$1 million awarded to biotech breakthroughs
Travelling to the United States for health care can be relatively expensive, but the key reason patients go to the United States for treatment is in large part because of the exceptionally advanced level of care that is available – innovative, evidence-based treatments that in many cases are not available in other parts of the world.
One of the main reasons why leading hospitals in the US are in a position to offer such advanced levels of care is because there is significant and broad spend on medical research in the US across the wide spectrum of healthcare. In addition, there is solid support for start-ups in the industry – to help them bring new tech to the market, for example. And there are many examples of this being announced across the country on a seemingly weekly basis.
The following story of the recent announcement of financial awards for ten biotech projects in the Boston area is a good example of what can be found happening in cities across the country on a regular basis and continues to be one of the main driving forces that ensure the United States remains a leading destination for the most cutting-edge diagnostics and treatments.
Innovation Discovery Grants
This year the Bank of America joined with Mass General Brigham as presenting sponsor at the World Medical Innovation Forum in Boston. The organisers say the Forum – established in 2015 – was set up “in response to the intensifying transformation of health care and its impact on innovation”. The Forum, they say, is rooted in “the belief that no matter the magnitude of change, the centre of health care needs to be a shared, fundamental commitment to collaborative innovation with industry and academia working together to improve patient lives.”
At the Forum the annual Innovation
Discovery Grants (IDG) were awarded.
Ten biotech advancements from Mass
General Brigham were awarded this highly competitive grant. Each of the potential patient care and healthcare delivery break-throughs received $100,000 toward ongoing development and future commercialization,
based on the potential to improve health outcomes, meet articulated milestones, and attract follow-on investment as assessed by independent industry experts.
Ravi Thadhani, MD, Chief Academic Officer, Mass General Brigham, explained: “The Innovation Discovery Grants support translation of biotech research from the lab to real-world products whereby they can help more patients.”
Since IDG’s inception, 65 research projects have been awarded a combined $4.2 million, and together have raised more than $199 million to further their development. This includes 14 new companies actively developing IDG-supported technologies and 19 license agreements.
Chris Coburn, Chief Innovation Officer, Mass General Brigham, noted: “Advancements in biotech are redefining the future of medicine. These grants recognize the tremendous commitment to spark innovation throughout Mass General Brigham and translate invention to the front lines of care.”
The Mass General Brigham Harvard faculty receiving this year’s IDG awards are:
- A Novel Convection Enhanced Delivery System for Brain Malignancies — Miles Cunningham, MD, PhD, McLean Hospital. Convection Enhanced Delivery (CED) is a method in which a large volume of therapeutic agent under hydrostatic pressure is infused directly within diseased brain tissue. CED is being investigated primarily for glioblastoma multiforme (GBM), an aggressive brain cancer that leads to approximately 200,000 deaths worldwide each year. Typically, patients live only a few months following diagnosis. There have been no significant improvements in treatment for GBM in 40 years. The proposed CED System addresses conventional CED shortcomings by replacing a large, cumbersome delivery tube with multiple precision micro-cannulas. These small tubes are positioned strategically in 3-D arrays using sophisticated surgical planning software and rapid prototyping to fabricate a patient-specific system within 1-2 days of diagnosis. GBM represents but one indication for this system, other indications include inoperable epilepsy and traumatic brain injury.
- FastLine: Single-handed Venous Access Device – Hilary Gallin, MD, Massachusetts General Hospital. FastLine is a device that enables placement of a central line with one hand using intuitive, ergonomic movements to enable real-time troubleshooting and allow the use of ultra-sound in the other hand throughout the entire procedure. The device eliminates poorly controlled movements that occur with the traditional placement technique and is designed to maximize control and dexterity. It also aggregates almost every required component of the procedure into one device with a safety needle, increasing central line kit organization and decreasing risk of contamination or needlesticks. FastLine will reduce complications, decrease procedure time, improve the patient experience, and serve to reduce the $2 billion spent on managing patient injuries arising from the 5 million central line placements in the U.S. alone each year.
- Development of KRAS Degraders in Cancer – Vid yasagar Koduri, MD, PhD, Brigham and Women’s Hospital. Mutation in the KRAS (Kirsten Rat Sarcoma virus) gene are the main drivers of lung, colon, and pancreatic cancer, which together kill 230,000 annually in the U.S. Treatment regimens are arduous, only partially effective, and cost (in aggregate) hundreds of millions of dollars. The pre-ponderance of data from studies in cell, animal, and patient-derived models of disease indicate that therapeutically targeting KRAS would be an enormous advance in the treatment of these cancers. The proposed research program uses a newly developed positive-selection screening platform as a tool to identify protein degraders of KRAS (rather than inhibitors) as potential therapeutic agents. This approach has been validated as a game-changer in other cancers such as multiple myeloma.
- Restoring Tumor Immunogenicity in Glioblastoma Multiforme (GBM) – Arpita Kulkarni, PhD, Brigham and Women’s Hospital. This program features a novel drug that may change the paradigm for glioblastoma (GBM) therapy. Traditional treatments for GBM (chemotherapy, radiation, and surgical resection) have remained unchanged for decades, and the prognosis for GBM patients is grim. While emerging cancer therapies (e.g., allogenic cell therapies) are being developed, so far, they remain unsuccessful in clinical trials, in addition to being very expensive and highly variable in outcomes between patients. This program has engineered and patented a new therapeutic that can cross the blood-brain barrier, induce tumour immunogenicity in models of GBM and other solid cancers, as well as synergize with cell therapies. Such a multi-pronged approach has never been tested in the clinic and could markedly improve outcomes in GBM while also improving the activity of cell therapy.
Developing First-in-Class mRNA Methyltransferase Inhibitor — Li Lan, MD, PhD, Massachusetts General Hospital. The goal of this study is to develop IND-enabled compounds targeting mRNA methyltransferase and validate their effectiveness in cancer therapy. The ultimate goal is to target a newly identified mRNA-dependent repair pathway and mRNA methylating enzymes in DNA repair in cancer therapy. This could lead to effective therapeutic strategies for around 90 percent of breast and ovarian cancer patients without HRD, as well as effective therapeutic strategies for other cancers. These first-in-class mRNA methyltransferase inhibitors for cancer treatment have a high potential for the cancer drug market and the treatment of a large population of cancer patients.
- Auditory Mirror Therapy for Tinnitus – Clas Linnman, PhD, Spaulding Rehabilitation Network. Current healthcare cost for tinnitus (ringing in the ears) in the U.S. is estimated at $17 billion. About one in ten adults suffer from tinnitus, yet there are no FDA approved drugs or devices. The proposed technology provides a new type of treatment for tinnitus that is non-invasive, affordable, and low risk. This neuro-modulatory therapy is based on disruption of multi-sensory integration. Like mirror box therapy for phantom pain, in this therapy, sound at the left ear is transmitted to the right ear canal, and sound at the right ear is transmitted to the left ear canal. Implemented in a wearable pair of headphones, a pilot trial of “auditory mirror therapy” (AMT) indicates that brief use of the headphones significantly and substantially reduced tinnitus. The effects of AMT will be verified in a larger, placebo-controlled study of persons with tinnitus. The technology can be implemented as a separate device, added to current hearing aids, or as a software ap- plication for microphone-equipped wireless earbuds, with the potential to help many with tinnitus.
- Noninvasive Diagnosis of Middle-Ear Pathologies – Hamid Motallebzadeh, PhD, Massachusetts Eye and Ear. According to National Institute on Deafness and Other Communication Disorders, approximately 15% of American adults (37.5 million) aged 18 and over report some trouble hearing, among which, over 50% of the cases are related to conductive hearing loss. Wideband tympanometry is a promising cost-effective tool for noninvasively probing the status of the ear. However, interpreting its complex outcome to provide reliable indicators of middle-ear pathologies has proven challenging. This project aims to automate objective differential diagnoses of middle-ear pathologies by taking advantage of powerful pattern-recognition capabilities of machine learning to infer details about the middle-ear status from clinical data. This tool could reduce the need for exploratory surgery, improve the specificity of preoperative preparations, and provide a low-cost tool for postoperative monitoring. This tool helps spot features that the human eye has difficulty identifying, which can help avoid subjective interpretations in differential diagnosis of the middle ear and conductive pathologies.
- Small-Molecule MMP Inhibitors for Allergic Inflammation – Jin Mo Park, PhD, Massachusetts General Hospital. Current allergy medications, ranging from corticosteroids to cytokine receptor-blocking antibodies to JAK inhibitors, are effective in only subsets of patients, produce partial responses, and often encounter treatment resistance. More importantly, they directly interfere with the workings of the immune system and can cause harmful side effects. The inadequacy of current medications to address these problems and the deficiency of new solutions prevent the pharmaceutical and healthcare industry from creating effective allergy treatment options for large patient populations. This project aims to develop small-molecule inhibitors of a specific protein-degrading enzyme as topical therapeutics for atopic dermatitis (AD), asthma, and other allergic inflammatory diseases. Preliminary data show that this enzyme plays a key role in driving allergic inflammation.
- Neutralizing Antibody to FSTL3 as an Immunotherapy in Ovarian Cancer – David Pepin, PhD, Massachusetts General Hospital. High-grade serous ovarian cancer is the most lethal gynaecologic malignancy, accounting for 70% of ovarian cancer-related deaths, and affecting over 14,000 women in 2020. The treatment options for these patients are limited, particularly for patients that do not respond to chemotherapy or PARP inhibitors, and the survival rates have remained low. The advent of immunotherapies, such as immune checkpoint blockade (ICB), has brought a paradigm shift in the treatment of many cancers, sometimes providing curative responses. Unfortunately, immune checkpoint blockade therapies have not been successful in ovarian cancer, which is generally resistant to these agents. Our technology consists of a neutralizing antibody to FSTL3, which we hypothesize drives this resistance, that would be used in combination with immune checkpoint blockade therapy and alongside the current standard of care for ovarian cancer. We expect that this technology will significantly increase the response rate of ovarian cancer to immune checkpoint therapies and lead to significantly better outcomes.
- Circulating Microparticle Proteins Predict Pregnancies Complicated by Placenta Accreta Spectrum – Hope Yu, MD, Brigham and Women’s Hospital. Placenta accreta spectrum, or PAS, is a potentially life-threatening pregnancy complication that occurs in approximately 1 in every 1000 – 2000 pregnancies. There is no bio-marker test for PAS currently, and diagnostics rely on imaging studies and assessment of clinical risk factors. Despite these efforts, approximately 50% of PAS cases remain unidentified prior to delivery. The proposed product is a blood test to assess a woman’s risk of PAS using circulating microparticle (CMP) proteins. The research team has previously identified a CMP protein panel that distinguishes pregnancies affected by PAS from unaffected pregnancies. This test would be used at the end of the second trimester with a woman’s routine outpatient blood work. Implementation of a blood test using this CMP protein panel has potential to improve the ante-natal detection of PAS and is critical in global efforts to reduce maternal morbidity and mortality.
