Success Stories

A success creates a "new" repurposed treatment for a single disease, or a repurposed treatment that can help patients across several diseases. A success may be used off-label by physicians and patients today, or it may have received funding for a larger clinical trial intended to lead to FDA marketing approval. We are proud to share the following successes, highlighting the power of repurposing to quickly, powerfully and inexpensively transform patient lives.

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teachey-davidPrinciple Investigator: Dr. David Teachey

Disease: Autoimmune Lymphoproliferative Syndrome

Research Description: Autoimmune Lymphoproliferative Syndrome (ALPS) is a rare childhood autoimmune disease. Patients suffer from enlarged lymph nodes and spleen, increased infections and anemia. ALPS patients may spend as many as 5-10 days in the hospital each month and may not survive beyond their teens. Dr. Teachey tested the repurposed generic drug sirolimus, a drug used to prevent rejection in organ transplants, first in mice and then in a pilot clinical study among six patients at the Children’s Hospital of Philadelphia, in which five of the six patients were in complete remission in 90 days. Patient hospitalizations declined, and patients have continued to survive and thrive on sirolimus. The cost to treat these patients has also significantly decreased, resulting in lower medical bills.

CWR funding role: Participating funder

Publication:  British Journal of Haematology

Since publication:

→ Data are supporting off-label clinical use

→ Findings from project led to additional applications of repurposed treatment in other pediatric autoimmune diseases.

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teachey-davidPrinciple Investigator: Dr. David Teachey

Disease: Autoimmune lymphoproliferative syndrome, Evans disease, systemic lupus erythematosus, autoimmune hemolytic anemia, idiopathic thrombocytopenic purpura, common variable immunodeficiency

Research Description: Children with autoimmune diseases that affect their blood cells require aggressive treatment with immunosuppressive drugs. These drugs are not very effective and have excessive side effects. Dr. Teachey previously investigated the use of sirolimus, a safe generic drug with few sides effects, to successfully treat autoimmune lymphoproliferative syndrome (ALPS). Based on these results. Dr. Teachey tested sirolimus in children with other autoimmune diseases. A total of 30 children were enrolled in the clinical trial, and sirolimus led to a response in 22 children. The results support sirolimus as a treatment option for children suffering from six types of autoimmune diseases. Sirolimus has the potential to improve quality of life and lower medical costs for many of these patients.

CWR funding role: Primary funder

Publication:  Blood 

Since publication

→ Data are supporting off-label clinical use

University of Wisconsin

Kurt KaczmarekYuri DanilovPrinciple Investigators: Dr. Kurt Kaczmarek and Dr. Yuri Danilov

Disease: Multiple sclerosis

Research Description: Multiple sclerosis (MS) is a debilitating disease that affects the nervous system and results in loss of function due to brain cell death. Drs. Kaczmarek and Danilov repurposed an electrical stimulation device called a PoNS (Portable Neuromodulation Stimulator) to make a direct impact on MS patient lives. In this clinical study, combining mild electrical stimulation of the tongue using the PoNS with physical therapy was able to create new nerve pathways in the brain, or to make current pathways work better. Following an intensive two-week training period, patients regained balance, movement, vision and hearing loss, reduced fatigue and improved sleep, cognitive and mood disorders. Use of the device at home can increase the functional improvements or maintain what was gained. This project led to testing on stroke, TBI and Parkinson's disease patients with similar impact (see Research in Progress). This device, with Cures Within Reach support, is now being managed by Helius Medical, and there are clinical trials underway to support FDA review and approval.

CWR funding role: Primary funder

Publication(s): Journal of NeuroEngineering and Rehabilitation

Since publication:

→ Data are supporting large scale clinical trials

→ Data leveraged into larger philanthropic or government funding for this treatment

→ Findings from project led to unanticipated discoveries in additional neurodegenerative diseases, now in testing

→ Data are supporting commercialization

 

 

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Luis Porrata HeadshotPrincipal Investigator: Dr. Luis Porrata

Disease: Non-Hodgkin Lymphoma

Research Description: Dr. Porrata’s clinical trial sought to improve the existing autologous stem cell transplant (ASCT) protocol used to treat non-Hodgkin's lymphoma patients. The existing method involved removing healthy blood cells from a patient, treating the patient with chemotherapy and then re-injecting the patient with their removed cells to regenerate a healthy blood system. By simply increasing both the total amount of cells and the relative amount of a specific cell type (natural killer cells) that are harvested and re-injected, the success rate of the procedure is increased. For almost no cost or additional risk, the rate of durable remissions seen in patients can increase from 50% to 70%. These protocol improvements can save lives and reduce healthcare costs for patients who suffer from a variety of blood cancers. Almost 600,000 ASCTs are performed around the world each year. This success could save up to 180,000 lives per year!

CWR funding role: Participating funder

Publication:  Biology of Blood and Marrow Transplantation

Since publication:

→ Data are supporting a change in clinical practice

→ Data are supporting trials in other diseases using the same repurposing treatment

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BerishSylviaPrinciple Investigators: Dr. Berish Rubin and Dr. Sylvia Anderson

Disease: Familial Dysautonomia

Research Description: Familial Dysautonomia (FD) is a genetic disorder that affects the development and survival of the nervous system in infants and children. Individuals with FD are affected with a variety of symptoms that include decreased sensitivity to pain and temperature, heart problems, recurrent pneumonias, vomiting crises, blood pressure swings and gastrointestinal dysfunction. Untreated FD can cause death before the age of three. Through a series of clinical trials, the research team has zeroed in on the functional protein tied to this disease. The research team then mapped out biomedical and dietary changes that eliminate the extreme symptoms of this disease. Implementing dietary protein regulation coupled with prescribing very powerful nutriceuticals from soy, Vitamin A and ECGC (green tea ingredient), these children are able to lead almost normal lives, with functioning nervous systems. In addition, if women who are rearing a child with this genetic mutation take theses nutriceuticals during pregnancy, the child will be born with a normal functioning autonomic nervous system. Funding for these discoveries was a cooperative effort among several organizations, including Cures Within Reach.

CWR funding role: Participating funder

Publication(s): Molecular Nutrition & Food Research

Since publication:

→ Data are supporting off-label clinical use

 

 

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Scott-EggenerAytekin Oto

Principal Investigators: Dr. Scott Eggener and Dr. Aytekin Oto

Disease: Prostate cancer

Research Description: Prostate cancer is both the second most common cancer and the second leading cause of cancer deaths in men. Treatment at earlier stages can increase survival rates. Drs. Eggener and Oto repurposed a laser device, originally approved to treat brain and breast cancers, to treat early-stage prostate cancers. In a pilot clinical trial with nine patients, the device was used to heat and kill the cancer cells, resulting in a successful and minimally-invasive treatment. Patients were able to resume their normal activities the next day. By using MRI technology to monitor the laser, the research team was able to eliminate or minimize damage to the surrounding normal tissue. This repurposed laser produced fewer side effects than other treatment options, and treatment was completed the same day, resulting in improved patient outcomes.

CWR funding role: Primary funder

Publication(s): Radiology 

Since publication:

→ Data are supporting off-label clinical use

→ Data leveraged into larger philanthropic or government funding for this treatment

→ University of Chicago and several other institutions are now offering this treatment to patients with prostate cancer

 

 

 

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Olga GuptaPrinciple Investigator: Dr. Olga Gupta

Disease: Type I/II Diabetes

Research Description: Type I and Type II diabetes are chronic diseases which require vigilant monitoring of blood glucose levels and insulin management. Developing self-care skills in youth skills is critical for effective life-long treatment. A number of other studies have found that social factors can help in diabetes control. The researchers investigated whether the responsibilities involved in pet care would also have beneficial results for diabetic management. Dr. Gupta randomly selected adolescent diabetic patients to give a gift card for a pet fish, along with equipment and instructions for both fish- and self-care. The researchers found that patients with a pet fish had fewer emergency room visits and better blood glucose levels than patients who did not receive a pet fish. This applies to both type I and type II diabetics. Pet care combined with self-care is an inexpensive and feasible strategy to improve patient outcomes among children with diabetes.

CWR funding role: Primary funder

Publication(s): The Diabetes Educator

Since publication

→ Data are supporting off-label clinical use

 

 

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June Robinson Photo

Principal Investigator(s): Dr. June Robinson

Institution(s): Northwestern University

Disease: Melanoma

Research Description: Melanoma is the deadliest type of skin cancer, with diagnosis rates rising dramatically over the past 30 years. Melanoma survival is improved with early detection and surgical treatment. An estimated 16% of all melanomas are discovered by physicians, and approximately half of all US doctor visits are with a primary care physician. These primary care visits are a prime opportunity to detect melanomas. However, many physicians do not conduct melanoma screenings due to lack of training and low confidence in their examination skills, resulting in missed opportunities for early detection. In order to combat these barriers, Dr. Robinson and her team created a new melanoma screening training simulation. This simulation repurposed existing teaching models and combined them with smartphone technology. After completing the simulation, participants were better able to identify and classify melanoma lesions and were more confident in their abilities. Results from this study were used to obtain a larger grant for a widely available, web-based screening program, currently in development.

CWR funding role: Participating funder

Publication(s): JAMA Dermatology

Since publication: 

→ Data leveraged into larger philanthropic or government funding

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