AI Therapeutics is a biopharmaceutical company that has created an artificial intelligence platform that is meant to match drugs to new indications. This platform, called Guardian Angel, has matched four drugs to indications, all of which are now in clinical trials. Two of these drugs are LAM-001, which treated those with lymphangioleiomyomatosis (LAM disease) in a clinical trial, and LAM-004, which is used for the treatment of facial angiofibroma and other skin diseases.
About Lymphangioleiomyomatosis (LAM Disease)
Lymphangioleiomyomatosis, also known as LAM disease, is a lung disease that occurs when the bronchial tubes and lymphatic vessels are blocked, along with holes and cysts in the lungs. These blockages and holes make breathing difficult. Women are primarily affected by this condition, and they are usually diagnosed between the ages of 20 and 40. It has also been associated with tuberous sclerosis, as about 30% of women with LAM disease also have tuberous sclerosis.
This disease occurs when the TSC1 and TSC2 genes are mutated. Some cases of LAM disease are hereditary, as they are associated with tuberous sclerosis. In the other 70% of cases, the mutations are sporadic. If one has these mutations, they can expect symptoms like shortness of breath, chest pain, a cough, wheezing, collapsed lungs, and pleural effusions.
An incorrect diagnosis is common for this disease, as it shares many of the symptoms of bronchitis and asthma. Cysts in the lungs are often misdiagnosed as emphysema. Certain tests can help to diagnose this condition, such as lung biopsies, CT scans, lung function tests, pulse oximetries, and VEGF-D blood tests. After a diagnosis is obtained, treatment can help to manage symptoms and stop progression. Sirolimus was recently approved by the FDA for the treatment of LAM disease. Oxygen therapy, medications that are inhaled to help air flow, procedures to remove fluid, and lung transplants in severe cases are all options for the treatment.
About LAM-001
LAM-001 is an inhaled medication for the treatment of LAM disease. It is an inhaled form of rapamycin, which is typically an oral drug that has the potential to have adverse effects. In the inhaled form adverse effects are minimized. Clinical trials have been completed, and they have proven to stabilize lung function. LAM-001 has been granted Orphan Drug designation in the United States and in Europe.
This drug is also being studied for the treatment of pulmonary arterial hypertension (PAH). It has been given Orphan Drug designation in the U.S. for the treatment of PAH. Researchers hope that it will be granted a Phase 2 trial so that it may be further studied.
About Facial Angiofibroma
Angiofibroma is a type of vascular tumor. They are made up of connective tissue and blood vessels and appear as small, red bumps on the face. Like LAM disease, facial angiofibroma is common in those with tuberous sclerosis. These tumors may also occur in the nasal cavities.
These tumors can cause symptoms such as nasal obstructions, nose bleeds, runny noses, and facial deformities like swollen cheeks, drooping eyelids, bulging eyes, and cranial nerve palsies. Once these symptoms are noticed, physical exams, biopsies, MRIs, and CT scans are used to obtain a diagnosis.
Surgery is the most common treatment for these tumors. Complementary treatments, such as radiation and chemotherapy, may be necessary after surgery. At times embolization happens before surgery.
About LAM-004
LAM-004 is a topical form of rapamycin. It has received Orphan Drug designation in the United States for the treatment of facial angiofibroma. The first phase of a clinical trial has been completed and it has shown to be safe and well-tolerated. This drug also has the potential to treat other skin diseases.
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