Optogenetics for Retinitis Pigmentosa
Identified and further supported by ReMedys, a highly innovative gene therapy project from the University of Bern was span out to Arctos Medical in 2019. A company that was acquired by Novartis in 2021, securing thus that a potential disease modifying therapy will be brought to the clinic (expected for 2023) and be made available to millions of patients suffering from retinal degeneration and blindness.
One of the most prevalent causes of blindness is photoreceptor degeneration and the only treatment presently available for patients with advanced photoreceptor loss are electrical retinal implants (e.g. Second Sight, Retina Implant). However, retinal implants only re-activate a very small area of the degenerated retina and the very few stimulating electrodes yield low image resolution, making face recognition and orientation in an unknown environment impossible.
The group of Dr Kleinlogel from the University of Bern, and others, have shown that expression of optogenetic, light-sensing protein in retinal neurons, recovers light sensitivity and vision at the cortical and behavioral levels in a mouse model of Retinitis pigmentosa.
Drug Repurposing for ALS
Identified and further supported by ReMedys since 2014, a drug repositioning project to treat Amyotrophic Lateral Sclerosis, from the laboratory of Prof Hornstein at the Weizmann Institute of Israel, secured appropriate resources, went through all preclinical development and manufacturing and is now available to patients in ongoing clinical trials. Results expected in 2023.
Altered expression of micro RNAs is recognized as a possible cause of neurodegenerative diseases.
It is believed to play roles in Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS) and Huntington's disease. miRNAs are abundantly expressed in the nervous system and play important roles in its regulation.
Research has shown that in ALS patient tissues and animal models of the disease deregulation of miRNAs are implicated in the pathology.
The laboratory of Prof Hornstein has shown that a well known and widely used antibiotic, enoxacin, can reestablish the maturation of miRNA and act as an anti-ALS agent in relevant preclinical models.
The ReMedys team worked since 2013 with Relief Therapeutics SA on the development of an innovative therapeutic that holds promise to stop and possibly reverse loss of nerves and make real difference for suffering patients.
The approach is to use Low-Dose Interleukin-6 As a Treatment Option for Diabetic Peripheral Neuropathy
The drug is entering in 2022 clinical trials supported by Sonnet Biotherapeutics.
Diabetic neuropathy (DN) is one of the most common complications of diabetes and occurs when nerve fibers are damaged by prolonged exposure to elevated levels of blood glucose. People suffering from DN often feel pain or burning sensations in their extremities, followed by numbness, ulceration and eventual limb damage and even amputation.
Over 50% of all people diagnosed with diabetes will suffer from diabetic neuropathy and it is estimated that, worldwide, over 374 million people have diabetes. Furthermore, those people most susceptible come from low- and middle-income countries where poor nutrition is prevalent.
There is no cure for diabetic neuropathy and most treatments focus on slowing disease progression, relieving pain and managing complications. Although tight glucose control and exercise can help reduce diabetic neuropathy in Type 1 (autoimmune) Diabetics, Type 2 sufferers (insulin resistance / deficiency) experience only modest improvements. Diabetic neuropathy affects all peripheral nerves including pain fibers, motor neurons and the autonomic nervous system. The burden of diabetic neuropathy for the patient is very significant with symptoms including numbness and tingling of extremities, burning or electric pain, vision changes, dizziness, muscle weakness, speech impairment, and fasciculation (muscle contractions). No therapy is available that could reverse or even stop progression.
Parasite Induced Cancer
In 2014 ReMedys recognize the urgent need to fight against a severe but neglected disease, Opisthorchiasis.
Thus in 2015 ReMedys, mobilized an international team of scientists and established a translational consortium to identify new treatments for this disease.
The work is ongoing and mainly supported by the Swiss Tropical and Public Health institute.
Opisthorchis or liver flukes are parasitic flatworms that are ingested with incompletely cooked food, especially infected fresh water fish, or due to poor sanitation. When ingested, the worms migrate from the small intestine into the bile duct where they can live for many years and produce eggs, which are excreted with the human feces.
Opisthorchis felineus is common in Russia where it is estimated that 1.5 million people are infected while Opisthorchis sinensis (or Clonorchis sinensis) and Opisthorchis viverrini are common in south and Southeast Asia. Especially in Laos and northeast Thailand the prevalence in humans is very high with roughly 10% of the population.
While infected people may show no symptoms, about 20 % have diarrhea, pain, lack of appetite, fatigue, jaundice, and/or fever.
In addition, Opisthorchis viverrini infections have been implicated in the etiology of malignant bile duct cancer (cholangiocarcinoma) in humans and have been categorized by the International Agency for Research on Cancer (IARC) as a Group 1 carcinogen. Cholangiocarcinoma is a metastatic tumor of epithelial cells derived from the bile duct, which is rapidly progressing and lethal. There is no treatment for the disease other than surgery, requiring the resection of the primary tumor and all metastases, and concomitant chemo- or radiation therapy.
The medical need for opisthorchiasis is early diagnosis. Many infected people do not show any symptoms that would make them consider seeing a doctor or get laborious and often difficult and expensive tests done to determine the cause of their problems. The parasite continues to produce eggs and enhances the chance for new worms going through their lifecycle and infecting other people.
But even more importantly the parasite will continue increasing the risk for the development of cholangiocarcinoma. Drugs are available to fight the parasite even if these have side effects and are only moderately effective. If a cheap, simple, early mass diagnosis could be achieved through the development of a simple kit similar to a pregnancy test, targeted treatment of infected would become possible and the development of cholangiocarcinoma could be prevented.