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Trial Shows Pain Drug Effective as Heroin Treatment

Heroin powder

Heroin powder (Drug Enforcement Administration)

7 April 2016. A clinical trial in Canada shows a drug already in use for chronic pain can serve as a substitute maintenance treatment for people addicted to heroin. The findings of the trial, conducted by University of British Columbia in Vancouver and other institutions in Canada, appear 6 April in the journal JAMA Psychiatry.

Opioid addition, particularly heroin, is taking an increasing toll on families and communities worldwide. Opioids work by reducing the intensity of pain signals to the brain, particularly regions of the brain controlling emotion, which reduces effects of the pain stimulus. The illegal drug heroin is an opioid derivative, and the strongest risk factor for heroin addiction is addiction to prescription opioid pain killers.

Among the treatment options for people addicted to heroin are substitute drugs that allow for control and reduction of their substance use. Among these substitute drugs are methadone hydrochloride and buprenorphine hydrochloride, known by its trade name Suboxone. However, these substitute drugs are not always available and some people with addictions cannot maintain their treatment regimens. Prescription-grade heroin, or diacetylmorphine hydrochloride, is often unavailable due to political or regulatory reasons.

The late-stage clinical trial, named Study to Assess Longer-term Opioid Medication Effectiveness or Salome, sought to test if the pain drug hydromorphone could provide another substitute treatment option. Hydromorphone, marketed under its trade names Dilaudid (Purdue Pharma) or Exalgo (Mallinckrodt Pharmaceuticals), is an opioid analgesic drug, approved for long-term chronic pain relief, and formulated for injection, as well as in liquid and tablets.

The study, led by Eugenia Oviedo-Joekes in UBC’s School of Population and Public Health, recruited 202 people age 19 and over with heroin addiction in Vancouver, randomly assigned to receive supervised injections of hydromorphone or diacetylmorphine, the prescription form of heroin. The drugs used in the trial were given at a clinic, up to 3 times a day, for 6 months. Average age of the sample was 44, and 69 percent were male.

The research team looked primarily at the number of self-reported days street heroin purchases were made by trial participants, supplemented by urine tests, during the study period, compared to before. Researchers also tracked number of days participants committed other crimes, as well as safety of the substitute injections.

The results show both hydromorphone and diacetylmorphine are about equally effective in reducing street drug purchases and other criminal behavior. Before the trial, participants were making almost daily purchases of heroin. After receiving hydromorphone or diacetylmorphine, the number of days in which participants bought heroin on the street dropped for both drugs to 3 to 5 days a month. Likewise, the number of days trial participants committed other crimes dropped on average from 14.1 to less than 4 days a month, with the rates about the same for both drugs.

In addition, injections of hydromorphone and diacetylmorphine given at a clinic, had very few adverse effects. Of the 88,451 injections, 14 overdoses and 11 seizures were reported, which were treated on the  spot. Had these adverse events happened on the street, few if any clinicians would have been available.

“Our study shows that hydromorphone is as effective as diacetylmorphine,” says Oviedo-Joekes in a university statement, “providing a licensed alternative to treat severe opioid use disorder. Providing injectable opioids in specialized clinics under supervision ensures safety of both the patients and the community, and the provision of comprehensive care.”

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$500K Challenge Seeks Mood Research Ideas with iPhone

iPhone in hand

(nvtriab, Pixabay)

6 April 2016. A new challenge seeks ideas for studying people’s moods using Apple’s iPhone as the technology platform. The competition, with a total purse of $500,000, is conducted by Robert Wood Johnson Foundation, and has an initial deadline of 22 May 2016 for submissions.

The Mood Challenge is looking for ways of systematically assessing mental and emotion conditions of individuals that collect data with Apple’s iPhone and ResearchKit to build the data collection instruments. Its sponsors are particularly interested in combining contextual and social factors associated with people’s moods, as well as the usual instruments for measuring emotions. Participants in the competition are encouraged to submit research ideas that collect both active information — entered directly by users — or passive data collected by sensors built in or attached to iPhones.

Apple’s ResearchKit is an open-source framework for collecting medical data with surveys or sensors connected to iPhones. The platform also contains templates to describe the conduct of studies and capture signatures for informed consent. In addition, ResearchKit integrates with HealthKit, Apple’s mobile platform for monitoring an individual’s health and fitness.

The Mood Challenge competition has three rounds. In the first round, entrants will provide a proposal detailing the research hypothesis and target audience, along with a plan for employing ResearchKit to conduct the study. Proposals should also explain the data and signals anticipated in their studies, as well as a plan for sharing data with individuals taking part. The deadline for these proposals is 22 May 2016.

From this first round, judges will select 5 semi-finalists, who will be asked to prepare visual mock-ups of proposed iPhone apps, with detailed descriptions of surveys and tasks in the proposed study. Semi-finalists will also be expected to develop plans for pilot testing, data management, and participant screening and consent. Judges will then select 2 finalists, who will write their apps with Apple’s xCode development platform and plan for full deployment through Apple’s App Store.

A total of $500,000 will be awarded. The 5 semi-finalists will each receive $20,000 to prepare their pilot testing and data management plans in the second round. Each of the two finalists will receive $100,000 to design their prototypes and pilot tests. The top winner will receive an award of $200,000 and guidance for submission of their ResearchKit study to the App Store.

“We know that mood is one of the keys to health,” says Robert Wood Johnson Foundation president Risa Lavizzo-Mourey in a foundation statement, “but much more can be learned about the relationship between mood and the many social and economic factors that affect it, and our health. We think platforms like ResearchKit have the potential to revolutionize how research is conducted, and we’re launching this competition to help explore that.”

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Stem Cell Biotech Licensing Blood Vessel Repair Technology

Induced pluripotent stem cells

Induced pluripotent stem cells reprogrammed from human skin (California Institute for Regenerative Medicine)

6 April 2016. Cellular Dynamics International, a developer of regenerative treatments from stem cells, is licensing university research that repairs blood vessels in people with peripheral artery disease. Financial aspects of the deal between Indiana University and Cellular Dynamics, a subsidiary of FujiFilm in Madison, Wisconsin, were not disclosed.

Cellular Dynamics designs and generates induced pluripotent stem cells, derived from live human tissue samples, cultured in the lab, and then reprogrammed with DNA molecules to transform into the desired human cells for clinical applications. The company provides specific cell types — heart, blood vessel, liver, and neurons — for drug discovery, toxicity testing, and simulation.

In addition, Cellular Dynamics, a spin-off enterprise from University of Wisconsin, provides a customized stem cell service for patients to grow their donated samples. The induced pluripotent stem cells, says the company, can transform into some 200 human cell types for an individual patient without the risk of rejection caused by donated tissue.

The company is licensing technology developed in the lab of Mervin Yoder, a professor of biochemistry and molecular biology at Indiana University’s medical school in Indianapolis. Yoder’s lab devised techniques with induced pluripotent stem cells to repair blood vessels damaged from peripheral artery disease. This disorder results from plaque building up and narrowing arteries, reducing the flow of blood to tissues, particularly in the legs. Smoking and age are key risk factors for peripheral artery disease causing pain and numbness in affected areas, and gangrene from infection in advanced cases, leading to amputation.

The technology developed by Yoder and colleagues transform induced pluripotent stem cells into endothelial colony forming cells, similar to cells found in umbilical cord blood, a rich source for regenerating blood vessels. In tests with lab mice, Yoder’s team found endothelial colony forming cells derived from stem cells could rescue and repair blood vessel damage in limbs affected by low blood flow similar to peripheral artery disease.

In addition, the Indiana lab’s techniques quickly and efficiently produce high volumes of regenerating cells, which can be delivered in a gel material with injections, or produced for transplantation in new blood vessels with three-dimensional bioprinting.

“About eight to 12 million Americans and 27 million people in Europe and North America are affected by peripheral arterial disease,” says Yoder in a university statement. “The technology licensed to Cellular Dynamics International may be useful to restore the delivery of blood and avoid amputation.”

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Study to Test Increasing Ethnic Genetic Disease Diversity

Diversity graphic

(Clker Free Vector Images, Pixabay)

5 April 2016. The personal genetics company 23andMe is beginning a study to test techniques for increasing the diversity of disease-causing genetic variations, which now overwhelmingly favor Europeans. The study is funded by a $260,000 Small Business Innovation Research grant from National Institutes of Health.

The study aims to provide better tools for genetics researchers that supplement genome-wide association studies, the standard means of identifying the underlying genetic basis for disease. A 2011 study published in Nature found that nearly all, 96 percent, of genome-wide association studies were of people of European descent. These studies succeeded in identifying associations between common genetic variations and diseases. But these variations explain only 5 to 50 percent of the diseases’ heritability, depending on the disease in question.

In this new project, 23andMe proposes to explore a technique known as admixture mapping, or more formally, mapping by admixture linkage disequilibrium. Admixed populations occur when historically isolated populations more recently combine, as is often found with African-Americans and Hispanics in the U.S., also descended from Europeans and Native Americans.

Admixture mapping seeks out areas in the genome that have an enrichment of one ancestral background in individuals with a disease. These genome regions can highlight risk variants that differ in frequency among the ancestral populations. Previous attempts to apply admixture mapping, however, relied on small samples that limit their representation of these populations at large.

The new study, funded by National Human Genome Research Institute at NIH, aims to take advantage of the company’s nearly 1 million customers that consent for their data to be used in this kind of research, to overcome the earlier small-sample limitations. The 23andMe databases of genetic factors and individual observable traits includes information from more than 68,000 people of Hispanic origin and 34,000 African-Americans. In an earlier phase of the project, also funded by an SBIR grant, 23andMe in Mountain View, California upgraded its data collection and management tools to better provide for greater ethnic diversity.

The research team led by computational biologist Kasia Bryc, plans to study people with admixed backgrounds in 23andMe’s databases having certain diseases in common. The investigation aims to reveal shared ancestries located in common regions of these individuals’ genomes, testing if these genetic regions of shared ancestry can be associated with diseases in common. These findings Bryc notes in a company statement, “could help reduce the research disparities among groups in the United States and elsewhere.”

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Stem Cells Shown to Improve Heart Failure Health Outcomes

EKG graphic

(PublicDomainPictures, Pixabay)

5 April 2016. Results from a clinical trial show a new therapy for heart failure using patients’ own blood-forming stem cells reduces worsening symptoms, hospitalizations, and deaths. Findings of the study testing the treatments developed by biopharmaceutical company Vericel Corp., which funded the trial, appear in today’s issue of the journal The Lancet (paid subscription required).

Heart failure is a condition where the heart cannot pump enough blood to meet the body’s needs, a condition affecting some 5.7 million people in the U.S. The treatments developed by Vericell address one form of heart failure, known as ischemic dilated cardiomyopathy, where failure to pump enough blood is caused by weakness in the left ventricle, the heart’s main pumping chamber, resulting from lack of blood supply to the heart muscle, called ischemia. Patients with this condition often have few treatment options, other than a heart transplant or heart-pumping device.

The treatments develop by Vericel, known as ixmyelocel-T, use a small sample, about 50 milliliters, of a heart failure patient’s own bone marrow as the source for blood-forming stem cells, in a minimally-invasive out-patient procedure. With this sample, Vericel’s lab cultures promote the expansion of mesenchymal cells that can transform into a variety of mature cell types, as well as specialized therapeutic white blood cells.

The company, based in Cambridge, Massachusetts, says it can produce as much as 300 times the number of these cells as found in the original bone marrow. The therapeutic cells produced by ixmyelocel-T are then injected back with a catheter into the patient’s heart muscles to regenerate healthy heart tissue and improve the heart’s pumping ability.

The intermediate-stage clinical trial, led by Timothy Henry at Cedars-Sinai Heart Institute in Los Angeles and Amit Patel at University of Utah, tested ixmyelocel-T with 109 individuals having severe and end-stage heart failure from ischemic dilated cardiomyopathy. Participants were randomized to receive ixmyelocel-T  treatments or a placebo, then tracked for 12 months. The research team looked mainly at the rate of adverse health outcomes of the patients, including further hospitalizations from cardiac causes, unplanned out-patient or emergency-room visits related to heart failure, and deaths from any cause.

The findings show patients receiving the treatments fared better than placebo recipients. Some 38 percent of patients receiving ixmyelocel-T treatments experienced adverse outcomes, compared to about half (49%) of those receiving the placebo. Among participants receiving ixmyelocel-T, 3 percent died and 38 percent had one or more heart-related hospitalizations. With placebo recipients, 14 percent died and 47 percent experienced heart-related hospitalizations. The company says there were fewer treatment-related adverse effects, mainly from the injections or use of a catheter, among treatment than placebo recipients.

In addition to The Lancet publication, results of the trial were presented by Henry yesterday (4 April) at a meeting of American College of Cardiology.

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Pharmacy Interventions Shown to Boost Meds Adherence

Walgreens store

Walgreens store in New Orleans, Louisiana (Musik Animal, Wikimedia Commons)

4 April 2016. Actions by community pharmacists and other customer-assistance programs at Walgreens pharmacies were shown to improve customers’ adherence to medications and reduce their health care costs. Results of the study conducted by health analysts at Walgreens appear in the 1 April issue of the journal Population Health Management.

The research team led by Michael Taitel, director of health analytics at Walgreens, sought to determine the impact, if any, of programs at Walgreens retail stores that aim to improve customers’ adherence to their prescribed medications. The authors note studies showing that only about half of individuals with chronic conditions take their medications as prescribed. In addition, non-adherence to medications is associated with increased hospitalization, progression of disease, and higher mortality.

Walgreens offers at many of its 8,173 retail stores a proactive program of customer counseling and medication therapy management, particularly for individuals starting drugs for the first time. Stores also provide for continuing customers, online and digital reminders to refill prescriptions and pick up their prescriptions once they’re filled. Earlier research indicates programs like those offered by Walgreens that aim to improve medication adherence can have some beneficial impacts.

The researchers conducted an analysis of electronic health records, with identifying information removed, in the databases of IMS Health, a data analytics company serving the health care industry. From these records, the team drew more than 528,000 records from February to July 2013, of which nearly 100,000 received at least one medication adherence intervention from a Walgreens retail store, and 428,000 who were contacted by other drug stores or not contacted at all.

From this sample, the team drew two sub-samples of 72,410 individuals each — in Walgreens and non-Walgreens groups — that were matched one-to-one based on demographics, clinical characteristics, and a number of baseline health care utilization and cost factors. The records were also matched according to 16 different types of prescription drugs covering heart, respiratory, metabolic, circulatory, and neurological conditions, then tracked for 6 months following the initial contact.

The results show customers who received medication adherence interventions from Walgreens stayed with their medications somewhat better than their counterparts, but also had better health outcomes and spent less money overall on their health care. Walgreens customers receiving counseling or reminders reported 3 percent better medication adherence than those in the comparison group. But the Walgreens intervention group also had 2 percent fewer hospital admissions, 3 percent fewer emergency room visits, and slightly fewer (0.5 on average) out-patient visits.

While these differences seem small, they were large enough to be statistically reliable. The differences also resulted in lower overall health care costs for Walgreens customers of about $226.00, particularly for out-patient visits and pharmacy costs: $120.00 and $92.00 less respectively.

Harry Leider, chief medical officer at Walgreens, says in a company statement, “Patients receiving a new chronic diagnosis and medication therapy are at very high risk for non-adherence to medication, and this important study demonstrates how a diverse set of pharmacy and digital interventions improves care while reducing total health care costs.”

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Intrexon Spin-Off Developing Type 1 Diabetes Therapy

Diabetes items

(Centers for Disease Control and Prevention)

4 April 2016. A new subsidiary of the synthetic biology company Intrexon Corp. is creating a pill for people with type 1 diabetes to stop the disease’s damage in its early stages. The enterprise, known as Intrexon T1D Partners LLC, is a joint venture between Intrexon, in Germantown, Maryland, and White Rock Capital Partners a New York investment bank providing an initial $10 million investment.

Type 1 diabetes is an inherited autoimmune disorder where the body does not produce insulin, and is diagnosed primarily in children or young adults. Autoimmune disorders are conditions where the immune system is tricked into attacking healthy cells and tissue as if they were foreign invaders, in this case, insulin-producing beta cells in the pancreas. About 1.25 million people in the U.S. have type 1 diabetes.

Once type 1 diabetes develops, people with the disorder need to regularly test for blood glucose levels and inject insulin to manage their condition. Intrexon T1D Partners plans to apply Intrexon’s technology, called ActoBiotics, to develop an oral treatment for type 1 diabetes. This pill would harness the immune system to prevent damage to beta cells in early stages of the disease, or to prevent the need for external insulin therapy.

Intrexon develops genetically engineered products for the pharmaceutical, food, energy, environmental, and consumer markets. The company operates several technologies derived from computational models and software that assemble DNA-based solutions on a commercial scale. The ActoBiotics platform creates engineered food-grade bacteria, known as Lactococcus lactis, similar to those found in yogurt, to deliver protein therapeutics into the gut, which would otherwise degrade in the gastrointestinal tract.

In 2014, a research team in Belgium tested delivery of a type-1 diabetes antigen and the anti-inflammatory protein interleukin-10, with engineered Lactococcus lactis bacteria into non-obese mice newly induced to express diabetes.  The study in this case used an oral drug that targeted gut-associated lymphoid tissue in the mice, the largest immune system in the body. The results show the treatments generated regulatory T cells in the mice, which stabilized inflammation and preserved beta cells in the pancreas, and restored normal blood glucose levels.

The deal with White Rock Capital Partners gives Intrexon a 50 percent interest in T1D Partners. Intrexon calls this type of agreement an exclusive channel collaboration that gives the partner companies an exclusive license to develop new products from Intrexon technologies, move those products through regulatory processes, and conduct marketing and sales.

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Gene-Editing Therapy Advances for Rare Immune Disorder

Human T-cell lymphocyte

Scanning electron micrograph of a human T-cell lymphocyte (National Institute of Allergy and Infectious Diseases, NIH)

1 April 2016. A key committee of the European Medicines Agency recommends approval of treatments for a rare children’s immune disease that uses edited genes as therapy. The Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency at its March 2016 meeting recommended approving Strimvelis by GlaxoSmithKline to treat adenosine-deaminase-deficient severe combined immunodeficiency or ADA-SCID.

ADA-SCID is an inherited disorder, where individuals are born without immune system protection from common bacteria, viruses, and fungi, making them susceptible to repeated infections, which can become life-threatening. The rare disorder — 1 case occurs in 200,000 to 1 million newborns worldwide — is usually diagnosed early in life, and without treatment, babies rarely live beyond the age of 2. In 10 to 15 percent of cases, the disease does not appear until after 6 to 24 months. The disorder is a result of a faulty gene inherited from both parents.

The only treatments for ADA-SCID are transplants of blood-forming stem cells, which requires genetically-matched siblings as donors. Unless the match is near-perfect, the individual risks developing graft-versus-host disease, an immune system rejection disorder. Bone-marrow recipients also may need to take drugs that suppress the immune system, which raises the risk for infection.

Strimvelis is the GlaxoSmithKline brand name for therapies where a sample of the individual’s bone marrow is removed and the person’s immature blood-forming cells are harvested. A healthy adenosine deaminase, or ADA, gene, is inserted into genomes of the blood-forming stem cells, carried by a benign virus, which enables the stem cells to produce healthy blood cells. Stem cells with corrected genes are infused back into the patient, where they flow into the bone marrow to produce healthy blood cells, including lymphocytes with normal immune system functions.

GlaxoSmithKline tested Strimvelis in a clinical trial where 12 children received the treatments over a 10-year period in Israel and Italy. The company says all 12 individuals are still alive today, including the first patient treated some 13 years ago. Nearly all of the participants in the trial (92%) did not require any follow-on stem cell transplants or enzyme replacement treatments. The most common side effects were fever, increased liver enzyme levels, and autoimmune reactions, such as anemia.

Strimvelis was given an orphan drug designation by the European Medicines Agency in 2005. The recommendation goes to the European Commission to authorize marketing Strimvelis in EU countries. No date was given for the European Commission to consider CHMP’s recommendation.

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On-Demand Pharma Manufacturing System Developed

Compact drug manufacturing system

Continuous-flow drug manufacturing system (Massachusetts Institute of Technology)

1 April 2016. Chemical engineers at Massachusetts Institute of Technology created a compact manufacturing system that produces small quantities of pharmaceuticals as needed. The system developed in the MIT labs of Alan Myerson, Klavs Jensen, and Timothy Jamison is described in the 31 March issue of the journal Science (paid subscription required).

Making drugs at commercial scale normally requires large amounts of materials often shipped from remote suppliers, large-scale facilities, and long production times to generate the quantities needed by customers. This batch production process may work for some prescription and over-the-counter drugs, but the industry still faces frequent spot shortages of pharmaceuticals — more than 200 per year, according to FDA — from variations in quality control and supply change interruptions.

Normal manufacturing processes are likewise not designed for small quantities or fast response. The long lead times make it difficult to respond quickly to natural disasters or other emergencies. In addition, the process does not work well for drugs needed in smaller quantities, such as those treating rare diseases or for clinical trials.

“The goal of this project,” says Jensen in an MIT statement, “was to build a small-scale, portable unit that was completely integrated, so you could imagine being able to ship it anywhere. And as long as you had the right chemicals, you could make pharmaceuticals.”

The authors’ manufacturing concepts are an outgrowth of a project with drug maker Novartis to develop a continuous flow process for drug manufacturing in a small-scale pilot facility that produced finished products from intermediate-stage chemicals. This new initiative, funded by Defense Advanced Research Projects Agency or Darpa, adds new processes for synthesis, purification, and formulation, with each process in a separate module. It also shrinks the size of the production facility to 1.0 by 0.7 by 1.8 meters, about the size of a home refrigerator.

A critical feature of the system is its use of tubes instead of large vats for the chemical reactions for drug production, which generate a lot of heat in the synthesis phase. Cooling the large vats is a challenge at many drug manufacturing plants, but a more manageable problem with the smaller quantities flowing through the tubes in the MIT prototype, even at temperatures of up to 250 degrees Celsius.

Purification of the product is done with crystallizing, followed by filtering and drying the product to remove solvents, then dissolving it in liquid to formulate the dosages. For this proof-of-concept, the team produced liquid drugs that meet U.S. Pharmacopeia standards. For solid formulations, such as tablets, additional steps, equipment, and space are needed, and the researchers are looking into miniaturization and 3-D printing for those processes.

With this platform, the team produced doses of the popular drugs diphenhydramine hydrochloride, lidocaine hydrochloride, diazepam, and fluoxetine hydrochloride — known by their brand names as Benadryl, Lidocaine, Valium, and Prozac respectively. The modular system design enabled the researchers to use the same technology, and swap out components to make each new batch, which they say could be done in a few hours.

The research team is now investigating adapting this platform to make more complex drugs, including solid-product formulations such as tablets, as well as shrinking its physical footprint further. In addition, first author and MIT research associate Andrea Adamo, founded the company Zaiput Flow Technologies in Cambridge, Massachusetts to commercialize liquid extraction and separation processes used in continuous flow chemistry.

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Foundation Supporting ALS Progression Model Study

Brain synapses illustration

(AllanAjifo, Wikimedia Commons)

31 March 2016. A study to validate computer models that predict progression and survival of patients with ALS is underway by a bioinformatics company and biopharmaceutical developer. The 3-year project is funded by a grant of nearly $500,000 from ALS Association in Washington, D.C.

ALS, or amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is a progressive neurological disease that attacks the nerve cells controlling voluntary muscles, such as those in the arms, legs, and face. In ALS, muscles gradually weaken and waste away, leading to individuals losing their strength and their ability to move their arms, legs, and body. When diaphragm and chest wall muscles fail, people lose the ability to breathe without ventilatory support, often leading to death from respiratory failure.

ALS patients experience varying degrees of progression of the disease, with some patients suffering complete paralysis in as few as two years, while for others the disease progression is much slower. In the early stages of the disease, it is difficult to determine whether a given patient will experience slow or fast disease progression. The ability to predict disease progression is also critical for those interested in planning ALS clinical trials for potential new treatments.

ALS Association sponsors open competitions to develop better models for predicting progression of the disease, which provide access to the Pooled Resources Open-Access Clinical Trial or PRO-ACT database that contains records from more than 8,500 ALS patients taking part in clinical studies. One of those challenge winners is David Ennist, now chief scientist at Origent Data Sciences, in Vienna, Virginia. Origent is a bioinformatics company that develops predictive models for neurological conditions, including for individual patients.

The ALS Association grant funds work by Ennist and colleagues at Origent to validate predictive models for progression of ALS built from the PRO-ACT database, with data from a clinical trial of the drug tirasemtiv, created by Cytokinetics Inc. in South San Francisco. Cytokinetics is a developer of therapies for disorders involving skeletal muscles, such as those in the respiratory system affected by ALS. Tirasemtiv is a small-molecule activator of troponin proteins that regulate calcium in skeletal and heart muscle contractions.

As reported on Science & Enterprise in July 2015, ALS Association is supporting a late-stage clinical trial by Cytokinetics testing tirasemtiv as a treatment for people with ALS to slow the decline of respiratory function. While the trial is still underway, data from the study and earlier trials will be made available to Origent to prospectively validate its ALS progression models. Those models are expected to make it possible for clinical trials of ALS treatments to require fewer patients and take less time.

“If validated,” says Ennist in a joint statement, “the models we have developed will be submitted to the FDA and may enable more nimble, cost-effective execution of ALS clinical trials, resulting in a potentially quicker path to new medicines.”

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