Stella went into the hospital with a pain so great it cripples her entire being. “It feels like a stabbing needle pain that shoots through my joints” she said. No longer able to carry on her day to day routine she finally gives in after a few pills of over-the-counter pain medication did not work, and heads to the emergency room. Her body is used to taking high doses of pain medication to alleviate the pain, so no regular pill will work. The doctors and nurses in the hospital are too familiar with the patient; the only medication they can give her is a highly potent medical narcotic. Stella, who wishes to remain anonymous, has been in the hospital at least twice before within the past 30 days, with each stay lasting more than a couple of days--Stella has Sickle Cell Disease.
While normal red blood cells are round and can move freely through the veins, blood cells within Sickle cell patients contain abnormal hemoglobin which causes the sickle-shapes, or crescent; these cells are stiff and sticky. They often block blood flow in the blood vessels around joints and in organs. The pain from the blockage of blood flow is referred to as a crisis. Most are able to overcome some of the pains, while others lose their life to the various medical diagnoses that are associated with treatments for Sickle Cell patients.
All patients with the chronic illness have built a high tolerance for most over-the-counter medication. Over the years, potent doses of medical narcotics cease to be effective in alleviating the pain. Though there is various medical research towards helping patients live a normal life with their disease, no single treatment has been widely known to be effective.
However, an innovative biopharmaceutical company based in Gaithersburg is setting the stage for a new era towards the treatment for Sickle Cell Disease. GlycoMimetics, co-founded by Rachel King, is focused on helping patients manage their pain at the onset of a crisis. King said that when it comes to treating Sickle Cell "there are still no drugs that can be given to somebody who is actually in a crisis. So we felt that it was an opportunity to really change the way that these patients are treated.”
GlycoMimetics is a privately held biotech company developing small-molecule drugs to treat a broad range of cancers and inflammatory diseases. GlycoMimetics’ current drug GMI-1070 is on the verge of unlocking a totally new approach towards treatment for vaso-occlusive crisis in Sickle Cell Disease.
The unconventional method of treatment for a disease that has been overlooked by the healthcare industry for decades-- is in such dire need-- the Food and Drug Administration (FDA) sought it critical that GlycoMimetics' drug be granted Orphan Drug and Fast Track status. The Orphan Drug Designation is given to developmental drugs that meet the need of 200,000 people or less in the U.S who have a rare disease. The Fast Track designation program provides expedited review of new drugs which are designed to treat unmet medical needs, so the drugs can get to patients quicker.
GMI-1070 is a synthetic glycomimetic molecule which mimics glycan properties-- that often make up the structure of molecules in antibiotics and anticancer therapies. The Sickle Cell treatment is designed to target and inhibit the inflammation process that occurs during a crisis. The advantage here--is that GMI-1070 will inhibit every one of the three selectins (cell -adhesion molecules associated with inflammation), while other drugs may only target one or imprecisely cover a broad range.
Currently, the most popular form of treatment provided to Sickle Cell patients is admission into the emergency room. Mitigating the pain often times involves powerful medical narcotics like Demerol, Percocet, and IV Fluid for dehydration. Some extreme cases may even call for blood transfusion. A few patients have opted to go as far as undertake a Bone Marrow transplant to help alleviate them from the sickle cell disease.
In the past ten years Hydroxyurea has been the drug of choice for some sickle cell patients. This drug was initially developed as a chemotherapy agent that had potent effects on the bone marrow. However, in 1995 the drug was prescribed to some Sickle Cell patients because it was found to change some red blood cells from having an abnormal shape. The drug is meant to control the symptoms of sickle cell but not cure the condition, and once the patient is on the medication they must be monitored constantly for possible side effects.
These treatments are not cheap and often times do not stop the painful crisis that every sickle cell patient experiences. However, GlycoMimetics’ drug GMI-1070 promises to change how sickle cell patients deal with the chronic pain brought by a Sickle Cell crisis. By inhibiting selectin interactions, GMI-1070 may be able to decrease the enhanced cell adhesion that results in vaso-occlusive crisis. In preclinical studies GMI-1070 restored blood flow to affected vessels of sickle cell animals experiencing vaso-occlusive crisis. The drug is currently in Phase II after successful trials during Phase I.
If the drug passes the Phase III process and receives FDA approval, the treatment will cut down on the costs of hospital stays; which can spiral to an expense range between $20,000 to as high as $40,000 in states like California for example, where hospital stays for patients are estimated to be the highest. Since the amount of Sickle Cell patients on Medicare/Medicaid is estimated to be 70%-if GlycoMimetics’ drug proves effective with measurable efficacy and stamp of approval from the FDA, it can end up decreasing the amount of government spending on Sickle Cell patients.
Instead of emergency room admissions King noted that “it would be optimal to have a much more streamline system where people didn’t have to go to the emergency room, and they can go directly to some sort of center where they can be treated immediately for their Sickle Cell crisis.” Her vision is that eventually "patients can give themselves the drug at home.”
For Stella and other people within the Sickle Cell Community a drug like this just might be what they have been waiting for. If the drug passes Phase III and it becomes available to patients it can liberate a large group of people who have had their life affected by their illness. People like athletes who have the disease can use the drug to stop the crisis from happening. The medical community will also be greatly affected by this potential new treatment, King noted that “if successful it could be an opportunity to hopefully change the way the disease is managed.”