Clinical Studies
Our
Clinical Studies
Alzheimer's Disease
It robs patients not only of their most precious memories but also the ability to care for themselves. Of the top 10 killers of Americans only 1 disease has no treatment that slows down, arrests, or stops the progression. It is Alzheimer's. This is a disorder in which over 5,000,000 Americans currently suffer. It is growing at a phenomenal rate. By the year 2050 is expected that over 15,000,000 Americans will suffer with this disease. That is if no treatment is found. In addition to the emotional toll that the disease takes, it also strikes Americans financially. It is estimated that over $200,000,000,000 are spent annually for this disease. This includes lost wages to care for sick family members, insurance premiums, medication costs, hospitalizations, etc. We hope to be part of the solution.
Parkinson's Disease
Although much less common than Alzheimer's disease, better treatments are urgently needed. Once thought to be a disease that only interfered with coordinated movement, it is now better understood to impact not only the whole nervous system but the whole body. Typically starting as a unilateral innocuous tremor, it eventually progresses to the point that it interferes with the patient’s ability to safely walk. The disease can cause random combinations of the following symptoms including: depression, difficulty swallowing or chewing, urinary problems, constipation, dry scaly skin, pain, sleep problems and cognitive problems, such as memory problems or slow thinking, unstable blood pressure, and acting out of dreams.
Essential Tremor
Up until approximately 2014 it was felt that essential tremor was 1 disorder where the patient would have involuntary rhythmic trembling of the hands voice or head. Classic essential tremor usually is not noticeable with the patient's hands at rest. However as soon as the patient attempts to use the hands they begin to shake. The shaking is described as a fast (8-12Hz) of low amplitude in most cases. Since 2014 however it is now felt that essential tremor is better described as essential tremors. This is because it was felt that it is more than 1 disease based on age of onset initial symptoms and progression of the symptoms over time. We are now beginning to understand that there are other features associated with tremor.
As described above the age of onset is variable. It is what we call bimodal, This means that it clusters around two time periods of life. Most individuals will get tremor either at a relatively young age (20-30s) or at a relatively old age (60s and 70s) with lower likelihood of acquiring tremor in the middle years. The evolution of tremor follows two typical patterns. These rules are not absolute but most patients will fall into 1 of these categories. There is the late life onset which has a tendency to be progressive with worsening of symptoms with passing years. Early-onset tremor under the age of 40 is typically stable for many years and then decompensates as the individual gets into their seventh and eighth decades of life. We are finding that there are genes associated with tremor that is seen in families. There is a growing list of possible genetic abnormalities associated with tremor. More research is required to determine exactly how these genetic abnormalities influence the disorder.
Typically to treat these medications propranolol, Mysoline, or topiramate are used. Many patients find that these medications are less than satisfactory and controlling their symptoms. Side effects from the propranolol may include but are not limited to lightheadedness dizziness shortness of breath swelling of the limbs and syncope. Most common side effects of Mysoline include dizziness fatigue and unsteadiness of gait. Topiramate most common side effects include mental slowing., tingling of the fingers, dizziness, and instability of gait.
Multiple Sclerosis
The team at AQR has years of extensive trial experience in the development of treatments for Multiple Sclerosis (MS). MS is a chronic and debilitating autoimmune disease of the central nervous system. MS is the leading cause of nontraumatic disability in young adults. The past decade has shown remarkable advances in the treatment of the inflammatory relapsing phase of the disease. Despite this, there is still gaps in our treatment paradigm.
We now recognize that early treatment with effective therapies favorably impacts long term outcomes of patients with MS. The progressive component of relapsing forms of MS (RMS) and Primary progressive MS (PPMS) appears to be driven by smoldering inflammation inside the brain and spinal cord. Presently, effective treatments for the progressive phase of MS are lacking.
Here at AQR we are actively recruiting for involvement in several trials which focus on small therapeutic molecules that are able to penetrate the central nervous system and address this process. This is coupled with cutting edge imaging and biomarker analysis that will lead to new ways of monitoring and assessing the effectiveness of these treatments.
Personalizing and tailoring therapy to the individual with MS is extremely important. Each patient has their own unique set of needs and goals. We are striving to develop therapies that safely capitalize on the unique nature of the immune system and harnessing the ability to safely safe regulate the misdirected immune response that results in MS. That is to say—turning on the bodies ability to dampen the specific overactive immunity the causes MS.
We are also interested and researching how the current treatment landscape affects our bodies abilities to mount responses to vaccine therapies, which is increasing becoming more important in the pandemic era.
Migraine
Migraine is a disorder of pain processing. It usually presents itself as attacks lasting typically between 4 and 72 hours with pain in the head of moderate to severe intensity.
The pain is frequently unilateral and pounding. With the pain frequently comes other features. This can include nausea, vomiting, diarrhea, light sensitivity, sound sensitivity, behavioral changes, and mental fog. It is estimated that up to 25% of women suffer from migraine headaches and up to 10% of men suffer from migraine headaches. Typical age of onset for men is about 10-11 years of age and for women 14-17 years of age. However there are clear exceptions to these guidelines and people well into late life have been described as starting to have migraines. Additionally women are more likely to be affected. Migraine is more likely if a first-degree relative (mother,father, or sibling) has the disorder. Overall this disease is relatively benign with very rare reports of people suffering permanent neurologic injury as a result of migraine headache.
Treatment options are numerous including blood pressure medications, anti seizure medications, over-the-counter medications, ergots, triptans, and most recently CGRP inhibitors. Despite a wide array of medications available for the management of both acute attacks and the prevention of future attacks some patients are still unresponsive to treatment.
Neuropsychiatric Conditions
Depression, anxiety, and other psychiatric symptoms are common alone or in combination with many neurologic diseases. AQR is actively involved in finding new therapies for treatment of these conditions.
Psychosis, agitation, and apathy (loss of interest) can complicate disease like Alzheimer and Parkinson’s disease. These symptoms are very challenging to treat and there is a lack of options to effective manage these symptoms. These are areas of active research interest at AQR
Twenty Million people in the United States suffer from clinical depression. The impact of depression on the individual and family can be profound. Currently approved therapies often take time to work. In about one third of individuals the medications are less than adequate ineffective. This is called treatment refractory depression. AQR is evaluating patients for enrollment in a treatment refractory depression study.
We continue to expand our role in the discovery of novel treatment of neuropsychiatric conditions where there is a need for safe and effective therapeutic interventions. With the help of patients and their families we are hopeful that we will be able to advance these treatments.
Anxiety
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