Tag: Multiple Sclerosis
This week, NYC-based Immune Response BioPharma, Inc. announced that the company has terminated partnering discussions on its multiple sclerosis vaccine NeuroVax with GlaxoSmithKline. The news per se may not trigger a lot of interest – but the company has managed to create a significant buzz around its announcement, with CEO David Buswell stating that (quote from corporate press release):
“GSK is a joke and seems very ignorant on how multiple sclerosis drugs work and how to develop one, we gave them a chance to develop NeuroVax but their management appears to be very poor. We have decided to terminate any collaboration or development with GSK. GSK is a loser in the MS market and will continue to be a loser.”
When we first saw this press release, we immediately did some research to verify if it was a joke or not. However, it was posted on the company’s website on May 24. While the CEO certainly has shut the door for any further discussions with GSK, the PR has generated a lot of attention in the industry and everyone will be out following the company and the further fate of its compound. Still, we believe that the impact on Immune Response BioPharma´s corporate reputation will not be as positive as the buzz suggests.
What was your first thought when you read this statement?[poll id=”5″]
Germany’s Institute for Quality and Efficiency in Health Care (IQWiG) this year put out several negative assessments of newly introduced drugs, stating the data did not prove “additional benefit” over existing treatments. In all cases, IQWiG came to the conclusion after deviating from the study design the companies had discussed with the regulators. Instead, IQWiG’s experts divided the patient population into subgroups, saying those subgroups needed different comparator treatments. As a result, these data were either not available or the subgroups were too small to demonstrate statistical significance.
One example is Pfizer’s Xiapex injectable collagenase, approved in early 2011 to treat Dupuytren’s contracture. IQWiG stated that Xiapex does not provide an additional benefit to patients because “it was not possible to derive such additional benefit from the dossier and because the manufacturer did not provide additional or suitable data” to substantiate the claim.
While the manufacturer had compared the Xiapex injection to a surgical treatment, partial fasciectomy (PF), IQWiG for its assessment established six subgroups of patients according to the severity of the disease and chose three different treatment options as comparator: no therapy, percutaneous needle fasciectomy (PNF) and partial fasciectomy (PF). As a result, IQWiG was able to state that Pfizer did not provide evaluable data because the company’s selected comparators differed from IQWiG’s comparators for all but one patient subgroup.
In the case of Eisai’s breast cancer drug Halaven eribulin, IQWiG’s verdict ruled that it could not find evidence for eribulin resulting in a prolonged life expectancy. IQWiG added that Halaven might provide an overall survival benefit for patients for whom taxanes or anthracyclines are no longer an option, but it was unclear whether the benefit was significant. Again, the assessment was made by subdividing the patient group. IQWig defined two subpopulations – one for which an additional anthracycline or taxane treatment was thought to be an option and one for which this was not.
Eisai, in contrast, had compared Halaven to a “Treatment of Physician’s Choice” (TPC) as there are no established national or international treatment guidelines for a standard therapy of women with metastatic or locally advanced breast cancer after failure of two standard chemotherapies including an anthracycline or taxane. This design of Eisai’s EMBRACE was established in discussions with the European Medicine’s Agency (EMA). Being a European study, the participating physicians sometimes opted for therapies not approved in Germany – a reason for IQWiG to not include these data in its assessment. As a result, only 69% of the EMBRACE study patients were regarded as suitable for an assessment.
The same approach was taken in the assessment of Novartis’ Gilenya fingolimod, the first oral treatment for Multiple Sclerosis (MS) approved in 2011. IQWiG once again performed separate assessments of the drug in three groups of patients, choosing three different comparators. Following this operation, IQWiG was able to find data only for one of these subgroups in the study, not enough to establish an additional benefit with sufficient statistical significance. However, one of the comparisons chosen by IQWiG – fingolimod against glatiramer acetate in patients with relapsing/remitting MS – would have been impossible as fingolimod is approved as second-line therapy in this indication while there are no studies of glatiramer acetate differentiating between first-line and second-line treatments.
In all cases, manufacturers may respond to the assessment, after which the Federal Joint Committee (G-BA) will review IQWiG’s recommendation before making a final decision. If G-BA deviates from IQWiG’s negative assessment, the manufacturers have to negotiate the price with the Statutory Health Insurance Funds Association (GKV-Spitzenverband) under the AMNOG pricing scheme. If G-BA agrees with the IQWiG assessment that a drug has no clinical benefit beyond available treatments, the drug will be added to the reference pricing system, which gives the same base price to all comparable drugs in the respective therapeutic group.
Ulrike von Leszczynski in Die Welt introduces a novel submersible which can dive up to 6 kilometers deep but weighs only 500 kg. The 3,5 meter long “autonomous underwater vehicle” named DNS Pegel does not need a pressure chamber as it is being flooded when diving. Instruments and electronics have been developed to withstand the conditions and most are protected by silicone.
In Der Spiegel, Steve Ayan, editor-in-chief of Gehirn & Geist, interviews Florian Holsboer, director of the Max Planck Institute of Psychiatry who explains how and why psychiatry will be revolutionized by tailor-made, personalized medicine to treat conditions such as anxiety, depression and others. Holsboer explains that psychiatric diseases are caused by a complex interplay between genes and environment in which the environment also influences the pattern of genes involved in a certain condition at a certain point in time. In the future, he predicts, “we will be able to generate biochemical snapshots using genetic tests and biomarkers.”
Marc-Denis Weitze in Neue Zürcher Zeitung (NZZ) introduces efforts by scientists from the Max Planck Institute for Biochemistry in Martinsried, the Natural and Medical Sciences Institute (NMI) at the University of Tuebingen and the Department of Biosystems Science and Engineering of ETH Zurich in Basle to record the activity of neurons in neuronal networks – a challenging task as chips and electronics elements need to withstand salty solutions for months. The latest innovation is a chip providing 32,000 contact points on a 2.6 square millimeter area. Nicola von Lutterotti, also in NZZ, reports on US and Swiss studies looking into the causes of hospitalizations. In Switzerland, up to 7% were due to overdosing of medications (either by doctors or accidentally by patients) or prescriptions of medications without observing warnings on potential interactions given on the label.
In the New York Times (NYT), Nicholas Wade reports on the successful genetic therapy of six patients with hemophilia B. The disease was corrected by transferring a working version of the factor IX gene via the adeno-associated virus-8 (AAV-8). The article points out that the therapy did not work or ceased to work in some of the patients. In other patients, the factor IX is produced in sufficient quantities for up to 22 months so that they can live without medications.
The New Scientist this week features a study by researchers from the University of Freiburg, Germany, in which symptoms of multiple sclerosis (MS) have been reverted in mice by injecting RNA oligonucleotides that stimulate the expression of interferon-B (IFNb). IFNb is known to be efficacious in humans with MS. However, 80% of people treated with IFNb injections develop antibodies against IFNb. If produced by the body itself the problem might be avoided.
And finally, “self-hacking” can be dangerous to your health, reports Klaus Vogt in Die Welt. Self hackers are promoting the “Quantified Self” movement and are recording, rating and sharing a wealth of body functions – from weight and blood pressure to feelings and data on sex and meditation – on a daily or even more frequent basis. While the movement already finds interest among medtech companies and data providers, medical professionals now warn that the underlying condition can become addictive. The akampioneer recommends software developers should program a meta app analyzing the quantified self data so that an addiction value can be posted on top.
Clemens Gleich in Die Welt reports on the development of super batteries able to power a smart phone or notebook for days without re-charging. While some researchers try to improve conventional lithium-ion batteries by modifying the carbon-based anode with silicon, others design lithium-oxygen or fluorine-oxygen batteries. Main challenges are safety, prevention of swelling and maintaining a high capacity.
Britta Verlinden in Die Zeit reports on the discovery that dimethyl fumarate, a standard drug used for the treatment of psoriasis since 1994, may also be used as a pill to treat multiple sclerosis. Preliminary results of a Phase III trial demonstrate its ability to significantly reduce the number of attacks. The drug candidate codenamed BG-12 is being developed by Biogen Idec. The paper raises the concern that BG-12 may be sold as MS medication at €15,000 a year – while based on the price of the same compound for psoriasis, costs would amount to €4,400 per year, which already “is clearly more costly than what might be expected based on the cheap basic material”.
The Economist this week features the discovery of Oxford University scientists that a small marine organism produces a water-resistant, flexible material which has the adhesive characteristics of barnacle glue and the structural properties of spider-silk fibres. Already, spider silk is being used for novel materials. A salt water tolerant silk might open up medical uses for silk where it would come in contact with salty body liquids. The paper also looks into the prospects of stem cell therapies. While Geron’s pulling out of the stem cell business is viewed as bad news for the field, the paper highlights good news coming from a Lancet paper describing how stem cells can be used to repair hearts. The injection of autologous heart stem cells into damaged heart muscles of patients which underwent coronary bypass surgery led to “remarkable” results, improving pumping volume and other parameters.
Linda Geddes in The New Scientist raises hopes that partial wave spectroscopic (PWS) microscopy some day may be used to screen the general population for diseases like cancer, Alzheimer’s Disease or autoimmune diseases. PWS microscopy can detect changes in the chromatin density of cells, and researchers already have shown that cancer patients even in apparently healthy cells have unusual chromatin densities not seen in cancer-free people.
Finally, Alex Knapp in Forbes proclaims the end is in sight: we may be approaching the day where coffee is both rare and expensive. For one, the demand is growing all over the world at an enormous rate, and second, at the same time yields are diminishing because of pests, climate changes and political instabilities. So enjoy your coffee while it lasts!