Q. I have a family history of Alzheimer’s on my mother’s side, and my younger brother was diagnosed with amyotrophic lateral sclerosis (ALS) three years ago at just 40 years old. I have been participating in fundraisers and awareness events to raise money for research for both of these diseases. I heard a new Alzheimer’s drug is on the horizon and that an ALS drug was recently approved by the FDA. Do you have any information about these drugs, and do they look promising for those suffering from these devastating diseases? Thanks for your help!
A. There is promising news on both fronts when it comes to drugs for Alzheimer’s and ALS. We will discuss the new ALS drug first, as it was just approved by the Food and Drug Administration yesterday, September 29, 2022!
Amyotrophic Lateral Sclerosis (ALS), which is sometimes called “Lou Gehrig’s disease” for the renowned baseball player diagnosed in 1939, paralyzes patients, robbing them of their ability to walk, talk, and eventually breathe. Patients typically die within three to five years, though some live much longer with the disease. About 30,000 people in the United States have ALS, with 6,000 new cases diagnosed every year.
As mentioned, yesterday the FDA approved a highly-debated drug for ALS, a move that encouraged patients and advocates who pushed for the medication but raised concerns among some experts about whether this new treatment has received enough scrutiny. Two other drugs — including Radicava, which came to the U.S. market in 2017 — are approved for the ailment but have extremely limited effectiveness.
Here’s what you need to know about the new ALS drug:
- The newly approved therapy, which will be sold under the brand name Relyvrio, is designed to slow the disease by protecting nerve cells in the brain and spinal cord destroyed by ALS.
- The drug consists of two components — a prescription drug called sodium phenylbutyrate used to treat rare liver disorders and a nutritional supplement called taurursodiol. The drug comes in a powder that is dissolved in water and can be swallowed or given through a feeding tube.
- Relyvrio is the first new therapy approved for ALS in five years.
- It was demonstrated in a 24-week study in which 137 patients were randomized to receive Relyvrio or placebo. The patients treated with the drug experienced a 25 percent slower rate of decline in performing essential activities such as walking, talking, and cutting food compared with those receiving a placebo.
- A long-term analysis showed that patients who originally received Relyvrio versus those who took the placebo lived longer. Amylyx, the Cambridge, Mass., biotech company that makes the drug, said that survival benefit was a median of about 10 months.
- During reviews of the drug, the FDA staff expressed concerns about the medication’s effectiveness and posed questions about the clinical trial.
- The agency acknowledged there were “limitations” to the data that resulted in uncertainty about the drug’s degree of effectiveness.
- They also said that regulatory flexibility was acceptable because of the “serious and life-threatening nature of ALS and the substantial unmet need” for treatments.
- Amylyx officials said they plan to move as quickly as possible to make the drug available.
- In 2014, the ALS Therapy Development Institute raised $115 million in six weeks from the Ice Bucket Challenge and provided $2.2 million of that to help pay for testing this particular drug. The medication is the first funded by the organization to receive FDA approval. Amylyx has agreed to use proceeds from sales of the medication to repay the organization 150% of its investment!
- Some drug policy experts, however, said insufficient evidence exists that the drug works. A trial with 600 patients won’t be completed until late 2023 or early 2024.
“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” Billy Dunn, director of the FDA’s Office of Neuroscience, said in a statement.
Jinsy A. Andrews, an associate professor of neurology and director of neuromuscular clinical trials at Columbia University, applauded the approval and said she plans to start prescribing the drug as soon as it is available. Other ALS specialists agreed. “I see patients living with this disease, and I diagnose them every day,” Andrews said. “So to have another therapy for the tool kit is helpful.” Andrews is an investigator in the large trial for the drug now under way.
Opponents of the drug being approved mention how the FDA approved Aduhelm, a drug for Alzheimer’s last year despite an overwhelming vote from the advisory committee that the standard had not been met and many arguing that the drug was too expensive and didn’t work. It was the first new Alzheimer’s drug approved by the FDA in almost 20 years. The Journal of American Medicine (JAMA) rejected Biogen’s key Aduhelm paper and shortly thereafter, Medicare chose to limit coverage of the drug.
With the new ALS drug being approved, those who are opposed to the quick approval fear that it could send a troubling message to pharmaceutical companies that they don’t have to show that a drug works, and that they only have to do the bare minimum to show that it might work.
New Alzheimer’s Drug Is Nearing Possible Approval Early Next Year
As mentioned, Aduhelm was approved last year but didn’t work out as everyone had hoped. But another promising Alzheimer’s drug may be on the horizon. The Japanese drugmaker Eisai’s experimental drug for Alzheimer’s disease has shown to help slow cognitive decline in patients in the early stages of the illness.
Initial data suggests that the new drug is proving quite successful in late-stage clinical trials. In fact, the company said that “in a phase 3 clinical trial, the drug, called lecanemab, slowed cognitive decline by 27% after 18 months.”
The results may offer renewed hope to Alzheimer’s patients after the U.S. drugmaker Biogen’s botched rollout of Aduhelm last year. Biogen partnered with Eisai in the commercialization of the new drug, although Eisai is leading its development and the phase three trial.
- The results are based on 1,795 patients, who were randomly assigned to receive either the drug or a placebo every two weeks over 18 months.
- Cognitive decline was measured using a clinical dementia rating scale that focused on six areas: memory, orientation, judgment and problem solving, community affairs, home and hobbies, and personal care.
- Eisai said that it will present the findings at an Alzheimer’s conference in late November and that it plans to submit the trial data to the Food and Drug Administration for approval by March.
- The company noted, however, that the FDA has already agreed to consider the drug under the agency’s so-called accelerated approval pathway — the same process used to approve Biogen’s Aduhelm. Eisai said the agency is expected to make a decision on conditional approval by early January.
Michel Vounatsos, Chief Executive Officer at Biogen, said in a press release, “We believe defeating this disease will require multiple approaches and treatment options, and we look forward to continuing the discussion about the significance of these findings with the patient, scientific, and medical communities.”
Hopefully, both of these drugs will prove to be effective in helping those suffering from ALS or Alzheimer’s Disease.
Planning for a Loved One with a Degenerative Disease
If you have a loved one who suffers from ALS, Alzheimer’s, or another degenerative disease, it’s important to plan in advance. Those with degenerative diseases and their families face special legal and financial challenges. At the Farr Law Firm, we are dedicated to easing the financial and emotional burden of individuals suffering from degenerative diseases and their loved ones.
If you or a family member are facing a diagnosis of ALS, Alzheimer’s, or any other degenerative disease, please call us to make an appointment for an initial consultation to discuss life care planning and asset protection in connection with obtaining Medicaid and Veterans special pension benefits:
Medicaid Planning Attorney Fairfax, VA: 703-691-1888
Medicaid Planning Attorney Fredericksburg, VA: 540-479-1435
Medicaid Planning Attorney Rockville, MD: 301-519-8041
Medicaid Planning Attorney Washington, DC: 202-587-2797
About Evan H Farr, CELA, CAP
