New Gene Therapies Show Promise for Genetic Neurology Conditions and Blood Disorders

• May 02, 2019
• Amber Specialty Pharmacy
• Amber Specialty Pharmacy

Breakthrough genetic therapy options are starting to complete the final stages of clinical trials and reach the market. Gene therapies provide hope where few treatment options are available. These potential treatment options are the result of significant medical advancements and years of research. As these exciting medications reach consumers, learn more about new gene therapies for neurology conditions and blood disorders.

What are Gene Therapy Treatments?

Genes are inherited traits that provide the information your body needs to function normally. Gene therapy treatments correct genetic problems at the source. New treatment options for people with genetic disorders target specific genetic mutations and DNA sequences. The treatments can insert properly functioning genetic copies to reduce the effects of the genetic disorder and prevent it from spreading. The Genetics Home Reference with National Institutes of Health (NIH) provides more information about how gene therapy works.

While researches have studied gene therapy for years, the first wave of treatment options is starting to complete clinical trials and receive approval from the Food and Drug Administration (FDA). These highly targeted treatments have spent years in the development pipeline. For the people managing serious genetic disorders with few treatment options, gene therapy medications offer tremendous hope.

The Importance of Genetic Testing

With new genetic medications making their way through the development pipeline, it’s important for healthcare providers to watch for these new treatment options. Genetic therapy is highly targeted, which means genetic testing is becoming more important before starting a patient on treatment plan. As more treatments target specific genetic abnormalities, healthcare providers need to be vigilant about ordering the right genetic lab tests before prescribing medications. Patients should understand that not every new genetic therapy option will be appropriate for their diagnosis. Information from genetic tests can also provide researchers with valuable insight to genetic conditions and pave the way for new treatments.

The trade relations team at Amber Specialty Pharmacy monitors product pipelines across the entire pharmacy industry. While there are many new genetic therapies nearing the market, here are four treatment options to watch.

New Gene Therapies for Genetic Neurology Conditions

Spinal muscular atrophy (SMA) is a genetic disorder that affects the motor neurons. It’s one of the most life-threatening neurological genetic disorders. In severe cases, it can leave children completely unable to move. A breakthrough new gene therapy to treat SMA is in the final phases of clinical trials. AVXS-101 is a single dose intravenous treatment that has shown exciting promise. While AVXS-101 is still in development, gene therapy is giving children and families with this diagnosis hope where there are currently few treatment options available.

Amyotrophic lateral sclerosis (ALS) is a disease characterized by motor neuron degeneration. As ALS progresses and motor neurons die, people with this condition gradually lose the ability to speak, move and breathe. VM202 is investigational gene therapy to slow the progression of ALS and increase the lifespan of people diagnosed with the disease. Injected directly into the affected muscles, VM202 helps the body produce more of a specific protein called hepatocyte growth factor (HGF). HGF can trigger neuron repair and growth. In addition to its potential for ALS patients, VM202 is being investigated for the treatment of coronary and peripheral artery disease and diabetic peripheral neuropathy.

New Gene Therapies for Genetic Blood Conditions

Beta-thalassemia is one of the most common genetic blood disorders. This potentially serious condition causes reduced hemoglobin production. Hemoglobin is an important part of blood that helps carry oxygen throughout the body. There are currently few treatment options available for people with beta-thalassemia. In severe cases, patients with this blood disease receive regular blood transfusions. Over time, regular blood transfusions can cause iron buildup and additional complications.

Investigational gene therapy treatments focus on reducing or eliminating a beta-thalassemia patient’s need for blood transfusions. One medication, called LentiGlobin™ gene therapy, shows promise in phase three clinical trials. The treatment takes a patient’s own blood cells, treats them outside of the body and returns a healthy, functioning copy of the cells to the patient. The findings of this study have the potential to help other patients with genetic blood disorders, including sickle cell disease.

Hemophilia A is another blood disorder with new genetic therapy treatment options making their way through the development pipeline. Hemophilia A is caused by a defective clotting protein. People with this condition have an increased risk of excessive bleeding and other complications. A breakthrough genetic therapy called Valoctocogene Roxaparvovec is currently in phase three clinical trials. This treatment aims to reduce or eliminate spontaneous bleeds and improve a patient’s overall quality of life.

Amber Specialty Pharmacy’s Trade Relation Team Monitoring Product Pipelines

Gene therapy is a new and developing treatment option. Medications are finishing clinical trials and receiving approvals, but research is ongoing and the cost of genetic therapy remains high. The trade relations team at Amber Specialty Pharmacy constantly monitors the drug development pipeline. We’re excited about the potential for new gene therapies and the potential to access new treatment options to help our patients manage genetic disorders.