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Start-Up Licensing Biologic Delivery Nanoparticles

Pharmacy lab

(University at Buffalo)

8 April 2016. A start-up enterprise is licensing research from a university pharmacy lab that harnesses nanoscale particles to boost the performance of biologic therapies. Financial details of the agreement between Zoetic Pharmaceuticals in Amherst, New York and University at Buffalo were not disclosed.

Biologic therapies are synthetic proteins derived from living systems, such as microbes or organism cells, often with engineered DNA molecules in human genes. Biologics, however, face obstacles from antibodies that react to their presence, creating immune reactions and thus complications for patients. This can be a particular problem for biologics given to treat autoimmune disorders, such as type 1 diabetes and rheumatoid arthritis, where the immune system is tricked into attacking healthy cells.

Zoetic Pharmaceuticals is developing a technology platform for delivery of biologics that addresses these immune system issues. That platform is based on and licensed from research by Buffalo pharmacy professor Sathy Balu-Iyer, who studies actions of protein therapies in the body. Among Balu-Iyer’s discoveries are drug delivery techniques harnessing lipids, natural insoluble oil or fat substances, as nanoscale particles to carry the active biologic ingredients.

Balu-Iyer and colleagues tested the technology on lab mice induced with hemophilia A, an inherited disorder where a clotting protein known as factor-8 is missing, causing excessive bleeding. Treatments to replace factor-8 proteins are often hampered by their short live duration in the body, as well as generation of antibodies and immune reactions. The findings show lipid nanoparticles carrying factor-8, however, extend its circulation in the test mice and reduce immune reactions. The study also showed through simulations the process can be scaled up for humans.

The technology can be applied as well to gene therapies, where healthy genes are inserted into an individual’s genome to replace missing or defective genes, similar to hemophilia A. Like biologic treatments, gene therapies can also trigger an immune reaction, and the lipid nanoparticles can shield the replacement genes from those reactions.

Zoetic was founded in November 2014 by life sciences and pharma industry veterans John Seman and Sven Beushausen, now president and chief scientist respectively. Balu-Iyer is a scientific adviser to Zoetic. The company is located at Baird Research Park, the university’s technology and business incubator site.

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Cancer Centers, Analytics, Pharma Plan Precision Medicine

Genetic testing illustration

(National Institute of General Medical Sciences, NIH)

7 April 2016. A cancer care center network, with pharmaceutical and informatics companies, plan to apply precision medicine techniques to increase the pool of patients for clinical trials of new cancer therapies. The coalition includes cancer centers in the Orien network — short for Oncology Research Information Exchange Network — with bioinformatics company M2Gen, and the pharmaceutical company Celgene that aims to generate many more cancer patients screened for clinical trials.

The Orien network includes founding members Moffitt Cancer Center in Tampa and Ohio State University’s James Cancer Center, along with 9 other cancer hospitals in the U.S. As reported in Science & Enterprise in May 2014, the Orien project collects de-identified clinical data from cancer patients, combined with samples of tumors and bodily fluids not needed for care. Data from these patients are stored and analyzed according to Moffitt Cancer Center’s protocols.

M2Gen, a spin-off company from Moffitt Cancer Center, will manage the high volume of genetic and clinical data, which will be made available as a subscription service, known as Orien Avatar, to pharmaceutical companies. The company identifies patients in the Orien databases using its algorithms for matching patients’ clinical and molecular profiles against a trial’s clinical and molecular eligibility criteria.

In Orien Avatar, patients’ data will be matched at a molecular level to cancer treatments in clinical development. The program anticipates focusing on patients with advanced primary or metastatic cancer, those with limited treatment options, and those whose condition is likely to worsen over time.

Patients are expected to benefit from Orien Avatar, by gaining greater access to more clinical trials and experimental treatments. Participating cancer centers anticipate sharing their findings and expanding clinical trial options for their patients.

William Dalton, founder and CEO of M2Gen says in a company statement, “The Orien Avatar program will use an in-silico analysis approach to better design clinical trials and match patients to promising clinical trials to achieve their accrual targets so that new and improved treatments can be brought to market more rapidly, and help millions of patients worldwide.”

The first pharma company to take subscribe to Orien Avatar is Celgene in Summit, New Jersey, where a large part of Celgene’s pipeline is devoted to treatments for solid tumor and blood-related cancers. “This wealth of clinical and molecular data,” says Michael Pehl, president of hematology and oncology at Celgene, “will potentially lead to a better understanding of molecular properties that are involved in a patient’s disease and what treatment designs might be most successful in battling their cancer.”

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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)

Update, 20 July 2016. The Robert Wood Johnson Foundation sponsoring the Mood Challenge announced the competition’s 5 semi-finalists:

Aware Study, to measure mood and posttraumatic stress symptoms among the millions of adults living with PTSD.

BiAffect, a system for understanding mood and neurocognitive functioning in bipolar disorder using keystroke dynamics, such as typing speed and errors, to track and predict mood episodes.

Mood Circle, to improve on mood detection and modeling using passive data tracking and self-reports on mood by incorporating social networking.

MoodSync, to identify how daily mood and social environments are associated with biological aging among family caregivers.

Mood Toolkit, to provide mental health researchers with a configurable toolkit to study daily emotional health and well-being through the ResearchKit framework.

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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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