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This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

Innovation Observatory > Reports

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Drugs

February 2020

Atezolizumab in combination with platinum-based chemotherapy for untreated locally advanced or metastatic urothelial cancer – first line

Atezolizumab is in clinical development, in combination with platinum-based chemotherapy, for the treatment of patients with locally advanced or metastatic urothelial cancer, who have received no prior systemic therapy (a drug which travels through the bloodstream and affects the whole body). Urothelial cancer, a subset of bladder cancer, occurs on the lining of the bladder, and other parts of the urinary system. In advanced urothelial cancer, the cancer has grown into deeper layers including connective tissue or muscle. Metastatic urothelial cancer occurs when the cancer has spread to other parts of the body, such as the liver or bones.

Drugs

February 2020

AMG 510 for KRAS G12c mutated metastatic non-small cell lung cancer – after prior standard therapy

AMG 510 is in clinical development for the treatment of adults with KRASG12C mutated, metastatic non-small cell lung cancer (NSCLC). NSCLC is the most common form of lung cancer and a small proportion of patients with NSCLC have tumours which carry the genetic mutation KRASG12C. Metastatic NSCLC describes tumours that have spread from the lungs to other parts of the body. Current NSCLC treatment depends largely on the stage of the cancer and any genetic mutations identified in the tumours and can include surgery, chemotherapy, radiotherapy, targeted cancer drugs and immunotherapy. Despite a wide range of treatments being available for lung cancers, there are currently no approved treatments for KRASG12C mutated, metastatic NSCLC.

Drugs

February 2020

Pembrolizumab in addition to docetaxel for chemotherapy-naïve metastatic castration-resistant prostate cancer – second line

Pembrolizumab in addition to docetaxel is in clinical development for patients with prostate cancer which has spread from its original site (i.e. is metastatic), is untreatable via testosterone suppression therapy (i.e. is castration resistant), and where the patient has not received chemotherapy (i.e. chemotherapy-naïve). Prostate cancer is a cancer of the prostate gland (a small organ in a man’s pelvis) and is the most common cancer in men in the UK. There are three stages: localised, locally advanced and advanced (or metastatic) prostate cancer. The symptoms may vary depending on the stage of cancer but can include pain, tiredness, and problems emptying the bladder and the bowels.

Drugs

February 2020

Tofersen for the treatment of amyotrophic lateral sclerosis caused by mutations in the SOD1 gene

Tofersen (BIIB067) is in clinical development for the treatment of amyotrophic lateral sclerosis (ALS – also known as motor neurone disease) caused by mutations in the SOD1 gene (SOD1-ALS). ALS is a progressive disease of the nervous system, where nerve cells in the brain and spinal cord that control voluntary movement gradually deteriorate, causing loss of muscle function and paralysis. ALS is a debilitating and life-threatening disease. The gradual loss of neurons leads to a paralysing effect on muscles used for breathing, which usually leads to death from respiratory failure.

Drugs

February 2020

Avapritinib for advanced systemic mastocytosis

Avapritinib is in clinical development for the treatment of advanced systemic mastocytosis (SM) in adults. SM is a condition where mast cells grow uncontrollably and accumulate in body organs/tissues such as the skin, internal organs, lymph nodes and bones. Mast cells are immune cells that release inflammatory mediators that are important in the body’s allergic responses. When mast cells are present in large numbers there is a high release of these mediators leading to symptoms such as itching, fever, abdominal pain, nausea and vomiting. In advanced SM, mast cells collect in such high quantities that they lead to organ damage and dysfunction, bone fractures and anaemia.

Drugs

February 2020

Garetosmab for preventing abnormal bone formation in fibrodysplasia ossificans progressiva

Garetosmab is in clinical development for the prevention of abnormal bone formation, outside of the normal skeleton (heterotopic ossification), and soft-tissue flare-ups in patients with fibrodysplasia ossificans progressiva (FOP). FOP is a very rare disease caused by a mutation in the gene ACVR1. The mutation results in the formation of unwanted bone in muscles, tendons, and ligaments throughout the body. Abnormal, misplaced and mis-shapen (heterotopic) bone can bridge across joints causing immobility, scoliosis, and other deformities. Patients usually require a wheelchair by the time they reach their 20s. Death often results in the 40s from complications, such as pneumonia, heart failure and loss of mobility in the chest, neck and jaw.

Drugs

February 2020

Naxitamab in combination with granulocyte-macrophage colony stimulation factor for relapsed/refractory high-risk neuroblastoma

Naxitamab is a type of protein that has been designed to recognise and attach to a specific structure called GD2 that is present in high amounts on the surface of neuroblastoma cells, but not normal cells. Naxitamab attaches to the neuroblastoma cells and activates the immune system, which then kills the cancer cells. If licensed, naxitamab in combination with GM-CSF may provide a treatment option for patients with relapsed/refractory high-risk neuroblastoma.

Drugs

February 2020

Daratumumab in addition to pomalidomide and dexamethasone for relapsed or refractory multiple myeloma

Daratumumab is a type of immune therapy that acts by inhibiting the growth of cancer cells in MM via a surface protein called CD38. Daratumumab monotherapy is already licenced for relapsed/refractory MM. Pomalidomide in combination with dexamethasone is also currently licenced to treat relapsed/refractory MM. Early findings from trials have demonstrated that the addition of daratumumab to pomalidomide and dexamethasone may further stimulate the immune system and directly act against cancer cells in MM, potentially providing another treatment option for patients whose disease has progressed on previous treatments.

Drugs

February 2020

Daridorexant for Insomnia

Daridorexant is a medicinal product in clinical development for the treatment of adults with insomnia. Insomnia means regularly having problems sleeping, which results in impaired daytime functioning. Insomnia can be categorised according to its duration, into short-term insomnia (lasting less than 4 weeks) and long-term (or persistent) insomnia (lasting 4 weeks or longer).

Drugs

February 2020

Niraparib for metastatic castration-resistant prostate cancer with DNA-repair anomalies

Niraparib is a medicinal product taken orally. It works by blocking a protein called poly (adenosine diphosphate-ribose) polymerase (PARP). It blocks the action of PARP-1 and PARP-2 enzymes that help in repairing damaged DNA in cells when they divide to make new cells. By blocking PARP enzymes, the damaged DNA in cancer cells cannot be repaired, and the cells die. If licensed, niraparib will offer an additional treatment option for men with mCRPC with DNA-repair anomalies.

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