ALS News Today brought you daily coverage of important discoveries, treatment developments, clinical trials, and other events dealing with amyotrophic lateral sclerosis (ALS) throughout 2018.
As a reminder of what mattered most to you in 2018, here are the 10 most-read articles of last year with a brief description of what made them interesting and relevant to ALS patients, family members, and caregivers.
Results from a Phase 2 trial (NCT02017912) in 48 patients with early-stage ALS showed that the investigative therapy can sustain immunomodulatory and protective effects by regulating levels of some small non-coding RNA molecules — identified as miR-34a, miR-132, miR-19, miR-376a, and miR-146a-5p — involved in several cellular processes.
The new private biotech company QurAlis announced in April that biotech investors MP Healthcare Venture Management (MPH), Amgen Ventures, and Alexandria Venture Investments had joined their efforts in the search for a cure for different forms of ALS.
The company is developing a device to remove toxic protein aggregates linked to the disease, a new therapy to target overactive nerve cells and prevent their death, and an investigational agent to restore normal activity of cells’ waste clearance system.
Upon the approval of Radicava (edaravone) by the U.S. Food and Drug Administration, marketed by Mitsubishi Tanabe Pharma America, the company asked Health Canada to consider the therapy as an intravenous treatment option for ALS.
The new drug submission was filed in March under the agency’s priority review program, and a positive decision was announced in October. Canadian patients with this progressive disease can now be treated with Radicava, which will be marketed by Mitsubishi Tanabe Pharma Canada.
The treatment is given in 28-day cycles by intravenous infusion, which may be done at an ALS center, a physician’s office, an infusion center, or a hospital outpatient department. Home infusion providers can also offer the treatment, depending on individual health insurance and physician oversight.
Interim results from an ongoing Phase 2 trial (NCT02714036) revealed that a combination of MediciNova’s investigational compound ibudilast (MN-166) with Rilutek (riluzole) can improve ALS patients’ functional activity, quality of life, and muscle strength.
Ibudilast is being developed to suppress specific pro-inflammatory molecules and increase the production of neurotrophic factors that will support the growth and survival of motor neurons (nerve cells that control movement).
Although the approval of Radicava was a remarkable event for the ALS community, ethical issues have been raised. ALS expects Crystal Yeo, MD, PhD, of Houston Methodist Hospital, and Zachary Simmons, MD, from the Pennsylvania State University College of Medicine, highlight the inability of the involved regulatory and ALS support entities to transmit the key fact that far from all patients are likely to benefit from the treatment.
In an article published in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, they presented aspects they argue should be part of every Radicava treatment discussion.
AT-1501 is an investigational antibody that targets the CD40L protein that can be found on the surface of some white blood cells that contribute to inflammation and neurodegeneration. It was developed by the ALS Therapy Development Institute (ALS TDI) with the support of several ALS organizations, including the ALS Association, ALS ONE, and ALS Finding a Cure.
The Phase 1 trial will evaluate the safety and tolerability of orally administrated AT-1501 in both healthy volunteers and eight people with ALS.
Results from Phase 1 (NCT01348451) and Phase 2 (NCT01730716) clinical studies have demonstrated that transplanting human spinal cord-derived neural stem cells (HSSC) into the spine of 20 ALS patients improved long-term survival and motor function.
Although the number of participants was low, the studies showed that this treatment strategy is safe. Additional studies are still warranted; these positive findings are expected to support the design of a Phase 3 clinical study.
More information about enrollment can be found here.
Following ibudilast’s positive results when administered in combination with Rilutek, the U.S. Food and Drug Administration approved MediciNova’s Phase 3 developmental plan for the investigational therapy.
To achieve the best result possible, the regulatory agency suggested that a future Phase 3 trial should include a broader ALS population. In addition, randomization and data analysis should be stratified according to a patient’s disease severity at the start of the study.
If researchers demonstrate clinically meaningful benefit of ibudilast in ALS patients’ functional activity over a placebo, MediciNova believes that further trials may not be necessary.
A preclinical study with cells collected from ALS patients revealed that Requip (ropinirole), a medication already approved for treatment of Parkinson’s disease, may be a potential therapeutic agent for ALS.
Requip acts as a substitute for dopamine in the brain. In ALS models, the therapy was found to modulate the activity of motor nerve cells as well as the activation of pro-inflammatory pathways, which suggests that it may hold therapeutic potential for ALS.
ALS News Today hopes that these stories, along with our continuing reporting throughout 2019, will help educate, inform, and improve the lives of patients living with ALS and their loved ones.
We wish all our readers a happy and inspiring 2019.
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