SCOTTISH HOSPITAL NEWS THREE NEW MEDICINES ACCEPTED BY SMC


The Scottish Medicines Consortium (SMC), has published advice accepting three new medicines for use by NHSScotland.


Olaratumab (Lartruvo) was accepted for the treatment of advanced soft tissue sarcoma, a rare type of cancer that affects the soft, supportive tissues of the body such as muscles, blood vessels and fat tissue. It is used in patients who cannot undergo curative surgery or radiotherapy. Olaratumab is given with doxorubicin (a chemotherapy medicine), and both medicines are given by intravenous infusion. Through SMC’s Patient


and Clinician Engagement (PACE) process for medicines used to treat very rare and end-of-life conditions, it was highlighted that sarcoma often occurs in younger patients who may have significant work and family commitments. Olaratumab can improve overall survival and PACE participants commented that treatment may give patients the ability to maintain a good quality of life.


Pegvisomant (Somavert) for the treatment of acromegaly was also accepted through the PACE process. Acromegaly is a rare condition


NOVEL TREATMENT


APPROACHES FOR FIBROTIC LIVER DISEASES Boehringer Ingelheim and MiNA


decades.


Therapeutics have announced that they have signed a collaboration and licensing agreement focused on the development of novel compounds to treat fibrotic liver diseases such as NASH, based on MiNA’s small activating RNA (‘saRNA’) therapeutics platform.


NASH is a major cause of liver fibrosis and cirrhosis and an area of high unmet medical need. It has an especially high prevalence among obese and diabetic patients and there are currently no specific treatments available. NASH is expected to become the most common cause of advanced liver disorders eventually necessitating liver transplantation in the coming


saRNAs have been demonstrated to activate transcription of specific genes resulting in upregulation of proteins with therapeutic potential. In the new collaboration, Boehringer Ingelheim and MiNA aim to identify targets to restore metabolic functionality of hepatocytes and prevent fibrotic tissue formation in patients with NASH. It will enable Boehringer Ingelheim to rapidly design, profile and develop novel compounds, potentially also creating opportunities for combination with Boehringer Ingelheim’s other NASH-pipeline assets.


‘This collaboration is a recognition


BLOOD TEST SPOTS OVERDOSE PATIENTS AT RISK OF LIVER DAMAGE


Around 50,000 people are admitted to hospital each year in the UK due to paracetamol overdose, mainly due to patients not knowing that they should abstain from taking it at the same time as cold and flu medications that also contain the drug.


Now, a new test may help to reduce the workload for already over- stretched Emergency Departments by


showing clearly if a patient is going to suffer liver damage.


Researchers from the Universities of Edinburgh and Liverpool say the test – which detects levels of specific molecules in blood – will help doctors identify which patients arriving in hospital need more intense treatment. It will also help speed the development of new therapies for liver damage by


usually caused by a non-cancerous tumour on the pituitary gland. In the PACE meeting, patient groups and clinicians spoke of how patients suffer significant symptoms, including extreme fatigue, joint pain and excess growth which can affect internal organs and external appearance. Pegvisomant provides a treatment option with a different side effect profile for patients where previous therapies have not been successful. Some patients receive radiation therapy, but it can take years for this to be fully effective and pegvisomant may reduce the signs


and symptoms of acromegaly until the benefits of radiation are fully realised.


Also accepted was glecaprevir/ pibrentasivir (Maviret) for the treatment of chronic hepatitis C, a blood-borne virus that predominantly infects the cells of the liver, resulting in inflammation and significant damage and potentially liver failure or cancer. Glecaprevir/pibrentasivir is given as a short-term course of oral treatment, which is associated with a high response rate, and offers the prospect of a cure across all types of hepatitis C.


of our platform and expertise in liver-based diseases as well as validation from an established pharmaceutical leader in what has been an exciting year for the company,’ said Robert Habib, CEO of MiNA. ‘This new collaboration is another sign of our ongoing commitment to patients with


targeting patients most likely to benefit.


The test detects three different molecules in the blood that are associated with liver damage – called miR-122, HMGB1 and FL-K18. Previous studies have shown that levels of these markers are elevated in patients with liver damage long before current tests can detect a problem.


The research team measured levels of the three markers in more than 1000 patients across the UK, who needed hospital treatment for paracetamol overdose. They found that the test can accurately predict


cardio-metabolic diseases, including NASH,’ said Clive R Wood, PhD, Corporate Senior Vice President Discovery Research at Boehringer Ingelheim. ‘It will combine MiNA’s pioneering work with saRNAs with our expertise in biopharmaceutical research and development.


which patients are going to develop liver problems, and who may need to be treated for longer before they are discharged. The test could also help identify patients who can be safely discharged after treatment, helping to free up hospital beds.


Patients with a life-threatening level of paracetamol in their blood can be treated with an antidote called acetylcysteine, given by intravenous drip. The researchers say the test could help to pinpoint patients who are unlikely to benefit from treatment.


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