Ionis’ third novel antisense medicine for ALS, its first designed to treat a broad ALS population, begins clinical trial

CARLSBAD, Calif., Oct. 22, 2020 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in antisense therapeutics, today announced that the first patients have been dosed with ION541 (also known as BIIB105), an investigational antisense medicine being developed as a potential therapy to treat most forms of amyotrophic lateral sclerosis (ALS) regardless of family history. This is another milestone in the continuing progress of Ionis’ ambitious program to develop novel treatments for ALS. Almost all cases of ALS share the pathological hallmark of TDP-43 protein aggregation in motor neurons. ION541 targets ataxin-2 RNA (ATXN2), which has been shown to prevent or reverse TDP-43 toxicity in preclinical models of ALS.

(PRNewsfoto/Ionis Pharmaceuticals, Inc.)

ALS is a rare, progressive and fatal neurodegenerative disorder that affects approximately 55,000 people globally.i  About 90 percent of ALS cases occur in people who have no apparent family history of the disease. People with ALS experience muscle weakness, loss of movement, and difficulty breathing and swallowing, resulting in a severely declining quality of life and potentially death.

“As our third medicine designed to treat different forms of ALS to enter clinical trials, ION541 represents yet another example of the power of Ionis’ antisense technology to potentially target root causes of devastating neurodegenerative diseases,” said Frank Bennett, Ph.D., Ionis’ chief scientific officer and franchise leader for neurological programs. “Initiation of this clinical trial for ION541 marks an important milestone in Ionis’ ALS program and reaffirms our commitment to the ALS community.”

Ionis received a payment of $10 million from Biogen for initiation of this Phase 1/2 clinical trial of ION541. Biogen is developing ION541 as part of a broad strategic collaboration with Ionis to advance novel antisense therapies for the treatment of neurological disorders.

Learn more about the Phase 1/2 trial of ION541 at: https://clinicaltrials.gov/ct2/show/NCT04494256?term=biib105&draw=2&rank=1

Ionis’ other leading investigational medicines to treat ALS are tofersen (BIIB067) and IONIS-C9Rx (BIIB078), both partnered with Biogen. Tofersen is designed to reduce the production of superoxide dismutase 1 (SOD1), the cause of a genetic form of ALS, referred to as SOD1-ALS, that results from mutations in the SOD1 gene. SOD1-ALS is the second most common genetic form of ALS, accounting for up to 20 percent of genetic ALS. Tofersen is currently in a Phase 3 clinical trial in SOD1-ALS patients with data expected in 2021. IONIS-C9Rx is designed to selectively reduce the mutant C9ORF72 RNA and associated neurotoxicity. Mutations in the C9orf72 gene account for greater than 30 percent of genetic ALS cases and five to 10 percent of all patients with ALS. It is the most common genetic form of ALS worldwide. IONIS-C9Rx is the first drug to enter clinical development that specifically targets the mutant C9ORF72 RNA and is a potentially first-in-class therapy for patients with C9orf72-ALS, referred to as C9-ALS. IONIS-C9Rx, which earlier this year received Fast Track designation from the U.S. Food and Drug Administration, is currently in a Phase 1/2 trial in C9-ALS patients.

Ionis’ Forward-looking Statement

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Treating ALS With Orthomolecular Medicine

Amyotrophic Lateral Sclerosis or Lou Gherig’s Disease is a degenerative disease where neurons die early due to a problem with the autoimmune system. Your immune system attacks its organs and nervous system. The muscles degenerate and the body may begin wasting. This occurs because ALS destroys the motor neurons in the brain and spinal cord. The brain looses its ability to control its muscles.

It is very important to understand that there may be a viral component to this disease. The body must be supported with adaptogenic botanicals to handle the possible viral component. These adaptogens include astragulus, ashwaganda, gotu kola, siberian ginseng, panax ginseng, and other botanical products found in Quantum Orthomolecular Medicine. The use of adaptogens may not have the desired effect for the patient that has a genetically acquired disease. If a family member has the disease, these adaptogenic herbs may not produce the desired outcome.

Treating and healing ALS with ordinary amino acids and botanicals is the basis for Orthomolecular Medicine. Large doses of naturally found proteins called amino acids stop the attack on the brain and repair its malfunction. These amino acids and botanicals are affordable and are used in therapeutic doses much larger than those levels normally found in food. The concept of orthomolecular medicine is based upon the use of very large doses of vitamins, minerals, amino acids, or botanical extracts for the cellular repair and enhancement of normal brain activities.

The ALS patient should be given a three week parasite cleanse with one of the many herbal parasite cleanses that are available at your local health food store. This patient should be treated for heavy metals toxins with oral chelation for a period up to five weeks. You can consider extending the oral chelation if for another two weeks if the patient improves under this treatment. This patient should be treated very conservatively and any treatment where the patient does not improve should be discontinued.

This patient may be a good candidate for adrenal or thyroid glandular support. You may try an organic product and closely monitor the progress. Chinese patent herbs would be very good for this patient and they might delay the progression of this disease by several years.

Lymphatic therapy or massage therapy would be a good addition to the overall patient care. This should be tried once or twice a week. Reflexology can also benefit ALS.

This progressive and degenerative disease can be treated with Orthomolecular Medicine and this may be a curable disease. Conventional medicine does not believe that this disease can be healed. The onset of some symptoms may be delayed or kept at bay with very aggressive use of IV amino acids, vitamins, and minerals along with oral orthomolecular medicine two to four times per day. There are several key amino acids for the amino acid therapy and they include NAC, Threonine, glutamine, methionine, and tryptophan. Glutamine and NAC are very valuable tools to fight progressive and degenerative neurological diseases.

It is very important to …

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