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2021 Brain Disease Research Recap

2021 Brain Disease Research Recap

Here are some of this year’s exciting breakthroughs and developments in brain disease research

As this year comes to a close, the American Brain Foundation would like to reflect on the groundbreaking research of 2021. We believe in the power of research to unlock advances in our understanding of the brain and connections between brain diseases so that when we cure one brain disease, we will be able to cure many. We know that this year’s findings will help lay the groundwork for future innovations and insights to improve brain disease prevention, diagnosis, treatments and cures. Here are a few of the brain disease research highlights of the past year.

Breakthroughs in Alzheimer’s Disease

In 2021, there have been multiple research developments in the area of Alzheimer’s disease. In the past, a definitive diagnosis of Alzheimer’s was only possible with an autopsy. Thanks to new technological discoveries, it is now possible to identify early signs of the disease with a blood test. This discovery reduces the need for costly, invasive brain scans and spinal fluid testing. It also contributes to improved treatments.

Among those treatments is Aducanumab, a controversial new drug that received FDA approval this year. The intravenous drug works to clear the plaques in the brain that disrupt cell function in people with Alzheimer’s. This is the first therapy to target these specific changes in the brain. This treatment gives hope of extending independence, daily activity, and memories for individuals with this disease. While there are still questions surrounding this drug, it is an indicator that exciting breakthroughs could be on the horizon.

This year also produced the groundbreaking discovery that Alzheimer’s presents in three distinct disease subtypes. This information can help identify which patients are most vulnerable to which subtype. It can also show how the disease may progress and pinpoint the best treatment for an individual. Another new study found that brain tissue inflammation may be a potential cause—not a consequence—of this disease’s progression. This breakthrough means that targeting neuroinflammation in people with early-stage Alzheimer’s may help reverse or slow down dementia.

Other Brain Disease-Specific Research

We’ve also seen many other disease-specific findings this year. These include breakthroughs for spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), glioblastoma, and more. Leading this year’s headlines is the extended availability of the world’s most expensive drug, Zolgensma, which costs over $2.5m per dose. Research led by Jerry Mendell, MD, 2019 recipient of the American Brain Foundation’s Scientific Breakthrough Award, uncovered this one-time gene therapy infusion that cures Type 1 SMA. The treatment has been available in the U.S. for some time. But it recently became available through NHS, the national healthcare service in the U.K. A five-month-old boy received the treatment just three weeks after his diagnosis.

Looking further, this year, researchers found a new compound that can repair ALS nerve damage. This finding also brings hope to people with other motor neuron diseases. Additionally, an early clinical trial showed that a new experimental drug crosses the blood-brain barrier to kill tumor cells in people with glioblastoma, a deadly brain cancer. This discovery may also have applications to other neurological diseases and brain tumor treatments.

Advancements Across Multiple Disease Areas

We have seen examples of our mantra, “cure one, cure many,” come to light in many ways in 2021. This year, there have been exciting research developments with implications for multiple disease areas. For example, the gene therapy for SMA led to research into treatments and cures for other types of muscular dystrophy. 2021 Next Generation Research Grant recipient Renatta Knox, MD, PhD, has been working to build on the SMA research and apply gene therapy to Facioscapulohumeral muscular dystrophy (FSHD) and other diseases.

Also, in recent news, Mayo Clinic and Google Research developed a new artificial intelligence algorithm to simplify data and gain a better understanding of how different areas of the brain interact during electrical brain stimulation. This technique will help improve electrical stimulation devices, which are currently used to treat epilepsy and movement disorders like Parkinson’s disease. The hope is that in the future, the algorithm could help expand the possibilities of electrical stimulation. This may lead to treatments for people with psychiatric disorders and brain injuries, such as stroke.

Highlights from the American Brain Foundation

The American Brain Foundation is proud to have launched new research funds and supported nine additional Next Generation Research Grant (NGRG) recipients in 2021. Within the NGRG program, the Foundation partners with the American Academy of Neurology and other organizations to invest in new generations of clinical neuroscientists.

This year, the Foundation and the American Academy of Neurology (AAN) introduced a $150,000 scholarship. This receives funding from Hearst Foundation and Eisai Inc to support research in understanding and reducing neurological healthcare disparities. The American Brain Foundation and the AAN also established a grant of up to $60,000 for autism research specifically among excluded and underrepresented populations.

To honor our Scientific Breakthrough Award recipient Peter Goadsby, MD, PhD, and his contributions to migraine research, the Foundation also started the Goadsby Headache Research Fund. Dr. Goadsby’s work includes the discovery of a key peptide’s role in migraine. The research has resulted in many clinical trials for preventative and acute treatments for migraine. His findings prove that the more research we do, the more answers we’ll find and the more people we’ll help. This fund will support innovative research projects to advance the understanding of migraine. It may also help develop treatments as well as research that addresses health disparities in headache and migraine.

The American Brain Foundation was founded to bring researchers and donors together in the fight against brain disease. Next Generation Research Grants fund the innovative research of early-career investigators, encouraging passion for research and laying the groundwork for future success. Learn more about brain disease or make a gift to support groundbreaking brain disease research.