Drug
Matthew Perrone
Drug Discovery & Development - March 31, 2011

WASHINGTON (AP) - Medicare officials said that the program will pay the $93,000 cost of prostate cancer drug Provenge, an innovative therapy that typically gives men suffering from an incurable stage of the disease an extra four months to live.
The Centers for Medicare and Medicaid said the biotech drug made by Dendreon Corp. is a "reasonable and necessary" medicine. The decision ensures that millions of men would be able to afford the drug through the government-backed health care coverage. With government reimbursement, analysts estimate Provenge could rack up $1 billion in sales next year. The decision, which will be finalized by June 30, is important for Dendreon because most prostate cancer patients are 65 or older.
Medicare is legally prohibited from considering price when deciding whether to pay for a new treatment. The Food and Drug Administration approved Provenge last April and in most cases Medicare automatically covers drugs cleared by the agency. But Medicare's decision to review Provenge last year prompted outrage from some patients and doctors who said the government was looking for a reason to avoid reimbursing for the pricey drug.
The infused drug is a first-of-a-kind treatment in that each dose is customized to work with a patient's immune system. Seattle-based Dendreon says Provenge's price reflects the more than $1 billion spent researching and developing the drug. And prostate cancer patients point out that the median survival time with Provenge is double that of chemotherapy, which is about two months and is marked by significant side effects.
"It's impossible to put a dollar figure on a human life, especially when you're talking about a drug that has such mild side effects," said Jim Kiefert, a prostate cancer patient and advocate who was part of the Provenge study. "Of all the treatments I've had - with surgery, radiation and hormone treatment - Provenge had fewer side effects than any of them."
But bioethicists who study health care decisions say Medicare's ruling on Provenge mirrors the bias of the overall U.S. health system, which emphasizes expensive treatments over basic medical care. Health care costs account for nearly one fifth of the U.S. economy, more than any other country.
"We tend to put our health care dollars into very high-tech interventions that produce very marginal improvements," said Dr. Steven Miles, a professor at the University of Minnesota's Center for Bioethics. "The problem is that we have created a health care system that is uniquely inadequate in terms of access to primary health care, which is where you get the most bang for your buck."
A growing number of biologically engineered cancer drugs are being priced in the $100,000 range, including therapies from Roche and Eli Lilly & Co. Last week, Bristol-Myers Squibb Co. received approval for a new melanoma drug that will be priced at roughly $120,000 per patient.
Provenge is the first FDA-approved cancer drug that uses the body's own immune system to fight the disease, offering an alternative to chemotherapy drugs that attack cancerous and healthy cells at the same time. The treatment is intended for men whose prostate cancer has spread elsewhere in the body and is not responding to hormone therapy or radiation.
Each regimen of Provenge must be tailored to the immune system of the patient using a time-consuming formulation process. Doctors collect special blood cells from each patient that help the immune system recognize cancer as a threat. The cells are mixed with a protein found on most prostate cancer cells and another substance to rev up the immune system, and then given back to the patient as three infusions two weeks apart.
Industry observers say much of the scrutiny of Provenge stems from the current political climate, as voters push lawmakers to cut the deficit and rein in government spending. At the same time, the new health care reform law has stoked debate about how much say the government should have in approving coverage of medical treatments.
Republicans have accused the acting chief of Medicare, Dr. Donald Berwick, of advocating health care rationing, based on statements he made as a professor at Harvard University. President Obama appointed Berwick to the post last year, but Senate Republicans have opposed his confirmation and have urged Obama to nominate someone else.
Dr. Sean Tunis, a former Medicare official, called Wednesday's decision a case of "the dog that didn't bark."
"You could almost guess that this would be outcome before they even started. So why raise all the anxiety and subject yourself to all the criticism of rationing?" said Tunis, who is director of the Center for Medical Technology Policy in Baltimore.
Medicare officials have said the agency's review of Provenge was aimed at clearing up bureaucratic confusion among Medicare carriers across the country, some of whom already pay for Provenge. Medicare on Wednesday called its online memo a "proposed decision," but it essentially amounts to agreeing to cover the drug for millions of seniors enrolled in its program.
Date: March 31, 2011
Source: Associated Press

Biolex Announces Hepatitis Trial Data

Biolex Announces Hepatitis Trial Data
Drug Discovery & Development - April 01, 2011

Biolex Therapeutics, Inc. announced that final 72-week results from its SELECT-2 Phase 2b trial of Locteron for the treatment of hepatitis C were presented at the 46th Annual Meeting of the European Association for the Study of the Liver (EASL) in Berlin, Germany.  Data presented show that Locteron achieved the SELECT-2 study objectives by demonstrating viral kinetics and response rates that were comparable with or exceeded the PEG-Intron control while also achieving a statistically significant reduction in flu-like adverse events, reduced rates of depression, lower use of concomitant medications and a reduced rate of discontinuation due to adverse events.  Locteron, the only controlled-release interferon alpha, is designed to offer key tolerability and dosing advantages over currently marketed interferons and serve as a core component of new combination therapies as the treatment of hepatitis C evolves to triple- and quad-drug regimens.
Locteron Dosing Convenience and Efficacy
Locteron is administered once every other week and requires half as many injections as the currently marketed interferons, each of which are injected once per week.  In SELECT-2, the sustained virologic response rate (SVR) for each of the three Locteron doses studied was comparable with or exceeded the response rate for the PEG-Intron control.
Locteron Tolerability Advantages
A major objective of the SELECT-2 trial was to further demonstrate the ability of Locteron’s controlled-release mechanism to improve patient tolerability.  In SELECT-2, patients treated with Locteron experienced a significant reduction in the frequency and severity of flu-like adverse events, reduced use of concomitant (analgesic/antipyretic) medications and reduced rates of depression compared to patients treated with the PEG-Intron control.
The SELECT-2 results were presented by the lead author, Eric Lawitz, MD, Medical Director and Principal Investigator, Alamo Medical Research, in a poster titled “SVR for Controlled-Release Interferon Alpha-2b (CR2b) + Ribavirin Compared to Pegylated Interferon Alpha-2b (Peg2b) + Ribavirin in Treatment-Naïve Genotype-1 (G1) Hepatitis C: Final Results from SELECT-2.”
“The results from the SELECT-2 trial are certainly consistent with the promise of this drug candidate as a next-generation interferon,” said Dr. Lawitz. “The ability of Locteron to contribute to the achievement of comparable viral cure rates with once-every-two-week dosing is a major improvement over current interferons.  I am also impressed by the consistency of the Locteron tolerability advantages seen across different outcome measures and reporting methodologies.  I look forward to seeing the development of Locteron expanded to testing in combination with direct-acting anti-viral agents.”
In SELECT-2, flu-like adverse events were predefined to include arthralgia, chills, fever, headache, and myalgia and were captured in two ways:
• Traditional weekly adverse event assessments performed by medical personnel at the clinical sites during the entire 48 weeks of treatment.
• Daily electronic patient reported outcome (ePRO) system where patients directly reported their flu-like adverse events each day for the first 12 weeks of the trial.
As the ePRO results were reported in real-time directly by the patients, they provide important insight into the patients’ real-world experiences with these side effects and the impact on their daily activities.  A comparison of the ePRO and clinic site reporting highlights the fact that flu-like adverse events may be even more important to patients than historically believed. The total flu-like adverse events reported directly by the patients using the ePRO system during the first 12 weeks of SELECT-2 were almost five times greater than the total flu-like adverse events recorded by the clinical sites.  Also of importance, patients rated 77% of their flu-like adverse events as moderate or severe in their ePRO reports, compared to the clinical site reporting in which only 16% of flu-like adverse events were rated as moderate or severe. Despite the apparent differences in sensitivity in the two adverse event reporting methods, the results from both the ePRO and weekly clinic visits complement each other and each demonstrates the substantial reduction in flu-like adverse events for patients treated with Locteron compared to patients treated with PEG-Intron.
“The ePRO results underscore the impact that flu-like adverse events have on patients’ daily lives and highlight the potential contribution of Locteron to the future success of multi-drug treatment combinations,” said Zobair Younossi, MD, Professor of Medicine and Executive Director of the Center for Liver Diseases at Inova Fairfax Hospital.  “Treatment of hepatitis C is likely to progress to triple and quad therapies in which interferons are combined with one or more direct-acting anti-viral drugs.  If confirmed in phase III clinical trials, Locteron’s potential advantages with regards to flu-like adverse events, depression and dosing frequency have the potential to enhance the overall tolerability of, and patient adherence to, these future combinations.”
Consistent with the reduction in flu-like adverse events, fewer Locteron patients used concomitant medications (analgesics and antipyretics) compared to PEG-Intron patients during the study period.
Date: March 31, 2011
Source: Polaris Group 

FDA Approves ADI-PEG 20 SPA

FDA Approves ADI-PEG 20 SPA
Drug Discovery & Development - April 01, 2011

Polaris Group announced that the U.S. Food & Drug Administration (FDA) has approved the company’s Special Protocol Assessment (SPA) for a Phase 3 clinical trial of pegylated arginine deiminase (ADI-PEG 20) in patients with advanced hepatocellular carcinoma (HCC). The randomized, placebo-controlled, global trial will enroll approximately 600 patients who have failed prior systemic chemotherapy. The primary endpoint of the study is overall survival.
Ghassan Abou-Alfa, M.D., of Memorial Sloan-Kettering Cancer Center in New York, is the principal investigator of the study.

“ADI-PEG 20 has demonstrated medical benefit in prior studies in multiple indications with a remarkable safety profile,” said Dr. Abou-Alfa. “I am delighted to lead this study to explore ADI-PEG 20 as a viable option for patients with advanced liver malignancies.”

SPA is a mechanism that allows the FDA and drug developers to reach an agreement prior to study initiation about the design and size of a Phase 3 trial. It will form the primary basis of an efficacy claim in a marketing application.
“The SPA agreement with the FDA is a key step in our plans for the clinical development of ADI-PEG 20,” said John Bomalaski, M.D., executive vice president, medical affairs, of Polaris. “We are pleased to move ADI-PEG 20 into a pivotal Phase 3 trial and to be working with such well-established hepatocellular carcinoma experts.”
Leo Chen, M.D., the lead investigator of the Phase 2 HCC study in Taiwan, stated, “Our Phase 2 study demonstrated that ADI-PEG 20 was effective and well-tolerated in patients with advanced liver cancer, and we look forward to the opportunity to further assess the efficacy and safety of this drug in a large randomized Phase 3 study.”

The lead investigator in China, Shukui Qin, M.D., president of the Chinese Society of Clinical Oncology and chair of the Chinese Liver Cancer Study Group, added, “Approximately half of the liver cancer-related deaths in the world are in China. We have been watching the development of this drug, and we are excited to become an active participant in this pivotal global study.”
ADI-PEG 20 is a biologic being developed to treat cancers carrying a major metabolic defect that renders them, unlike normal cells, unable to make arginine internally. Because arginine is one of the 20 amino acids that are essential for protein synthesis and survival of cells, these cells become dependent upon the external supply of arginine to survive and grow. ADI-PEG 20 works by systemically depleting the external supply of arginine and causes these arginine-dependent cancer cells to die while leaving the normal cells unharmed.

In addition to HCC, multiple other cancers have been reported to have a high degree of arginine-dependency. Early-to-mid-stage clinical trials have yielded positive results in patients with metastatic melanoma, and Phase 2 trials for small cell lung cancer (SCLC) and mesothelioma are currently ongoing. Polaris also plans to initiate clinical studies in prostate cancer, pancreatic cancer, leukemia, lymphoma and sarcoma this year.
Date: March 31, 2011
Source: Polaris Group 

BMS Releases HCV Study Results

BMS Releases HCV Study Results
Drug Discovery & Development - April 01, 2011

Bristol-Myers Squibb Company announced results from a Phase 2 clinical trial in which treatment with the investigational direct-acting antiviral (DAA) BMS-790052, an NS5A replication complex inhibitor, in combination with PEG-Interferon alfa and ribavirin (RBV), achieved sustained virologic response 12 weeks post-treatment (SVR12) in up to 92% of treatment-naïve patients chronically infected with hepatitis C (HCV) genotype 1 (10 mg dose arm, n=12). Adverse events and serious adverse events were consistent with those reported in the PEG-Interferon alfa and ribavirin arm and were comparable across all doses of BMS-790052. These data were reported today for the first time in a late-breaker poster session at the International Liver Congress, the 46th annual meeting of the European Association for the Study of the Liver (EASL) in Berlin, Germany.
“There currently exists a medical need for new medicines or new combinations of medicines for hepatitis C patients as many hepatitis C patients have limited success on the currently available treatments,” said Stanislas Pol, MD, PhD, Professor of Hepatology at Université Paris V (René Descartes), Paris, France and head of the Hepatology unit at Cochin Hospital, Paris, France. “The results of this study warrant further clinical investigation of adding Bristol-Myers Squibb’s investigational compound BMS-790052 to the current medicines to evaluate its potential to address this unmet treatment need.”
Date: March 31, 2011
Source: Bristol-Myers Squibb Company 

Sylatron Approved in Melanoma

Sylatron Approved in Melanoma
Drug Discovery & Development - April 01, 2011

The U. S. Food and Drug Administration approved peginterferon alfa-2b (Sylatron, Schering Corporation), for the treatment of patients with melanoma with microscopic or gross nodal involvement within 84 days of definitive surgical resection including complete lymphadenectomy.
The approval was based on a single trial, EORTC 18991, an open label, multi-center trial enrolling 1256 patients.  Patients who had been adequately surgically resected for their primary cutaneous melanoma and affected regional lymph nodes were randomized (1:1) to receive either Sylatron or observation for a 5 year period. Stratification factors included type of nodal involvement (microscopic versus gross), number of positive nodes (1, 2-4, 5 or more, or not assessed), Breslow primary thickness (less than 1.5 mm, greater than or equal to 1.5 to 4 mm, greater than or equal to 4 mm), ulceration of primary tumor (present or absent or unknown), sex, and study center.  Patients were assessed for local and regional recurrence or distant metastases every three months for the first two years of treatment and subsequently every six months through the end of the trial.  An independent review committee, blinded to randomization assignment and to information that would break the treatment blind, reviewed the case report form data to determine the occurrence, and the date of loco-regional recurrence, or distant metastasis.
The primary efficacy endpoint, relapse-free survival (RFS), was defined as the time to the earliest of local or regional recurrence, distant metastases, or death. Based on 696 RFS events, an improvement in RFS for Sylatron-treated patients [hazard ratio 0.82 (95% CI: 0.71, 0.96); unstratified log-rank p = 0.011] was observed.  The estimated median RFS was 34.8 months (95% CI: 26.1, 47.4) and 25.5 months (95% CI: 19.6, 30.8) in the Sylatron and observation arms, respectively.  Following 525 deaths on study, there was no difference in overall survival between the Sylatron and the observation arms [hazard ratio 0.98 (95% CI: 0.82, 1.16)].
Safety was evaluated in 608 Sylatron-treated patients in EORTC 18991.  The most common (>60%) grade 1-4 adverse reactions experienced by Sylatron-treated patients were fatigue, increased ALT, increased AST, pyrexia, headache, anorexia, myalgia, nausea, chills, and injection site reactions.  The most common serious adverse reactions occurring in Sylatron-treated patients were fatigue, increased ALT, increased AST, and pyrexia.
Thirty-three percent of patients receiving Sylatron discontinued treatment due to adverse reactions.  The most common adverse reactions present at the time of treatment discontinuation were fatigue, depression, anorexia, increased ALT, increased AST, myalgia, nausea, headache, and pyrexia.  Five deaths were reported within 30 days of the last Sylatron dose.  Two were attributed to recurrent disease, two to cardiovascular disease possibly related to Sylatron, and one to an accident.
The recommended dose and schedule for Sylatron is 6 mcg/kg/week, subcutaneously for 8 doses, followed by 3 mcg/kg/week subcutaneously.  The maximum treatment period is 5 years (260 weeks).
Date: March 30, 2011
Source: U. S. Food and Drug Administration 

New Study into Possible Cholesterol-Autism Link

New Study into Possible Cholesterol-Autism Link
Drug Discovery & Development - April 01, 2011

Researchers at The Ohio State University Medical Center are studying whether simple nutritional intervention – adding cholesterol to the diets of children with autism spectrum disorders after a test to see if they need it – can improve core autism symptoms.
In excess, cholesterol can be harmful, but a certain amount is crucial for the proper development and maintenance of the brain. So it stands to reason that lower levels of cholesterol, particularly during crucial periods of growth, can lead to mental dysfunction, said principal investigator Dr. L. Eugene Arnold, a child psychiatrist at Ohio State’s Nisonger Center who specializes in researching and treating autism.
“We think low cholesterol may be contributing to the core autism symptoms of a subgroup of children with autism spectrum disorders,” said Arnold. “Cholesterol is not only in the food we eat, but we also make cholesterol, which is necessary for normal brain development and for the manufacture of vitamin D from sunlight. Vitamin D is necessary for brain development, so there are several possible ways that having low levels of cholesterol can affect the brain.”
Some children with autism appear to be typically developing before age 2 and then suddenly “regress,” losing language or social skills they had previously developed. Children with autism often have difficulty with pretend play, social interactions and verbal and nonverbal communication.
Researchers have noticed that cholesterol levels in children with autism spectrum disorders tend to be below the normal range compared to the general population. Normal or healthy cholesterol levels range from 120 to 190, but in up to 10 percent of children with autism, cholesterol levels are below 120, and in some cases, even below 100, Arnold said.
“The lower levels of cholesterol could be attributed to poor absorption or a missing enzyme to make the cholesterol,” said Arnold. “There are many different causes of autism, of course, from genetic through environmental, so what we’re investigating here will not apply to all children or people with autism, but it may help a subgroup.”
Too much cholesterol can do more harm than good, especially when it comes to cardiovascular health, so dosages will be closely monitored to find the optimal dose.
“It’s very important that we not just rush out and try to give everyone with autism more cholesterol, because for some of them it may be harmful,” Arnold said.
Ohio State’s Nisonger Center is teaming up with Dr. Elaine Tierney of Johns Hopkins University/Kennedy Krieger Institute and Dr. Forbes D. Porter of  the National Institutes of Health to conduct a phase I/II double-blind study for children ages 4-11 who have been diagnosed with autism spectrum disorders: autistic disorder, Asperger’s disorder, or pervasive developmental disorder – not otherwise specified (PDD-NOS).
Potential study participants will be screened, and 60 children who are found to have abnormally low cholesterol will participate in a 12-week double-blind study in which they will receive either the cholesterol or a placebo, followed by 12 weeks of “open” cholesterol supplementation.
The study, which is currently enrolling participants, is open to children with autism spectrum disorders who are ages 4-11.
Date: March 31, 2011
Source: The Ohio State University Medical Center 

Omeros OMS103HP Study Inconclusive

Omeros OMS103HP Study Inconclusive
Drug Discovery & Development - April 01, 2011

SEATTLE (AP) - Omeros Corp. said that its experimental knee drug did not meet goals of a late-stage clinical trial.
Omeros said it couldn't draw any conclusions about the drug's effectiveness in the study because of "confounding factors," or variables that made it impossible to tell if its drug was responsible for changes in the condition of patients. The company didn't elaborate on the nature of those factors.
Omeros was testing OMS103HP in patients undergoing arthroscopic surgery to reconstruct an anterior cruciate ligament. The anterior cruciate ligament is a major ligament in the knee, and Omeros' drug is intended to improve joint motion and function and reduce pain after surgery.
"Given the strength of the data from previous clinical studies of OMS103HP, we are obviously disappointed and surprised by the outcome," said Gregory A. Demopulos, M.D., Omeros Chairman and CEO. "As we learn more from our data analysis, we will provide additional information on our plans for OMS103HP."
Shares of Omeros plunged $3, or 37.5 percent, to $5 in aftermarket trading. The stock had the regular session up 20 cents at $8.
The company said the results don't show the drug is ineffective and do not undermine another late-stage study of OMS103HP in patients who are having surgery on the meniscus, a cartilage pad inside the knee.