failed before coming to me ,” Dr . Saleh says . “ It ’ s extremely difficult for these patients to meet the eligibility criteria . We need to think about phase I studies differently than we have thought about them before . It used to be an end-of-the-line option , and it ’ s no longer that . In many cases , it may very well be closer to the front of the line .”

Dr . Saleh ’ s message for patients and referring physicians is that phase I studies should be considered sooner rather than later . “ We shouldn ’ t wait until the end of the line to refer a patient to a phase I study ,” he says . “ We are here to say if you don ’ t have an FDA-approved option , consider a phase I trial .”

“ What we can best do for the patient should be our goal . We have a highly trained and compassionate team , all dedicated to this concept of giving the patient the drug called hope .”

Mansoor Saleh , M . D . UAB Comprehensive Cancer Center senior scientist and director of the Phase I Program

The Role of the Cancer Center

The Cancer Center has long been a tremendous supporter of the Phase I Unit and was instrumental in its creation . The program currently has 16 open trials , nearly all of which are oncology-related and more than double from when the program started . Some are studying hematologic malignancies , while others involve solid tumors and the rest are considered “ catch-all .” “ We are ambitious in our goal , but pragmatic and focused in our approach . With our disease-specific as well as catch-all , first-in-human trials , we have something for every patient ,” Dr . Saleh says .

“ While the objective of a phase I study is safety , tolerability and pharmacokinetic profile of novel drugs never administered to patients before , we are seeing more and more combination phase I studies , where an FDA-approved agent is delivered with the study agent . In many ways , such phase I trials also offer the real potential for some biologic and clinical activity ,” Dr . Saleh says . “ But , it is important to know that we are still in the experimental phase and do not have data to make any claim of benefit .”

The Phase I Unit has a number of trials that are target- or pathway-specific . For example , if researchers are studying a drug ’ s effect on a certain gene mutation or cell-survival pathway , only patients with that “ actionable signal ” would be eligible for the trial . That target , however , could appear in a number of cancers . Thus , a number of the phase I studies are geared toward a broad range of cancers .

The Phase I Program also has a number of trials involving the human body ’ s natural disease fighter – the T-cell . “ There is an entire army of drugs that stimulate the immune system and its T cells ,” Dr . Saleh says . “ However , we have not been able to activate the T-cell and help it fight cancer cells effectively , because cancer cells produce proteins and chemicals that inhibit the actions of the T-cell . We now have drugs that activate those T-cells and overcome the inhibitory impact of the tumor cell .” An example of these types of agents are the “ check-point ” inhibitors , including , for example , the drug given to former President Jimmy Carter to treat his melanoma . “ In our Phase I Program , we have a number of FDA-approved check-point inhibitors being administered with novel new drugs aimed at activating the cancer-fighting potential of the body ’ s own immune mechanism ,” Dr . Saleh says .

The challenge with any clinical trial , including phase I studies , is that patients must meet an often extensive list of eligibility criteria . Overall health – termed Performance Status – is an important factor , as are baseline laboratory studies , the stage of the cancer and having measurable target lesions , just to name a few . This goes a long way in explaining why not all patients can qualify for a clinical trial , because the only way to study new agents is to have a strictly defined patient population and to strictly adhere to the protocol .

“ When I see patients in my clinic , many have had five or more prior treatment regimens that have